In a revolutionary advancement for neurology, scientists have successfully slowed the progression of Huntington’s disease for the first time using a groundbreaking new treatment. Researchers from University College London (UCL) have announced findings that could “change everything” for individuals suffering from this debilitating condition, which progressively worsens over time and currently has no cure. Huntington’s disease significantly impacts a person’s movement, thinking, and mood.
The study focused on a novel gene therapy called AMT-130, which is administered through brain surgery. Initial clinical trials involving 29 patients demonstrated that those receiving a high dose of AMT-130 experienced a remarkable 75 percent reduction in disease progression over a period of 36 months. This promising data was reported by uniQure, a leading gene therapy company with a presence in both the Netherlands and the US.
One of the most compelling aspects of this treatment is that a single dose of AMT-130 is anticipated to last for a patient’s entire lifetime, offering a long-term solution for managing the symptoms of Huntington’s disease.
Professor Ed Wild, the principal investigator from UCL’s Huntington’s Disease Centre, expressed his optimism about the findings, stating, “This result changes everything. Based on these results, it seems likely that AMT-130 will be the first licensed treatment to slow Huntington’s disease, which is truly world-changing stuff.”
He further emphasized the importance of making this treatment accessible to all patients in need, while continuing the search for even more effective therapies. “Trial results come through in numbers and graphs, but behind each data point is an incredible patient who volunteered to undergo major neurosurgery to be treated with the first gene therapy we’ve ever tested in Huntington’s disease. That is an extraordinary act of bravery for the benefit of humanity,” said Professor Wild.
Notably, he shared that some patients enrolled in the trial are demonstrating stability over time, a shift from what is typically observed in Huntington’s disease cases. Remarkably, one patient who had medically retired due to the disease has been able to return to work.
Professor Sarah Tabrizi, the lead scientific adviser on the trial and also affiliated with UCL’s Huntington’s Disease Centre, expressed her excitement regarding the findings. “I am thrilled that this study of AMT-130 showed statistically significant effects on disease progression at 36 months. These groundbreaking data are the most convincing evidence in the field to date, underscoring the disease-modifying effect in Huntington’s disease, where an urgent need persists,” she stated.
Tabrizi highlighted that AMT-130 has the potential to not only preserve daily functioning for patients but also to allow them to remain in the workforce longer, ultimately meaningfully slowing the progression of this devastating disease.
The development of AMT-130 marks a monumental step forward in the fight against Huntington’s disease, potentially altering the lives of countless patients and their families. As further research and clinical trials continue, there is hope that effective treatments will become widely available, offering a brighter future for those impacted by this challenging condition.
