Gene Therapy Holds Promise as Parkinson’s Disease Treatment

Posted on 2009-10-20 06:00:00 in Gene Therapy | Parkinsons Disease |

Affecting more than 4 million people worldwide, Parkinson’s Disease (PD) is a chronic motor system disorder characterized by tremor, rigidity; bradykinesia (slowness of movement) and postural instability.  There presently is no cure, and medications, while offering symptom control, can become less effective over time.  Bechir Jarraya, from the department of neurosurgery at Henri-Mondor Hospital (Paris, France), and colleagues inserted genes into brain cells of Macaque monkeys induced to have PD-like symptoms, in an effort to resume production of dopamine, the neurotransmitter that is thought to be deficient in PD patients and the cause of the neurological manifestations.  They restored dopamine at about 50% of normal concentrations, resulting in motor improvements without the side effects seen with conventional drugs. The benefits were sustained for up to 44 months.   Commenting that: “Gene therapy–mediated dopamine replacement may be able to correct Parkinsonism in patients without the complications of dyskinesias,” the team is hopeful that these advancements will herald innovations that may be applied to humans afflicted with Parkinson’s Disease.

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Bechir Jarraya, Sabrina Boulet, G. Scott Ralph, Caroline Jan, Gilles Bonvento, Mimoun Azzouz, James E. Miskin, Masahiro Shin, Thierry Delzescaux, Xavier Drouot, Anne-Sophie Herard, Denise M. Day, Emmanuel Brouillet, Susan M. Kingsman, Philippe Hantraye, Kyriacos A. Mitrophanous, Nicholas D. Mazarakis, Stephane Palfi.   “Dopamine Gene Therapy for Parkinson’s Disease in a Nonhuman Primate Without Associated Dyskinesia.”  Sci Transl Med 14 October 2009 1:2ra4. DOI:10.1126/scitranslmed.3000130


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