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Center for Cell and Gene Therapy

Houston, Texas

Center for Cell and Gene Therapy
Center for Cell and Gene Therapy
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Cell and Gene Therapy Program

Introduction

Improvements in the understanding of the molecular basis of disease have encouraged the development of novel cellular and gene therapy strategies that have the potential to revolutionize the therapy of a wide variety of human diseases including cancer, cardiovascular disease, diabetes, HIV, and a number of other conditions.

Baylor College of Medicine has made the comprehensive cell and gene therapy program, aimed at developing innovative treatments for both adults and children, a high priority. Such a program requires both a strong basic science research effort as well as targeted clinical research programs.

The program was created in January, 1997, through the cooperative effort of Baylor, The Methodist Hospital, and Texas Children’s Hospital. Dr. Malcolm Brenner was recruited to lead the multi-institutional program.

The basic research laboratories of gene therapy are located at Baylor in the Alkek Building.

The 30,000-square-foot, state-of-the-art adult stem cell transplant unit at Methodist, opened in October of 1999. The unit consists of a 15-bed inpatient unit with a contiguous outpatient unit and infusion area for providing seamless care between inpatients and outpatients.

The 16,200-square-foot Pediatric Stem Cell Transplant Unit opened in June of 2001 and has a contiguous unit with nine inpatient beds.

Laboratory space to support the pre-clinical research component, and the Gene Vector Laboratory for the production of clinical grade vectors both reside in the Feigin Center at Texas Children’s. The Feigin Center also contains Cell and Molecular Therapy Laboratories which operate under Good Manufacturing Practice guidelines. Additional space for the GVL and the Translational Research Laboratory is available at the Feigin Center.

These laboratories process patient components for use in clinical trials according to the novel cellular and gene therapy strategies developed through the integrated efforts of the clinical faculty and the basic and pre-clinical research faculty. Creation of the financial and administrative infrastructure necessary to support such a multi-institutional program has been a joint effort between the institutions.

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.

Program Projections

Optimizing the therapeutic potential of cell and gene therapy requires a fundamental understanding of the molecular pathophysiology of disease as well as expertise in gene transduction (transfer) techniques, vector development, stem cell biology, cellular immunology, and stem cell transplantation. Baylor College of Medicine has made a significant long-term commitment to further enhance the basic research areas related to cell and gene therapy, including the identification of genes, gene expression and regulation, target cell identification, development of animal models of disease, and vector development and large scale production.

The development of comprehensive adult and pediatric Cell and Gene Therapy Transplant units at The Methodist Hospital and Texas Children’s Hospital will lead to the expansion of the therapeutic applications for stem cell transplantation, and will facilitate the translation of laboratory breakthroughs into clinical cell and gene therapies. These innovative units will provide the capability for performing all types of stem cell transplantation, cellular therapy, and gene therapy, as well as provide seamless care for the patient by providing inpatient, outpatient, and infusion areas within the same unit. These units will also provide the environment for the implementation of clinical research studies in gene therapy, transplantation involving novel conditioning regimens, and the potential role of transplantation as novel therapy for diseases such as systemic lupus erythematosis, rheumatoid arthritis, and multiple sclerosis. Included in the comprehensive program plan is the Cell and Gene Therapy Protocol office which will facilitate the preparation of protocols for FDA approval and compliance for novel cell and gene therapy studies, as well as provide the resources for data management.

The Cell and Gene Therapy Program provides an exciting opportunity for all three institutions and will be unique to the Texas Medical Center and Houston. This program will attract patients throughout the United States and around the world. With the support of Baylor College of Medicine, The Methodist Hospital, and Texas Children’s Hospital, the program will be instrumental in developing and providing innovative cellular and gene therapies in a cost-effective and affordable manner.

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