New Drug for SMA: Close to a Cure!

New experimental gene therapy for SMA
New experimental gene therapy for SMA(WGEM)
Updated: Jul. 21, 2021 at 3:28 PM CDT
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COLUMBUS, Ohio (Ivanhoe Newswire)— Spinal muscular atrophy, or SMA is a disease that attacks and kills nerve cells in the brain and spinal cord. In the most severe cases, people lose their ability to walk, speak, swallow, and breathe on their own. Babies with the most severe form have not survived past age two. Now, new treatments, including an FDA approved oral medication are giving young patients a second chance.

Five-year-old Donovan Weisgarber can’t get enough of the playground.

His dad, Matt, soaks in every minute. At six weeks old, doctors diagnosed Donovan with type one spinal muscular atrophy. They told the family that their newborn son would quickly waste away.

“But as far as stopping the progress of the disease, there was nothing available. So, we were told, go home and love him for as long as you have him,” shared Matt.

But shortly after his diagnosis, doctors learned about an experimental gene therapy for SMA. Donovan became the fifteenth person in the world to have his missing gene replaced with a synthetic one. Then, last year, Donovan began taking a new oral medication for SMA called Evrysdi . Evrysdi is taken by the mouth, or directly into a patient’s feeding tube. The drug works by increasing a protein that SMA patients are missing.

“This really is a game changer for our field,” said Perry Shieh, MD, PhD, Professor of Neurology and Pediatrics, David Geffen School of Medicine at UCLA.

“In my book, it’s as close to a cure as you can get. And we scientists actually hate using that word cure, but I would say that we’re pretty close,” stated Karen Chen, PhD, CEO, Spinal Muscular Atrophy Foundation.

Matt repeatedly hoists his 48-pound son up in the air—using playtime to ensure Donovan maintains muscular strength while Evrysdi works from within.

“Since he started taking it, we noticed he’s able to stand longer. SMA should not be the thing that does him in,” said Matt.

The FDA approved Evrysdi last August after several clinical trials. In one trial, after 23 months or more of treatment, 81 percent of SMA patients were alive without permanent ventilation. Researchers say that is a noticeable improvement from the typical progression of the disease.

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