Fudan researchers achieve breakthrough in Huntington's disease treatment

Researchers at Fudan University in Shanghai have come up with an innovative method of drug discovery to treat Huntington's disease, a neurodegenerative disease similar to Alzheimer's and Parkinson's.

The discovery was published on science journal Nature on Thursday.

Huda Zoghbi, agenetic neurologist and the winner of the Breakthrough Prize in Life Sciences, said that this "therapeutic strategy might be useful not only for Huntington's disease, but also other diseases involving expanded polyglutamine tracts".

Huntington's disease, also known as Saint Vitus' dance, is a fatal genetic disorder that causes the progressive breakdown of nerve cells in the brain. It is usually accompanied byclinical symptoms such asuncontrolled dance-like behavior, also known as chorea, as well as cognitive deficiency and psychiatric abnormalities.

The occurrence rate of the disease is estimated to be around 15 out of 100,000 people in Western countries. In China, there are about 30,000 patients with the disease.

There is currently no cure for the disease. Current treatment methods are focused on the prevention of jerky and uncontrollable physical movements.

Researchers from Fudan University managed to identify four small molecule compounds that specifically reduce the protein that causes Huntington's disease, bringing hope to the disease-progression-modifying treatment ofHuntington's disease and similar conditions.

This breakthrough is a result of the cross-discipline collaborationbetween researchers from the field of life sciences and that of information sciences and engineering. The latter group had stepped in and helped to develop a faster, more sensitive screening platform that could better identify the target protein-interacting compounds from a library of thousands of small molecule compounds.

"If applied, the drug could effectively slow down the development of the disease, and greatly improve the quality of life of patients," said Lu Boxun, one of the lead researchers of the project who is also a professor from the School of Life Sciences at Fudan University.

Studies have found that some of the most distressing aspects of the disease include psychiatric disorders like anxiety and depression, which could result in thoughts and attempts of suicide.