An announcement of the top-line results from a European phase 3 clinical trial of TUDCA revealed that the primary endpoint of the study was not met as TUDCA failed to demonstrate an ability to significantly alter the progression of ALS as measured by the ALS Functional Rating Scale – Revised (ALSFRS-R). Additional measurements (secondary endpoints) were also unable to demonstrate a benefit of TUDCA. These included an evaluation of effect on survival and key biomarkers including neurofilament light chain (NfL), and several other analyses.
The TUDCA-ALS phase 3 clinical trial was a randomized, double-blind, placebo-controlled study, testing a regimen of four oral capsules (250 mg), twice daily in 336 participants over 18 months. It was an academic (also referred to as investigator-initiated) trial, run by the TUDCA-ALS consortium of UK and European ALS clinicians, which means it was not sponsored by a pharmaceutical company. The consortium has stated that they will be exploring further statistical analyses to better understand if there was any potential benefit of TUDCA in a subgroup of individuals, and it is anticipated that some of this data will be presented in June at the European Network for the Cure of ALS (ENCALS) meeting in Sweden. The ALS Society of Canada will keep the community updated as more information becomes available to share.
TUDCA or tauroursodeoxycholic acid (also referred to as TURSO) is also one of the two drugs compounded in Albrioza (also referred to as Relyvrio and AMX0035), which was recently announced to have failed an effect on primary and secondary outcome measures in a phase 3 clinical trial.
For anyone interested in discussing TUDCA use, it is recommended to speak to your clinician.
Eight projects receive funding to find answers and
fuel discoveries in ALS research
Toronto – In their effort to fund progress and innovation in amyotrophic lateral sclerosis (ALS) research, the ALS Society of Canada (ALS Canada) and Brain Canada are proud to announce the eight recipients of the 2023 Discovery Grants, with a total investment of $1,350,000.
The ALS Canada-Brain Canada Discovery Grants provide the research community with crucial funding for projects focused on identifying causes of ALS, treatments for ALS or related neurological diseases, and avenues to maximize function, minimize disability, and optimize the quality of life for people and their families living with ALS.
“ALS Canada is incredibly grateful for our partnership with Brain Canada and the support of our donors to fund these individuals and the projects they are leading,” says Dr. David Taylor, Vice-President of Research and Strategic Partnerships at ALS Canada. “These projects represent Canada’s best new ideas to impact our current understanding of ALS. Our hope is that discoveries made through this funding will take us closer to effective treatments and, someday, a world free of ALS.”
The Discovery Grant recipients are selected following a rigorous peer-reviewed grant competition that engages an international panel of experts to choose the best work grounded in scientific excellence and with the potential to advance the field of ALS research quickly.
“We take great pride in supporting these eight projects, which are driving forward scientific discovery on a global scale,” says Dr. Viviane Poupon, President and CEO of Brain Canada. “With these funds, the ALS research community is empowered to push the boundaries of innovation, and we eagerly anticipate the remarkable discoveries that will arise from their dedicated efforts.”
Summary of the 2023 ALS Canada-Brain Canada Discovery Grants:
- Can this new way of analyzing brain imaging data help researchers predict and monitor the progression of ALS?
Dr. Mahsa Dadar, McGill University, in collaboration with Dr. Sanjay Kalra, University of Alberta, awarded $125,000 - Can boosting a vital protein in the brain help to slow the progression of ALS?
Dr. Jean-Pierre Julien, Université Laval, in collaboration with Dr. Angela Genge, McGill University, awarded $125,000 - Is this newly discovered tag on TDP-43 key to understanding and treating ALS?
Dr. Dale Martin, University of Waterloo, in collaboration with Dr. Max Rousseaux, University of Ottawa, and Dr. Christine Vande Velde, Université de Montréal, awarded $125,000 - Can these worm models help researchers better understand and even block the spread of ALS pathology?
Dr. Alex Parker, Université de Montréal, in collaboration with Dr. Guy Rouleau, McGill University, awarded $125,000 - How does fatty acid metabolism influence motor neuron health?
Dr. Chantelle Sephton, Université Laval, in collaboration with Dr. Liang Li, University of Alberta, awarded $125,000 - Can investigating protein shapes reveal important puzzle pieces for future treatment strategies?
Dr. Valerie Sim, University of Alberta, in collaboration with Dr. Sumit Das, University of Alberta, and Dr. Sanjay Kalra, University of Alberta, awarded $125,000 - Can a deeper understanding of nerve-muscle connections uncover new ways to treat ALS?
Dr. Richard Robitaille, Université de Montréal, in collaboration with Dr. Sandrine Da Cruz, KU Leuven (Belgium), and Dr. Danielle Arbour, Université de Montréal, awarded $300,000 - Can targeting the stress response within cells lead to more effective therapies for ALS?
In partnership with Dr. Jean-Pierre Canuel Fund – SLA Québec and Brain Canada, Dr. Christine Vande Velde, Université de Montréal, in collaboration with Dr. Jonathan Watts, UMass Chan Medical School (USA), awarded $300,000
Funding for one Discovery Grant was made possible by the Dr. Jean-Pierre Canuel Fund – SLA Québec, who generously contributed $150,000 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.
For more information on these funded projects, visit als.ca.
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.
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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
Quebec researcher receives $250,000 over three years to investigate
gene-based research to treat ALS
Toronto – The ALS Society of Canada (ALS Canada) and Brain Canada are dedicated to a shared mission to advance amyotrophic lateral sclerosis (ALS) research. Together, ALS Canada and Brain Canada are proud to announce the recipient of the 2023 Career Transition Award. Dr. Sahara Khademullah at the CERVO Brain Research Centre at Université Laval will receive $250,000 over a period of three years to support her innovative ALS research.
This award aims to develop the next generation of scientists across various disciplines within basic and clinical sciences, advance knowledge generation and translation in ALS research, and support the recipient in both salary and research costs as they transition into their first faculty position.
“ALS Canada, alongside our partners at Brain Canada, remain committed to investing in early career researchers. These funds are necessary to develop and support the future leaders of ALS research in Canada,” says Dr. David Taylor, Vice-President of Research and Strategic Partnerships at ALS Canada. “Our organizations have been fortunate to support Dr. Khademullah’s work over the past decade, and the resulting discoveries are quite novel and exciting. We are proud to reinforce her successes by helping her to achieve an independent academic position and an opportunity to drive this therapeutically minded research forward towards potential human impact.”
The recipients of the Career Transition Award are chosen by a peer review process based on the merits of the applicant, the quality of their project, the relevance of the work to ALS, and their likelihood for future success. This funding aims to identify and engage rising stars pursuing innovative research in labs and academic institutions in Canada. Successful candidates will expand their research horizons and develop independence to become a future leader in the field.
