Stephan A. Grupp Cell and Gene Therapy Laboratory, Department of Pathology and Laboratory Medicine Children’s Hospital of Philadelphia Biography Publications Institution JoVE Articles Stephan A. Grupp has not added a biography. If you are Stephan A. Grupp and would like to personalize this page please email our Author Liaison for assistance. Publications In Vivo Hematopoietic Stem Cell Modification by MRNA Delivery Science (New York, N.Y.). Jul, 2023 | Pubmed ID: 37499029 INSPIRED Symposium Part 1: Clinical Variables Associated with Improved Outcomes for Children and Young Adults Treated with Chimeric Antigen Receptor T Cells for B Cell Acute Lymphoblastic Leukemia Transplantation and Cellular Therapy. Jul, 2023 | Pubmed ID: 37481241 Defibrotide Prophylaxis for Prevention of Sinusoidal Obstruction Syndrome - Authors' Reply The Lancet. Haematology. Jul, 2023 | Pubmed ID: 37407140 Incidence, Clinical Presentation, Risk Factors, Outcomes, and Biomarkers in De Novo Late Acute GVHD Blood Advances. Aug, 2023 | Pubmed ID: 37315175 Phase 2 Study of Natalizumab Plus Standard Corticosteroid Treatment for High-risk Acute Graft-versus-host Disease Blood Advances. May, 2023 | Pubmed ID: 37235690 Prevention of Veno-occlusive Disease/sinusoidal Obstruction Syndrome: a Never-ending Story and No Easy Answer Bone Marrow Transplantation. Aug, 2023 | Pubmed ID: 37231094 Impact of Diagnostic and End-of-induction Curie Scores with Tandem High-dose Chemotherapy and Autologous Transplants for Metastatic High-risk Neuroblastoma: A Report from the Children's Oncology Group Pediatric Blood & Cancer. Aug, 2023 | Pubmed ID: 37199022 The Utility of Biomarkers in Acute GVHD Prognostication Blood Advances. May, 2023 | Pubmed ID: 37142257 Defibrotide Plus Best Standard of Care Compared with Best Standard of Care Alone for the Prevention of Sinusoidal Obstruction Syndrome (HARMONY): a Randomised, Multicentre, Phase 3 Trial The Lancet. Haematology. May, 2023 | Pubmed ID: 37001534 Reply to W.H. Tong Et Al Journal of Clinical Oncology : Official Journal of the American Society of Clinical Oncology. May, 2023 | Pubmed ID: 36898064 Automation of Hematopoietic Cell Transplantation Outcomes Reporting Leads to Dramatic Reduction in Errors Reported to Real-World Data Registry Transplantation and Cellular Therapy. Mar, 2023 | Pubmed ID: 36610491 Three-Year Update of Tisagenlecleucel in Pediatric and Young Adult Patients With Relapsed/Refractory Acute Lymphoblastic Leukemia in the ELIANA Trial Journal of Clinical Oncology : Official Journal of the American Society of Clinical Oncology. Mar, 2023 | Pubmed ID: 36399695 Impact of Poverty and Neighborhood Opportunity on Outcomes for Children Treated with CD19-directed CAR T-cell Therapy Blood. Feb, 2023 | Pubmed ID: 36351239 Characterization of Leukemic Resistance to CD19-Targeted CAR T-cell Therapy Through Deep Genomic Sequencing Cancer Immunology Research. Jan, 2023 | Pubmed ID: 36255409 A Novel Antibody-TCR (AbTCR) T-cell Therapy is Safe and Effective Against CD19-positive Relapsed/refractory B-cell Lymphoma Journal of Cancer Research and Clinical Oncology. Jul, 2023 | Pubmed ID: 35776199 Preinfusion Factors Impacting Relapse Immunophenotype Following CD19 CAR T Cells Blood Advances. Feb, 2023 | Pubmed ID: 35482927 Biomarker Correlates with Response to NY-ESO-1 TCR T Cells in Patients with Synovial Sarcoma Nature Communications. Sep, 2022 | Pubmed ID: 36075914 Correction for Bagashev Et Al., "CD19 Alterations Emerging After CD19-Directed Immunotherapy Cause Retention of the Misfolded Protein in the Endoplasmic Reticulum" Molecular and Cellular Biology. Sep, 2022 | Pubmed ID: 35976030 Statistical Considerations for Analyses of Time-To-Event Endpoints in Oncology Clinical Trials: Illustrations with CAR-T Immunotherapy Studies Clinical Cancer Research : an Official Journal of the American Association for Cancer Research. Sep, 2022 | Pubmed ID: 35838646 Low Rate of Subsequent Malignant Neoplasms After CD19 CAR T-cell Therapy Blood Advances. Sep, 2022 | Pubmed ID: 35834728 Comprehensive Serum Proteome Profiling of Cytokine Release Syndrome and Immune Effector Cell-Associated Neurotoxicity Syndrome Patients with B-Cell ALL Receiving CAR T19 Clinical Cancer Research : an Official Journal of the American Association for Cancer Research. Sep, 2022 | Pubmed ID: 35705524 Single-cell Antigen-specific Landscape of CAR T Infusion Product Identifies Determinants of CD19-positive Relapse in Patients with ALL Science Advances. Jun, 2022 | Pubmed ID: 35675405 Cytosine Base Editing Enables Quadruple-edited Allogeneic CART Cells for T-ALL Blood. Aug, 2022 | Pubmed ID: 35560156 Tisagenlecleucel in Pediatric and Young