The federal election is scheduled for April 28, 2025, and it’s a key opportunity to make sure the voices of Canadians affected by ALS are heard.
ALS is a devastating disease that can affect anyone at any time. It can move with startling swiftness, and with no cure, the ALS community has no choice but to measure time not by months or years but by loss–loss of function and loss of life. As more families are impacted by this disease, the need for federal leadership and investments in research is becoming increasingly urgent.
Raising our voices and engaging with candidates during the election is a critical way to keep ALS on the radar. To support community advocacy during the election, ALS Canada has created the Candidate Handout – ALS in Canada: What Candidates Need to Know, a practical resource that helps inform candidates about the realities of ALS and how they can support the Canadian ALS community.
Download the Candidate Handout and start engaging with your candidates!
Download the candidate handout
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How you can use this resource
Start the conversation about the realities of ALS with your local candidates.
Share it with candidates at your door or events.
Email it directly to candidates.
Post on your social media, tagging your candidates, and encourage others to do the same.
If you’re interested in learning more about the political parties, visit the Elections Canada website, and you can find a complete list of registered political parties and information on where and when to vote in your riding.
Advocacy Success
On October 30, 2024, the Government of Ontario announced an investment of $13 million over three years to implement the recommendations of the Ontario Provincial ALS Program. Your contributions have brought us to this monumental achievement!
In Canada, what the provincial health care systems provide does not always match the needs of people living with ALS. This statement rings true for Ontario, where the current ALS care and support landscape in the province presents people living with ALS with significant challenges that demand immediate action.
The impact of an ALS diagnosis on the person living with the disease and their family, caregivers, and community is tremendous and pervasive – physically, psychologically, and financially.
In Ontario, more than people living with ALS not only face the harsh realities of this disease daily but also a health care system that fails to meet their complex and progressive needs, leaving them without adequate care and support.
Our Solution
To address this urgent issue, the ALS Society of Canada (ALS Canada), in collaboration with the five regional multi-disciplinary ALS Clinics in Ontario (Hamilton, Kingston, London, Ottawa, Toronto), developed the Ontario Provincial ALS Program.
The Ontario Provincial ALS Program is a comprehensive solution that addresses the complex, critical needs of the ALS community. The program includes four key recommendations:
Due to the complex nature of ALS, people living with the disease have substantial care and equipment needs that evolve and increase over time. Effective management of ALS requires access to multi-disciplinary ALS clinics where health care providers, such as speech language pathologists, dietitians, social workers, respiratory therapists, and occupational therapists, play a key role in optimizing care. However, Ontario’s five regional ALS clinics are beyond capacity, under-resourced and unable to meet the unique levels of care identified in the Canadian Best Practice Recommendations for the Management of ALS.
Solution:
The Ontario government to provide incremental investments to ALS clinics to standardize and enhance existing care models, optimizing ALS care and ensuring each person living with ALS in the province receives the highest quality care.
ALS is an incredibly isolating disease due to the lack of awareness and the increasingly difficult physical realities. To support a person living with ALS, their caregiver, and the people closest to them, they are connected to ALS Canada Community Leads located throughout Ontario. ALS Canada Community Leads provide direct, in-home support and individualized information and resource navigation to ensure people are well-supported, helping to augment the health care system.
As a person living with ALS progresses in their disease, so does their reliance on mobility and communication equipment and other assistive devices that help them maintain their independence, dignity, and safety. However, Ontario’s medical equipment programs do not meet the needs of people living with ALS as they are left with equipment for short-term use only or with devices that are outdated for their needs or not right for them, which can put both themselves and their caregivers or families at significant risk. In the end, people living with ALS and their caregivers must turn to ALS Canada, a donor-funded organization, to fill the significant gaps that exist in Ontario’s health care system.
Solution:
The Ontario government to allocate funds to ALS Canada’s Community Services and Equipment Programs, ensuring people have access to the right equipment and assistive devices at the right time, improving quality of life and helping people living with ALS maintain independence, dignity, and safety for themselves and their caregivers.
The lack of comprehensive oversight and coordination for ALS care in Ontario hinders the province’s ability to gather critical patient information, which would inform evidence-based decisions to shape a health care system that meets the needs of the ALS community. Comprehensive data capture, knowledge dissemination, and system planning are essential for a coordinated approach to ALS care.
Solution:
Formation of a secretariat to oversee and coordinate ALS care and facilitate comprehensive data capture, efficient knowledge dissemination, and strategic system planning.
Many Ontarians living in northern and rural regions face unique challenges in health care access due to vast distances and limited health care infrastructure. This is the case for people living with ALS in northern and rural Ontario, as they must endure long journeys to attend appointments at one of the five ALS clinics located in Toronto, London, Hamilton, Kingston, and Ottawa, leading to disparities and barriers in access to care.
Solution:
The development of a regional strategy to provide equitable and accessible ALS care in northern and rural Ontario ensures that people living with ALS receive timely care regardless of their geographic location.
Our Advocacy
In partnership, ALS Canada, with Sunnybrook Health Sciences Centre, the five regional ALS Clinics in Ontario (Hamilton, Kingston, London, Ottawa, and Toronto), and the ALS community, we have advocated for this program since 2023.
Our collective efforts have highlighted the devastating impact that ALS can have on both individuals and families. We have demonstrated the urgent need for access to standardized and equitable care, community services, and equipment. And now, we’ve been heard.
We are incredibly grateful for the dedication and passion of our community advocates. Their efforts have been instrumental in achieving this significant milestone.
We would like to acknowledge the following ALS community advocates for their vital contributions:
Denis Blais, passed away in November 2024 Carmen Cels, caregiver to her husband, Mike Mike Cels, diagnosed with ALS in 2017 Patsy Ellis-Ma, bereaved caregiver to her husband, David Steven Gallagher, diagnosed with ALS in 2019 Wade Hall, diagnosed with ALS in 2022 Simon Kuzyl, Patient Care Manager, Sunnybrook ALS Clinic Steve Parker, bereaved caregiver to his brother, Peter Paula Rodriguez, bereaved caregiver to her sister, Carla Dr. Lorne Zinman, Director of the Sunnybrook ALS Clinic
Since June 2023, ALS Canada, along with ALS community members, met with Ontario MPPs to discuss the implementation of the Ontario Provincial ALS Program, including the Minister of Health, Sylvia Jones, and the office of the Premier, Doug Ford.
Since we launched our advocacy campaign in January 2024, the ALS community has come together to raise their voices in an inspiring show of solidarity. More than 150 ALS community members have utilized the toolkit developed by ALS Canada to contact their elected officials, the Minister of Health and the Premier of Ontario to advocate for the urgent need for the Provincial Program.
ALS Canada submitted a written submission as part of Ontario’s 2024 Budget Consultations, urging the government to invest $6.6 million to implement the recommendations outlined in the Ontario Provincial ALS Program.
Throughout November and December 2023, ALS Canada participated in six pre-budget public hearings held across Ontario by The Standing Committee on Finance and Economic Affairs (SCFEA) and local MPPs.
On February 21, 2024, members of the ALS community joined ALS Canada at Queen’s Park, urging the Ontario government to include the Ontario Provincial ALS Program as part of Budget 2024. With participation from more than 40 MPPs, including the Minister of Health, Sylvia Jones, ALS Canada’s Queen’s Park Day of Action raised awareness of the current realities of the province’s health care system, which fails to meet the complex and urgent needs of people living with ALS.
On October 30, 2024, Ontario government announced their commitment of $13 million over three years in the 2024 Fall Economic Statement to support the Ontario Provincial ALS Program.
This pivotal investment represents a meaningful commitment from the provincial government to address the urgent and evolving needs of people affected by ALS. The program will deliver vital services aligned with the best practice model of care to provide better health outcomes for the 1,400 Ontarians living with , supporting their independence, safety, and dignity while reducing stress on Ontario’s healthcare system by helping prevent unnecessary hospitalizations and emergency visits.
This pivotal investment represents a meaningful commitment from the provincial government to address the urgent and evolving needs of people affected by ALS. The program will deliver vital services aligned with the best practice model of care to provide better health outcomes for the 1,400 Ontarians living with ALS and their families, supporting their independence, safety, and dignity while reducing stress on Ontario’s healthcare system by helping prevent unnecessary hospitalizations and emergency visits.
As we gain more insights into the implementation and funding allocations of the Program, we will keep you informed with updates on this news page and through our social media channels.
“The government is committing more than $13 million over three years, starting in 2024–25, to establish the Ontario Provincial ALS Program. This program will support patient care at Ontario’s five regional multi-disciplinary ALS clinics, as well as for the ALS Society of Canada’s Community Leads program and Equipment Program. It will also enable the expansion of regional multidisciplinary ALS services into Northern Ontario for improved access to care. The government is also exploring the creation of a comprehensive assistive devices loan and recycling program to allow Ontarians living with physical disabilities to have improved access to a larger range of affordable recycled devices.”
Government of Ontario. 2024 Fall Economic Statement, p. 90. 30 Oct. 2024.
Next Steps & Updates
Looking ahead, our work is not done! With the Ontario Provincial ALS Program now funded, it has the potential to set a national benchmark. We are eager to demonstrate its impact in Ontario and explore how we can bring a unified model of ALS care and support to every province.
This marks an exciting step toward building a national standard of ALS care, and we are hopeful for the future of ALS advocacy and support across the country.
Thank you for being a part of this significant milestone. This is an achievement we can all celebrate, and it’s a testament to what we can accomplish together.
Ontario Election 2025: Take Action
February 27, 2025 is election day in Ontario. This is our opportunity to make sure Ontario’s ALS community’s priorities remain at the forefront of the next government’s agenda.
We’ve made progress – in 2024, the Ontario Provincial ALS Program received a commitment of $13 million to improve access to multi-disciplinary care, support and equipment for people living with ALS. Now, we must ensure the Ontario Provincial ALS Program is sustainable for the future.
Timely and equitable access to ALS therapies remains a priority for the community. Ontario’s provincial reimbursement process needs to reflect the diversity and urgency of ALS. Ensuring Health Canada approved therapies are reimbursed in a timely and equitable manner is critical.
That’s why we created the Candidate Handout – a simple, practical resource to help candidates understand what they can do to support the ALS community.
Download the Candidate Handout and start engaging with your candidates!
Download the candidate handout
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How you can use this resource
Print it and share it with candidates at your door or at local events
Email it to candidates
Share it on your social media and tag your local candidates
Bring it to townhalls or debates and use it to ask informed questions
Share with friends and family to amplify the ALS community’s resource
Amyotrophic lateral sclerosis (ALS) is a complex disease with varying symptoms and severity for people affected. Due to its heterogeneity, diagnosing the disease can be challenging and finding effective treatments can also be difficult.
Through a collective effort, researchers aim to better understand the genetic causes of ALS, identify biomarkers for early detection, and impact how clinicians can treat the disease.
Participating for future generations
Looking back, Kris Noakes can see how her genetic form of ALS has impacted her family through generations. Living with the disease has meant letting go of parts of her life, including her love for running and reimagining her career.
After Kris recovered from the initial shock of her diagnosis, she turned to her First Nations teachings for guidance. Embracing her feeling of responsibility for the community, she is determined to assist in research, advocacy, and support for people like herself living with ALS.
Kris journeys to Sunnybrook Hospital in Toronto four times a year, where the team looks at the progression of the disease, and she undergoes MRI scans, cognitive and speech testing, and has her blood collected.
“People aren’t necessarily thinking of this at the time of diagnosis,” Kris shares. “It’s half a day that can really have an impact on our lives. It’s a small sacrifice to help lead us to finding a cure for this devastating disease.”
