Slowing ALS symptom progression
Treatment with dexpramipexole — a novel drug believed to prevent dysfunction of mitochondria, the subcellular structures that provide most of a cell’s energy — appears to slow symptom progression in the neurodegenerative disease amyotrophic lateral sclerosis (ALS). Promising results of a phase 2 trial of dexpramipexole are receiving advance online publication in Nature Medicine. Some preliminary results of the study were presented at the 2009 International Symposium on ALS/MND (motor neuron disease) and the 2010 American Academy of Neurology annual meeting. “Today there are only two FDA-approved drugs used to treat ALS — riluzole, which extends life about 10 percent, and Nuedexta, which treats the emotional instability that characterizes ALS and other neurological disorders,” said Merit Cudkowicz, director of the Harvard-affiliated Massachusetts General Hospital (MGH) Neurology Clinical Trials Unit and ALS Center, lead author of the study. “We need more therapies to slow, halt, and ultimately reverse the course of disease and also...