Adult Patients with Down Syndrome-associated Relapsed/refractory Acute Lymphoblastic Leukemia Leukemia. Jun, 2022 | Pubmed ID: 35422096 Next-Generation Sequencing of Minimal Residual Disease for Predicting Relapse After Tisagenlecleucel in Children and Young Adults with Acute Lymphoblastic Leukemia Blood Cancer Discovery. Jan, 2022 | Pubmed ID: 35019853 Potential Role of IFNγ Inhibition in Refractory Cytokine Release Syndrome Associated with CAR T-cell Therapy Blood Cancer Discovery. Mar, 2022 | Pubmed ID: 35015687 Unrelated Donor α/β T Cell- and B Cell-depleted HSCT for the Treatment of Pediatric Acute Leukemia Blood Advances. Feb, 2022 | Pubmed ID: 34872106 Impact of High-risk Cytogenetics on Outcomes for Children and Young Adults Receiving CD19-directed CAR T-cell Therapy Blood. Apr, 2022 | Pubmed ID: 34871373 Blinatumomab Nonresponse and High-Disease Burden Are Associated With Inferior Outcomes After CD19-CAR for B-ALL Journal of Clinical Oncology : Official Journal of the American Society of Clinical Oncology. Mar, 2022 | Pubmed ID: 34767461 CD19-targeted Chimeric Antigen Receptor T-cell Therapy for CNS Relapsed or Refractory Acute Lymphocytic Leukaemia: a Post-hoc Analysis of Pooled Data from Five Clinical Trials The Lancet. Haematology. Oct, 2021 | Pubmed ID: 34560014 Distinct Bioenergetic Features of Human Invariant Natural Killer T Cells Enable Retained Functions in Nutrient-Deprived States Frontiers in Immunology. 2021 | Pubmed ID: 34434191 Tisagenlecleucel Immunogenicity in Relapsed/refractory Acute Lymphoblastic Leukemia and Diffuse Large B-cell Lymphoma Blood Advances. Dec, 2021 | Pubmed ID: 34432863 Pooled Safety Analysis of Tisagenlecleucel in Children and Young Adults with B Cell Acute Lymphoblastic Leukemia Journal for Immunotherapy of Cancer. Aug, 2021 | Pubmed ID: 34353848 Humanized CD19-Targeted Chimeric Antigen Receptor (CAR) T Cells in CAR-Naive and CAR-Exposed Children and Young Adults With Relapsed or Refractory Acute Lymphoblastic Leukemia Journal of Clinical Oncology : Official Journal of the American Society of Clinical Oncology. Sep, 2021 | Pubmed ID: 34156874 CAR-T Cells: Early Successes in Blood Cancer and Challenges in Solid Tumors Acta Pharmaceutica Sinica. B. May, 2021 | Pubmed ID: 34094824 Single-cell Multiomics Dissection of Basal and Antigen-specific Activation States of CD19-targeted CAR T Cells Journal for Immunotherapy of Cancer. May, 2021 | Pubmed ID: 34006631 Antigen-independent Activation Enhances the Efficacy of 4-1BB-costimulated CD22 CAR T Cells Nature Medicine. 05, 2021 | Pubmed ID: 33888899 Absolute Lymphocyte Count Proliferation Kinetics After CAR T-cell Infusion Impact Response and Relapse Blood Advances. Apr, 2021 | Pubmed ID: 33881465 Immune Reconstitution Following TCRαβ/CD19-Depleted Hematopoietic Cell Transplantation for Hematologic Malignancy in Pediatric Patients Transplantation and Cellular Therapy. Feb, 2021 | Pubmed ID: 33830028 Integrative Bulk and Single-Cell Profiling of Premanufacture T-cell Populations Reveals Factors Mediating Long-Term Persistence of CAR T-cell Therapy Cancer Discovery. Sep, 2021 | Pubmed ID: 33820778 Improving and Maintaining Responses in Pediatric B-Cell Acute Lymphoblastic Leukemia Chimeric Antigen Receptor-T Cell Therapy Cancer Journal (Sudbury, Mass.). Mar-Apr 01, 2021 | Pubmed ID: 33750075 Will Allogeneic CAR T Cells for CD19 Malignancies Take Autologous CAR T Cells 'off the Shelf'? Nature Reviews. Clinical Oncology. Apr, 2021 | Pubmed ID: 33608691 Practical Guidelines for Monitoring and Management of Coagulopathy Following Tisagenlecleucel CAR T-cell Therapy Blood Advances. Jan, 2021 | Pubmed ID: 33496754 Beyond the Storm - Subacute Toxicities and Late Effects in Children Receiving CAR T Cells Nature Reviews. Clinical Oncology. Jun, 2021 | Pubmed ID: 33495553 Risk-Adapted Preemptive Tocilizumab to Prevent Severe Cytokine Release Syndrome After CTL019 for Pediatric B-Cell Acute Lymphoblastic Leukemia: A Prospective Clinical Trial Journal of Clinical Oncology : Official Journal of the American Society of Clinical Oncology. Mar, 2021 | Pubmed ID: 33417474 Analysis of Time to Complete Response After Defibrotide Initiation in Patients with Hepatic Veno-Occlusive Disease/Sinusoidal Obstruction Syndrome After Hematopoietic Cell Transplantation Transplantation and Cellular Therapy. Jan, 2021 | Pubmed ID: 32950693 Society for Immunotherapy of Cancer (SITC) Clinical Practice Guideline on Immune Effector Cell-related Adverse Events Journal for Immunotherapy of Cancer. Dec, 2020 | Pubmed ID: 33335028 Diagnostic Biomarkers to Differentiate Sepsis from Cytokine Release Syndrome in Critically Ill Children Blood Advances. 