Phase II Finds Umbilical Cord Blood Transplant With Pooled Stem Cell Product Shows 96% Survival and No Graft-Versus-Host Disease in Leukemia Patients

Key Points

• A phase 2 trial tested a single cord blood transplant plus dilanubicel, a lab-expanded stem cell product made from pooled cord blood donors, in patients with leukemia and myelodysplastic syndrome.
• At one year, 27 of 28 patients were alive, and no patients developed severe acute or chronic graft-versus-host disease.
• The pooled donor product did not engraft long-term, but the study found that it supported early immune recovery after transplant.

Trial Results

Fred Hutch Cancer Center reported phase 2 results showing that adding dilanubicel to a single-unit umbilical cord blood transplant produced strong one-year outcomes in patients with leukemias and myelodysplastic syndrome. The study, published in the Journal of Clinical Oncology on April 27, found that 27 of 28 patients, or 96%, were alive at one year. None developed severe acute or chronic graft-versus-host disease.

Cord blood transplantation is used for patients with blood cancers and other blood disorders who need a stem cell transplant but do not have a closely matched donor. This can be especially relevant for multiethnic patients, since cord blood stem cells do not need to be matched as closely as other donor sources. However, the practical limitation is that a single donated cord blood unit often does not contain enough cells for treatment.

How the Approach Worked

In this trial, patients received a standard cord blood unit along with dilanubicel, a second stem cell product developed from six to eight different cord blood units. The cells were isolated, expanded in the lab, and then infused into the patient. According to the study, this pooled donor product gave early immune support after transplant, even though it did not persist long term.

One week after transplant, patients consistently showed blood recovery driven by the pooled donor product.

At the end of follow-up, all but one patient were alive and in remission. One patient experienced non-relapse mortality. Another patient relapsed 324 days after transplant and was given another treatment, and is now at least one year into remission.

“This is the first time transplant patients received cells from what amounts to nine different human beings,” said the study’s principal investigator, Filippo Milano, MD, PhD, who is the first author of the study and directs the Cord Blood Program at Fred Hutch Cancer Center.

“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.

“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,” Milano said.

Next Steps

The clinical trial has closed. Milano said he hopes additional funding will allow the team to continue treating more patients, as they have found that cord blood is an important transplant option for patients with high-risk disease, particularly when a close donor match is unavailable.

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