Samples and data are de-identified and will be shared globally for researchers to use for scientific advancement. Kris believes the more her samples are used, the better. She likes the idea of her samples travelling around the world and going to the places she might not have the opportunity to see in her lifetime.
“People can study me any way they want. My life depends on it; how can I not participate? My children’s lives, my cousins. The clock is ticking,” she adds.
Importance of personal engagement
CAPTURE ALS prioritizes the voices of people affected by ALS, their families, friends, and communities. It has an active Participant Partner Advisory Council (PPAC) that advises on research priorities, participant recruitment, and engagement guidelines.
“By including the voices of people living with ALS, the research gains an elevated sense of purpose and urgency,” said Shelagh Genuis, a CAPTURE ALS member who works with the PPAC. “Research projects sometimes fall behind schedule, but for someone living with ALS, every day counts. With their presence on our council, researchers are reminded of the time-sensitive nature of their work and are motivated to prioritize the needs of people affected by the disease.”
Participant engagement has been a priority since the beginning of the initiative. Early consultation with focus groups of people living with ALS and family members informed and shaped the funding application, including study design and CAPTURE ALS’ data sharing plan. Today, their participation ensures that the lived experience is embedded in all the research the team engages in.
Healthy Canadians are also needed in research
The ALS Society of Canada (ALS Canada) is proud to support CAPTURE ALS, and our team is actively participating in it. Carolina Jung, research specialist at ALS Canada, recently participated in the study as a healthy control participant.
“Healthy controls are an important part of any research study,” Carolina shared. “For researchers to obtain reliable and impactful results, they need healthy volunteers to participate. It’s a small contribution of your time and effort that can ultimately have a huge impact on people living with ALS.”
Join CAPTURE ALS
The team at CAPTURE ALS is seeking people living with ALS, their families, and control participants who can travel to clinics located near Edmonton, Toronto, Montreal, or Quebec City. To learn more about getting involved, visit captureals.ca.
Sodium phenybutyrate is a histone deacetylase inhibitor that is believed to reduce a mechanism known as endoplasmic reticulum stress (ER stress) that may contribute to motor neuron degeneration in ALS. Ursodoxicoltaurine, also known as TUDCA or TURSO aims to relieve stress on the mitochondria, which produce energy for cells to live. The combination of ER and mitochondrial stress reduction is believed to explain the beneficial effects of ALBRIOZA.
It is available as individual sachets, each containing 10 g of powder (3 g sodium phenylbutyrate and 1 g ursodoxicoltaurine) to be reconstituted in 250 mL of room temperature water and taken orally or administered via feeding tube within one hour of preparation.
The recommended dose in the product monograph is one sachet daily for the first three weeks and one sachet twice daily thereafter.
The drug is now commercially available in Canada. As of June 15, 2023, Amylyx and the pCPA have successfully concluded negotiations for ALBRIOZA and entered into a Letter of Intent (LOI) for the terms and conditions under which ALBRIOZA would qualify for reimbursement through federal, provincial, and territorial public drug plans in Canada.
As of September 1, 2023, following provinces have made decisions regarding the public reimbursement of ALBRIOZA:
Ontario: With criteria under the Ontario Drug Benefit Formulary (Exceptional Access Program)
Québec: With criteria under the Régie de l’assurance maladie du Québec (RAMQ) formulary (special authorization)
British Columbia: Under BC PharmaCare Formulary (special authorization)
New Brunswick: Under the New Brunswick Drug Plan (NBDP) formulary
Alberta: Under the Alberta Drug Benefit List
Nova Scotia: Under the Nova Scotia Pharmacare Program
Newfoundland and Labrador: Under the Newfoundland and Labrador Prescription Drug Program (NLPDP)
Manitoba: Under the Manitoba Drug Benefits and Interchangeability Formulary (special authorization)
Saskatchewan: Under the Saskatchewan Drug Plan Formulary
The remaining provinces have not yet made a reimbursement decision.
Health Canada conducts a review of the drug to assess its safety, efficacy, and quality. The process begins when the drug manufacturer files a New Drug Submission (NDS) with Health Canada. Health Canada must accept the New Drug Submission before regulatory review begins.
There are three timelines under which Health Canada conducts drug reviews:
Standard Review, which is almost a one-year process (300 days)
Notice of Compliance with Conditions, which is an approximately 200-day process.
Priority Review, which is a six-month process (180 days)
If Health Canada concludes the benefits of the drug outweigh the risks and that the risks can be mitigated, they will issue a Drug Identification Number (DIN) and a Notice of Compliance (NOC). Health Canada may also issue a Notice of Compliance with conditions (NOC/c). This is authorization to market a drug with the condition that the sponsor undertake additional studies to verify the clinical benefit. An NOC or NOC/c means the drug is approved to market in Canada and physicians can begin prescribing it. At this point, however, the drug is only accessible by paying for it out-of-pocket or through private insurance.
If the drug does not receive a Notice of Compliance, the drug’s manufacturer has the option of providing additional information, re-submitting the application at a later date with additional supporting data, or asking Health Canada to reconsider its decision.
We believe any new innovative ALS therapy should be granted priority review status. Due to the swift progression and terminal nature of the disease, people living with ALS cannot afford to wait almost a year for Health Canada to review a therapy. During this time, people will experience further loss of function, and ultimately, approximately 1,000 Canadians will die of ALS. Visit this blog post on How new drugs become approved and accessible to Canadians for more information.
ALBRIOZA (AMX0035) is now commercially available in Canada. Once the drug is commercially available, a Canadian ALS clinician can prescribe the drug for treatment. Please speak with your ALS clinician to determine eligibility for ALBRIOZA. If eligible, your physician can then enroll you into the Amylyx Care Team (ACT) Patient Support Program to determine next steps.
If you are from outside of Canada, please speak with your ALS clinician for more information. You can also contact Amylyx medical information or the Amylyx Care Team (ACT) Patient Support Program:
Amylyx Medical Information: Medinfo@amylyx.com
(877) 374-1208 (For the US and Canada Only)
Not yet in all provinces. As of June 15, 2023, Amylyx and the pCPA have successfully concluded negotiations for ALBRIOZA and entered into a Letter of Intent (LOI) for the terms and conditions under which ALBRIOZA would qualify for reimbursement through federal, provincial, and territorial public drug plans in Canada. It is now up to individual provinces and territories to make a reimbursement decision for the therapy. If negotiations are successful, provincial and territorial drug plans will provide reimbursement for ALBRIOZA.
As of July 25, 2023,the following provinces have made decisions regarding the public reimbursement of ALBRIOZA:
Ontario: Yes, with criteria under the Ontario Drug Benefit Formulary (Exceptional Access Program)
Québec: Yes, under the Régie de l’assurance maladie du Québec (RAMQ) formulary (special authorization)
British Columbia: Under BC PharmaCare Formulary (special authorization)
New Brunswick: Under the New Brunswick Drug Plan (NBDP) formulary
ALS Canada will continue to be involved in the CADTH Health Technology Assessment process and is actively working to support the pan-Canadian Pharmaceutical Alliance (pCPA) and the provincial drug programs to make informed and expedited reimbursement decisions by bringing forward the perspective and experiences of people affected by ALS. We are hopeful that pan-Canadian and provincial decision-makers in the reimbursement process will work quickly to provide expedited and equitable access for all Canadians who may benefit. There is simply no time to wait for people affected by ALS.
ALBRIOZA (AMX0035) is now commercially available in Canada. Once a drug is commercially available, a Canadian ALS clinician can prescribe the drug for treatment. Please speak with your ALS clinician to determine eligibility for ALBRIOZA. If eligible, your physician can then enroll you into the Amylyx Care Team (ACT) Patient Support Program to determine next steps.
If you are from outside of Canada, please speak with your ALS clinician for more information. You can also contact Amylyx medical information or the Amylyx Care Team (ACT) Patient Support Program:
Amylyx Medical Information: Medinfo@amylyx.com
(877) 374-1208 (For the US and Canada Only)
Please speak with your ALS clinician to determine eligibility for ALBRIOZA. If eligible, your physician can then enroll you into the Amylyx Care Team (ACT) Patient Support Program to determine next steps.
No. ALS treatments (disease-modifying therapies) are only available through prescription from a Canadian physician.
ALS Canada is aware of the use of online pharmacies, but we do not maintain a list of these pharmacies. There are potential serious risks surrounding the use of online pharmacies. Please visit: Health Canada and US Food and Drug Administration for more information.
Any questions regarding shipment or crossing the border with therapies should be addressed by checking with your country’s customs or food and drug administration authorities for their rules and requirements.
The drug is now commercially available in Canada. Once a drug is commercially available, a Canadian ALS clinician can prescribe the drug for treatment. If you are from outside of Canada, please speak with your ALS clinician for more information. You can also contact Amylyx medical information or the Amylyx Care Team (ACT) Patient Support Program:
Amylyx Medical Information: Medinfo@amylyx.com
(877) 374-1208 (For the US and Canada Only)
There may be an ALS Society or ALS/MND clinic in the country where you live. Please visit the International Alliance of ALS/MND Associations for a listing of organizations around the world.
Many treatments offered in Canada are also offered in other countries. Please speak with your healthcare provider in your local region to learn about treatment options available to you.
In Canada, there are specialized ALS/MND clinics. A very limited number of these clinics may accept international patients on a referral basis only. It is up to the discretion of the Canadian ALS clinician on whether they prescribe a treatment or not. The ALS Society of Canada cannot comment on wait times and does not provide assistance for medical appointments once in Canada. Costs for all medical appointments including tests (blood work, MRI, etc.), transportation, accommodation, medication, translation services etc. are the responsibility of the individual seeking medical services in Canada.
ALBRIOZA (AMX0035) is now commercially available in Canada. In Canada, please speak with your ALS clinician to determine eligibility for ALBRIOZA. If eligible, your physician can then enroll you into the Amylyx Care Team Patient Support Program to determine next steps.
Any questions regarding shipment or crossing the border with therapies should be addressed by checking with your country’s customs or food and drug administration authorities for their rules and requirements. If you are from outside of Canada, please speak with your ALS clinician for more information. You can also contact Amylyx medical information or the Amylyx Care Team (ACT) Patient Support Program:
Amylyx Medical Information: Medinfo@amylyx.com
(877) 374-1208 (For the US and Canada Only)
Each person’s experience with ALS is unique. People living with ALS face different challenges and symptoms of the disease. It is important to be aware of these challenges and to consider both the benefits and risks when deciding on a treatment. This includes travelling abroad. Gathering as much information as possible will enable you to make well-informed decisions for you, and your loved ones.
When making the decision to travel abroad for medical treatments, some key considerations to be aware of both before you go, and when you return include things like, cost, safety, aftercare, rules for travelling with medications, immigration laws and visas.
Please speak with your ALS clinician for more information and consult your government’s healthcare authorities.
Additional Blog Posts
Advocacy
ALS Canada Advocacy Update – April – June 2022
June 17, 2022
Advocacy
ALS Canada Advocacy Update – January-March 2022
April 4, 2022
Advocacy
ALS Canada Advocacy Update – December 2021
December 9, 2021
Top Research Stories of 2021
January 20, 2022
Top Research Stories of 2020
February 9, 2021
AMX0035 approved as a treatment for ALS in Canada with Conditions
June 13, 2022
CADTH launches call for patient input on AMX0035
October 27, 2021
Long-Term Data for CENTAUR clinical trial of AMX0035 supports a further possible benefit for people with ALS
October 16, 2020
Phase 2 CENTAUR clinical trial for AMX0035 shows promising results
Earlier this year, manufacturer Amylyx announced that it plans to pursue Health Canada approval for its AMX0035 therapy. We’ve updated the blog post we first published back in 2018 about how new drugs become accessible in Canada to help orient you to the steps involved, their purpose, the milestones along the way and the timelines involved.
