What Are the Successful Therapeutic Effects of Fatal Brain Disorder Huntington's Disease?

by Kim Eunha

Published 27 Sep.2025 14:54(KST)

Updated 27 Sep.2025 15:21(KST)

First Successful Gene Therapy Trial for Huntington's Disease in the UK

Huntington's disease, a fatal degenerative brain disorder that is inherited within families, has demonstrated successful therapeutic effects in the world's first gene therapy clinical trial conducted in the United Kingdom.

Yonhap News, citing The New York Times, reported on the 27th that this trial has, for the first time, suggested the possibility of treating Huntington's disease, which has until now been considered incurable.

Gene therapy that slows the progression of Huntington's disease has shown effectiveness in clinical trials. Yonhap News

According to the report, Professor Ed Wild of the Department of Neurology at University College London directly injected a newly developed gene therapy into the brains of patients exhibiting early symptoms of Huntington's disease.

Researchers identified toxic proteins as the main factor causing Huntington's disease. Therefore, they conducted gene therapy designed to prevent the production of these toxic proteins in the human body. They surgically delivered specially designed DNA fragments to brain cells using a harmless virus as a carrier.

The medical team drilled a very small hole in the patient's head, inserted a microcatheter deep into the brain, and then slowly infused the gene therapy agent into the brain over a period of 12 to 18 hours.

Among the 12 patients who received the maximum dose of the therapy over three years, the rate of disease progression-measured by comprehensive assessments of motor skills, cognitive abilities, and daily living activities-was reduced by 75% compared to the control group. Indicators of brain cell damage were also significantly lower.

(This photo is not directly related to the article.) Pixabay

One of the patients was able to return to work. In addition, several patients, while still requiring the use of a wheelchair, recovered enough to be able to walk.

Meanwhile, Huntington's disease is a degenerative brain disorder caused by the deterioration of brain nerve cells. If one parent has the disease, their children have a 50% chance of developing it. Because the disease causes involuntary movements that resemble dancing, it is also nicknamed "chorea." It is estimated that there are about 40,000 patients worldwide, with particularly high numbers in Europe and the United States. In South Korea, there are approximately 1,000 patients.

Huntington's disease typically presents initial symptoms in people in their 30s or 40s. It begins with mood changes, anger, and depression, and gradually progresses to involuntary movements, dementia, and paralysis. Some patients die within 10 years of diagnosis. If this treatment becomes commercially available, patients are expected to be able to live independently for much longer. There is also a possibility that early administration before the onset of symptoms could prevent the disease from developing altogether.