Forum for Science, Industry and Business

Sponsored by:     3M 
Search our Site:

 

MIT research holds promise for Huntington’s treatment

08.03.2006


Researchers at MIT and Harvard Medical School have identified a compound that interferes with the pathogenic effects of Huntington’s disease, a discovery that could lead to development of a new treatment for the disease.



There is no cure for Huntington’s, a neurodegenerative disorder that now afflicts 30,000 Americans, with another 150,000 at risk. The fatal disease, which is genetically inherited, usually strikes in midlife and causes uncontrolled movements, loss of cognitive function and emotional disturbance.

"There are now some drugs that can help with the symptoms, but we can’t stop the course of the disease or its onset," said Ruth Bodner, lead author on a paper appearing online the week of Mar. 6 in the Proceedings of the National Academy of Sciences (PNAS). Bodner is a postdoctoral fellow in MIT’s Center for Cancer Research.


The compound developed by Bodner and others in the laboratories of MIT Professor of Biology David Housman, Harvard Medical School Assistant Professor Aleksey Kazantsev and Harvard Medical School Professor Bradley Hyman might lead to a drug that could help stop the deadly sequence of cellular events that Huntington’s unleashes.

"Depending on its target, any one compound will probably block only a subset of the pathogenic effects," Bodner said.

Huntington’s disease is caused by misfolded proteins, called huntingtin proteins, that aggregate and eventually form large clump-like "inclusions." The disease is characterized by degeneration in the striatum, an area associated with motor and learning functions, and the cortex. The proteins may disrupt the function of cellular structures known as proteasomes, which perform a "trash can" function for the cell -- disposing of cellular proteins that are misfolded or no longer needed, said Bodner.

Without a functional proteasome, those cellular proteins accumulate, poisoning brain cells and impairing patients’ motor and cognitive function.

Until now, most researchers looking for Huntington’s treatments have focused on compounds that prevent or reverse the aggregation of huntingtin proteins. However, recent evidence suggests that the largest inclusions may not necessarily be harmful and could in fact be protective, said Bodner. So, the MIT and Harvard scientists decided to look for compounds that actually promote the formation of large inclusions.

The highest concentration of protein inclusions was found when the researchers applied a compound they called B2 to cells cultivated in the laboratory. The compound also had a strong protective effect against proteasome disruption, thus blocking one of the toxic effects of the huntingtin protein.

The B2 compound also promoted large inclusions and showed a protective effect in a cellular model of Parkinson’s disease, another neurodegenerative disorder caused by misfolded proteins.

In Parkinson’s disease, the mutant proteins destroy dopamine-producing cells in the substantia nigra. Normally, the dopamine transmits signals to the corpus striatum, allowing muscles to make smooth, controlled movements. When those dopamine-producing cells die, Parkinson’s patients exhibit the tremors that are characteristic of the disease.

The researchers are now working on finding a more potent version of the compound that could be tested in mice.

This work was funded by the Hereditary Disease Foundation, Massachusetts Biotechnology Research Council, National Institutes of Health, American Parkinson’s Disease Association and the MassGeneral Institute for Neurodegenerative Disease.

Elizabeth A. Thomson | MIT News Office
Further information:
http://www.mit.edu

More articles from Life Sciences:

nachricht For a chimpanzee, one good turn deserves another
27.06.2017 | Max-Planck-Institut für Mathematik in den Naturwissenschaften (MPIMIS)

nachricht New method to rapidly map the 'social networks' of proteins
27.06.2017 | Salk Institute

All articles from Life Sciences >>>

The most recent press releases about innovation >>>

Die letzten 5 Focus-News des innovations-reports im Überblick:

Im Focus: Can we see monkeys from space? Emerging technologies to map biodiversity

An international team of scientists has proposed a new multi-disciplinary approach in which an array of new technologies will allow us to map biodiversity and the risks that wildlife is facing at the scale of whole landscapes. The findings are published in Nature Ecology and Evolution. This international research is led by the Kunming Institute of Zoology from China, University of East Anglia, University of Leicester and the Leibniz Institute for Zoo and Wildlife Research.

Using a combination of satellite and ground data, the team proposes that it is now possible to map biodiversity with an accuracy that has not been previously...

Im Focus: Climate satellite: Tracking methane with robust laser technology

Heatwaves in the Arctic, longer periods of vegetation in Europe, severe floods in West Africa – starting in 2021, scientists want to explore the emissions of the greenhouse gas methane with the German-French satellite MERLIN. This is made possible by a new robust laser system of the Fraunhofer Institute for Laser Technology ILT in Aachen, which achieves unprecedented measurement accuracy.

Methane is primarily the result of the decomposition of organic matter. The gas has a 25 times greater warming potential than carbon dioxide, but is not as...

Im Focus: How protons move through a fuel cell

Hydrogen is regarded as the energy source of the future: It is produced with solar power and can be used to generate heat and electricity in fuel cells. Empa researchers have now succeeded in decoding the movement of hydrogen ions in crystals – a key step towards more efficient energy conversion in the hydrogen industry of tomorrow.

As charge carriers, electrons and ions play the leading role in electrochemical energy storage devices and converters such as batteries and fuel cells. Proton...

Im Focus: A unique data centre for cosmological simulations

Scientists from the Excellence Cluster Universe at the Ludwig-Maximilians-Universität Munich have establised "Cosmowebportal", a unique data centre for cosmological simulations located at the Leibniz Supercomputing Centre (LRZ) of the Bavarian Academy of Sciences. The complete results of a series of large hydrodynamical cosmological simulations are available, with data volumes typically exceeding several hundred terabytes. Scientists worldwide can interactively explore these complex simulations via a web interface and directly access the results.

With current telescopes, scientists can observe our Universe’s galaxies and galaxy clusters and their distribution along an invisible cosmic web. From the...

Im Focus: Scientists develop molecular thermometer for contactless measurement using infrared light

Temperature measurements possible even on the smallest scale / Molecular ruby for use in material sciences, biology, and medicine

Chemists at Johannes Gutenberg University Mainz (JGU) in cooperation with researchers of the German Federal Institute for Materials Research and Testing (BAM)...

All Focus news of the innovation-report >>>

Anzeige

Anzeige

Event News

Plants are networkers

19.06.2017 | Event News

Digital Survival Training for Executives

13.06.2017 | Event News

Global Learning Council Summit 2017

13.06.2017 | Event News

 
Latest News

Touch Displays WAY-AX and WAY-DX by WayCon

27.06.2017 | Power and Electrical Engineering

Drones that drive

27.06.2017 | Information Technology

Ultra-compact phase modulators based on graphene plasmons

27.06.2017 | Physics and Astronomy

VideoLinks
B2B-VideoLinks
More VideoLinks >>>