The bottom line is that it simply takes too long for a drug to move from regulatory approval through to public reimbursement (where a provincial drug plan decides to cover some, or all, of the cost of a drug). Ultimately, we want to see a streamlined series of processes that see Health Canada approved ALS therapies made accessible quickly and equitably regardless of where in the country someone lives. Our advocacy efforts will be ramping up to support this need. In the meantime, here is an overview of the current approach.
Health Canada must authorize the drug to be sold in Canada
How it works
Health Canada conducts a review of the drug to assess its safety, efficacy and quality.
The process begins when the drug manufacturer files a New Drug Submission (NDS) with Health Canada. Health Canada must accept the New Drug Submission before regulatory review begins.
If Health Canada concludes the benefits of the drug outweigh the risks and that the risks can be mitigated, they will issue a Drug Identification Number (DIN) and a Notice of Compliance (NOC). This means the drug is approved to market in Canada and physicians can begin prescribing it. At this point, however, the drug is only accessible by paying for it out-of-pocket or through private insurance.
If the drug does not receive a Notice of Compliance, the drug’s manufacturer has the option of providing additional information, re-submitting the application at a later date with additional supporting data, or asking Health Canada to reconsider its decision.
How long it takes
Standard review is one year; Priority Review is 180 days. ALS therapies are typically Priority Review.
Public and private drug plans must determine if they will cover the costs associated with the drug
How it works
The drug manufacturer makes a submission to the Common Drug Review, which is administered by a federal agency called the Canadian Agency for Drugs and Technologies in Health, or CADTH. (In Quebec, this process is managed by the Institut national d’excellence en santé et en services sociaux, or INESSS.)
The Common Drug Review conducts an evaluation of the clinical, economic, patient, and clinician evidence of the drug. It results in recommendations and advice that are used by federal, provincial, and territorial drug plans to make decisions about whether the drug costs will be covered whether through public programs.
Patient input and, since 2020, clinician input, are key parts of the Common Drug Review. Patient groups are invited to share their experiences, perspectives and expectations for the drug under review and clinicians have the opportunity to comment on the benefits of the therapy from a clinical perspective. Together the patient and clinician input help to inform CADTH about the realities of an ALS diagnosis and how therapies that have even modest clinical impact can make a significant difference in quality of life.
ALS Canada is planning a CADTH patient input submission for AMX0035 and will be inviting the Canadian ALS community to contribute to it. If you would like to participate, sign up for our e-newsletter to be notified.
At the conclusion of the Common Drug Review, one of the following recommendations is made:
That the drug be reimbursed;
That the drug be reimbursed with clinical criteria and/or conditions; or
That the drug not be reimbursed.
The final recommendation is shared with participating drug plans and the manufacturer and is posted on cadth.ca.
Following the completion of the Common Drug Review, the federal, provincial and territorial drug plans can choose to work together through the pan-Canadian Pharmaceutical Alliance (pCPA) to determine what public reimbursement could look like within each jurisdiction based on negotiation with the manufacturer.
Each jurisdiction then typically does its own independent review to determine if and how the drug will be covered by its particular public drug plan. Once the review is complete, the jurisdiction’s health authority (e.g. Ministry of Health) will make the final decision about public drug plan coverage. Federal, provincial and territorial drug plans choosing to cover the therapy in whole or in part will have a Product Listing Agreement with the manufacturer.
Increasingly, private health plans are referencing the CADTH recommendation when making decisions about whether they will cover the drug.
How long it takes
In 2018 Health Canada, CADTH and INESSS announced an aligned review process where the Common Drug Review will accept submissions six months before they expect to receive a Notice of Compliance from Health Canada.
The Common Drug Review has a target timeline of approximately six months. However, there are multiple variables that can affect the process and extend it upwards of one year or more.
Timelines associated with the pan-Canadian Pharmaceutical Alliance and subsequently the Product Listing Agreements with individual federal, provincial and territorial drug plans can easily take up to two years complete, depending on the jurisdiction. Throughout the process there are often ways for patients, caregivers, patient groups and health care professionals to push for an expedited process.
The price of the drug is set
How it works
While Health Canada approval and reimbursement reviews are in process, and throughout the duration of the drug’s patent, the drug is subject to price controls, which fall under the authority of the Patented Medicine Prices Review Board (PMPRB). The PMPRB is a federal agency that sets the maximum price at which companies sell their drug products in the Canadian marketplace (e.g. to hospitals, pharmacies, public drug plans, private drug plans, out of pocket payers).
Drug manufacturers must submit specific pricing information to the PMPRB when the drug is first introduced and then on a semi-annual basis. PMPRB publishes information on its price reviews on its website.
The federal government recently updated the guidelines that set out how patented therapies are priced in Canada. They are set to come into effect on July 1, 2021. ALS Canada is one of many organizations that have voiced concerns about the potential impact of these reforms. We believe that the model used to determine pricing must balance affordability with access.
How I access a therapy if a price has not yet been set?
A final price does not have to be determined by PMPRB before the drug can be purchased by a patient. In general, as long as Health Canada has approved the therapy and supply is available, clinicians are able to prescribe it based on the product monograph. However, depending on the status and outcome of the pCPA negotiations and reimbursement decisions by public and private drug plans, a patient may have to pay out of pocket to ultimately get access.
Case study: Radicava
Here are the timelines associated with the approval and reimbursement of Radicava, the most recent ALS therapy to become available in Canada. ALS Canada made a patient input submission and initiated an advocacy campaign that saw more than 3,600 Canadians email their provincial Health Ministers (or equivalent) to advocate for the province to make a quick decision in favour of covering the drug. As of May 2021, the province of PEI has still not made a reimbursement decision, so if you are a PEI resident please consider contacting your Health Minister.
How support services are changing
In the wake of the COVID-19 pandemic, our number one priority was to support the safety of the ALS community and our staff, and as a result, we quickly pivoted to moving all ALS Canada office staff to working remotely from home and made changes to our service model to include more virtual and remote support services in place of in-person visits.
Now, with more regions throughout Ontario moving into Phases 2 and 3, many of you have been asking how ALS Canada’s services will evolve. For the foreseeable future, here is what our services will look like:
Individual support. ALS Canada will be providing virtual and remote support in place of in-person visits. Regional Managers will continue to communicate with people and families living with ALS through videoconferencing, phone, email and/or text. We have received feedback from the community that this model has been working well.
Support groups. Support groups are now being offered virtually through videoconferencing technology removing geographical barriers, enabling increased participation. These sessions will continue to be offered in this format. For more information about upcoming support groups, please contact your Regional Manager.
Educational webinars. Webinars featuring a range of expert speakers on a broad range of topics will continue to be developed. Initially, the webinars were created with a focus on the impact of COVID-19 and will now transition to key topics of interest to the ALS community. To access previous sessions, please visit ALS Canada’s archived webinar playlist on YouTube.
ALS Canada Equipment Program. We will continue to provide mobility and accessibility equipment working in partnership with vendors from across the province with practices informed by recommendations provided by public health experts. We are currently accepting equipment donations prioritizing items of greatest need to our community.
Support services survey results
Given how much has changed in the past few months, in May we emailed a survey to people and families who access our services to collect feedback about the level of support they are receiving during the COVID-19 pandemic. We are pleased to report that:
These have been challenging times and unchartered waters for all of us. Our commitment to innovation and your openness to changes in how we deliver support have resulted in an understanding that the way we support the ALS community and the way in which you need our support had to evolve. Even as COVID-19 restrictions are lifted, we know that the virus is still a risk and that the ALS community is considered a vulnerable population. The health and safety of our community and employees continues to be of utmost importance to us.
With all of this in mind, along with financial shortfalls that will be faced across the charitable sector, we are embracing the opportunity to do things differently going forward. We know that there will still be times when in-person support will be optimal and we hope to reintroduce it for specific circumstances (e.g. someone recently diagnosed with ALS, complex advocacy or support situations that are challenging to discuss virtually, etc.) in the fall, based on consideration of public health officials’ recommendations.
We appreciate your understanding and flexibility as our service model evolves to meet the needs of the ALS community while those needs and the external environment continue to change.
With the 2019 federal election campaign underway, are you curious about what the parties are saying about the issues that could affect Canada’s ALS community?
ALS Canada asked each of the major parties what they would do, if elected, to help people and families affected by ALS.
Specifically, we asked them about how they would improve equitable, timely and affordable access to proven ALS therapies and patient engagement, provide dedicated ALS research funding, and implement a palliative care strategy.
As you go to the polls on October 21, we hope this blog post helps inform you about the issues affecting people and families living with ALS. Read each of the party’s responses below to learn more.
ALS Canada would like to thank the parties for their participation and responses.
*NOTE: All responses are directly from the parties. ALS Canada did not edit or change the responses in any way.
Question #1
The existing pathway for drug access is too long for a person living with a terminal illness. As recently experienced with one new ALS therapy, it has been a year since Health Canada priority-review approval and there is still no timeline in sight for public reimbursement and pricing decisions. With more than 60 prospective ALS therapies in development right now, what will your party do to improve equitable, timely and affordable access to proven ALS therapies and how will you ensure that the perspectives of people with lived experience are meaningfully considered throughout the process?
Conservative Party of Canada
No response was received.
Green Party of Canada
The Green Party will expand the single-payer Medicare model to include Pharmacare for all Canadians. We will also create a bulk drug purchasing agency, and reduce drug patent protection periods.
Liberal Party of Canada
Liberals believe that all Canadians – but especially those living with ALS – deserve consistent, timely access to the treatments they rely on. That’s why our government has taken several steps over the past four years to streamline this process. We are reviewing drugs and medical devices more quickly and finding ways to support innovative treatments coming to Canada.
Health Canada has accelerated the review of many new medicines, and we continue to expedite reviews so that Canadians get quicker access. We’ve also taken steps to reduce the time between our approval and the recommendation for reimbursement, which means that new drugs will be covered by drug plans sooner. Finally, we are modernizing the Special Access Programme to better meet the needs of patients and physicians. Most recently, Health Canada approved Edaravone (Radicava) in Canada for the treatment of patients with ALS in October 2018.
As well, we are in the process of establishing the new Canada Drug Agency which will help to further streamline the process through which new treatments are approved in Canada. A re-elected Liberal government will also move forward, working with provinces, territories and stakeholders, with the creation of a national formulary and the implementation of a $500 million per year rare disease strategy so that Canadians can have better access to high-cost treatments.
New Democratic Party of Canada
Decades of Conservative and Liberal cuts and inaction have left too many Canadians digging deep into their pockets to pay for prescription medication. Many people cannot take the medication they are prescribed because it’s too expensive or because the medication is not readily available.
Instead of making things better, Andrew Scheer and the Conservatives have closed the door on implementing universal pharmacare and negotiating fair prices and timely
access to new therapies with drug companies.
The Trudeau Liberals claim that pharmacare is on its way but they have made no realistic investments that would help implement pharmacare soon rather than decades
from now.
This hurts Canadians, especially those who need expensive, life-saving medication. It’s time for Canadians to have a federal government that’s in it for them.
Jagmeet Singh and the NDP have a bold vision to provide Canadians with head-to-toe health coverage. Affordable health care is our top priority – this is why we’re making a historic investment in universal pharmacare.
Our pharmacare plan ensures that all Canadians will have access to prescribed medication, at no cost.
