For scientists who study stem cells, the ability to produce hematopoietic stem/progenitor cells (HSPCs) in the lab and then transplant them into patients with blood disorders has been a long-sought-after goal. Recently, the field took a step closer to that milestone when researchers at the Icahn School of Medicine at Mount Sinai identified cells in the embryos and placentas of mice that are actually precursors to HSPCs. Hematopoiesis is the daily process by which the human body generates all of the different types of cells found in the blood and immunological system.
The results of the team’s work—led by Kateri Moore, DVM, Associate Professor of Developmental and Regenerative Biology at the Icahn School of Medicine—were published online in the March 7, 2016, issue of Developmental Cell. The findings could eventually provide new information that will help develop patient-specific HSPCs and more differentiated blood products for cell-replacement therapy.
In a previous study, Dr. Moore’s team successfully reprogrammed mouse skin cells to resemble HSPCs. That reprogramming process was used to inform the group’s latest research, which was focused on maturing precursor cells into hematopoietic stem/progenitor cells that are suitable for transplanting.
“We’ve been able to isolate precursor cells from the placentas of mice and under special culture conditions, mature them in our laboratory to become definitive hematopoietic stem cells,” says Dr. Moore. “That means they could be transplanted into an adult mouse and reconstitute its blood system.”
The work being done by the Mount Sinai team is particularly critical due to the severe shortage of human donors for blood stem cell transplants. The scientists’ ultimate goal—to create a significant new source of transplantable stem cells—could represent a breakthrough treatment for patients with blood diseases such as leukemia, lymphoma, multiple myeloma, and immune deficiency. Indeed, patient-specific HSPCs grown in the laboratory could “provide a whole new blood system of renewable cells that wouldn’t be rejected by the body because it would be from the patient’s own cells,” according to Dr. Moore.
Another member of the research team, Ihor Lemischka, PhD, Professor of Developmental and Regenerative Biology, Pharmacology and Systems Therapeutics, says, “Many investigators have attempted to do what we’ve done in terms of growing HSPCs in vitro, but we’ve been able to build on this process in a way that could move the field forward. The next step is to test these findings in humans.”
Drs. Lemischka and Moore have been investigators in the field of hematopoiesis and stem cell biology for 20 years, moving their lab from Princeton University to Mount Sinai in 2007. As part of The Black Family Stem Cell Institute at Mount Sinai, they are committed to pursuing the therapeutic use of stem cells as a promising area of medicine.
Their lab has enlisted the help of Mount Sinai Innovation Partners, which assists Mount Sinai’s researchers in bringing in outside partners and funding, with an eye toward eventually commercializing its cell-reprogramming process.