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Case study: Gene Therapy for Parkinson's Disease

Find out more about the translational research the NIHR carries out in dementia

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"Helping develop treatments for a disease which affects thousands of people in the UK is something we should be proud of.“

NIHR researchers collaborate with industry to conduct a UK first-in-man gene therapy trial for Parkinson’s disease, delivering the synthetic enzymes for dopamine (ProSavin®) into patient’s brains. 

UK First-in-Man Gene Therapy Trial for Parkinson’s Disease

Investigators in the Dementia and Neurodegeneration theme from the NIHR Cambridge Biomedical Research Centre have collaborated with industry partner Oxford BioMedica to conduct a UK first-in-man gene therapy trial for Parkinson’s disease.

The trial used the tricistronic vector (the vehicle used to transfer genetic material) developed by Oxford BioMedica to deliver the synthetic enzymes for dopamine (ProSavin®) into the brain of
patients with Parkinson’s disease. The trial commenced with nine patients in Paris, France, but now involves NIHR facilities in Cambridge, UK as a second site. Treatment is designed to improve the delivery of dopamine within the brain and by so doing, smooth out the motor fluctuations of patients with advancing Parkinson’s disease.

Vital research

Parkinson’s disease affects approximately 4 million patients globally (127,000 people in the UK) and is projected to rise to almost 9 million by 2030. While there is no cure for Parkinson’s
disease and no treatment can prevent the disease from progressing, current treatments can usually ease symptoms. In Parkinson’s disease there is a loss of specific nerve cells in the
brain that release a chemical called dopamine.

Dopamine plays a vital role in the control of movement, hence patients with Parkinson’s disease find they have problems with normal movement. Movement is typically slow and stiff, but
drugs (e.g. L-DOPA) have been developed that replace this lost neurochemical. Over time these drugs do begin to lose their effectiveness, producing their own side-effects such as
the involuntary jerking of the body’s muscles, called L-DOPA induced dyskinesias.

These involuntary movements can be disabling, but can be managed if a smoother delivery of dopamine can be achieved. The new gene therapy is injected directly into the brain of Parkinson’s disease patients, improving the patient’s ability to produce uniform amounts of dopamine at the site of treatment. This helps smooth the motor control in Parkinson’s disease, whilst allowing the patient to reduce the amount of dopamine required to be taken orally.

Findings

In April 2013, 15 patients had received this treatment: 12 in Paris and three at the NIHR’s Cambridge facilities. Treatment began with very low doses. As the trial progressed, dose of the agent increased and the delivery method was been improved upon. The treatment was shown to be well tolerated, with significant improvements in motor behavior from baseline at 1 year. The results were published in the Lancet with a further follow-on paper in Hum Gene Ther Clin Dev. 2018 Sep; 29(3): 148-155. A new gene therapy trial started in 2018 with the company (Oxford BioMedica). The first patient was treated in London in the beginning of November 2018 and the first patient in Cambridge was treated in mid-November 2018. Oxford BioMedica (the company working on this therapy) was bought by Axocant during summer 2018 for ~$850million.

Patient impact

This treatment has the potential to help patients, but more research is required to progress this treatment to a stage where patients with advanced Parkinson’s disease can have smoother control of their disease and allow them to reduce oral drug therapies, so reducing the risk of side-effects.

Professor Roger Barker, Theme Lead for Dementia and Neurodegenerative Disorders at NIHR Cambridge Biomedical Research Centre commented: “This collaboration with Oxford BioMedica is a bold step forward not only in the use of an advanced gene-based treatment, but the results being achieved from a new infusion technique which requires fewer needle tracks into the brain. I’m most excited to be progressing this vital research with a UK biopharm company. Helping develop treatments for a disease which affects thousands of people in the UK is something we should be proud of.“


 

Back to NIHR Dementia Translational Research Collaboration page.