Dr. 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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*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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*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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*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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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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AB 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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Dr. 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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*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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Lindsay 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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*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.
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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.
Dr. Jan Veldink
Professor Martin Turner
Nick Ramsey
