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 disorderthat 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.

Occurrence rate of the disease is estimated to be around 5~10 out of 100,000 people in Caucasian populations. In China, there are about 30,000 sufferers of the disease based on the estimation using the epidemiology studies in east Asia populations.

There is no cure for the disease. The currently approved treatment drugs only suppress the chorea symptoms (jerky and uncontrollable physical movement) for a few years, and it cannot delay the disease progression.

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 fromthe 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 slacken the development of the disease, and greatly improve life quality of sufferers. Meanwhile, extensive preclinical and clinical experiments are still required before their application in Huntington's disease patients" said Lu Boxun, professor of School of Life Sciences of Fudan University, and a leading researcher with the project.

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.