The Clinical Research Division's Dr. Colleen Delaney has received a $12.6 million award from the National Heart, Lung and Blood Institute to test her pioneering method for expanded umbilical cord blood transplants in a clinical trial of 160 patients.
This phase 2 multicenter trial—headquartered at the Hutchinson Center—builds on the success of Delaney's original studies that have demonstrated the technique’s power in 20 patients.
Bone marrow transplants from adult donors can be lifesaving for patients with blood cancers when a match is available, but nearly 40 percent of patients who need a transplant, or 16,000 per year, are unable to find a match from a family member or the donor registry. For most ethnic minorities the chance of finding a donor is even lower.
This is where umbilical cord blood has the potential to shine. Because the immune system found in an umbilical cord after a baby is delivered is less developed than adult stem cells, the cord blood stem cells do not need to be as closely matched to the patient.
With less stringent matching requirements, cord blood donors can be found for nearly all patients in need of a stem cell donor. But a single unit of cord blood contains so few stem cells that the patient would have a long period before his or her blood cells and immune system are fully repopulated, increasing the chances of a dangerous infection. Delaney, continuing work started in Dr. Irwin Bernstein's laboratory, optimized methods to expand cord blood stem cells in the laboratory prior to transplant.
The first trial using expanded cells required that the cord blood unit used to generate the expanded cells be at least partially matched to the patient and the process of cell expansion had to occur while the patient was undergoing chemotherapy and radiation treatments needed prior to the transplant. While this approach demonstrated that the median time to blood count recovery was cut in half in patients receiving the expanded cells, the process is logistically difficult and does not easily allow patients around the country access to this approach.
To address these challenges, Delaney and her group developed an approach to cord blood stem cell transplantation in which matching is not at all required. This is possible because the stem cells are isolated prior to expansion and the final expanded cord blood product does not contain the original immune system of the donor.
This approach allowed Delaney to develop a supply of cord blood units that are pre-expanded in the Center’s Shared Resources Cell Processing Facility and then frozen for future use as a universal donor. This "off-the-shelf" approach means the universal donor units can be made available for all patients, regardless of matching, and shipped at the time of transplant without affecting the timing of chemotherapy or radiation treatment.
Delaney’s new trial will compare patients undergoing cord blood transplants with or without the expanded, off-the-shelf products. Her study will ask whether patients who receive expanded cord blood cell transplants repopulate their immune and blood cells more quickly and whether the universal donor products provide clinical benefit to the patient in reducing infections, toxicity and length of their hospital stay.