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.