Canada continues solidifying its reputation as a leader in ALS research innovation, as evidenced by the magnitude and quality of conversations, knowledge sharing, and relationship building at the 2024 ALS Canada Research Forum. From April 27 to 29, researchers, industry partners, ALS Canada supporters, and more attended the in-person, three-day event, that aimed to move the needle on research toward effective therapies and avenues to improve the quality of life for people affected by ALS. 

The weekend kicked off with a keynote presentation by neurologist Prof. Orla Hardiman on PRECISION ALS, a pan-European collaborative platform that brings together researchers, industry experts, and more to accelerate drug discovery and treatment for ALS. Later that evening, attendees heard from community members Mehboob and Sophie Damji. They also learned more about the global work of the International Alliance of ALS/MND Associations from the CEO, Cathy Cummings, and the role ALS Canada plays in working toward a world free of ALS. 

Throughout the weekend, attendees were treated to engaging presentations from researchers of all levels, including students, doctoral candidates, postdoctoral fellows, and senior researchers and clinicians. Session topics were diverse – from using virtual reality to improve breathing in people living with ALS, to identifying environmental risk factors for ALS, to poster presentations by up-and-coming junior researchers, to a panel discussion led by seasoned researchers on what makes a great principal investigator. These presentations and conversations further cemented the ALS research community’s deep-rooted commitment to finding a cure for ALS through collaboration and partnership. 

For Canadian ALS Learning Institute graduate and ALS Canada Community Ambassador Andrew Dundas, the Research Forum was fascinating and an opportunity to share his lived experiences with researchers. “I’m hoping to understand the research [on] not only for advancement and the cause of ALS, ultimately [to find] a cure, but also for opportunities to improve the quality of life for patients with ALS like myself while a search for a cure is found.” 

Andrew reminds us of the reason knowledge-sharing events like the ALS Canada Research Forum are needed – to foster hope, collaboration, and solutions in the pursuit of creating a world free of ALS. He reminds us that finding a cure is not the only goal to strive for and that working towards improving quality of life is just as important.  

For Jenny Zhang, a junior researcher at the University of Ottawa and the winner of the Research Forum’s Minute to Win It and poster competitions, the event was a valuable experience that allowed her to network and receive feedback on her work. “As someone currently exploring potential career possibilities, the 2024 ALS Research Forum allowed me to learn more about various ALS research pathways that I wasn’t aware of…All of these experiences will support my progress and improve my work.”  

Jenny also echoes the sentiment of many Research Forum attendees that collaboration is critical for advancing ALS research. “I believe that uniting researchers with varying scientific backgrounds and perspectives can help drive innovation and speed up the development of treatments for complex diseases like ALS,” she said. “Bridging together researchers and clinicians and people living with or affected by ALS has provided a sense of hope for the community to continue making progress in ALS research and improve quality of life.” 

The 2024 ALS Canada Research Forum is made possible thanks to generous support from our platinum sponsor, Mitsubishi Tanabe Pharma Canada; our gold sponsor, Amylyx Pharmaceuticals; and our silver sponsors, Biogen and QurAlis. We thank our sponsors for their support and generosity in fostering collaboration, knowledge sharing, and capacity building at the 2024 ALS Canada Research.

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.

CAPTURE (Comprehensive Analysis Platform To Understand, Remedy, and Eliminate) ALS is a platform established in Canada to gather biological samples, data, and experiences of people with ALS and healthy volunteers from across the country.

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.

 

On World Science Day, the latest ALS Canada-Brain Canada research awards, with support from Fondation Vincent Bourque, highlight the importance of funding early-career researchers and clinicians

 

Toronto — In recognition of World Science Day, together with Brain Canada, the ALS Society of Canada (ALS Canada) is pleased to announce the 2023 ALS Canada-Brain Canada Clinical Research Fellowship and Trainee Award recipients. These early career grants are designed to support the training and research of clinicians in clinical care and PhD students and postdoctoral fellows engaged in research related to amyotrophic lateral sclerosis (ALS) across Canada.

With an estimated 3,000 Canadians living with ALS, investing in the next generation of researchers is critical to learning more about the disease, improving therapies, and eventually finding a cure.

“Our commitment to invest in clinicians and researchers early in their careers through the Clinical Research Fellowship and Trainee Award programs ensures that we have top medical and scientific talent working hard to find treatments for people living with ALS,” said Dr. David Taylor, Vice-President of Research and Strategic Partnerships, ALS Canada. “These are critical parts of our national Research Program that drive optimal care and discovery toward our vision of a future without ALS.”

