ALS: New treatment extends lives

WGEM News at Five
Updated: Dec. 1, 2022 at 5:19 PM CST
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BOSTON, Mass. (WGEM) – Every year in the United States, about 5,000 people are diagnosed with ALS, also known as Lou Gehrig’s disease. There is no cure, and patients often die within two years of receiving a diagnosis. A social media movement several years ago brought ALS to the forefront while raising millions of dollars for research. Now, one of the drugs developed as a result of that funding has just been FDA approved for use.

In 2014, the “Ice Bucket Challenge” was all over social media. People challenged their friends to dump ice water over their heads and then make a donation to the ALS Association. A portion of the $115 million raised went to research and contributed to the development of a new ALS drug.

Dr. Sabrina Paganoni, MD, PhD, at Massachusetts General Hospital, was the lead investigator on the clinical trial that led to approval of this new therapy.

“The new drug is called AMX 35, and it’s a combination of two different drugs.” Dr. Paganoni explains.

Now marketed as Relyvrio, the drug comes in a powder form that patients can drink or take by feeding tube. It works by slowing the loss of spinal and brain nerve cells.

Dr. Paganoni adds, “Things that we do every day that we take for granted, like talking or swallowing or walking or climbing stairs, people who took the drug did better on those functions for longer.”

Patients taking the drug also lived, on average, five months longer. Giving some valuable time.

“People want to see their kids graduate from school or they want to see their first child get married. So, really five or six months could be the difference between making it to that event or not,” Dr. Paganoni emphasizes.

One drawback that has been a major concern for patient advocates is the cost of the medication – around $158,000 a year. The company that developed the drug says the cost to patients will be lower because they anticipate insurance will cover it. Relyvrio is the third FDA-approved drug for ALS and its symptoms.

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