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:10/12/2017)... (PRWEB) , ... October 12, 2017 , ... Leading pediatric ... peers in Washington, D.C., for the 49th Congress of the International Society of ... Vice President of the Center for Cancer and Blood Disorders at Children’s ...
(Date:10/12/2017)... RAPIDS, Mich. (PRWEB) , ... October 12, 2017 ... ... Wellness, has been named one of Michigan’s 2017 Best and Brightest in Wellness® ... and Brightest in Wellness® awards program on Friday, Oct. 20 from 7:30 a.m. ...
(Date:10/12/2017)... ... October 12, 2017 , ... ... property taxes a year. In some states—like New York, New Jersey, Massachusetts, Texas, ... many overseas retirement havens have extremely low property-tax rates, which contributes to the ...
(Date:10/12/2017)... ... October 12, 2017 , ... Farm Forward ... Berkeley, and other leading institutions in announcing the launch of the Leadership ... the way animals are raised for food. , Founding members of the Leadership ...
(Date:10/12/2017)... ... 12, 2017 , ... Nvision Biomedical Technologies , a ... addition of industry sales leader, Thomas (Tom) Slott, as Senior Vice President of ... sales network, direct the efforts of the surgical sales teams, manage contractual agreements, ...
Breaking Medicine News(10 mins):
(Date:10/12/2017)... 12, 2017   Divoti USA will engrave ... the standard of the latest FDA requirements, which stipulates new criteria ... Anyone in need of Medical ID jewelry such as Medical ID ... are engraved in terms of the new FDA requirements . ... Divoti offers this dark mark ...
(Date:10/11/2017)... IRVING, Texas , Oct. 11, 2017  Caris ... science focused on fulfilling the promise of precision medicine, ... Institute has joined Caris, Precision Oncology Alliance™ (POA) as ... leading cancer centers, the St. Jude Crosson Cancer Institute ... to advance the use of tumor profiling, making cancer ...
(Date:10/10/2017)... ORANGE COUNTY, Calif. , Oct. 10, 2017  NDS received ... Mobile  — a medical-grade battery-powered display stand specifically designed for ... aims to transform technology into a clinical solution to support the ... costs. Innovative Design ... NDS ZeroWire Mobile Wireless Solution ...
Breaking Medicine Technology: