Investigational Gene Therapy Trial for Parkinson’s Disease



A Phase I clinical trial for the treatment of Parkinson’s with Glial Cell Line Derived Neurotrophic Factor (GDNF) has completed the first procedure on the first patient at the University of California, San Francisco.  Krystof Bankiewicz, M.D., Ph.D. of UCSF and John D. Heiss, M.D., from the National Institutes for Neurological Diseases and Stroke are the joint chief investigators.  This trial will treat 24 patients in four cohorts that will receive varying doses of the GDNF. This is the first stage of testing for safety and dose response.

GDNF provides neuro regeneration and is expected to provide neuro-protective properties to strengthen dopamine producing cells in the brain.  It will be delivered directly to the putamen region of the brain, where dopamine is normally produced but is disrupted by Parkinson’s disease.   According to Dr. Bankiewicz “The success of gene therapy in patients requires accuracy in delivery.”  To achieve this accuracy, a program from the company MRI Interventions called “ClearPoint” is used.  “ClearPoint” is a system of technology to enable precise and exact placement of the gene therapy using MRI.  The infusion of the gene therapy to this very small and precise region of the brain is actually considered “minimally invasive” surgery.  The first patient received treatment on May 20th and there have been no safety issues.

This study is a collaboration between MRI Interventions, the manufacturers of the “ClearPoint” system for precise visualization, uniQure V.B. a Dutch Company that is providing the GDNF and UCSF.   It is sponsored by National Institutes of Health.  MRI Interventions “ClearPoint” system has received FDA approval.  UniQure specializes in human gene therapy treatments with potentially curative results.  They currently have one product that has been successful and are eager to bring new therapies to patients with severe disorders.

Additional note: This treatment of Parkinson’s with Glial Cell Line Derived Neurotrophic Factor (GDNF) was used in the UK in 2003 with remarkable results.  After one year, there were no serious clinical side effects, a 39% improvement in the off-medication motor sub-score of the Unified Parkinson’s Disease Rating Scale (UPDRS) and a 61% improvement in the activities of daily living sub-score. Medication-induced dyskinesias were reduced by 64% and were not observed off medication during chronic GDNF delivery. Positron emission tomography (PET) scans of [(18)F]dopamine uptake showed a significant 28% increase in putamen dopamine storage after 18 months, suggesting a direct effect of GDNF on dopamine function.

Nat Med. 2003 May;9(5):589-95. Epub 2003 Mar 31.Direct brain infusion of glial cell line-derived neurotrophic factor in Parkinson disease. Gill SS, Patel NK, Hotton GR, O’Sullivan K, McCarter R, Bunnage M, Brooks DJ, Svendsen CN, Heywood P. Source: Frenchay Hospital, Institute of Neurosciences, Bristol, UK.

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