An NDP government will invest $10 billion in our pharmacare plan. Investing in people and expanding access to medication will improve Canada’s position as a priority country for new drug launches and health research.
We will work with drug companies, experts, provinces and territories, and patients with lived experiences to develop a national formulary and ensure that patients and their
families have timely access to new drug therapies covered by our plan.
Question #2
There has been more progress in ALS research in the past five years than in the previous 100 – momentum that needs to be built upon in order to develop effective treatments. Right now, the ALS Canada Research Program is the only program in Canada that provides dedicated funding for ALS research, and it is funded entirely by donors. We believe government has a role to play in providing dedicated ALS research funding. What will your party do to invest in dedicated funding for ALS research?
Conservative Party of Canada
No response was received.
Green Party of Canada
The federal government can and should lead the way in demonstrating a better model of health care. It is important that health care challenges are addressed at a federal level and that vulnerable populations receive equal access to care.
Scientific research is the foundation of innovation. The Green Party will invest in scientific research and implement the full funding recommendations from Canada’s Fundamental Science Review. We are happy to work with members of the ALS Society of Canada to determine research priorities at the federal level.
Liberal Party of Canada
Liberals know that health research is one of the most important investments we can make as a nation – transforming lives at home and abroad. Unlike the last government – who cut funding and muzzled scientists – we strongly support our health researchers, investing over $1.1 billion annually in the Canadian Institutes of Health Research, with an additional $350 million in 2018. This includes significant investments in ALS research, with close to $20 million in ALS research over the past five years.
This supports research from institutions across Canada through investigator-initiated research programs and is focused on understanding the underlying mechanisms of ALS and trying to find treatments. For example, we’re supporting the work of Dr. Janice Robertson from the University of Toronto, who is investigating the potential cause of ALS. CIHR has a long history of partnering with the ALS Society of Canada to advance ALS research and supporting the Canada Brain Research Fund. Finally, we were thrilled to appoint world leading ALS researcher Dr. Michael Strong as the new President of the Canadian Institutes of Health Research in 2018. Dr. Strong actually developed the international criteria for diagnosing ALS, the aptly named “Strong Criteria.”
A re-elected Liberal government will continue to make major investments in health research – including ALS research – to ensure our researchers keep making life changing breakthroughs that make a meaningful difference in the lives of Canadians with ALS.
New Democratic Party of Canada
In 2017, the NDP supported ALS Society of Canada’s pre-budget request for $25 million over five years in research to help build upon the momentum of the ice-bucket challenge at the time. The Trudeau Liberals rejected the request – the Liberals have made its priorities quite clear.
An NDP government will ensure that Canada is a leader in innovative health research. We will work with universities and health professionals to make sure that public
research on critical health issues continues to flourish. This is especially needed for rare diseases like ALS.
Question #3
Whereas challenges and gaps in Canada’s palliative care system are increasing, how will your party implement the Framework on Palliative Care in Canada and improve the access and quality of palliative care for Canadians, including for people living with ALS.
Conservative Party of Canada
No response was received.
Green Party of Canada
The Green Party is committed to implementing the Framework on Palliative Care in Canada . We support innovative home-sharing plans and other measures to allow people to stay in their own homes as long as possible. We will work to create more long-term care beds in neighbourhood facilities. We will also consult with caregivers and employers to create a work environment where caregivers are not penalised for taking care of a loved one, exploring options to increase flexibility, EI and tax credits, and work sharing.
Liberal Party of Canada
Liberals believe that Canadians approaching the end of their lives should live out their days in comfort and dignity, in the setting of their choice. We know that palliative care is both one of the most essential and most difficult parts of our health care system – it’s literally a matter of life and death.
We were proud to unveil Canada’s first-ever Palliative Care Framework, but we know that it alone is not enough – so we’re putting our money where our mouth is. This starts with the largest investment in Canadian history in home and community care – which includes palliative care – an unprecedented six billion dollars. We’re also supporting targeted initiatives that – bit-by-bit – help more Canadians get access to palliative care. These range from $1.9 million for the Canadian Hospice Palliative Care Association to improve advance care planning to two million for the Canadian Virtual Hospice to expand virtual services and resources to $600,000 for the Canadian Home Care Association to improve delivery of care at home.
We also know that in order to make palliative care more accessible it needs to be well integrated all throughout the health care system. That is why we have provided more than $6 million to Pallium Canada in order to expand palliative care training across the country so that health professionals are fully able to support Canadians and their families in their end of life needs.
These investments will help bring the comfort of palliative care to more Canadians. But we’re just getting started. A re-elected Liberal government will continue to expand access to to palliative care nationwide, supported by an additional six billion dollar towards our health care system.
New Democratic Party of Canada
New Democrats have made health care our priority, which is why we are advocating for head-to-toe health coverage, including palliative care.
We successfully put forward the motion in 2013 that helped establish the Framework on Palliative Care in Canada.
Now, our historical investment in pharmacare means that people can afford expensive medication they need if they choose to receive palliative care outside hospital settings.
This is important because drug therapy helps control symptoms at the end of life.
We will improve access and quality of palliative care as part of our National Seniors Strategy so that we have a stronger, cohesive approach to planning care for our aging
population.
We will ensure that caregivers are supported as part of this strategy. In order to help make life a little more affordable for caregivers, who are overwhelmingly women, we’ll
make the Canada Caregiver Tax Credit refundable. This will provide thousands of dollars to the most low-income caregivers, many of whom have given up work
completely to care for a loved one.
New Democrats believe Canadians and their families have the right to make the choice for the kind of care they would like to receive that is dignified and compassionate.
Antibodies are produced by the immune system to protect the body against foreign invaders like bacteria and viruses. They work by binding to specific proteins on harmful agents and triggering their removal or destruction. Antibodies are also commonly used as a tool in research because they bind to particular proteins. This allows researchers to see where and how much of that protein is in a cell. In ALS research, this means antibodies can be designed to test for ALS specific proteins.
Research funded in part by an ALS Canada–Brain Canada Arthur J. Hudson Translational Team Grant published today in eLife, an open-access scientific journal, shows just how important it is to have effective antibodies in ALS research.
Led by researchers at the Montreal Neurological Institute, the paper highlights the need for better antibody validation to obtain reliable data. The more reliable the data, the better the ability to replicate results and accelerate our understanding of how ALS happens.
A researcher usually makes antibodies or purchases them from a manufacturer for use in laboratory settings. However, commercially available antibodies do not always detect the protein they are supposed to identify. To demonstrate how inconsistent commercially available antibodies can be, the team led by Dr. Peter McPherson and Dr. Carl Laflamme, ALS Canada’s 2016 Ronald Peter Griggs Memorial Postodoral Fellowship recipient, used different engineered antibodies to test for C9orf72. C9orf72 is a protein that results from the gene most commonly mutated in ALS and frontotemporal dementia (FTD).
This validation method clearly showed which antibodies could and could not accurately detect the C9orf72 protein – highlighting that antibody validation is vital to in ALS to ensure reliable products are being used. Validated antibodies mean scientific findings will be more accurate and better able to be replicated in future research. They also save large amounts of time and money.
The paper and validated antibodies are also part of the ALS Reproducible Antibody Platform (ALS-RAP), an open science collaborative effort to ensure the highest quality, validated antibodies are shared with the ALS research community.
ALS-RAP is a partnership between scientists at the Structural Genomics Consortium (SGC) and its associated labs at the Montreal Neurological Institute (MNI) McGill University in Montreal (Canada), the University of Oxford (UK), and the Karolinska Institute (Sweden). It is funded by the MND Association, ALS Association and the ALS Society of Canada.
For more information on the study, you can read the full press release from The Neuro (Montreal Neurological Institute and Hospital).
In late 2018, Facebook began offering the option for Canadians to create fundraisers for nonprofit organizations or for personal causes. Since then many of you have reached out to us with questions about how these giving options can be used to support ALS Canada. We know Facebook can be a quick and easy way to raise money and engage your friends and family, and are grateful to all who have chosen to support us in this way.
There are some important things to be aware of when creating or donating to Facebook fundraisers, so here’s a primer on the options available and how they work – along with using our ALS fundraising website.
1. Facebook: Raising money for a nonprofit organization
Visit facebook.com/fundraisers. You can create a fundraiser for ALS Canada by searching for “ALS Society of Canada” from the list of registered charities. Donations are processed at no cost to the donor or to the individual who set up the fundraiser. For your donation, you will receive two separate emails: a transaction receipt for the payment and your official donation receipt. These are sent by Facebook on behalf of their fundraising partner, the PayPal Giving Fund, which delivers the funds to ALS Canada.
Some things to be aware of when creating or donating to Facebook fundraisers to support ALS Canada:
If you or your donors have any questions about setup, donations, donation receipts or refunds, you must contact theFacebook Fundraiser Support Centre.
When you donate through Facebook, we do not receive your contact details and therefore are not able to communicate with you.
As per Canada Revenue Agency regulations, we are not allowed to issue tax receipts for donations that are made to a third-party charity—in this case, the PayPal Giving Fund.
2. Facebook: Raising money for a personal cause
Personal causes generally refer to fundraisers in response to individual needs – for example a family that requires financial support after losing their home to a fire, or an education fund for a child whose parents have suddenly passed away.
If you create or give to a personal fundraiser on Facebook, a portion of the donation is used to cover processing fees. Funds are deposited to the bank account specified by the person who set up the fundraiser. The person who set up the fundraiser is responsible for making sure the donations are sent to ALS Canada.
Some things to be aware of when creating or donating to personal causes on Facebook:
Tax receipts are not issued when a person donates to a personal fundraiser because the donation doesn’t go directly to a charity but instead goes to the bank account specified by the person who set up the fundraiser.
Only a charity with a registered charitable number from the Canada Revenue Agency can issue tax receipts.
Example of a successful ALS fundraiser
Adam Foley used My ALS Fundraiser to meet (and surpass) his $12,000 fundraising goal for his 900km bike ride in memory of his brother, Matthew.
3. ALS Canada’s “My ALS Fundraiser” platform
Visit My ALS Fundraiser at www.als.ca/myalsfundraiser to create your own fundraising event or find an existing event to give to. When you fundraise through our platform, ALS Canada will receive the contact information for each donor and automatically issue them a tax receipt by email when they donate online. This helps us stay in touch and provide support for your tax receipting requirements. We also have lots of resources available here, including an event organizer toolkit, templates for things like posters or social media, and best of all, your very own ALS Canada event coach to support you every step of the way.
Interested in fundraising with us? Contact our team at teamals@als.ca or head over to the My ALS Fundraiser website to get started.
Thank you to all our donors for their generosity and continued support—in any way you choose to give.
For someone caring for a loved one living with ALS
Looking after a family member or close friend who is living with ALS is a precious act of love and devotion. It is often a full-time commitment that demands a lot of physical and emotional energy, making it challenging to maintain your own health and well-being. If you know someone in this role, the holiday season is a perfect time to say, “here’s something to help take care of you.”
Many of you from our ALS caregiving community recently shared some gift ideas you thought would be most appreciated by others providing care for a loved one. Keep in mind that it may be hard for caregivers to ask for what they need. They often prioritize the needs of their loved one over their own. They can even feel guilty attending to their own needs, putting self-care at the bottom of their list.
Don’t be afraid to reach out and spread your love around! If some of the suggestions below seem like a good idea, chances are, they will be received with gratitude and appreciation.
Here are some gift ideas to offer a caregiver you care about:
Time for yourself
The number one request from caregivers was some time to re-charge. As the disease progresses, someone living with ALS requires an increasing level of support managing even the most basic daily tasks, such as eating, getting dressed, and showering. Giving a caregiver the time to take a break allows them to return to the role feeling re-energized and better able to cope. A day off might provide the respite someone needs, but even giving the gift of a short break can be helpful.