“Investing in the future of ALS research means investing in the brilliant young minds who possess the insights needed to unravel the complexities of the disease,” says Dr. Viviane Poupon, President and CEO of Brain Canada. “Through the Clinical Fellowship Award and the Trainee Award program, Brain Canada proudly supports these up-and-coming scientists, as they are shaping a more hopeful future for those battling ALS.”

The Clinical Research Fellowship is designed to support a clinician’s training in clinical care and research skills related to ALS, which is crucial to building better clinical infrastructure across Canada.

The Clinical Research Fellowship will provide $200,000 in funding.

 

Summary of 2023 Clinical Research Fellowship

The Trainee Awards include two streams of funding: Doctoral students and postdoctoral fellows receive financial support to cover their salaries for up to three years, providing Canadian labs with the necessary funds to have top researchers working on the best projects to better understand the disease and drive toward new treatments for individuals living with ALS.

A total of $303,000 was awarded through the 2023 Trainee Awards.

 

Summary of 2023 Doctoral Awards

 

Summary of 2023 Postdoctoral Fellowship

 

World Science Day reminds us of the impact science has in all our lives. ALS Canada is proud to support emerging scientists and clinicians whose ideas and discoveries will advance treatment and eventually lead to a world free of ALS.

Funding for Anna Huynh’s Doctoral Award was made possible through partnership with Fondation Vincent Bourque, who generously contributed $25,000 to ALS Canada, which was matched by Brain Canada through the Canada Brain Research Fund (CBRF).

The CBRF is an innovative arrangement between the Government of Canada (through Health Canada) and Brain Canada Foundation, which increases Canadians’ support for brain research and expands the philanthropic space for funding brain research to achieve maximum impact. To date, Health Canada has invested more than $145 million in brain research through the CBRF which has been matched by Brain Canada Foundation and its donors and partners.

 

About ALS Canada and the ALS Canada Research Program

The ALS Society of Canada (ALS Canada) is working to change what it means to live with amyotrophic lateral sclerosis, an unrelenting and currently terminal disease.

Grounded in and informed by the Canadian ALS community, we respond to the urgent unmet need for life-changing treatments by investing in high-quality research that will fuel scientific discovery and by engaging industry, supporting increased clinical capacity and advocating for equitable, affordable, and timely access to proven therapies.

Responding to the tremendous need for current and credible ALS knowledge, awareness, and education, we empower Canadians affected by ALS to navigate the current realities of ALS, be informed consumers of ALS information, and advocate effectively for change.

Through the ALS Canada Research Program, we fund peer-reviewed research grants, foster collaboration and build capacity within Canada’s ALS research and clinical community, and invest in new areas of research positioned to have high impact. As the only national dedicated source of funding for ALS research across Canada, the ALS Canada Research Program aims to accelerate research impact by providing funding for the most promising ALS projects focused on translating scientific discoveries into treatments for ALS. We are grateful for the support of our donors and the contributions from participating provincial ALS Societies through the Walk to End ALS.

 

About Brain Canada

Brain Canada is a national non-profit organization that enables and supports excellent, innovative, paradigm-changing brain research in Canada. It plays a unique and invaluable role as the national convener of the brain research community. We join people, labs and platforms across the country, as well as institutions, organizations and sectors – to drive innovation and foster an interconnected brain research system. Our work enables Canada to excel and make even greater contributions to the global quest to understand the brain and brain disorders. Join us in funding brilliance daily, braincanada.ca.

 

About Fondation Vincent Bourque
Vincent was diagnosed with ALS in 2015. He knew that ALS was not an incurable disease, but an underfunded one. With his wife, Isabelle Lessard, and many friends, he created a Fondation to help families living with ALS and to support research in order to eradicate ALS. He passed away in 2018, but his legacy to the ALS community is immeasurable. The Fondation Vincent Bourque has raised more than $1,200, 000 since 2018, given five research grants to star researchers dedicated to ALS, and distributed more than $250, 000 to help families. The Fondation Vincent Bourque is proud to be working with ALS Canada and Brain Canada toward a future without ALS.

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Join the conversation and connect with the ALS community online. Find ALS Canada on Twitter, Instagram, or like our page on Facebook. Visit als.ca to find out more.

For more information
ALS Society of Canada
media@als.ca 
437-703-5402

Brain Canada
Brielle Goulart
brielle.goulart@braincanada.ca
450-915-2253

In March 2023, the ALS Society of Canada and Brain Canada announced the latest round of Discovery Grant recipients from their 2022 research grant competition. With support from SLA Québec and the Dr. Jean-Pierre Canuel Fund, nine Canadian researchers and their teams received a total of $1,475,000 in funding to fuel innovation that will accelerate our understanding of amyotrophic lateral sclerosis (ALS) and identify pathways for future therapies.

