Marina Cavazzana

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.

Marina Cavazzana
Marina Cavazzana
Born1959
Venice
Alma materUniversity of Padua
Paris Diderot University
Known forGene therapy
Scientific career
InstitutionsNecker-Enfants Malades Hospital
Paris Descartes University

Early life and education

Cavazzana was born in Venice. Her father worked on the railway and her mother was a teacher. She studied medicine in Padua, where she completed her residency at the University of Padua.[1] She moved to Paris, where she earned her doctorate at the Paris Diderot University studying bone marrow transplants with Élaine Gluckman at the Hôpital Saint-Louis.[1]

Research and career

Cavazzana joined the Necker-Enfants Malades Hospital in 1987, where she worked with Alain Fischer and Claude Gricelli. She was appointed to Professor of Immunology in 1994. She served as Director of the Institut national de la santé et de la recherche médicale (INSERM) in 1994. In 2003 she was made Head of the Haematology Department and Director of Biotherapy at the Necker-Enfants Malades Hospital.[2] She established the first Clinical Investigation Center in the Assistance Publique – Hôpitaux de Paris, which is the only French academic department is authorised to produce cell and gene therapies.

Her research considers the haematopoietic immune system[3] and gene therapy.[4] She has designed clinical trials based on lentiviral vector in gene therapy and pioneered the use of haematopoietic stem cell gene therapy for genetic immune and haematopoietic disorders.[1] In 2002 Cavazzana started working with Philippe Leboulch trying to develop clinical prootocols for the treatment of anemia.[5] Cavazzana was the first doctor to successfully treat a boy with sickle cell disease, a severe form of hereditary chronic anemia. Sickle cell disease is a blood disorder that results from an abnormality in red blood cells. It occurs due to a mutation on the HBB gene, and results in a sickle-like deformity of red blood cells. It is the most common genetic disorder in France. The first gene therapy trail of young boys with X-linked severe combined immunodeficiency was performed by Cavazzana in collaboration with bluebird bio in 2000.[6] When it was established that two of the boys developed leukaemia from the gene therapy, Cavazzana coordinated the medical efforts of biologists, virologists, clinicians and regulators to develop novel medical therapies.[1] In 2010 she performed gene therapy on a child with beta thalassemia.[7][8][9] Children who are born with Thalassemia Major often develop anaemia within their first years of life.[5] Cavazzana went on to provide gene therapy to sickle cell disease in 2014[5] and the first patient with Wiskott–Aldrich syndrome.[10] Wiskott–Aldrich syndrome is an immunodeficiency associated with microthrombocytopenia. The child with sickle cell disease had a restored clinical and biological phenotype within sixth months of the gene therapy.[5]

In 2017 Cavazzana was involved in re-writing the French bioethics laws, in particular, Law 2011-814.[11] The law requires close family members to be informed when individuals are at risk of developing a genetically transmitted illness.

Awards and honours

References
  1. "Luigi Naldini, MD, PhD, and Marina Cavazzana, MD, PhD, to Present 2017 American Society of Hematology Ernest Beutler Lecture". www.hematology.org. 2017-08-24. Retrieved 2019-11-01.
  2. "Necker's site clinical research". www.institutimagine.org. Retrieved 2019-11-01.
  3. "24 research labs". www.institutimagine.org. Retrieved 2019-11-01.
  4. Gene therapies, today: Marina Cavazzana at TEDxLakeComo, retrieved 2019-11-01
  5. "Necker-Enfants Malades Hospital: The gene-ius treating sickle cell disease". Research Features. 2017-07-26. Retrieved 2019-11-01.
  6. Cavazzana-Calvo, M. (2000-04-28). "Gene Therapy of Human Severe Combined Immunodeficiency (SCID)-X1 Disease". Science. 288 (5466): 669–672. Bibcode:2000Sci...288..669C. doi:10.1126/science.288.5466.669. ISSN 0036-8075. PMID 10784449.
  7. Cavazzana-Calvo, Marina; Payen, Emmanuel; Negre, Olivier; Wang, Gary; Hehir, Kathleen; Fusil, Floriane; Down, Julian; Denaro, Maria; Brady, Troy; Westerman, Karen; Cavallesco, Resy (2010). "Transfusion independence and HMGA2 activation after gene therapy of human β-thalassaemia". Nature. 467 (7313): 318–322. Bibcode:2010Natur.467..318C. doi:10.1038/nature09328. ISSN 1476-4687. PMC 3355472. PMID 20844535.
  8. Hacein-Bey-Abina, Salima; Pai, Sung-Yun; Gaspar, H. Bobby; Armant, Myriam; Berry, Charles C.; Blanche, Stephane; Bleesing, Jack; Blondeau, Johanna; de Boer, Helen (2014-10-08). "A Modified γ-Retrovirus Vector for X-Linked Severe Combined Immunodeficiency". New England Journal of Medicine. 371 (15): 1407–1417. doi:10.1056/nejmoa1404588. PMC 4274995. PMID 25295500.
  9. Thompson, Alexis A.; Walters, Mark C.; Kwiatkowski, Janet; Rasko, John E. J.; Ribeil, Jean-Antoine; Hongeng, Suradej; Magrin, Elisa; Schiller, Gary J.; Payen, Emmanuel; Semeraro, Michaela; Moshous, Despina (2018-04-18). "Gene Therapy in Patients with Transfusion-Dependent β-Thalassemia". New England Journal of Medicine. 378 (16): 1479–1493. doi:10.1056/NEJMoa1705342. PMID 29669226.
  10. Ribeil, Jean-Antoine; Hacein-Bey-Abina, Salima; Payen, Emmanuel; Magnani, Alessandra; Semeraro, Michaela; Magrin, Elisa; Caccavelli, Laure; Neven, Benedicte; Bourget, Philippe (2017-03-01). "Gene Therapy in a Patient with Sickle Cell Disease". New England Journal of Medicine. 376 (9): 848–855. doi:10.1056/nejmoa1609677. PMID 28249145.
  11. Atwill, Nicole (2011-07-18). "France: New Bioethics Law | Global Legal Monitor". www.loc.gov. Retrieved 2019-11-01.
  12. "Marina Cavazzana-Calvo - Prix de Recherche, clinique et thérapeutique 2006 / Histoire de l'Inserm". histoire.inserm.fr. Retrieved 2019-05-06.
  13. "Marina Cavazzana-Calvo, lauréate du Prix de la "Femme Scientifique de l'année" - hopital.fr - Fédération Hospitalière de France". www.hopital.fr. Retrieved 2019-05-06.
  14. "Lauréats 2016". Académie nationale de médecine | Une institution dans son temps (in French). 2016-06-29. Retrieved 2019-05-06.
  15. "Fondation Guillaumat-Piel". Fondation pour la Recherche Médicale (in French). Retrieved 2019-11-01.
  16. "Ernest Beutler Lecture and Prize Recipients". www.hematology.org. 2018-08-29. Retrieved 2019-05-06.
  17. "University of Zurich Awards Six New Honorary Doctorates". www.media.uzh.ch. Retrieved 2019-05-06.
  18. "Newly elected NAM members". NAM. Retrieved 2019-11-01.
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