A phase 1 clinical trial of the experimental drug ISIS-SOD1-Rx in patients with the SOD1-related type of familial (inherited) amyotrophic lateral sclerosis (ALS) is expected to begin before the end of 2009 at Washington University in St. Louis, Massachusetts General Hospital in Boston and four additional U.S. sites.
MDA supported Timothy Miller at Washington University in St. Louis to work with Isis Pharmaceuticals of Carlsbad, Calif., to develop ISIS-SOD1-Rx (formerly ISIS-333611).
The trial will test the safety and tolerability of this "antisense" compound, which is designed to block production of the SOD1 protein in people who have developed ALS because of mutations in the SOD1 gene. Such mutations cause approximately 1 percent to 3 percent of all ALS cases.
The investigators will infuse ISIS-SOD1-Rx into the fluid that surrounds the brain and spinal cord, a delivery method that targets the cells that produce the toxic SOD1 protein.
Details of genetic testing will be discussed with potential study participants.