“At Brain Canada, we firmly believe in the power of investing in early-career researchers to transform lives and shape a better future for individuals and families affected by ALS,” says Dr. Viviane Poupon, President and CEO of Brain Canada. “Our collaboration with ALS Canada holds immense promise, fostering innovation and paving the way for breakthroughs.”
To date, the ALS Canada – Brain Canada partnership has resulted in more than a $25 million investment in a wide range of research projects that have helped further the study of the disease.
Summary of the 2023 Career Transition Award
- Can these gene-based strategies help restore a normal balance in the brain?
Therapeutic validation of gene-based strategies aimed at restoring neuronal KCC2 in ALS
Dr. Sahara Khademullah, supervised by Dr. Yves De Koninck, at the CERVO Brain Research Centre, Université Laval, awarded $250,000
The Career Transition Award competition is funded through a matching contribution from 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.
For more information on this funded project, visit als.ca.
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.
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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
Funding for first two projects to total $200,000
Toronto – Amyotrophic lateral sclerosis (ALS) is a disease that can progress with startling swiftness. The ALS Society of Canada (ALS Canada) recognizes the need to move quickly in funding promising research and, with a new grant program, intends to condense lengthy processes between the selection and funding of research projects while also expanding the scope of eligible projects globally.
The ALS Canada Acceleration Grant Program was created with the goal of propelling an exciting line of research forward seamlessly, without the need for lengthy applications, reviews, and administrative phases. It relies on a unique form of global peer review, where experts in the field proactively determine the best work to support rather than reactively evaluating applications. This could potentially shift the ability to fund some of the most promising projects anywhere in the world from taking years to find funding to just a few short months.
“Researchers around the globe are working tirelessly to uncover new discoveries that will advance our understanding of ALS and develop effective treatments for the disease,” says Dr. David Taylor, Vice-President of Research and Strategic Partnerships, ALS Canada. “To have an impact on people currently living with the disease, we must move faster to fund research and look at promising, innovative ideas beyond our borders to fuel discoveries that will lead to a world free of ALS.”
Nearly 4,000 Canadians live with ALS and know firsthand the urgency to fund new treatments. After receiving a diagnosis, eighty per cent of people will die from the disease within two to five years.
“We are incredibly proud to be bringing forward an innovative opportunity to fund new projects at an expedited speed with the ALS Canada Acceleration Grant Program,” says Tammy Moore, CEO, ALS Canada. “ALS is a devastating disease; by bringing together global leaders in ALS to identify and support projects from around the world, we look to fund research that will change the outcome of an ALS diagnosis and have an impact on future ALS discoveries.”
The 2023 ALS Canada Acceleration Grant Program is pleased to provide a total of $200,000 in funding to two projects over one year.
Summary of 2023 Acceleration Grants
Epidemiological study of ALS in Colombia and Ethiopia
Mónica Povedano, Hospital de Bellvitge-IDIBELL (Barcelona, Spain), awarded $100,000
Professor Mónica Povedano leads a collaborative effort with colleagues in Colombia and Ethiopia. The goal of the study is to enhance clinical and research infrastructure in South America and Northern Africa.
With the support of an ALS Canada Acceleration Grant, Professor Povedano is conducting the first-ever comparative study in ALS across different ethnic backgrounds alongside data from European individuals and aims to strengthen research capabilities in these regions. This study is significant in the Canadian context as it addresses the underrepresentation of global populations in ALS research and will contribute to our overall understanding of ALS.
Beyond learning about people living with ALS in these regions, Professor Povedano’s work will create the infrastructure to conduct clinical trials. This will potentially provide beneficial experimental therapeutic access to these countries and accelerate opportunities to discover treatments that could be available to Canadians if successful.
Unravelling early ALS: An EEG-MRI investigation of presymptomatic C9ORF72
Dr. Stefan Dukic, postdoctoral researcher at University Medical Centre Utrecht (The Netherlands), awarded $100,000
Dr. Stefan Dukic is developing a new method of sub-grouping people with ALS using a resting-state electroencephalogram (EEG). This technique measures the brain’s electrical activity through simple electrodes placed on the scalp. Dr. Dukic and his colleagues have shown that EEG can capture abnormal patterns of motor and cognitive brain activity in ALS and potentially help to predict how someone’s ALS will progress.
The Acceleration Grant funding will provide support to speed up this work, recruit more participants and to explore a novel combination of EEG with Magnetic Resonance Imaging (MRI). This combined tool could be much more powerful in understanding the different forms of ALS, potentially paving the way toward personalized medicine and more effective clinical trials of experimental treatments. The project will also focus on individuals who may have underlying disease processes but have not yet experienced symptoms, suggesting that it could help to someday treat earlier or even prevent ALS from happening.
Funding for Dr. Dukic’s The Stevie Fever For ALS Foundation-ALS Canada Acceleration Grant was made possible through a partnership with The Stevie Fever For ALS Foundation, which generously contributed $100,000 to ALS Canada.
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 The Stevie Fever For ALS Foundation
The Stevie Fever for ALS Foundation is a registered Canadian charity created in loving memory of Steve Daly – affectionately known as ‘Stevie’ – a vibrant soul, devoted husband, and father of four. His spirited zest for life echoed loudly, even in the face of adversity. In 2022, at the age of 50, Stevie passed away after valiantly battling ALS for nine months, leaving an indelible mark on all who knew him. To pay tribute to Stevie’s remarkable life and propel the fight against ALS, his family established the Stevie Fever for ALS Foundation. This registered Canadian charity is on a mission to not only raise awareness but also generate crucial funds for groundbreaking ALS research and compassionate care. Since Steve’s diagnosis in 2021, the Stevie Fever Foundation has rallied an astounding $225,000+ in his name. This impressive sum serves as a testament to the love and support pouring in from friends, family, and the community, all united in the common cause of advancing research and providing care for those affected by 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
Updated on: January 31, 2024
Pharmaceutical company Ferrer has announced topline results from the ADORE (ALS Deceleration with ORral Edaravone) clinical trial in ALS. The Phase 3 study, conducted in Europe, investigated the efficacy of an oral formulation of edaravone (FAB122) over a 48-week period. The study did not meet primary or key secondary endpoints.
The results indicate no significant benefit from daily treatment with the oral edaravone formulation (FAB122) compared to placebo, as measured by changes in the ALS Functional Rating Scale-Revised (ALSFRS-R) score. Long-term survival, measured by the Combined Assessment of Function and Survival (CAFS), also showed no improvement. Despite being safe and well-tolerated, the lack of efficacy led to the company’s decision to also conclude the open-label extension study.
