Good News on the Research Front

In several articles over the last eighteen months I have mentioned ASC-J9 as another potential treatment for Kennedy’s Disease.

Background:  In 2006, the KDA’s Scientific Review Board recommended the awarding of a research grant to Chawnshang Chang, Ph.D., of the University of Rochester.  Dr. Chang’s research was focused on developing a treatment regimen for Kennedy’s Disease targeting the poly Q-expanded mutant AR.  A few years later, AndroScience purchased the rights to this technology.



Tuesday, AndroScience and NIH announced a grant to move this potential treatment’s research along.  Follow this link (ASC-NINDS) to the PR-Newswire site to read the entire press release.  A summary of the release can be found below.

“AndroScience Corporation Awarded a $3.8 Million, 3-Year Milestone-Driven, Cooperative Translational Research Grant from the NIH to Develop an Oral Treatment for Spinal Bulbar Muscular Atrophy (Kennedy's Disease)

 

SAN DIEGO, Jan. 25, 2011 /PRNewswire/ -- AndroScience Corp. (ASC), based in San Diego, California, announced receiving a $3.8 Million, 3-year milestone-driven, cooperative translational research grant from the National Institute of Neurological Disorders and Stroke (NINDS) of the National Institutes of Health (NIH).  Through a joint research effort with the Neurogenetics Branch of the NINDS, ASC will use the funding to pursue development of an oral drug treatment for spinal and bulbar muscular atrophy (SBMA) or Kennedy's Disease, a rare hereditary neurodegenerative disease, which currently has no approved drug available to patients.  Key pathological features of SBMA include progressive motor neuropathy and androgen insensitivity syndrome caused by a distinctive mutation within the androgen receptor (AR) gene.  ASC has developed a unique platform of therapeutic small molecule drugs, which selectively and potently enhance degradation of the AR protein, termed AR degradation enhancers (ARD enhancers).
 
"Given encouraging pre-clinical results and the clear need for a new therapeutic option for SBMA patients, ASC is excited to continue advancing preclinical development of this promising novel drug candidate," said ASC President Charles Shih, Ph.D. "The funding provided by the NINDS/NIH will significantly propel our efforts in validating ARD enhancers as a disease-modifying therapeutic intervention against such a rare and devastating neurodegenerative illness."
 
This $3.8 Million cooperative translational research grant will leverage expertise from the NINDS and draw upon ASC's innovative approach to targeting the mutant androgen receptor (AR).  The goals of the grant will be to first validate an orally administered ARD enhancer drug is efficacious in the SBMA transgenic animal model, and further, to complete preclinical toxicology, safety pharmacology, and ADME studies necessary in supporting of an IND filing to commence human clinical studies.  To date, ASC has provided robust proof of concept data using an ARD enhancer compound; demonstrating treatment ameliorates cardinal features of SBMA neuromuscular pathology, restores functional activity, and improves survival in a SBMA transgenic mouse model.”
 
This is very good news for those of us living with Kennedy’s Disease because it moves another major project forward to the pre-clinical testing phase.  I will be watching this project closely.