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.