The Center for Cell and Gene Therapy is designed to provide a cell and gene therapy resource for the whole of Baylor College of Medicine and its affiliated hospitals. The organization of the center reflects the following proposed functions. It will provide support from the earliest level of vector development to preparation of clinical grade materials, and submission of clinical protocols and INDs. Since advances in three areas will be critical for most cell or gene therapy strategies, pre-clinical research within the center will focus on transgene regulation, vector targeting, and stem cell biology. In addition, there is a vector development core which will consist of three investigators and nine additional scientific staff. This core will work with investigators throughout the institution to prepare a range of vectors expressing genes of interest. The core will also be involved in improving the function of currently available vector systems, and will import newly discovered vector systems and develop them for clinical use.

Research Area Working Groups

The center is also forming a series of working groups in specific areas such as ocular gene therapy, neurodegenerative diseases, cardiovascular diseases, solid tumors, leukemias and lymphomas in order to identify suitable targets for a cellular and gene therapy approach. These small groups are composed of six to eight individuals from basic science departments such as genetics, from relevant clinical science departments such as Cardiovascular Medicine and from the Cell and Gene Therapy Center itself. Other relevant individuals may be brought in on an ad hoc basis. The intent of these working parties is to identify candidate genes and diseases that will be amenable to currently available technologies in cell and gene therapy, and to provide a scheme of experimental development to allow pre-clinical observations to evolve into clinical trials. These working parties are also responsible for identifying resource requirements and for the appointment of support staff and/or provision of equipment and reagents for the relevant investigators.

While scientists may be appointed directly to the center with an academic appointment in a relevant pre-clinical or clinical department, we can also offer partial salary support to scientists who are working on gene therapy related projects in other departments. We also support technical staff and post-doctoral fellows working with such investigators outside the center. Hence, the center has considerable flexibility in the way its resources are used for the institution. The working groups overall are coordinated by Dr. Brenner, the director of the Center for Cell and Gene Therapy, who works with his executive committee containing representatives from major pre-clinical and clinical departments.

Center Work Flow

Once an appropriate clinical target has been identified and the pre-clinical studies have been completed, the center contributes to the clinical protocols by providing expertise from its Protocol Development Core in developing clinical protocols that will be acceptable to the institutional and federal regulatory authorities, including the Recombinant DNA Advisory Committee and the Food and Drug Administration. Thus, clinical investigators with no previous experience in gene therapy can be assisted by a team that has successfully submitted over 25 successful INDs in the field.

Finally, when the gene therapy protocols have been approved, the Good Manufacturing Protocols (GMP) core works with the principal investigator to prepare the vectors and cells for infusion. The treatment itself can be carried out in the investigator's own inpatient facility or in the stem cell transplant unit at Texas Children's Hospital or Houston Methodist Hospital, as appropriate. These stem cell transplant units will be particularly valuable for studies which require isolation of the treated individual or in which infusions of large cell numbers with subsequent monitoring are part of the protocol.