By Sy Mukherjee
July 31, 2017

Veteran actor, director, and playwright Sam Shepard—star of films such as the space drama The Right Stuff and war epic Black Hawk Down, as well as the scribe behind classic plays like Buried Child (which won him a Pulitzer prize) and True Westdied last Thursday following a battle with amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s disease, according to his spokespeople. He was 73.

Shepard’s battle with ALS wasn’t public knowledge until Monday. But the rare, progressive, and degenerative disease afflicts some 20,000 Americans in any given year (including 6,000 new annual diagnoses). Patients lose the ability to control their motor functions as nerve cells that connect the brain to various muscles decay, eventually leaving many unable to eat or breathe. It can amount to a death sentence; there is no cure, and many patients die within five years of diagnosis.

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There has been some progress on fighting ALS in recent years, though. The disease was newly thrust into the public limelight in 2014 when the nonprofit ALS Association’s (ALSA) “Ice Bucket Challenge” fundraiser became a veritable social media craze and raised millions of dollars ($115 million in just eight weeks, to be exact).

While that effort was derided by some as a stunt that would do little to help ALS research, it was actually credited with helping lead to the discovery of a new gene associated with ALS almost exactly one year ago.

More recently, the Food and Drug Administration (FDA) approved the first new ALS drug in 22 years in May—Mitsubishi Tanabe Pharma America’s treatment Radicava. It’s just the second Lou Gehrig’s therapy on the U.S. market besides French pharma giant Sanofi’s Rilutek.

But Rilutek isn’t a cure despite its potential to manage ALS symptoms. And its limits highlight the enormous challenges to creating drugs for rare, intractable diseases.

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