The Interdisciplinary Center for Human Stem Cell Research and Regenerative Medicine

Stem cells and cell therapy hold enormous potential for both science and medicine. Studying stem cells is expected to improve our understanding of fundamental biological processes and allow human disease modeling in vitro; using these cells may also prove useful for drug discovery and the development process. Ultimately, the biggest hope is that stem cell-based therapies could offer promise for curing serious diseases for which limited or no treatment options are available at present, such as Alzheimer’s and Parkinson’s diseases, paralysis due to spinal cord injury, heart failure, liver disease, and Type I diabetes. By some estimates, if successful cell-based therapies could be a $30 billion market.

Stem cells are unspecialized cells that are capable of self-renewal and, under certain conditions or when given certain cues, can differentiate into tissue/organ-specific cell types. Stem cells can be derived from three major sources: embryos, adult tissues, and umbilical cord blood. Stem cells from different sources vary in their abilities to differentiate or to produce mature cell types. Embryonic stem cells (ESC) can give rise to any cell type in the adult body, as well as to extra-embryonic tissues (placenta and other supporting structures), whereas adult and cord blood-derived stem cells are more restricted in their ability to differentiate into various cell lineages. Understanding the versatility of a given stem cell type is crucial to unlocking its therapeutic potential.

A goal of a cell-based therapy is to restore normal organ function by transplanting cells that can replace those lost and/or can create an environment that will stimulate the organ’s self-repair mechanisms. The latter should also be possible to achieve through stimulation of the adult stem cells that already exist within a damaged organ or tissue by using appropriate growth factors or other agents.

Although the general public is keenly aware of the stem cell promise for curing many diseases, many people do not realize that stem cell therapy is already a reality in clinics today. In fact, hematopoietic stem cell (HSC) transplantation for the treatment blood diseases, such as leukemias, lymphomas, and anemias, has been around for more than 40 years. Originally such transplants were performed using bone marrow, but more recently, they are performed using mobilized stem cells from peripheral blood, as well as cord blood stem cells.