About

Stephan A. Grupp, MD, PhD, is a Professor of Pediatrics (Oncology) at the University of Pennsylvania and a leading expert in CAR T cell therapy and pediatric transplant/cell therapy. He serves as an Attending Physician in Pediatrics at the Children's Hospital of Philadelphia, where he is also the Medical Director of the Cell and Gene Therapy Laboratory. Grupp's research focuses on the development and preclinical testing of engineered cell therapies and signal transduction inhibitors in leukemia, with extensive experience in pediatric trials utilizing these therapies. He has led most of the CTL019 (CD19 CAR) clinical trials at CHOP and globally, contributing to the field of highly active CAR T cell therapy, culminating in the FDA approval of CTL019/tisagenlecleucel as Kymriah, the first CAR T product and gene therapy approved in the US.

Research topics

• Medicine
• Internal medicine
• Immunology
• Biology
• Oncology
• Genetics
• Cancer research
• Pathology
• Cell biology
• Bioinformatics
• Pediatrics
• Virology
• Intensive care medicine

Selected publications

• CD19 CAR T-cell outcomes in relapsed/refractory extramedullary B-ALL: a multisite, retrospective cohort review

  Blood Advances · 2026-02-17 · 1 citations

  articleOpen access

  ABSTRACT: CD19 chimeric antigen receptor T cells (CD19-CAR) are effective at eradicating bone marrow (BM) B-cell acute lymphoblastic leukemia (B-ALL), but efficacy data for the treatment of extramedullary leukemia are lacking. We conducted a multisite, retrospective review of 308 children and young adults who received CD19-CAR and report efficacy in patients with active central nervous system (CNS) disease (CNS cohort, n = 36), active non-CNS extramedullary disease (EMD; n = 21), or isolated BM disease (iBM; n = 251) at infusion. The overall survival (OS) at 24 months in the iBM, EMD, and CNS cohorts was 71.5% (95% confidence interval [CI], 66.0-77.6), 66.7% (95% CI, 49.3-90.2), and 57.0% (95% CI, 42.6-76.3), respectively (P = .032); the corresponding event-free survival (EFS) was 51.8% (95% CI, 45.8-58.6), 45.8% (95% CI, 28.2-74.4), and 35.2% (95% CI, 22.4-55.3) (P = .035). All patients with isolated EMD (n = 5) achieved complete response and did not relapse. Eight patients (80%) with isolated CNS disease (n = 10) had clearing of the CNS, and 3 (37.5%) subsequently relapsed. Concurrent EMD or CNS disease with high BM disease burden (HD) was associated with inferior outcomes when compared with low BM disease burden (LD) in terms of OS (EMD-HD, 41.7% vs EMD-LD, 100%; P = .005; CNS-HD, 33.3% vs CNS-LD, 92.3%; P = .001) and EFS (EMD-HD, 8.3% vs EMD-LD, 100%; P< .001; CNS-HD, 14.3% vs CNS-LD, 63.6%; P< .001). In summary, CD19-CAR may be an effective option for relapsed/refractory B-ALL with active EMD or CNS disease, but concurrent HD predicts inferior outcomes, prompting consideration of enhanced preinfusion evaluation and treatment.

  PublisherOA PDFDOI

• Next Generation Sequencing Minimal Residual Disease in Pediatric B-ALL after CD19 and/or CD22 CAR T-cell Therapy: Depth and Kinetics of Response Predict Relapse Risk.

  Transplantation and Cellular Therapy · 2026-02-01

  article
  PublisherDOI

• Operational and Data Quality Alerting in a Cell Therapy Laboratory

  Transplantation and Cellular Therapy · 2026-02-01

  article
  PublisherDOI

• Preinfusion risk factors for the development of severe neurotoxicity following CART therapy in pediatric patients

