Has anybody heard anything further regarding this study? It seemed so promising but then..nothing but crickets.

https://www.stjude.org/media-r...logical-disease.html

I think it was stated at the KDA conference last November they have a pharmaceutical development company partner but I don't recall any noteworthy details such as an expected date for start of clinical trials.

Hi Jon. The study is complete and the results of the study have been published. The abstract is at this link https://www.nature.com/articles/nm.4500 and the full text of the article is at this link. https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5975249/

As you know, the results say it was beneficial in a mouse model. I wish I knew more about a possible next step. Might be testing in a more complex organism or even testing in humans, but apparently no pharma company has stepped up to sponsor that kind of testing.

Thanks Todd & Dan for the feedback. Guess I'll keep my fingers crossed but have little hope since there doesn't appear to be any kind of a rush for treatment of a rare disease that provides little drug profit on the back end.

I share your frustration. Testing drugs in humans is very expensive, and getting drugs through the FDA approval process is even more expensive. Over the past ten years, I can think of at least ten drug candidates that were listed as possibly beneficial for SBMA, or shown to be beneficial in a mouse model. Only one or two of them have been tested in humans.

Apart from the expense issues with testing any drug, we also have the problem that the population of men with SBMA is small, therefore the potential market for the drug is small. Would be helpful if a government agency such as the NIH would sponsor a drug test, but apparently they don't have the funding either.