August 29, 2019

What’s happening in the world of ALS research at this point in the year? Read about the progress researchers have made in developing new and better models to study ALS in the lab, insights gained into the progression of ALS on a cellular level, new compounds identified as potential treatment strategies for ALS and the results of a promising Phase 2/3 clinical trial.

New insights into the progression of ALS on a cellular level

ALS symptoms typically first appear in one area of the body and eventually progress leading to widespread paralysis. For the first time, researchers have been able to track this progression throughout the body on a cellular level using an innovative new approach called spatial transcriptomics.

It is well established that genetic factors play a role in the development of ALS. On a cellular level, scientists can determine whether certain genes are activated, or “expressed,” by looking for the substances that gene creates, called RNA. Using a precise new technology, researchers at the New York Genome Center analyzed gene expression in animal models of ALS throughout disease progression.

In this study, researchers collected 76,000 gene expression measurements from 1,200 mouse spinal cord sections. Samples were taken from different regions of the spinal cord and at various stages of disease (pre-symptomatic, onset, symptomatic and end-stage), allowing researchers to track changes over time.

Researchers also collected 60,000 gene expression measurements from 80 post-mortem spinal cord sections donated from seven ALS patients. These samples were representative of the end-stage of disease but were taken from different sections of the spinal cord, enabling researchers to detect differences between distinct areas of the human spinal cord.

The large amount of data provided researchers with numerous insights into the progression of ALS.  For example, researchers found that specialized cells called microglia, which are the primary immune cells of the brain and spinal cord, became dysfunctional before ALS symptoms appeared. The work helped to provide researchers with a map of disease-related changes in many cell types and revealed ways in which the various cells communicate with one another.

Further research is required to determine whether the changes observed were part of the root cause of ALS, or rather a part of the body’s response to the disease. However, this work greatly increases our understanding of the nervous system on a cellular level and researchers believe the information garnered from these types of studies may one day lead to the development of biomarkers, which are needed to help diagnose ALS earlier and test new ALS treatments.

AB Science announces results of Phase 2/3 clinical trial of masitinib in ALS

Pharmaceutical company AB Science recently published positive results from their Phase 2/3 clinical trial of masitinib in ALS that showed that the drug was able to slow functional decline. The study enrolled 394 participants and lasted 48 weeks.

Masitinib is a drug that targets cells within the body that play an important role in the immune system. Preclinical studies suggest that masitinib may reduce inflammation within the nervous system, which is believed to be a factor in the progression of ALS.

The study achieved its main goal, with participants who received the higher dose of masitinib (4.5 mg/kg/day) in combination with riluzole showing a 27% slowing of functional decline (as measured by the ALSFRS-R). This masitinib and riluzole combination also delayed decline in quality of life by 29% (as measured by the ALSAQ-40 survey) and respiratory function by 22% (as measured by forced vital capacity).

Similar to other treatments, such as the recently approved edaravone, subsequent analyses showed that masitinib has the largest effect for those who are earlier in their disease progression.

Overall, researchers are encouraged by these early results. A global Phase 3 clinical trial of masitinib is set to begin to confirm the drug’s effectiveness, and will be led by Canadian researcher Dr. Angela Genge at the Montréal Neurological Institute. This new study is expected to enroll 495 participants from Canada, the US and Europe. Please speak to your neurologist to find out if the study will be recruiting in an area near you.

Researchers develop a model to study ALS-resistant motor neurons in the laboratory

It is common for people with ALS who have progressed to the point of advanced paralysis to retain the ability to move their eyes, yet researchers still don’t know exactly why. For some reason the motor neurons that control eye movements, called oculomotor neurons, are more resistant in ALS.

Studying oculomotor neurons in animals or humans can be difficult due to the relatively low abundance of this cell type in the brain. As a result, researchers from the Karolinska Institute in Sweden set out to develop a way to better model oculomotor neurons in the laboratory.

In the June 2019 study, researchers used stem cell technology to generate a high proportion of oculomotor neurons from mouse embryonic stem cells. After thorough analysis, the researchers were able to confirm that the cells generated in the lab were in fact oculomotor neurons with very similar properties to those found in the human brain.

Preliminary studies of the oculomotor neurons revealed that a specific survival-enhancing signaling pathway, referred to as Akt, was boosted in these cells. This led the researchers to conclude that Akt signaling may in part underlie the oculomotor neurons’ resistance in ALS.

Researchers are hopeful that further studies using these oculomotor neurons, a new ALS-resistant model, will provide a better understanding of the mechanisms responsible for the resistance, as well as important insights that could be used to help slow or stop degeneration in other, more vulnerable cell types.

The ALS Canada Research Program is currently funding a project led by Dr. Richard Robitaille, a researcher and professor at the Université de Montréal, investigating the resistance of oculomotor neurons in ALS, specifically in a region called the neuromuscular junction. Learn more about this exciting project.

Small-molecule compounds identified that can reduce TDP-43 clumping in cells

In a study recently published in the journal Neuron, researchers from UC San Diego School of Medicine set out to identify small-molecule compounds that may be able to reduce the accumulation of stress granules within cells. Researchers are hopeful that the compounds identified in this study can provide a starting point for the development of new ALS therapies.

Stress granules are structures that form within cells in response to various stresses (such as being exposed to high/low temperatures, toxins or disease) in order to protect important cellular components. Stress granules are only meant to form temporarily; however, in ALS these structures often do not disassemble and proteins, such as TDP-43, can become trapped.

When caught in these stress-induced clumps, proteins liked TDP-43 are unable to complete their normal function which can be detrimental to the cell’s health. Thus, researchers believe that strategies aimed at restoring stress granule dynamics in cells may be an important avenue to explore for the treatment of ALS.

Using motor neurons derived from the stem cells of ALS patients with either a TDP-43 or FUS mutation as a model, researchers exposed the motor neurons to a toxin called puromycin to induce stress. They then screed thousands of small-molecule compounds for their ability to change the stress granule dynamics observed and found that several compounds were able to reduce the size and number of clumps formed.

Since TDP-43 displays abnormal behaviour in cells in 97% of all ALS cases, treatment strategies aimed at restoring the normal function and dynamics of the protein, such as the one described in this study, could have broad implications for the treatment of ALS. The researchers recognize, however, that the therapeutic benefit of the small molecules identified still needs to be confirmed in model organisms (such as mice) before a potential therapy could one day be tested in patients.

