Words of the president

Hematopoietic stem cell transplantation (HSCT) remains the only curative treatment for many malignant and non-malignant hematological diseases. Over the past several decades, outcomes have steadily improved, and indications for HSCT have become increasingly refined.
In the 1970s, HSCT was typically limited to patients under 30 years of age who had an HLA-identical sibling donor. Today, the landscape has changed considerably. The number of transplants has risen markedly, driven by the use of alternative graft sources such as unrelated adult donors, umbilical cord blood, and, more recently, haploidentical family donors. These advances have enabled transplantation for patients without an HLA-identical family donor. Furthermore, optimized donor selection criteria, improved post-transplant supportive care, and the development of reduced-intensity conditioning regimens have all contributed to lowering transplant-related toxicity. As a result, both the range of indications and the acceptable age limits for transplant recipients have expanded. To date, more than two million patients worldwide have undergone HSCT.
International registries were first established to identify adult volunteer donors who were HLA-identical to recipients. Later, it was discovered that partially HLA-matched donors could also be used, a major breakthrough that greatly expanded the pool of available donors. Umbilical cord blood collected at birth became a valuable graft source for transplantation from partially HLA-matched donors. Because cord blood cells are immunologically immature, they provoke fewer immune reactions than adult stem cells, making them an excellent option in this context. Another significant advance was the use of mobilized peripheral blood stem cells from partially HLA-matched family members, a procedure known as haploidentical transplantation.
Currently, over 35 million unrelated adult donors and more than 783,000 cord blood units are registered worldwide, making donor availability less of a limiting factor for most patients.
While transplant outcomes continue to improve, substantial challenges remain. Post-transplant mortality is still significant, primarily due to graft rejection, infections, graft-versus-host disease (GVHD), and relapse in patients with malignant diseases.
HSCT continues to benefit from advances in biomedical science and technology, including genomics, molecular biology, immunology, imaging, and data analysis powered by artificial intelligence (AI). New strategies to enhance transplant outcomes are under development, involving gene therapy, monoclonal antibodies, novel immunosuppressive agents, and targeted therapies against tumor-specific markers.
Among the most recent breakthroughs is gene therapy, with promising early results. These therapies target hereditary diseases such as immune deficiencies and red blood cell disorders including thalassemia and sickle cell anemia. Two main gene therapy approaches are in use: inserting corrective genes into patients’ cells using lentiviral vectors, and CRISPR-Cas9 gene editing – a revolutionary technology honored with the 2020 Nobel Prize in Medicine. These methods aim to cure hereditary disorders by using patients’ own genetically corrected cells, thereby eliminating the need for allogeneic transplantation.
Immunotherapy has also advanced significantly. The development of monoclonal antibodies has enabled targeted attacks on tumor cells and infectious agents, including the Epstein-Barr virus (EBV), which causes infectious mononucleosis and is linked to certain lymphomas.
Cellular immunotherapy is rapidly evolving, particularly through the use of laboratory-modified immune cells engineered to recognize and destroy tumor or infectious antigens. Notably, chimeric antigen receptor T-cell (CAR-T) therapy involves modifying lymphocytes to target and eliminate specific cancer cells, such as B-cell leukemias and lymphomas. This approach has produced remarkable remissions in patients resistant to conventional chemotherapy.
Other immunotherapeutic modalities are under investigation for a broad range of leukemias and cancers, with similar technologies also being explored for certain infectious diseases.
Molecular genomics has facilitated the mapping of the human genome and the identification of cancer-associated mutations. This has enabled the development of highly specific targeted therapies that eradicate malignant cells with greater precision and fewer side effects than traditional chemotherapy. A landmark example is the treatment of chronic myelogenous leukemia (CML) through inhibition of the BCR-ABL fusion protein, which results from a chromosomal translocation. This targeted therapy has allowed many patients to achieve long-term remission, often eliminating the need for bone marrow transplantation. Numerous additional targeted treatments are currently in clinical trials.
These advances result from close collaboration between basic science and translational research, leading to the development and commercialization of innovative therapies. The establishment of clinical research registries and the integration of AI have greatly enhanced outcome monitoring and data analysis.
The 21st century has opened a new era of diagnostic and therapeutic innovation. Yet, it is important to remember that these achievements are all built on the foundation of decades of dedicated research. Discoveries in stem cell biology have expanded our therapeutic arsenal and opened the door to transformative treatments for the future.