Cord blood transplant with a pooled stem cell product (dilanubicel) achieved 96% one-year survival in leukemia patients with no severe graft-versus-host disease.
A new method of using umbilical cord blood to treat blood diseases could make stem cell transplants easier for patients to access, according to a new study.
Cord Blood Stem Cell Therapy Shows 96% One-Year Survival in Cancer Trial
A phase 2 clinical trial of patients undergoing a cord blood transplant plus a stem cell product derived from pooled cord blood units showed that 27 of 28 patients (96%) with leukemias and myelodysplastic syndrome survived at least one year and none of the patients experienced severe acute or chronic graft-versus-host disease, which are common complications of stem cell transplantation.
The findings of the study are published in the Journal of Clinical Oncology (1✔ ✔Trusted Source
Safety and Clinical Outcomes of Pooled Donor, Nonengrafting Expanded Progenitor Cells in Single-Unit Cord Blood Transplantation
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Leukemia Treatment Breakthrough: Nine-Donor Cord Blood Transplant Shows Success
“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 first author of the study and directs the Cord Blood Program at Fred Hutch Cancer Center.
Milano added that many of the 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.
Cord Blood Transplant Offers Hope for Blood Cancer Patients Without Donor Match
Cord blood transplantation can help patients with blood cancers or other blood diseases who need a stem cell transplant but who lack a close donor match, especially multiethnic patients. This is because stem cells in cord blood do not have to be as stringently matched to be safe and effective.
But the number of cells in a single unit of donated cord blood is often too small to treat a patient.
In the clinical trial, Milano took a two-unit approach by treating patients with both a cord blood unit along with a second unit of a stem cell product developed by the study’s senior author Colleen Delaney, MD, MSc, a former Fred Hutch physician-scientist who started the Fred Hutch Program in Cord Blood Research and Transplantation in 2006 and who is now at Seattle Children’s Hospital.
Dilanubicel Stem Cell Therapy Boosts Early Immune Recovery in Cord Blood Transplants
The product, called dilanubicel, combines blood stem cells isolated from six to eight different units of cord blood. Then, in the lab, the stem cells are nurtured and allowed to grow and expand before they are infused into the patient.
The study showed that although these pooled stem cells do not engraft long term, they provided essential early immune support. One week after transplant, patients’ blood consistently showed 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 now is at least one year into remission.
“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.
The clinical trial has now closed. Milano hopes with additional funding that his team can continue to treat more patients.
“Cord blood continues to be an important option for people who need a stem cell transplant, especially those with high-risk disease” Milano said.
Reference:
- Safety and Clinical Outcomes of Pooled Donor, Nonengrafting Expanded Progenitor Cells in Single-Unit Cord Blood Transplantation – (https://ascopubs.org/doi/10.1200/JCO-25-02510)
Source-Eurekalert