Remember the ALS Ice Bucket Challenge? Well, apparently the more than $200 million raised worldwide through the challenge helped fund an experimental medication that may slow the progression of amyotrophic lateral sclerosis, or ALS.
Although this medication is not a cure, it may slow the inexorable disability caused by ALS, which destroys the nerve cells that control the muscles that allow us to move, speak, eat and even breathe. The treatment targets two cellular structures damaged by the disease: the mitochondria, which are the cells’ power plants, and the endoplasmic reticulum, the cellular dump trucks that cart away waste that can gunk up the cells’ machinery.
The multicenter, randomized, double-blind study is the second step — a phase 2 trial — in a three-step process required by the Food and Drug Administration for drug approval. In a double-blind study, neither the patients nor the researchers know who is receiving the drug. If a phase 2 study generates positive results, the FDA typically requires a larger and longer phase 3 trial.
During the six months of the study, which recruited 137 people with ALS, the control group of patients saw a 25 percent better functional outcome than the placebo group. Dr. Sabrina Paganoni, a neuromuscular specialist at Massachusetts General Hospital’s Sean M. Healey & AMG Center for ALS, said that if the drug is approved, it would likely be just one part of a combination of medications that could keep ALS at bay.