September 28, 2005

CytRx Corporation Testing 'Fast Track' ALS Drug in Clinical Trial

By Gary Wosk, ALSA Staff Writer

People with ALS can enroll in a Phase IIa clinical trial for arimoclomol, which slowed down the progression of the disease in a mouse model of ALS (amyotrophic lateral sclerosis), also commonly referred to as Lou Gehrig’s disease. The drug was developed by the biotechnology company CytRx.

CytRx, engaged in the development and commercialization of human therapeutics, needs to recruit 80 people for the clinical trial at 10 centers across the United States. According to the company, the drug was “well absorbed and well tolerated in two Phase I clinical trials in healthy volunteers” and was granted orphan drug status by the FDA earlier last spring.

“We are very excited about the potential of this compound to extend survival in patients and encourage participation in the trial,” said ALSA Science Director and Vice President Lucie Bruijn, Ph.D.

U.S. regulators also notified CytRx that arimoclomol has been given a “fast track” designation, a process under the FDA Modernization Act of 1997 that “facilitates the development and expedites the review of a new drug that demonstrates potential to address a significant unmet medical need for the treatment of a serious or a life-threatening condition.”

Arimoclomol is one of CytRx’s three orally-administered, small molecule compounds believed to function by stimulating a normal cellular protein through the activation of “molecular chaperones.” Since damaged proteins called aggregates are thought to play a role in many diseases, CytRx believes that activation of molecular chaperones could have therapeutic efficacy for a broad range of issues.

The co-principal investigators of the trial are Merit Cudkowicz, M.D.M. Sc., professor of neurology at Harvard Medical School, Robert H. Brown, Dphil, M.D., professor of neurology at Harvard Medical School, and Jeremy Shefner, M.D., professor of neurology at SUNY Upstate Medical University.

“We are very excited to move forward to begin testing of arimoclomol for ALS,” Cudkowicz said. “The work from Dr. Linda Greensmith’s laboratory supports moving forward to develop arimoclomol for ALS.”

For more information, contact Liz Simpson at the Massachusetts General Hospital Neurology Clinical Trials Unit Coordination Center at (617) 726-9122.

To read a summary of animal findings with the drug arimoclomol, please click here.

For more information on other drug trials, click on ALSA's Drug Development Update Information.