Huntington's disease is an inherited genetic disease, causing progressive damage to certain nerve cells in the brain. This damage worsens over time and affects movement, cognition, and behavior. The symptoms of this disease are similar to dementia, Parkinson's and motor neuron disease before eventually causing death.
This week, the company uniQure announced that the gene therapy treatment they developed successfully slowed the progression of Huntington's disease in patients by an average of 75% in clinical trials. This is the first time that a treatment that slows the progression of the disease has been successfully carried out. In Malaysia, this disease is rare with the first case reported in 1994.
Through gene therapy called AMT-130, a virus modified to carry a DNA chain is injected directly into the striatum of the brain that is susceptible to Huntington's disease. When it enters the brain cell, this DNA chain instructs the neuron to produce its own therapy that prevents its death. Without treatment, the mutated huntingtin protein will slowly kill brain neurons until the patient shows symptoms of dementia, Parkinson's and motor neuron disease.
A total of 26 patients who participated in the 3-year clinical trial had a better quality of life than patients who did not receive any treatment. In one case, a patient who should have been in a wheelchair is still able to walk, and another who retired due to health reasons is now back to work.
Huntington's disease is usually diagnosed in the 30s and 40s with an estimated life expectancy of only 20 years. The AMT-130 treatment allows patients to live for several more decades even if it is not completely cured.
uniQure, a company based in the Netherlands and the United States, has applied to sell this treatment as early as 2026. However, not everyone will be able to enjoy the treatment because the cost is expected to reach millions of dollars, similar to the sickle cell anemia gene therapy treatment that was created two years ago.