Clinical results show a positive impact of Huntington’s disease gene therapy

Huntington’s disease has long been considered one of the cruelest and devastating diseases; the disease has been successfully treated for the first time, according to doctors. Huntington’s disease runs through families, and it kills brain cells, resembling a combination of dementia, Parkinson’s, and motor neurone disease. 

Until now, treatments could only manage symptoms but could not slow down the progression of the disease. Here is groundbreaking news regarding this most feared neurological disorder. For the first time, a gene therapy called AMT-130 has successfully slowed the progression of the disease in clinical trials. 

This success contributes hope to thousands of families impacted by this feared condition. AMT-130  targets the root cause of the disease, a hazardous protein called huntingtin. The protein slowly damages the brain cells, leading to the severe symptoms associated with HD. The gene therapy instructs the neurons to slow down their production, thus, it slows down the brain damage before it can further harm.

During the clinical trials, patients receiving AMT-130 showed a positive impact on the slowdown of the disease. Many people were able to live their normal lives, including their daily activities and independence, much better than expected. 

Even the patients receiving AMT-130 have shown cognitive abilities such as memory and decision making, offering patients and their families a ray of hope.

The therapy has been conducted through a specialized surgical procedure where a modified virus has been introduced with genetic material directly into the brain. Indeed, this surgery is complex, but there is a probability that long-term benefits from this single treatment could be life-changing for affected people and their families.

Experts believe that this gene therapy can mark the start of an era in treating genetic brain disorders. Scientists and researchers are working to find ways to refine the therapy. Families affected by Huntington’s disease have expressed optimism. The milestone is more than a medical achievement, and for the first time, this disease is no longer seen as completely untreatable.

The first symptom of Huntington’s disease is likely to appear between the 30s and 40s. It is normally fatal within two decades; early treatment can prevent symptoms from ever emerging. 

The director of the University College London Huntington’s Disease Centres, Prof Tabrizi, expressed the result of gene therapy as “spectacular”.She added that they never in their wildest dreams would have expected a 75% slowing of clinical progression.

The treatment could be costlier; however, this is a moment of real hope that hits people in their prime and devastates families.

The success of AMT-130 is a turning point in neurodegenerative disease research. A treatment is slowing the progression of a genetic brain disorder for the first time.