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Experimental Treatment Slows Huntington’s Disease Progression


Experimental Treatment Slows Huntington’s Disease Progression

Huntington’s disease is basically a dominant genetic disorder characterized by chorea and progressive cognitive deterioration with a sudden onset during the middle age period. It can only be diagnosed by genetic testing. To be more specific, Huntington’s disease leads to sudden uncontrolled movements, mental and emotional deterioration. Lifetime expectancy ib about fifteen years after the disease has been diagnosed. Until now, the only management of the disease consisted of  treating the symptoms it presents, and the progressive nature of the disease could not be reversed. Over the past decade, hopes for a cure for this debilitating disease are up as several breakthroughs in the medical field emerged.

Researchers have discovered that specific enzymes are in fact the culprit behind the effects and the onset itself of Huntington’s disease. These enzymes are called HDACs. Their role is to enhance the action of the mutation that is responsible for causing the disease, thus worsening its devastating effects. Experimental drugs that work by blocking the HDACs drugs drastically reduce the risk of further mutations.

MRI Brain image of a patient with Hungtington Disease

MRI Huntington Disease

The main author of this research, Professor Robert Lahue of National University of Ireland Galway’s Centre for Chromosome Biology explains the idea on which the study was based “Ongoing mutations in the brain of Huntington’s patients are thought to drive progression of the disease, thus 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.”

Currently, several research laboratories in the US and have begun testing the HDAC inhibitor in order asses its efficiency and safety, using laboratory models of Huntington’s disease. Professor Lahue along with his research team expressed their desire to collaborate with these labs in order to thoroughly evaluate the effect of HDAC inhibitors.

However, the discovery is still in the experimental phase and Professor Lahue clearly states that “It is very exciting that basic research at National University of Ireland Galway, funded by Science Foundation Ireland, has created a new possibility for helping Huntington’s patients and their families.”

The results of the study may also consist of an excellent base for further research towards new treatment options for several other neurological disorders, such as myotonic dystrophy type I, which also seems to be caused by the same type of mutation as the Huntington’s disease.