Drug Research
Drug Discovery & Development

Canadian researchers discover medication to treat amyotrophic lateral sclerosis

PBR Staff Writer Published 20 November 2017

Canadian researchers have discovered an effective medication for the treatment of individuals with amyotrophic lateral sclerosis (ALS) or Lou Gehrig’s disease.

Researchers from the University of Montréal Hospital Research Centre (CRCHUM) and the Cumming School of Medicine (CSM) at the University of Calgary have discovered a medication that can treat individuals with ALS.

Université de Montréal professor and CRCHUM researcher Alex Parker said: “This medication alleviates the symptoms of ALS in animal models.

"Riluzole and edaravone, the drugs currently used, have modest effects. Other studies must be conducted to confirm our results, but we believe that we’ve found a medication that may prove to be more effective in improving patients’ quality of life.” 

Six years back, Parker genetically modified millimeter-long nematode worms called C. elegans to exhibit aspects of the human form of ALS.

Simultaneously, his colleague Pierre Drapeau also carried out the similar procedure with another animal zebrafish, which is a tiny tropical fish with 5cm long.

The US Department of Defense provided the funding for two scientists to test medications on these worms and fish born with ALS.

With around 25 ALS patients, the first preclinical trial for ALS was launched in 2015.

The initial clinical trial was modest in scope, while the researchers had a first indication of the drug's efficacy only after six weeks.

Loss of control of the thenar muscles situated in the palm of the hand between thumb and index finger is generally one of the first signs of ALS. This function remained stable for patients who took pimozide.

A phase II clinical trial on 100 volunteers will start in next few weeks, which will be carried out in nine hospital centres across Canada.

The study is being conducted to confirm that pimozide is safe and to measure its effect on the progression of the disease and its symptoms and on patients' quality of life over a six-month period.


Image: The limited clinical trial on worms, zebrafish, mice and human subjects conclude that pimozide may be effective in treating ALS. Photo: courtesy of University of Montréal Hospital Research Centre.