Maria Ester Bernardo
EBMT CIC 813
Milano, Italy"I am a pediatrician and physician scientist working in the fields of HSCT and HSC-gene therapy with a view on both clinical activity and experimental/translational research. Given my scientific and human aptitudes, I see the Call for IEWP Chair as a unique opportunity to contribute to the central activities of EBMT.
If elected I intend to: i) drive innovative clinical trials for the cure of Inborn Errors, ii) guide the development of impactful research on transplantation (from basic to observational studies), iii) leverage the fruitful collaboration within IEWP members and with other WPs to further advance the EBMT stimulating ecosystem."
My Manifesto
I am a pediatrician and transplant physician who, during my education and professional path, always combined clinical activity with experimental and translational research.
After getting my Residency in Pediatrics in 2005 at the University of Pavia, Italy, I obtained my PhD degree in Experimental Hematology in 2010 at the University of Leiden, The Netherlands. I currently hold the roles of Responsible for the Pediatric Transplant Unit at the San Raffaele Hospital, Clinical Coordinator of the Pediatric Clinical Research Unit at San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget) and Associate Professor of Pediatrics at the San Raffaele Vita Salute University Medical School. In addition, I am a Group Leader at SR-Tiget where I manage my own experimental research unit.
My clinical focus has always revolved around hematopoietic stem cell transplantation (HSCT) in pediatric patients. Since my recruitment to SR-Tiget in 2014 I applied my clinical and pre-clinical expertise to hematopoietic stem cell-gene therapy (HSC-GT) for rare genetic defects, being an active Investigator in several institutional clinical trials. In particular, my contribution has been essential to the successful clinical implementation of HSC-GT for Mucopolysaccharidosis Type I, Hurler variant (MPSIH). The preliminary results of this study highlight the substantial therapeutic potential of this treatment strategy for MPSIH patients and have been published in the New Engl. J. Med in 2021. On the experimental research side, I have been actively pursuing the study of mesenchymal stromal cells from their biological and functional characterization to their application as therapeutic support in the context of HSCT and HSC-GT.
I have been prolific in terms of publications having authored >120 peer-reviewed scientific papers and I have been awarded a number of grants as PI with government (Young Researcher, Italian Ministry of Health, National University Grant PRINN PNRR) and charities (Italian Association for Cancer Research; Telethon Foundation) funding. Moreover, I have been actively engaged with scientific societies such as the European Hematology Association (EHA) where I played the role of Councilor of the Board, Vice-Chair of the Specialized Working Group in Pediatrics, as well as member of several scientific Committees.
I am well trained for GCP and regulatory aspects of clinical trials, particularly on gene and cell therapy for rare diseases, and I am aware of the challenges of establishing collaborative studies in our field and potential solutions to overcome them.
Being a physician scientist with human and scientific aptitudes and given my open collaborative approach in scientific endeavors, I see the Call for IEWP Chair as a unique opportunity to contribute to the core activities of our Society. I always enjoyed the collaborative and stimulating scientific environment of the IEWP whose activities I have been actively involved since the last 10 years by contributing to the preparation of IEWP annual conferences programs and co-organizing the IEWP annual meeting in Milan in 2021, as well as by taking part to the IEWP scientific committee.
If elected as Chairman I intend to:
- Drive innovative clinical trials for the cure of Inborn Errors and act to pave the way towards the development of new advanced therapy medicinal products.
- Guide the development of impactful translational research within the WP
- Allow space for young and emerging investigators in our field and promote their education
- Foster even more strongly the collaboration within the WP members since this has been proven to be very successful in the past years under the guidance of the previous WP Chairmen. Stimulate the collaboration with other WPs, as well as with other Scientific Societies as recently personally experienced with the ESID EHA SIOPE Focused Symposium which will be held in Vienna in November 2025.
- Promote retrospective analysis of HSCT in Inborn Errors under the EBMT umbrella, comparing HSCT with alternative treatments, refining conditioning regimens and other aspects of transplantation.
Thanks to my positive, inclusive and constructive approach I believe I could represent a catalyst for energies and further development of the lively ecosystem of the WP.
In conclusion, I would be extremely proud to be the Chairman of the IEWP for the next 4 years and I will do all my best to continue the line of achieving relevant and challenging scientific outputs, supported by the valuable members of the WP.
Note: The online voting period will run from Tuesday, 18 March 2025, to Monday, 31 March 2025. Principal Investigators (PI's) from active EBMT member centres will be able to cast their vote online for their preferred candidate within the open positions. The elections results will be announced live at the General Assembly Meeting on Tuesday, 1 April (07:45 am CET), during the 51st Annual Meeting of the EBMT. Instructions on how to vote will be sent out to PI's closer to the voting start date.