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Experimental Treatment Slows Huntington’s Disease Progression


Experimental Treatment Slows Huntington’s Disease Progression

Huntington’sdisease is basically a dominant genetic disorder characterized by chorea and progressive cognitive deteriorationwith a sudden onset during the middle age period. It can only be diagnosedbygenetic 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 oftreating the symptoms it presents, andthe progressive nature of the disease could not be reversed. Over the past decade, hopes for a curefor thisdebilitatingdisease are up asseveral breakthroughsin themedical 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 isresponsible for causing the disease, thus worsening its devastating effects. Experimental drugsthat work by blockingthe HDACsdrugs drastically reduce the riskof further mutations.

MRI Brain image of a patient with Hungtington Disease

MRI Huntington Disease

The main author of this research, Professor Robert Lahueof National University of Ireland Galway’s Centre for Chromosome Biology explains theidea on which the study was based“Ongoing mutationsin the brain of Huntington’s patients are thought to drive progression of the disease, thus our discovery suggests that inhibiting HDACfunction 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 USand have beguntesting the HDACinhibitor in orderasses itsefficiency and safety, using laboratorymodels ofHuntington’s disease. Professor Lahuealong with his research team expressed theirdesire tocollaboratewith these labs in order to thoroughly evaluate the effect of HDAC inhibitors.

However, the discovery is still in the experimental phase andProfessor Lahue clearly statesthat “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 thestudy may also consist ofan excellentbase forfurther research towardsnew treatment options forseveral other neurological disorders, such as myotonic dystrophy type I, which also seems to be caused by the same type of mutation asthe Huntington’s disease.