1 After Gene Therapy: Issues in Long-Term Clinical Follow-up and Care

Abstract

Publisher Summary This chapter focuses on the clinical trials of somatic gene therapy that began in 1989 after several decades of debate concerning the biological, ethical, and social implications of genetic engineering. To a large extent, this analysis arose independent of the traditions of clinical investigation and the practical problems associated with clinical research and clinical practice. By the end of 1994, more than 80 clinical trials involving gene transfer into human subjects can be approved by the National Institutes of Health (NIH) through its recombinant DNA Advisory Committee and more than 200 patients could have participated in these trials. The chapter discusses various distinct issues related to this. The first is the need to have effective follow-up for patients who receive somatic gene therapy to fully assess potential adverse experiences resulting from these therapies. The second is the need to provide continuous clinical care to patients who are treated with somatic gene therapy. This relates to both the responsibility of investigators and institutions to ensure that patients with adverse experiences receive appropriate care for these complications and to their responsibility to provide continuing therapy to patients who may benefit from clinical trials. Several characteristics of the current clinical activity in the gene therapy make follow-up and the commitment to long-term care particularly difficult.