Cell and Gene Therapy Program

Elements of Cell and Gene Therapy

Bone Marrow and Stem Cell Transplantation: BMT and/or PBSC are forms of cellular therapy that have become standard treatment for an increasing number of both malignant and nonmalignant diseases. Stem cell therapy in combination with conventional modalities has become accepted therapy for leukemia and other neoplasms such as breast cancer, certain brain tumors, and ovarian cancer. Stem cell transplantation also plays a role in the treatment of a variety of conditions including hematological disorders, autoimmune diseases, and immunodeficiency conditions. The approaches to transplantation are distinguished by the type of donor involved in providing the stem cell component. This includes autologous (self) and allogeneic, which can be either a family member (related) or unrelated donors procured through the various donor registries. The recognition of the therapeutic value of stem cell transplantation has led to the general acceptance of this procedure by third party providers.

Gene Therapy: This newer, more innovative form of therapy involves the transfer of genetic material into patients for therapeutic purposes. It is an approach that holds promise for the treatment of many human diseases including those with currently ineffective or nonexistent treatment. Recombinant DNA technology has progressed at an extraordinary pace over the past few years and has allowed for the identification and manipulation of genes. This process has allowed for a better understanding of the molecular basis of disease. Thus, gene therapy has become a natural extension of this research. As a potential alternative to drug administration, pathophysiological defects in disease may be corrected by altering the genetic material of somatic cells in one or two ways. In one approach, the applicable genes or DNA (transferred by a vector) are administered directly (in vivo) via blood or tissues. A second approach, a form of cell therapy, involves the infusion of cells into the patient which have been modified by gene therapy in the laboratory (ex vivo). The rationale and strategies for treating particular disease are varied. In addition to the treatment of cancer, gene therapy has many potential applications including single-gene inherited disorders (such as sickle cell anemia, hemophilia, hypercholesterolemia), infectious diseases (e.g. HIV, hepatitis, herpes), and multi-factorial disorders (e.g. diabetes, coronary heart disease).

Cellular Therapy: Cell therapy involves the transfer of autologous or heterologous cells for therapeutic purposes. As an example, tumor-infiltrating lymphocytes and other immune effector cells can be manipulated in vitro, using gene transfer and related techniques, with the aim of increasing their specificity and/or reactivity against tumor cells. Recent advances in tissue culture techniques permits the large scale in vitro expansion of these cells which can then be infused into the patient with therapeutic intent. These strategies have been clinically applied and show significant therapeutic promise for diseases such as cancer. In addition to tumor-infiltrating lymphocytes, Epstein-Barr Virus (EBV) specific lymphocytes can be selected, amplified in vitro, and infused into transplanted patients. This strategy has had a high success rate in preventing and even curing EBV-induced lymphomas in transplanted patients. There are a multitude of similar applications for cellular therapy being developed for clinical trials in the near future.

©2002, Cell And Gene Therapy, Baylor College of Medicine
One Baylor Plaza, N1002, Houston, TX 77030, (713)798-1246
URL: http://www.bcm.edu/genetherapy Email:cagt@bcm.edu
(Modified: September 21st, 2007 - jb)