Hey there-

I read these releases all the time but seldom post them- except that this one is also in a peer reviewed journal. Marsha- any sense of how fast would these sorts of things get to phase 1 clinical trials?

[www.eurekalert.org]

Got the journal article and am reading now. I think this will get to clinical trials within the next two years. I'll be posting more details on the Lighthouse research section tomorrow!

I think this is VERY good news.

Fingers crossed for rapid drug development. This could be the one. I'll be anxious to hear Marsha's take.

Will

Dr. Cleveland says he thinks they can get this into clinical trials in eighteen months.

I started crying as I read this! I feel like I have been living in a land of acceptance. I accept that Hd will affect my child in one way or another. But now, it seems like there is hope! I am overwhelmed!

Great!

That's awesome!!

Yes! Promising news!

Keep hoping and wish and praying!

Great news!!

Here's my HDSA artilcle. [www.hdsa.org]

I am going to write on for the Lighthouse as well.

Marsha, this is great news, and thank you so much for doing your article. I dont usually read hd buzz, just not my style is all, but i did want to see what they also had to say about this. The doctor in your article says this could mean a one time dose of a med could possibly be the result, but hd buzz said people would maybe need the med about once a year. So im having a bit of trouble understanding this, and what they mean. Something about a transient dose to the spinal fluid, what does that mean? I hope you understand what i mean Marsha, and i value your articles so much. The lighthouse is the only place i've ever gone for my research news, because in the beginning, it was the ONLY place that had information and support for us...thats why your articles are important to me. How come your job with hdsa is ending soon?
PS Also, what is the difference between this and the trial that Will is trying? Also, the article mentions transient infusion, so i tried to google that to find out what that means, and couldn't find out anything...help?



Edited 2 time(s). Last edit at 06/21/2012 11:54PM by Barb.

I pray to God that finally this monster will be slain.

Barb, I think that this will definitely have to be a recurring treatment, not a one time thing. I don't see how it is possible to 'reset the clock.' I understand the IDEA here but I don't think it works. The idea is that during this 'Huntington's holiday' the cells would have time to clear away the HD protein that has been piling up and getting into the nucleus of the cell and then you (as an example) would be just like you were at 20 when the HD protein hadn't done any damage and then maybe it would take another 30 years for you to become symptomatic just like before. The problem is you aren't 20 any more, you are 50 and all those cellular defense mechanism aren't working at peak efficiency any more - which in my opinion is why people become symptomatic later in life and not at birth. So once the HD protein is being made at regular levels again, it won't take all that long before it overwhelms the cells again and you'd need another treatment. So I agree with HD Buzz on this.

So this is not going to be a one time treatment and it's not going to be as easy as taking a pill. There would be some kind of implanted pump in the spinal cord so that the drug can be delivered. But that's okay, IMO, if it works we will take it and then researchers will work on improving delivery and effectiveness and will learn what needs to be done and do it. Maybe there will be some way to do a one time RNAi allele specific treatment, But this is a good way to start because the treatment is temporary.

Transient infusion means that it was short lived, not a continuous infusion. The infusion occurred over a two week period and then was stopped.

The drug that Will is trying is not a genetic intervention. It's an effort to reduce the amount of HD protein through a post translational modification, in other words changing the protein AFTER its made, not stopping it from being made.

I had goosebumps reading this!!! Thanks for writing this, Marsha. You have a gift for writing...you explain things so well. Now even if there needs to be recurring treatments, it still sounds like a miracle. I pray that this is going to be effective and that it gets out for trials SOON!!! Thanks for bringing this to our attention noreaster. I am in such a happy mood right now!

So often we read about something promising and then we wait and wait for a follow up because the researcher hasn't gotten more money or the pharmaceutical company doesn't have the funds to go forward. I don't anticipate those problems with the ASOs since CHDI, Novartis, Isis, and Genzyme are all involved in this.

Thank you marsha for taking the time to answer all my questions, i get it now, thanks

WOW I will be keeping an eye on this one!