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Doctors have reported a significant breakthrough in the treatment of Huntington’s disease, a hereditary neurological disorder known for its severe impact on patients and families. A new gene therapy has demonstrated the ability to slow the disease’s progression by 75%, marking a potential turning point in the management of this condition.
Huntington’s disease typically emerges in a person’s 30s or 40s and is ultimately fatal within about two decades. The disease is characterized by involuntary movements, cognitive decline, and various behavioral changes. If one parent carries the faulty gene responsible for the disease, there is a 50% chance their child will inherit it. The new therapy targets the toxic huntingtin protein, which is responsible for the neurodegeneration associated with Huntington’s.
The innovative treatment involves a single administration via a specially engineered virus, which is infused deep into the brain during a complex surgical procedure lasting 12 to 18 hours, guided by real-time MRI scans. This virus delivers DNA instructions that enable brain cells to produce microRNA fragments, which then disable the messenger RNA that builds the harmful huntingtin protein. This process effectively reduces the levels of the toxic protein and protects neurons from further damage.
Initial trial results involving 29 patients have shown remarkable outcomes. Three years post-surgery, participants displayed an average 75% slowing in disease progression, assessed through cognitive, motor, and daily functioning metrics. Additionally, biomarker data indicated a reduction in neurofilaments—an indicator of cell death—in spinal fluid samples, suggesting that the treatment is preserving brain cells.
While the results have generated optimism among families affected by Huntington’s disease, some challenges remain. A number of patients experienced inflammation linked to the viral delivery system, resulting in temporary headaches or confusion, although these side effects generally resolved with time or steroid treatment. The cost of the therapy is expected to be high, given the intricacies of the neurosurgery involved.
Despite these hurdles, experts see this as a transformative moment. Plans are underway for further trials, including preventive approaches for individuals who carry the Huntington’s gene but have not yet developed symptoms. UniQure, the biotech company behind the therapy, is preparing to seek regulatory approval in the U.S. by early 2026, with discussions for the UK and European markets to follow.
This groundbreaking advancement not only brings hope to those currently living with Huntington’s disease but also opens new avenues for potential prevention, changing the narrative from a grim inevitability to one of possibility for a brighter future.
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