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Scientific result | Parkinson's disease | Gene and cell 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.
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:
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.
 Tested for the first time in humans with three levels of increasing doses (1x,
2x and 5x).
CEA is a French government-funded technological research organisation in four main areas: low-carbon energies, defense and security, information technologies and health technologies. A prominent player in the European Research Area, it is involved in setting up collaborative projects with many partners around the world.