Luigi Naldini, MD, PhD
Marina Cavazzana, MD, PhD
THE AMERICAN SOCIETY OF HEMATOLOGY (ASH) will honor Luigi Naldini, MD, PhD, of the San Raffaele Telethon Institute for Gene Therapy in Milan, and Marina Cavazzana, MD, PhD, of Paris Descartes University, Necker-Enfants Malades Hospital, and Imagine Institute of Genetic Diseases, AP-HP, Inserm in Paris with the 2017 Ernest Beutler Lecture and Prize for their significant research advances in the area of hematopoietic stem cell (HSC) gene therapy.
The Ernest Beutler Lecture, named for the late Ernest Beutler, MD, Past President of ASH and a physician-scientist for more than 50 years, is a two-part lectureship that recognizes major translational advances related to a single topic. The award honors two individuals: one who has enabled advances in basic science and another for achievements in clinical science or translational research.
Professors Naldini and Cavazzana will present their lecture, Turning Foes Into Friends: Exploiting HIV to Generate Efficient and Safe Vectors to Cure Inherited and Acquired Blood Diseases, at 1:30 PM on Monday, December 11, at the 59th ASH Annual Meeting and Exposition in Atlanta. Their lecture will detail the advent of lentiviral vectors derived from human immunodeficiency virus (HIV) and how they have revolutionized gene therapy for blood disorders. Prof. Naldini will review the development and the molecular analyses showing safe and efficient HSC gene transfer in preclinical and clinical studies, and Prof. Cavazzana will review the current stage of clinical application and benefit of use of these vectors in HSC gene transfer.
Prof. Luigi Naldini
PROF. NALDINI, the 2017 Ernest Beutler Lecturer for Basic Science, is Director of the San Raffaele Telethon Institute for Gene Therapy in Milan and Professor of Cell and Tissue Biology and Gene and Cell Therapy at the “Vita Salute San Raffaele” University School of Medicine in Milan. He obtained his medical degree at the University of Torino in 1983 and his PhD in cell and developmental biology at the Sapienza University of Rome in 1987. He went on to complete his postdoctoral training in tyrosine kinases and signal transduction.
Prof. Naldini is known for his major scientific contributions to the understanding of stem cell transplantation and cell and gene therapy. Many of his accomplishments contributed to the safe and efficient use of gene therapy, which made these therapies more viable for the clinic. Early in his career, he described the use of HIV-derived hybrid lentiviral vectors for gene transfer into nondividing cell, working with Didier Trono, MD, and Inder Verma, PhD, at the Salk Institute for Biological Studies in La Jolla, California. This work laid the foundation for the broad use of lentiviral vectors.
Building on that foundation, Prof. Naldini’s continued studies led to a more advanced design of lentiviral vectors that caused fewer side effects than previous models of gene therapy. He also made it possible to monitor HSC activity in patients and developed a strategy for micoRNA regulation that is now widely adapted to develop safer vectors.
In addition to his seminal work with lentiviral vector therapy, Prof. Naldini’s laboratory has pioneered the use of engineered zinc-finger nucleases to edit the human genome. His studies have allowed researchers to correct, rather than replace, genes.
Prof. Naldini has published 245 papers in international scientific journals, which have been cited over 30,600 times, and was awarded a European Research Council advanced grant in 2011. He was appointed member of the European Molecular Biology Organization in 2008; President of the European Society of Gene and Cell Therapy from 2012 to 2014; member of the Human Gene Editing Study Committee by the National Academies of Sciences and of Medicine (2015–2017); and member of the Italian National Advisory Committee on Biosafety, Biotechnology, and Life Sciences in 2016. He has also received numerous awards, including the Outstanding Achievement Award from the American Society of Gene and Cell Therapy, the Human Gene Therapy “Pioneer Award,” and the Outstanding Achievement Award from the European Society of Gene and Cell Therapy.
Prof. Marina Cavazzana
PROF. CAVAZZANA, the 2017 Ernest Beutler Lecturer for Clinical Science, is Professor of Hematology at Paris Descartes University; Head of the Department of Biotherapy at Hospital Necker-Enfants Malades, AP-HP; and Chief of Research of the Laboratory of Human Lympho-hematopoiesis U1163 Inserm in the Institut Imagine in Paris. She also serves as Coordinator of the Biotherapy Clinical Investigation Center at Necker-Enfants Malades, hospital from AP-HP, Greater Paris University Hospitals, and INSERM. She completed her residency at the University of Padua in Italy and obtained her PhD in blood cell biology at Paris VII University.
Prof. Cavazzana has made seminal discoveries that have led to clinical trials with lentiviral vector gene–modified cells for treatment of nonmalignant hematologic diseases. She has spearheaded translational and clinical advances achieved in HSC gene therapy for multiple genetic immune, hematopoietic, and metabolic disorders. She is well known for her work to design, lead, and implement a clinical trial of retroviral gene therapy for X-linked severe combined immunodeficiency (X-SCID), a rare, inherited disorder of the immune system. In 2002, Prof. Cavazzana reported the first benefit in a human gene therapy trial when she demonstrated reconstitution of the immune system in young boys with X-SCID. When it was discovered that two of these boys developed leukemia from the therapy, she coordinated the efforts of a highly skilled group of clinicians, virologists, molecular biologists, and regulators to develop novel therapies. In addition to this work, in 2017, she published the proof of principle that using a lentiviral vector expressing an antisickling beta chain into the hematopoietic stem cells provides a stable clinical benefit to a patient with sickle cell disease.
Prof. Cavazzana is the author of more than 240 publications in peer-reviewed journals and was awarded two European Research Council grants (2011 and 2016). Her work was rewarded by ASH (Award on Clinical Research in Gene Therapy in 1999) and by the French Academy of Sciences (Special Medical Award in 2000 and Jean-Pierre Lecocq Award on Gene Therapy in 2004). She was awarded with the Matmut Prize for Medical Innovation in 2008; the Outstanding Achievement Award of the European Society of Gene and Cell Therapy in 2009; the title of Officier de l’Ordre National de la Légion d’honneur in 2011; and the Irène Joliot-Curie “Scientific Woman of the Year” award in 2012 by the French Academy of Sciences, French Ministry of Education and Research, and EADS group corporate foundation. In 2016, she was awarded the French National Medicine Academy Prize.
She currently serves as a member of UNAIDS Science Panel and has previously served as Chairwoman of the Scientific Committee for Strategies and Evaluation of Clinical Research Protocols (INSERM). She has also served as a member of the Blood editorial board.
“Professors Naldini and Cavazzana have made outstanding contributions to medicine that resulted in safer and more efficient use of gene therapy to treat genetic blood disorders,” said ASH President Kenneth C. Anderson, MD, of the Lebow Institute for Myeloma Therapeutics and Jerome Lipper Myeloma Center at Dana-Farber Cancer Institute. “I am pleased that the Society has chosen them for this honor to present one of our most prestigious lectures.” ■