Projects were selected following a competitive peer-review process, in which international ALS experts considered the merit of the applicant, the quality of the project, and the potential to advance the field of ALS research. In 2022, the Discovery Grant program introduced two grants of $300,000, an increase from the traditional amount of $125,000, as part of the partnership’s efforts to support more ambitious projects.

Real hope for Canadians living with ALS

For Canadians living with ALS, like Paula Trefiak, this funding represents a source of hope. 

Paula has a familial variation of ALS, and she has lost 27 family members to the disease. Since 2018, she has served as a Community Ambassador for ALS Society of Canada, as well as on its Scientific and Medical Advisory Council (SMAC). 

“This partnership means a lot,” she said. “We have brilliant Canadian minds working on these innovative projects. It’s incredible to see their ideas end up in trials or give us more information about how ALS works so we can develop treatments. That’s really important to me, and of course, to my family.” 

Paula has personal experience with the potential of ALS research. For the past five years, she has been receiving tofersen, a therapy designed for people with SOD1 gene mutations, through a clinical trial in Montreal and now through the Calgary ALS clinic. The treatment was recently approved by the FDA in the United States but is not yet approved by Health Canada, though it can be accessed via a Special Access Program. Tofersen was developed after many years of work to understand and target the precise biology of SOD1-ALS, which represents a small proportion (~2%) of ALS cases. That same understanding hasn’t yet been achieved for most people with ALS. 

So far, she says, the treatment seems to have slowed her disease progression and even reversed some of her symptoms. 

“I’m very excited for people with other variations of ALS to see the same success that I have,” she said. “And I have no doubt that Discovery Grant research will help that.”

Canadian research with global impact 

Canadian ALS research is known for punching above its weight, thanks in part to a history of bold funding through the Discovery Grant program. 

“These exciting projects led by teams across Canada are contributing to global scientific discovery,” says Brain Canada President and CEO, Dr. Viviane Poupon. “Our unique partnership with ALS Canada has the potential to lead to improve diagnosis and treatment for people living with ALS, and it’s something that we are very proud of.”  

Since 2014, ALS Canada’s partnership with Brain Canada has resulted in more than $25 million being invested in ALS research that has helped further our understanding of the disease. 

“The Discovery Grant program continues to support critical Canadian research that contributes to the global effort on understanding and treating ALS. Over the past 15 years, many discoveries made through this program have provided a foundation for studies that are impacting humans today, whether through clinical trials or critical initiatives like CAPTURE ALS,” says Dr. David Taylor, Vice-President, Research and Strategic Partnerships, ALS Canada. 

Meet This Year’s Discovery Grant Recipients 

Over the coming months, we will share more about each of the research projects, going behind the science to find out how–and why–some of Canada’s top ALS researchers do what they do. Check back for new stories regularly.

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Best practice recommendations establish national standard and address issues important to care in Canada

Toronto – The Ice Bucket Challenge continues to make an impact. Proceeds from the highly successful 2014 viral sensation have helped fund a comprehensive new resource to guide efforts to ensure people living with ALS in Canada receive the best possible care no matter where they live.

The first Canadian Best Practice Recommendations for the Management of Amyotrophic Lateral Sclerosis, published in the latest issue of the CMAJ (Canadian Medical Association Journal), recommends people living with the disease receive specialized multidisciplinary care, and addresses issues important to caring for people living with ALS in Canada, including timeliness of care, disease-modifying therapies, medical assistance in dying (MAiD) and caregiver support.

ALS, also known as Lou Gehrig’s disease, gradually paralyzes people because the brain and spinal cord are no longer able to communicate with the muscles of the body that we are typically able to move at will. With four out of five people dying within five years of diagnosis in Canada, providing timely, optimal care from coast-to-coast is critical.

“These Best Practice Recommendations are an important step forward for improving the lives of people living with ALS in Canada. We hope they will help enable ALS clinics across the country to meet a common national standard, offering the best possible care to their patients and helping them navigate this complex and devastating progressive disease,” says Dr. Christen Shoesmith, Neurologist & Motor Neuron Disease Clinic Director at London Health Sciences Centre and Chair of the ALS Best Practice Recommendations Working Group.

With the new guideline, people living with ALS in Canada, their families and healthcare providers are also now empowered to make informed, collaborative decisions and advocate for optimal care consistent with best available evidence and expert consensus.

