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expert reaction to announcement of a successful human trial of a drug for Huntington’s disease

A phase 1 trial for Huntington’s disease – which took place at UCL – has announced positive results.


Prof. Roger Barker, Professor of Clinical Neuroscience and Honorary Consultant Neurologist, University of Cambridge, said:

“We were involved in this trial, here in Cambridge with 6 patients. I would say that the results are encouraging as it shows delivering therapeutic agents of this type is feasible and well tolerated and the treatment has done what it said it would- namely drop the relevant protein levels in the CSF. The question is whether this is enough to make a difference to patients and their clinical course and that we will have to wait for bigger trials.”


Dr Philippa Brice, PHG Foundation, said:

“This is a potential game-changer, not only for Huntington’s disease patients but also for genomic medicine in general. Though more work needs to be done, if gene silencing lives up to this promise we could be on the brink of some of the personalised treatments that patients with severe genetic diseases need so badly. But this will also raise further questions for society and for policy-makers about how best to use such a powerful technique.”




Declared interests

Prof. Roger Barker: “The University of Cambridge were involved in this trial.”

None others received

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