In This Section
Dr. Grupp develops and conducts preclinical testing of engineered cell therapies and signal transduction inhibitors in leukemia, in pediatric immunotherapy trials, and in the manufacture and use of cellular therapeutics in preclinical, good manufacturing practices, and clinical trial settings. Dr. Grupp leads most CTL019 (CD19 CAR) clinical trials, and his colleagues are the global leaders in highly active CAR T cell therapy.
The primary focus of Dr. Grupp’s research is the development of targeted and cell therapies and study of molecular signaling pathways in acute lymphoblastic leukemia (ALL). His group has leveraged studies using primary human ALL xenografts into treatments tested in a number of clinical trials, including national phase III randomized and FDA registration trials. : In addition, as the director of the Cancer Immunotherapy Frontier Program and translational research for the Center for Childhood Cancer Research, he oversees research into clinical use of CAR T cells and hematopoietic stem cells.
Dr. Grupp and his team have demonstrated the importance of the mTOR pathway in B cell cancer, and demonstrated that mTOR inhibitors are effective agents against ALL as well as lymphoproliferative disorders. Their mTOR pathway findings have had direct translational significance in ALL, leading to phase I, II, and III trials.
These studies have also shown the importance of the graft versus leukemia effect in ALL, and demonstrated the power of next generation sequencing to detect clinically relevant minimal residual disease in the transplant setting.
In addition to ALL, Dr. Grupp has worked with autoimmune lymphoproliferative syndrome (ALPS) in patients and animal models. Leveraging their ALL work, Dr. Grupp and his team have also shown the power of mTOR inhibition in ALPS, and their recently published pilot trial of sirolimus in ALPS supports the first-line use of this drug in ALPS.
As part of his role as medical director of the stem cell lab at Children’s Hospital of Philadelphia and transplant discipline chair for the Children’s Oncology Group (COG), Dr. Grupp and his team have performed trials to improve outcomes in neuroblastoma. His team developed tandem transplant approaches at CHOP, piloted them in the COG and then designed a national phase III trial (ANBL0532) in the COG, the results of which are positive.
Education and Training
BS, University of Cincinnati (Biology), 1981
PhD, University of Cincinnati (Immunology), 1985
MD, University of Cincinnati, 1987
Titles and Academic Titles
Director of Translational Research, Center for Childhood Cancer Research
Director, Cancer Immunotherapy Frontier Program
Chief, Cellular Therapy and Transplant Section
Medical Director, Stem Cell Laboratory
Yetta Deitch Novotny Endowed Chair in Pediatric Oncology
Professor of Pediatrics
Society for Pediatric Research, 2003-
American Pediatric Society, 2010-
The American Society of Pediatric Hematology/Oncology, 2011-2014
Stanford Young Investigator in Molecular Oncology, 2000
Research Recognition Award, Leukemia and Lymphoma Society, 2002
Eagles Fly for Leukemia 2007 Lifetime Achievement Award, 2007
Chai Lifeline Community Service Award, 2013
Audrey Evans Service Award, Ronald McDonald House Charities, 2014
van Bekkum Prize, European Bone Marrow Transplantation Society, 2014
Clinical Research Forum Herbert Pardes First Place Achievement Award, 2014
Clinical Research Forum Top 10 Clinical Research US Achievement Award, 2014
Pennsylvania Bio Patient Impact Award, 2014
Oski Lectureship and Award, American Society of Pediatric Hematology/Oncology, 2015
Fred Saunders Lectureship and Award, Canadian Blood and Marrow Transplant Society, 2015
William Osler Patient Oriented Research Award, University of Pennsylvania, 2018
Election to National Academy of Medicine, 2019