Florence, Italy — March 30, 2025 – The 51st Annual Meeting of the European Society for Blood and Marrow Transplantation (EBMT) commenced in Florence with a compelling and multifaceted opening ceremony that captured the essence of what the EBMT represents. Far beyond a traditional welcome, the evening unfolded as a rich tapestry of scientific achievement, heartfelt remembrance, and forward-looking vision.
Set against the backdrop of one of Europe’s most historic cities, the nearly two-hour ceremony offered a unique convergence of cutting-edge research, emotional tributes to trailblazers, and impassioned calls for equity and global access to advanced therapies. It was a stirring reflection of EBMT’s unwavering commitment to patient-centered care, scientific excellence, and international collaboration, setting the stage for what promises to be a landmark week in the field of transplantation and cellular therapy.
Setting the Stage: A United Vision
The 51st Annual Meeting of the EBMT began with a warm welcome from Dr. Anna Sureda (Spain), followed by the opening address from Dr. Fabio Ciceri (Italy), who set the tone for what promises to be a landmark year for the organization. Speaking from the stage in Florence, Ciceri reflected on the enduring strength of the EBMT community, emphasizing its resilience in the face of global challenges—geopolitical tensions, economic uncertainties, and the fast-paced evolution of medical science.
He introduced the guiding themes for EBMT 2025: fostering the multidisciplinary nature of the society, reinforcing the basic biology–translational research axis, and empowering the next generation of young investigators. These principles will shape not only the scientific program in Florence but also the future of hematopoietic cell transplantation and cellular therapy. Ciceri also presented the extensive roster of the Scientific Committee, showcasing a wide range of expertise across Europe—an embodiment of the collaborative spirit he described as essential to driving progress forward.
“We are so glad you are here with us,” read the message welcoming attendees both in person and virtually, underscoring the dual ambition of the meeting: scientific excellence and inclusive participation.
Patient Advocacy: A Shared Responsibility
One of the evening’s first major addresses came from Natacha Bolaños, who spoke on behalf of the EBMT Patient Advocacy Committee. She urged all professionals to attend two pivotal sessions on equitable access to CAR-T cell therapies and standardized transplant protocols for autoimmune diseases. These, she stressed, were not merely patient-centered events but essential engagements for physicians, researchers, and decision-makers alike. Her address reminded attendees that the responsibility for equity lies with everyone—not just those directly affected.
Reflections from the Nursing Frontlines
Michelle Kenyon, President of the EBMT Nurses Group, delivered one of the most emotional and deeply resonant speeches of the evening. As she neared the end of her presidency, Kenyon welcomed the audience to Florence by acknowledging both the rich history of the EBMT and the rapid evolution of the healthcare landscape in recent years.
She spoke candidly about the ongoing impacts of the COVID-19 pandemic—educational setbacks, staff shortages, and increased patient complexity—while highlighting how the Nurses Group had responded by building inclusion and removing barriers to education. Her moving speech was grounded in personal narrative, including the story of a transplant recipient diagnosed with acute myeloid leukemia (AML) at 63, whose anonymous donor gave him a second chance at life. The letter exchanged between the two served as a reminder of the deeply human relationships forged through transplantation.
Kenyon closed by acknowledging the transformative power of nursing leadership, the resilience of caregivers, and the moral imperative to “see the person before the patient.”
Celebrating Dedication: EBMT Honorary Membership Awards
The ceremony continued with the announcement of this year’s EBMT Honorary Members, celebrating four professionals whose lifetime contributions have shaped the field and the society.
Professor Catherine Cordonnier was recognized for her leadership in infectious disease management and for founding EBMT’s first training course on infections, which continues to this day. She reflected on the importance of combining hematology and infectious disease knowledge to deliver safe and effective care.
Dr. Myriam Labopin, unable to attend in person, shared a letter read on her behalf by Dr. Mohamed Montini. In it, she described EBMT as a sanctuary for creativity and collaboration, noting that scientific innovation arises not from strict bureaucracy but from open-minded teamwork. Her work as a senior statistician over three decades has been foundational to EBMT’s data-driven success.
