Maria Grazia Roncarolo

Last updated

Maria Grazia Roncarolo
Born
Turin, Italy
Alma mater University of Turin
Scientific career
Institutions Stanford University

Maria Grazia Roncarolo (born 17 December 1954) is an Italian pediatrician who is currently George D. Smith Professor in Stem Cell and Regenerative Medicine and Professor of Medicine (Blood and Marrow Transplantation and Cellular Therapy) at Stanford University. [1] She is also the Director of the Stanford Institute of Stem Cell Biology and Regenerative Medicine along with Irving Weissman and Michael Longaker and the Director for Center for Definitive and Curative Medicine at Stanford. [2]

Contents

She was educated at the University of Turin. [1] She is a past President of the Federation of Clinical Immunology Societies, [3] and is a member of the Austrian Academy of Sciences. [4] She is an academic founder of Graphite Bio. [5] She has an h-index of 91. [6]

Awards and honors

Research career

Roncarolo is well known for identifying a peripheral subset of regulatory T cells, called type 1 regulatory T (Tr1) cells. Her team was the first to describe Tr1 cells [12] while at DNAX Research Institute of Molecular and Cellular Biology, which was later acquired by Schering-Plough and now a part of Merck. She was the principal investigator of the first clinical trial using Tr1 cells that are generated ex vivo to treat graft-versus-host disease in leukemia patients receiving a haploidentical haematopoietic stem cell transplant [13] and is the principal investigator of an additional clinical trial utilizing these cells in the United States. [14]

Roncarolo has made major contributions in the field of cell and gene therapy.  She performed fetal stem cell transplants given before birth [15] to treat inherited diseases of the immune system such as Wiskott-Aldrich Syndrome [16] She led the first stem cell-based gene therapy trial for patients with adenosine deaminase-severe combined immunodeficiency. [17] The clinical trial results led to the European Commission approval. [18] The therapy was licensed to GlaxoSmithKline and is marketed under the name Strimvelis, making it the first commercially approved ex vivo gene therapy in Europe. [19]

Related Research Articles

<span class="mw-page-title-main">Gene therapy</span> Medical field

Gene therapy is a medical technology that aims to produce a therapeutic effect through the manipulation of gene expression or through altering the biological properties of living cells.

<span class="mw-page-title-main">Severe combined immunodeficiency</span> Genetic disorder leading to severe impairment of the immune system

Severe combined immunodeficiency (SCID), also known as Swiss-type agammaglobulinemia, is a rare genetic disorder characterized by the disturbed development of functional T cells and B cells caused by numerous genetic mutations that result in differing clinical presentations. SCID involves defective antibody response due to either direct involvement with B lymphocytes or through improper B lymphocyte activation due to non-functional T-helper cells. Consequently, both "arms" of the adaptive immune system are impaired due to a defect in one of several possible genes. SCID is the most severe form of primary immunodeficiencies, and there are now at least nine different known genes in which mutations lead to a form of SCID. It is also known as the bubble boy disease and bubble baby disease because its victims are extremely vulnerable to infectious diseases and some of them, such as David Vetter, have become famous for living in a sterile environment. SCID is the result of an immune system so highly compromised that it is considered almost absent.

<span class="mw-page-title-main">Adenosine deaminase deficiency</span> Medical condition

Adenosine deaminase deficiency is a metabolic disorder that causes immunodeficiency. It is caused by mutations in the ADA gene. It accounts for about 10–20% of all cases of autosomal recessive forms of severe combined immunodeficiency (SCID) after excluding disorders related to inbreeding.

<span class="mw-page-title-main">Wiskott–Aldrich syndrome</span> Medical condition

Wiskott–Aldrich syndrome (WAS) is a rare X-linked recessive disease characterized by eczema, thrombocytopenia, immune deficiency, and bloody diarrhea. It is also sometimes called the eczema-thrombocytopenia-immunodeficiency syndrome in keeping with Aldrich's original description in 1954. The WAS-related disorders of X-linked thrombocytopenia (XLT) and X-linked congenital neutropenia (XLN) may present with similar but less severe symptoms and are caused by mutations of the same gene.

<span class="mw-page-title-main">Cell therapy</span> Therapy in which cellular material is injected into a patient

Cell therapy is a therapy in which viable cells are injected, grafted or implanted into a patient in order to effectuate a medicinal effect, for example, by transplanting T-cells capable of fighting cancer cells via cell-mediated immunity in the course of immunotherapy, or grafting stem cells to regenerate diseased tissues.

Enzyme replacement therapy (ERT) is a medical treatment which replaces an enzyme that is deficient or absent in the body. Usually, this is done by giving the patient an intravenous (IV) infusion of a solution containing the enzyme.