Until now, there have been no recommendations explicitly for the care of people living with ALS in Canada. Clinicians and care teams have relied on older guidelines published in the US and Europe, which do not address issues important to care in Canada.

“It’s critical that each person living with ALS, along with their family and caregivers – no matter where they live in Canada – have access to the best and most appropriate care for their disease,” says Tammy Moore, Chief Executive Officer, ALS Society of Canada. “That’s why it’s important that these guidelines reflect Canadian experience, values and the healthcare system. We congratulate the Working Group and everyone involved in developing this important resource that will help to improve the standard of care.”

A 13-member pan-Canadian Working Group developed the guideline’s more than 130 recommendations across 13 areas of focus to provide an update on evolving best practices of care in ALS. In doing so, they assessed hundreds of published peer-reviewed articles, and sought the input of people living with ALS in Canada, ALS Societies across the country, as well as other health providers and organizations.

Funding for the guideline was made possible through donations from the 2014 Ice Bucket Challenge and the Canadian ALS Research Network (CALS), a partnership of clinicians across Canada that specialize in ALS research and clinical care.

“We thank Canadians who enthusiastically supported the ALS community through the Ice Bucket Challenge which helped bring these recommendations to life. We continue to strive to make significant impacts with the ongoing generosity and support of Canadians coast to coast,” Moore continues.

About the ALS Canada Research Program and Canada’s ALS Societies
The ALS Canada Research Program funds peer-reviewed research grants and fosters collaboration amongst Canadian researchers, helping to nurture new ideas and build capacity. As the only dedicated source of funding for ALS research in Canada, the ALS Canada Research Program aims to accelerate research impact by providing funding for the best ALS projects focused on translating scientific discoveries into treatments for ALS. Collectively through initiatives like the Walk to End ALS, ALS Societies across Canada support the ALS Canada Research Program. ALS Societies across Canada work together to maximize our collective impact and make the greatest difference for people affected by ALS. Our approach as eight independent organizations working in partnership enables us to respond to the variation that exists between provincial healthcare systems, where we each play a role in filling gaps by providing community-based support. ALS Societies advocate federally, provincially and locally on behalf of people and families living with ALS for policy changes that will have a meaningful impact today and in the future.

The ALS Canada Research Program awards $650K for three innovative new research initiatives, with an additional $1 million to be announced in 2021

TORONTO – The ALS Society of Canada today announced that as part of its 2020 research commitment, the ALS Canada Research Program is investing $650,000 in three new initiatives that will contribute to the development of potential ALS therapies and strengthen ALS clinical care in Canada. The investments are in keeping with the need for Canadians living with ALS to have access to quality care, and for innovative new treatments that change the reality of the disease and its devastating impact.

As the only dedicated source of ALS research funding in Canada, the ALS Canada Research Program plays an essential role in the sustained research investment needed to address unanswered questions about the causes and progression of ALS so that targeted treatments can be developed. The three initiatives are being funded following of a peer-reviewed grant competition that engaged a panel of scientific experts in prioritizing projects grounded in scientific excellence and with the potential to most quickly advance the field of ALS research, contributing to the knowledge base needed to develop treatments.

The research being funded seeks to answer the following questions:

  • Will advanced brain imaging help to support ALS clinical trial enrolment and evaluation? Being able to objectively measure the progression of ALS is critical both for determining someone’s prognosis, and in assessing the effectiveness of clinical trials. With this $200,000 award Dr. Collin Luk, a clinician scientist at the University of Alberta, will study an advanced MRI technique called texture analysis to learn if it could be a biomarker for the onset and progression of ALS. He will collaborate with the Canadian ALS Neuroimaging Consortium (CALSNIC) to validate his findings. If successful, his research could help predict disease progression, aiding clinical trial recruitment and giving researchers an objective tool to better evaluate the effectiveness of promising new treatments. Dr. Luk will also study how this technique may be evolved to expedite ALS diagnosis, which is currently a lengthy and stressful process.
  • Can wearable sensors improve the convenience and quality of clinical trials? Typically, people living with ALS provide their care team with updates on limb function only during clinic visits, which can occur months apart. Dr. Gordon Jewett from the University of Calgary will study if wearing arm and leg sensors could help to track changes in limb movement as people’s ALS symptoms progress, enabling movement data to be collected between clinic visits and improving the quality of movement reporting during clinical trials. This $200,000 grant is supported in partnership with the Mitsubishi Tanabe Pharma Canada, Inc. (MTP-CA) Fellowship Program., which enabled his Fellowship training. It could also make clinical trial participation more convenient, with fewer clinic visits required at a time when new advances in remote evaluation are more important than ever.
  • Can identification of new biological targets represent promising new antibody treatment strategies for ALS? Dr. Silvia Pozzi, currently a postdoctoral fellow in Dr. Jean-Pierre Julien’s lab at the Université Laval CERVO Brain Research Centre, is aiming to develop antibodies that can delay or stop the progression of ALS. Her work will validate how the interaction of two specific proteins can activate a biological pathway that eventually leads to motor neuron death. While she will initially try to prevent this protein interaction in mouse models, if successful, she hopes to determine if similar effects can be seen in humans, which would result in a new target for the development of ALS treatments for clinical trial. This $250,000 grant is supported in partnership with la Fondation Vincent Bourque.