Researchers develop a new animal model to better understand C9ORF72-linked ALS

A team of researchers based in China has developed a new mouse model of ALS that allows them to study the movement impairments linked to the most common genetic mutation in ALS.

Mutations in a gene called C9ORF72 represent the most common genetic cause of ALS and frontotemporal dementia (FTD). The C9ORF72 gene normally contains a short repeating segment of DNA that, in some people living with ALS, is drastically expanded with up to hundreds or thousands of repeats observed.

Within cells, mutations in C9ORF72 lead to the production of five different small proteins, referred to as dipeptide repeat proteins (DPRs). It is believed that these DPRs contribute to the neurodegeneration seen in ALS. Previous studies looking at the effects of DPR proteins in cell lines and fruit fly models of ALS indicate that one of these proteins, referred to as poly-PR, is exceptionally toxic. The exact role that poly-PR plays in human C9ORF72 linked-ALS is unclear.

To better understand the role that poly-PR may play in ALS, researchers developed an ALS mouse model where the mice produce poly-PR specifically in neurons.  The researchers found that these mice display the movement impairments, loss of motor neurons, and inflammation in the brain and spinal cord that is typical of ALS.

This newly developed mouse model will help researchers to study the mechanisms that underlie the movement impairments seen in C9ORF72-linked ALS. In previous mouse models, the animals would often only display the behavioural/cognitive impairments that are common in FTD. Thus, these mouse models provide a new tool for ALS researchers to gain a more comprehensive view of the symptoms associated with C9ORF72 mutations.

ALS Society of BC Lifetime Directors and researchers; Drs Charles Krieger and Andrew Eisen on ALS Research

ALS News Today

Clinical trials

Clinical trials are research studies that use human volunteers to test new therapies. After scientists test experimental therapies in the laboratory, those with promising results move to clinical trial to determine whether the therapy is safe and effective for use in humans. A new therapy must successfully pass through a series of phases before ultimately being approved by Health Canada and being made widely available to the Canadian public (learn more in our Clinical Trials FAQs).

In general, ALS clinical trials are therapeutic or observational in nature. Therapeutic clinical trials test potential drug therapies or interventional devices that aim to either slow the progression of the disease or help to manage symptoms. Observational trials aim to learn more about the disease and are essential to understanding, diagnosing and ultimately treating ALS. In many cases people who participate in clinical trials will not benefit from the therapy, but their generous involvement will help to find a successful therapy for those diagnosed in the future. One day, a clinical trial will test a therapeutic that slows the progression of ALS and those involved may directly benefit from taking part.

For more information, please speak with your clinician (preferably at an ALS clinic) and visit, where all legitimate, recognized ALS clinical trials are registered globally. You can also visit the EU Clinical Trials Register and the World Health Organization International Clinical Trials Registry for additional information.

You can learn more about the clinical trials currently being conducted at sites across Canada below.

Current Clinical Trials for ALS in Canada

*NEW* A Study to Assess the Efficacy and Safety of H.P. Acthar® Gel in the Treatment of Subjects With Amyotrophic Lateral Sclerosis

Study Type: Therapeutic (Drug: Acthar); Phase 2
Status: Recruiting
Sites: Edmonton, Ottawa, Montreal (Neuro & CHUM), Greenfield Park
Sponsor: Mallinckrodt

H.P. Acthar® Gel is an injectable drug that contains ACTH, a hormone normally found within the body that stimulates the release of natural steroids, such as cortisol. It is believed that ACTH may also affect immune cells within the body. This Phase 2 clinical trail will test whether H.P. Acthar® Gel can slow the progression of ALS. Participants will receive daily subcutaneous injections (i.e. injections under the skin) of the drug for a period of 36 weeks. To determine the effectiveness of the drug, researchers will measure participants’ functional decline via telephone calls using the ALS Functional Rating Score Revised (ALSFRS-R). H.P. Acthar® Gel is currently used to treat other conditions, such as lupus and multiple sclerosis, and so researchers are hopeful that there may be a positive effect for people living with ALS as well.

Learn More and Contact

*NEW* A Phase 3, Randomised, Placebo-Controlled Trial of Arimoclomol in Amyotrophic Lateral Sclerosis

Study Type: Therapeutic (Drug: Arimoclomol); Phase 3
Status: Recruiting
Sites: Toronto, Montreal (Neuro), London
Sponsor: Orphazyme

One of the defining characteristics of ALS and other neurodegenerative diseases is that proteins can become misfolded and clump together, potentially damaging nerve cells. Arimoclomol is an oral drug that increases the production of a group of proteins called heat shock proteins (HSPs) that work to prevent protein misfolding in cells. This Phase 3 clinical trial will test whether treatment with Arimoclomol can slow the progression of ALS. Researchers will determine the effectiveness of the drug through a combined assessment of functional decline (measured using the ALSFRS-R) and survival. Researchers will also monitor changes in participant’s slow vital capacity (SVC), a measure of lung function. The study is expected to last 76 weeks, after which participants will have the option to partake in an open-label extension trial.

Learn More and Contact

REFALS: Effects of Oral Levosimendan (ODM-109) on Respiratory Function in Patients With ALS

Study Type: Therapeutic (Drug: levosimendan); Phase 3
Status: Recruiting
Sites: Calgary, Edmonton, Toronto, Montreal (Neuro & CHUM), Fredericton, Moncton
Sponsor: Orion Corporation, Orion Pharma

This Phase 3 clinical trial will determine whether levosimendan (ODM-109) can improve respiratory function (breathing abilities) in people living with ALS. Levosimendan is an oral drug shown in previous studies to help muscles (such as the diaphragm) contract more easily. This study will enroll approximately 450 people living with ALS. Participants will take the drug daily for a period of 48 weeks. The effectiveness of the treatment will be assessed by monitoring changes in a participant’s slow vital capacity (SVC), a measure of the volume of air exhaled without forced effort after a full breath. Overall functional decline will also be measured using the ALS Functional Rating Score Revised (ALSFRS-R).

