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Maria Grazia Roncarolo, MD is the George D. Smith Professor in Stem Cell and Regenerative Medicine, Professor of Pediatrics and of Medicine, director of the Center for Definitive and Curative Medicine, and co-director of the Institute for Stem Cell Biology and Regenerative Medicine. Dr. Roncarolo leads efforts to translate scientific discoveries in genetic diseases and regenerative medicine into novel patient therapies, including treatments based on stem cells and gene therapy.A pediatric immunologist by training, she earned her medical degree at the University of Turin, Italy. She spent her early career in Lyon, France, where she focused on severe inherited metabolic and immune diseases, including severe combined immunodeficiency (SCID), better known as the "bubble boy disease." Dr. Roncarolo was a key member of the team that carried out the first stem cell transplants given before birth to treat these genetic diseases.While studying inherited immune diseases, Dr. Roncarolo discovered a new class of T cells. These cells, called T regulatory type 1 cells, help maintain immune system homeostasis by preventing autoimmune diseases and assisting the immune system in tolerating transplanted cells and organs. Dr. Roncarolo completed the first clinical trial using T regulatory type 1 cells to prevent severe graft-versus-host disease in leukemia patients receiving blood-forming stem-cell transplants from donors who were not genetic matches. Dr. Roncarolo worked for several years at DNAX Research Institute for Molecular and Cellular Biology in Palo Alto, where she contributed to the discovery of novel cytokines, cell-signaling molecules that are part of the immune response. She studied the role of cytokines in inducing immunological tolerance and in promoting stem cell growth and differentiation.Dr. Roncarolo developed new gene-therapy approaches, which she pursued as director of the Telethon Institute for Cell and Gene Therapy at the San Raffaele Scientific Institute in Milan. She was the principal investigator leading the successful gene therapy trial for SCID patients who lack an enzyme critical to DNA synthesis, which is a severe life-threatening disorder. Based on the results of this trial, gene therapy for ADA-SCID has obtained Orphan drug status from both the FDA and EMEA and it was licensed to Glaxo Smith Klein, which has received European Commission approval to market under the name of Strimvelis. Under her direction, the San Raffaele Scientific Institute has been seminal in showing the efficacy of gene therapy for otherwise untreatable inherited metabolic diseases and primary immunodeficiencies.Dr. Roncarolo established the Stanford Center for Definitive and Curative Medicine to cure patients with currently incurable diseases through the development of innovative stem cell-and gene-based therapies.
Research InterestsImmunetolerance: Mechanisms underlying T-cell tolerance, induction of T-cell anergy and regulatory T cells; Immunomodulation: mAbs, proteins and low molecular weight compounds which can modulate T-cell activation; Primary immunodeficiencies: Characterization of molecular and immunological defects; Gene therapy: Gene transduction of hematopoietic cells for gene therapy in primary immunodeficiencies and metabolic diseases; Hematopoiesis: Mechanisms underlying growth and differentiation of hematopoietic stem cells; Transplantation: Immune reconstitution and T-cell tolerance after allogenic stem cell transplantation; Cytokines/Cytokine receptors: Role in regulation of immune and inflammatory responsesClinical InterestsPrimary ImmunodeficienciesMonogenic Autoimmune DisordersAllogenic Bone Marrow Transplantation Gene Therapy Clinical Trials Cell Therapy Clinical TrialsClinical Trials in Autoimmune Diseases and Organ TransplantationClinical Trials in Hemoglobinopathies
CD4^LVFOXP3 in Participants With IPEX
This first-in-human, Phase 1 clinical trial will test the feasibility of the manufacturing
and the safety of the administration of CD4^LVFOXP3 in up to 36 evaluable human participants
with IPEX and evaluate the impact of the CD4^LVFOXP3 infusion on the disease.
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Stem Cell Transplant From Donors After Alpha Beta Cell Depletion in Children and Adults With T-allo10 Cells Addback
The purpose of this study is to determine the safety of a cell therapy, T-allo10, after
αβdepleted-HSCT in the hopes that it will boost the adaptive immune reconstitution of the
patient while sparing the risk of developing severe Graft-versus-Host Disease (GvHD).
The primary objective of Phase 1 is to determine the recommended Phase 2 dose (RP2D)
administered after infusion of αβdepleted-HSCT in children and young adults with hematologic
A Phase 1b extension will occur after dose escalation, enrolling at the RP2D for the T-allo10
cells determined in the Phase 1 portion to evaluate the safety and efficacy of infusion of
T-allo10 after receipt of αβdepleted-HSCT. Additionally, Phase 1b aims to explore
improvements in immune reconstitution.
All participants on this study must be enrolled on another study: NCT04249830