Biogen Idec and Knopp Neurosciences announced Aug. 18, 2010, that they have entered into an agreement to continue developing KNS-760704 (dexpramipexole) as an experimental treatment for ALS. The drug helps protect nerve cells under adverse conditions. A phase 2 trial by Knopp showed the drug had favorable effects on motor function and survival in people with ALS. KNS-760704 has received Orphan Drug and Fast Track designations from the U.S. Food and Drug Administration (FDA). See Biogen Idec and Knopp Neurosciences Announce License Agreement for Late-Stage ALS Drug Candidate.
Mutations in the gene for the FUS protein, recently identified as a cause of ALS, have been found to cause the FUS protein to become part of so-called "stress granules" in cells that are subjected to elevated temperatures and other challenges. The findings indicate a potential link between FUS gene mutations and cellular pathways involved in stress responses. See Mutant FUS proteins that cause amyotrophic lateral sclerosis incorporate into stress granules.
At least some of the many ALS-causing mutations in the SOD1 gene appear to interfere with the energy-producing activities of mitochondria in nerve cells. The mutations the researchers tested, which cause SOD1 proteins to misfold, apparently also cause them to stick to a channel on the surface of nerve-cell mitochondria and disrupt the ability of the mitochondria to import needed substances for energy production. The findings could suggest new therapeutic strategies for SOD1-related ALS. See Misfolded mutant SOD1 directly inhibits VDAC1 conductance in a mouse model of inherited ALS and Is amyotrophic lateral sclerosis a mitochondrial channelopathy?