Multicenter Trial Of Arimoclomol in ALS To Begin

A multicenter clinical trial of the experimental compound arimoclomol, developed by CytRx (www.cytrx.com), a Los Angeles biopharmaceutical company, is set to begin testing in people with amyotrophic lateral sclerosis (ALS) during the first half of 2006.

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 in a recently completed phase 1 study.

CytRx received a green light for a large-scale, phase 2 study from the U.S. Food and Drug Administration on Sept. 21 and has begun identifying potential trial participants at some of its sites. The FDA has 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 for a 12-week, 80-patient, phase 2a study are Robert Brown, who directs the MDA ALS Center at Massachusetts General Hospital; Merit Cudkowicz, an MDA research grantee and MDA-associated clinician at that institution; and Jeremy Shefner, director of the MDA ALS Center at SUNY Upstate Medical University in Syracuse, N.Y.

The study is likely to take place at eight to 10 U.S. sites. Participants will receive either a placebo (inactive substance) or one of three arimoclomol dosage levels.

Depending on the results of the phase 2a study and FDA approval, an 18-month, phase 2b study with approximately 300 participants at 25 sites, is planned.

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