Edaravone is currently approved by Health Canada in two different formulations: RADICAVA® IV administered through intravenous (IV) infusions, and RADICAVA® Oral Suspension, an oral suspension taken by mouth or via a feeding tube. Both formulations are distributed by the pharmaceutical company Mitsubishi Tanabe Pharma Canada.
The ALS Society of Canada will work with clinicians and other key experts to better understand the implications of these results for Canadians living with ALS and remains committed to keeping the community updated as more information becomes available.
A study published today in Muscle & Nerve on the long-term data from the CENTAUR clinical trial and open-label extension shows that AMX0035 has promise for extending survival in people living with ALS.
The results demonstrated that early treatment with AMX0035 resulted in longer survival for people living with ALS on the therapy during the Phase 2 clinical trial compared to those who only received it during the open-label extension. The study, sponsored by the pharmaceutical company Amylyx, followed participants for up to three years from the CENTAUR trial. This data complements the study published in September 2020 in the New England Journal of Medicine, which demonstrated AMX0035 was safe and slowed the rate of decline in people living with ALS compared to those on placebo.
ALS Canada continues to actively engage with Amylyx to advocate for the company to include Canada in their future plans.
You can read more about the results here.
A study published today in the New England Journal of Medicine shows that AMX0035, an experimental therapy, has promise for slowing the progression of ALS.
AMX0035 is an oral drug that contains two small molecules, which when combined, aim to preserve motor neuron health in people living with ALS, thereby delaying the progression of symptoms. Results from the Phase 2 CENTAUR clinical trial, sponsored by the pharmaceutical company Amylyx, showed that AMX0035 was safe and slowed the rate of decline in people living with ALS on the therapy compared to those on placebo. All clinical trial participants were measured and assessed over 24 weeks primarily based on the ALS Functional Rating Scale-Revised (ALSFRS-R). The preliminary data also showed potential benefits in areas including measures of muscle strength, breathing and hospitalization frequency. In addition, many participants in the trial were also already taking riluzole and Radicava (edaravone), meaning the effects observed were on top of that for the current standard of care.
ALS Canada has been in contact with Amylyx for many years and has continually advocated to have the company include Canada in their immediate future plans. We remain committed to bringing forward the needs of people living with ALS and to engage in meaningful dialogue that demonstrates the urgent need for access within the ALS community. The positive results from this study represent another step forward in developing new therapies that will one day transform the reality of living with ALS.
You can read more about the results here.
July 9, 2020 – A study published today in the New England Journal of Medicine shows that an experimental drug, called tofersen, has promise for treating a genetic form of ALS caused by mutations in the gene SOD1.
Results from the Phase 1/2 clinical trial, sponsored by the pharmaceutical company Biogen, revealed that tofersen was safe and able to lower levels of the toxic SOD1 protein thought to contribute to disease progression. The preliminary data also showed potential for slowing loss of function and preserving neuronal health. Canadian researchers have played a key role in the studies so far as Dr. Angela Genge (Montreal) and Dr. Lorne Zinman (Toronto) have been involved since the beginning. The positive results from this study led to launch of the Phase 3 VALOR study that will further test the safety and effectiveness of tofersen and is ongoing at a number of sites globally, including at four Canadian sites: Calgary, Edmonton, Toronto and Montreal (Neuro).
If the Phase 3 trial is successful and tofersen moves forward as a new and proven treatment for ALS, it will directly benefit the small percentage of ALS patients who have a mutation in the SOD1 gene. However, the lessons learned from this pivotal study will help researchers when developing new approaches to treating all forms of ALS.
TORONTO – After a rigorous competition, the ALS Society of Canada today announced the recipients of the 2019 research funding grants. Through the ALS Canada Research Program, the only dedicated source of ALS research funding in Canada, nearly $1.4 million will be invested in leading-edge ALS research that will further the study of the disease. For a terminal disease like ALS, that gradually paralyzes people because the brain is no longer able to communicate with the muscles of the body typically moved at will, transformative research is needed to help accelerate understanding of disease progression and provide pathways for future therapies. Continued investment in ALS research fuels the scientific discoveries that will bring hope for a future without the disease.
More than $850,000 is being invested in ten Project Grants, and $540,000 is being invested in six Trainee Awards that will provide momentum for the next generation of promising ALS researchers. It is because of the generosity of ALS Societies, individual donors and community-based efforts, including 40 per cent of net proceeds from the Walk to End ALS fundraising events that take place across the country, that these innovative research projects aimed at providing a greater understanding of the onset and progression of ALS can happen.
“The spectrum of Canadian ALS research at the moment is incredibly rich and deep, touching on so many aspects of the disease,” said Chris Loudon, who was diagnosed with ALS in 2015 and had the opportunity to observe the peer review process that resulted in the funding of the 10 project grants. “I am impressed by the passion of the ALS research community and the dedication of the ALS Canada Research Program on furthering such vital research. As someone living with ALS, I am more hopeful than ever that ALS can and will be beat.”
“ALS is a complex disease and while we’ve made amazing advancements in recent years towards understanding of the disease processes, there is still a lot to learn,” said Dr. David Taylor, VP Research, ALS Society of Canada. “By focusing on multiple diverse projects, new avenues of understanding and treatment can be explored. Everything we support is with our vision of a future without ALS.”
Funding partners for the ALS Canada Research Program’s 2019 grants and awards are the provincial ALS Societies across Canada, Orangetheory Fitness, La Fondation Vincent Bourque, and the Brain Canada Foundation (with financial support from Health Canada) which provided funds to support three of the trainee awards as part of a commitment made after the Ice Bucket Challenge.
“The members and franchisees of Orangetheory Fitness are extremely passionate about Augie’s Quest’s vision to help the ALS community live longer, more vibrant lives. We are proud to partner with the ALS Canada Research Program to dedicate funds from the 2019 Augie’s Quest initiative in support of Canadian ALS research,” said Blake MacDonald, President, OTF Canada Inc. “We hope to continue striving for a cure, while supporting scientific discovery that will one day improve the lives of individuals and families who are affected by this rare and yet, terrible disease.”
The projects funded will answer questions that will help to accelerate research discovery contributing to the development of potential ALS therapies:
- How do unique protein interactions explain TDP-43 behaviour in different people with ALS? $100,000 awarded to Dr. Mohan Babu at the University of Regina.
- How do environmental marks on RNA play a role in how ALS is caused? $100,000 awarded to Dr. Patrick Dion at the Montréal Neurological Institute at McGill University.
- Does prior exposure to common viruses influence ALS onset and disease progression? $100,000 awarded to Dr. Matthew Miller at McMaster University.
