Study identifies potential drug for treatment of SBMA

In March of last year I mentioned this article:

Study identifies potential drug for treatment of debilitating inherited neurological disease

An excerpt of the article can be found below Dr. Taylor's update.

I wrote Dr. Taylor today and asked for an update on the research. Below is his reply.

We have been working with the group in Vancouver at the University of British Columbia who developed the MEPB compound and have identified analogs that have even better potency and drug properties. UBC owns the rights. My understanding is that UBC is discussing a licensing agreement with a drug company to further develop this – meaning they will run independent preclinical trials in a mouse model using the newer compound, and pending those results will initiate human clinical trials. This typically takes ~ 2years.

The following is an excerpt from the original Medical Press article. To read the entire article follow this link: Potential Drug Treatment. The St. Jude's website also has the article:  Study Identifies PDT

As always, I appreciate Dr. Taylor and his team's support for finding a treatment for Kennedy's Disease.

March 15, 2018

Dr. Paul Taylor of St. Jude Children's Research Hospital ...

"... and his colleagues were led to seek drugs to treat SBMA because of findings from a previous study in his laboratory. The study pinpointed a molecular niche in the mutant androgen receptor protein that appeared to be a key to driving SBMA symptoms. However, that niche did not seem to be essential to the normal function of the androgen receptor. The study started with fruit flies genetically engineered to have the human androgen receptor, giving the scientists a living "test tube" to explore the effects of mutating the receptor.

"This identification of a small patch of this protein that appeared to be functionally important for driving the disease, but is not essential for most androgen receptor functions, gave us a potential drug target," Taylor said.

Pharmaceutical companies have been developing drugs to target this small patch, called the "activator function-2" or "AF2" domain. The companies were testing the drugs as possible treatments for prostate cancer, which also involves the androgen receptor. Taylor obtained a collection of the test drugs to evaluate for use with SBMA.

Using the genetically engineered flies, the researchers identified two drugs—whose long chemical names are abbreviated TA and MEPB—that alleviated SBMA symptoms. Then, using mice, the scientists determined that MEPB more effectively reached target tissues in the brain and spinal cord.

For their trials of TA and MEPB as potential SBMA treatments, the researchers developed a new genetically engineered mouse model to more accurately mimic the mutation found in men with SBMA. The transgenic mice showed many of the symptoms of humans with the disease.

Researchers found that MEPB effectively alleviated symptoms of SBMA in the mice. "Treating the mice with MEPB forestalled muscle atrophy and prevented loss of their motor neurons, with recovery of their testicles to normal size," Taylor said. "The treatment also protected their ability to walk and their muscle strength and endurance." ..."