Learn More and Contact

Safety and Efficacy of Repeated Administrations of NurOwn® in ALS Patients

Study Type: Therapeutic (Drug: NurOwn®); Phase 3
Status: Recruiting
Sites: University of Massachusetts Medical School
*Canadians may contact
Sponsor: Brainstorm-Cell Therapeutics
Collaborator: California Institute for Regenerative Medicine

Stem cells are cells in the body that have not yet matured into a specific type of cell with a function (such as skin, muscle, bone, etc.). Because of this, stem cells have the ability to become part of any organ in the body. This special ability has led scientists to believe that stem cells may have the ability to repair and replace tissue within the human body. In this Phase 3 clinical trial, researchers are testing the safety and effectiveness of a new potential ALS treatment called NurOwn®. In this study, a person’s own stem cells are taken from their bone marrow and are then combined with the NurOwn® drug. The drug converts the stem cells into cells that secrete neurotrophic factors (NTFs), substances thought to support motor neuron health and delay their degeneration in ALS. These transformed cells are then injected back into the spinal cord of the person living with ALS (intrathecal injection). Researchers will be monitoring participants to assess the safety and ability of NurOwn® to slow the progression of ALS.

Learn More and Contact

A Study of GDC-0134 to Determine Initial Safety, Tolerability, and Pharmacokinetic Parameters in Participants with Amyotrophic Lateral Sclerosis

Study Type: Therapeutic (Drug: GDC-0134); Phase 1
Status: Recruiting
Sites: Montreal (Neuro)
Sponsor: Genentech, Inc.

This Phase 1 clinical trial is designed to test the safety and tolerability of a new oral medicine called GDC-0134 in people living with ALS. GDC-0134 is a drug designed to block the activity of a protein called dual leucine zipper kinase or DLK. DLK is found in nerve cells in the brain and spinal cord and previous studies have shown that activation of DLK may cause these nerve cells to die when they become stressed. Preclinical studies using ALS animal models also revealed that when DLK is blocked it may help to delay motor neuron death. Based on these positive results, GDC-0134 is now being tested as a potential treatment for ALS with approximately 70 people living with ALS to be enrolled in this study. Researchers will monitor participants to ensure that the drug is safe, determine the appropriate dosage and learn more about how the body breaks down the drug internally.

Learn More and Contact

NeuroCognitive Communicator: Safety Study (NCC-1701)

Study Type: Therapeutic (Device: NeuroCognitive Communicator)
Status: Not yet recruiting
Sites: Ottawa
Sponsor: Ottawa Hospital Research Institute

The progressive paralysis experienced by people living with ALS can eventually make communicating with others difficult as the ability to gesture and speak lessens with the weakening of the muscles. To help improve quality of life for people affected by ALS, researchers from the Ottawa Hospital Research Institute are testing the safety of a new assistive device that uses brain-computer interface (BCI) technology to help people who have motor impairments to communicate. This device can convert brain signals into single letters on a computer screen, allowing people to spell words simply with their thoughts. The technology requires surgical placement of two sensors into the areas of the brain that support motor and cognitive function. Researchers will be monitoring the two participants to ensure that the procedure is safe, and to assess the ability of this technology to support effective communication and improve quality of life.

Learn More and Contact

A Clinical Trial of Pimozide in Patients with Amyotrophic Lateral Sclerosis

Study Type: Therapeutic (Drug: pimozide); Phase 2
Status: Recruiting
Sites: Calgary, Edmonton, Fredericton, Hamilton, London, Toronto, Greenfield Park, Montreal (CHUM)
Sponsor: University of Calgary
Collaborators: ALS Canada, Brain Canada

Some people believe that the loss of muscle function that occurs in ALS is caused by the muscles and the nerves not being able to communicate anymore. The area in the body where this communication occurs is called the neuromuscular junction. Pimozide is a medication originally used in schizophrenia that has been shown to enhance communication at the neuromuscular junction in laboratory worms, fish and mice. This Phase 2 study will investigate whether treatment with pimozide slows the progression of ALS in humans. Pimozide will be evaluated primarily using the ALS Functional Rating Score Revised (ALSFRS-R), a 12-item questionnaire that assesses function in certain daily activities. This Phase 2 clinical trial is supported by an ALS Canada-Brain Canada Arthur J Hudson Translational Team Grant.

Learn More

Advancing Research and Treatment for Frontotemporal Lobar Degeneration (ARTFL) (ARTFL)

Study Type: Observational
Status: Recruiting
Sites: Vancouver, Toronto
Sponsor: University of California

Frontotemporal dementia (FTD) is present in 15 to 18 per cent of people living with ALS. FTD is the second most common cause of early onset dementia and results in degeneration of the frontal and temporal lobes of the brain, which can cause changes in personality and language difficulties. The Advancing Research and Treatment for Frontotemporal Lobar Degeneration (ARTFL) project is an observational study with the goal to ultimately discover new biomarkers to better understand disease, improve diagnostic criteria, and identify a large group of potential participants for future clinical trials of new treatment options. Participants in this study include people living with FTD and/or ALS, as well as those living with related frontotemporal lobar degeneration (FTLD) syndromes. On-site evaluations will include medical exams, clinical assessments of cognition and function, questionnaires and surveys, and biological samples.

Learn More and Contact

Diagnosing Frontotemporal Lobar Degeneration

Study Type: Observational
Status: Recruiting
Sites: Toronto
Sponsor: University Health Network, University of Toronto

Diagnosing neurodegenerative conditions such as FTD or ALS can be difficult, as symptoms and disease progression often differ significantly from person to person. The goal of this observational study is to use a variety of tests to determine the best criteria for diagnosing frontotemporal lobar degeneration (FTLD) syndromes. These tests include brain imaging, skin biopsy, cognitive and functional assessments and biological samples for genetic testing. Researchers hope the results of this study will not only guide FTLD diagnosis but also provide a better understanding of the mechanism of disease and aid in the development of new treatments in the future. These advancements may also prove to be helpful in understanding and ultimately treating ALS due to the close overlap in the pathological and genetic features of ALS and FTD.

Learn More and Contact

Phenotype, Genotype & Biomarkers in ALS and Related Disorders

Study Type: Observational
Status: Recruiting
Sites: Edmonton
Sponsor: University of Miami

This observational study is recruiting people living with ALS and related diseases, including primary lateral sclerosis (PLS), hereditary spastic paraplegia (HSP), progressive muscular atrophy (PMA), and frontotemporal dementia (FTD). Biological samples will be collected from study participants with the aim of discovering new biomarkers of disease, and the hope to better understand the links between genetic data (genotype) and observable symptoms (phenotype).

Learn More and Contact

On Wednesday, October 17, 2018, Dr. David Taylor, VP Research at ALS Canada hosted speakers from Canada’s world-class ALS research community as well as international researchers and experts to share their updates with you.