- Does a substance in gut or oral bacteria influence the disease course of ALS? $100,000 awarded to Dr. Minh Dang Nguyen at the University of Calgary.
- Can a new ALS mouse model provide important information for understanding and treating ALS? $100,000 awarded to Dr. Jeehye Park, at the Hospital for Sick Children (SickKids) Research Institute.
- Are the same faulty nerve-muscle connections in ALS mice also occurring in humans? $100,000 awarded to Dr. Richard Robitaille at the Université de Montréal.
- What is the role of the annexin A11 gene in ALS disease processes? $100,000 awarded to Dr. Peter St. George-Hyslop at the University of Toronto.
- Can advanced technology reveal the role of multiple cell types affecting ALS in humans? $98,400 awarded to Dr. Stefano Stifani at the Montréal Neurological Institute at McGill University.
- Can earlier palliative care consultation improve patient and caregiver quality of life? $55,437 awarded to Dr. Jocelyn Zwicker and Dr. Christine Watt at the Ottawa Hospital.
- Can speech-recognition technology help diagnose ALS? In partnership with Orangetheory Fitness, $100,000 awarded to Dr. Yana Yunusova at the Sunnybrook Research Institute.
- Can an animal model provide new insights into the formation of stress granules? $75,000 awarded to Alicia Dubinski, a PhD student in Dr. Christine Vande Velde’s lab at the Université de Montréal.
- What is the role of a newly discovered protein in ALS? La Fondation Vincent Bourque | ALS Canada – Brain Canada Ph.D. Studentship of $75,000 awarded to Myriam Gagné, a PhD student in Dr. Christine Vande Velde’s lab at the Université de Montréal.
- Is the loss of normal function of C9ORF72 in a particular cell type a key driver of ALS disease processes? $75,000 awarded to Rahul Kumar, a PhD student in Dr. Peter McPherson ‘s lab at the Montréal Neurological Institute at McGill University.
- Is an experimental drug that can prevent abnormal protein behaviour in ALS already out there? La Fondation Vincent Bourque | ALS Canada – Brain Canada Ph.D. Studentship of $75,000 awarded to Marc Shenouda, a PhD student in Dr. Janice Robertson’s lab at the University of Toronto.
- Could newly discovered tags on TDP-43 protein explain its abnormal behaviour in ALS? In partnership with Brain Canada, $75,000 awarded to Terry Suk, a PhD student in Dr. Maxime Rousseaux’s lab at the University of Ottawa.
- Can new understandings about nuclear speckles lead to new treatment options for ALS? $165,000 awarded to Dr. Ulises Rodríguez Corona, a post-doctoral student in Dr. Marlene Oeffinger’s lab at Institut de recherches cliniques de Montréal (IRCM).
The funding of the 16 research projects followed a competitive peer-review process, which engaged global ALS experts to identify projects grounded in scientific excellence and with the potential to most quickly advance the field of ALS research. The peer review was observed by people who have personal experience with ALS.
About the ALS Canada Research Program and Canada’s ALS Societies.
The ALS 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.
This article was originally posted on the Motor Neurone Disease Association research blog on January 15, 2019. Thank you to the author Dr. Nicholas Cole, Head of Research, as well as the Motor Neuron Disease Association for giving ALS Canada permission to re-post this content.
There has recently been a flood of news stories on the outcomes of the Australian Phase 1 clinical trial investigating Copper ATSM (CuATSM) which is a small man-made compound that can selectively deliver copper to cells. The results were first presented at our International Symposium in Glasgow back in December.
MND is a terrible disease and anyone affected by it is looking for good news. We really hope that CuATSM will provide a new treatment for MND that is going to have a positive effect on people’s disease progression.
However, CuATSM is not yet at a stage where a clinician can prescribe it as a treatment. Drug development is a long journey, where any drug has to pass important rigorous checks before approval as a medicine. This trial is an important ‘first’ in the drug development process.
The reported Phase 1 trial was only designed to test the safety and dosage of the compound CuATSM and the researchers have demonstrated that this drug is safe to consume. Historically, many promising drugs have failed at this stage, and so to get to this stage and pass is vital for its success and further development. The next and important stage is to perform the trial on a larger number of people and really see if it can alter the disease progression.
We have a duty to people with MND, their families and carers to be as accurate and factual on these stories as possible. We must be realistic and not over-interpret promising early data.
Although the data showed the average rate of decline of the ALSFRS-R (ALS Functional Rating Scale – revised) score is lower than observational control cohorts, is it right to claim a 70% slowing of disease from a small open label Phase 1 study, and can this be substantiated? To find this out we must take a deep dive into the details of the actual study and its data.
The Study
Firstly, the trial was a Phase 1 trial (see our clinical trials info sheet) which is primarily designed and performed to find a safe dose of medicine (monitoring side effects) and is not designed to determine efficacy (effectiveness against the disease). These trials normally only use a small number of participants.
The primary objective was to define a recommended dose for Phase 2, the dose that can be taken with minimum side effects. This is then the dosage, that can then be tested in a larger clinical trial, which will also look at effects on disease symptoms.
The study was an ‘open-label’ trial. Open-label means that everyone involved, including the participants, know that they were receiving the drug rather than being given a placebo. Whether a trial is open-label or ‘blind’ is a crucial consideration, the consensus being a double blind trial is more reliable when determining drug effectiveness. Double blind means that neither the participant nor clinician know whether the patient is receiving the trial drug or a placebo.
In the study, 32 participants (29 finished the trial) were split into five ‘dosage’ groups (with no more than seven participants in any one group) where each dosage group was given a different amount of CuATSM (either 3, 12, 36, 72 or 144mg/day) to determine the highest safe amount of the drug. Based on safety and pharmacokinetic (PK) data (to determine how the body handles the substance) the recommended dose was determined as 72 mg/day.
During the trial, each person was given an assessment of disease severity by ALSFRS-R score, breathing function by forced vital capacity (FVC), and cognitive function by Edinburgh Cognitive and Behavioural (ECAS) score. These were measured at the start (baseline) and after the first and sixth month of daily CuATSM treatment. Sniff nasal pressure (SNP) and the urinary p75 biomarker of disease progression was also measured but this data is not reported.
The reported data (ALSFRS-R and FVC) comes from the 72mg/day group. Their averaged data was compared to a historical control group of 8,600 people with MND in the PRO-ACT database.
When compared to the historical cohort over a six-month period the results from the 72mg/day group showed:
- improved lung function (+1.1 % predicted/month vs -2.24 % predicted/month for FVC)
- improved cognitive ability (+10 points vs no changes* for ECAS score)
- slowed rate of averaged disease progression (-0.29 points/month vs the expected -1.02 points/month on ALSFRS-R)
* Some improvements in cognitive function have been reported in control subjects in other studies. This may be related to participants becoming more familiar with the tests.
