Isis Pharmaceuticals of Carlsbad, Calif., has begun a phase 1 clinical trial of its experimental compound ISIS-SOD1-Rx in people with familial (inherited) ALS caused by toxic SOD1 protein molecules.
“This is a watershed moment,” said R. Rodney Howell, a metabolic disease specialist and chairman of the MDA Board of Directors. “More than 30 people with familial ALS caused by mutations in the SOD1 gene soon will receive infusions of a SOD1 inhibitor directly into their central nervous systems.”
A so-called "antisense" compound, ISIS-SOD1-Rx is designed to block production of the toxic SOD1 (superoxide dismutase 1) protein in people who have developed ALS because of mutations in the SOD1 gene. Antisense compounds are pieces of genetic information that keep other genetic information from being processed.
SOD1 mutations account for approximately 1 percent to 3 percent of all cases of ALS (amyotrophic lateral sclerosis), and about 20 percent of all familial ALS cases.
The SOD1 form of ALS is perhaps the best-understood form of the disease and almost the only form used in animal-based ALS studies
ALS SOD1 Trial: A ‘Watershed Moment’ | Quest Magazine Online
