Jason, have you heard of this before now? Their website looks really thought-out.
Clare,i too have had a look at the content of this site.I will admit most of it is way over my head,i not being much of a sciences person.However,their concept sounds pretty interesting and if it is an innovation able to accomplish what they seem to be implying,well,then it is something worth keeping an eye on.
Concept looks interesting, but the website only calls out funding sources outside of the Duchenne non-profit community, which makes me wonder why. All the non-profits have scientific advisory boards with some pretty sharp and informed people. If it was a promising concept for Duchenne, I'd imagine someone within our community would be funding it.
Perhaps Sharon can comment.
This is the first time I have heard of this drug and think its worth PPMD looking into. I see no reason to doubt them at this time the research seems well sited on the web site. I would be interested in Sharon and PPMD's science advisor's opinions of the reasearch on this drug.
TV interview in Doctor working on the research
Dr. Frederick Sachs:
Yes, we have reviewed this project numerous times (I first investigated some of this data when I was still at MDA) and Dr. Sachs spoke at the PPMD conference two years ago. Dr. Sachs has discovered an interesting molecule in spider venom that may block a particular kind of channel in muscle cells. I can't say much beyond this except to point you to TREAT NMDs Therapeutic Advisory Committee (TACT) review: http://www.treat-nmd.eu/resources/tact/reviews/past/gsmtx4/. Although this summary is somewhat technical, the gist is that the drug needs a lot more data behind it before it's clear if this is a feasible approach to treating Duchenne.
PPMD supports the TACT meeting and relies on the outcomes of these reviews, in part, for making funding decisions. We always have more requests for funding than we are able to accomodate so we have to make a decision about what projects we think are most likely to succeed based on the available data.
I think it's likely that there has been a small buzz about this particular drug right now because of the release of the next Spider Man movie.