Drug may prove to be first effective treatment for Huntington’s disease | Slone Partners

Researchers at the University of California, San Diego School of Medicine have developed a drug that may quell the gene mutation responsible for causing Huntington's disease – a rare and incapacitating genetic disorder.

In animal trials, test subjects were able to show long-term improvement, beginning with improved motor functioning within two months. Prolonged effects were observed even after the immediate impact of the drug had subsided.

"This finding raises the prospect of a 'huntingtin [gene] holiday,' which may allow for clearance of disease-causing species that might take weeks or months to re-form," lead researcher Dr. Don Cleveland said in a statement. "If so, then a single application of a drug to reduce expression of a target gene could 'reset the disease clock,' providing a benefit long after huntingtin suppression has ended."

Prior to news of this treatment, the best hope for those with a family history of Huntington's was that a genetic test would be able to inform them that they did not possess the gene that predisposes them toward developing the condition.

Earlier this month, this blog told the story of Kristen Powers, a teenager whose mother died from Huntington's disease and who was concerned she too would develop the condition. Following a negative genetic test, though, she found out she would avoid the debilitating symptoms associated with Huntington's, which include memory loss and a deterioration of coordination.

For Powers and other individuals who could be at risk for suffering from a genetic condition, negative test results can set their minds at ease or help them receive timely treatment if necessary. This is why diagnostic laboratories and other stakeholders in the genetic testing process must have the best industry talent on their side.

This article is brought to you by Slone Partners, a leading laboratory recruitment firm in the emerging sciences of molecular, clinical, and in-vitro diagnostics, anatomic pathology and personalized medicine.

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