Gene transfer for neurologic disease: agencies, policies, and process.
Advances in molecular biology have contributed to a growing interest in gene therapy as a form of management for neurologic diseases. However, implementation requires knowledge of the regulatory policies governing this field of research, especially in view of the greater stringency imposed by the serious adverse events affecting some patients participating in gene therapy protocols. Educational resources for neurologists, or any clinicians, who hope to serve as potential principal investigators for a gene therapy protocol are not available through any single source, requiring considerable effort to discover appropriate guidance. Summarized here are the regulatory agencies and their requirements, the phases of clinical development with emphasis on a Phase I study, and specific steps leading to an Investigational New Drug application for a biologic product to be used in a gene therapy clinical trial. The links provided to all appropriate Web sites will facilitate the process for the clinician investigator.
Mendell
Jerry R
JR
Center for Neuromuscular Disorders, Children's Research Institute, Department of Neurology, The Ohio State University, WA 3024, 700 Children's Drive, Columbus, OH 43205, USA. mendell.1@osu.edu
Miller
Andra
A
D002985:Clinical Protocols / Q000592:standards; D017321:Clinical Trials, Phase I as Topic; D004274:DNA, Recombinant / Q000627:therapeutic use; D015507:Drugs, Investigational; D028744:Ethics Committees, Research; D005568:Forms and Records Control; D018014:Gene Transfer Techniques / Q000009:adverse effects / Q000592:standards / Q000639:trends; D015316:Genetic Therapy / Q000009:adverse effects / Q000331:legislation & jurisprudence / Q000592:standards / Q000639:trends; D005822:Genetic Vectors / Q000009:adverse effects / Q000627:therapeutic use; D006077:Government Agencies; D006291:Health Policy; D006801:Humans; D017387:Investigational New Drug Application / Q000331:legislation & jurisprudence; D009316:National Institutes of Health (U.S.); D009422:Nervous System Diseases / Q000628:therapy*; D012306:Risk; D014481:United States; D014483:United States Dept. of Health and Human Services; D014486:United States Food and Drug Administration; D018172:United States Office of Research Integrity
D004274:DNA, Recombinant; D015507:Drugs, Investigational