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New Drug Could Extend Life for People With ALS
  • Robert Preidt
  • Posted October 16, 2020

New Drug Could Extend Life for People With ALS

An experimental drug combination lengthens survival for patients with amyotrophic lateral sclerosis (ALS), new research shows.

A previous clinical trial found that the two-drug combo -- called AMX0035 -- slowed progression of the neurodegenerative disease over six months.

The new clinical trial of 137 patients with the disease, also known as Lou Gehrig's disease, found that those who took AMX0035 lived a median of 6.5 months longer than those who received a placebo. Median means half lived longer, half for less time.

The study, published Oct. 16 in the journal Muscle & Nerve, was conducted by researchers at Massachusetts General Hospital in Boston and Amylyx Pharmaceuticals, Inc., which makes the drug.

According to The New York Times, the drug combination was dreamed up by two Brown University students seven years ago. It combines an existing supplement and a medication for a pediatric urea disorder.

There is no cure for ALS, in which increasing damage to brain and nerve cells in the spinal cord results in a progressive loss of one's ability to move, speak, eat and even breathe.

AMX0035 is designed to reduce the death and dysfunction of motor neurons, according to the new report.

Findings published last month in the New England Journal of Medicine reported that the drug slowed progression of ALS paralysis by about 25% more than a placebo, according to the Times.

The latest results "provide substantial evidence supporting the role of AMX0035 for the treatment of ALS," said study leader Dr. Sabrina Paganoni, an investigator at the Healey and AMG Center for ALS at Mass General. "Next steps will depend on ongoing discussions with regulatory agencies," she said in a hospital news release.

Senior author Dr. Merit Cudkowicz, director of the Center for ALS, described the study as groundbreaking.

"This is one of the first studies to show effect on both function and survival," she said. "We are hopeful that this is just the beginning of many new treatments for ALS."

Both researchers are on the faculty at Harvard Medical School. Paganoni is an assistant professor of physical medicine and rehabilitation and Cudkowicz is chief of neurology.

More information

The ALS Association has more on amyotrophic lateral sclerosis.

SOURCE: Massachusetts General Hospital, news release, Oct. 16, 2020
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