A New Drug For A Relentless Brain Disease
People participate in one last ice bucket challenge during the last annual "Plunge 4 Pete" on what would have been Pete Frates 35th birthday on Dec. 28, 2019 in Gloucester, MA. Jessica Rinaldi/Boston Globe via Getty Images hide caption
People participate in one last ice bucket challenge during the last annual "Plunge 4 Pete" on what would have been Pete Frates 35th birthday on Dec. 28, 2019 in Gloucester, MA.
Jessica Rinaldi/Boston Globe via Getty ImagesALS is a disease that gradually destroys the nerve cells in the brain and spinal cord we need for voluntary movement. Most people diagnosed with amyotrophic lateral sclerosis, also known as Lou Gehrig's disease, die within two to five years of diagnosis.
There is no cure for ALS, but as of September, there is a newly approved medication that may slow down the disease and extend patients' lives. The drug, called Relyvrio, got its start with a couple of college students, some "ice bucket challenge" money, and a new approach to targeting this disease. The U.S. Food and Drug Administration greenlit Relyvrio based mostly on a single, relatively small study. They approved it over the objections of some FDA advisors, who were not persuaded that the drug works.
Today, NPR's neuroscience correspondent Jon Hamilton checks in with Emily about this tough call for regulators: how do you weigh promising but limited evidence – and the pleas of patients desperate for hope – against the backdrop of a 100% fatal disease with hardly any other treatment options?
This episode was produced by Thomas Lu, edited by Gabriel Spitzer and fact checked by Margaret Cirino. The audio engineer was Gilly Moon. Our Senior Supervising Editor is Gisele Grayson. Beth Donovan is our Senior Director of Programming and Anya Grundmann is our Senior Vice President of Programming.