Our speaker line-up included clinical trial experts from three pharmaceutical companies who shared the latest updates on these new studies:

Drug iconPhase 3 study of arimoclomol

A common feature of ALS is the accumulation of misfolded proteins in motor neurons that make it difficult for them to function correctly. In lab research, the drug arimoclomol demonstrated an ability to reduce the buildup of misfolded proteins by increasing the heat shock response. (Learn more about the heat shock response.)

Clinical research among a small group of human volunteers with ALS found that arimoclomol administered in an oral capsule format was safe and well-tolerated. But to further confirm the drug’s safety and to find out whether it can slow disease progression and/or improve survival, larger numbers of study participants are needed. (Learn more about clinical trial phases in our Clinical Trial FAQs or in this blog post from last year’s clinical trials webinar.)

The drug manufacturer Orphazyme recently launched a Phase 3 clinical trial. The first study site opened in Miami, Florida at the end of July 2018. Multiple locations in Canada, the United States and Europe will open soon.

Headshot of Richard BennettRichard Bennett is a senior clinical project manager with Orphazyme A/S, a biopharmaceutical company based in Copenhagen, Denmark. During the Virtual Research Forum, he provided an update on the arimoclomol study and shared details about timing for Canadian study sites. He also discussed what he believes matters most to people who are living with ALS and how Orphazyme is including patient-centric approaches in the study.

Drug iconPhase 3 study of levosimendan (ODM-109)

ALS is characterized by a progressive inability to use muscles involved in movement, speech, swallowing and breathing. Eventually, most people with ALS need breathing support and adaptive aids to assist mobility. Levosimendan (ODM-109) is an oral medication that helps muscles contract more easily and exert a stronger force, possibly slowing down their loss of function.

An intravenous form of levosimendan called Simdax is already marketed in more than 60 countries around the world, (but not in Canada), for the treatment of acute heart failure. A Phase 2 study of an oral form of the drug for people with ALS was conducted in Europe and the United Kingdom in 2017. The results for a small number of study volunteers were promising, but not conclusive.

Orion, the maker of levosimendan, launched a Phase 3 clinical trial in April 2018 to determine whether an oral capsule format of the drug can improve respiratory function in people with ALS. The trial is called REFALS (Respiratory Function in Patients with ALS).
Six Canadian trial sites will open for recruiting soon in Fredericton, Moncton, Montreal (Neuro and CHUM), Calgary and Edmonton.

Paula Rytilä, MD, PhD is Chief Medical Officer at Orion, a pharmaceutical company based in Finland. During the Virtual Research Forum, Dr. Rytilä presented the latest update on the REFALS clinical trial for oral levosimendan (ODM-109).

Drug iconPhase 2 study of H.P. Acthar Gel
Mallinckrodt Pharmaceuticals

One of the hallmarks of ALS disease progression is the over-activation of immune cells in the central nervous system. H.P. Acthar Gel is a medication that is injected beneath the skin. It contains a synthetic version of a hormone normally secreted by the pituitary gland called adrenocorticotrophic hormone (ACTH) that plays a role in regulating inflammation.

The drug is already approved in the United States for the treatment of flares associated with inflammatory diseases including lupus and multiple sclerosis.

Results from a Phase 1 study among a small group of people with ALS suggested that H.P. Acthar Gel might help reduce inflammation. Based on those results, Mallinckrodt Pharmaceuticals has launched a Phase 2 trial to examine whether the drug can slow functional decline in adults living with ALS. The trial is already underway and open for recruiting. Researchers are looking for 213 volunteers across 43 study sites located in Canada, the United States and Colombia. Currently, there are three locations recruiting in Canada — Edmonton, Longueuil and Montreal.

Susan VanMeter, MD, is Senior Director of Clinical Research at Mallinckrodt Pharmaceuticals, a company based in the United Kingdom. She shared more details about the clinical trial’s objectives at the Virtual Research Forum, how it is designed and how Canadians with ALS can participate.

Clinical research is essential towards realizing a future without ALS. Participating in clinical trials gives Canadians living with ALS an opportunity to try new therapies earlier and helps others who may one day be diagnosed with the disease.

NEWS RELEASE       November 15, 2018

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.

Webinars and education

Free to access and open to all, our webinars will help you learn about the latest advancements in ALS research, how donor dollars are supporting one of the best ALS research programs in the world and how to get involved in clinical trials to help make ALS a treatable, not terminal, disease. Some webinars feature guest speakers from the Canadian ALS research community who share details about their work and address key topics in the field.

ALS and the environment

Can exposure to toxins and other environmental factors play a role in the development of sporadic ALS?

Zebrafish can be a useful model to study motor neuron degeneration, and are particularly useful for understanding the role that different cells, such as glial cells, may play in the onset and progression of ALS.

Jessica Morrice, a PhD student in Dr. Christopher A. Shaw’s lab at the University of British Columbia, uses zebrafish to understand how toxins can cause motor neuron degeneration – an understanding that could eventually lead to slowing down or stopping ALS.

Register today

Archived Webinars

From models in the laboratory to ALS therapies

Approaches for better modeling of human disease

Webinar artwork with information and headshot of Vincent Picher-Martel

When it comes to testing new therapeutic approaches for ALS, researchers have a wide variety of animal models to choose from. However, many drugs that have been shown to be effective in animal models have failed in human clinical trials, leading to the question: which animal models are the most effective for modeling human disease?

In this webinar, Dr. Vincent Picher-Martel from Laval University presents some advantages and disadvantages of the animal models currently available for the study of ALS. He also discusses his work using mice models to simulate mutations in Ubiquilin-2 and TDP-43, two genes known to contribute to the development of ALS.

This webinar was presented in both French and English. Click on the Français tab for the link to the French webinar.

View the webinar

Thinking Inside the Box

How touchscreen technology may help to identify cognitive impairments in ALS mice and pave the way for future treatments

Link to archived webinar: Thinking inside the box

This webinar was presented by Dr. Flavio Beraldo, Adjunct Professor and research associate at Western University.

Learn about how touchscreen technology could improve testing for cognitive impairment in ALS, and how these tests could help find treatments in the future. Please note that this webinar was conducted in English only.

