Nirali N. Shah, M.D., M.H.Sc.
Dr. Shah is a physcian scientist who serves as the Head of the Hematologic Malignancies Section of the Pediatric Oncology Branch. Her primary research interests focus on translation of immunotherapeutic approaches to treat high-risk hematologic malignancies in children, adolescents and young adults. She has a particular interest in prevention and treatment of relapsed disease after allogeneic hematopoietic stem cell transplantation. Her clinical trials focus on exploring and optimizing chimeric antigen receptor (CAR) T-cell based strategies and other antibody based therapies to target surface proteins found on leukemia cells to improve outcomes for chemotherapy refractory disease. Additional areas of expertise include pediatric hematopoietic stem cell transplantation in both malignant and non-malignant diseases, development of early phase clinical trials in pediatric oncology, and ethical considerations for inclusion of minors in research. Dr. Shah also serves as the NCI Fellowship Program director for the joint Johns Hopkins Hospital-National Cancer Institute Pediatric Hematology-Oncology Fellowship Program.
1) chimeric antigen receptor (CAR)-T cell therapy for pediatric leukemia, 2) immunotherapy for relapsed/refractory pediatric leukemia and lymphoma, 3) early-phase clinical trials in
pediatric oncology, 4) hematopoietic stem cell transplantation, 5) bioethics
Dr. Shah’s research focuses on translation of immunotherapeutic approaches to treat high-risk hematologic malignancies in children, adolescents and young adults. Her clinical work has included implementation of conduct of several Phase I trials for the treatment of relapsed/refractory pediatric acute lymphoblastic leukemia. Prior trials have included a pediatric phase I trial of vincristine sulfate liposomal injections (Marqibo®), a pediatric phase I trial of moxetumomab pasudotox, (an anti-CD22 targeted immunotoxin based therapy), and a pilot trial using WT1 dendritic cell vaccines for the treatment of post-transplant relapsed leukemia. Results from the trials have included establishment of a safety profile, identification of a dose and an understanding of pharmacokinetics leading to development of Phase 2 multicenter studies that she will be leading. More recently she has led the effort in CAR-T cell therapy targeting CD22 for the treatment of relapsed/refractory ALL and will be leading a trial using a combinatorial CD19/CD22 targeted CAR-T cell approach. In addition to her work in leukemia, she also serves to support pediatric transplantation at the NCI by serving as an associate investigator on several transplant trials focused on patients with primary immunodeficiency and is leading the effort on transplantation for children with DOCK8 deficiency in collaboration with investigators in NIAID.
Sequential loss of tumor surface antigens following chimeric antigen receptor T-cell therapies in diffuse large B-cell lymphoma..Haematologica. [Epub ahead of print], 2018. [ Journal Article ]
CD22-targeted CAR T cells induce remission in B-ALL that is naive or resistant to CD19-targeted CAR immunotherapy..Nat Med.. 24: 20-28, 2018. [ Journal Article ]
- Blood. 135(5): 303-4, 2020. [ Journal Article ]
Haploidentical Related Donor Hematopoietic Stem Cell Transplantation for Dedicator-of-Cytokinesis 8 Deficiency Using Post-Transplantation Cyclophosphamide..Biol Blood Marrow Transplant. 6: 980-990, 2017. [ Journal Article ]
- Nat Rev Clin Oncol. 16(6): 372-85, 2019. [ Journal Article ]
Dr. Shah received her M.D. in 2004 from the University of Illinois College of Medicine in Chicago. She subsequently completed a dual residency program in Internal Medicine and Pediatrics at the Harvard Combined Residency program and trained jointly at the Children’s Hospital Boston and Massachusetts General Hospital, during which time she also served as Chief Resident. She then joined the Pediatric Hematology and Oncology Fellowship joint training program between the National Cancer Institute, Pediatric Oncology Branch (POB) and Johns Hopkins University and was an Associate Research Physician in the Pediatric Oncology Branch. In 2019 she was appointed as an NIH Lasker Investigator. Additional training has included obtaining a Masters of Health Science in Clinical Research through the joint NIH-Duke University School of Medicine; and completion of the American Society for Blood and Marrow Transplantation Clinical Research Training Course (2012). She is a member of multiple societies, including the American Society of Hematology, the American Society of Blood and Marrow Transplantation, the Children’s Oncology Group, the Therapeutic Advances in Childhood Leukemia & Lymphoma consortium and the Pediatric Blood and Marrow Transplant Consortium. Dr. Shah is board certified in General Internal Medicine, General Pediatrics and Pediatric Hematology Oncology.