Last month, not all news was bad news; the FDA approved a new drug called endaravone to treat those who suffer from ALS (amyotrophic lateral sclerosis). The neurological disease, also known as Lou Gehrig’s disease, is diagnosed in over 5,000 Americans each year, and doctors are still searching for a cure.
ALS is a degenerative brain disease that eventually deteriorates a person’s voluntary muscle control, rendering a person unable to walk, talk, eat, or breath independently. On average, people diagnosed with the disease live approximately 2-5 years, though there are many cases of people who live with the disease for much loner; a famous example is the brilliant Steven Hawking.
The medicine doesn’t cure ALS; but it can slow the disease down and help with symptoms; potentially increasing both the quality and quantity of a person’s life. So far, this drug, brand named Radicava, is only the second drug in history to be approved by the FDA specifically for ALS.
The announcement of its approval has given many patients and doctors new hope for the future of ALS. Because there are such limited treatment options for ALS sufferers, there are many who could potentially benefit from the latest pharmaceutical offering.
However, there is a catch; treatment with Endaravone is expensive and intensive, which means that realistically the treatment will be inaccessible for many. The drug must be administered intravenously at a clinic, and a single transfusion costs over $1000. Since patients need roughly 12-15 infusions a month, a year of treatments could cost a patient $150,000.
The company that created the drug, Mitsubishi Tanabe Pharma Corp., reports that they are currently negotiating with health insurance companies to make the drug an option for all patients diagnosed with ALS.
This exciting development is cause for celebration; but we hope that it is only the first (or second) step toward a better understanding of ALS and the discovery of a cure. In the meantime, be grateful for science (and Endaravone)!
H/T: Science Alert
