Navigation Links
Proposed drug may reverse Huntington's disease symptoms
Date:6/20/2012

With a single drug treatment, researchers at the Ludwig Institute for Cancer Research at the University of California, San Diego School of Medicine can silence the mutated gene responsible for Huntington's disease, slowing and partially reversing progression of the fatal neurodegenerative disorder in animal models.

The findings are published in the June 21, 2012 online issue of the journal Neuron.

Researchers suggest the drug therapy, tested in mouse and non-human primate models, could produce sustained motor and neurological benefits in human adults with moderate and severe forms of the disorder. Currently, there is no effective treatment.

Huntington's disease afflicts approximately 30,000 Americans, whose symptoms include uncontrolled movements and progressive cognitive and psychiatric problems. The disease is caused by the mutation of a single gene, which results in the production and accumulation of toxic proteins throughout the brain.

Don W. Cleveland, PhD, professor and chair of the UC San Diego Department of Cellular and Molecular Medicine and head of the Laboratory of Cell Biology at the Ludwig Institute for Cancer Research, and colleagues infused mouse and primate models of Huntington's disease with one-time injections of an identified DNA drug based on antisense oligonucleotides (ASOs). These ASOs selectively bind to and destroy the mutant gene's molecular instructions for making the toxic huntingtin protein.

The singular treatment produced rapid results. Treated animals began moving better within one month and achieved normal motor function within two. More remarkably, the benefits persisted, lasting nine months, well after the drug had disappeared and production of the toxic proteins had resumed.

"For diseases like Huntington's, where a mutant protein product is tolerated for decades prior to disease onset, these findings open up the provocative possibility that transient treatment can lead to a prolonged benefit to patients," said Cleveland. "This finding raises the prospect of a 'huntingtin holiday,' which may allow for clearance of disease-causing species that might take weeks or months to re-form. If so, then a single application of a drug to reduce expression of a target gene could 'reset the disease clock,' providing a benefit long after huntingtin suppression has ended."

Beyond improving motor and cognitive function, researchers said the ASO treatment also blocked brain atrophy and increased lifespan in mouse models with a severe form of the disease. The therapy was equally effective whether one or both huntingtin genes were mutated, a positive indicator for human therapy.

Cleveland noted that the approach was particularly promising because antisense therapies have already been proven safe in clinical trials and are the focus of much drug development. Moreover, the findings may have broader implications, he said, for other "age-dependent neurodegenerative diseases that develop from exposure to a mutant protein product" and perhaps for nervous system cancers, such as glioblastomas.


'/>"/>
Contact: Scott LaFee
slafee@ucsd.edu
619-543-6163
University of California - San Diego
Source:Eurekalert  

Related medicine news :

1. Proposed testosterone testing of some female olympians challenged by Stanford scientists
2. Treatment of childhood OSA reverses brain abnormalities
3. Combination of 2 drugs reverses liver tumors
4. In Mice, Drug Reverses Symptoms of Condition Linked to Autism
5. Kidney Disease May Be as Harmful to Heart as Heart Attack: Study
6. New Treatments Emerging from Immune and Inflammatory Disease R&D Pipelines
7. Gum Disease, HPV May Play Role in Head & Neck Cancers
8. Device implanted in brain has therapeutic potential for Huntingtons disease
9. Control gene for conveyor belt cells could help improve oral vaccines, treat intestinal disease
10. Fruit flies reveal mechanism behind ALS-like disease
11. 1960s-era anti-cancer drug points to treatments for Lou Gehrigs disease
Post Your Comments:
*Name:
*Comment:
*Email:
Related Image:
Proposed drug may reverse Huntington's disease symptoms
(Date:7/26/2017)... ... 26, 2017 , ... Six greater Bay Area roller derby leagues are partnering with Brown Paper ... widely attended series of blood drives in California, beginning July 29. Make ‘Em Bleed roller ... years -- enough to have helped to save up to 2700 lives. , “The Make ...
(Date:7/26/2017)... (PRWEB) , ... July 26, 2017 , ... A major ... processes. Inconsistent results freeze drying samples and a lengthy freeze dry cycle are a ... will discuss freeze drying techniques, as well as new accessories and advancements in laboratory ...
(Date:7/26/2017)... ... July 26, 2017 , ... “We are dentists and we thought there ... Jose, Calif., “so we invented the MAGNETIC/ LOCK BRACKETS.” , The patent-pending MAGNETIC/ LOCK ... In doing so, it offers an effective alternative to traditional braces. As a result, ...
(Date:7/26/2017)... ... ... to sign up for the new One-A-Month Essential Oil Club for fun and wellness. ... and author of numerous books on essential oils. , , Members ... Two items are included in the December mailing. , A HOW ...
(Date:7/26/2017)... ATLANTA, Ga. (PRWEB) , ... July 26, 2017 , ... ... and equal access to medicine for everyone affected by diabetes, is excited to announce ... of its Global Ambassador Program. , Established in 2005, the Team Type 1 ...
Breaking Medicine News(10 mins):
(Date:7/14/2017)... , July 14, 2017 Endo International plc (NASDAQ: ... on Tuesday, August 8, 2017.  Members of its senior management team ... markets open at 8:30 a.m. ET. The dial-in ... (866) 497-0462, International (678) 509-7598, and the passcode is 45397076. Please ... A replay of the call ...
(Date:7/12/2017)... , July 12, 2017 CarpalAID is a revolutionary ... braces or surgery. Carpal tunnel syndrome affects more than ... at twice the rate of men. The common methods of treating ... mobilization with uncomfortable hand braces or gloves. ... CarpalAID is a clear patch ...
(Date:7/11/2017)... , July 11, 2017  Sysmex America, Inc., ... diagnostic testing equipment as well as middleware information ... way to make quality assurance easier and more ... is well known for the innovation that it ... Monitor elevates quality assurance processes to a new ...
Breaking Medicine Technology: