Current status of gene therapy trials for Parkinson's disease.
The incidence of Parkinson's disease (PD) increases greatly with age, and the baby-boomer population can expect to generate a large number of individuals with the disease, all of whom will have significantly increased medical care needs over periods of 20 years or more. This emerging healthcare burden to our society calls for accelerated efforts to understand this disease better and treat it more effectively. The growing interest in gene therapy grew out of a recognition that new medicines may be needed to combat the relentless progression of the disease in the face of conventional pharmaco-therapies and surgical interventions that have so far failed to offer more than palliative relief. The potential of gene therapy to alter dramatically the course of the disease lies very much with the challenge of converting a research tool into a medical option, a process that clearly requires a unique combination of rigor and flexibility. In this review, we examine the unique aspects of gene therapy that make its use in PD attractive, but also analyze the difficulties of employing a medicine that acts for the rest of the patient's life.
Fiandaca
Massimo
M
Department of Neurological Surgery, Movement Disorder Research Program, University of California, Mission Center Building, San Francisco, CA 94103, USA.
Forsayeth
John
J
Bankiewicz
Krystof
K
D000818:Animals; D017321:Clinical Trials, Phase I as Topic; D000229:Dependovirus / Q000235:genetics; D004195:Disease Models, Animal; D004353:Drug Evaluation, Preclinical; D015316:Genetic Therapy / Q000331:legislation & jurisprudence / Q000639:trends*; D005822:Genetic Vectors; D006801:Humans; D010300:Parkinson Disease / Q000628:therapy*