Promising New Treatment for ALS

ALS, commonly known as “Lou Gehrig’s disease,” is named for the famous Yankee first baseman who succumbed to the disease at the age of 38, in 1941.

Amyotrophic lateral sclerosis (ALS) is a neurodegenerative disease that progressively paralyzes the skeletal muscles. ALS affects nerve cells in the brain and spinal cord, causing nerves in the muscle to die, thus affecting voluntary muscle movement. On average, people with the disease die within two to five years after being diagnosed. Very little is still known about ALS, including its cause(s), or why it strikes some people and not others. There is no effective cure for ALS; the medications riluzole (approved in 1995) and edaravone (approved in May 2017, the first drug approved specifically for ALS in 22 years), have modest effects.

Old Drug, Repurposed. However, the future of disease treatment may be about to change. Canadian scientists from the University of Montreal Hospital Research Centre and the Cumming School of Medicine at the University of Calgary have discovered that pimozide, a drug used to treat schizophrenia, may be effective in treating ALS. Neuroscientists Alex Parker, PhD and Pierre Drapeau, PhD, first identified and confirmed the potential of pimozide intwo model organisms: a tiny worm and zebrafish.

How They Did It. They used the worms to create a high-volume system that enabled them to screen hundreds of drugs to see how they affected the worms’ symptoms. In all, they screened 3,850 existing drugs and found that 24 affected the worms’ activity. The most promising drugs were the antipsychotic drugs, notably pimozide. Lawrence Korngut, MD, director of the Calgary ALS and Motor Neuron Disease Clinic, used mouse models in his research, arriving at the same results.

Positive Results. Preliminary results from six years of research show that the drug could stabilize progression of ALS. The drug was found to alleviate symptoms of ALS in animal models (worms, zebrafish, mice), and most recently, in humans, in a limited clinical trial. The trial revealed that the optimal dose for ALS patients was much lower than for patients with schizophrenia, and the drug also mitigated some adverse side effects, such as restlessness and a need to move constantly.

Pimozide apparently works by strengthening the electrical connections between nerves and muscles, says Dr. Korngut. All scientists involved in the research caution that larger clinical trials are needed. A Phase 2 trial is planned, and will enroll 100 patients in two groups: a placebo group and a low-dose pimozide group.

“There is a lot of reason to be optimistic about the research going on in the ALS community right now,” Dr. Korngut concluded.

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