Taken at face value it is clear to see why the data looks and sounds promising. However, this was over a relatively short time period (most large-scale trials last for at least 12 months) with an open label study with very small number of participants. When a test is performed only in a small number of people you can sometimes get a result that is a little misleading. Therefore, we must exercise optimistic caution in reading too much into the data at this point.
Reasons for caution in interpreting the results.
- This was a trial with only a small number of participants. MND is a heterogeneous disease, meaning every person’s disease is different. Disease progression is non-linear and can slow, pause or even reverse for short periods in people with MND. The trial was performed for 24 weeks. It has been shown that, in control datasets from people with MND, over a six-month period, one quarter of all participants enrolled in a trial will show periods of slow or stopped decline in ALSFRS score even though they are not being given any active compound. If a few of these events occur in a trial with very small numbers of participants involved, the data can be severely affected in either direction. For example, if there is just one person whose progression has a natural (unrelated to the treatment) periods of slowing of progression, (e.g. they ‘appear’ stable on the drug) then in a small study there will not be enough other participants to smooth out this single datapoint.
- The trial was ‘open label’, meaning the participants know they were taking the drug, opening the data to the very real but not really understood ‘placebo’ effect. The open label means that both the participant and the clinician, nurse, (person recording the ALSFRS or ECAS score) knows that the participant is taking the real drug, all of which changes their outlook and approach (consciously or otherwise).
- Historical controls were used. This gives only an average, effectively ‘virtual’ person with MND to compare the experimental data with. We know from other clinical studies in the past, such as diaphragm pacing, that the use of historical control data can be highly misleading.
- The attention-grabbing headline ‘the copper-delivery drug CuATSM slowed the progression of the disease in patients by 70 per cent’ is really overinterpretation and overstatement given the potential for progression variation with only a small number of participants in the group. Also, the points scale of the ALSFRS does not directly correspond to life expectancy. The figure comes from the difference in final ALSFRS-R score in the 72mg/day group compared to the historical controls used (-0.29 points/month vs the expected -1.02 points/month taken from the historical controls).
What next?
What is needed now is to test CuATSM in a follow up trial with a large number of people. The great news is that this is going to happen. Further work is needed and is planned in a randomized, placebo-controlled Phase 2 clinical trial of CuATSM. The Phase 2 trial begins later this year in Sydney and Melbourne.
We really hope that these results from this small dose-determining trial can be confirmed and that CuATSM will provide an effective treatment for MND.
It is positive to see such a clear example of how the money raised by charity supporters can effectively drive research to the point of a Phase 1 clinical trial for this compound which has been in development for some time. Everyone involved, (from the school child selling cupcakes, the walkers, swimmers, to the marathon runner, patients, families, supporters, nurses, clinicians and volunteers) should be congratulated on this achievement.
Please note: CuATSM is a specialised compound and is not the same as taking copper supplements, which can be poisonous in high doses.
2018 funding competition with Brain Canada results in $720,000 for six trainee awards – more trainee awards in a single year than ever before
TORONTO December 13, 2018 – The ALS Society of Canada (ALS Canada), together with Brain Canada, today announced $720,000 in funding for six new trainee awards, thus completing the last of the $20 million research partnership with the Brain Canada Foundation (with financial support from the Government of Canada) following the Ice Bucket Challenge. ALS Canada is also pleased to partner with La Fondation Vincent Bourque who will be providing financial support for one of these awards. Each grant has been awarded with the aim of sustaining high-quality Canadian ALS research by providing salary support for the next generation of ALS researchers currently pursuing PhDs or postdoctoral research positions.
At a given time, approximately 3,000 Canadians are living with ALS, a terminal disease that gradually paralyzes people because the brain is no longer able to communicate with the muscles of the body that we are typically able to move at will. As the connection with the brain and muscles breaks down, someone living with ALS will lose the ability to walk, talk, eat, swallow, and eventually breathe. Each year approximately 1,000 Canadians die from ALS. With no cure and few treatment options available that have a significant impact on the progression of the disease, 80 per cent of people with ALS die within two to five years of diagnosis.
“This was a tremendous opportunity to collaborate with Brain Canada and La Fondation Vincent Bourque to support more trainees in a given year than ever before,” said Dr. David Taylor, VP Research, ALS Society of Canada. “For more than a decade the ALS Society of Canada has funded exceptional students and postdoctoral candidates with the goals of advancing key Canadian ALS research and creating the next generation of dedicated ALS researchers in the country. It’s a program that has impact today and for many years to come.”
“Since 2015, Brain Canada’s much-valued partnership with the ALS Canada Research Program has resulted in a $20-million investment in ALS research. The funded projects will lead to new discoveries in the development of effective treatments for this devastating disease, and enable the next generation of ALS researchers to start or continue research in promising areas of investigation,” said Inez Jabalpurwala, President and CEO, Brain Canada Foundation. “In addition, the discoveries that will result from this partnership have the potential to inform how we approach other neurodegenerative diseases with similar underlying mechanisms, such as MS, and Alzheimer’s, Parkinson’s and Huntington’s diseases.”
“An ALS diagnosis has significant impact on all areas of life – physical, emotional and financial – but the progress of research in recent years has brought hope to those living with ALS that life-changing treatments could be possible,” shared Vincent Bourque, the founder of La Fondation Vincent Bourque, shortly before his passing in November 2018. Mr. Bourque was diagnosed with ALS in 2015 at the age of 40. “Future advancements in research are only limited by the amount of funding available, which is why we are proud to be helping to build the capacity for future ALS research discovery by supporting an early-career researcher.”
This year’s six trainee award recipients seek to answer the following questions about how ALS occurs and how it could be treated:
- Can advanced imaging techniques effectively pinpoint validated biomarkers in ALS and track disease progression?
$75,000 has been awarded to Abdullah Ishaque, a MD/PhD student from Dr. Sanjay Kalra’s lab at the University of Alberta. Abdullah’s research involves analysing images obtained from the Canadian ALS Neuroimaging Consortium (CALSNIC), a project that was funded through the single largest grant given by ALS Canada and the Brain Canada Foundation following the Ice Bucket Challenge. - Can restoring motor neuron inhibition prevent or stop ALS progression?
In partnership with La Fondation Vincent Bourque, $165,000 has been awarded to Dr. Sahara Khademullah, a postdoctoral fellow from Dr. Yves De Koninck’s lab at Université Laval. This research builds on two projects previously funded by ALS Canada that explore whether targeting the activity of upper motor neurons in the brain could be a new and unexplored way to treat ALS. - Is a loss of C9orf72, responsible for the most common protein abnormality in ALS?