Debbie Henderson appreciated a few hours away from her role as caregiver so that she could go shopping or out for a meal with a friend. “I also used the time off to attend an ALS support group for caregivers. I got so much from the others in the group. It picked me up when I was feeling down.”
Relaxation for your body
Caring for someone with ALS is physically taxing. As a loved one gradually loses the ability to move, the physical demands of the caregiver role increase. Dealing with equipment like wheelchairs and specialized assistive devices for personal care can be hard on the muscles.
Many of you emphasized the importance of physical therapies such as massage, reiki, float pods, spa treatments like manicures and pedicures to help soothe sore muscles and rejuvenate the body. Offering a gift certificate for any of these services can provide temporary relief from pain, stiffness, or fatigue.
Jessica Gustafson also included yoga as a way to de-stress. “I had the gift of a yoga class once a week, coupled with a reliable person to come and stay with my hubby. I think it got me through some of the darkest times and also, it felt so good for my tired and stressed body. Super lovely and thoughtful idea.”
Visits for your loved one
ALS is such a devastating disease. Many people become uncomfortable with the physical effects of the disease when it happens to someone they know, even when it is a close friend or relative. When people turn away, it can cause tremendous pain.
Offering a friendly visit can be a wonderful gift for the caregiver and the person living with ALS. “My dad was such a social and friendly guy,” says Andrea Fairweather. “When he developed ALS, people stopped visiting. It broke my heart that people didn’t know what to say, so they stayed away completely. It would have been so much easier for me with regular visitors.”
If you’re not sure whether a visit would be helpful or not, you can always ask. Caregivers will let you know if they prefer privacy, or if it is too much for the person being cared for to receive a visit on any given day, or at any time during the progression of the disease.
Taking care of chores
Finally, think of a chore you can take on that might relieve some of the work of caregiving. It doesn’t have to be something big. A gift card offering to do the dishes, mow the lawn, clean the house, pick up groceries, walk the dog, or cook a meal is something many caregivers would appreciate.
Use your imagination. The possibilities are endless and the potential for making someone feel appreciated when they are giving so much of themselves is a gift in itself!
Updated October 17, 2018
The first ALS gene was discovered over two decades ago. By the end of 2017, scientists had found more than 25 genes. By studying these genes that are involved in familial ALS, which accounts for 5 to 10 per cent of ALS cases, researchers hope to learn more about the 90 per cent of sporadic ALS cases that are not linked to a family history.
One of the most common ways researchers study the role of genes in ALS is in animal models in the lab. However, within each study, they are investigating one gene mutation at a time. As a result, the animals display similar disease onset and progression, unlike human ALS that affects people in many different ways. “Finding new ALS-related genes is definitely important. However, we also need human-focused research so that we can learn why people with ALS experience such a wide range of onset, symptoms, progression and duration,” says Dr. David Taylor, VP Research at ALS Canada.
Understanding how ALS affects people so differently will guide the development of personalized treatments. It will also help researchers determine which participants should be included in clinical trial groups to ensure the best chance of finding out whether new treatments may work.
Large-scale research involving humans with ALS would not have been possible a decade ago, but it is today, thanks to advances in biobanking, DNA sequencing, stem cell technologies and computer analytics. The ALS Canada 2018 Virtual Research Forum on Wednesday, October 17, 2018, included speakers from three international organizations that are collecting clinical information and biological samples from people with ALS and studying massive amounts of data to uncover new insights.
Answer ALS Research Program
The Answer ALS Research Program is the most comprehensive study of ALS ever conducted. The overall goal of the program is to discover biological signatures of the disease that may help speed up diagnosis and match people with ALS to targeted therapies to counteract disease progression, based on the underlying biology of their ALS.
In 2016, researchers started collecting whole blood, plasma, serum and optionally, cerebrospinal fluid from people living with ALS. Their goal is to collect biological samples from 1,000 volunteers with ALS at eight ALS clinics in the United States.
The Answer ALS Research Program is a collaboration of experts from multiple scientific fields: clinical research, regenerative medicine, cell biology, multi-omics, big data and machine learning. Scientists at the New York Genome Center are extracting DNA from blood samples and performing whole genome sequencing. At Cedars-Sinai Medical Center (CSMC) in Los Angeles, researchers are isolating stem cells from blood samples and reprogramming them to become motor neurons; the cell type affected in ALS. Scientists at other collaborating centers are examining the motor neurons using multi-omics and generating massive amounts of information about how they function. Throughout the collection and analysis of samples, the ALS clinics are tracking disease progression in study participants.
“Incredible advances in biological techniques, including stem cell biology, have made it possible for us to generate motor neurons from each individual patient to begin to understand the true variability of the disease,” says Dr. Emily Baxi, Program Director for Answer ALS. “Multi-omics analyses are generating a huge amount of data. Together with the clinical data, we estimate a total of six billion data points will be generated per study participant. We’re analyzing that data using different methods of machine learning and artificial intelligence to pull out the subtle signatures that may distinguish one form of ALS from another.”
“We are on a mission to generate more knowledge than we’ve ever accumulated about ALS before,” says Dr. Baxi. “Nobody can solve ALS in isolation. We will share our data with the global ALS research community.”
At the ALS Canada Virtual Research Forum, Dr. Jeffrey Rothstein, founder and executive director of the Answer ALS Research Program provided an update about Answer ALS, including how close they are to reaching their goal and what’s next for analyzing the data for results. Dr. Rothstein is also the John W. Griffin Director for the Brain Science Institute (BSI), a professor of neurology and neuroscience and founding director of the Robert Packard Center for ALS Research at Johns Hopkins University’s School of Medicine.
Project MinE
Project MinE is a multinational initiative of collaborating organizations from 19 participating countries. It was founded by two entrepreneurs, Robbert Jan Stuit and Bernard Muller, who are both living with ALS and want to find faster answers about the cause of ALS.
To find out why some people develop ALS and others do not, researchers are analyzing full DNA profiles of 22,500 people to compare results for 15,000 people with ALS to 7,500 people without ALS. By investigating the differences in DNA between these two groups they hope to uncover gene variations associated with ALS. The information will help identify biological clues that may help accelerate the development of new treatments for slowing or halting disease progression and help to find a cure.
As of mid-September, Project MinE has sequenced over 10,480 DNA profiles. ALS Canada is leading Canada’s participation in Project MinE through fundraising to support the mapping and analysis of up to 1,000 DNA profiles. By the end of 2018, Canada will be more than a third of the way there. Our contribution brings together four leading geneticists from across the country. Further, we recently awarded Jay Ross, a PhD student at McGill University, who is working in the lab of Dr. Guy Rouleau at the Montreal Neurological Institute, a $75,000 Trainee Award to process a subset of DNA profiles collected for Project MinE.
At the ALS Canada Virtual Research Forum, Dr. Jan Veldink, one of the Project MinE initiators, provided an overview of the project and an update on discovery achievements to date. Dr. Veldink is a professor of neurology and neurosurgery and head of the Human Neurogenetics Unit at the University Medical Center, Utrecht, Netherlands.
AMBRoSIA
A Multicenter Biomarker Resource Strategy In ALS (AMBRoSIA) is a research project led by the Motor Neurone Disease Association in London, United Kingdom. The project goal is to speed up diagnosis and understand the causes of ALS by identifying the unique biological fingerprints, or “biomarkers,” that could be used to diagnose ALS and predict its progression.
Researchers are collecting blood, urine and skin samples from 900 people with ALS and 400 people without ALS. Since 2016, scientists have been collecting and analyzing blood and urine samples to search for chemicals that might act as biomarkers of ALS. They are also engineering participants’ skin cells into stem cells and then reprogramming the stem cells to become motor neurons. In the lab, they are studying how the motor neurons respond to drugs and whether different drugs affect subtypes of ALS differently.
Biomarkers are also important for measuring responses to experimental therapies in clinical trials. Typical ALS studies to date have measured ALS responses using the ALS Functional Rating Score Revised (ALSFRS-R) and slow vital capacity, a measure of the volume of air exhaled without forced effort after a full breath. A better understanding of ALS may lead to targeted treatments for subtypes of the disease in the future.
At the ALS Canada Virtual Research Forum, Dr. Martin Turner, principal investigator of Project AMBRoSIA, discussed the project status two years into the five-year mandate and the biomarkers that have been discovered to date. Dr. Turner is a professor of clinical neurology and neuroscience at the John Radcliffe Hospital, University of Oxford, UK.
Updated October 17, 2018
The opportunity to be involved in clinical trials for experimental ALS treatments is something that is often on the radar for people who are living with ALS. Did you know that there are three clinical trials in Canada that recently started recruiting volunteers or will open soon?
On Wednesday, October 17, 2018, Dr. David Taylor, VP Research at ALS Canada hosted speakers from Canada’s world-class ALS research community as well as international researchers and experts to share their updates with you.
Our speaker line-up included clinical trial experts from three pharmaceutical companies who shared the latest updates on these new studies:
Phase 3 study of arimoclomol Orphazyme
A common feature of ALS is the accumulation of misfolded proteins in motor neurons that make it difficult for them to function correctly. In lab research, the drug arimoclomol demonstrated an ability to reduce the buildup of misfolded proteins by increasing the heat shock response.
Clinical research among a small group of human volunteers with ALS found that arimoclomol administered in an oral capsule format was safe and well-tolerated. But to further confirm the drug’s safety and to find out whether it can slow disease progression and/or improve survival, larger numbers of study participants are needed. (Learn more about clinical trial phases in our Clinical Trial FAQs or in this blog post from last year’s clinical trials webinar.)
The drug manufacturer Orphazyme recently launched a Phase 3 clinical trial. The first study site opened in Miami, Florida at the end of July 2018. Multiple locations in Canada, the United States and Europe will open soon.
Richard Bennett is a senior clinical project manager with Orphazyme A/S, a biopharmaceutical company based in Copenhagen, Denmark. During the Virtual Research Forum, he provided an update on the arimoclomol study and shared details about timing for Canadian study sites. He also discussed what he believes matters most to people who are living with ALS and how Orphazyme is including patient-centric approaches in the study.
Phase 3 study of levosimendan (ODM-109) Orion
ALS is characterized by a progressive inability to use muscles involved in movement, speech, swallowing and breathing. Eventually, most people with ALS need breathing support and adaptive aids to assist mobility. Levosimendan (ODM-109) is an oral medication that helps muscles contract more easily and exert a stronger force, possibly slowing down their loss of function.
An intravenous form of levosimendan called Simdax is already marketed in more than 60 countries around the world, (but not in Canada), for the treatment of acute heart failure. A Phase 2 study of an oral form of the drug for people with ALS was conducted in Europe and the United Kingdom in 2017. The results for a small number of study volunteers were promising, but not conclusive.
Orion, the maker of levosimendan, launched a Phase 3 clinical trial in April 2018 to determine whether an oral capsule format of the drug can improve respiratory function in people with ALS. The trial is called REFALS (Respiratory Function in Patients with ALS). Six Canadian trial sites will open for recruiting soon in Fredericton, Moncton, Montreal (Neuro and CHUM), Calgary and Edmonton.
Paula Rytilä, MD, PhD is Chief Medical Officer at Orion, a pharmaceutical company based in Finland. During the Virtual Research Forum, Dr. Rytilä presented the latest update on the REFALS clinical trial for oral levosimendan (ODM-109).
