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Marina Cavazzana MD
Head of Service of the Department of Biotherapy at the Necker Hospital
Necker Hospital
Paris
France
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Marina Cavazzana is the Head of Service of the Department of Biotherapy at the Necker Hospital - Sick Children since 2003 and The Director of the Clinical Investigation Center (CIC) specializing in Biotherapy at the Imagine Institute. Since her internship in Pediatrics, she is primarily concerned with hematopoietic stem cell transplantation seeking to improve prognosis. She very quickly associated her clinical activity with fundamental research around the development of the lympho-hematopoietic system through the leadership of a group she led for 25 years. Combining fundamental research and stem biology she conducted more than a dozen phase I/II therapeutic trials based on the use of autograft of hematopoietic stem cells genetically modified for patients with primary immune deficits or hemoglobinopathies, such as sickle cell disease and thalassaemia major; The vast majority of patients treated in the trials have seen improvement from the prognosis of their disease or fully healed. She also discovered or participated in the discovery of genes involved in rare diseases of the hematopoietic system. She used pathophysiological knowledge of the action of these genes to try to develop new drugs. Major advances in the use of RNA as a new way to change the trajectory of genetic diseases suggest a new therapeutic route for conditioning patients requiring an allogeneic hematopoietic stem cell transplant. This new means is being explored in the CIC that she leads under the scientific responsibility of Dr. C. Lagresie. If this approach proves effective in preclinical studies patients should no longer suffer adverse effects of conditioning based today on the use of chemotherapy and/or radiotherapy.