<span class="mw-page-title-main">IPEX syndrome</span> Medical condition

Immunodysregulation polyendocrinopathy enteropathy X-linked syndrome is a rare autoimmune disease. It is one of the autoimmune polyendocrine syndromes. Most often, IPEX presents with autoimmune enteropathy, dermatitis (eczema), and autoimmune endocrinopathy, but other presentations exist.

Primary immunodeficiencies are disorders in which part of the body's immune system is missing or does not function normally. To be considered a primary immunodeficiency (PID), the immune deficiency must be inborn, not caused by secondary factors such as other disease, drug treatment, or environmental exposure to toxins. Most primary immunodeficiencies are genetic disorders; the majority are diagnosed in children under the age of one, although milder forms may not be recognized until adulthood. While there are over 430 recognized inborn errors of immunity (IEIs) as of 2019, the vast majority of which are PIDs, most are very rare. About 1 in 500 people in the United States are born with a primary immunodeficiency. Immune deficiencies can result in persistent or recurring infections, auto-inflammatory disorders, tumors, and disorders of various organs. There are currently limited treatments available for these conditions; most are specific to a particular type of PID. Research is currently evaluating the use of stem cell transplants (HSCT) and experimental gene therapies as avenues for treatment in limited subsets of PIDs.

Hans Dieter Ochs, is an immunologist and pediatrician. He is Professor of Pediatrics, Division of Immunology, Department of Pediatrics, University of Washington School of Medicine, Seattle.

In 1992 Doctor Claudio Bordignon working at the Vita-Salute San Raffaele University, Milan, Italy performed the first procedure of gene therapy using hematopoietic stem cells as vectors to deliver genes intended to correct hereditary diseases. This was a world first, but was unfortunately unsuccessful because it did not lead to sustained correction of the hematopoietic stem cells. In 2002 this work led to the publication of the first successful gene therapy treatment for adenosine deaminase deficiency (SCID). He expanded this work to stem cell gene therapy of other genetic diseases and AIDS, and for the immunotherapy of cancer.

TK is an experimental cell therapy which may be used to treat high-risk leukemia. It is currently undergoing a Phase III clinical trial to determine efficacy and clinical usefulness.

Elizabeth Simpson OBE FRS FMedSci is a British biologist. She is the Emeritus Professor of Transplantation Biology at Imperial College London. Simpson is particularly known for her elucidation of the nature of male-associated minor transplantation antigens, and their roles in the generation of immunological tolerance, graft versus host disease, and transplant rejection.

Guo Mei is a hematologist and associate director of 307th Hospital of Chinese People’s Liberation Army and deputy director of Radiation Research Institute.

Autologous CD34+ enriched cell fraction that contains CD34+ cells transduced with retroviral vector that encodes for the human ADA cDNA sequence, sold under the brand name Strimvelis, is a medication used to treat severe combined immunodeficiency due to adenosine deaminase deficiency (ADA-SCID).

Type 1 regulatory cells or Tr1 (TR1) cells are a class of regulatory T cells participating in peripheral immunity as a subsets of CD4+ T cells. Tr1 cells regulate tolerance towards antigens of any origin. Tr1 cells are self or non-self antigen specific and their key role is to induce and maintain peripheral tolerance and suppress tissue inflammation in autoimmunity and graft vs. host disease.

Infectious tolerance is a term referring to a phenomenon where a tolerance-inducing state is transferred from one cell population to another. It can be induced in many ways; although it is often artificially induced, it is a natural in vivo process. A number of research deal with the development of a strategy utilizing this phenomenon in transplantation immunology. The goal is to achieve long-term tolerance of the transplant through short-term therapy.

<span class="mw-page-title-main">Shimon Slavin</span> Israeli professor of medicine

Shimon Slavin is an Israeli professor of medicine. Slavin pioneered the use of immunotherapy mediated by allogeneic donor lymphocytes and innovative methods for stem cell transplantation for the cure of hematological malignancies and solid tumors, and using hematopoietic stem cells for induction of transplantation tolerance to bone marrow and donor allografts.

<span class="mw-page-title-main">Graziella Pellegrini</span> Italian Professor of Cell Biology

Graziella Pellegrini is an Italian Professor of Cell Biology and the Cell Therapy Program Coordinator at the University of Modena and Reggio Emilia. She has developed and championed cell therapy protocols in hospitals across Italy.

<span class="mw-page-title-main">Marina Cavazzana</span> Italian physician and cellular biologist

Marina Cavazzana is a professor of Paediatric Immunology at the Necker-Enfants Malades Hospital and the Imagine Institute, as well as an academic at Paris Descartes University. She was awarded the Irène Joliot-Curie Prize in 2012 and elected to the National Academy of Medicine in 2019.

<span class="mw-page-title-main">Cynthia E. Dunbar</span> American hematologist

Cynthia Dunbar is an American scientist and hematologist at the National Heart Lung and Blood Institute (NHLBI), which is part of the National Institutes of Health (NIH). She is the Branch Chief of the Translational Stem Cell Biology Branch.