$75,000 has been awarded to Lilian Lin, a PhD student in Dr. Janice Robertson’s lab at the University of Toronto. - Does a specific type of viral infection play a role in triggering ALS and its progression?
$75,000 has been awarded to Yasir Mohamud, a PhD student in Dr. Honglin Luo’s lab at the University of British Columbia. - Can using worm and stem cell models of ALS to screen for new ALS drugs identify something that slows disease progression?
$165,000 has been awarded to Dr. Prateep Pakavathkumar, a postdoctoral fellow in Dr. Alex Parker’s lab at Université de Montréal. - Can antibodies detect a key misfolded protein associated with ALS in cerebral spinal fluid?
$165,000 has been awarded to Dr. Yulong Sun, a postdoctoral fellow in Dr. Avi Chakrabartty’s lab at the University of Toronto.
The granting of these trainee awards follows a competitive peer review process, which engaged a panel of Canadian ALS experts in evaluating applications to identify the most exceptional young researchers whose projects have great potential to advance the field of ALS research. All aspects of the peer review process were executed in full partnership with Brain Canada, whose funds are provided through the Canada Brain Research Fund (with financial support from Health Canada).
With Brain Canada’s commitment of matched funds now complete, the ALS Canada Research Program will now return to its historic funding levels with $1.5 million to $2 million in research grants awarded each year.
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. ALS Societies across Canada fundraise on a regional basis to provide services and support to people and families living with ALS in their province and contribute to the funding of the ALS Canada Research Program. 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 registered charity that enables and supports excellent, innovative, paradigm-changing brain research in Canada. Since 1998, 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 has committed $115 million from private donors and non-federal partners—now numbering more than 100—which Health Canada has matched with $120 million. For more information, visit www.braincanada.ca or follow us on Twitter or Facebook.
For more information:
416-497-2267 x213
media@als.ca
The ALS Canada Research Program awards eight new project grants in the pursuit of new therapy targets.
TORONTO, November 15, 2018 – The ALS Society of Canada (ALS Canada) today announced an investment of $1 million in eight new research projects being funded in 2018 through the ALS Canada Research Program, which is the only dedicated source of funding for ALS research in Canada. The ALS Canada Research Program is generously funded by Canadians committed to a future without ALS through individual donations and community-based efforts, including 40 per cent of net proceeds from the WALK for ALS fundraising events that take place across the country.
The eight new research projects being funded include multiple studies using cutting-edge techniques, never before applied to ALS, further examination of newly discovered proteins that may be critical to understanding how ALS works in the body, the use of specialized models of ALS to better understand how the disease occurs, a new spin on targeting abnormal immune and inflammatory mechanisms to treat ALS, and development of a unique Canadian protocol to measure the value of therapies on quality of life alongside medical evaluation in clinical trials.
“As someone living with ALS, when I see the passion and dedication of the Canadian ALS research community, I am hopeful that we will soon find the answers we need to change the lives of people living with this disease,” said Denis Blais, who was diagnosed with ALS in 2015 and last year had the opportunity to observe the peer review process. “While there has been significant progress in the last few years, there is more work to be done and we must continue to invest in ALS research to bring us closer to a future without ALS.”
The ALS Canada Research Program aims to accelerate research impact by providing funding support for the best ALS projects focused on someday translating scientific discoveries into treatments for ALS. The ongoing commitment of donors and partnership with provincial ALS Societies provides the program the ability to support new and innovative basic research, as well as help established researchers maintain momentum on vital ALS research projects.
“The Project Grant program directs donor dollars towards the best ALS research projects in Canada,” said Dr. David Taylor, VP Research, ALS Society of Canada. “These include great new ideas that need pilot funding to gather a foundation of data, ongoing ALS research that is already impacting the global understanding of ALS, and studies that directly affect people living with ALS today. Canadians are important contributors to the field and we hope the program can expand to fund even more projects in the future.”
The research being funded will seek to answer questions that will enable the exploration of new therapeutic targets, extend existing research to support further discoveries and help researchers gain a greater understanding about why ALS progresses differently in each individual. The research questions to be answered are:
- Can unique fish models of ALS be used to understand nervous system signaling in ALS?
$125,000 has been awarded to Dr. Gary Armstrong at the Montréal Neurological Institute. - Can a protein that affects immune cells in the brain be reprogrammed to prevent or slow ALS?
$125,000 has been awarded to Dr. Jasna Kriz at Université Laval. - Can an effective measure of ALS-specific health related quality of life in clinical trials be developed?
$124,993.81 has been awarded to Dr. Ayse Kuspinar and Dr. Vanina Dal Bello-Haas, both from McMaster University - Are abnormal stress granules a unifying factor in ALS?
$125,000 has been awarded to Dr. Eric Lécuyer at Institut de recherches cliniques de Montréal (IRCM). - Is ALS pathology different in one region of the brain vs. another within a single person with ALS?
$125,000 has been awarded to Dr. Janice Robertson at the University of Toronto. - Can environmental factors affecting genes explain why ALS affects people differently?
$125,000 has been awarded to Dr. Ekaterina Rogaeva at the University of Toronto. - Does a unique, hidden protein called altFUS play a role in ALS?
$125,000 has been awarded to Dr. Xavier Roucou at Université de Sherbrooke. - Does a previously unstudied protein called hnRNP A1B play an important role in ALS?
$125,000 has been awarded to Dr. Christine Vande Velde at the University of Montréal.
The project grants for Dr. Ayse Kuspinar and Dr. Vanina Dal Bello-Haas, Dr. Janice Robertson and Dr. Xavier Roucou are supported in part by the Bernice Ramsay Estate.
Approximately 3,000 Canadians are living with ALS, a terminal disease that gradually paralyzes people because the brain is no longer able to communicate with the muscles of the body that we are typically able to move at will. Each year approximately 1,000 Canadians die from ALS and a similar number are diagnosed. With no cure and few treatment options available that have a significant impact on the progression of the disease, most people with ALS die within two to five years of diagnosis.
The funding of the eight research projects followed a competitive peer review process, which engaged global ALS experts who evaluated a larger pool of applications to identify the projects that are grounded in scientific excellence and have the potential to most quickly advance the field of ALS research in order to develop effective treatments. The peer review was observed by people who have personal experience with ALS.
About the ALS Canada Research Program and Canada’s ALS Societies
The ALS Research Program funds peer-reviewed research grants and fosters collaboration amongst Canadian researchers, helping to nurture new ideas and build capacity. 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 better government support and access within the healthcare system.