View the webinar

The ALS Treatment Pipeline

A webinar by Neurologists for Canadians affected by ALS

Link to archived webinar: The ALS Treatment Pipeline

Presented by the Canadian ALS Research Network
Sponsored by the ALS Canada Research Program

The Canadian ALS Research Network (CALS), a group of ALS clinicians and researchers across Canada, held a webinar on November 30, 2017 offering perspectives on ALS treatments including edaravone as well as others in development. The webinar was a panel discussion featuring:

  • Dr. Wendy Johnston, Director of the Edmonton ALS Clinic and Co-Chair of CALS
  • Dr. Geneviève Matte, Director of the ALS Clinic at CHUM – Hôpital Notre-Dame
  • Dr. Colleen O’Connell, Director of the Fredericton ALS Clinic and Co-Chair of CALS
  • Dr. Christen Shoesmith, Director of the London ALS Clinic

The webinar was moderated by Dr. David Taylor, Vice President Research for ALS Canada, which sponsored the webinar. He provided a brief overview of edaravone, masitinib, NurOwn, tirasemtiv and other experimental treatments currently in clinical trials.

The webinar was conducted primarily in English, but efforts were made to provide some information in French.

Note that the archived version of the webinar has been modified from the live version that originally aired, in which the possibility of placebo effect was discussed using an example of an individual who had reported positive results with treatment. This statement was based on assumption and has since been retracted and removed. An explanation of placebo effect remains because it is important for webinar listeners to be aware of the possibility of placebo responses when pursuing any type of treatment.

View the webinar

Virtual Research Forum

2017 ALS Canada Virtual Research Forum: a free, two-day, webinar featuring world-class researchers in the field of ALS

For a disease like ALS that has no cure and few effective treatments, research is a tremendous source of hope. For the second year running, the ALS Canada Research Program presented a free Virtual Research Forum open to anyone interested in learning more about some of the ALS research currently underway and therapies in development. Hosted by Dr. David Taylor, VP Research at ALS Canada, the Virtual Research Forum featured more than twenty speakers and panelists and took place over two days.

Select presentations from the Virtual Research Forum are available for on-demand viewing below. Please note that the forum was conducted in English – we will be identifying future opportunities to offer research updates in both official languages.

Biomarkers for C9ORF72-associated ALS

Link to Dr. Tania Gendron's Virtual Research Forum presentationDr. Tania Gendron
Mayo Clinic

Mutations in the C9ORF72 gene have been identified as the most common genetic cause of ALS. Toxic substances, called dipeptide repeats (DPR), produced as a result of these mutations are believed to play a key role in the development of ALS. In this webinar, Dr. Gendron, a Canadian working at the Mayo Clinic in Jacksonville, Florida, describes her work to determine whether these cellular byproducts can be used as biomarkers of ALS and allow researchers to more effectively test new ALS treatments. Dr. Gendron explains that the levels of these toxic DPR substances in biological fluid samples of people living with C9ORF72-associated ALS can help researchers to determine things like whether the drug is interacting with the correct target, how much of the drug is needed, and when the drug should be given.

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Cannabinoid use in ALS

Link to Dr. Mark Ware's Virtual Research Forum presentation*Dr. Mark Ware
McGill University

With a clinical management grant through the ALS Canada Research Program, Dr. Mark Ware is leading a team of Canadian researchers who will participate in the upcoming Phase 2 clinical trial designed to test the safety and efficacy of using cannabis (marijuana) extracts to better manage the symptoms of ALS. Previous studies have shown that cannabis-based medicines may improve quality of life by reducing pain, drooling, speech difficulties, breathing issues, depression and sleep disorders. Dr. Ware explains how cannabis can influence such a variety of different symptoms and presents preliminary work that highlights the positive effects cannabis-based medications can have in ALS animal models.

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Cognition in ALS – it’s not what you think

Link to Dr. Michael Strong's Virtual Research Forum presentation*Dr. Michael Strong
Western University

In this webinar, Dr. Michael Strong discusses the relationship between ALS and frontotemporal dementia. It is well-known that a subset of people living with ALS will develop or present with some form of frontotemporal dementia. Frontotemporal dysfunction is linked to problems with memory, language and behaviour (e.g. irritability, loss of sympathy, etc.). Dr. Strong introduces revised criteria for the diagnosis of frontotemporal dysfunction in ALS that will be valuable to researchers, clinicians and people affected by ALS. He also goes on to present recent work that identifies the toxic protein modification responsible for frontotemporal dysfunction in many ALS cases and highlights drug treatments that have proven to be effective at preventing this toxic modification in laboratory studies.

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Getting to know pimozide in ALS

Link to Dr. Lawrence Korngut's Virtual Research Forum presentation*Dr. Lawrence Korngut
University of Calgary

Pimozide is a medication originally used to treat schizophrenia that has shown promise in treating people living with ALS. In this webinar, Dr. Lawrence Korngut describes the evolution of pimozide from laboratory studies on worms and fish to the Phase 2 clinical trial that will launch in late 2017. This Canadian clinical trial is supported by an ALS Canada-Brain Canada Arthur J Hudson Translational Team Grant and will test whether treatment with pimozide slows the progression of ALS in humans. Dr. Korngut, the trial’s principal investigator, outlines the design of the trial as well as the eligibility criteria for participation.

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Lazarus by Lunasin? Untangling an ALS X-file

Link to Dr. Richard Bedlack's Virtual Research Forum presentation Dr. Richard Bedlack
Duke University

Many people living with ALS self-experiment with alternative treatments that are advertised to slow, stop or reverse ALS without trustworthy scientific evidence that backs up these claims. Often these alternative treatments have no therapeutic benefit and in some cases can even be harmful. This is why Dr. Richard Bedlack created ALSUntangled, an online forum designed to engage people living with ALS in shared decision-making about what alternate treatments are worth trying. The ALSUntangled team conducts comprehensive reviews of the most-requested therapies using a scientific approach of gathering evidence and reporting results. In this webinar Dr. Bedlack describes the ALSUntangled process, as well as two new programs he is founding based on the study of ultra-rare cases where ALS symptoms have reversed (referred to as an ALS reversal). The first program is called Replication of ALS Reversals (R.O.A.R) and is a pilot study designed to test alternative treatments that have been associated with ALS reversals. The second is the Study of ALS Reversals (St.A.R.) and is a program designed to identify and gather data on very rare cases where ALS reversals are reported to occur.

