Huntington’s Disease gene therapy shows 75 per cent slowing of disease progression

A new gene therapy to treat Huntington’s disease has shown positive data from the study of AMT-130 which is being run at UCLH and other sites.

The phase I/II study demonstrates a statistically significant slowing (75 per cent) of disease progression measured at 36 months compared to the control.

Jack May-Davis who is 30 from Sussex found out when he was just 19 that he carried the Huntington’s Disease gene. Jack has already lost two family members including his father, a Met Police detective, who started to develop symptoms in his mid-40s and quickly required fulltime care as the disease progressed. 

Since finding out he also carries the gene Jack has been involved with trials at the National Hospital for Neurology and Neurosurgery (NHNN) at UCLH helping support research and to do something positive with the situation he found himself in.

Jack said; “The results are astonishing – I’m lost for words. It is just amazing. When I started participating in trials I never thought something would be developed in a timeframe that might be actually be useful for me. This feels like a huge moment that will mean so much to families who carry the Huntington’s gene.”

Sarah Tabrizi, Professor of Clinical Neurology and Honorary Consultant Neurologist at the National Hospital for Neurology and Neurosurgery, UCLH is the lead scientific advisor for the study. Sarah said:

“I am thrilled that this pivotal study of AMT-130 showed statistically significant effects on both cUHDRS and TFC at 36 months, supported by mean CSF NfL remaining below baseline. I believe these groundbreaking data are the most convincing in the field to date and underscore potential disease-modifying effects in Huntington’s disease, where an urgent need persists. These data indicate that AMT-130 has the potential to meaningfully slow disease progression – offering long-awaited hope to individuals and families impacted by this devastating disease.”

Prof Edward Wild consultant neurologist at the National Hospital for Neurology and Neurosurgery, UCLH said;

“This result is the good news we’ve been working and waiting for: not just a treatment that slows progression of this terrible disease, but one that does so with truly stunning effectiveness. It is nothing less than the dawn of a new age for families impacted by Huntington’s disease. We must now work no less diligently to turn this breakthrough into something that benefits everyone who needs it.”

The gene therapy company uniQure believes that the consistently favourable results in functional, motor and cognitive endpoints at 36 months observed in the high dose group, compared to the variable trends observed in the low dose group, reflect a dose-dependent response to AMT-130.

AMT-130 was generally well-tolerated, with a manageable safety profile at both doses.

uniQure plans to submit for regulatory approval in the United States next year.

Read more on the BBC website