HDMeetings : Message: Scientists close to "discovering cure for HD" [C2-8 MIND Study] 10-20-2007

Don't we all wake each day with the same thought [hope/prayer] in our hearts/mind that this will be the day the headline will announce "A Cure for Huntington's Disease has been found"? I'm sure each of you feel as I do: every time I see or hear the words "Huntington's Disease" and "Cure" appear in the same headline, my heart starts to race and, I swear, I stop breathing momentarily.

Most of us want to know about the latest, and most hopeful, results of scientific studies or clinical trials in HD being done around the globe. Yet, after digesting what we've just read/heard, we realize those headlines may be a bit misleading because the news relates to animal studies [not the here and now], or the scientific aspects of the announcement are so technical in content, we really don't know anymore then we did before because we couldn't understand the significance of what was just reported.

For years, I was guilty of sending every tidbit of scientific news I found out to my list of on-line groups and HD friends, including abstracts that even had me scratching my head, wondering "HUH?" What? Because, sometimes it can be that small ray of "hope" we hear about that helps us fight this battle a little bit harder, longer. For the past few years I still share published news articles, but the scientific abstracts or copies of the full studies I receive I only share with a small group of people. Included in that group are the editors of the HD Lighthouse and HDAC and the editor of the HD Drugs Works [HDDW] because I know they will dissect those documents, often doing their own research or talking with the author, then publish a summary paper that ordinary HD families can understand - a clear and honest interpretation!

Jumping forward to today:

Many of you may have read the below news article yesterday or had it forwarded to you by an excited family member, friend or co-worker because those two magical words "Huntington's Disease" and "Cure" appear together in the headline. Once again, you're heart jumping into your throat, you wonder "how soon" only to read the entire thing and realize this news release doesn't provide you with an answer.

Well this MIND C2-8 news was released when the abstract was published and the HDSA made it headline news early in October. We received a full copy of the study on the 17th and, as you will see from Marsha Miller/HDL's email message, below, the HDL will be publishing its report on this study in the near future. Like Marsha, I and many others have agreed for years that one day an "HD Cocktail" would offer the most hope to those with HD and their families.

So, please don't be discouraged when you read news like the below, then feel almost broken-hearted when you realize they don't mean today. The only difference between then and now is the fact that, because of all the dedication and hard work by our scientists world-wide, the "HD Cocktail" to slow down the progression of HD is a lot closer to becoming a reality today then it was when we first started talking about it over 10 years ago!! When you read Marsha Miller's message, I think you'll agree! Continue with your support by participating in clinical studies when you can, supporting fund raising efforts for HD research, creating awareness by educating those around you about HD but whatever you do , do NOT give up hope! And, before you get too excited by the hottest" thing off the press on HD you just heard about, please check out the HD Lighthouse www.hdlighthouse.com or HDDW http://www.hddrugworks.org/ to see what they have to say about it!

Love

Jean

Scientists close to discovering cure for Huntington's Disease

http://www.bodyofwealth.com/entry/scientists-close-to-discovering-cure-for-huntingtons-disease/

Oct 20 2007

There appears to be a ray of hope for patients with Huntington�s disease, which is a rare inherited genetic disorder, characterized by un-coordinated body movements and decreased mental ability. Researchers at Mass General Institute for Neuro-degererative Disorders (MIND) claim to have developed a molecule, which has been proven to slow down disease progression.

According to the scientists, the molecule C2-8 was tested on mice and appeared to delay the degenerative process of Huntington�s disease. Researcher Steven Hersch has stated that although the course of action and the bio-chemical interaction of the molecule with body cells still remain unknown, it is speculated that the molecule is likely to have similar effects in humans as well. The molecule or its derivatives still remain to be converted into a drug form.

Aleksey Kazantsev, was the first scientist to propose C2-8 as a potential therapeutic drug considering its unique ability to �ability to block the aggregation of the mutant huntingtin protein in yeast and animal tissue and to improve function in a fruit fly model�. Subsequent research also established that this molecule did not have any toxic side effects on the mouse-model and possessed the ability to penetrate the blood-brain barrier.

Although mice treated with C2-8 molecule seemed to fare better in terms of strength, endurance and stability, the molecule did not appear to prolong their life spans as compared to untreated mice. This animal study is an important milestone in the treatment of HD and taking the research further would definitely yield promising results.

================

Marsha Miller - HD Lighthouse

Email: m.miller2001@...

Sat, 20 Oct 2007

I'm working on a Lighthouse update right now about C2-8. I don't think the Mass General researchers would have selected that headline "...close to a cure." I think this is an encouraging development but it's only one of a number of possible treatments in the research pipeline. It's a novel compound, it is probably going to have to be developed further, then there will be more animal testing, and safety studies. So even if it continues to show promise as it moves through the pipeline, you are right in thinking that it's years away from being available.

At the same time though, there are other compounds which are farther along in the research pipeline. Research into treatments is being done in parallel and lines of research which don't pan out are dropped for more that do. Treatments can't come soon enough, but I think that with the strategic plan that Robert Pacifici of CHDI has drawn up, and all the research now going on into screening, developing and testing promising drugs that we have every reason to feel hopeful about the future. I also feel encouraged because we've seen some outstanding work by Michael Hayden and Ray Truant and Elena Cattaneo which has identified some specific targets for drugs to address.

It would be nice if we could wake up one morning to the news that there's a cure, but it won't happen that way. We are hearing about the milestones along the way and I think it's good to recognize them and feel encouraged. HD families are volunteering for research so we're making things happen, too. I think we'll be seeing treatments within the next couple of years, each addressing a different problem in the disease process and delaying onset and slowing progression more and more. I think we'll get an HD cocktail much like AIDS patients have their own cocktail of drugs.

So getting back to C2-8, there may be a point in the research when a developed version of the drug is available, it addresses a problem, and it's added to
the cocktail.

Eventually we may get a virtual cure through RNA interference but it's hard to predict the timing on that. A lot of good people are working on it
though!

======================
Proc Natl Acad Sci U S A. 2007 Oct 9; [Epub ahead of print]
http://www.ncbi.nlm.nih.gov/sites/entrez
A small-molecule therapeutic lead for Huntington's disease: Preclinical pharmacology and efficacy of C2-8 in the R6/2 transgenic mouse.
Chopra V, Fox JH, Lieberman G, Dorsey K, Matson W, Waldmeier P, Housman DE, Kazantsev A, Young AB, Hersch S.
Department of Neurology, Harvard Medical School, Massachusetts General Hospital, Charlestown, MA 02129;

HDSA news link http://www.hdsa.org/site/PageServer?pagename=c28
announced by Coalition Member Steve Hersch
Molecule Promising Against Huntington's Disease
C2-8 battles the brain illness, mouse study shows

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