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Masitinib for the treatment of ALS

Link to AB Science's Virtual Research Forum presentationAB Science

AB Science’s Alain Moussy (Founder & CEO), Laurent Guy (CFO) and Dr. Olivier Hermine (President of the Scientific Committee) discuss the results of the recently completed Phase 2/3 clinical trial of masitinib. Masitinib is a drug currently in development that reduces inflammation in the body believed to be linked to the development of ALS. The clinical trial results show that masitinib does have a therapeutic benefit by slowing the progression of ALS. A global clinical trial for masitinib, led by Canadian investigator Dr. Angela Genge, is set to begin in late 2017 to confirm the results of this study.

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Protein misfolding in ALS

Link to Dr. Neil Cashman's Virtual Research Forum presentationDr. Neil Cashman
Vancouver Coastal Health ALS Clinic; University of British Columbia

Within a cell, a protein must fold into the appropriate 3D shape in order to perform its intended function. When a protein does not fold into correct shape the outcome can be harmful to cells. Prion-like proteins are especially dangerous because they can cause other normally folded proteins to adopt an abnormal shape creating a domino effect of toxic protein misfolding that spreads throughout the nervous system. Many years ago Dr. Neil Cashman hypothesized that ALS may be caused by the misfolding of prion-like proteins. In this webinar, Dr. Cashman describes data that support this hypothesis as well as new drugs that may be able to stop the domino-like protein misfolding thought to be a problem in ALS.

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Skin models for the study of ALS

Link to Bastien Paré's Virtual Research Forum presentation*Bastien Paré
Laval University

Bastien Paré, a PhD student in Dr. François Gros-Louis’ lab at Laval University and recipient of an ALS Canada Doctoral Research Award, is studying ALS in a new way: he is investigating the relationship between the skin and ALS. Neurological conditions, like ALS, are often accompanied by changes in the elasticity or texture of the skin which may be a result of both skin and brain tissue having the same origin during fetal development. By studying the unique skin characteristics of people living with ALS, Bastien hopes to develop new biomarkers that will help researchers to better understand the disease and even lead to the development of new ALS treatments. Furthermore, he is interested in the possibility of a simple skin test that could someday allow for earlier diagnosis.

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The role of ataxin-2 in ALS

Link to Lindsay Becker's Virtual Research Forum presentationLindsay Becker
Stanford University

Abnormalities in a protein called TDP-43 are present in approximately 97% of all ALS cases. TDP-43 is normally found in the nucleus of a cell (a central compartment where our DNA is located); however, in people living with ALS it is often found in the cytoplasm (the area outside of the nucleus) where it does not belong. This altered location of TDP-43 is thought to be harmful to cells. Previous work looking at TDP-43 in cell models revealed that another protein called ataxin-2 can actually make TDP-43 more toxic. Building on this work, Lindsay Becker, a PhD student in Dr. Aaron Gitler’s lab, studied the effects of changing the amount of ataxin-2 in mice with ALS. Lindsay found that when the amount of ataxin-2 is decreased, ALS mice live longer with increased muscle function suggesting that reducing ataxin-2 levels may represent a promising new strategy to treat ALS in humans.

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Understanding the role of the neuromuscular junction in ALS

Link to Elsa Tremblay's Virtual Research Forum presentation*Elsa Tremblay
Université de Montréal

When you want to perform a voluntary movement, a signal moves from your brain through the motor neurons in your spinal cord and into the muscle where the movement is achieved. The area in your body where the muscles and the nerves communicate is called the neuromuscular junction (NMJ). Elsa Tremblay, a PhD student in Dr. Richard Robitaille’s lab, and recipient of an ALS Canada Cycle of Hope Doctoral Research Award, uses animal models to investigate changes in the NMJ caused by ALS. Experimental tests reveal clear differences in the NMJs of mice with ALS that affect muscle function and fatigue. The hope is that by targeting these differences new ALS treatments can be developed. A Phase 2 clinical trial of pimozide, a medication shown to enhance communication at the NMJ in animal studies, is being funded through the ALS Canada Research Program.

View the webinar

Research projects marked with an asterisk (*) have been funded by the ALS Canada Research Program thanks to the generosity of donors and partnerships with ALS Societies across Canada.

Project MinE logo “Why have I been diagnosed with ALS when so many other people have not?”

This is an all-too-common question of people living with the devastation of an ALS diagnosis, and the ALS Society of Canada (ALS Canada) wants to help answer it. We are leading Canada’s fundraising efforts for Project MinE to support the mapping and analysis of up to 1,000 DNA profiles.

Project MinE is a multi-national initiative with more than 15 participating countries. It will map the full DNA profiles of 15,000 people with ALS and 7,500 control subjects, establishing a global resource of human data that will enable scientists worldwide to understand the genetic signature that leads someone to develop ALS.

By accumulating such a large amount of data that no one country could achieve alone, it is expected that Project MinE will identify new genetic causes of the disease that will significantly accelerate our ability to advance treatment possibilities that will slow down or stop ALS.

Chris McCauley served as ALS Canada’s ambassador for Project MinE until his death in August 2017. In this video, shot in September 2016, he shares his hope for the initiative:

            ALS Research Update, June 2018

June 7, 2018

ALS research is at a time of unprecedented advancement. Our biggest hope is to stop ALS from stealing dreams, abilities, and lives – and researchers are closer than they’ve ever been to making this a reality. With such unprecedented momentum, we can’t afford to be stagnant.

This ALS Awareness Month, the ALS Canada Research Program team has summarized what we believe are the most significant research discoveries. This is our second installment for 2018 – for more information see past updates from August 2017, November 2017and February 2018.

New insights into how mutations in the protein FUS leads to the development of ALS

Mutations in the protein FUS have been linked to ALS. In a healthy cell, FUS can be found in one of three states: (1) as a single protein molecule, (2) grouped together in a liquid droplet state (like a drop of oil in water) or (3) grouped together in a dense gel state (like jelly). In order for FUS to be able to complete one of its intended functions within cells, it must easily be able to switch back and forth between the liquid and gel states. This process, which is referred to as phase separation, appears to be disrupted in ALS. Scientists found that mutations in FUS cause the protein to become trapped in the gel state which is damaging to neuronal cells and over time can lead to cell death. Researchers knew that in order to be able to prevent FUS from becoming trapped in the gel state they needed to better understand the factors that control the liquid to gel transition in cells. Remarkably, four papers published in the April 19 Issue of Cell, a prominent peer-reviewed scientific journal, made significant advances in this understanding. Researchers from all four laboratories were investigating different aspects of FUS and independently reached the same findings, highlighting two specific cellular pathways that control FUS phase separation and may be able to prevent neurodegeneration. One of these studieswas funded in part by the Ice Bucket Challenge and the ALS Society of Canada’s partnership with Brain Canada. Together, these findings open up new avenues to explore when developing treatments for FUS-linked ALS, and are of broad importance as these biological mechanisms are part of our evolving understanding of other forms of ALS as well as frontotemporal dementia (FTD).