10, 2020 | Pubmed ID: 33095872 Multisystem Inflammatory Syndrome in Children and COVID-19 Are Distinct Presentations of SARS-CoV-2 The Journal of Clinical Investigation. Nov, 2020 | Pubmed ID: 32730233 Thoracic Duct Lymphatic Fluid Harbors Phenotypically Naive T Cells for Use in Adoptive T-cell Therapy Cytotherapy. Oct, 2020 | Pubmed ID: 32622753 Prospective Evaluation of Radiation Dose Escalation in Patients With High-Risk Neuroblastoma and Gross Residual Disease After Surgery: A Report From the Children's Oncology Group ANBL0532 Study Journal of Clinical Oncology : Official Journal of the American Society of Clinical Oncology. Aug, 2020 | Pubmed ID: 32530765 Chimeric Antigen Receptor T Cell Therapy During the COVID-19 Pandemic Biology of Blood and Marrow Transplantation : Journal of the American Society for Blood and Marrow Transplantation. Jul, 2020 | Pubmed ID: 32298807 Dissecting the Tumor-Immune Landscape in Chimeric Antigen Receptor T-cell Therapy: Key Challenges and Opportunities for a Systems Immunology Approach Clinical Cancer Research : an Official Journal of the American Association for Cancer Research. Jul, 2020 | Pubmed ID: 32127393 Impaired Death Receptor Signaling in Leukemia Causes Antigen-Independent Resistance by Inducing CAR T-cell Dysfunction Cancer Discovery. 04, 2020 | Pubmed ID: 32001516 CD19-targeting CAR T Cell Immunotherapy Outcomes Correlate with Genomic Modification by Vector Integration The Journal of Clinical Investigation. 02, 2020 | Pubmed ID: 31845905 Subcutaneous Immunoglobulin Replacement Following CD19-specific Chimeric Antigen Receptor T-cell Therapy for B-cell Acute Lymphoblastic Leukemia in Pediatric Patients Pediatric Blood & Cancer. Mar, 2020 | Pubmed ID: 31793170 Partially CD3-Depleted Unrelated and Haploidentical Donor Peripheral Stem Cell Transplantation Has Favorable Graft-versus-Host Disease and Survival Rates in Pediatric Hematologic Malignancy Biology of Blood and Marrow Transplantation : Journal of the American Society for Blood and Marrow Transplantation. Mar, 2020 | Pubmed ID: 31765697 Efficacy and Safety of Tisagenlecleucel in Japanese Pediatric and Young Adult Patients with Relapsed/refractory B Cell Acute Lymphoblastic Leukemia International Journal of Hematology. Feb, 2020 | Pubmed ID: 31709501 Retention of CD19 Intron 2 Contributes to CART-19 Resistance in Leukemias with Subclonal Frameshift Mutations in CD19 Leukemia. Apr, 2020 | Pubmed ID: 31591467 The MAGIC Algorithm Probability is a Validated Response Biomarker of Treatment of Acute Graft-versus-host Disease Blood Advances. Dec, 2019 | Pubmed ID: 31816061 CAR T-cell Therapy is Effective for CD19-dim B-lymphoblastic Leukemia but is Impacted by Prior Blinatumomab Therapy Blood Advances. Nov, 2019 | Pubmed ID: 31738832 More Precisely Defining Risk Peri-HCT in Pediatric ALL: Pre- Vs Post-MRD Measures, Serial Positivity, and Risk Modeling Blood Advances. Nov, 2019 | Pubmed ID: 31714961 Systemic and Local Immunity Following Adoptive Transfer of NY-ESO-1 SPEAR T Cells in Synovial Sarcoma Journal for Immunotherapy of Cancer. Oct, 2019 | Pubmed ID: 31651363 Patient-reported Quality of Life After Tisagenlecleucel Infusion in Children and Young Adults with Relapsed or Refractory B-cell Acute Lymphoblastic Leukaemia: a Global, Single-arm, Phase 2 Trial The Lancet. Oncology. Dec, 2019 | Pubmed ID: 31606419 CAR T Cell Viability Release Testing and Clinical Outcomes: is There a Lower Limit? Blood. 11, 2019 | Pubmed ID: 31554634 Effect of Tandem Autologous Stem Cell Transplant Vs Single Transplant on Event-Free Survival in Patients With High-Risk Neuroblastoma: A Randomized Clinical Trial JAMA. Aug, 2019 | Pubmed ID: 31454045 Clinical Utilization of Chimeric Antigen Receptor T-cells (CAR-T) in B-cell Acute Lymphoblastic Leukemia (ALL)-an Expert Opinion from the European Society for Blood and Marrow Transplantation (EBMT) and the American Society for Blood and Marrow Transplantation (ASBMT) Bone Marrow Transplantation. Nov, 2019 | Pubmed ID: 31092900 Reply Biology of Blood and Marrow Transplantation : Journal of the American Society for Blood and Marrow Transplantation. Jun, 2019 | Pubmed ID: 30910604 Tisagenlecleucel Model-Based Cellular Kinetic Analysis of Chimeric Antigen Receptor-T Cells CPT: Pharmacometrics & Systems Pharmacology. 