New Drug Provides a Breakthrough in the Treatment of Huntington’s disease

Published By : 12 Dec 2017 | Published By : QYRESEARCH

For the first time ever an experimental drug has successfully diminished the level of toxic protein that has been causing Huntington’s disease in human beings. An inherited disorder, Huntington’s disease in which huntingtin protein mutates and leads to the damage of nerve cells of the brain.

New Drug Lowers the Level of Huntingtin Protein

Ionis-HTTRx, the new drug has successfully reduced the level of mutated huntingtin protein without causing any side effect to the patient. Hailing the result as a revolutionary, Sarah Tabrizi, director of UCL’s Huntington’s Disease Centre, said that now the emphasis would be to go for a larger trial to examine if the drug to delay the progression of the disease.

The trial involved 46 patients who were in the early stages of Huntington’s disease and came from various nine centers of Canada, the U.K., and Germany. The initial results were made public on last Monday.

The patients were given four doses and were injected with either a placebo or the new drug. It was injected directly into their spinal fluid so that the drug could easily and promptly reach the human brains. The dosage was increased over time for those patients who were receiving the drugs and they were kept under close watch as well. Toxic protein concentration in the spinal fluid was measured both after and before the dosage of new drug.

The result revealed substantial decline in level of mutated huntingtin protein. The researchers are looking forward to go for bigger trails so as to determine if the drug could successfully delay the progression of the disease and make a significant difference in the clinical course of the patient. The next larger trial will involve hundreds of patients coming from various centers.

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