“ALS Canada is incredibly proud to support these promising young researchers. We look forward to each of them becoming the leaders of tomorrow and permanent contributors to the Canadian ALS research and care landscape,” said Dr. David Taylor, VP Research ALS Society of Canada. “The critical areas of clinical care, biomarker identification and development, clinical trial outcome measures, and identification of new treatment targets all contribute to improved care, treatments and quality of life for people living with ALS.”

Generous support from donors and partners have made this competition possible during the COVID-19 pandemic when health research funding is so challenged. In addition to the grants awarded in partnership with la Fondation Vincent Bourque and MTP-CA, all three initiatives have received funding support from provincial ALS Societies across Canada who contribute 40 percent of net proceeds from their local Walk to End ALS fundraising events.

Based on an additional grant competition taking place this fall, more projects receiving funding will be announced in early 2021.

About the ALS Canada Research Program and Canada’s ALS Societies
The ALS Canada Research Program funds peer-reviewed research grants and fosters collaboration amongst Canadian researchers, helping to nurture new ideas and build capacity. As the only dedicated source of funding for ALS research in Canada, the ALS Canada Research Program aims to accelerate research impact by providing funding for the best ALS projects focused on translating scientific discoveries into treatments for ALS.

Collectively through initiatives like the Walk to End ALS, ALS Societies across Canada support the ALS Canada Research Program. ALS Societies across Canada work together to maximize our collective impact and make the greatest difference for people affected by ALS. Our approach as eight independent organizations working in partnership enables us to respond to the variation that exists between provincial healthcare systems, where we each play a role in filling gaps by providing community-based support. ALS Societies advocate federally, provincially and locally on behalf of people and families living with ALS for policy changes that will have a meaningful impact today and in the future.

 

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.

Read more about the ALS Canada Research Program and consider donating today.

 

ALS Canada and Brain Canada award $4.5 million in research funding

The ALS Society of Canada (ALS Canada), in partnership with Brain Canada, today announced $4.5 million in funding for nine new ALS research projects. This means that since the Ice Bucket Challenge became a social media phenomenon in 2014, nearly $20 million has been invested in Canadian ALS research at a time when it has the potential to make the greatest impact.

ALS, or amyotrophic lateral sclerosis, is a disease that gradually paralyzes the body, leaving people without the ability to move, talk, swallow and eventually breathe. Most people die within two to five years of being diagnosed with ALS because the disease has no effective treatment or cure. However, ALS research has advanced to a point that many ALS research experts believe effective treatments are now a matter of ‘when’ not ‘if.’

“We hear often from people and families living with ALS that the promise of research discovery is something they can be hopeful about. The challenge is that research takes time, which is exactly what people living with ALS don’t have – and why the Ice Bucket Challenge has been such a game-changer,” said Tammy Moore, CEO of ALS Canada. “Because of the increased funding that the Ice Bucket Challenge has made available, we have been able to make more significant research investments than ever before. We are grateful to Canadians who donated to the Ice Bucket Challenge, to our ALS Society partners across the country and to Brain Canada and the federal government’s Canada Brain Research Fund for making this research investment possible.”

“Brain Canada’s partnership with ALS Canada has enabled greater investment in ALS research, which will in turn accelerate progress towards the development of effective treatments,” said Inez Jabalpurwala, President and CEO, Brain Canada Foundation. “In addition, the discoveries that will result from this research funding have the potential to inform how we approach other neurodegenerative diseases with similar underlying mechanisms.”

The nine projects include two large-scale, multi-year team initiatives – one of which is using stem cell technology to better understand and potentially treat ALS, while the other is studying in a new way the gene most commonly linked to ALS development – and seven smaller studies that enable investigators to explore out-of-the-box research.

