The Tissue Engineering & Regenerative Medicine program was established to advance the field of cell-based therapies, and to support research involving pluripotent stem cells to treat human diseases. Stem cells currently have many uses in research and a virtually unlimited potential in the clinical setting: they can be used to study human development and the complex genetic and molecular controls that affect cell differentiation; new drugs can be tested on stem cells; and cells and tissues can be generated for cell-based therapies.
Amyotrophic Lateral Sclerosis (ALS)
Approximately 30,000 people in the United States are afflicted with amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s disease. ALS is an adult-onset neurodegenerative disease that selectively destroys motoneurons, cells in the spinal cord and brain that cause muscle movement. Following diagnosis, the average life expectancy of ALS patients currently ranges from 4 - 6 years. Since ALS patients seek medical attention only after disease onset, therapies directed at slowing disease progression are vitally needed. Drs. Stanley Appel and Ericka Simpson, and colleagues at the Methodist Neurological Institute, are conducting translational research with a long-term goal of developing novel drugs and treatments to significantly slow disease progression, prolong life expectancy, and enhance the quality of life for their ALS patients. An initial trial with 6 ALS patients using unmodified human blood-derived stem cells was not clinically beneficial, however, the stem cells were documented to reach the spinal cord and provided important evidence that such cells could act as a cellular vehicle for future central nervous system gene therapy. Additional cell-based therapies are currently being developed and tested in the Appel laboratory.
Chronic heart failure remains a major public health burden affecting almost 5 million Americans, with an overall annual mortality rate of 10%. Drs. Michael Reardon and Brian Bruckner of the Methodist DeBakey Heart and Vascular Center are conducting a groundbreaking clinical trial using stem cells from a patient’s own bone marrow to treat this often terminal condition. The study uses a specific type of stem cells called cardiac repair cells and injects them directly into the damaged areas of the patient’s beating heart using a minimally invasive surgical procedure.
Myeloma and Lymphoma
Drs. Malcolm Brenner and Helen Heslop of the Center for Cell and Gene Therapy are currently conducting a wide range of studies to develop and test cell-based therapies to treat hematological and Epstein-Barr virus-associated malignancies. Their research aims to improve cancer therapies using adoptive immunotherapy with gene-modified effector cells. The research in the Center for Cell and Gene Therapy has been continuously funded by the National Institutes of Health since 2001. These investigators are also collaborating with neurosurgery and hematology oncology clinicians to bring these approaches to patients with non-small cell lung cancer and brain tumors (glioblastoma multiforme).
Bone marrow transplants are used to treat a variety of diseases, most often hematological malignancies and autoimmune disorders. Hematopoietic stem cells (HSCs) are the critical component of a marrow transplant due to their ability to self-renew and differentiate into all blood cell types. Patients in need of bone marrow transplants continue to far outnumber available compatible bone marrow donors. Dr. Aleksandar Babic is conducting vital translational research at TMHRI to directly address this disparity. His studies, funded by the National Institutes of Health, aim to identify novel methods to amplify the number of HSCs in donated bone marrow transplants and thereby improve engraftment rates and the number of recipients that can benefit from a single bone marrow donation.
The leading causes of blindness in the United States are diabetic retinopathy, glaucoma, and age-related macular degeneration. Tissue engineering stands poised to play a key role in the stabilization and restoration of vision in these and other conditions, including retinal detachment. Dr. William Foster, a vitreoretinal surgeon, develops intraocular materials and nanotechnology-based drug delivery systems to improve surgical and medical treatments for vision loss.
For more information on stem cells and their potential to treat human disease, please visit: http://stemcells.nih.gov/