Professor Norbert Schmitz was celebrated for his pioneering contributions to the treatment of Hodgkin’s lymphoma. His work helped establish autologous stem cell transplantation as a standard of care, and he served numerous leadership roles within the organization.
Professor Gérard Socié, though absent, was also honored for his extensive research into graft-versus-host disease (GVHD) and transplant complications. His leadership in EBMT’s scientific and educational activities has had a lasting impact on multiple working parties and task forces.
Each of these individuals was described not only as a leader in science but also as a mentor and example of humility and service.
A Legacy Remembered: Prof. Riccardo Saccardi
The ceremony shifted into a more solemn and reflective tone with a tribute to the late Professor Riccardo Saccardi. Introduced by Dr. Fabio Ciceri, the tribute highlighted Saccardi’s instrumental role in establishing hematopoietic stem cell transplantation for autoimmune diseases. He was remembered as a man of principle and quiet influence, someone who championed standardization in graft processing, led the JC accreditation effort, and helped benchmark registry data in autoimmune disease settings.
Professor Paolo Muraro followed with a personal reflection on their collaboration, particularly in the treatment of multiple sclerosis (MS). Saccardi was a founding force behind efforts to integrate HSCT into the treatment of MS, co-authoring critical consensus guidelines alongside neurology societies such as ECTRIMS. His work enabled lasting change, not only in Italy but across international borders.
Tribute Lecture: Advancing HSCT in Multiple Sclerosis
In a powerful tribute lecture, Professor Gianluigi Mancardi reflected on the remarkable evolution of hematopoietic stem cell transplantation (HSCT) for multiple sclerosis (MS), now widely recognized as a transformative therapy for relapsing-remitting MS (RRMS). Once viewed as experimental, HSCT is now included in national guidelines and endorsed by multiple scientific societies as a standard of care.
Prof. Mancardi began by honoring the late Professor Riccardo Saccardi, whose calm leadership and vision for interdisciplinary collaboration were instrumental in bridging hematology and neurology. As a founder of the GITMO-Neuro Intergroup, Saccardi helped neurologists overcome uncertainty toward cell therapy and laid the groundwork for its adoption across Italy and Europe. He then presented robust long-term data showing that, among RRMS patients, disability progression-free survival reached 85.5% at five years and 71.3% at ten years. In progressive MS, survival rates remained notable—71.0% at five years and 57.2% at ten years—demonstrating the therapy’s broad efficacy.
Comparative studies further support HSCT’s superiority. In a phase II trial versus mitoxantrone, HSCT significantly reduced new T2 MRI lesions over four years, with a rate ratio of just 0.21 and consistent significance across all timepoints (p < 0.01). These findings confirm HSCT’s ability to halt both clinical and subclinical disease activity. Equally important, safety has improved substantially. Once associated with high treatment-related mortality, HSCT is now delivered with a mortality rate of approximately 1.3%, thanks to better patient selection and supportive care. International momentum continues through four major Phase III trials—RAM-MS, BEAT-MS, STAR-MS, and NET-MS—comparing HSCT to high-efficacy disease-modifying therapies like alemtuzumab and ocrelizumab, with outcomes including NEDA and relapse-free survival.
Closing his lecture, Prof. Mancardi stressed that HSCT is no longer an option of last resort. “We are now in a position to offer patients a therapy that not only stabilizes MS but may reverse early disability,” he said. With mounting evidence and unified clinical support, HSCT is positioned to become a global standard in treating severe MS.
Gene Therapy Breakthroughs and Equity Challenges
In his keynote lecture, Prof. Franco Locatelli offered a comprehensive overview of the rapid evolution of gene therapy for β-thalassemia and sickle cell disease, emphasizing both its clinical promise and the ethical urgency of ensuring access.
