Arimoclomol May Combat Misfolded Proteins in ALS

A trial of arimoclomol, an experimental compound developed by Cytrx (www.cytrx.com), a Los Angeles biopharmaceutical company, will likely begin this summer or fall, pending its approval by the U.S. Food and Drug Administration. The FDA has classified arimoclomol for ALS as an “orphan drug,” a status that allows Cytrx some financial benefits as an incentive for developing a drug for a rare disorder.

Neurologist Robert Brown, who directs the MDA ALS Center at Massachusetts General Hospital in Boston, is slated to be the trial’s principal investigator.

“Arimoclomol amplifies cellular methods used to combat protein misfolding,” said David Haen, director of business development at Cytrx, citing studies in mice with genetic ALS that showed the drug increased their life span by 22 percent. A key factor in the mouse studies, he said, is that arimoclomol was effective even after the onset of ALS symptoms, which hasn’t been the case with many compounds tested.

Dairin Kieran at University College London (England) and colleagues published the results of the mouse experiments in the April 2004 issue of Nature Medicine.