Disease Pathogenesis Program

Lauren Pachman, MD, Interim Program Director

By far the greatest growth in external funding to CMIER has been in the area of translational research, which involves the study of the role that basic mechanisms play in a clinical disease. At Children’s Memorial Hospital, clinician-scientists are studying a wide-range of pediatric diseases and conditions including studies of the mechanisms of: (i) immune recognition and regulation; (ii) injury to the gastrointestinal tract in the newborn period; (iii) single-cell transplantation approaches to liver diseases; (iv) angiogenesis and the regulation of vascular tone and tumor cell biology; and (v) mechanisms of drug resistance. While the clinical focus of these investigators may vary, the intersecting studies in the mechanisms that alter disease susceptibility and response were brought together formally in 2002 by the creation of the Disease Pathogenesis Program to foster the sharing of ideas among scientific groups.

With a gift from The Dr. Ralph and Marian Falk Medical Research Trust, CMIER recently established the cross-disciplinary Stem Cell and Immunobiology Initiative within the Disease Pathogenesis program. Stem cell research offers unprecedented opportunities to develop novel therapies and treatments for diseases for which there are few or no known cures. Immunobiology is another growing area where new insights have already led to breakthroughs in the treatment of infectious diseases and autoimmune diseases such as arthritis and diabetes. The arrival this year of William Tse, MD, PhD, signifies a major step forward for this initiative. Dr. Tse’s research focuses on the developmental potential of bone marrow stem cells for tissue regeneration and clinical transplantation for the treatment of hematopoietic disorders and other diseases.