New therapy successful in treating Huntington’s disease

Researchers on Wednesday released results of a clinical trial aimed at slowing the progression of the genetic disorder that gradually diminishes a person’s ability to move and think. Scientists with the company uniQure developed a gene therapy that reduced the disease’s acceleration in patients who received a high dose by 75% over three years. Patients received the therapy during an hours-long brain surgery and researchers compared their outcomes against a group of Huntington’s patients who received standard care. One of the 29 patients in the trial previously had to retire due to the disease but was able to return to work after treatment, said researcher Ed Wild. The company behind the treatment plans to apply for approval with the U.S. Food and Drug Administration early next year, and will seek approval in the United Kingdom and Europe as well.

What is Huntington’s? The progressive disease causes nerve cells in the brain to decay over time and is caused by a genetic mutation often passed down by a parent. It is quite rare and only occurs in about one of every 10,000 to 20,000 people, according to University of California San Francisco Health. Most people with the disease begin experiencing symptoms in their 30s or 40s that can include mood swings and depression. As the disease progresses, it causes uncontrolled movements, loss of brain function, and eventually paralysis. The majority of patients die within one to three decades of diagnosis. There is currently no other treatment to stop, slow, or prevent symptoms from developing and most patients receive a variety of therapies aimed at maintaining physical and mental capabilities.

Dig deeper: Read my report about the FDA approving two genetic treatments for sickle cell disease.