Arimoclomol Trial Open to ALS Patients

A multicenter clinical trial of the experimental compound arimoclomol, developed by CytRx (www.cytrx.com), a Los Angeles biopharmaceutical company, is set to now open to people with amyotrophic lateral sclerosis (ALS) at 10 U.S. medical centers.

The CytRx-sponsored study is designed to include 80 people with ALS who are at least 18 years old, not dependent on mechanical ventilation, have had ALS symptoms for no more than five years, and meet other study criteria.

The investigators will test the safety of arimoclomol; will analyze how the drug is metabolized; and will see what effect, if any, it has on disease progression. Some participants will receive the study drug, while others will receive a placebo (an inactive, look-alike substance).

Arimoclomol is a small molecule that’s designed to stimulate a natural cellular repair pathway by activating compounds called “molecular chaperones.” It has been shown to extend life in ALS-affected mice and was well tolerated in healthy human volunteers.

For additional background information on arimoclomol, see “Arimoclomol Multicenter Trial” in the December 2005 issue of the MDA ALS Newsmagazine; and “Multicenter Trial,” in Research News on the MDA Web site.

CytRx received a green light for this trial from the U.S. Food and Drug Administration on Sept. 21 and has begun identifying potential trial participants. The FDA also granted arimoclomol “fast-track” status, a program begun in 1997 to hasten review of potential treatments for serious or life-threatening diseases.

The principal investigators are Merit Cudkowicz, an MDA research grantee and MDA-associated clinician at Massachusetts General Hospital in Boston; and Jeremy Shefner, director of the MDA ALS Center at SUNY Upstate Medical University in Syracuse, N.Y.

For details and contact information, see www.clinicaltrials.gov, a Web-based trial posting service overseen by the National Institutes of Health. Enter “arimoclomol” into the search box.