Is this a possible cure for my wife who was diagnosed with Huntington's Disease 8 Years ago. We had no family history or knowledge prior to eventually finding out Kay was suffering with this condition. None the less this possibility is giving my family hope.

Sustained Therapeutic Reversal of Huntington's Disease by Transient Repression of Huntingtin Synthesis

Link to full article is here
[www.cell.com]

Neuron, Volume 74, Issue 6, 1031-1044, 21 June 2012
Copyright 2012 Elsevier Inc. All rights reserved.
10.1016/j.neuron.2012.05.009

Refers to: Huntingtin Holiday: Progress toward an...

Authors

Holly B. Kordasiewicz, Lisa M. Stanek, Edward V. Wancewicz, Curt Mazur, Melissa M. McAlonis, Kimberly A. Pytel, Jonathan W. Artates, Andreas Weiss, Seng H. Cheng, Lamya S. Shihabuddin, Gene Hung, C. Frank Bennett, Don W. ClevelandSee Affiliations
Highlights
ASOs suppress huntingtin throughout the rodent and nonhuman primate CNS
ASO therapy reverses disease, prevents brain loss, and promotes survival in HD models
ASO-mediated disease reversal persists for longer than huntingtin suppression
Suppression of mutant huntingtin reverses disease independent of wild-type levels
Summary

The primary cause of Huntington's disease (HD) is expression of huntingtin with a polyglutamine expansion. Despite an absence of consensus on the mechanism(s) of toxicity, diminishing the synthesis of mutant huntingtin will abate toxicity if delivered to the key affected cells. With antisense oligonucleotides (ASOs) that catalyze RNase H-mediated degradation of huntingtin mRNA, we demonstrate that transient infusion into the cerebrospinal fluid of symptomatic HD mouse models not only delays disease progression but mediates a sustained reversal of disease phenotype that persists longer than the huntingtin knockdown. Reduction of wild-type huntingtin, along with mutant huntingtin, produces the same sustained disease reversal. Similar ASO infusion into nonhuman primates is shown to effectively lower huntingtin in many brain regions targeted by HD pathology. Rather than requiring continuous treatment, our findings establish a therapeutic strategy for sustained HD disease reversal produced by transient ASO-mediated diminution of huntingtin synthesis.



Edited 3 time(s). Last edit at 06/24/2012 04:47AM by markacarey.

Here's my review of the article: [www.hdsa.org]

It's also been discussed under the thread 'possible good news'

I believe gene silencing coupled with a stem cell treatment will come very close to being a cure. Given the involvement of CHDI, Novartis, Isis, and Genzyme in the work reported, I believe that the work necessary for a clinical trial to be approved will be completed swiftly. Dr. Cleveland is estimating a clinical trial in 18 months.

I hope so!

Any idea where the trial would take place?

Marsha,

You say a clinical trial could start in 18 months. Do you mean a Phase 1? Or have they already completed the early trials and you mean phase 3?

Thank you

Katy x

Phase One.

I read this before my vacation and it is truly great hope. Keep the inform coming Marsha!!