Huntington’s disease is an inherited condition that causes neurodegeneration - the progressive loss of neurons in the brain. This can lead to changes in movement, thinking, and mood. Reports of a breakthrough hit the headlines last week: a press release from a clinical trial reported that using gene therapy they have managed to slow disease progression for the first time.
Some are warning that it is too early to say we have a cure for Huntington’s given that these results are very early and have not yet been reviewed.
What does it mean to treat a neurodegenerative disease like Huntington’s? How is slowing the progression of a disease different from curing it? And what do the results of this trial mean for families living with the condition today?
In this episode, we speak with Professor Rachael Scahill, Associate Director of the Huntington’s Disease Centre at University College London. We discuss how Huntington’s affects the brain, what this gene therapy involves, and what these results could mean for the future of neurodegeneration research.
This episode is part of our neuroscience series, produced by PhD student Chloe Carrick, Youth Advisor Anushka De, and Research Fellow Dr. Kathryn Bates. Click the subscribe button on this page to get episodes straight to your inbox!
Resources
Read more about the work of the Huntington’s Disease Centre directed by Sarah Tabrizi
Read about Huntington’s Disease news in plain language on HDBuzz
Read about uniQure, the company who led this trial
What you’ll find in this episode
3:05 - What is a neurodegenerative disease like Huntington’s?
8:42 - Gene therapy explained
10:50 - Interpreting results of the trial
16:00 - What role do companies play in clinical trials?
20:00 - Slowing a disease’s progression vs curing a disease
23:00 - Implications of the trial’s findings
26:35 - Next steps in working towards a treatment
The views and opinions expressed by guests on this podcast are their own and do not necessarily reflect those of the host or The Science or Fiction Podcast.
This episode was supported by the King’s Public Engagement Small Grant funding scheme.