05, 2019 | Pubmed ID: 30848084 Higher Reported Lung Dose Received During Total Body Irradiation for Allogeneic Hematopoietic Stem Cell Transplantation in Children With Acute Lymphoblastic Leukemia Is Associated With Inferior Survival: A Report from the Children's Oncology Group International Journal of Radiation Oncology, Biology, Physics. Jul, 2019 | Pubmed ID: 30807822 Naïve T-cell Deficits at Diagnosis and After Chemotherapy Impair Cell Therapy Potential in Pediatric Cancers Cancer Discovery. Apr, 2019 | Pubmed ID: 30630850 ASTCT Consensus Grading for Cytokine Release Syndrome and Neurologic Toxicity Associated with Immune Effector Cells Biology of Blood and Marrow Transplantation : Journal of the American Society for Blood and Marrow Transplantation. Apr, 2019 | Pubmed ID: 30592986 Clinical Utilization of Chimeric Antigen Receptor T Cells in B Cell Acute Lymphoblastic Leukemia: An Expert Opinion from the European Society for Blood and Marrow Transplantation and the American Society for Blood and Marrow Transplantation Biology of Blood and Marrow Transplantation : Journal of the American Society for Blood and Marrow Transplantation. Mar, 2019 | Pubmed ID: 30576834 CD3/CD19 Depleted Matched and Mismatched Unrelated Donor Hematopoietic Stem Cell Transplant with Targeted T Cell Addback Is Associated with Excellent Outcomes in Pediatric Patients with Nonmalignant Hematologic Disorders Biology of Blood and Marrow Transplantation : Journal of the American Society for Blood and Marrow Transplantation. Mar, 2019 | Pubmed ID: 30312755 Purification of MRNA Encoding Chimeric Antigen Receptor Is Critical for Generation of a Robust T-Cell Response Human Gene Therapy. Feb, 2019 | Pubmed ID: 30024272 Chimeric Antigen Receptor (CAR) T Therapies for the Treatment of Hematologic Malignancies: Clinical Perspective and Significance Journal for Immunotherapy of Cancer. Dec, 2018 | Pubmed ID: 30514386 A Novel Antibody-TCR (AbTCR) Platform Combines Fab-based Antigen Recognition with Gamma/delta-TCR Signaling to Facilitate T-cell Cytotoxicity with Low Cytokine Release Cell Discovery. 2018 | Pubmed ID: 30479831 Genetic Mechanisms of Target Antigen Loss in CAR19 Therapy of Acute Lymphoblastic Leukemia Nature Medicine. Oct, 2018 | Pubmed ID: 30275569 Induction of Resistance to Chimeric Antigen Receptor T Cell Therapy by Transduction of a Single Leukemic B Cell Nature Medicine. 10, 2018 | Pubmed ID: 30275568 Clinical Pharmacology of Tisagenlecleucel in B-cell Acute Lymphoblastic Leukemia Clinical Cancer Research : an Official Journal of the American Association for Cancer Research. 12, 2018 | Pubmed ID: 30190371 Neurotoxicity After CTL019 in a Pediatric and Young Adult Cohort Annals of Neurology. Oct, 2018 | Pubmed ID: 30178481 Tisagenlecleucel for the Treatment of B-cell Acute Lymphoblastic Leukemia Expert Review of Anticancer Therapy. Oct, 2018 | Pubmed ID: 30111196 CD19 Alterations Emerging After CD19-Directed Immunotherapy Cause Retention of the Misfolded Protein in the Endoplasmic Reticulum Molecular and Cellular Biology. Nov, 2018 | Pubmed ID: 30104252 Reducing Culture Improves the Antileukemic Activity of Chimeric Antigen Receptor (CAR) T Cells Cancer Immunology Research. 09, 2018 | Pubmed ID: 30030295 CAR-T in the Clinic: Drive with Care Gene Therapy. Jun, 2018 | Pubmed ID: 29961080 Nonviral RNA Chimeric Antigen Receptor-modified T Cells in Patients with Hodgkin Lymphoma Blood. 09, 2018 | Pubmed ID: 29925499 Defibrotide for the Treatment of Hepatic Veno-occlusive Disease/sinusoidal Obstruction Syndrome Following Nontransplant-associated Chemotherapy: Final Results from a Post Hoc Analysis of Data from an Expanded-access Program Pediatric Blood & Cancer. Oct, 2018 | Pubmed ID: 29873895 False-positive Results with Select HIV-1 NAT Methods Following Lentivirus-based Tisagenlecleucel Therapy Blood. Jun, 2018 | Pubmed ID: 29669777 Disease Burden and Conditioning Regimens in ASCT1221, a Randomized Phase II Trial in Children with Juvenile Myelomonocytic Leukemia: A Children's Oncology Group Study Pediatric Blood & Cancer. Jul, 2018 | Pubmed ID: 29528181 Grading of Cytokine Release Syndrome Associated with the CAR T Cell Therapy Tisagenlecleucel Journal of Hematology & Oncology. Mar, 2018 | Pubmed ID: 29499750 Toxicity Management After Chimeric Antigen Receptor T Cell Therapy: One Size Does Not Fit 'ALL' Nature Reviews. Clinical Oncology. Apr, 2018 | Pubmed ID: 29434335 Tisagenlecleucel in Children and Young Adults with B-Cell Lymphoblastic Leukemia The New England Journal of Medicine. 02, 2018 | Pubmed ID: 29385370 Preclinical Efficacy of Daratumumab in T-cell Acute Lymphoblastic Leukemia Blood. Mar, 2018 | Pubmed ID: 29305553 High-Affinity GD2-Specific CAR T Cells Induce Fatal Encephalitis in a Preclinical Neuroblastoma Model Cancer Immunology Research. 01, 2018 | Pubmed ID: 29180536 Single Agent and Synergistic Combinatorial Efficacy of First-in-class Small Molecule Imipridone ONC201 in Hematological Malignancies Cell Cycle (Georgetown, Tex.). 