For more information:
416-497-2267 x213
media@als.ca
Washington, D.C. (March 8, 2018) – The ALS Association, in partnership with the Motor Neurone Disease Association (MND Association) and the ALS Society of Canada, is pleased to announce $600,000 in funding to support the ALS Reproducible Antibody Platform (ALS-RAP). The funding will support the creation of an open-access pipeline to validate antibody research and provide the ALS research community with the highest quality reliable, renewable antibodies for ALS genes.
ALS-RAP was created as a public-private partnership comprised of world experts in antibody generation and validation, including 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).
Standard operating procedures will be established to characterize ALS antibodies – both commercially available and newly created – to ensure these exceed SGC and the Neuro’s stringent quality criteria to establish a public list of “gold-standard” antibodies that will ultimately lay a solid foundation for successful ALS therapies.
ALS is a progressive neurodegenerative disease that affects nerve cells in the brain and spinal cord. Eventually, people with ALS lose the ability to initiate and control muscle movement, which leads to total paralysis and death, usually within two to five years of diagnosis. The vast majority of ALS cases are believed to be caused by a diverse combination of genetic and environmental factors. There is no cure.
Antibodies are among the most frequently used tools in basic science research and clinical assays. Researchers either purchase or generate their own antibodies to detect proteins in their model systems. In part due to lack of a common and transparent validation framework, antibodies often turn out to have high variability and low specificity. This may yield questionable results, leading to wasted time and money, and more importantly, compromising steady scientific progress to understand this challenging disease.
In response, ALS-RAP was formed to ensure the availability of the highest quality, validated antibodies developed using standard operating procedures that will be openly shared with the ALS research community. Notably, no form of intellectual property protection or patents will be filed for all new reproducible antibodies fully discovered and developed by ALS-RAP. This collaborative effort, based on open science and complete freedom to operate, will ensure the use of the highest-quality tools to increase the success of future drug discovery.
This partnership brings together antibody experts from institutions all over the world. The SGC is a global network of experienced academic and industry scientists that will contribute expertise in the development of reagents required for target validation and drug discovery.
ALS-RAP will be working with Lucie Bruijn, Ph.D. MBA, Chief Scientist, The ALS Association; Brian Dickie, Ph.D., Director of Research Development, the MND Association; and David Taylor, Ph.D., Vice President, Research, ALS Society of Canada. Janice Robertson, Ph.D., Professor at University of Toronto; Aaron Gitler, Ph.D., Professor of Genetics at Stanford University; Joe Lewcock, Ph.D., Head of Biology Discovery at Denali; and Janine Kirby, Ph.D., Reader in Neurogenetics at The University of Sheffield will serve as advisors and provide input and guidance into the panel of proposed antibodies to be generated.
An ALS Gene Prioritization Panel with additional experts will also be created to ensure that nomination of genes into ALS-RAP will be transparently responsive to requests for antibody creation and validation from the global ALS research community. Taken together with Open Science model and patent-free access to all outputs, it is expected that the ALS-RAP will galvanize and enable a faster and even more efficient development of therapies to address the ALS challenge, globally.
“The ALS Association is delighted to fund this exciting initiative and believe that it will be an extremely valuable open resource for researchers globally,” stated Lucie Bruijn, Ph.D.
“We are delighted to partner on this exciting new initiative to provide researchers with the ‘best tools for the job’, which will help improve the quality and pace of ALS research,“ said Brian Dickie, Ph.D, Director of Research, MND Association.
“This platform has the potential to accelerate our ability to understand and treat ALS by removing a long-standing hurdle to discovery, and ALS Canada is thrilled to be supporting it as part of this international partnership,” stated David Taylor, Ph.D., Vice President, Research, ALS Society of Canada.
“We are excited to be part of such a visionary initiative from the three leading ALS foundations to build a truly groundbreaking model to underpin the future of therapeutics discovery,” said Wen Hwa Lee, Ph.D., Director of Disease Foundations at the Structural Genomics Consortium and based at the University of Oxford.
About The ALS Association
The ALS Association is the only national nonprofit organization fighting Lou Gehrig’s Disease on every front. By leading the way in global research, providing assistance for people with ALS through a nationwide network of chapters, coordinating multidisciplinary care through certified clinical care centers, and fostering government partnerships, the Association builds hope and enhances quality of life while aggressively searching for new treatments and a cure. For more information about The ALS Association, visit our website at www.alsa.org.
About the Motor Neurone Disease Association
The MND Association was founded in 1979 by a group of volunteers with experience of living with or caring for someone with MND. They are the only national charity in England, Wales and Northern Ireland focused on MND care, research and campaigning. The MND Association improves care and support for people with MND, their families and carers, funds and promotes research that leads to new understanding and treatments, and brings us closer to a cure for MND, and campaigns and raises awareness so the needs of people with MND and everyone who cares for them are recognized and addressed by wider society.
About the ALS Society of Canada
Founded in 1977, the ALS Society of Canada (ALS Canada) and our provincial partners are dedicated to supporting Canadians living with ALS and investing in research to make ALS a treatable, not terminal, disease. We are a registered charity that receives no government funding – all of our services and research are funded through the generosity of our donors. Through the ALS Canada Research Program, we fund peer-reviewed research grants, foster collaboration and build capacity within Canada’s ALS research community, and participate in new areas of research where we are well-positioned to have an impact. Within Ontario, ALS Canada has a role similar to that of the provincial ALS societies providing services and support to help meet the needs of people living with ALS. ALS Canada advocates federally, provincially, and locally for better government support and access within the healthcare system for people touched by ALS. www.als.ca
About the Structural Genomics Consortium
The SGC is a pre-competitive public-private partnership that accelerates research in human biology and drug discovery by making all of its research output freely available to the scientific community. To achieve its mission, the organization is building an open and collaborative network of scientists. The SGC has active research facilities at seven leading academic institutions across the globe (Toronto and Montreal-Canada, Oxford-UK, UNICAMP-Brazil, Karolinska-Sweden, UNC Chapel Hill-USA and Frankfurt-Germany), and SGC scientists collaborate with more than 300 researchers in academia and industry. The SGC is a registered charity (number 1097737) that receives funds from AbbVie, Bayer Pharma AG, Boehringer Ingelheim, Canada Foundation for Innovation, Eshelman Institute for Innovation, Genome Canada, Innovative Medicines Initiative (EU/EFPIA), Janssen, MSD, Merck KGaA, Novartis Pharma AG, Ontario Ministry of Economic Development and Innovation, Pfizer, São Paulo Research Foundation-FAPESP, Takeda, and Wellcome Trust. For more information, visit www.thesgc.org.
