Cord Blood Highlights from the 2025 Tandem Transplant Meeting in Hawaii

Warren Fingrut, MD 

The 2025 Tandem Meetings of the American Society for Transplantation and Cellular Therapy (ASTCT) and Center for International Blood and Marrow Transplant Research (CIBMTR) in Honolulu, Hawaii (February 12-15, 2025) included a spotlight session outlining new frontiers cord-blood therapies.  

First, Dr. Colleen Delaney from Fred Hutchinson Cancer Research Center, USA spoke about leveraging cord blood stem cell expansion for the generation of multiple off-the-shelf immunotherapy products. Dr. Delaney reviewed the cell and gene therapy landscape, highlighting the major role of cord-blood derived therapies, and discussed the promise of these therapies as well as challenges limiting their development and use. Her talk outlined how cord blood is an attractive source of starting material for cell therapies, and shared data demonstrating the safety of infusing cord blood derived-products that are from pooled donors and which are not human leukocyte antigen (HLA)-matched to the recipient. 

Next, Dr. Partow Kebriaei from The University of Texas MD Anderson Cancer Center reviewed cord tissue derived mesenchymal stromal cells (MSCs) for graft-versus-host disease (GVHD) and regenerative medicine. She highlighted that, currently, there are over 1400 active clinical trials registered on ClinicalTrials.gov which are studying clinical applications of MSCs. She shared data outlining that cord blood derived MSCs have unique properties compared with MSCs derived from bone marrow, and showed how cord blood derived MSCs are manufactured at the MD Anderson GMP facility. Dr. Kebriaei then discussed three active trials of cord blood-derived MSCs at MD Anderson, studying their therapeutic roles in steroid-refractory acute GVHD, acute respiratory distress syndrome (ARDS), and cardiac toxicity resulting from anthracycline chemotherapy. 

Dr. Takanori Teshima from Hokkaido University Faculty of Medicine, Sapporo, Japan spoke about optimized cord blood transplantation practices in Japan. He explained that cord blood remains a major source for allogeneic stem cell transplantation in Japan, with 38% of transplant recipients there receiving cord blood grafts. He reviewed Japanese data that has guided optimization of cord blood transplantation conditioning chemotherapy, GVHD prophylaxis, and unit selection. 

The next presentation was delivered by Dr. Guy Sauvageau from the Institute for Research in Immunology and Cancer (IRIC) at University of Montreal, Montreal, Canada. Dr. Sauvageau spoke about the epigenetic rejuvenation of stem cells for better transplants. He outlined the properties of young, balanced hematopoietic stem cells which are present in cord blood grafts, and discussed approaches under investigation to harness these stem cells. He reviewed the UM171 treatment, including results from phase II clinical trials studying this therapy in patients with high-risk acute myeloid leukemia (AML). 

Finally, Dr. Rafet Basar from the laboratory of Dr. Katy Rezvani at The University of Texas MD Anderson Center Center spoke about cord blood derived natural killer (CB-NK) cells for cancer therapy. Dr. Basar shared data highlighting the use of chimeric antigen receptor (CAR) transduced CB-NK cells in lymphoid malignancies. He outlined ongoing clinical trials studying the safety and efficacy of CAR-NK cells targeting CD70 and CD5 in patients with a range of hematologic malignancies which express these antigens.