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:5/31/2016)... ... 31, 2016 , ... To meet a growing demand for ... The University of Scranton is adding a Certificate in Health Informatics to its ... rapidly growing field of healthcare information. , Healthcare organizations are under growing ...
(Date:5/30/2016)... ... 30, 2016 , ... As the CDC relaxes its stance on traditional No-Nit ... keep their households lice free. , According to a May 26 article from ... kids in the classroom despite the fact that they may be harboring an infestation. ...
(Date:5/30/2016)... ... May 30, 2016 , ... Shaolin Institute officially starts the ... special intensive summer training camp starts on June 17th on Shaolin Institute Atlanta ... children a fun and unique experience with an opportunity to learn KungFu martial ...
(Date:5/29/2016)... ... May 29, 2016 , ... Whole Health Supply is happy to ... KP-240L clipper is available to the public. This is an unusual clipper because it ... the average clipper. , Everything about this product is concentrated on ease of use, ...
(Date:5/28/2016)... (PRWEB) , ... May 28, 2016 , ... "Color Grading ... drag and drop a preset onto their media," said Christina Austin - CEO of ... Studios, editors can quickly and easily add stylish color grades to their footage. A ...
Breaking Medicine News(10 mins):
(Date:5/30/2016)... , May 30, 2016 On ... eliminate viral hepatitis by 2030. At the 69 th ... ever Global Viral Hepatitis Strategy, signalling the greatest global commitment ... a goal of eliminating hepatitis B and C by 2030 ... if reached, will reduce annual deaths by 65% and increase ...
(Date:5/27/2016)... 27, 2016  A new study highlights the necessity of health literacy within the ... the American College of Radiology , a majority of oncology patients undergo imaging screenings ... http://photos.prnewswire.com/prnh/20160527/373022 ... ... Medical Diagnostic Imaging Ampronix ...
(Date:5/27/2016)... 27, 2016 Kitov ... focused on late-stage drug development, today announced the ... of pivotal batches required for registration of KIT-302 ... This follows Kitov,s announcement in December ... met its primary efficacy endpoint. "We ...
Breaking Medicine Technology: