"molecular chaperone" protein drug for neurodegenerative diseases
 

http://www.rttnews.com/Content/Break...um=2682_3781_2

FDA Lifts Clinical Hold On CytRx's ALS Drug Arimoclomol - Update
12/2/2009 12:28 PM ET

(RTTNews) - Wednesday, drug developer CytRx Corp. (CYTR: News ) said the U.S. Food and Drug Administration lifted a clinical hold on its molecular chaperone drug candidate arimoclomol for the treatment of amyotrophic lateral sclerosis, commonly known as ALS or Lou Gehrig's disease. FDA removed the two-year ban after accepting a revised clinical trial protocol.

FDA placed the phase 2b trial with arimoclomol on hold in January 2008 citing the need for additional analysis from previously-completed animal toxicology studies... In September 2005, the FDA granted Fast Track designation for arimoclomol for the treatment of ALS. The drug candidate also got orphan status from the FDA in May 2005 and from the European Union in October, 2006.

CytRx now estimates that data from the trial protocol accepted by the FDA would be available 18 months after the trial starts...

Daniel Levitt, chief medical officer of CytRx, said, "The revised protocol allows for a safety assessment and a preliminary efficacy evaluation in ALS patients treated with arimoclomol at levels up to four times the dose administered in our prior Phase 2a clinical trial and open label extension study...

Steven Kriegsman, chief executive officer said, "Arimoclomol's underlying molecular chaperone amplification method of action has shown promising clinical and preclinical study results in inhibiting the progression of ALS, a debilitating disease that currently lacks effective therapeutic treatment. We believe that this drug candidate has the potential to be a breakthrough drug with application in a number of other neurodegenerative diseases, such as Alzheimer's disease, Parkinson's disease, and Huntington's disease, and as a treatment for stroke recovery and neuropathy."..

(Explanation of molecular chaperone" proteins:
http://www.cytrx.com/molecular_chape...egulation.html

and:

http://www.cytrx.com/neurodegenerati...e_program.html
CytRx believes arimoclomol represents a potentially powerful breakthrough in the treatment of multiple neurological disorders including Amyotrophic Lateral Sclerosis (ALS or Lou Gehrig's disease), Alzheimer's, Huntington's and Parkinson's diseases, and stroke. Arimoclomol is believed to function by amplifying "molecular chaperone" proteins, normally found in all cells of the body and thought to enhance the cell's natural ability to, in essence, mend damaged, misfolded proteins. Arimoclomol may thus provide cellular protection from misfolded, toxic proteins, repairing those that are believed to cause many diseases, including ALS. Arimoclomol has demonstrated the ability to protect motor nerves subjected to physical trauma and to accelerate the regeneration of previously damaged nerves in animals. These observations provide additional evidence that, in addition to ALS, it is possible that the drug may have a much broader application profile in the area of neurodegenerative diseases. Arimoclomol is an orally available small molecule compound that has been shown to be well tolerated in seven previous Phase I clinical trials with healthy human subjects