As a cardiologist, Dr. Marc Litt has plenty of arrows in his quiver for patients with heart disease: Medications that thin blood. Drug-coated stents that widen blocked arteries. Implants that replace damaged valves in minimally invasive surgery.

But when the 63-year-old physician was diagnosed with ALS in March 2019 at Massachusetts General Hospital, he was dismayed to see how little medicine had to offer him. Despite at least 50 clinical trials of potential drugs since 1995, only two drugs have been approved to slow the progression of the deadly degenerative neurological disease, and neither is all that effective.

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