In 2017, major scientific advances in Canada and internationally have led to significant and exciting progress in ALS research. Discoveries that increase our understanding of the disease are happening far more often than ever before and as a result many new experimental treatments are set to begin human clinical trials in the next few years. Due to the rapid progress of ALS research it can sometimes be hard to stay on top of the latest discoveries. To help with this, the ALS Canada Research Program team regularly summarizes what we believe are the most significant research discoveries. We have two installments from 2017, August and November. The prominent scientific journal, Nature, also produced a fantastic open-access publication on the state of ALS research in October. Below we have summarized what we believe are the most newsworthy research stories of 2017.

January: Canada joins international research collaboration Project MinE

Project MinE is a large-scale global research initiative aimed to determine the genetic basis of ALS. In 2014, the project was initiated by researchers and two individuals living with ALS in the Netherlands. Today, 18 different countries are participating in Project MinE with the goal of mapping the full DNA profiles of 15,000 people living with ALS and 7,500 controls. By doing so, researchers hope to identify genetic signatures that confer a susceptibility to both sporadic and familial (inherited) ALS and ultimately identify new targets for potential treatments. On January 10, 2017 it was announced that Canada was joining this international research partnership. The Canadian component of Project MinE brings together four of Canada’s leading ALS genetics researchers in Vancouver (Dr. Ian Mackenzie), Toronto (Dr. Ekaterina Rogaeva), Montreal (Dr. Guy Rouleau) and Quebec City (Dr. Nicolas Dupré) for their first ever cross-country collaboration. Canada is committed to reading and analyzing up to 1,000 DNA profiles. ALS Canada is leading Canada’s fundraising efforts for Project MinE and our contribution started in 2017 with the first 200 samples. To support Canada’s efforts to contribute to Project MinE, make a donation today.

March: Global Phase 3 clinical trial of masitinib set to begin in 2018

Masitinib is a drug that targets cells within the body that play an important role in the immune system. Previous studies suggest that masitinib may reduce inflammation within the nervous system that is thought to be a factor in the progression of ALS. In March 2017, pharmaceutical company AB Science announced positive results from their Phase 2/3 clinical trial testing the safety and effectiveness of masitinib at slowing the progression of ALS. The study showed that over a 48-week period, treatment with masitinib reduced loss of function and increased quality of life. A global Phase 3 clinical trial intended to confirm the results of the previous Phase 2/3 study is set to begin recruiting in 2018. This clinical trial, led by Canadian researcher Dr. Angela Genge from the Montréal Neurological Institute, will take place in Canada, the US and Europe. When recruitment information becomes available we will update the clinical trails page of our website.

April: New technology allows people to communicate using brain waves

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 conducting a clinical trial to test 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 placement of two sensors into the areas of the brain that support with motor and cognitive function. Researchers will be monitoring 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. The team in Ottawa is one of many BCI groups working globally to improve communication for people living with ALS. If proven to be safe and effective, the impact of this type of technology could be significant, for example allowing people with limited motor function to control computer cursors, wheelchairs or even play video games. This study, which was first posted on in April 2017, is not yet recruiting.

May: Approval of a second ALS treatment, edaravone, in the United States

On May 5, 2017 the United States Food and Drug Administration approved edaravone (also referred to as Radicava® and Radicut®) for the treatment of ALS. Edaravone is the first drug to be approved by the FDA for the treatment of ALS since the approval of riluzole over 20 years ago. The results of a Phase 3 clinical trial showed that for a certain segment of people living with ALS – those who are early in their ALS disease progression with mild symptoms and a good breathing capacity – treatment with edaravone may help to slow functional decline. ALS Canada is in conversation with Health Canada and with Mitsubishi Tanabe Pharma America about the status of edaravone and the need for Canadians to have equitable access to this drug. Canadians interested in learning more about edaravone are advised to connect with their neurologist. Further information is also available here.

October: Phase 2 clinical trial of pimozide now recruiting in Canada

Pimozide is a medication originally used to treat schizophrenia that has more recently shown promise in treating ALS. Pimozide is thought to work by strengthening the electrical connections between muscles and nerves, allowing muscles to function better. Preclinical studies in Canada using worms, fish and mice engineered to have a human form of ALS, showed that treatment with pimozide increased mobility and slowed progression of the disease. Due to the positive results of these laboratory studies, researchers in Calgary conducted a small clinical trial consisting of 25 human volunteers living with ALS. That trial helped to determine a dose that was safe enough for testing in a larger scale clinical trial to hopefully observe whether treatment with pimozide slows the progression of ALS in humans. This Phase 2 clinical trial, supported by an ALS Canada-Brain Canada Arthur J. Hudson Translational Team Grant, began recruiting in Calgary in October and will eventually be enrolling participants at eight clinics across Canada. You can find up to date information on participating sites here.

October: First patients enrolled in Phase 3 clinical trial of NurOwn®

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 be able to repair and replace tissue within the human body. Using stem cells, BrainStorm Cell Therapeutics Inc. is conducting a Phase 3 clinical trial to test 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). On October 16, 2017, BrainStorm Cell Therapeutics Inc. announced that the first patients have been enrolled in the Phase 3 clinical trial of NurOwn®. Researchers will be monitoring participants to assess the safety and ability of NurOwn® to slow the progression of ALS. Final results are expected by mid- 2019.

November: Disappointing results from Phase 3 clinical trial of tirasemtiv

On November 21, 2017 negative results from the phase 3 clinical trial of the muscle activating drug tirasemtiv were announced. This international clinical trial, sponsored by pharmaceutical company Cytokinetics, involved 743 participants from 81 investigative centers in 11 different countries. Researchers had hoped that tirasemtiv, a drug previously shown to increase muscle strength while also prolonging the amount of time before muscles become fatigued, would help to improve breathing in people with ALS as well as slow progression of the disease. Unfortunately, no difference was observed between participants who received tirasemtiv versus those who received placebo, indicating that tirasemtiv had no clinical benefit. Although disappointed by the results, Cytokinetics has expressed renewed commitment to developing treatments for people living with ALS. Cytokinetics is hopeful that a next generation muscle activating drug, CK-2127107, which is currently in a Phase 2 clinical trial, may represent a promising new strategy for the treatment of ALS.

November: ALS Canada awards $3 million for 12 new research projects

The ALS Canada Research Program, which is supported by the generosity of our donors and partnerships with ALS Societies across Canada, funds peer-reviewed research grants and fosters collaboration amongst Canadian researchers. After rigorous scientific assessment by a panel of global ALS experts, ALS Canada announced 12 new exciting research projects being funded in 2017. The announcement was made in partnership with the Brain Canada Foundation, which through the Canada Brain Research Fund (with financial support from Health Canada) is co-funding the largest grant of $1.8 million. You can learn more about these exciting new projects here.

Continued hope for the future

At this year’s International Symposium on ALS/MND in Boston from December 8-10, a record-setting 1,200 researchers and clinicians gathered together to share their work and hopefully continue this unprecedented momentum in ALS research. While there is still a lot left to learn, we are more hopeful than ever that the next several years will see more treatment options with the potential to significantly improve the lives of people living with ALS.

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