Researchers have found that a drug used to treat schizophrenia has the potential to slow the progression of amyotrophic lateral sclerosis (ALS) or Lou Gehrig’s disease, a fatal neurodegenerative condition for which there is currently no effective cure.
This neurodegenerative disease normally leads to a progressive paralysis of the skeletal muscles and, on average, three years after the onset of symptoms, to death.
After six years of research on several animal models and a few patients, the researchers discovered that the drug, pimozide, stabilises the disease in the short term.
The study, published in the journal JCI Insight, found the medication to be safe.
“This medication alleviates the symptoms of ALS in animal models,” said Alex Parker, a researcher at University of Montreal Hospital Research Centre in Canada.
“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,” Parker added.
Although pimozide has been well-known for 50 years, recent studies uncovered genetic links between schizophrenia and ALS.
The researchers said that their next step is to conduct a phase-2 clinical trial on 100 volunteers in nine hospital centres across Canada.
“At this stage, people with ALS should not use this medication,” Lawrence Korngut, Associate Professor at the Cumming School of Medicine at the University of Calgary in Canada, said.
“We must first confirm that it is really useful and safe in the longer term. It is also important to be aware that pimozide is associated with significant side effects. Therefore, it should only be prescribed in the context of a research study,” Korngut added.