2016 ALS Research Review

Investments in ALS research have always been a reason to be hopeful that one day there will be effective treatments for this devastating disease. As a result of generous donor contributions and our partnership with the ALS Society of Canada, we – and you! – helped to fund more than $6 million in research in 2016 through the ALS Canada Research Program.

Research Highlights

The research investments made in 2016 will fund 20 projects across the country, enabling 31 researchers at eight academic institutions to engage in important ALS research.

These include:

  • With co-funding from Brain Canada (with financial support from Health Canada), 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 (see sidebar);
  • $1.5 million awarded to early-career researchers through three different grant programs that invest in the future of ALS research by developing the next generation of scientists and enabling them to focus their work on ALS;
  • $700,000 to seven smaller studies, co-funded with Brain Canada (with financial support from Health Canada) that enable investigators to explore outside-of-the-box research;
  • The first recipients of the ALS Canada Clinical Management Grant. This program funds research focused on avenues to maximize function, minimize disability and optimize quality of life through symptom management and support to families and persons living with ALS. The funding allowed a group of researchers to explore the use of cannabinoids, substances that have demonstrated therapeutic effects including anti-inflammatory, analgesic, and anti-anxiety, for ALS symptom management.


A research team led by Dr. Guy Rouleau of McGill University and the Montreal Neurological Institute was 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.

p21-janice-robertsonA research team led by Dr. Janice Robertson of University of Toronto was 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.

The ALS Canada Research Program by numbers

6 million dollars invested in ALS research across Canada in 2016
20 projects funded representing 31 investigators at 8 universities in seven provinces
7 studies, co-funded with Brain Canada (with financial support from Health Canada)
3 grant programs that invest in the future of ALS research by supporting the next generation of scientists

Collaboration behind ALS Research

In the field of research, collaboration is key. Many ALS research projects have resulted in important discoveries, thanks to this interaction. Therefore, the ALS Society of Quebec is proud to support Quebec’s initiatives that aim to encourage exchange between today’s and tomorrow’s researchers, all over the world.

Dr. Christine Vande Velde
Associate Professor in the Department of Neuroscience at Université de Montréal and the Centre hospitalier de l’Université de Montréal (CHUM), Research Center.
On September 27, 2016, many researchers, clinicians and students gathered in an informal setting in order to share their projects and ideas in ALS research during the very first ALS Research Network meeting, initiated by Dr. Christine Vande Velde and Dr. Angela Genge.

“In the last few years, ALS researchers have been collaborating more and more. As a result, we have made significant findings. I think in my lifetime we will discover a therapy to treat ALS. We’ll finally get it. The therapy might not benefit all patients, but it will be a start.”

Dr. Jean-Pierre Julien
Professor at the Faculty of Medicine, Université Laval and Neurosicence Director at l’Institut universitaire en santé mentale de Québec
When he helped spearhead the organization of the first ALS Annual Symposium on behalf of the Fondation André Delambre in 2005, Dr. Jean-Pierre Julien was hoping for an international collaboration to emerge from this event. Twelve years later, he thinks he achieved this goal.

“Since 2005, nearly 300 guest speakers coming from 14 countries have taken part in these encounters. One particular thing about this symposium is that the friendly environment encourages participants to discuss the unpublished results of their research projects. ALS is a complex disease and the collaboration between experts is essential in trying to understand both molecular and cellular mecanisms involved.”

Dr. Nicolas Dupré
Associate Professor, Faculty of Medicine, l’Université Laval and Director of the ALS Clinic at Centre hospitalier de l’Université Laval (CHUL).
Dr. Dupré studies the genes linked to ALS and other motor neuron diseases. Mapping these mutant genes will significantly advance treatment that could slow down or stop the disease.

“I am very excited by advances in genomic research which is allowing us to create motor neurons in the lab. By using skin samples from those with ALS we are able to recreate motor neurons with the same genetic background. This is helping us gain a better understanding of the degeneration of motor neurons and will eventually open the door to new therapies and treatments and diagnostic methods for ALS.”

Dr. Angela Genge
Director of the Clinical Research Unit and the ALS Research Program at the Montreal Neurological Institute and Hospital(The Neuro).
Dr. Genge’s research focuses on testing new ALS treatments in clinical trials to improve the quality of life of people living with ALS.

“2017 promises to be a huge year with a variety of new clinical trials. Plus, many medications currently in the review process are showing a lot of promise.  We’ll be going to Health Canada for approval soon. Also, the Neuro will be the first Open Science institute in the world. Starting in 2017, research data will move freely between research teams. The aim is to speed up discoveries of all neurological diseases with ALS at the forefront of this initiative.”

Dr. Pierre Drapeau
Professor, Department of Neuroscience at Université de Montréal and the Centre hospitalier de l’Université de Montréal (CHUM), Research Center
Dr. Drapeau studies zebra fish with mutant TDP-43 or FUS genes that causes them to develop a disease which mimics human ALS. In fact, Dr. Drapeau discovered the drug Pimozide slows down ALS in fish.

“We are about to publish the promising results and in early 2017, phase two clinical trials will get underway.  Worldwide, a number of clinical trials are underway to test the effectiveness and safety of various ALS treatments. Within a year or two, several new medications should be available. These treatments will improve the quality of life of those living with the disease.”

“For those of us with ALS, we keep hoping, every second, that researchers will find a way to extend our lives and ultimately save us.“
-Michel Tremblay, ALS patient, Montérégie

Making Progress

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. Significant progress in ALS research is being made — researchers now believe that finding effective treatment options for ALS is now a matter of “when,” not “if,” and that research discovery is limited only by the amount of funding available to pursue it.

As just one example of a recent development in ALS research, July 2016 saw researchers announcing 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 Canada Research Program for their work during this discovery. This finding will trigger future studies that focus specifically on the NEK1 gene.

Investing with Integrity

The ALS Canada Research Program invests in the best science in order to pursue our unwavering commitment to make ALS treatable, not terminal. Decisions about which projects to invest in are made through a peer review panel, which is the international standard of excellence for research funding. This process provides an objective assessment on the best science that is most able to advance the field.

Thank you

Thank you for your continued support of the ALS Canada Research Program. It is because of your generosity that we can continue to make significant investments in the research that is needed to make ALS treatable, not terminal.

Thank you to all Canadian researchers for feeding hope of a future without ALS!

For more information, please visit als.ca