Gene Therapy Page

Gene Therapy

• Availability of GCRC Resources for Long Term Follow Up of Participants in Gene Transfer Protocols (NOT-RR-04-005)
  
  In 2002, three years after receiving retrovirally transduced stem cells, two subjects who were enrolled in a gene transfer research study developed T cell leukemia. These events highlighted the potential for serious adverse events to emerge long after the subject's active involvement in the study . Gene transfer research investigators must confront these risks in order to maximize the protection of human subjects who participate in gene transfer research protocols and to help advance understanding of the underlying mechanisms involved. To address these concerns, the FDA now recommends that all subjects enrolled in gene transfer studies be followed for 15 years after administration of the study agent. FDA implements this recommendation by requiring gene transfer investigators to document their long-term follow-up plans in their IND applications.
  
  
• NIH Guidance on Informed Consent for Gene Transfer Research
  
  Since before the first clinical gene transfer trial began enrolling subjects, the National Institutes of Health (NIH) and its Recombinant DNA Advisory Committee (RAC) have sought to assist investigators in developing good consent forms and processes for clinical gene transfer research. These sections address issues unique to gene transfer, as well as issues that gene transfer has in common with other forms of clinical research.
  
  
• National Gene Vector Laboratories (NGVL)
  The National Gene Vector Laboratories (NGVL) "Policy and Procedures" and an "Application for NGVL Resources" (Appendix I) to be used by investigators wishing to obtain clinical grade vectors or pharmacology/toxicology support from the NGVL. The "Policy and Procedures" document describes the goals of the NGVL, the application and review process for obtaining NGVL resources, and the responsibilities of NGVL members and investigators. Investigators must agree to follow these guidelines to be considered for receipt of NGVL resources.
  
  
• Clinical Trials in Human Gene Transfer: Recombinant DNA and Gene Transfer
  
  The NIH Office of Biotechnology Activities website which contains information on a database of gene therapy clinical trials.