2018 | Pubmed ID: 29157092 Defibrotide for the Treatment of Hepatic Veno-occlusive Disease/sinusoidal Obstruction Syndrome with Multiorgan Failure International Journal of Hematologic Oncology. Nov, 2017 | Pubmed ID: 30302228 Cellular Kinetics of CTL019 in Relapsed/refractory B-cell Acute Lymphoblastic Leukemia and Chronic Lymphocytic Leukemia Blood. 11, 2017 | Pubmed ID: 28935694 Monocyte Lineage-derived IL-6 Does Not Affect Chimeric Antigen Receptor T-cell Function Cytotherapy. 07, 2017 | Pubmed ID: 28506444 Earlier Defibrotide Initiation Post-diagnosis of Veno-occlusive Disease/sinusoidal Obstruction Syndrome Improves Day +100 Survival Following Haematopoietic Stem Cell Transplantation British Journal of Haematology. Jul, 2017 | Pubmed ID: 28444784 IFN-γ Directly Inhibits Murine B-cell Precursor Leukemia-initiating Cell Proliferation Early in Life European Journal of Immunology. 05, 2017 | Pubmed ID: 28295300 Defibrotide for Patients with Hepatic Veno-Occlusive Disease/Sinusoidal Obstruction Syndrome: Interim Results from a Treatment IND Study Biology of Blood and Marrow Transplantation : Journal of the American Society for Blood and Marrow Transplantation. Jun, 2017 | Pubmed ID: 28285079 Refining Megatherapy, Improving Outcome in Neuroblastoma The Lancet. Oncology. Apr, 2017 | Pubmed ID: 28259607 Optimized Depletion of Chimeric Antigen Receptor T Cells in Murine Xenograft Models of Human Acute Myeloid Leukemia Blood. 04, 2017 | Pubmed ID: 28246194 Repeated Loss of Target Surface Antigen After Immunotherapy in Primary Mediastinal Large B Cell Lymphoma American Journal of Hematology. Jan, 2017 | Pubmed ID: 27779774 Potent Efficacy of Combined PI3K/mTOR and JAK or ABL Inhibition in Murine Xenograft Models of Ph-like Acute Lymphoblastic Leukemia Blood. Jan, 2017 | Pubmed ID: 27777238 Cytokine Release Syndrome After Chimeric Antigen Receptor T Cell Therapy for Acute Lymphoblastic Leukemia Critical Care Medicine. Feb, 2017 | Pubmed ID: 27632680 Corrigendum to "Advances in T-cell Therapy for ALL" [Best Pract Res Clin Haematol 27 (2014) 222-228] Best Practice & Research. Clinical Haematology. Mar, 2016 | Pubmed ID: 27742067 Dual CD19 and CD123 Targeting Prevents Antigen-loss Relapses After CD19-directed Immunotherapies The Journal of Clinical Investigation. Aug, 2016 | Pubmed ID: 27571406 Cytokine Release Syndrome After Haploidentical Stem Cell Transplantation Biology of Blood and Marrow Transplantation : Journal of the American Society for Blood and Marrow Transplantation. Oct, 2016 | Pubmed ID: 27543158 Inhibition of Precursor B-cell Malignancy Progression by Toll-like Receptor Ligand-induced Immune Responses Leukemia. Oct, 2016 | Pubmed ID: 27220664 CAR T Cell Therapy in Acute Lymphoblastic Leukemia and Potential for Chronic Lymphocytic Leukemia Current Treatment Options in Oncology. Jun, 2016 | Pubmed ID: 27098534 Identification of Predictive Biomarkers for Cytokine Release Syndrome After Chimeric Antigen Receptor T-cell Therapy for Acute Lymphoblastic Leukemia Cancer Discovery. 06, 2016 | Pubmed ID: 27076371 T Cells Targeting NY-ESO-1 Demonstrate Efficacy Against Disseminated Neuroblastoma Oncoimmunology. 2016 | Pubmed ID: 26942053 Phase 3 Trial of Defibrotide for the Treatment of Severe Veno-occlusive Disease and Multi-organ Failure Blood. Mar, 2016 | Pubmed ID: 26825712 Early Memory Phenotypes Drive T Cell Proliferation in Patients with Pediatric Malignancies Science Translational Medicine. Jan, 2016 | Pubmed ID: 26738796 Sirolimus is Effective in Relapsed/refractory Autoimmune Cytopenias: Results of a Prospective Multi-institutional Trial Blood. Jan, 2016 | Pubmed ID: 26504182 Convergence of Acquired Mutations and Alternative Splicing of CD19 Enables Resistance to CART-19 Immunotherapy Cancer Discovery. Dec, 2015 | Pubmed ID: 26516065 Chimeric Antigen Receptor T Cells Persist and Induce Sustained Remissions in Relapsed Refractory Chronic Lymphocytic Leukemia Science Translational Medicine. Sep, 2015 | Pubmed ID: 26333935 Chimeric Antigen Receptor- and TCR-Modified T Cells Enter Main Street and Wall Street Journal of Immunology (Baltimore, Md. : 1950). Aug, 2015 | Pubmed ID: 26188068 Eradication of B-ALL Using Chimeric Antigen Receptor-expressing T Cells Targeting the TSLPR Oncoprotein Blood. Jul, 2015 | Pubmed ID: 26041741 CD19-targeted Chimeric Antigen Receptor T-cell Therapy for Acute Lymphoblastic Leukemia Blood. Jun, 2015 | Pubmed ID: 25999455 Risk Factors and Timing of Relapse After Allogeneic Transplantation in Pediatric ALL: for Whom and when Should Interventions Be Tested? Bone Marrow Transplantation. Sep, 2015 | Pubmed ID: 25961775 IgH-V(D)J NGS-MRD Measurement Pre- and Early Post-allotransplant Defines Very Low- and Very High-risk ALL Patients Blood. May, 2015 | Pubmed ID: 25862561 Efficacy of JAK/STAT Pathway Inhibition in Murine Xenograft Models of Early T-cell Precursor (ETP) Acute Lymphoblastic Leukemia Blood. Mar, 2015 | Pubmed ID: 25645356 TNF-receptor Inhibitor Therapy for the Treatment of Children with Idiopathic Pneumonia Syndrome. A Joint Pediatric Blood and Marrow Transplant Consortium and Children's Oncology Group Study (ASCT0521) Biology of Blood and Marrow Transplantation : Journal of the American Society for Blood and Marrow Transplantation. Jan, 2015 | Pubmed ID: 25270958 Will Post-transplantation Cell Therapies for Pediatric Patients Become Standard of Care? Biology of Blood and Marrow Transplantation : Journal of the American Society for Blood and Marrow Transplantation. Mar, 2015 | Pubmed ID: 25064748 Roadblocks to Success for RNA CARs in Solid Tumors Oncoimmunology. Dec, 2014 | Pubmed ID: 25964863 Chimeric Antigen Receptor T-cell Therapy for ALL Hematology. American Society of Hematology. Education Program. Dec, 2014 | Pubmed ID: 25696911 Advances in T-cell Therapy for ALL Best Practice & Research. Clinical Haematology. 2014 | Pubmed ID: 25455270 Chimeric Antigen Receptor T Cells for Sustained Remissions in Leukemia The New England Journal of Medicine. Oct, 2014 | Pubmed ID: 25317870 Nature of Tumor Control by Permanently and Transiently Modified GD2 Chimeric Antigen Receptor T Cells in Xenograft Models of Neuroblastoma Cancer Immunology Research. Nov, 2014 | Pubmed ID: 25104548 Engineered T Cells for Cancer Therapy Cancer Immunology, Immunotherapy : CII. Sep, 2014 | Pubmed ID: 24943274 Current Concepts in the Diagnosis and Management of Cytokine Release Syndrome Blood. Jul, 2014 | Pubmed ID: 24876563 Managing Cytokine Release Syndrome Associated with Novel T Cell-engaging Therapies Cancer Journal (Sudbury, Mass.). 2014 | Pubmed ID: 24667956 Preclinical Targeting of Human Acute Myeloid Leukemia and Myeloablation Using Chimeric Antigen Receptor-modified T Cells Blood. Apr, 2014 | Pubmed ID: 24596416 Antibody-modified T Cells: CARs Take the Front Seat for Hematologic Malignancies Blood. Apr, 2014 | Pubmed ID: 24578504 INKT Cell Cytotoxic Responses Control T-lymphoma Growth in Vitro and in Vivo Cancer Immunology Research. Jan, 2014 | Pubmed ID: 24563871 The Addition of Sirolimus to Tacrolimus/methotrexate GVHD Prophylaxis in Children with ALL: a Phase 3 Children's Oncology Group/Pediatric Blood and Marrow Transplant Consortium Trial Blood. Mar, 2014 | Pubmed ID: 24497539 Relation of Clinical Culture Method to T-cell Memory Status and Efficacy in Xenograft Models of Adoptive Immunotherapy Cytotherapy. May, 2014 | Pubmed ID: 24439255 Toxicity Management for Patients Receiving Novel T-cell Engaging Therapies Current Opinion in Pediatrics. Feb, 2014 | Pubmed ID: 24362408 Purged Versus Non-purged Peripheral Blood Stem-cell Transplantation for High-risk Neuroblastoma (COG A3973): a Randomised Phase 3 Trial The Lancet. Oncology. Sep, 2013 | Pubmed ID: 23890779 Challenges and Opportunities for International Cooperative Studies in Pediatric Hematopoeitic Cell Transplantation: Priorities of the Westhafen Intercontinental Group Biology of Blood and Marrow Transplantation : Journal of the American Society for Blood and Marrow Transplantation. Sep, 2013 | Pubmed ID: 23883618 Regimen-specific Effects of RNA-modified Chimeric Antigen Receptor T Cells in Mice with Advanced Leukemia Human Gene Therapy. Aug, 2013 | Pubmed ID: 23883116 Cytokine Release Syndrome After Blinatumomab Treatment Related to Abnormal Macrophage Activation and Ameliorated with Cytokine-directed Therapy Blood. Jun, 2013 | Pubmed ID: 23678006 Chimeric Antigen Receptor-modified T Cells for Acute Lymphoid Leukemia The New England Journal of Medicine. Apr, 2013 | Pubmed ID: 23527958 Allogeneic Hematopoietic Cell Transplantation for Neuroblastoma: the CIBMTR Experience Bone Marrow Transplantation. Feb, 2013 | Pubmed ID: 23419433 A Pilot Study of Tandem High-dose Chemotherapy with Stem Cell Rescue As Consolidation for High-risk Neuroblastoma: Children's Oncology Group Study ANBL00P1 Bone Marrow Transplantation. Jul, 2013 | Pubmed ID: 23334272 Sirolimus Pharmacokinetics in Early Postmyeloablative Pediatric Blood and Marrow Transplantation Biology of Blood and Marrow Transplantation : Journal of the American Society for Blood and Marrow Transplantation. Apr, 2013 | Pubmed ID: 23266742 Children's Oncology Group's 2013 Blueprint for Research: Stem Cell Transplantation Pediatric Blood & Cancer. Jun, 2013 | Pubmed ID: 23255402 Adoptive Transfer of Autologous T Cells Improves T-cell Repertoire Diversity and Long-term B-cell Function in Pediatric Patients with Neuroblastoma Clinical Cancer Research : an Official Journal of the American Association for Cancer Research. 