Featured Research

from universities, journals, and other organizations

Potential Treatment For Huntington's Disease Identified

Date:
October 9, 2007
Source:
Massachusetts General Hospital
Summary:
Researchers have identified a compound that may lead to a treatment that could protect against the effects of Huntington's Disease. A small molecule called C2-8 appears to delay the loss of motor control and reduce neurological damage in a mouse model of the disorder.

MassGeneral Institute for Neurodegererative Disorders (MIND) researchers have identified a compound that may lead to a treatment that could protect against the effects of Huntington's Disease (HD). Their report, which will appear in the Proceedings of the National Academy of Sciences, describes how a small molecule called C2-8 appears to delay the loss of motor control and reduce neurological damage in a mouse model of the disorder.

Related Articles


"We found that C2-8 slows the progress of HD in a mouse model and might do the same thing in human patients, if it or its biochemical relatives can be translated into a drug," says Steven Hersch, MD, PhD, of MIND and the Massachusetts General Hospital (MGH) Department of Neurology, who led the study. "What we don't know yet is precisely how it works, what molecules it interacts with in cells and how potent it might be."

C2-8 was first identified as a candidate treatment for HD by MIND researcher Aleksey Kazantsev, PhD, based on its ability to block the aggregation of the mutant huntingtin protein in yeast and animal tissue and to improve function in a fruit fly model. The current study was designed to further investigate its potential as a therapeutic drug. The researchers first confirmed that oral doses of C2-8 can cross the blood-brain barrier and are nontoxic in a mouse model of HD. They also found that C2-8 does not interact with a number of molecules predictive of negative side effects.

HD mice that were treated with C2-8 starting at the age of 24 days scored significantly better on tests of strength, endurance and coordination than did HD mice that did not receive the molecule. While treatment significantly delayed progressive motor disability, the animals receiving C2-8 did not live longer. Examination of brain cells from the striatum, the area of the brain where the deterioration of HD occurs, showed that treated mice had less shrinkage of brain cells and smaller aggregates of huntingtin protein than did untreated HD mice.

"We've both validated that compounds reducing the aggregation of mutant huntingtin are potential HD drugs -- so that strategy is one that other scientists should pursue -- and shown that C2-8 has potential as the basis of a neuroprotective treatment," says Hersch. "We now need to confirm those results in a different mouse model, see whether similar compounds may be more potent than C2-8 and search for the enzyme or receptor it is binding to." Hersch is an associate professor of Neurology at Harvard Medical School.

Vanita Chopra, PhD, and Jonathan Fox, PhD, of MIND and MGH-Neurology are co-first author of the PNAS study. Additional co-authors are Greg Lieberman, Kathryn Dorsey, Kazantsev and Anne B. Young, MD, PhD, of MIND/MGH-Neurology; Wayne Matson, PhD, Boston University School of Medicine; Peter Waldmeier, PhD, Novartis Institute for Biomedical Research, Basel, Switzerland; and David Houseman, PhD, Massachusetts Institute of Technology. The study was supported by the Discovery of Novel Huntington's Disease Therapeutics Fund, MIND and the Massachusetts General Hospital.


Story Source:

The above story is based on materials provided by Massachusetts General Hospital. Note: Materials may be edited for content and length.


Cite This Page:

Massachusetts General Hospital. "Potential Treatment For Huntington's Disease Identified." ScienceDaily. ScienceDaily, 9 October 2007. <www.sciencedaily.com/releases/2007/10/071008171037.htm>.
Massachusetts General Hospital. (2007, October 9). Potential Treatment For Huntington's Disease Identified. ScienceDaily. Retrieved March 3, 2015 from www.sciencedaily.com/releases/2007/10/071008171037.htm
Massachusetts General Hospital. "Potential Treatment For Huntington's Disease Identified." ScienceDaily. www.sciencedaily.com/releases/2007/10/071008171037.htm (accessed March 3, 2015).

Share This


More From ScienceDaily



More Mind & Brain News

Tuesday, March 3, 2015

Featured Research

from universities, journals, and other organizations


Featured Videos

from AP, Reuters, AFP, and other news services

This Nasal Treatment Could Help Ease Migraine Pain

This Nasal Treatment Could Help Ease Migraine Pain

Newsy (Mar. 2, 2015) Researchers gave lidocaine to 112 patients, and about 88 percent of the subjects said they needed less migraine-relief medicine the next day. Video provided by Newsy
Powered by NewsLook.com
How Facebook Use Can Lead To Depression

How Facebook Use Can Lead To Depression

Newsy (Mar. 1, 2015) Margaret Duffy of the University of Missouri talks about her study on the social network and the envy and depression that Facebook use can cause. Video provided by Newsy
Powered by NewsLook.com
The Best Foods to Battle Stress

The Best Foods to Battle Stress

Buzz60 (Feb. 26, 2015) If you&apos;re dealing with anxiety, there are a few foods that can help. Krystin Goodwin (@krystingoodwin) has the best foods to tame stress. Video provided by Buzz60
Powered by NewsLook.com
Sleeping Too Much Or Too Little Might Increase Stroke Risk

Sleeping Too Much Or Too Little Might Increase Stroke Risk

Newsy (Feb. 26, 2015) People who sleep more than eight hours per night are 45 percent more likely to have a stroke, according to a University of Cambridge study. Video provided by Newsy
Powered by NewsLook.com

Search ScienceDaily

Number of stories in archives: 140,361

Find with keyword(s):
Enter a keyword or phrase to search ScienceDaily for related topics and research stories.

Save/Print:
Share:

Breaking News:

Strange & Offbeat Stories


Health & Medicine

Mind & Brain

Living & Well

In Other News

... from NewsDaily.com

Science News

Health News

Environment News

Technology News



Save/Print:
Share:

Free Subscriptions


Get the latest science news with ScienceDaily's free email newsletters, updated daily and weekly. Or view hourly updated newsfeeds in your RSS reader:

Get Social & Mobile


Keep up to date with the latest news from ScienceDaily via social networks and mobile apps:

Have Feedback?


Tell us what you think of ScienceDaily -- we welcome both positive and negative comments. Have any problems using the site? Questions?
Mobile: iPhone Android Web
Follow: Facebook Twitter Google+
Subscribe: RSS Feeds Email Newsletters
Latest Headlines Health & Medicine Mind & Brain Space & Time Matter & Energy Computers & Math Plants & Animals Earth & Climate Fossils & Ruins