“Five years ago, the breadth of ALS research we are funding today would not have been possible simply because we didn’t know enough about the disease to be able to ask the kinds of questions that today’s researchers are investigating in their work,” said Dr. David Taylor, Vice President of Research at ALS Canada. “The fact that we now have the ability to explore ALS from different angles reflects the growing body of knowledge about the disease and the increasing likelihood of effective treatments being developed.”

A research team led by Dr. Guy Rouleau of McGill University and the Montreal Neurological Institute has been awarded $2.2 million to study motor neurons and astrocytes created from people living with different forms of ALS via stem cell technology. Dr. Rouleau and his team, which includes other collaborators at McGill University and the Montreal Neurological Institute as well as Université de Montréal and Université Laval, will study the biology of these stem cells to determine if their characteristics in the laboratory can represent different forms of human disease and further develop them as a potential screening mechanism for therapeutics. The tools and tests created in this project will be valuable for both Canadian and global ALS researchers as new resources to understand the disease and find new ways to treat it.

A research team led by Dr. Janice Robertson of University of Toronto has been awarded $1.6 million to understand whether the most common genetic abnormality in ALS, which occurs in the C90RF72 gene, causes or contributes to the disease through a loss of the gene’s normal biological function. The majority of the research community is focusing on how the genetic abnormality in C9ORF72 might lead to an extra, toxic function, but in this five-year study, the team – which includes other collaborators at the University of Toronto as well as Sunnybrook Health Sciences Centre, McGill University and the Montreal Neurological Institute, and the University of British Columbia – will comprehensively analyze the potential damage to motor neurons lacking C9ORF72, and examine if both loss and gain of function mechanisms combine to cause ALS. This knowledge could fundamentally alter how therapeutics are developed for common forms of ALS as well as frontotemporal dementia (FTD), which often occurs with ALS.

Other projects that have been awarded $100,000 each in funding are:

All of the research projects were selected through a competitive peer review process, regarded as the international benchmark of excellence in assessing projects for research funding. The peer review process engages a panel of international experts in ALS and other neurodegenerative diseases in evaluating and ranking all proposed research projects based on their scientific merit and on the potential to most quickly advance the field of ALS research in order to develop effective treatments. All aspects of the peer review process are executed in full partnership with Brain Canada, whose funds are provided through a partnership with Health Canada known as the Canada Brain Research Fund.

Approximately 1,000 Canadians are diagnosed with ALS each year. At any time, there are approximately 2,500 to 3,000 people living with the disease in Canada, and the average cost of caring for one person with ALS is between $150,000 and $250,000. Every day, two to three Canadians will die of ALS.

About Canada’s ALS Societies
ALS Societies across Canada fundraise on a regional basis to provide services and support to people and families living with ALS and to contribute to the funding of the ALS Canada Research Program. The ALS Canada Research Program funds peer-reviewed research grants and fosters collaboration amongst Canadian researchers, helping to nurture new ideas and build capacity. ALS Societies advocate federally, provincially and locally on behalf of people and families living with ALS for better government support and access within the healthcare system.

About Brain Canada and the Canada Brain Research Fund
Brain Canada is a national non-profit organization headquartered in Montreal, Quebec, that enables and supports excellent, innovative, paradigm-changing brain research in Canada. For more than one decade, Brain Canada has made the case for the brain as a single, complex system with commonalities across the range of neurological disorders, mental illnesses and addictions, brain and spinal cord injuries. Looking at the brain as one system has underscored the need for increased collaboration across disciplines and institutions, and a smarter way to invest in brain research that is focused on outcomes that will benefit patients and families. Brain Canada’s vision is to understand the brain, in health and illness, to improve lives and achieve societal impact.

The Canada Brain Research Fund is a public-private partnership between the Government of Canada and Brain Canada, designed to encourage Canadians to increase their support of brain research, and maximize the impact and efficiency of those investments. Brain Canada and its partners have committed to raising $120 million, which is being matched by Health Canada on a 1:1 basis for a total of $240 million. For more information, visit www.braincanada.ca.

The generosity of Canadians has helped three early-career researchers to make ALS the focus of their work in the country’s labs and academic institutions. The research funding, which totals more than $1 million, has been awarded through the ALS Canada Research Program and Brain Canada as a result of money raised through the Ice Bucket Challenge.

