International Research Updates


Today Biogen Idec reported results from a Phase 3 trial investigating dexpramipexole (dex) in people with amyotrophic lateral sclerosis (ALS). The company said that the trial failed to show slowing of functional decline or improved survival and failed to demonstrate efficacy. Based on these results, the company is discontinuing development of dexpramipexole in ALS.
Mutations in the gene for an enzyme called valosin-containing protein (VCP) are known to directly cause a disease called Inclusion Body Myopathy associated with Paget’s disease of bone and frontotemporal dementia.
Belgian scientists, under the direction of Dr. Wim Robberecht of the University of Leuven, have discovered that motor neuron degeneration is strongly affected by a protein called Epha4 and humans with ALS that have non-functional Epha4 have delayed disease onset and live considerably longer.