Phase 2 study of H.P. Acthar Gel Mallinckrodt Pharmaceuticals
One of the hallmarks of ALS disease progression is the over-activation of immune cells in the central nervous system. H.P. Acthar Gel is a medication that is injected beneath the skin. It contains a synthetic version of a hormone normally secreted by the pituitary gland called adrenocorticotrophic hormone (ACTH) that plays a role in regulating inflammation.
The drug is already approved in the United States for the treatment of flares associated with inflammatory diseases including lupus and multiple sclerosis.
Results from a Phase 1 study among a small group of people with ALS suggested that H.P. Acthar Gel might help reduce inflammation. Based on those results, Mallinckrodt Pharmaceuticals has launched a Phase 2 trial to examine whether the drug can slow functional decline in adults living with ALS. The trial is already underway and open for recruiting. Researchers are looking for 213 volunteers across 43 study sites located in Canada, the United States and Colombia. Currently, there are three locations recruiting in Canada — Edmonton, Longueuil and Montreal.
Susan VanMeter, MD, is Senior Director of Clinical Research at Mallinckrodt Pharmaceuticals, a company based in the United Kingdom. She shared more details about the clinical trial’s objectives at the Virtual Research Forum, how it is designed and how Canadians with ALS can participate.
Clinical research is essential towards realizing a future without ALS. Participating in clinical trials gives Canadians living with ALS an opportunity to try new therapies earlier and helps others who may one day be diagnosed with the disease.
How do you tell your friends and family you have ALS?
There is no easy way to tell family and friends you have been diagnosed with ALS. And there is no right or wrong way, either. Individual personalities, family circumstances, and the degree to which the symptoms are visible can all play a role in when, how, and with whom to share the news.
Lianne Johnston has worked with hundreds of families coping with ALS in the nine years she has been a Regional Manager for ALS Canada. She lists plenty of reasons people find it hard to talk about their diagnosis: feelings of denial, fear of upsetting family members such as elderly parents, a reluctance to receive pity from others, or lack of energy necessary to comfort others are just a few examples.
Fred Gillis: One family’s approach to sharing the news
For Fred Gillis, who was diagnosed with ALS in 2015, being open about the diagnosis and communicating the news quickly and extensively came very naturally. He and his wife, Lana, both peace officers in the RCMP, had experience delivering bad news in their professional lives that helped them to be proactive about informing the people around them. They describe themselves as “forthright and not the type of people to hide stuff,” so for them, putting off talking about Fred’s diagnosis was simply not an option although they are the first to admit this might not be everyone’s chosen path.
Fred’s willingness to talk about his ALS diagnosis doesn’t mean it wasn’t an emotional experience for him. Even today, he chokes up when he says that the hardest part was telling his three children, who were 16, 18, and 20 at the time. Within a week of receiving the news from their neurologist, Fred and Lana came up with a strategy for informing their children. They created a PowerPoint presentation to explain the disease and its progression, stress important information such as the fact that Fred’s ALS is not hereditary, and provide an opportunity to ask questions. Sharing with the children one at a time gave each of them a chance to react in their own way. For Fred and Lana, that was an important way to recognize their individual personalities.
When it came to telling their broader circle of family and friends, Fred and Lana created a communication strategy that recognized the power of all social media platforms. For Fred and Lana, it was very important that the news come directly from them as opposed to other sources. They chose to deliver the news in stages – all within a day or two – making use of personal email, internal work email and Facebook as their main modes of communication. Lana said that sharing information in this way was “extremely important” for Fred in accepting the disease and brought an “outpouring of love and support.” It also told people that Fred was open to talking about his disease and answering questions if anyone wanted to ask. And it meant that he and Lana didn’t have to tell the story over and over again.
Communicating can bring relief
As Fred explains, after sharing the news of his diagnosis, he felt “a tremendous sense of relief.” For him, it meant he could move on and spend his time in the best way possible. He understands that not everyone in his circle can journey alongside him. He doesn’t judge people. He relies on his sense of humour to deflect some of the sad feelings. As a 6’2” man who used to weigh 260 pounds, he jokes to his friends who he played hockey with: “Now you’ll finally be able to skate around me!”
When people are considering how to have some of the more difficult conversations about their ALS diagnosis, ALS Canada Regional Managers can provide guidance and insight as a result of their extensive experience working with families coping with ALS. Sarah McGuire, ALS Canada Regional Manager for Central Ontario & North Simcoe Muskoka, describes situations that can make delivering the news particularly difficult. “I have met people who recently lost close family members or friends for other reasons just prior to receiving their ALS diagnosis. Delivering this news on top of a loss can cause a lot of distress.” Similarly, in her work with parents who have young children, Sarah says, “I let people know that they are under no obligation to do anything immediately. However, it is important to know that our loved ones who know us very well – even young children – can eventually pick up on physical changes, stress levels, potential information being kept in confidence.” She says one of the benefits of being open about an ALS diagnosis is that you are fully in charge of the information that is shared.
You don’t have to do it alone
Melissa Van Tuyl, who supports people with ALS living in Hamilton, Niagara & Haldimand Brant, explains that she and her ALS Canada Regional Manager colleagues can attend family meetings to help answer some of the more difficult questions. “You don’t have to do it alone if you don’t want to,” she says, and recounts her experience with a woman who had worked in the school system for 25 years when she was diagnosed with ALS. The woman wanted to write a message to her colleagues so they wouldn’t assume she was simply taking early retirement. She and Melissa sat down together and wrote a personal message the woman could share with her colleagues in a way that was heartfelt and honest. ALS Canada can also provide families with resources such as “A manual for people living with ALS” or guides designed specially to help children and teens who have a family member living with ALS.
A neurologist’s perspective
Telling a patient that he or she has ALS is something that medical professionals take seriously so that the information is relayed in a way that is clear but also compassionate. In addition to being a neurologist and Medical Director of the ALS Clinic at the University of Saskatchewan, Dr. Kerri Schellenberg is the author of a paper called “Breaking bad news in amyotrophic lateral sclerosis: the need for medical education.” She stresses the importance of being sensitive to each of her patients’ individual needs in terms of how much information they want at any given time, and how to balance clear communication and empathy with respect to some of the common fears people express. She encourages her patients to ask as many questions as they want, and even to write down questions prior to meeting with her but as she admits, “it is a lot to take in at once.” She recommends a staged approach where she and her patient can revisit the same questions and introduce new information as time elapses.
In the same way, after someone has told family and friends about their diagnosis they can anticipate additional questions and the need to share new information as time passes. Using social media, creating a blog or sending group e-mails can be very helpful as a way to keep people informed. As Lana and Fred point out, “people just want to help.” Letting them know how can be difficult to articulate, but it can also open the door to just the kind of help, kindness, and support that is needed and appreciated.
What do you give someone living with ALS to show you care? There’s no ‘one-size-fits-all’ gift that works for everyone, but there are special ways to say “I am thinking of you.” The holiday season can be a perfect time to do just that. We recognize that it’s not always easy to know how to help, so we recently canvassed our regional managers and our online community for their gift ideas. They came back to us with practical suggestions, many of them suitable for someone regardless of where they are in the progression of their disease. Here’s what they had to say.
The gift of time
The gift of time was the number one response from across the board. Gail Renshaw, whose husband, Cliff, passed away from ALS in August, stressed the importance of sharing time together. “The most important thing for Cliff was to spend time with family and friends. Since his diagnosis two years ago, we planned and enjoyed many themed dinner parties to make things fun. ‘Spaghetti Western Night’ was his favourite. Everyone had to come dressed as a cowboy. We watched spaghetti western movies, ate spaghetti, cowboy beans, ribs and corn bread. We made some wonderful memories that we can cherish forever.”
Precious time together can take many forms. It can be a private moment between the two of you or a larger gathering among friends or family. Going out for dinner, or to a sports event or music concert can be a way to maintain connection and enjoy your time together in a different way. When planning a social outing, make sure to consider accessibility if the person you are with uses a cane, a walker, or a wheelchair. A gift of time can also be as simple as reading a newspaper or book aloud if someone you know with ALS is no longer able to turn the pages. Simply offering your company can provide comfort and support to a friend or loved one who is too tired to communicate, but would welcome a caring presence by their side. For the full-time caregiver, offering to spend an hour or so with the person with ALS can also provide some much-needed respite.
The gift of words
Another way to create cherished memories is to help videotape special messages or write letters for loved ones. If someone in your life has ALS and has expressed interest in documenting their feelings, hopes and desires, you could offer to help them by setting up a camera or typing out a message they dictate.
The gift of food
Many respondents also suggested preparing and freezing meals for the family as a way to reduce the load on caregivers. Heather Cockerline, whose dad had ALS, talks about what a relief it was for her parents to have help with cooking meals. “While he was sick, his care was the priority which made meal preparation daunting on some days. What Mom and Dad enjoyed was a steady supply of homemade soups and healthy meals. All they had to do was warm them up.”
The gift of service
The burden of completing daily chores can become overwhelming for a caregiver who is focused on attending to the needs of a loved one with ALS. Relieving some of the stress by offering your services is a wonderful way to help. Some of the most popular suggestions offered by our respondents included snow-shoveling, grass-cutting, babysitting, picking kids up from school, dog-walking, doing laundry, filling out forms, sending emails, and shopping. Housekeeping services were also high on the list, whether it was paid help on a regular basis or an offer from a friend to come and clean the house themselves.
The gift of relaxation
Stephanie Marshall-White suggested offering gift cards for physiotherapy, massage, manicures or pedicures. “My mom recently passed away from ALS and throughout her diagnosis she really liked physiotherapy or massage. I think it would be a great gift to be able to donate an hour of massage or whatever it might be. It really made her feel so much better.”
The gift of entertainment
Carol Skinner, ALS Canada Ambassador and someone who is living with ALS, describes the boredom she experienced as she lost mobility. She also struggles with the physical tiredness that accompanies the progression of the disease. She suggests offering to pay for streaming services such as Netflix, Spotify, or Apple Music to help pass the time. She also suggested smart devices such as smart thermostats, wireless lighting, and wireless speakers and home sound systems that enable a person to control the temperature, lights, and music in the house from their phone without having to get up.
The gift of flowers
Finally, respondents suggested a weekly flower delivery service as a cheerful gift to brighten the house. When you are housebound, a fresh bouquet of flowers every week can be a welcome sight to behold.
Updated October 20, 2017
Staying active and independent for as long as possible is important for people living with ALS as the disease progresses. There are many ways in which to manage symptoms or receive care, but there often isn’t enough research performed to determine which approaches make the greatest difference.
To help address these knowledge gaps and pave the way for better care, ALS Canada funds Clinical Management Grants thanks to donations from the Ice Bucket Challenge. So far, two clinical management grants have been awarded: one that focuses on helping people living with ALS to drive safely for longer, and another that is researching the use of cannabis (marijuana) to help manage pain and discomfort.
Driving Assessment: Helping People with ALS to Drive Safely, Longer
Associated with independence, driving is a complex activity that requires physical, emotional and mental focus. Unfortunately, many of these abilities decline as ALS progresses. Unlike other diseases like stroke or Parkinson’s, there are few published resources to measure when a person’s ALS has progressed to the point that driving becomes unsafe. As a result, ALS clinical care teams regularly face decisions about when to discuss driving with patients and families, whether someone living with ALS can drive safely, or if a referral for an on-road test is needed.
A retrospective review conducted by members of the ALS Clinic at the Montreal Neurological Institute & Hospital (MNI/H) found that many people with ALS were not making changes to their vehicles that had the potential to enable them to prolong driving: for example, adapted foot controls can help with pedal access, a ball installed on the steering wheel can help with steering; and a BiPAP machine secured in the vehicle can help with breathing.
