Arimoclomol Safe, Well Tolerated In ALS

The Los Angeles-based company CytRx has announced that its experimental drug arimoclomol is safe and well tolerated in people with amyotrophic lateral sclerosis (ALS). The study involved 84 people and lasted 12 weeks.

Arimoclomol’s developers believe this orally administered compound activates proteins called “chaperones,” which assist other proteins to fold into their correct shapes.

There were no statistically significant differences noted on measures of disease progression, but the study wasn’t designed to detect these.

Arimoclomol appears to have entered the fluid that surrounds the brain and spinal cord, which is considered important for treating ALS.

If regulatory approvals are obtained, CytRx plans to launch a trial early in 2007 that will likely involve some 390 ALS patients, last 18 months, and be designed to detect changes in disease progression.