One of the largest pediatric programs of its kind in the world, the St. Jude Bone Marrow Transplantation Program has performed nearly 3,000 transplants since 1982. Patients have included newborns, infants, children, adolescents and young adults who have a malignant or nonmalignant life-threatening disorder. Such disorders must be treatable with a hematopoietic stem cell or bone marrow transplant with either autologous (own) cells or allogeneic (donor) cells. The program is also an active research enterprise with clinical and pre-clinical investigators collaborating across departments to develop novel therapeutic strategies informed by biologic discoveries. New protocols are routinely in development and are often generated directly from pre-clinical work conducted at St. Jude.
Bone Marrow Tranplantation Experts
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Stephen Gottschalk, MD
Gottschalk
Stephen Gottschalk, MD, St. Jude Department of Bone Marrow Transplantation and Cellular Therapy chair, focuses his research on cancer immunotherapy, cell therapy and stem cell transplantation. He leads a team of clinical and scientific researchers conducting clinical studies with highly specific T cells for patients with blood cancers and severe combined immunodeficiency (SCID), as well as brain and other solid tumors. The Gottschalk laboratory focuses on Epstein-Barr virus (EBV)-specific T cells for treating EBV-associated cancers and using genetically modified T cells for cancer immunotherapy. He can also speak on the successful cure, pioneered at St. Jude, of X-linked severe combined immunodeficiency disease (SCID), also known as Bubble Boy Disease.
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Giedre Krenciute, PhD
Krenciute
Giedre Krenciute, PhD, St. Jude Department of Bone Marrow Transplantation and Cellular Therapy is a leading investigator in the field of chimeric antigen receptor (CAR) T cells. Her research focuses on understanding T-cell regulation to improve immunotherapeutic approaches for patient populations. Krenciute’s recent work has focused on developing and characterizing CARs specific to brain tumors, and she has made several key contributions to improving the understanding of CAR T cell function. By re-educating the immune system, this work may help improve tumor detection along with the persistence, efficacy and safety of immune effector cells to optimize immunotherapy for patients.
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Ewelina K. Mamcarz, MD
Mamcarz
Ewelina Mamcarz, MD, St. Jude Department of Bone Marrow Transplantation and Cellular Therapy leads clinical research into the treatment of patients with X-linked severe combined immunodeficiency (X-SCID). Mamcarz was instrumental in the development of the first lentiviral gene therapy to successfully treat these patients. X-SCID, commonly known as Bubble Boy Disease, is a genetic disorder that causes patients to have an inactive immune system, leaving them vulnerable to the simplest infections, resulting in death, often before the patient is two years old. The therapy needed to cure these patients was heralded worldwide and earned the distinction of Smithsonian Magazine’s American Ingenuity Award.
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Akshay Sharma, MBBS
Sharma
Akshay Sharma, MD, St. Jude Department of Bone Marrow Transplantation and Cellular Therapy, coordinates the bone marrow transplantation program for patients with sickle cell disease. Sharma is leading clinical research using gene editing, sometimes referred to as CRISPR therapeutics, for treating sickle cell disease. Gene editing for treating human disease is pushing the boundaries of science in healthcare. Sharma is also interested in the development of novel reduced-intensity and non-myeloablative conditioning regimens to improve sickle cell care.
