Huntington's disease: Enzyme offers hope, study finds

  • 3 June 2011
  • From the section Health
Fruit fly eye and body
Image caption Researchers reduced the death of neurons in fruit flies by blocking a particular enzyme.

A new approach to treating Huntington's disease may have been found, a UK study carried out in fruit flies suggests.

The incurable disease, caused by degeneration of nerve cells, gradually impairs a person's ability to function.

Researchers slowed the development of neurodegeneration - a key factor in Huntington's disease - by targeting a particular enzyme in fruit-flies.

The study, led by the University of Leicester, is published in Current Biology.

"This breakthrough is important as no drugs currently exist that halt progression, or delay onset of Huntington's disease," said Dr Giorgini, who led the study.

The researchers found decreasing the activity of a certain enzyme - known as KMO - reduced the number of neurons dying in the fruit-flies.

"Since we have identified an actual target and there are chemicals that can act on the target, there is a possibility that we could develop drugs to delay the onset of the disease," Dr Giorgini said.

Scientists also found that targeting other related enzymes with chemicals improved Huntington's symptoms in fruit flies.

"We would like to look in closer detail at the other enzymes in the same pathway and to develop new inhibitors of KMO," Dr Giorgini said.

Brain inflamation

Research from America also suggests the enzyme could be a key target for therapies.

The US research, published in Cell, found symptoms of Huntington's disease in mouse models could be reduced by blocking the same enzyme.

They developed a new compound - known as JM6 - that blocks the enzyme, leading to reduced brain inflammation in mice.

Working with mouse models of another neurodegenerative condition - Alzheimer's disease - they found that JM6 also prevents memory loss.

"The discovery has significant implications for two devastating diseases and suggest that the KMO enzyme is a good protein for us to target with medications in diverse neurodegenerative disorders," said Professor Lennart Mucke, of the Gladstone Institute, where the research took place.

Huntington's Disease Association chief executive Cath Stanley said: "This is an exciting piece of research that will offer hope to the many people affected by Huntington's disease."

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