07 June 2011

Treatment of Alzheimer's and Huntington's diseases: another hope

Researchers at the Gladstone Institute, working under the leadership of Dr. Paul Muchowski, have identified a new potential drug that eliminates the symptoms of Alzheimer's and Huntington's diseases in animal models. Currently, there are no effective methods of treating these serious diseases.

When conducting experiments on genetically modified mice with Alzheimer's and Huntington's diseases, scientists demonstrated that the experimental drug JM6 blocks the activity of the enzyme kynurenin-3-monooxygenase (CMOS), which has long been suspected by experts of involvement in the development of neurodegenerative diseases.

When working with a mouse model of Alzheimer's disease, the drug prevented the development of memory disorders and the loss of synaptic contacts between brain neurons, that is, the main signs of a human disease, which is the most common variant of dementia. In a mouse model of Huntington's disease – the most common hereditary neurodegenerative brain disease – JM6 blocked the development of inflammation of brain tissue and loss of synapses, while increasing the life expectancy of animals.

The peculiarity of JM6 is that it does not penetrate directly into the brain tissue and acts by inhibiting kynurenin-3-monooxygenase in the blood. After that, blood cells send a signal to the brain that stabilizes the functioning of brain cells and prevents neurodegeneration. The fact that the experimental drug does not penetrate the blood-brain barrier and its effectiveness can be assessed using a simple blood test should speed up the process of obtaining permission to conduct clinical trials.

The results of a parallel study conducted by a former employee of Mukhovsky, Flaviano Giorgini from the University of Leicester, provided indisputable genetic and pharmacological evidence of the important role played by kynurenin-3-monooxygenase in the body of fruit flies with simulated Huntington's disease. The results of this work will be published in the on-line version of the journal Current Biology in the near future.

Currently, the researchers are considering possible options for conducting phase I clinical trials of JM6, which they plan to start in 2013.

Article by Daniel Zwilling et al. Kynurenine 3-Monooxygenase Inhibition in Blood Ameliorates Neurodegeneration was published on June 2 in the preliminary on-line version of the journal Cell.

Evgeniya Ryabtseva
Portal "Eternal youth" www.vechnayamolodost.ru based on the materials of Gladstone Institutes:
Gladstone Scientists Discover Drug Candidate for Alzheimer's, Huntington's disease.

07.06.2011

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