Hi -

This may be one for Marsha or someone more clinically minded than myself!

I have been reading this article about Stem Cell Research at UC Davis -

[www.ucdmc.ucdavis.edu]

One sentence that jumped out at me was - " The awards are specifically designed to support collaborative research that will bring potential therapies to the Food and Drug Administration for approval within four years"

Are they actually saying that they could begin treating patient's with HD within 4 years?

Kathy x

Yes they are! They've done primate research and need to get an Investigational New Drug Application into the FDA and get it approved before they can start with a Phase One trial. This grant will enable them to get the application done.

But this group suggested in November 2009 that they were "12 months" from human phase I trials, which have yet to begin. Given that they haven't published efficacy in even a mouse, it's probably safe to assume that their goal of "treating patients in 4 years" will be as successful.

Good to see you Jeff!!!! My thoughts on it are this. There's lots of dif ways of doing stem cells now, but they are going to get the cells from a donor, to me that means anti rejection drugs possibly. The other problem is this. With the hd gene still at play, stem cells will only work for a certain amount of time. They need to do stem cells WITH gene silencing, is what i think. So i think any research is great, but i don't feel any excitement about this one myself

Actually this trial is not intended to be restorative but neuroprotective. They are using mesenchymal stem cells which don't cause rejection problems and they are going to be engineered to deliver BDNF.

The researchers have not been able to follow their projected timeline but there again, they wouldn't be the only HD group to underestimate how long it takes to be ready for clinical trials. They haven't published an efficacy article but another group of researchers doing the same thing in mice did report good results. [www.hdsa.org]

Any news is good news!

Thanks for the replies.

So does anyone know of any clinical trials that are due to start in the next 12 - 18 months that could offer an actual "cure" or stall the disease?

I know there are late stage trials for creatine, COQ10 etc, and, of course Huntexil - but are we on the verge of seeing at least a few trial participants "cured"?

Kathy x

I am not sure when a clinical trial will start for RNAi or antisense but it's possible that it could be sometime within the next two years. Another possibility within the next two years is a KMO inhibitor. I have an article that should go up on the HDSA website sometime today about this. It looks very promising; the results in the mice are the best we've seen for a drug. The researcher, Dr. Paul Muchowski, found an inhibitor that worked in principle but was too unstable to use as a medication. Drug development is expensive and often delays a promising approach but not this time -- he asked his father, a chemist, to do it and he did!

Marsha-

I looked into both of the advances you referred to. Re: the KMO inhibitor, it seems that KMO may be a byproduct of tryptophan digestion..

should people look into eating less tryptophan? two HUGE components of my sib's diet are high in that amino- turkey and bananas. and when i say HUGE components, we are talking maybe 8oz of turkey and 3-4 bananas a day...