09 January 2019

Scientists have developed a new technique which they hope will improve the process of stem cell transplants using umbilical cord blood, it has been announced.

A team led by Prof Mitchell Horwitz at Duke University Medical Centre, North Carolina, USA, applied a technique known as NiCord. It involves using nicotinamide to expand the stem cells from a single unit of cord blood over three weeks ex vivo. The expanded stem cells appeared to be safe once transplanted, and superior to standard cord blood units at restoring blood count, the team report in the Journal of Clinical Oncology.

The new technique was tested at 11 clinical trial sites in the US on 36 patients with various types of blood cancer. Most patients (94%) had successful engraftment in six weeks. The transplant failed in two patients due to viral infections.

Blood count recovery happened much earlier among patients receiving NiCord, compared to a historical cohort of patients receiving standard umbilical cord blood units. Neutrophil recovery took an average of 11.5 days with NiCord, versus 21 days for standard cord blood, and platelet recovery took 34 days versus 46 days.

This expansion technology "represents an opportunity to improve the results for adult patients", says Prof Horwitz, adding: "This study shows that a single unit of this product appears to be delivered safely to patients around the world."

Testing on a larger number of patients is already under way in a phase III trial directly comparing NiCord with standard cord blood transplantation.

Source: Horwitz, M.E., Wease, S., Blackwell, B., Valcarcel, D., Frassoni, F., Boelens, J.J., Nierkens, S., Jagasia, M., Wagner, J.E., Kuball, J., Koh, L.P., Majhail, N.S., Stiff, P.J., Hanna, R., Hwang, W.Y.K., Kurtzberg, J., Cilloni, D., Freedman, L.S., Montesinos, P., Sanz, G. (2018) “Phase I/II Study of Stem-Cell Transplantation Using a Single Cord Blood Unit Expanded Ex Vivo With Nicotinamide”, Journal of Clinical Oncology, available at doi: 10.1200/JCO.18.00053


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