Headlines Published on 22 June 2011

MEDICAL
Title New treatment method for Huntington's disease on the cards

Patients suffering from Huntington's disease take note: scientists from the United Kingdom and the United States have discovered a new method to treat the neurodegenerative disease that affects muscle coordination and leads to cognitive decline and dementia, and eventually death. In two separate studies, the researchers say their findings could potentially help slow down the development of Huntington's disease as well as Parkinson's and Alzheimer's diseases. The research is presented in the journal Current Biology.

Eye colour mutations in the fruit-fly are being used to study degeneration of photoreceptor neurons, which may have relevance to conditions such as Huntington's disease. Image taken using a light microscope © University of Leicester
Eye colour mutations in the fruit-fly are being used to study degeneration of photoreceptor neurons, which may have relevance to conditions such as Huntington's disease. Image taken using a light microscope
©  University of Leicester

Experts say Huntington's is an inherited disease of the central nervous system. Brain cells are progressively degenerated, leading to a person's inability to walk, talk and even think logically. Around 1 in 10 000 people suffer from this debilitating disease.

In the first study, led by the University of Leicester in the United Kingdom, the researchers stopped the development of neurodegeneration linked to Huntington's disease by targeting a specific enzyme — kynurenine 3-monooxygenase, KMO — in fruit flies. Dr Flaviano Giorgini and Professor Charalambos Kyriacou and their team directly manipulated metabolites in the KMO cellular pathway with drugs in order to manipulate the symptoms the flies displayed.

'This work provides the first genetic and pharmacological evidence that inhibition of a particular enzyme — KMO — is protective in an animal model of this disease, and we have also found that targeting other points in this cellular pathway can improve Huntington's disease symptoms in fruit flies,' says senior author of the study Dr Giorgini from the University of Leicester.

'This breakthrough is important as no drugs currently exist that halt progression or delay onset of Huntington's disease. We are tremendously excited about these studies, as we hope that they will have direct ramifications for Huntington’'s disease patients. Our work combined with the study in our companion publication in Cell, provides important confirmation of KMO inhibition as a potential therapeutic strategy for these individuals. As many KMO inhibitors are available, and more are being developed, it is hoped that such compounds can ultimately be tested in clinical trials for this as well as other neurodegenerative disorders.'

Dr Giorgini says the findings of the researchers offer the first genetic and pharmacological evidence that inhibition of KMO is protective in an animal model of the disease.

The researchers that contributed to the studies plan to continue their efforts, targeting the enhancement and development of medical intervention in Huntington's and other neurodegenerative disorders.

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

Dr Giorgini also collaborated with Professor Robert Schwarcz of the University of Maryland School of Medicine and Dr Paul Muchowski of the University of California, San Francisco in the United States, providing more insight on the potential role of KMO for the treatment of Huntington's and Alzheimer's diseases.









More information:

  • University of Leicester
  • Current Biology
  • 'Skin cells converted into brain cells, EU-funded team takes steps towards the future'







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