To help empower people living with ALS, the clinic at the MNI/H, led by Dr. Angela Genge, is implementing a multidisciplinary, systematic approach to assess driving safety. “This initiative focuses on how the whole care team can help people with ALS drive safely for a longer length of time,” said Kendra Berry, the clinic’s occupational therapist, in an interview with ALS Canada.
Berry, who recognized the gap in care several years ago, is collaborating with the entire MNI/H multidisciplinary ALS clinic team on this initiative. After reviewing existing broad guidelines for other conditions, the clinical team is trying to establish a standardized driving assessment to be used in their clinical setting. The complete assessment incorporates the ALSFRS-R, a cognitive behavioural screen, and questionnaires for people with ALS and caregivers. Berry has also accumulated a battery of tasks to simulate driving actions that she is incorporating into regular clinic assessments.
With all this input, the team will develop checkpoints that the whole care team can use to help people with ALS decide when they may benefit from an adaptation, need to be referred for a road test or transition to giving up driving.
“We start the conversation at the first or second visit, rather than the usual timing of a year or two after diagnosis,” said Berry. Earlier discussion and driving assessment mean that people with ALS are empowered to access driving adaptations earlier.
The project is set to launch the week of August 28, 2017, and the team will follow about 200 study participants for two years. When the project is complete, they plan to share their learning with ALS clinicians across Canada and the SAAQ in Quebec, the provincial licensing authority for drivers’ licenses, which could help pave the way for other provincial and national groups to adopt them in the future.
Studying the use of cannabis extracts to manage ALS pain and discomfort
In surveys, people with ALS have reported that cannabis (marijuana) helps to alleviate weakness, speech difficulties, excessive saliva production, difficulty swallowing, breathing issues, pain and stiff muscles, and can improve sleep, appetite and mood.
Yet while cannabis has been used for symptom management by anywhere from 15 to 40 per cent of people with HIV/AIDS, epilepsy, chronic pain, multiple sclerosis and ALS, often the doctors providing care aren’t aware that their patients are using it.
In 2012, ALSUntangled conducted a review of cannabis studies and potential in ALS.
Studies of the effect of cannabis on ALS to date have used animal models or small numbers of people with single doses of only one ingredient – whereas cannabis contains over 480 compounds. The two most well-known active ingredients are tetrahydrocannabinol (THC), the psychoactive component that has been linked to analgetic action, appetite stimulation, decreased muscle stiffness and improved sleep; and cannabidiol (CBD), which has been studied for anti-inflammatory, anti-anxiety and anti-nausea benefits.
“THC, CBD and other ingredients in cannabis affect many different systems in the body,” said Dr. Mark Ware during the ALS Canada Virtual Research Forum on August 9, 2017. “For example, THC works by binding to a specific receptor found in the brain, spinal cord, the gastrointestinal tract and other body tissues. By affecting these receptors, cannabis has an impact on the ways our nerves talk to one another.”
Dr. Ware has been conducting cannabis research for medical purposes for almost 18 years, mostly in the area of pain management. He is executive director of the Canadian Consortium for the Investigation of Cannabinoids, director of Clinical Research at McGill University Health Centre’s Alan Edwards Pain Management Unit, and an associate professor in the Departments of Family Medicine and Anesthesia at McGill University in Montreal, Quebec.
With a clinical management grant through the ALS Canada Research Program, Dr. Ware is leading a team of Canadian researchers who will conduct a pilot study of the use of cannabis in 32 people with ALS starting later this fall. The other co-investigators include Dr. Angela Genge, director of the ALS Clinic and Clinical Research Unit at the MNI in Montreal, Quebec, and Dr. Colleen O’Connell, a neurologist specializing in physical medicine and rehabilitation who leads the ALS Clinic at the Stan Cassidy Centre for Rehabilitation in Fredericton, New Brunswick and is an assistant professor at Dalhousie University in Halifax, Nova Scotia.
The researchers are working with Green Sky Labs, a Canadian company licensed by Health Canada to grow marijuana for medicinal purposes, to obtain oral capsule products with different ratios of THC and CBD formulated in a medical-grade oil. Study participants with ALS will be part of a crossover study over an eight week period, with five days on one dose, followed by a nine day washout period, then another five days on a different dose, until all four ratios have been administered. Researchers will collect information about ALS symptoms, pain, mood, sleep, appetite, cardiovascular and respiratory effects, and any adverse effects.
Health Canada is currently reviewing Green Sky Labs’ application for product approval. The researchers expect to begin the study by the early 2018. Results from this pilot study will inform future research studies that could seek to explore whether cannabis can slow disease progression.
Clinical Management Grants are one category of high-quality research funded through the ALS Canada Research Program. Read more about the ALS Canada Research Program and consider donating today.
ALS Canada Virtual Research Forum
Update: Dr. Ware was one of more than 20 speakers who participated in the ALS Canada Virtual Research Forum in August. You can listen to his full presentation online here.
ALS is difficult to diagnose because no single test or procedure can firmly identify the disease. Current diagnostic tests for ALS focus on ruling out other diseases that share similar initial symptoms. For example, magnetic resonance imaging (MRI) is a test typically used to eliminate a diagnosis of cancer, multiple sclerosis or pressure on the spinal cord due to arthritis. A standard MRI analysis of a person with ALS, however, usually shows normal results.
The lack of a reliable diagnostic test for ALS means that it takes on average about a year for a diagnosis to be confirmed. “The delay means we can’t help people sooner, nor identify them early enough to enter a clinical trial,” said Dr. Sanjay Kalra in an interview with ALS Canada. “This issue is actually hindering the clinical investigation of drug therapies. If we could identify people with different types and progressions of ALS more quickly, we could find a breakthrough therapy faster. A test is desperately needed that can determine if a drug is working in clinical trial.” Dr. Kalra is a professor in the department of medicine (neurology) and member of the Neuroscience and Mental Health Institute at the University of Alberta.
Looking for ALS in Brain Images
Dr. Kalra has been intrigued with finding a way to harness the power of imaging to uncover the early signs of ALS since his medical residency when he met neurologist Dr. Douglas Arnold, an MRI specialist at the Montréal Neurological Institute and Hospital. “My residency research rotation was only supposed to last six months, but after realizing the potential power of this technology in research and really enjoying working with people with ALS, I decided to continue and eventually stayed as a postdoctoral fellow specializing in MRI for ALS,” said Dr. Kalra.
Dr. Kalra has secured funding for his imaging research program from a number of sponsors including the Canadian Institutes of Health Research (CIHR), the major agency of the Canadian government responsible for funding health research in Canada. His focus has been to develop and validate advanced MRI methods that can be used as a biomarker, a biological marker that allows physicians to detect disease earlier, monitor disease progression and evaluate new therapies.
In 2013 he founded the Canadian ALS Neuroimaging Consortium (CALSNIC), a multidisciplinary team of experts from across Canada that includes neurologists, MRI scientists, computing scientists, neuropathologists and a biostatistician. Since then, the CALSNIC team has been working together on a national scale to develop advanced MRI methods to find biomarkers in people with ALS and related conditions.
Dr. Kalra and three colleagues at the University of Alberta in Edmonton conducted a preliminary study in 2014-2015 to look for biomarkers in brain images using MRI scans of 19 people with ALS and 20 healthy participants for comparison. They analyzed the images with 3D texture analysis, an advanced method that allowed them to look for statistically significant patterns of brain degeneration not normally visible to the naked eye. They examined voxels, tiny 3D spaces in the brain about a cubic millimetre in size.
The researchers found different texture features in regions of the brain affected by ALS and frontotemporal dementia in people with ALS compared to people without ALS. They also discovered that some features were associated with clinical observations, such as disease duration and differences in finger tapping speed. The study was funded in part by an ALS Canada Discovery Grant.
Going Big: The First Large Imaging Study in the World
Based on encouraging research results in this preliminary study and other work, Dr. Kalra wants to confirm the findings in a larger group of people with ALS. “The ALS field has seen an explosion of imaging studies in the last five years, but for the most part, they have been single-centre studies that used different methods in small groups of patients, so it has been difficult to draw conclusions on the best method to use,” he said. “To develop and validate the use of MRI biomarkers , especially for their potential use in clinical trials, we need to confirm that our these methods will work well on a large scale, in every clinic.”
In 2015, Dr. Kalra and a team of 13 other investigators applied for a grant from ALS Canada and were successful. The resulting ALS Canada – Brain Canada Arthur J. Hudson Translational Team Grant of $2.94 million – the largest grant awarded in ALS Canada’s history – is funding the first large multicentre imaging study focused on ALS in the world, according to Dr. Kalra. The study seeks to enroll over 700 volunteers split between people with ALS and people without ALS for comparison in seven locations: Calgary, Edmonton, Vancouver, London, Toronto, Montreal and Quebec City. Some sites are currently recruiting and others will be up and running soon. Participants receive a baseline MRI and clinical evaluation followed by two follow-up visits.
Always thinking ahead, Dr. Kalra is already considering how to expand CALSNIC further to increase the value of the network. “Another purpose of setting up the CALSNIC infrastructure is that it allows us to probe other questions. I’m excited that it has spurred other ALS research and collaborations,” said Dr. Kalra, “such as at the University of Toronto where Dr. Yana Yunusova is studying speech changes in patients across the CALSNIC network and will be able to compare the findings with our imaging data. In the future, I would like to see CALSNIC build a comprehensive resource of tissue, imaging and clinical descriptions that all scientists can access to understand the disease better.”
ALS Canada has been funding world-class research across Canada for over 30 years to enable discovery of new treatments and therapeutic interventions that have the potential to make an impact on altering the course of the disease or improving the quality of life for people with ALS.
Finding new treatments that can change the course of ALS requires a robust research program, one that supports a large community of researchers exploring multiple avenues of inquiry. ALS Canada has been supporting world-class research across Canada for more than 30 years with a variety of grants and awards that are helping to learn more about how ALS works in the body with the goal of making ALS a treatable, not terminal disease.
It is an exciting time in ALS research: the amount of activity and discovery has increased more in the last five to seven years than in the last century – to the point that there are more projects, ideas and tools to explore than funds available, particularly in the case of the ALS Canada Research Program which is supported entirely by donor dollars (including the Ice Bucket Challenge) and partnerships with provincial ALS Societies. With limited funds available, ALS Canada must be able to make the most of them.
“ALS Canada uses a comprehensive peer-review process to decide which projects receive funding, ensuring that donor dollars will have the maximum impact to advance learning in the field,” said Dr. David Taylor, Vice President of Research at ALS Canada during a webinar presentation on July 20, 2017. The webinar focused on the history of the ALS Canada Research Program, the different types of grants available, and the status of the research program today.
Over the past several years, donations raised through the Ice Bucket Challenge have boosted the amount that ALS Canada Research Program has been able to direct to grants and awards, which previously averaged $1.5 to $2 million per year. In 2016, ALS Canada awarded $3.5 million in research grants, $2.7 million of which was matched by the Brain Canada Foundation through the Canada Brain Research Fund (with the financial support of Health Canada) for a total of $6.2 million.
The ALS Canada Research Program: A brief history
In 1977, a neurologist named Dr. Arthur J. Hudson in London, Ontario, co-founded ALS Canada to provide supportive care for people with ALS and their families. He also established the country’s first multi-disciplinary clinic where people with ALS could see a neurologist as well as speech pathologists, dietitians, respirologists and other specialists. The research program started slowly in the 1980s when ALS Canada began awarding small grants of a few thousand dollars. By 1999, the program included multi-year grants that encouraged new researchers to enter the field, and it continued to evolve with an expanded range of grant programs that set the stage for the ALS Canada Research Program as it exists today.