Contact:
Brian Frederick
The ALS Association
bfrederick@alsa-national.org
TORONTO, November 22, 2017 – Today, the ALS Society of Canada announced 12 exciting new research projects being funded in 2017 through the ALS Canada Research Program, which is supported by ALS Societies across Canada. The research being funded includes a multi-year study of a promising drug combination, three trainee grants that will help to nurture the next generation of Canadian ALS researchers currently pursuing their PhDs, two projects that explore how ALS treatments could be delivered through the bloodstream, and an initiative that seeks to understand why the muscles of the eyes are often more resistant to ALS as other muscle groups shut down. The announcement was made in partnership with the Brain Canada Foundation, which through the Canada Brain Research Fund (with financial support from Health Canada) is co-funding the largest grant of $1.8 million through matching funds committed following the Ice Bucket Challenge.
Approximately 3,000 Canadians are living with ALS, a disease that paralyzes the body, taking away the ability to move, talk, swallow and eventually breathe. Each year about 1,000 Canadians are diagnosed with the disease and a similar number die. Most people die within two to five years of their ALS diagnosis because the disease has no cure and few treatment options.
“ALS is an incredibly complex disease, but the research community is making tremendous headway and has come very far with very little in comparison to annual funding levels of diseases that have treatment options,” says Dr. David Taylor, Vice President of Research, ALS Canada. “For the ALS Canada Research Program this has only been possible because of the generosity of donors, partnerships with provincial ALS Societies across Canada, and organizations like Brain Canada, which by matching donations made through the Ice Bucket Challenge has essentially doubled Canada’s ALS research investment over the past several years.”
Combined with research grants jointly awarded by ALS Canada and Brain Canada in 2015 and 2016, the grants awarded in 2017 are putting to work the $20 million earmarked for Canadian ALS research following the Ice Bucket Challenge that in 2014 raised unprecedented awareness for the disease and funds for ALS charities including ALS Societies across Canada. With Brain Canada’s commitment of matched funds now complete, the ALS Canada Research Program will soon return to its historic funding levels with $1.5 million to $2 million in research grants awarded each year.
“Canadians responded generously to the Ice Bucket Challenge, and by matching the $10 million earmarked for research through our partnership with Health Canada, Brain Canada doubled the impact of this generosity,” said Inez Jabalpurwala, President and CEO, Brain Canada Foundation. “Since 2015, in partnership with the ALS Canada Research Program, we have funded 20 grants for novel, outside-the-box research; nine grants for collaborative, translational research projects that will move discovery from the lab to the clinic; and five grants that enable the next generation of researcher professors to start or continue research in promising areas of investigation. While the focus of all these research investments remains ALS, Brain Canada has brought its one system approach, whereby researchers from other areas were encouraged to apply their knowledge, and every discovery carries the potential to impact other neurodegenerative disorders which share common underlying mechanisms with ALS.”
Of the research grants announced today, the $1.8-million project co-funded by ALS Canada and Brain Canada will seek to find out whether a promising drug combination can address misfolded proteins, one of the defining biological characteristics of ALS. It was awarded to a research team led by Dr. Heather Durham from McGill University, with collaborators Dr. Josephine Nalbantoglu (McGill University); Dr. Richard Robitaille (Université de Montréal), and Dr. Chantelle Sephton (Université Laval).
The other 11 projects were funded through the ALS Canada Research Program through two different grant competitions as a direct result of donations and partnerships with ALS Societies across Canada including 40% of net proceeds from the WALK for ALS fundraising events that take places across the country.
The research being funded in 2017 seeks to answer the following questions that will help to move us from greater understanding of ALS to the development of therapies for human use:
- Can adjusting the levels of a “guardian” protein protect a protein that becomes toxic in most cases of ALS? $125,000 awarded to Dr. Marco Prado with collaborators Dr. Martin Duennwald and Dr. Flavio Beraldo, all from Western University
- Can image-guided focused ultrasound technology be used safely in people living with ALS as a means of delivering future treatment? $124,948 awarded to Dr. Lorne Zinman with collaborators Dr. Nir Lipsman, Dr. Kullervo Hynynen, Dr. Sandra Black, Dr. Todd Mainprize, and Dr. Agessandro Abrahao, all from the University of Toronto
- Can microscopic bubbles in our bodies be used to deliver ALS treatments through the bloodstream? $125,000 awarded to Dr. Derrick Gibbings with collaborators Dr. Baptiste Lacoste and Dr. Maxim Berezovski, all from the University of Ottawa
- Could targeting the activity of motor neurons in the spinal cord be a new way to diagnose and treat ALS? $125,000 awarded to Dr. Yves De Koninck, Université Laval
- Could the change in communication processes between motor neurons and the immune cells of the nervous system after an ALS diagnosis help to identify new treatment targets? $124,930 awarded to Dr. Stefano Stifani, McGill University
- Could touchscreen technology help to improve testing for the cognitive impairment that occurs in some cases of ALS? $110,770 awarded to Dr. Flavio Beraldo with collaborators Dr. Marco Prado and Dr. Vania Prado, all from Western University
- Could whole genome sequencing reveal new areas of genetic mutations that make some people more likely to develop ALS? $75,000 awarded to Jay Ross, a PhD student in Dr. Guy Rouleau’s lab at McGill University
- How might misfolded proteins that occur in ALS cause cells to die? $50,000 awarded to Sonja Di Gregorio, a PhD student in Dr. Martin Duennwald’s lab at Western University
- What can we learn from mice that are able to walk almost normally despite significant loss of motor neuron function? $125,000 awarded to Dr. Turgay Akay, Dalhousie University
- Why are eye muscles more resistant to ALS, and what can we learn about this that could help to preserve the function and use of other muscles? $121, 048 awarded to Dr. Richard Robitaille with collaborator Danielle Arbour, both from Université de Montréal
- Will probiotics that improve ALS symptoms in worms also work in mice? $75,000 awarded to Audrey Labarre, a PhD student in Alex Parker’s lab at the Université de Montréal
The funding of these 12 research projects follows a rigorous scientific assessment by global ALS experts who evaluated a larger pool of applications to identify the projects that are grounded in scientific excellence and have the potential to most quickly advance the field of ALS research in order to develop effective treatments. The scientific evaluation was observed by people who have personal experience with ALS.
About Canada’s ALS Societies and the ALS Canada Research Program
ALS Societies across Canada fundraise on a regional basis to provide services and support to people and families living with ALS in their province and 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 registered charity headquartered in Montreal that enables and supports excellent, innovative, paradigm-changing brain research in Canada. For nearly two decades, 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.
For more information:
416-497-2267 x234
media@als.ca