Pooled Cord Blood Stem Cell Product Achieves 96% Survival Rate in Blood Cancer Transplant Trial
Key Insights
A phase 2 clinical trial demonstrated that 27 of 28 patients (96%) with leukemias (search) and myelodysplastic syndrome (search) survived at least one year after receiving a novel cord blood transplant approach.
The innovative treatment combines traditional cord blood transplantation with dilanubicel, a pooled stem cell product derived from six to eight different cord blood units.
None of the patients experienced severe acute or chronic graft-versus-host disease (search), addressing a major complication of stem cell transplantation.
A groundbreaking phase 2 clinical trial has demonstrated remarkable survival outcomes for blood cancer patients using a novel approach to cord blood transplantation. The study, published in the Journal of Clinical Oncology on April 27, 2026, showed that 27 of 28 patients (96%) with leukemias (search) and myelodysplastic syndrome (search) survived at least one year, with none experiencing severe acute or chronic graft-versus-host disease (search).
The innovative treatment represents the first time transplant patients have received cells from what amounts to nine different human beings, according to principal investigator Filippo Milano, MD, PhD, who directs the Cord Blood Program at Fred Hutch Cancer Center (search).
Novel Two-Unit Transplant Approach
The clinical trial addressed a fundamental limitation of cord blood transplantation: the insufficient number of cells in a single unit of donated cord blood to effectively treat patients. Milano's team implemented a two-unit approach, combining a traditional cord blood unit with a second unit containing dilanubicel, a specialized stem cell product.
Dilanubicel, developed by senior author Colleen Delaney, MD, MSc, combines blood stem cells isolated from six to eight different units of cord blood. These stem cells are nurtured and expanded in laboratory conditions before being infused into patients. Delaney, a former Fred Hutch physician-scientist who established the Fred Hutch Program in Cord Blood Research and Transplantation in 2006, is now at Seattle Children's Hospital.
Exceptional Clinical Outcomes
The trial results exceeded expectations across multiple measures. At the end of follow-up, all but one patient remained alive and in remission. One patient experienced non-relapse mortality, while another patient who relapsed 324 days after transplant received additional treatment and achieved at least one year of remission.
Milano noted that many patients are now approaching two years post-transplant with continued strong outcomes. "I am grateful for the boldness and courage from our patients and clinical care team to move the transplant field forward with this new approach," said Milano, who holds the Endowed Chair of Cord Blood Research at Fred Hutch.
Mechanism of Action
The study revealed that while the pooled stem cells do not engraft long-term, they provide essential early immune support. One week after transplant, patients' blood consistently showed recovery driven by the pooled donor product. Milano explained that "the cells from the pooled donor stem cell product did not remain long term, but they all helped the matched cord blood donor establish a new, healthy immune system in the patient."
Addressing Transplant Accessibility
Cord blood transplantation serves as a crucial option for patients with blood cancers (search) or other blood diseases who need a stem cell transplant but lack a close donor match, particularly benefiting multiethnic patients. This accessibility stems from the fact that stem cells in cord blood do not require as stringent matching to be safe and effective compared to other transplant sources.
"Cord blood continues to be an important option for people who need a stem cell transplant, especially those with high-risk disease," Milano stated. The clinical trial has now closed, though Milano hopes to secure additional funding to treat more patients with this approach.
Research Support and Future Directions
The research received support from multiple sources, including the George & Fay Young Foundation, Robert J. Kleberg, Jr. and Helen C. Kleberg Foundation, and Cord Blood Protocol Support Funds. Additional funding came from a SHRM Foundation grant designated to the cord blood program by a former patient. Deverra Therapeutics (search) provided the dilanubicel product for the study.