References

  1. 1 2 "Maria Grazia Roncarolo". Stanford University . Retrieved 23 June 2021.
  2. ↑ "Stanford announces new Center for Definitive and Curative Medicine". News Center (in Samoan). Retrieved 8 February 2022.
  3. ↑ "Leadership". Federation of Clinical Immunology Societies . 4 June 2018. Retrieved 26 June 2021.
  4. ↑ "Maria Grazia Roncarolo". Human Technopole. Retrieved 26 June 2021.
  5. ↑ "About Us: Academic Founders". Graphite Bio. Retrieved 26 June 2021.
  6. ↑ "Maria Grazia Roncarolo - Top Italian Scientist in Biomedical Sciences". Top Italian Scientists. Retrieved 26 June 2021.
  7. ↑ "Academy of Europe: ListMembersByAlphabet". www.ae-info.org. Retrieved 8 February 2022.
  8. ↑ "Academy of Europe: Roncarolo Maria". www.ae-info.org. Retrieved 8 February 2022.
  9. ↑ "Awards". www.esgct.eu. Retrieved 29 September 2023.
  10. ↑ "EURORDIS - The Voice of Rare Disease Patients in Europe". www.eurordis.org. Retrieved 8 February 2022.
  11. ↑ "Outstanding Achievement Award | ASGCT - American Society of Gene & Cell Therapy | ASGCT - American Society of Gene & Cell Therapy". asgct.org. Retrieved 8 February 2022.
  12. ↑ Groux, Hervé; O'Garra, Anne; Bigler, Mike; Rouleau, Matthieu; Antonenko, Svetlana; de Vries, Jan E.; Roncarolo, Maria Grazia (October 1997). "A CD4 + T-cell subset inhibits antigen-specific T-cell responses and prevents colitis". Nature. 389 (6652): 737–742. Bibcode:1997Natur.389..737G. doi:10.1038/39614. PMID   9338786. S2CID   4422991.
  13. ↑ Bacchetta, Rosa; Lucarelli, Barbarella; Sartirana, Claudia; Gregori, Silvia; Lupo Stanghellini, Maria T.; Miqueu, Patrick; Tomiuk, Stefan; Hernandez-Fuentes, Maria; Gianolini, Monica E.; Greco, Raffaella; Bernardi, Massimo (2014). "Immunological Outcome in Haploidentical-HSC Transplanted Patients Treated with IL-10-Anergized Donor T Cells". Frontiers in Immunology. 5: 16. doi: 10.3389/fimmu.2014.00016 . ISSN   1664-3224. PMC   3907718 . PMID   24550909.
  14. ↑ Roncarolo, Maria Grazia (25 June 2021). "Use of T-allo10 Cell Infusions Combined With Mismatched Related or Mismatched Unrelated Hematopoietic Stem Cell Transplantation (HSCT) for Hematologic Malignancies". Stanford University.{{cite journal}}: Cite journal requires |journal= (help)
  15. ↑ "Ailing fetuses to be treated with stem cells". www.science.org. Retrieved 8 February 2022.
  16. ↑ Aiuti, Alessandro; Biasco, Luca; Scaramuzza, Samantha; Ferrua, Francesca; Cicalese, Maria Pia; Baricordi, Cristina; Dionisio, Francesca; Calabria, Andrea; Giannelli, Stefania; Castiello, Maria Carmina; Bosticardo, Marita (23 August 2013). "Lentiviral Hematopoietic Stem Cell Gene Therapy in Patients with Wiskott-Aldrich Syndrome". Science. 341 (6148). doi:10.1126/science.1233151. PMC   4375961 . PMID   23845947.
  17. ↑ Aiuti, Alessandro; Cattaneo, Federica; Galimberti, Stefania; Benninghoff, Ulrike; Cassani, Barbara; Callegaro, Luciano; Scaramuzza, Samantha; Andolfi, Grazia; Mirolo, Massimiliano; Brigida, Immacolata; Tabucchi, Antonella (29 January 2009). "Gene therapy for immunodeficiency due to adenosine deaminase deficiency". The New England Journal of Medicine. 360 (5): 447–458. doi: 10.1056/NEJMoa0805817 . ISSN   1533-4406. PMID   19179314.
  18. ↑ Aiuti, Alessandro; Roncarolo, Maria Grazia; Naldini, Luigi (June 2017). "Gene therapy for ADA‐SCID, the first marketing approval of an ex vivo gene therapy in Europe: paving the road for the next generation of advanced therapy medicinal products". EMBO Molecular Medicine. 9 (6): 737–740. doi:10.15252/emmm.201707573. ISSN   1757-4676. PMC   5452047 . PMID   28396566.
  19. ↑ "Gene Therapy's First Out-and-Out Cure Is Here". MIT Technology Review. Retrieved 8 February 2022.
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