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NDCN’s Associate Professor Andrea Nemeth is leading the Oxford part of a clinical trial for Hungtinton’s disease.

None © Oxford University Hospitals NHS Foundation Trust
Professor Andrea Nemeth with patient Danny Atkins

This international collaborative trial, sponsored by Roche pharmaceuticals, is the first aiming to lower the amount of the disease-causing toxic protein produced in the nervous system of patients. If successful, the trial could have implications not just for Huntington’s disease but also genetically similar conditions such as the spinocerebellar ataxias.

This trial is the culmination of over 15 years of collaboration between Oxford University Hospitals NHS Foundation Trust clinical departments and key local research teams at the University of Oxford and Oxford Brookes University, supported by the NIHR Clinical Research Network.
- Andrea Nemeth

Huntington's disease is an inherited genetic condition that typically begins in mid-life. It results in movement control difficulties and dementia, and can cause significant behavioural problems. It progresses to severe disability and death over 20 years. Children of affected individuals have a 50% risk of inheriting the same disease, meaning that Huntington’s has a profound effect on whole families.

Three patients at Oxford University Hospitals NHS Foundation Trust are among the first in the world to receive a pioneering experimental treatment. The GENERATION HD1 trial being conducted at the John Radcliffe Hospital is investigating a compound that is attempting to limit the production and lower levels of the toxic protein that kills nerve cells and causes the disease. An experimental compound, an antisense oligonucleotide, is injected directly into the spinal fluid at the base of the spine. “We hope that this compound will then reach the areas of the brain that are affected, and so reduce the amount of toxic protein in those cells,” Prof Nemeth explained.

Patients will receive the drug every two months for two years, and will be assessed frequently, both clinically and using MRI scans and other biochemical tests. They will also wear innovative smart-trackers that monitor other aspects of their daily lives.

One of the patients taking part in the Oxford trial is Danny Atkins, a 39-year-old father of five from Milton Keynes. He was diagnosed with HD nearly a year ago. “Everybody hopes for the condition to slow down. Even if we can get to a point where there’s a treatment, so my children don’t have to worry about Huntington’s. I’m so happy to be taking part, and the team here have been absolutely amazing.”

This trial, the outcome of which is expected in three years, represents the culmination of a massive international collaborative effort to treat this disease, which gained momentum when the causative genetic mistake was identified in the early 1990s. The team at Oxford University Hospitals, led by Professor Nemeth of the Oxford Centre for Genomic Medicine, and Dr Richard Armstrong in the Department of Neurology, have been at the forefront of this collaboration, having played an instrumental role in establishing the UK and European HD Networks and now contributing to large international efforts. Other key researchers involved include NDCN’s Chrystalina Antoniades and Gwenaelle Douaud, and Prof Helen Dawes of Oxford Brookes University.