12, 2012 | Pubmed ID: 23092876 Targeting JAK1/2 and MTOR in Murine Xenograft Models of Ph-like Acute Lymphoblastic Leukemia Blood. Oct, 2012 | Pubmed ID: 22955920 Genetic Alterations Activating Kinase and Cytokine Receptor Signaling in High-risk Acute Lymphoblastic Leukemia Cancer Cell. Aug, 2012 | Pubmed ID: 22897847 Targeting the PI3K/AKT/mTOR Signaling Axis in Children with Hematologic Malignancies Paediatric Drugs. Oct, 2012 | Pubmed ID: 22845486 Feasibility of a Tandem Autologous Peripheral Blood Stem Cell Transplant Regimen for High Risk Neuroblastoma in a Cooperative Group Setting: a Pediatric Oncology Group Study: a Report from the Children's Oncology Group Pediatric Blood & Cancer. Nov, 2012 | Pubmed ID: 22744917 The Future is Now: Chimeric Antigen Receptors As New Targeted Therapies for Childhood Cancer Clinical Cancer Research : an Official Journal of the American Association for Cancer Research. May, 2012 | Pubmed ID: 22589486 Neuroblastoma: Issues in Transplantation Biology of Blood and Marrow Transplantation : Journal of the American Society for Blood and Marrow Transplantation. Jan, 2012 | Pubmed ID: 22226119 Randomized Trial of Hydroxychloroquine for Newly Diagnosed Chronic Graft-versus-host Disease in Children: a Children's Oncology Group Study Biology of Blood and Marrow Transplantation : Journal of the American Society for Blood and Marrow Transplantation. Jan, 2012 | Pubmed ID: 21689773 Noninvasive Bioluminescent Imaging of Primary Patient Acute Lymphoblastic Leukemia: a Strategy for Preclinical Modeling Blood. Oct, 2011 | Pubmed ID: 21856863 Treatment of Advanced Leukemia in Mice with MRNA Engineered T Cells Human Gene Therapy. Dec, 2011 | Pubmed ID: 21838572 T Cells with Chimeric Antigen Receptors Have Potent Antitumor Effects and Can Establish Memory in Patients with Advanced Leukemia Science Translational Medicine. Aug, 2011 | Pubmed ID: 21832238 Phase I Study of Temsirolimus in Pediatric Patients with Recurrent/refractory Solid Tumors Journal of Clinical Oncology : Official Journal of the American Society of Clinical Oncology. Jul, 2011 | Pubmed ID: 21690471 Early and Late Extensive Chronic Graft-versus-host Disease in Children is Characterized by Different Th1/Th2 Cytokine Profiles: Findings of the Children's Oncology Group Study ASCT0031 Biology of Blood and Marrow Transplantation : Journal of the American Society for Blood and Marrow Transplantation. Dec, 2011 | Pubmed ID: 21669298 Statins Are Active in Acute Lymphoblastic Leukaemia (ALL): a Therapy That May Treat ALL and Prevent Avascular Necrosis British Journal of Haematology. Nov, 2011 | Pubmed ID: 21554258 Adoptive Cellular Therapy Current Topics in Microbiology and Immunology. 2011 | Pubmed ID: 20700700 Cutting Edge: Lymphoproliferation Caused by Fas Deficiency is Dependent on the Transcription Factor Eomesodermin Journal of Immunology (Baltimore, Md. : 1950). Dec, 2010 | Pubmed ID: 21076068 Anti-GD2 Antibody with GM-CSF, Interleukin-2, and Isotretinoin for Neuroblastoma The New England Journal of Medicine. Sep, 2010 | Pubmed ID: 20879881 Autologous and Allogeneic Cellular Therapies for High-risk Pediatric Solid Tumors Pediatric Clinics of North America. Feb, 2010 | Pubmed ID: 20307711 Anti-CD13 Abs in Children with Extensive Chronic GVHD and Their Relation to Soluble CD13 After Allogeneic Blood and Marrow Transplantation from a Children's Oncology Groups Study, ASCT0031 Bone Marrow Transplantation. Nov, 2010 | Pubmed ID: 20190833 Identifying Autoimmune Lymphoproliferative Syndrome in Children with Evans Syndrome: a Multi-institutional Study Blood. Mar, 2010 | Pubmed ID: 20068224 Advances in the Management and Understanding of Autoimmune Lymphoproliferative Syndrome (ALPS) British Journal of Haematology. Jan, 2010 | Pubmed ID: 19930184 Interferon-gamma-dependent Infiltration of Human T Cells into Neuroblastoma Tumors in Vivo Clinical Cancer Research : an Official Journal of the American Association for Cancer Research. Nov, 2009 | Pubmed ID: 19825945 A Phase I/II Study of the Safety and Efficacy of the Addition of Sirolimus to Tacrolimus/methotrexate Graft Versus Host Disease Prophylaxis After Allogeneic Haematopoietic Cell Transplantation in Paediatric Acute Lymphoblastic Leukaemia (ALL) British Journal of Haematology. Dec, 2009 | Pubmed ID: 19744131 Long-term Protection from Syngeneic Acute Lymphoblastic Leukemia by CpG ODN-mediated Stimulation of Innate and Adaptive Immune Responses Blood. Sep, 2009 | Pubmed ID: 19636062 Chimeric Receptors Containing CD137 Signal Transduction Domains Mediate Enhanced Survival of T Cells and Increased Antileukemic Efficacy in Vivo Molecular Therapy : the Journal of the American Society of Gene Therapy. Aug, 2009 | Pubmed ID: 19384291 Mammalian Target of Rapamycin Inhibitors and Their Potential Role in Therapy in Leukaemia and Other Haematological Malignancies British Journal of Haematology. Jun, 2009 | Pubmed ID: 19344392 Treatment with Sirolimus Results in Complete Responses in Patients with Autoimmune Lymphoproliferative Syndrome British Journal of Haematology. Apr, 2009 | Pubmed ID: 19208097 Novel Molecular and Cellular Therapeutic Targets in Acute Lymphoblastic Leukemia and Lymphoproliferative Disease Immunologic Research. 