I am the Chair of the Department of Pediatrics and oversee all aspects of the clinical, medical education, and research endeavors the department is privileged to provide. My clinical expertise is in bone marrow transplantation and the treatment of children with congenital and acquired immunological deficiencies. I have particular expertise in treating genetic diseases that impair hematopoiesis — the formation of blood cells — as well as pediatric leukemias. I am also an authority on transplantation immunology and cellular therapies.
I've always been fascinated by the immunology of transplantation and our ability to use this approach to cure diseases. I was involved in the first transplant of bone marrow from an unrelated donor to a patient in 1973. This opened the possibility of a transplant to the majority of patients who lack a matched brother or sister. Since then, we have greatly refined this approach. In fact most of the transplants we perform are now from unrelated donors.
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I also conduct laboratory and clinical research, and have led national studies of new therapies for children with genetic immunodeficiencies and both adults and children with leukemia and other life-threatening blood diseases. I and my colleagues developed and introduced methods for removing T cells from the donor’s transplant. (T cells are the white blood cells that can attack the tissues of the patient and cause a complication called graft-versus-host disease [GvHD]). This approach has proven to be an effective way to prevent GvHD, and has allowed us to transplant marrow from genetically half-matched donors, such as a mother or father, without causing severe or fatal GvHD. We've also developed and introduced in clinical trial, new immune cell therapies to manage other potential complications of transplantation, such as Epstein-Barr-virus lymphomas and cytomegalovirus infections. Today there is no more “boy in the bubble,” thanks to the advances we've made in bone marrow transplantation.
My fellow scientists and I are now developing cellular therapies that could be used to enhance the resistance of bone marrow transplant recipients to not only serious infections but also their underlying cancers. We're aiming to create novel systems to elicit an immune response in the patient against infectious organisms or residual cancer cells.
I am privileged to work with a dedicated and experienced team of professionals who have the capacity to assess clinical challenges and develop innovative solutions. We share a passion for delivering good medicine. We've also developed support systems for patients and their families that are second to none.
I have shared my experience and knowledge with fellows and residents, many of whom have gone on to establish bone marrow transplant programs at institutions around the world. I am past-President and currently a member of the Board of the Damon Runyon-Walter Winchell Cancer Fund, and on the Board of the Children's Oncology Society of New York, which runs the local Ronald McDonald House. I have won several awards for my work, including honors from the American Society of Clinical Oncology, the Society of Translational Oncology, the American Society for Blood and Marrow Transplantation, and Leiden University in The Netherlands.
