In This Section

Stephan A. Grupp, MD, PhD
Stephan A. Grupp
Director of Translational Research, Center for Childhood Cancer Research

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.



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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

Attending Physician

Professor of Pediatrics

Professional Memberships

Society for Pediatric Research, 2003-

American Pediatric Society, 2010-

The American Society of Pediatric Hematology/Oncology, 2011-2014

Professional Awards

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


Publication Highlights