2008 | Pubmed ID: 18716718 Late Effects in Survivors of Tandem Peripheral Blood Stem Cell Transplant for High-risk Neuroblastoma Pediatric Blood & Cancer. Nov, 2008 | Pubmed ID: 18623215 MTOR Inhibitors Are Synergistic with Methotrexate: an Effective Combination to Treat Acute Lymphoblastic Leukemia Blood. Sep, 2008 | Pubmed ID: 18544682 Immunotherapy for Pediatric Cancer Biology of Blood and Marrow Transplantation : Journal of the American Society for Blood and Marrow Transplantation. Jan, 2008 | Pubmed ID: 18162219 Targeting Notch Signaling in Autoimmune and Lymphoproliferative Disease Blood. Jan, 2008 | Pubmed ID: 17925488 Thymic Stromal-derived Lymphopoietin Induces Proliferation of Pre-B Leukemia and Antagonizes MTOR Inhibitors, Suggesting a Role for Interleukin-7Ralpha Signaling Cancer Research. Oct, 2007 | Pubmed ID: 17942929 Immunosurveillance and Survivin-specific T-cell Immunity in Children with High-risk Neuroblastoma Journal of Clinical Oncology : Official Journal of the American Society of Clinical Oncology. Dec, 2006 | Pubmed ID: 17179106 Rapamycin Improves Lymphoproliferative Disease in Murine Autoimmune Lymphoproliferative Syndrome (ALPS) Blood. Sep, 2006 | Pubmed ID: 16757690 Use of G-CSF in Matched Sibling Donor Pediatric Allogeneic Transplantation: a Consensus Statement from the Children's Oncology Group (COG) Transplant Discipline Committee and Pediatric Blood and Marrow Transplant Consortium (PBMTC) Executive Committee Pediatric Blood & Cancer. Apr, 2006 | Pubmed ID: 16463346 Collection, Storage, and Infusion of Stem Cells in Children with High-risk Neuroblastoma: Saving for a Rainy Day Pediatric Blood & Cancer. Jun, 2006 | Pubmed ID: 16429413 The MTOR Inhibitor CCI-779 Induces Apoptosis and Inhibits Growth in Preclinical Models of Primary Adult Human ALL Blood. Feb, 2006 | Pubmed ID: 16195324 Unmasking Evans Syndrome: T-cell Phenotype and Apoptotic Response Reveal Autoimmune Lymphoproliferative Syndrome (ALPS) Blood. Mar, 2005 | Pubmed ID: 15542578 RNA-transfected CD40-activated B Cells Induce Functional T-cell Responses Against Viral and Tumor Antigen Targets: Implications for Pediatric Immunotherapy Blood. Mar, 2004 | Pubmed ID: 14630810 Rapamycin is Active Against B-precursor Leukemia in Vitro and in Vivo, an Effect That is Modulated by IL-7-mediated Signaling Proceedings of the National Academy of Sciences of the United States of America. Dec, 2003 | Pubmed ID: 14657335 Role of the BCR Complex in B Cell Development, Activation, and Leukemic Transformation Immunologic Research. 2003 | Pubmed ID: 12857978 Cytoplasmic Micro Heavy Chain Confers Sensitivity to Dexamethasone-induced Apoptosis in Early B-lineage Acute Lymphoblastic Leukemia Cancer Research. Aug, 2002 | Pubmed ID: 12154021 Advancements in CAR-T Cell Manufacturing and Gene Therapy Production (Video) | JoVE Rene Machietto1, Nicholas Giacobbe1, Jessica Perazzelli2, Ted J. Hofmann2, Allison Barz Leahy2,3, Stephan A. Grupp1,2,3, Yongping Wang1,3,4, Stephan Kadauke1,3,4 1Cell and Gene Therapy Laboratory, Department of Pathology and Laboratory Medicine, Children’s Hospital of Philadelphia, 2Division of Oncology, Department of Pediatrics, Children’s Hospital of Philadelphia, 3Perelman School of Medicine at the University of Pennsylvania, 4Division of Transfusion Medicine, Department of Pathology and Laboratory Medicine, Children’s Hospital of Philadelphia JoVE 65488 Medicine
Advancements in CAR-T Cell Manufacturing and Gene Therapy Production (Video) | JoVE Rene Machietto1, Nicholas Giacobbe1, Jessica Perazzelli2, Ted J. Hofmann2, Allison Barz Leahy2,3, Stephan A. Grupp1,2,3, Yongping Wang1,3,4, Stephan Kadauke1,3,4 1Cell and Gene Therapy Laboratory, Department of Pathology and Laboratory Medicine, Children’s Hospital of Philadelphia, 2Division of Oncology, Department of Pediatrics, Children’s Hospital of Philadelphia, 3Perelman School of Medicine at the University of Pennsylvania, 4Division of Transfusion Medicine, Department of Pathology and Laboratory Medicine, Children’s Hospital of Philadelphia JoVE 65488 Medicine
