New treatments for ALS
Until recently, only one drug, riluzole (Rilutek), was approved by the Food and Drug Administration (FDA) specifically as an amyotrophic lateral sclerosis treatment. Rilutek can extend patients’ survival for a few months, but it won’t reverse nerve damage or stall the forward progress of the disease.
In May 2017, the FDA gave its approval to edaravone (Radicava) – a drug that has previously been used to restore mental function in people who’ve had a stroke. Radicava works by cleaning up free radicals – toxic byproducts of cellular processes. Research finds that the drug improves function in people with ALS compared to placebo.
Other new ALS drugs in development work by different mechanisms. Some are gene therapies, meaning they deliver a healthy gene into the body to replace one that is damaged in ALS. VM202, which the FDA has given fast-track status, codes for the production of hepatocyte growth factor (HGF) – a protein that helps regenerate damaged nerve cells.
March 16, 2020
Christopher Nystuen, MD, MBA