Canada is home to many world-class ALS researchers who have played a significant role in landmark discoveries about the disease. Ensuring that our country continues to have a strong community of talented ALS researchers is the goal of the research funding, which supports senior postdoctoral trainees as well as recently hired junior faculty members to secure or maintain a faculty job in Canada. Recipients of this funding are all pursuing forward thinking, high-impact ALS research aimed squarely at helping ALS Canada to achieve its vision of making the disease treatable, not terminal. Furthermore, this research will have a broader impact on our understanding of other neurodegenerative diseases.

2016 marks the second year this particular research program has been funded – it was introduced in 2015 following the Ice Bucket Challenge and provides young investigators with the financial stability to pursue their studies in ALS research at the Assistant Professor level. Without this type of funding, it would be very difficult for ALS research to be a viable area for young Canadian researchers to pursue within our country’s borders.

Partnership with Brain Canada (with the financial support of Health Canada) and funds from the ALS Ice Bucket Challenge bolstered the implementation of this new program and allowed for funding to support the early careers of three promising young ALS researchers from a very strong pool of applicants. By the end of 2016, $20 million in research funding will be awarded through the ALS Canada Research Program as a result of the Ice Bucket Challenge.

Please read on to learn more about the recipients of the 2016 ALS Canada-Brain Canada Career Transition Award.

Dr. Jeehye Park
Assistant Professor, Department of Molecular Genetics
Hospital for Sick Children, Toronto, ON
Title: Characterization of MATR3 mutations associated with ALS
$315,000 over three years

Dr. Park has made significant contributions to neurodegenerative disease research since the beginning of her career. During her PhD work in South Korea with Dr. Jongkyeong Chung, Dr. Park discovered a key connection between two Parkinson’s disease pathways that had a major impact on the field and was published in the elite scientific journal Nature. She subsequently pursued postdoctoral research at Baylor College of Medicine under the guidance of Dr. Huda Zoghbi, where Dr. Park helped to create a network of laboratories with expertise across different animal models to screen for treatments for the neurodegenerative disease spinocerebellar ataxia 1, which led to yet another paper in Nature. Her research then led her to study RNA binding proteins (RBP), where she not only developed a new tool to study them, but became interested in the multiple RBPs that are linked to ALS.

In her lab, Dr. Park will examine how abnormalities in RBPs – in particular, one called Matrin 3 (MATR3) – can lead to ALS. MATR3 was discovered to be a genetic cause of ALS in 2014 and has yet to be studied in any detail. By creating the first-ever cell, fruit fly and mouse models of MATR3, Dr. Park will learn both about the functions of MATR3 and how mutations can confer motor neuron degeneration. Dr. Park will then search for other genes that may increase or reduce mutant MATR3 toxicity in both human cells and fruit fly models to find potential targets for treatment, and follow up with the most promising candidates being tested in the new MATR3 mouse models with an aim to eventually move them forward translationally into the clinic.

As a member of the Canadian ALS research community, Dr. Park will be able to integrate the knowledge gained about MATR3 with the work of others here and around the world as yet another puzzle piece in understanding ALS. By focusing the early stages of her independent career on a less understood ALS mechanism, she intends to find connections between MATR3 and more prominently studied RBPs like TDP-43 and FUS to ultimately unravel key mechanisms in the development of ALS, as well as new targets to treat the disease.

Dr. Veronique Belzil
Postdoctoral Fellow
Mayo Clinic, Jacksonville, Florida
Supervisor: Dr. Leonard Petrucelli
Title: Discovery of transcriptomic biomarkers and epigenetic therapeutic targets for c9ALS and sALS
$110,000 over two years; eligible for an additional $315,000 over three years

Dr. Belzil began her research career as a PhD student at the Université de Montréal under the guidance of world renowned geneticist and Director of the Montreal Neurological Institute and Hospital, Dr. Guy Rouleau. During this time, Dr. Belzil pursued a better understanding of the genetics behind familial/hereditary ALS and led or contributed to more than 20 manuscripts, an amazing accomplishment for a graduate student.

For the past four years, Dr. Belzil has spent her postdoctoral studies pursuing the complex understanding of how alterations in genetic regulation may lead to ALS not just in certain familial forms, but in sporadic ALS that makes up 90-95% of cases. She has led or contributed to a large number of important discoveries.

The high impact work that Dr. Belzil has been pursuing during her postdoctoral training translates very well into an expanded program for an independent laboratory and she aims to continue to tackle these mechanisms as an Assistant Professor. The program she has outlined is also designed to apply the knowledge of these discoveries into a strategy to develop novel and exciting new treatments for ALS that would be based on an intricate understanding of the disease.

