ALS Research

Clinical trials are research studies that involve human volunteers to test new therapies. After scientists test experimental therapies in the laboratory, those with promising results move to clinical trials to determine whether the therapy is safe and effective for use in humans. 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.

For more information on clinical trials, please refer to our complete Clinical Trials FAQ. The following resources are available:

• ALS Clinical Trials Unboxed: In this webinar series, join ALS Canada to learn more about ongoing ALS clinical trials and hear from pharmaceutical companies. Watch past recordings here.
• ALS Research and Clinical Trials 101: Join ALS Canada in this new monthly virtual series for people living with ALS, their families, caregivers, and anyone close to someone living with the disease who might have questions about ALS research and/or clinical trials.

For inquiries about clinical trials in Canada and globally, you can also contact the ALS Canada Research Team at research@als.ca.

Most clinical trials are run at ALS specialized ALS (CALS clinics). To participate, an individual should ask the clinician at their CALS clinic for information on any available trials they may be eligible for. Resources provided in this FAQ can be used to locate clinical trial sites in Canada and internationally. If someone would like to participate in a clinical trial that is not offered at their clinic, they can contact the clinic hosting the trial and ask for more information and eligibility criteria. They may also ask their clinician to refer them to another clinic with a trial of interest.

Please visit our Clinical Trials page for up to date information on clinical trials in Canada and participating sites. Additionally, you can find ongoing and upcoming clinical trials in Canada on clinicaltrials.gov. This database also includes information on trials worldwide, as well as past ALS clinical trials. ALS Signal is another useful resource to find ALS clinical trials in Canada and the US.

You can also visit the EU Clinical Trials Register and the World Health Organization International Clinical Trials Registry for additional information.

Observational studies do not actively investigate a potential therapeutic. Rather, researchers collect information from participants living with ALS. These studies aim to learn more about the disease and are essential to understanding, diagnosing, and ultimately treating ALS. An example of an observational study in Canada is CAPTURE-ALS.

The Canadian ALS Research Network (CALS) is a network of clinicians across Canada that specialize in ALS research and clinical care. Established in 2008, CALS is recognized within Canada and world-wide for rapid and effective study recruitment. The mission of CALS is to connect Canadian ALS clinics, to improve both patient and clinic participation in clinical research.

You can find more information on CALS and a list of clinics across Canada here.

For a list of commonly used ALS research terms, please refer to ALS Canada’s Research Glossary.

Scientific articles (or papers) are the published results of a scientific study. You can search for articles on databases such as PubMed. ALS Canada’s Guide to Reading Research Papers can help you to start navigating these articles.

Biomarkers are biological measures that provide real-time insights into processes happening in the body, and tell us information about a person’s health status. These measures can vary from checking protein levels in the blood, to measuring a person’s weight and temperature, to utilizing MRI scans as an imaging biomarker. As an example, the level of cholesterol in the blood can serve as a biomarker for the risk of heart disease.

Validated biomarkers that are specific to ALS are urgently needed to help clinicians diagnose ALS, track progression of the disease, and measure response to potential therapies in clinical trials.

While researchers are still working to understand the exact causes of ALS, it is known that some cases are caused by a genetic variant. Several genes have been identified that when altered, can contribute to disease. Approximately five to ten percent of people living with ALS will have a family history of the disease, meaning multiple family members were affected due to a hereditary variant in a gene related to ALS. This is commonly referred to as “familial” ALS (FALS).

For the ninety to ninety-five percent of individuals living with ALS without an obvious family history, traditionally referred to as “sporadic”, it is estimated that between eleven to sixteen percent of cases are caused by known ALS variants.

Please refer to ALS Canada’s Gene Hub for more information on ALS genetics.

Access to ALS genetic testing depends on whether you have a known family history and/or are showing ALS symptoms.

Please refer to ALS Canada’s Gene Hub (Section 2.6: Accessing Genetic Testing in Canada) for more information.

Researchers are currently studying environmental risk and protective factors associated with ALS, which can influence a person’s risk of developing the disease.

Proven risk factors for ALS are genetic variants and advanced age. ALS is also slightly more common in men than women.

For environmental risk factors, the only factor that is generally accepted to be associated with ALS is smoking. The exact mechanism and reasons why smoking could lead to a higher risk for ALS is unknown. However, given that smoking is a major risk factor for many, more common health problems, the risk of ALS is only one of the many reasons for recommending against it.

More research is needed and is currently being conducted to assess the influence of environmental factors on the onset and progression of ALS. There are current observational studies investigating the link between ALS and other risk factors. However, it is difficult for these studies to prove with certainty that the risk factor caused or contributed to the disease, as it is not a controlled environment, and several different factors might be at play. Due to this, we are unable to pinpoint any other specific causal factors for ALS.

A workshop on guidance for pre-symptomatic genetic ALS and frontotemporal dementia (FTD), held in September 2023, concluded that, aside from not smoking for reasons beyond ALS and FTD, our current understanding of risk factors is not sufficient to warrant any recommendations for lifestyle changes to individuals at high genetic risk of these diseases. Once any summarized documentation from this workshop is available, a link will be provided.

ALS Canada uses a rigorous peer-review process to determine which projects receive funding, ensuring that donor dollars will have the maximum impact to advance learning in the field. You can learn more about our peer review process here.

ALS Canada’s Research Program has been supporting world-class research across Canada for more than 30 years through a variety of grants and awards. These initiatives aim to deepen our understanding of how ALS works in the body, with the ultimate goal of making ALS a treatable, not terminal disease. Our Research Program funds projects in several areas of ALS research, including therapeutic, genetic, clinical management, & biomarker work. To explore all of our projects funded since the 2000s, please visit our Research Program & Funded Projects pages.