Has anyone heard of any updates on the Biostrophin trial going on? Dr. Mendell, Samulski and Xiao Xiao are the ones spear heading this trial. It made big news when it was starting and now there has been no mention.
I have emailed a few times and the first emails did receive a response. However, I emailed the same day as I posted this and have yet to receive an email. I can't remember if there was an update at the annual conference.
I just read this on the Bio site and I am praying this is for real. Is biostrophin the same as utrophin?
BMN-195 - Utrophin upregulator for Duchenne Muscular Dystrophy:
BioMarin is in the final stages of preparing the regulatory filing and
expects to initiate a Phase I trial by the first quarter of 2010.
BMN-195 is an orally available small molecule which may upregulate
utrophin, a potential substitution for the missing dystrophin protein
in DMD patients.
Biostrophin is not an utrophin upregulator. It is a miniaturized dystrophin gene that is hoped to be functional. If it worked, and it looks like it doesn't, the boys would be infected with a virus that would alter their genes to include the new dystrophin gene. I am saying that it doesn't look like it works because it's been years since any further real news has come out afer it was first administered. The maker, Asklepios, has not even bothered to update its website on this product since 2006. http://www.askbio.com/news.html
As you note in your post, Biomarin makes an utrophin upregulator that has gone from being off the radar to Phase I clinical trials over that same time period. I didn't use to buy into the idea behind utropin upregulation because the boys who have naturally high utrophin levels do better, but only by a little bit, than the rest of the DMD population.
However, I was told by my son's doctor, Kevin Flanigan that the utrophin upregulators will increase the utrophin levels up far beyond the highest naturally occuring levels. The Phase I test for utrophin upregulators will only be in healthy adults to find out whether these elevated levels are too toxic.