A promising lead for treatment of an inherited form of ALS

A team of researchers at the University of Liverpool has identified a possible new treatment strategy for an inherited form of ALS. Mutations in the SOD1 protein represent the second most common cause of inherited ALS. It is thought that mutations cause the protein to fold into the wrong 3D shape, a process referred to as misfolding, which then causes it to gain a toxic function. In study findings published in April 2018, researchers set out to identify a drug that may be able to prevent the misfolding of SOD1 and restore its normal function in human cells. After testing a variety of different compounds the researchers found that one, called ebselen, had a positive effect. Ebselen has previously been tested for the treatment of multiple disorders, including stroke, bipolar disorder, and hemorrhage. Since this study was only conducted using human cells outside the body, the results are preliminary but suggest to researchers that ebselen should be further tested in ALS animal models to confirm the positive effect. Ebselen represents one of many unique treatment options being explored for SOD1-linked ALS. As the first genetic mutation discovered for ALS, SOD1 has been studied for more than two decades. In the near future we hope to see a similar number of treatment options in development for all forms of ALS.

What we can learn from extremely rare ALS reversal cases

In extremely rare cases, a person diagnosed with ALS may stop progressing and regain motor function. These cases are referred to as “ALS reversals” and while highly uncommon, they are important to study because they can help researchers to identify genetic differences that may make a person more resistant to ALS and even lead to the development of an effective treatment. This was the case for HIV where the study of a rare group of HIV resistant “elite controllers” led to development of a successful drug treatment. In an April 2018 study, researchers compared the demographics, disease characteristics and self-administered alternative treatments of verified reversal cases to those of patients with typical progressive ALS. The researchers found that people who experienced ALS reversals were more likely to be male, have limb onset disease, and initially progress faster. The results of the study indicated that there are differences that should be further explored between people who experienced ALS reversals compared to typical progressive cases. Despite the limited number of cases to work with, researchers hope continued study will help to identify the mechanism of disease resistance in reversal cases so that it can one day be applied to the vast majority of people who show typical progressive ALS.

Researchers identify a new link between two cellular processes disrupted in ALS

In recent years, researchers have made significant progress in identifying the various cellular pathways that are disrupted in ALS. This is important because a better understanding of the pathways involved, as well as how they interact with each other to cause ALS, is crucial to developing an effective treatment. Two important cellular pathways previously shown to be affected in ALS are (1) stress granule formation and (2) nucleocytoplasmic trafficking. Stress granules are structures that form temporarily when a cell is stressed (i.e. exposed to heat, cold or radiation) to protect important cellular components. Stress granules are only meant to form temporarily; however, in ALS cellular components can become trapped in these structures preventing them from completing their normal functions. Nucleocytoplasmic trafficking, on the other hand, involves the transport of cellular components between two important compartments of the cell and is crucial to cell survival. In a May 2018 study, researchers identified a link between these two pathways. The researchers found that many of the cellular elements that become trapped in stress granules play an important role in the trafficking process. When these cellular elements are trapped in stress granules, trafficking is stalled. Based on these results, researchers are optimistic that if they can regulate stress granule formation within cells they may also be able to restore nucleocytoplasmic trafficking. In fact, gene therapy techniques to reduce stress granule formation are currently being explored as a potential treatment option for ALS.

Advances in models used to study the biology of ALS

In its early stages, medical research is often conducted using disease models (ranging from nerve cells in a dish, to worms, to mice). The use of disease models allows researchers to study the biology of the disease in ways they could not in people. In order to be useful, however, these models must accurately represent how the disease develops in the human body. In an April 2018 study, researchers described a new technique that allowed them to grow a patient’s own neurons and blood vessels together outside the body for the first time. Using what is referred to as “Organ-Chip” technology, the researchers were able to grow spinal motor neurons from stem cells in a more life-like environment than previously possible in petri dishes. This research allows scientists to mimic important parts of the human nervous system and is part of a bigger collaborationthat aims to use this technique to advance personalized medicine, a new medical approach to determine the most effective treatment for a person based on their unique biology. Since ALS is causes by many different gene mutations, researchers are hopeful that by creating a living model of the disease with a patient’s own cells they may be able to better predict which treatment option would be most beneficial to that patient and avoid the risk of giving a drug that may be costly and ineffective. Organ-Chip technology is not specific to the study of ALS, but rather can be applied to a variety of different conditions including Parkinson’s and Crohn’s disease, and so is an important new tool for the medical community as a whole.

Note: We have included links to the publications because we know people may be interested in the original source papers. While abstracts are always available, since many journals are subscription based in some cases full papers may only be accessed at a cost.

ALS research is at a time of unprecedented advancement, with researchers zeroing in on creating a future without ALS. Our biggest hope is to stop ALS from stealing dreams, abilities, and lives – and researchers are closer than they’ve ever been to making this a reality. With such unprecedented momentum, we can’t afford to be stagnant.

Other initiatives

In addition to funding ALS research across Canada, we bring together the national ALS research community to share ideas and information and collaborate as a unified group in support of our vision to make ALS a treatable, not terminal disease.

We also support the Canadian ALS research community in contributing to international ALS initiatives that pool our resources and knowledge, enabling us to work together on solving problems that can’t be tackled alone.

By bringing the national and international ALS research communities together, the opportunities for ALS breakthroughs grow.

ALS Canada Research Forum

This annual event hosted by ALS Canada is a venue for researchers to share ideas, form new collaborations and connect with people living with ALS. The forum is attended mainly by the Canadian research community and also, people living with ALS and their family members, volunteers, donors, sponsor representatives and ALS Canada board members.

Event Sponsorships and Travel Awards

To be able to respond nimbly to new ALS research discoveries, ALS Canada sponsors ad-hoc gatherings of ALS researchers to share knowledge on timely topics related to ALS.  Sponsorship requests are reviewed and assessed by ALS Canada’s Scientific and Medical Advisory Council. Recent examples of sponsored events include a bi-annual international workshop on frontotemporal dementia in ALS, the global ALS Clinical Trials Guidelines workshop and the Symposium de la Fondation André-Delambre sur la sclérose latérale amyotrophique (SLA).

