A multi-institutional research team led by Indiana University School of Medicine scientists has developed a new way to harvest blood stem cells for bone marrow donation that is faster and more effective than the current standard of care—a finding that could make the donation process easier on donors and therefore help increase the number of people willing to donate stem cells for this life-saving procedure.

The research team, including scientists at IU School of Medicine, Massachusetts General Hospital Cancer Center, the Harvard Stem Cell Institute, and Glaxo SmithKline, was led by Louis M. Pelus, PhD, a professor of microbiology and immunology at IU School of Medicine, and Jonathan G. Hoggatt, PhD, a principal faculty member at Harvard Stem Cell Institute, an IU School of Medicine alumnus, and a former graduate student in Dr. Pelus’s lab. Their findings were recently published in the journal Cell.

Blood stem cell transplants can help prolong the lives of people with certain cancers or other serious diseases, such as multiple myeloma and non-Hodgkin's lymphoma, for whom these transplants of healthy adult stem cells can help stave off their diseases.

The current method of donating stem cells requires people to undergo twice daily injections of the drug G-CSF for multiple days, which prompts the blood-forming stem cells to be released from the bone marrow into circulation so they can be collected. But this process can be a significant lifestyle disruption for donors.

In contrast, the new method, as tested in mice, required just one injection of a combination of the signaling molecule GROβ and a bone marrow-stimulating drug called AMD3100 (Plerixafor). Dr. Pelus, senior author on the paper, said the study builds on research he started while working as a lead scientist at SmithKline Beecham, now known as Glaxo SmithKline.

“This new stem cell collection method is the culmination of 25 years of my work from laboratory bench to bedside, back to bench,” Dr. Pelus writes in a blog post about the discovery. “My lab team started by purifying and cloning a molecule that was induced by a previously known hematopoiesis regulator. The new molecule was found to be synergistic for regulating hematopoiesis, but unexpectedly also resulted in mobilization.”

Furthermore, the combination of that molecule, GROβ, and AMD3100 seems to result in not only the release of just as many blood-forming stem cells into the bloodstream as G-CSF, but it also resulted in higher-quality stem cells that were more easily transplanted and accepted into the mice.

“If we can do the whole blood stem cell donation process with one combination injection, then that makes it a lot easier for the process to happen, and it makes it a lot more enticing for normal donors to donate for a transplant for someone else,” Dr. Pelus said.

Dr. Pelus said the next step for this research will likely be clinical trials in humans to test the safety and effectiveness of this new method of stem cell donation.

This research was funded by the National Institutes of Health, the Massachusetts Life Sciences Center and Glaxo SmithKline.