Short Wave A New Drug For A Relentless Brain Disease
Oct 5, 2022
Jon Hamilton, a seasoned neuroscience correspondent, dives into the world of ALS and the newly approved drug Relyvrio. He shares the inspiring story of two college students who turned ice bucket challenge funds into a potential lifeline for ALS patients. The discussion highlights the cautious optimism surrounding Relyvrio, reflecting on the mixed reactions from experts regarding its effectiveness. Hamilton also addresses the ethical dilemmas in drug approval processes, especially when facing a 100% fatal disease and the desperate need for treatment options.
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Amilix's Origins
- Two Brown University undergraduates, Josh Cohen and Justin Klee, formed Amilix to develop ALS treatments.
- They focused on a new approach targeting later-stage symptoms with a combination of two existing products.
Relyvrio's Initial Results
- Amilix's drug, Relyvrio, showed promise in a small study, extending lifespan and improving function in ALS patients.
- Despite this, an FDA advisory committee initially voted against approval due to the study's limited size.
Patient Advocacy and FDA Approval
- Following the initial rejection, ALS patients and families advocated for Relyvrio's approval, emphasizing their need for treatment options.
- The FDA reconsidered and eventually approved the drug after this public pressure and calls for wider access.