I am really excited about this. We are on our way!

[www.hdlighthouse.org]

Fantastic!!

Thanks Marsha for posting this! I will be praying that this research goes well.

We really appreciate this update Marsha (and all of them for that matter). Thank so much for all you do for us, keeping us in the loop. This news also gives us hope - and that is a good thing.
(o:

Great ,as always

As the resident layman around here, I have to ask:

Is this collaboration based on encouraging research, or is it more akin to "well, let's try something new"

It's based on a solid body of research that we have been following for years. Google RNAi on the Lighthouse and you can read about the cumulative research. We have proof of principle in mouse models and there's a good possibility that this could be developed into a virtual cure for HD.

That's the vibe I got from the article, but HD or not, comprehending journal articles written for the scientific community has never been my strong suit.

If I'm reading correctly, it can prevent the onset of symptoms for those pre-symptomatic, and can halt but not reverse symptoms in those already affected?

So good news for the next generation, and to prevent further suffering of the current generation?

Patty

When the HD gene is silenced in mice, the symptoms are substantially reversed even late in the disease, so hopefully people will actually get better. Another possibility is the use of stem cells, either new ones or inducing one's own to develop, to repair the brain.

Both RNAi and stem cell technology have wider target populations than HD so we can expect work to continue to advance in those areas -- the profits are waiting.

So Marsha, are we looking at 2 years research and then, if successful, on to trials?

[www.hdyo.org]

yes, exactly.

In the meantime there is another pharmaceutical company attempting to advance to non-allele specific RNAi trials (Alnylam), and another moving to trials with antisense oligonucleotides (Isis). Isis already has an ASO in a phase one trial with patients with the genetic form of ALS. Also exciting is that a researcher in the Hayden lab has discovered a way to make ASOs allele specific.

I'm betting that one of these three will get to the finish line.

Ok, so we could end up with a few different trials going on at the same time? All of which looking promising?

[www.hdyo.org]

yes.

How long do trials normally take?

Marsha,
Thank you for posting this! I really needed to read something positive this week . . . something hopeful!!

Thanks, again!

This sounds great! About research firms...I have a question I'm hoping you can help me with.
I live in Grand Rapids, MI and we have a fabulous place named the VanAndel Research center. It's HUGE and has recently sparked a development in our community of many wonderful research opportunities, and medical centers ...we now have an area of town dubbed "Medical Mile" because of the impact this research growth has had. They focus on cancer and Parkinsons and Alzheimers.
They're right here in town...How do you go about contacting a place like this one about adding HD to their list? DO you have any ideas ??
Thanks!
Sara

what do the timelines look like on trials for the three of these promising treatments???

Very promising and exciting!!

Is RNAi the same thing that Dr Hayden did with silencing the HD gene in mice? Sorry, i have trouble understanding if this is the same thing or something similar, thanks Marsha