University College London Achieves Breakthrough in Huntington's Disease Gene Therapy

What's Happening?

University College London has successfully treated Huntington's disease for the first time using a breakthrough gene therapy. The disease, which is caused by a single gene defect, leads to severe neurological degeneration. The therapy, led by Prof Sarah Tabrizi, involves a surgical procedure that delivers a drug to the brain, inactivating the mutant protein responsible for the disease. This treatment has slowed the disease's progression by 75% in patients over three years. The therapy is expected to be costly due to the complexity of the procedure, which takes 12 to 20 hours. The trial involved 29 patients in the UK and US, and results show significant slowing of disease progression and preservation of neurons.

Why It's Important?

This development is significant as it offers hope to those affected by Huntington's disease, which has been incurable until now. The therapy could allow patients to maintain independence and work for longer periods, potentially preventing symptoms if administered early. With an estimated 6,000 to 10,000 people affected in the UK, and many more carriers of the gene, this breakthrough could lead to increased genetic testing and early intervention. The success of this trial may pave the way for similar treatments for other genetic disorders, revolutionizing the approach to neurodegenerative diseases.

What's Next?

The company uniQure plans to submit the drug for approval in the US early next year. If approved, this could lead to wider availability of the treatment, offering new hope to families affected by Huntington's disease. The therapy's success may encourage further research into gene therapies for other conditions, potentially leading to new treatments for various genetic disorders.