In 2017, ALS Canada is funding research grants and awards through three separate competitions:
The ALS Canada – Brain Canada Arthur J. Hudson Translational Team Grant was created in 2014 and is currently a partnership between ALS Canada and Brain Canada. In 2017, this grant will help to accelerate the development of effective ALS treatments by providing up to $1.8 million to teams of three or more researchers from at least two institutions, fostering collaboration and promoting the sharing of information. Since 2014, over $18 million dollars in Hudson Grants have been awarded thanks to the generosity of Canadians through the Ice Bucket Challenge.
ALS Canada Trainee Awards support Canada’s emerging ALS researchers, whether they are doctoral students, post-doctoral researchers, or clinical research fellows. Trainee awards are instrumental in encouraging young researchers to choose ALS as their area of focus – helping to ensure that Canada has a strong community of talented ALS researchers. Successful recipients receive salary support awards appropriate for their level from a minimum funding pool of $200,000.
ALS Canada Project Grants support a range of promising research projects, including those at an early pilot stage that need seed funding to obtain initial data, those focused on improving the symptoms or experience of someone living with ALS, or those projects that are well established, but have not been successful in securing one of the very limited awards given by the Canadian Institutes of Health Research, the primary health research funding agency of the Canadian federal government. Project grants are designed to fund the best ALS work in Canada that will have significant impact on the global effort to understand the disease and find new ways to treat it.
Deciding which research projects to fund
Simply put, there is more ALS research to fund than money available – making it necessary for ALS Canada to have a proven method for making difficult decisions about which research receives funding. ALS Canada has always awarded funding based on a peer review process that is continually evolving and being optimized. Known within the scientific community as the gold standard for assessing all types of research, peer review engages independent experts in evaluating the quality and rigour of research papers and proposals.
For ALS Canada, the awarding of grants begins with an invitation to the Canadian ALS research community to submit their research proposals. Information about funding opportunities is publicly available on the ALS Canada website and includes eligibility criteria and application deadlines. ALS Canada reviews all applications to ensure they are eligible and complete before they are moved on to the next phase.
Next, ALS Canada recruits panels of experts in ALS and other neurodegenerative diseases to review the proposals. In order to engage experts who don’t have a connection to any of the proposals being reviewed, it is common to recruit panel members from beyond Canada. To enable panel members to freely critique research proposals, their names are not disclosed.
The panel meets to discuss the merits of each submission in detail after which all panel members assign a score to the proposal. ALS Canada Board representatives and senior staff observe the process, along with at least one person who is living with ALS, but they do not participate in the discussion or scoring. The average scores are ranked and the top proposals are considered for funding. The panel then determines the list of projects they recommend for funding to the ALS Canada Board of Directors.
Finally, the ALS Canada Board of Directors reviews the panel’s funding recommendations to reach a funding decision. The goal is to fund as many project as possible based on the research dollars that are available in a given year. ALS Canada notifies all applicants as to whether their proposals were successful, and provides feedback to unsuccessful applicants so they can strengthen their proposals if they wish to re-apply in the future.
While the peer review process is not without its downsides – a common criticism is the length of time the entire process takes – it is a proven, well-respected and internationally recognized approach. ALS Canada continues to review its approach to research funding so we can be responsive to new and emerging opportunities.
In 2014, the Ice Bucket Challenge became a viral social media sensation … and ALS researchers across the country – along with ALS Canada staff and supporters – responded to the challenge. For a full list of the researchers in the video, see below.
Dr. Angela Genge and Dr. Rami Massie, Montreal Neurological Institute
Dr. Stuart Cleary and Dr. Kelvin Jones, University of Alberta
Dr. François Gros-Louis, CRCHU, and Bastien Paré, Université Laval
Dr. Pierre Drapeau, Dr. Alex Parker, Dr. Martine Therrien, Guillaume Caron, Alexandria Lissouba and Dr. Gary Armstrong, CRCHUM
Dr. Lorne Zinman, Dr. Janice Robertson, Dr. Aaron Izenberg, Dr. Laura MacNair and Dr. Jesse McLean, Sunnybrook Research Institute
Dr. Colleen O’Connell, Dalhousie University
Cindy Lister and Robin Farrell, ALS Cycle of Hope
Dr. Richard Robitaille, Elsa Tremblay, Eric Martineau and Dr. Danielle Arbour, Université de Montréal
Dr. Sanjay Kalra, University of Alberta
Cindy Lister and Robin Farrell, ALS Cycle of Hope
Kristiana Salmon, Montreal Neurological Institute
Dr. Michael Strong, Michael Tavolieri and Alexander Moszczynski, Western University
Denrell Mah, Edmonton ALS Clinic
Dr. Jean-Pierre Julien, Dr. Jasna Kriz, Vincent Picher-Martel and Louis-Charles Beland, Université Laval
Dr. David Taylor, VP Research, ALS Canada
Dr. Victor Rafuse, Dalhousie University
Marie Beaudin, lab of Dr. Nicolas Dupré, Université Laval
Dr. Wendy Johnston, University of Alberta
Dr. Christen Shoesmith, London Health Sciences Centre ALS Clinic
Dr. Geneviève Matte, Notre Dame Hospital
Dr. Heather Durham, Dr. Benoit Gentil and Dr. Michael Tibshirani, Montreal Neurological Institute
ALS Canada Board of Directors
Tammy Moore, CEO, ALS Canada
Dr. Fabio Rossi, University of British Columbia
Brian Parsons, ALS Canada advocate
Brad Butt, former MP, Mississauga — Streetsville
Dr. Agessandro Abrahao, Sunnybrook Hospital
Robert Blum, President and CEO, Cytokinetics
Dr. Guy Rouleau, Dr. Patrick Dion and Dr. Martine Therrien, McGill University
ALS Canada Staff
Please note that this is not a comprehensive list – many researchers across the country participated in the challenge but were not featured in this video.
Many people living with ALS and their families want to know how scientific discoveries move from basic research in the laboratory through different phases of clinical trials using human volunteers. They also want to understand why clinical research is required before a new therapy is approved and why it takes so long for researchers to determine if a promising new treatment works.
Dr. David Taylor, Vice President of Research at ALS Canada discussed these topics and more during a lively webinar presentation about clinical trials on June 29, 2017. The webinar was the second of four presentations in the ALS Canada Webinar Series to provide an update on the latest ALS research, clinical trials and funding programs.
How are researchers improving clinical trial design to find better answers, faster? What are the best resources for finding reputable information about treatment options? Which clinical trials are recruiting participants? What are some of the most promising trials underway? Dr. Taylor addressed these questions and many more.
The Spectrum of Research Discovery
At the beginning of the research discovery spectrum, scientists conduct preclinical research in the laboratory, looking for new understandings about the biology of the disease, designing new therapies that address how the disease works and conducting studies in cells or animals such as mice, rats, fish, flies or worms. These animal models have a simpler biological structure than humans, which helps researchers to better understand how a potential therapy works in a very general way and what the side effects are.
Before a new therapy can advance from the laboratory to human clinical trials, a lot of work takes place to ensure it will be safe to test in people. Researchers must apply to Health Canada for approval, showing data from toxicity studies in animal models and how they expect the drug will be processed by the human body.
After approval to proceed, clinical trials move through Phases 1, 2 and 3 in order, as long as a drug or intervention continues to show strong results. Participants visit a clinical trial site, usually a hospital or medical clinic, to receive an assigned treatment. Researchers measure their progress by collecting information about how their body is reacting to the intervention. The study objectives, the number of participants, cost and timing vary for each phase:
Why Does It Take So Long?
As you can see from the table above, it can take five to eight years or more for a new therapy to move through all three clinical research phases. Why does it take so long? Because each phase involves securing approvals and funding, establishing study sites, finding and screening patients that meet the eligibility criteria for what’s being tested, and collecting and analyzing the data.
After Phase 3, if the evidence shows that a new therapy can slow disease progression, the drug manufacturer decides whether to apply to Health Canada for market approval. Expedited review for a drug application takes about six to 12 months. After that, each province may take an additional two years or more to decide whether to cover the cost of the drug.
In the event of a positive Phase 3 trial, ALS Canada will advocate with drug manufacturers and Health Canada to help expedite the process of making the treatment available to Canadians and to have costs covered.
Designing Clinical Trials to Ensure Answers
Clinical trials must be designed properly to ensure collecting the right information about whether a new therapy is safe, and if it works or doesn’t work.
Given the wide variability in the rate of disease progression among people with ALS, researchers are exploring new indicators to measure responses to therapies in clinical trials. The ALS Functional Rating Scale – Revised (ALSFRS-R) remains the most common outcome measure, but a range of new measures for functional abilities in the clinic and using consumer wearables or mobile apps are currently being examined.
Biomarkers, biological markers in tissue or blood, have been identified recently as essential for improving clinical trial design. One way they can be used is to screen patients to ensure inclusion of those most likely to benefit in a trial. For example, in the new trial of NP001 in progress, participants are pre-screened for C-reactive protein, a known biomarker for inflammation. Beyond this specific example, biomarkers are crucial to the success of ALS clinical trials in general because they explain whether the therapy is doing what it’s expected to. Without knowing this, it becomes very difficult to interpret results.
Beware Unproven Treatments Found on the Internet!
It’s easy to find alternative treatments for ALS promoted on the Internet, but unfortunately, many are unproven, or worse, unsafe. ALSUntangled is an excellent resource for determining if an alternative treatment is based on sound science. ALSUntangled is led by Dr. Richard S. Bedlack, Duke University ALS Clinic Director and sponsored by the ALS Association and the Motor Neurone Disease Association. Dr. Bedlack and a team of ALS researchers evaluate the science behind alternative treatment claims and issue a comprehensive review of the potential mechanism of action, the available evidence and risks. Dr. Bedlack will be discussing ALSUntangled at the ALS Canada Virtual Research Forum coming up in August 2017.
How to Find a Reputable Clinical Trial for ALS How to Find a Reputable Clinical Trial for ALS
The best sources for finding reputable information about treatments that have already been tested, or to find a clinical trial that is currently recruiting new participants are:
ClinicalTrials.gov – a registry and results database of human clinical trials conducted around the world, including both publicly and privately funded studies. Listing information includes the study objectives, phase, whether the study is currently recruiting participants, inclusion and exclusion criteria and locations.
ALS Canada maintains a list of the current clinical trials in Canada
Clinical Trials to Watch
Phase 3 trials for masitinib, NurOwn® and tirasemtiv are ones to watch closely. Dr. Taylor spoke about these clinical trials and others during the first webinar.
ALS Canada Virtual Research Forum
ALS Canada will host a Virtual Research Forum on Wednesday, August 9 and Thursday, August 10, 2017 from 11:00 a.m. – 5:00 p.m. Eastern time. The forum is free and open to anyone interested in learning more about ALS research currently underway and therapies in development. Hosted by Dr. David Taylor, the forum will feature more than 20 speakers and panelists over two days. Advance registration is recommended as spots are limited.
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Phase 3 study of arimoclomol Orphazyme
Richard Bennett is a senior clinical project manager with 
Paula Rytilä, MD, PhD is Chief Medical Officer at 
For Fred Gillis, who was diagnosed with ALS in 2015, being open about the diagnosis and communicating the news quickly and extensively came very naturally. He and his wife, Lana, both peace officers in the RCMP, had experience delivering bad news in their professional lives that helped them to be proactive about informing the people around them. They describe themselves as “forthright and not the type of people to hide stuff,” so for them, putting off talking about Fred’s diagnosis was simply not an option although they are the first to admit this might not be everyone’s chosen path.
