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Possible new treatment targets found for Huntington’s disease

February 23rd, 2012
by Tania

Scientists in Ireland claim to have made a major breakthrough in the understanding of Huntington's disease.

Over 100,000 people worldwide are affected by the condition, which causes uncontrolled movements, emotional disturbances, and severe mental deterioration, and a further 300,000 are expected to develop symptoms during their lifetime.

Currently, the only therapies available manage the symptoms, rather than halt the progression of the disease.

Researchers from the National University of Ireland Galway have identified histone deacetylase complexes (HDACs) as acting as agents in the underlying mutations that cause Huntington's disease.

They found that active HDACs worsen the mutation in cells, which may be what makes the disease so severe.

Blocking these with experimental HDAC inhibitors, greatly reduces the risk of further mutations from occurring, the research claims.

Separate research from Northwestern University also recently identified a number of new genes and pathways associated with protein misfolding diseases, which include Huntington's. A total of 150 genes were found to be linked with protein aggregation, nine of which made all the proteins healthier.

Lead author Professor Robert Lahue of National University of Ireland Galway's Centre for Chromosome Biology said: "Ongoing mutations in the brain of Huntington's patients are thought to drive progression of the disease.

"Our discovery suggests that inhibiting HDAC function slows down the mutation process, and thereby could slow disease progression. A key finding of the research was to pinpoint specific HDACs for selective inhibition."

The development of drugs based on HDAC inhibitors is some way off. Researchers in the US are in the process of testing experimental HDAC inhibitors for efficacy and safety in not just laboratory models of Huntington's but also other diseases.

It is thought the research could hold clues into other neurological disorders, such as myotonic dystrophy type I, which is caused by the same type of mutation seen in patients with Huntington's.

Professor Lahue said: "Huntington's is a particularly cruel disease, as it is passed from parent to child, often with increased severity or earlier onset."

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