We also provide travel stipends to up to 15 Canadian ALS researchers each year to present their work at the International Symposium on ALS/MND. Organized by the International Alliance of ALS/MND Associations,  it is the premier international ALS research conference in the world.

Contact us to learn more about sponsorship funds or travel awards.

National Research Network

ALS Canada is actively facilitating the development of a formalized network of researchers across the country. In previous years, The Canadian ALS Research Network (CALS) sought to bring world-class clinical trials to sites across Canada. This clinical network is now being enhanced and broadened in order to better integrate preclinical research studies. Once established, the new network will reflect a true “bench to bedside” approach that bridges basic laboratory science and clinical application. More information will be available on this site as the network evolves.

ALS Disease Registry

The ALS Canada research program has also supported building the foundation of a national registry for ALS, housed in the Canadian Neuromuscular Disease Registry (CNDR). This database is designed to learn more about the clinical aspects of Canadians living with ALS and the care they receive. This tool, alongside the Canadian ALS Clinical Practice Guidelines, are working towards a standard of care for people living with ALS across the country.

Clinical Practice Guidelines

Since 2014, a working group of ALS specialists has been creating a set of Canadian guidelines that will represent standards of care for people living with ALS. Once completed, the guidelines will support physicians across the country in delivering appropriate care to people living with ALS. They will also serve as a tool for clinicians and other stakeholders to advocate at local, provincial and federal levels with hospitals, lawmakers and others who can affect change to ensure the standards of care are being met. ALS Canada has supported all aspects of the evidence-based process to develop the guidelines, including convening the working group and professional literature searches.

Projects funded

ALS Canada-Brain Canada Arthur J. Hudson Translational Team Grant

Named after Dr. Arthur J. Hudson, the co-founder of ALS Canada, this grant program brings together researchers from across the country to accelerate therapeutic development by 1) identifying and testing a relevant therapeutic target or candidate therapy and/or 2) addressing critical needs for early diagnosis and biomonitoring of clinical progression applied to clinical research. Research in all stages of development is welcomed, from basic/preclinical to Phase I, II and III clinical trials. ALS Canada partners with Brain Canada (with the financial support of Health Canada) on this program in order to leverage contributions made through the Ice Bucket Challenge.

Project Grants

Project Grants provide funding for three different types of ALS research: 1) outside-the-box research that would not easily be successful in receiving funding from other agencies due to the high risk/high reward nature of the work (previously called Discovery Grants); 2) Research already underway that has demonstrated promise and would benefit from continued funding in order to maintain momentum (previously called Bridge Grants, with the requirement of having applied to CIHR for funding); and 3) research focused on avenues to maximize function, minimize disability and optimize quality of life through symptom management and support to people living with ALS (previously called Clinical Management Grants). The Project Grant category represents a combination of three different grant programs prior to 2017.

Trainee Awards

By providing salary support to PhD students, trainees with a postdoctoral position, or clinical fellows or recently-hired junior faculty members, these grants attract the brightest young minds to ALS research, bringing new ideas to the field and maintaining Canadian ALS research excellence into the future. The Trainee Grant category represents a combination of different grant programs prior to 2017.

ALS Canada-Brain Canada Discovery Grants

The discovery grant program encourages new basic research focused on identifying causes of or treatments for ALS. The goal of this program is to build a foundation of data for novel, out-of-the-box ideas in ALS research or to attract investigators from related fields who bring new expertise to ALS research. ALS Canada partners with Brain Canada (with the financial support of Health Canada) on this program in order to leverage contributions made through the Ice Bucket Challenge.

ALS Canada-Brain Canada Career Transition Award

Ensuring that Canada continues to have a strong community of talented ALS researchers is the goal of this 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 to make the disease treatable, not terminal. Furthermore, this research will have a broader impact on our understanding of other neurodegenerative diseases. ALS Canada partners with Brain Canada (with the financial support of Health Canada) on this program in order to leverage contributions made through the Ice Bucket Challenge.

Ronald Peter Griggs and Tim E Noël Postdoctoral Fellowships

Fellowships provide salary support for promising young investigators who at this point in their careers are making critical decisions about the areas they will study in the future.  Supporting the highest calibre applicants at this stage provides the best possible chance for maintaining Canadian ALS research excellence in the future.

ALS Canada Doctoral Research Awards

These awards provide $25,000 per year over three years for young researchers to pursue a PhD in a Canadian laboratory. This funding also assists the hosting laboratory by offsetting funds that will help them to achieve their goals. As a result, it is a wise investment with the potential to launch the career of a future leader in the field and further secure our ability to achieve the vision of making ALS a treatable, not terminal disease.

ALS Canada Bridge Grants

This program is designed to maintain the momentum of the best ongoing ALS research projects in Canada that applied to federal government grant competitions through the Canadian Institutes of Health Research (CIHR). CIHR grant competitions typically have over 2,000 applicants spanning all forms of health research from across the country, and only the top 10-15% receive funding. This can make it very difficult for ALS grants to be supported. The ALS Canada bridge grant program combines CIHR scores and an independent assessment of the impact on the field of ALS through peer review, to determine the recipients of $100,000 for use over one year.

ALS Canada Clinical Management Grant

Many of the choices that clinicians make in treating symptoms, or that families make on seeking care are anecdotal or based on evidence from other diseases. Formal assessment of these avenues in ALS patients could address potential gaps in care. Examples include, but are not limited to, management of secretions and cramps, psychological interventions to address mental health issues, nutritional interventions, respiratory care, engineering applications to reduce physical limitations, and programs to address the needs of caregivers.

Bernice Ramsay Clinical Research Fellowship

This program supports specialized training in clinical care and research skills related to ALS. The program awards a researcher $100,000 per year for two years and is designed to strengthen the Canadian ALS clinical community. These were also made possible by the generous estate of Bernice Ramsay, which donated $2.28 million to ALS Canada in 2006. Peer review is performed by a panel of not-in-conflict Canadian ALS clinicians.

ALS Cycle of Hope Doctoral Research Award

In 2015, the ALS Cycle of Hope sponsored a PhD student to pursue their studies in ALS for three years at $25,000 per year. The student was chosen as the top-ranked individual in the 2015 Doctoral Research Award competition.

ALS Canada Postdoctoral Fellowship

In times where extra funding has been available and following the awarding of any named fellowships, additional recipients have been named for ALS Canada.