Compound may provide drug therapy approach for Huntington's disease

June 23, 2011

UT Southwestern Medical Center researchers have identified compounds that appear to inhibit a signaling pathway in Huntington's disease, a finding that may eventually lead to a potential drug therapy to help slow the progression of degenerative nerve disorders.

"Our studies have uncovered a new for Huntington's disease treatment and possibly for other ," said Dr. Ilya Bezprozvanny, professor of physiology and senior author of the study, published in today's issue of . "In addition, we now have this new series of compounds that gives us a tool to study the pathogenesis of Huntington's disease."

Huntington's disease is a fatal genetic disorder in which certain waste away. More than 250,000 people in the U.S. have the disorder or are at risk for it. The most common form is adult-onset, with symptoms usually developing in patients in their mid-30s and 40s.

The disease results in uncontrolled movements, psychiatric disturbance, gradual dementia and eventually death. There is no therapy available currently to slow the progression of the disease.

Scientists at UT Southwestern found that quinazoline-derived compounds effectively block what is known as the store-operated calcium entry signaling pathway, which was never before implicated in Huntington but that might be a therapeutic target in the disease.

Dr. Bezprozvanny's laboratory research has contributed to growing scientific evidence that suggests abnormalities in neuronal calcium signaling play an important role in the development of Huntington's disease. UT Southwestern researchers demonstrated in the current study that the quinoline compounds – supplied by EnVivo – protected brain cells.

"If this holds, this compound can be considered to have potential therapeutic application for Huntington's," he said. "As we ultimately seek a cure, we are encouraged to have found something that may slow the progress or delay the onset of the disease."

Explore further: Test reveals effectiveness of potential Huntington's disease drugs

Related Stories

Duke team finds compounds that prevent nerve damage

September 23, 2008

Duke University Medical Center scientists have made a significant finding that could lead to better drugs for several degenerative diseases including Huntington's disease and Alzheimer's disease. Compounds that block the ...

Research offers clue to halt Huntington's disease

March 25, 2011

( -- Surprising findings from a study into the brains of transgenic mice carrying the Huntington's disease mutation could pave the way for treatments which delay the onset and progression of this devastating genetic ...

New drugs target delay of Huntington's symptoms

May 31, 2011

(Medical Xpress) -- McMaster researchers have discovered a new drug target that may be effective at preventing the onset of Huntington's disease, working much the same way heart medications slow the progression of heart disease ...

Recommended for you

A new form of real gold, almost as light as air

November 25, 2015

Researchers at ETH Zurich have created a new type of foam made of real gold. It is the lightest form ever produced of the precious metal: a thousand times lighter than its conventional form and yet it is nearly impossible ...

Getting under the skin of a medieval mystery

November 23, 2015

A simple PVC eraser has helped an international team of scientists led by bioarchaeologists at the University of York to resolve the mystery surrounding the tissue-thin parchment used by medieval scribes to produce the first ...

Moonlighting molecules: Finding new uses for old enzymes

November 27, 2015

A collaboration between the University of Cambridge and MedImmune, the global biologics research and development arm of AstraZeneca, has led researchers to identify a potentially significant new application for a well-known ...


Please sign in to add a comment. Registration is free, and takes less than a minute. Read more

Click here to reset your password.
Sign in to get notified via email when new comments are made.