He described how lentiviral gene addition has revolutionized treatment for patients with transfusion-dependent β-thalassemia, particularly those with the β⁰/β⁰ genotype—one of the most severe forms of the disease. In recent trials, over 90% of these patients achieved sustained transfusion independence, a milestone that until recently seemed out of reach. These outcomes not only reflect the success of gene addition strategies but also demonstrate the increasing maturity of personalized cellular approaches in hematology.
Turning to sickle cell disease, Prof. Locatelli highlighted the transformative impact of gene editing using CRISPR-Cas9 technology to inactivate BCL11A. Among patients treated in clinical studies, 95% experienced complete resolution of vaso-occlusive crises—the painful and often life-threatening hallmark of the disease. These results, observed over a median follow-up of 24 months, suggest a profound shift in the therapeutic trajectory for sickle cell disease, potentially allowing patients to live free of both symptoms and hospitalizations.
Despite these remarkable outcomes, Locatelli raised a note of caution. He pointed to the troubling trend of pharmaceutical companies withdrawing advanced therapy medicinal products (ATMPs) from the European market due to regulatory and economic challenges. He stressed that such actions threaten to widen the global equity gap in access to curative therapies. “The science is mature,” he stated, “but access must catch up.” He urged the EBMT community and academic institutions to step forward where industry steps back, forming international consortia that can sustain development, manufacturing, and delivery of gene therapies—especially for patients in low-resource settings.
Future Frontiers: Genetic Engineering with Precision
Professor Luigi Naldini, a leading figure in gene therapy, concluded the opening ceremony with a forward-looking lecture on the progress and future of hematopoietic stem cell (HSC) gene therapy. He shared long-term follow-up data from over 400 patients treated with lentiviral vectors, emphasizing the durability and safety of these approaches across multiple monogenic diseases.
A key highlight of his presentation was the demonstration of stable long-term polyclonal reconstitution in patients years after treatment. Tracking vector copy numbers and clonal contributions over time, his data showed that genetically modified HSCs not only persist but maintain diverse lineage contributions—a sign of both safety and regenerative integrity. This observation, he noted, affirms the capacity of lentivirally corrected HSCs to support lifelong hematopoiesis without signs of clonal dominance or malignant transformation.
Yet, he addressed safety with scientific rigor. In studies involving X-linked adrenoleukodystrophy (ALD), a small proportion of patients developed MDS/AML. These events were associated with vector insertions near genes such as MECOM and PRDM16, highlighting the importance of vector design and integration site profiling. While rare, they underscore the necessity of continuous long-term monitoring and safer vector evolution.
Looking ahead, Professor Naldini described the rise of precise genome editing tools—CRISPR-Cas9, base editors, and prime editors—which allow correction of disease-causing mutations at the nucleotide level. Still, he cautioned that these methods come with risks such as chromosomal translocations and large deletions. To preserve HSC viability, his team is exploring strategies like transient p53 inhibition (e.g., GSE56) and advanced delivery methods to limit genotoxic stress and enhance editing outcomes.
He also discussed in vivo gene editing platforms under investigation in neonatal models and stressed the need to move away from traditional chemotherapy-based conditioning toward safer antibody-based regimens.
Closing his lecture, Professor Naldini delivered a clear call to action:
“We must now move from treating rare diseases to globalizing these therapies,” he stated. “Access is not a by-product—it’s the purpose.”
Closing Remarks: A Ceremony of Significance
Dr. Fabio Ciceri returned for closing remarks, expressing gratitude to all speakers, organizers, and attendees. He described the ceremony as “an emotional journey” that combined the best of science with the best of humanity. He invited attendees to continue discussions at the welcome reception held at the historic Arsenale Basilica and encouraged them to carry the spirit of reflection and collaboration throughout the conference.
Conclusion
The EBMT 2025 Opening Ceremony was a momentous beginning to a week that promises groundbreaking science, dynamic collaboration, and patient-centered innovation. Through tributes to pioneers, reflections on clinical progress, and inspiring calls for equity, the evening underscored EBMT’s enduring role as both a scientific leader and a compassionate community. In Florence a city steeped in history and rebirth the EBMT charted its course for a future defined by courage, inclusion, and excellence.