Dr. Petrucelli and a mentoring committee at Mayo Clinic are committed to assisting Dr. Belzil to not only reach her goal of becoming an independent investigator at a Canadian institution, but to become an internationally recognized leader in translational ALS research.

Dr. Kessen Patten
Assistant Professor, Genetics and Neurodegenerative Disease
Centre INRS–Institut Armand-Frappier, Laval, QC
Title: Pathogenic mechanisms of C9ORF72 repeat expansion in ALS and development of therapeutics
$315,000 over three years

Dr. Patten started his research career as a PhD student at the University of Alberta under the supervision of Dr. Declan Ali in 2004. There he trained in electrophysiology, cell biology and imaging using zebrafish as a model to study neurodevelopment. After publishing several manuscripts on his discoveries and receiving multiple awards, including national recognition for the outstanding quality of his PhD thesis, Dr. Patten pursued a postdoctoral fellowship in Montreal with Drs. Florina Moldovan and Pierre Drapeau. During that time, among other achievements, he developed zebrafish models of human disease including ALS, and used those models to develop a high-throughput method for drug discovery. This procedure was then used by Dr. Patten in the identification of pimozide as a lead compound in a translational pipeline that has led to a multi-centre Canadian clinical trial to start in 2017. The trial is being supported by the first ALS Canada-Brain Canada Arthur J. Hudson Translational Team Grant that was awarded in 2014.

In the initial years of his independence as an Assistant Professor, Dr. Patten will pursue the development and use of zebrafish models of the most common genetic cause of ALS, C9ORF72, as well as use of the high-throughput screening method to examine more promising compounds for further examination. As a key addition to his work, he has formed strong collaborations with international ALS experts with proficiency in developing motor neurons from induced pluripotent stem cells (iPSCs) that will undoubtedly strengthen the ability to translate zebrafish discoveries to the clinic via the use of human cells.

Dr. Patten has been a regular attendee at the ALS Canada Research Forum for the past several years and has formed relationships with a number of other investigators in the community. Combined with multiple other Canadian investigators using ALS model zebrafish, C. elegans worms, Drosophila fruit flies, mice, rats and iPSC derived motor neurons, Dr. Patten will strengthen this country’s expertise on forming a pipeline of drug discovery that can efficiently reach the clinic and ultimately help make ALS a treatable, not terminal disease.

Earlier this week researchers announced that the gene NEK1 has been found to play a significant role in the development of amyotrophic lateral sclerosis (ALS). This landmark discovery is the result of an 11-country research collaboration that was funded through the Ice Bucket Challenge. The research team included 3 Canadians, one of whom was directly funded by the ALS Society of Canada for their work during this discovery.

This finding will trigger future studies that focus specifically on the NEK1 gene. It is a compelling example of how donor dollars can lead to landmark research results, and speaks to the ongoing need to fund ALS research. In Canada, the Ice Bucket Challenge resulted in more than $20 million in research funding through ALS Canada – more than 10 times the annual research investment the organization has typically been able to make. Your support of further research will support further advances that will make ALS a treatable, not terminal, disease.

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A large consortium of researchers from six countries have definitively identified a new ALS gene called TANK-binding kinase 1 (TBK1) by performing a rigorous study that involved sequencing of more than 2800 people with ALS compared with more than 6000 control samples. Canadian researcher Dr. Guy Rouleau, Director of the Montreal Neurological Institute and his colleague Dr. Patrick A. Dion, along with current Tim E. Noël (ALS Canada) Postdoctoral Fellowship recipient Dr. Claire Leblond are authors on this landmark manuscript, recently published early online for the top journal Science.

The discovery of TBK1 as an ALS gene is interesting because it not only provides new pathways to focus on, but it links quite closely with a number of other previously discovered ALS genes. In particular, TBK1 is closely related to the ALS gene optineurin (OPTN) and a process of recycling things inside cells called autophagy. Both OPTN and autophagy have been studied, but this new discovery places increased importance on their potential roles for understanding the disease. TBK1 is also an important part of an inflammatory mechanism that will now certainly be explored in more depth.

This study is another example of how the ALS research community is working collaboratively to drive the field forward. Typically discovery of a new ALS gene by a single institute or lab requires proof that it is important in the disease by confirmation in other sets of genetic samples studied elsewhere. In this case, the large number of combined sample sets and world renowned scientists involved confirms TBK1 as a bona fide gene/protein to focus further work on. Furthermore, there are still stronger, more advanced techniques that will be employed to hopefully identify even more ALS genes, each which represent targets (puzzle pieces) to study for understanding the disease, but also a potential place where treatments can be targeted.