The sarcoma team oversees the largest single institution program of clinical research and treatment devoted to sarcomas of childhood, and has pioneered effective combination chemotherapy regimens for the treatment of osteosarcoma and Ewing sarcoma. Current clinical research is focused on development of pharmacologic and immunologic therapies targeting unique molecular characteristics of osteosarcoma and fusion gene-induced features of Ewing sarcoma, rhabdomyosarcoma, and desmoplastic small round cell tumors.
The neuroblastoma team, which directs the care of over 100 new patients with neuroblastoma annually, has pioneered dose-intensive chemotherapy combined with tumor-seeking monoclonal antibody conjugates in the treatment of neuroblastoma; introduced revised staging criteria for evaluation of disease in infants; and developed novel strategies for detection of minimal residual disease. Current clinical research includes studies of genetically engineered immunotoxins, radio-conjugates, idiotype vaccines, and novel biologicals to enhance tumor-targeted immunotherapies; and exploration of intrathecally administered radio-conjugates for the eradication of leptomeningeal metastases of neuroectodermal and selected epithelial tumors.
This team pioneered the development of effective approaches for patients with high-risk forms of acute lymphoblastic leukemia, and has recently introduced promising new agents, such as clofarabine, in the treatment of childhood leukemias. The Developmental Therapeutics Program within the group is also conducting phase I and II trials of a series of new agents in the treatment of leukemias and lymphomas, as well as new agents to treat selected solid tumors resistant to existing combinations of chemotherapeutic agents.
This team has developed and explored dose-intensive chemotherapy combined with autologous marrow transplants in the treatment of brain tumors and widely metastatic retinoblastoma, and introduced radiation-sparing regimens for the treatment of central nervous system (CNS) germ cell tumors. Recently, the team introduced the adjuvant use of intrathecally administered tumor-reactive monoclonal antibodies conjugated to radioisotopes to treat and prevent leptomeningeal metastases of medulloblastoma. Analyses of genomic and proteomic signatures of gliomas and medulloblastomas are also being conducted to identify distinctive factors of these tumors that could be targeted by drugs, biologicals or targeted immunotherapies.
Hematopoietic Cell Transplantation
The hematopoietic cell transplantation team pioneered the use of matched unrelated donor stem cell transplants; first introduced the use of T cell depletion for leukemia and genetic diseases of immunity to prevent graft-versus-host disease (GvHD) following Human Leukocyte Antigen (HLA)-matched and haplotype-disparate transplants; delineated the effector cells and allo-antigenic targets contributing to graft rejection; developed effective strategies to circumvent graft rejection; pioneered effective techniques for treating life-threatening Epstein-Barr virus (EBV) and cytomegalovirus (CMV) infections; and developed strategies to achieve a graft versus leukemia response without GvHD. Current clinical research is focused on the genetic determinants of innate resistance to leukemia; the exploration of adoptive immunotherapies for the treatment of viral diseases, as well as for the treatment and prevention of leukemic relapse; and the evaluation of new biologicals and cytokines to potentiate immune reconstitution.
This multidisciplinary program of clinical care and research oversees the follow-up of over 1500 children and adults who are long-term survivors of childhood cancer. This program is a major component of the NIH-sponsored Childhood Cancer Survivor Study. Major objectives of the clinical research program are the identification, characterization, and treatment of adverse medical sequelae of childhood cancer and its treatment; the development of improved models of care for earlier detection and prevention of these sequelae; and the elimination of barriers to the effective monitoring and long-term evaluation and care of these patients particularly as they transition from adolescence to adulthood.
Pediatric Oncologic Surgery Service
- David H. Abramson, MD
Chief, Ophthalmologic Oncology
Pediatric Medical Subspecialties
- Charles A. Sklar, MD (Endocrinology)
- Julia Kearney, MD (Psychiatry)
- Suzanne L. Wolden, MD (Radiation Oncology)
- Bruce M. Greenwald, MD (Pediatric Intensive Care)
- Steven Pon, MD (Pediatric Intensive Care)
- Joy D. Howell, MD (Pediatric Intensive Care)
- Chani Traube, MD (Director, Pediatric Observation Unit)
- James Killinger, MD (Medical Director, Pediatric Intensive Care Unit)
- Michael F. Walsh, MD (Geneticist & Pediatric Oncologist)
- Rachna K. Sheth, MD
- Manjusha Namuduri, MD
- Daniel Ortiz, MD