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A group of researchers from University College London (UCL) in the United Kingdom announced on the 24th of last month, according to local time, that they have verified the possibility of slowing the advancement of Huntington’s disease in the world’s initial gene therapy clinical trial focused on individuals affected by this condition. Huntington’s disease is a progressive brain disorder resulting from damage to nerve cells in the brain. It is an uncommon genetic illness that affects between 5 and 10 people per 100,000, with roughly 75,000 patients in the United States and Europe. The number of patients within the country is estimated to be approximately 1,000.

A group of researchers carried out a clinical study with 29 individuals diagnosed with Huntington’s disease from the UK and the US. They provided gene therapy aimed at stopping the creation of harmful proteins thought to be responsible for the condition. For this, they executed a detailed surgical process where therapeutic genes were inserted into a carefully modified virus, which was then directly introduced into the brain. A narrow tube was placed into a deep region of the brain, and the gene therapy solution was gradually administered over a period of about 12 to 18 hours. This gene therapy, known as ‘AMT-130,’ is a microRNA-based treatment developed by the Dutch biotech firm Uniqure.

The group noted that 12 individuals who were given a high dose of the gene therapy drug experienced a decline of just 0.38 points in motor and cognitive abilities three years post-surgery. In contrast to the usual decrease of 1.52 points seen in Huntington’s patients over the same period, this indicates an approximately 75% reduction in the rate of disease advancement. The team mentioned, “A condition that typically progresses within one year took four years to progress in those treated with gene therapy,” and also highlighted, “One individual showed improvement enough to return to work.”

Nevertheless, certain critics contend that the limited number of participants and the absence of peer-reviewed publication make it premature to consider the results as conclusive. Although patients were monitored for three years following treatment, it is still uncertain if the effects will last over time, and potential adverse reactions might arise, necessitating careful observation. The elevated expense of the therapy is also regarded as a major obstacle. Uniqure mentioned that the gene therapy drug is anticipated to cost approximately 2 million dollars, roughly 2.8 billion South Korean won.