Graziella Pellegrini and Michele De Luca, of Italian nationality, will share the 2020 Jeantet-Collen Prize for Translational Medicine for the development of epithelial stem cell-based regenerative therapy in patients with severe eye and skin disease.
Born in Genoa in 1961, Graziella Pellegrini (“GP”) earned her degrees in Chemistry and Pharmaceutical Technologies in 1988 and Pharmacy in 1989 from the University of Genoa. She carried out her postdoctoral work at the Advanced Biotechnology Center in Genova before becoming deputy head of the Laboratory of Tissue Engineering of Istituto Dermopatico dell’Immacolata (IDI) in Rome and Director of the Regional Research Center on Epithelial Stem Cells in Venice.
Michele De Luca (“MDL“) was born in Savona in 1956. He received his M.D. from the University of Catania and his specialization in endocrinology from the University of Rome. He was a fellow at the National Institutes of Health in Bethesda, USA and a visiting scientist at Harvard Medical School, USA. He was senior investigator at the Laboratory of Stem Cell Differentiation at the National Institute for Cancer Research in Genova, head of the Laboratory of Tissue Engineering at IDI in Rome and scientific director of the Veneto Eye Bank Foundation in Venice.
Currently, they both are full professors of Biochemistry (MDL) and Applied Biology (GP) and coordinators of cell (GP) and gene (MDL) therapy at the Centre for Regenerative Medicine of the University of Modena and Reggio Emilia, Italy. They are co-founders, members of the Board, Scientific Director (MDL), and Director of Research & Development (GP) of Holostem Terapie Avanzate S.r.l., of Modena, Italy.
Epithelial stem cells in cell and gene therapy
Few people today dispute the enormous potential of stem cells for regenerative medicine. Yet, despite an ever-increasing number of reports where stem cells are used to treat various disorders, proven stem-cell therapies remain few and far between. Graziella Pellegrini and Michele De Luca have dedicated most of their scientific activities to the study of epithelial stem cell biology aimed at clinical application in regenerative medicine, where their work has been pivotal.
In 2015, the European Medicines Agency recommended Holoclar, a treatment for moderate to severe limbal stem cell deficiency (“LSCD“) and the first advanced therapy medicinal product containing stem cells, for approval in the European Union. Limbal stem cells are stem cells located in the basal epithelial layer of the cornea. Damage caused by external factors such as chemical or thermal burns, can result in LSCD, leading to visual impairment and blindness. Holoclar, developed by De Luca and Pellegrini, allows full recovery of normal vision and has already cured hundreds of patients. To achieve this, the team first characterized the molecular mechanisms regulating long term proliferative potential, clonal evolution and self-renewal of epithelial stem cells, before developing the treatment, thus bridging basic science and medicine.
Their pioneering work on the use of human epidermal stem cell cultures also allowed hundreds of patients to be treated from their life-threatening burns as well as treating patients affected by vitiligo and piebaldism. Recently, Michele De Luca and Graziella Pellegrini took on the challenge of severe genetic skin disease. This required a combination of stem cell and gene therapy by means of transplantation of autologous epidermal sheets prepared from genetically corrected epidermal stem cells. They reported life-saving regeneration of the entire, fully functional, epidermis on a 7-year-old “Butterfly Child” suffering from a devastating, life-threatening form of Junctional Epidermolysis Bullosa and showed that the human epidermis is sustained only by a limited number of long-lived stem cells detected as holoclones. Based on their rigorous basic research work, De Luca and Pellegrini have developed new stem cell therapies that are now applied worldwide. Their approach to combine transplantation of cultured adult stem cells with gene therapy provides a blueprint that can be applied to the development of other adult stem cells for clinical therapies.