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Scientific result | Parkinson's disease | Gene and cell therapy

Parkinson’s disease: a major breakthrough thanks to gene therapy

​An Anglo-French team (AP-HP, Inserm, CEA/Mircen, UPEC, Oxford Biomedica, Cambridge University) coordinated by Professor Stéphane Palfi (head of the neurosurgery department at the hôpital Henri-Mondor) conducted a gene therapy trial in 15 patients with an advanced form of Parkinson’s disease. Their promising results are being published in The Lancet today.

Published on 10 January 2014

​An Anglo-French team (AP-HP, Inserm, UPEC, CEA/Mircen, Oxford Biomedica, and Cambridge University) conducted a phase I/II¹ clinical gene therapy study in patients with an advanced form of Parkinson’s disease. Fifteen patients were able to benefit from this treatment that consists of injecting a vector expressing the genes of three enzymes essential to dopamine biosynthesis (which is missing in Parkinson’s disease).

Thanks to this therapy, certain cells in the brain begin again to produce and secrete dopamine. In all patients, the motor symptoms of the disease showed improvement up to 12 months after treatment delivery. Looking back on the last 4 years, this study demonstrates at this point the safety and tolerability of the lentiviral vector used for the first time in humans: ProSavin®, developed from the equine infectious anemia virus (EIAV).

This study was coordinated by Professor Stéphane Palfi, head of the neurosurgery department at the hôpital Henri-Mondor (AP-HP), within the neurolocomotor center led by Professor Pierre Cesaro. The CEA’s contribution involved the two following biomedical platforms:

  • MIRCen, a translational research infrastructure dedicated to biotherapy and imagery in the field of neurodegenerative diseases;
  • the Service Hospitalier Frédéric Joliot, which develops functional and molecular imaging using positron emission tomography, mainly for applications in neurology, oncology, psychiatry.


This clinical trial follows a preclinical study published in 2009, which showed for the first time the effectiveness and safety of this drug in an animal model. Performed within the MIRCen translational platform at the CEA, it opened the way for the ProSavin® clinical study.


Principe du traitement thérapeutique via un « gêne médicament » (© CEA)
Key figures:
15 patients treated
1 lentiviral vector, used for the first time in humans
3 dose levels tested
Zoom : Parkinson's disease
With approximately 120,000 patients in France, Parkinson’s disease is the most common neurodegenerative disorder after Alzheimer’s disease. It is expressed mainly through motor symptoms of progressive and increasing severity, such as tremors, rigidity of the limbs and reduced body movements. This condition is due to the degeneration of neurons that produce dopamine, a neurotransmitter involved in motor control. Currently, the treatment of people with this disease consists of taking drugs that mimic the action of the missing dopamine in the brains of these patients. If this treatment provides a good improvement in motor activity in the early stages of the disease, severe side effects occur over time, including fluctuations in the treatment effect and involuntary movements (dyskinesia).

[1] Tested for the first time in humans with three levels of increasing doses (1x, 2x and 5x).

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