  HemaSphere · 2026-04-01

  articleOpen access

  Neurotoxicity is a common and potentially life-threatening complication after chimeric antigen receptor T-cell therapy (CART) for pediatric B-cell acute lymphoblastic leukemia (B-ALL). The only consistently demonstrated clinical risk factor for severe neurotoxicity in prior studies is high, preinfusion bone marrow disease burden (DB). To better prognosticate this syndrome, we sought to identify preinfusion clinical, laboratory, and imaging risk factors for the development of severe neurotoxicity, stratified by DB. We determined the incidence of severe neurotoxicity (defined as Grade ≥3 neurologic event[s]) in children and young adults treated with investigational anti-CD19 or anti-CD22 CART products across eight clinical trials or with commercial tisagenlecleucel. We comprehensively examined the association of putative clinical, laboratory, and imaging factors with the development of severe neurotoxicity. The occurrence of a seizure was included as a secondary outcome. Severe neurotoxicity was observed in 45/442 (10%) patients and was more frequent in the high DB (26/107, 24%) than the low DB (17/312, 5%) cohort. In the high DB cohort, history of a prior transient neurologic insult was associated with severe neurotoxicity (adjusted odds ratio, 5.73; 95% CI, 1.63, 21.7; P = 0.007). In the low DB cohort, no studied risk factors were robustly associated with severe neurotoxicity. Risk factors associated were different in the high and low DB cohorts. Patients with high DB and a previous history of transient neurologic events had a higher risk of severe neurotoxicity. Although the frequency of severe neurotoxicity was much lower in low DB patients, low DB patients accounted for 40% of severe neurotoxicity cases. Future studies should consider patients with high or low DB separately.

  PublisherDOI

• Improved Hematopoietic Stem Cell Mobilization for Gene Therapy of Hemoglobinopathies Using Single Agent Motixafortide

  Transplantation and Cellular Therapy · 2026-02-01

  article1st author
  PublisherDOI

• Comment on Management Practices of CAR T-cell-Related Inflammatory Toxicities: A Survey of Pediatric CAR T-cell Providers

  Transplantation and Cellular Therapy · 2026-01-30

  article1st authorCorresponding
  PublisherDOI

• High Rates of Clinically Significant Late Infections and Recurrent Sinopulmonary Disease in Long-Term Survivors after CD19 CAR T-cell Therapy for Pediatric B-ALL

  Transplantation and Cellular Therapy · 2026-02-01

  article
  PublisherDOI

• Pharmacokinetically-Adjusted Busulfan Dosing in Adults and Adolescents with Sickle Cell Disease or Transfusion-Dependent β-Thalassemia Treated with Exagamglogene Autotemcel

  Transplantation and Cellular Therapy · 2026-02-01

  article
  PublisherDOI

• Multi-Week Forecasting of Pediatric BMT Census: Deployment and Operationalization

  Transplantation and Cellular Therapy · 2026-02-01

  article
  PublisherDOI

• Durable Clinical Benefits with Exagamglogene Autotemcel for over 5 Years of Follow-up in Transfusion-Dependent Thalassemia and Sickle Cell Disease with Recurrent Vaso-Occlusive Crises

  Transplantation and Cellular Therapy · 2026-02-01

  articleSenior author
  PublisherDOI

Recent grants

• NIH Grant R29AI040111

  NIH · $612k · 2001

• Defining and overcoming intrinsic T cell dysfunction to enable pediatric immunotherapy

  NIH · $3.7M · 2019–2024

• Radiobiology and Imaging Program

  NIH · $93.0M · 1997–2027

• NIH Grant R01CA082156

  NIH · $1.1M · 2005

• NIH Grant R21CA110516

  NIH · $661k · 2008

Frequent coauthors

• David T. Teachey

  Children's Hospital of Philadelphia

  410 shared

• Shannon L. Maude

  University of Pennsylvania

  405 shared

• Carl H. June

  Parker Institute for Cancer Immunotherapy

  336 shared

• David M. Barrett

  Kite (United States)

  313 shared

• Michael A. Pulsipher

  282 shared

• Franco Locatelli

  Bambino Gesù Children's Hospital

  250 shared

• Donna A. Wall

  Children’s Hospital at TriStar Centennial

  239 shared

• Haydar Frangoul

  University College London

  201 shared

Education

• B.S.

  University of Cincinnati

  1981

• Ph.D.

  University of Cincinnati College of Medicine

  1985

• M.D.

  University of Cincinnati College of Medicine

  1987