The researchers Murdoch Children's Research Institute (MCRI, Melbourne, Australia) have developed a better way to engineer hematopoietic stem cells in the laboratory that are suitable for transplant into humans.
Many blood-related diseases, including bone marrow failure disorders and cancers such as leukemia, ultimately require patients to undergo bone marrow or blood stem cell transplants from healthy donors. However, finding donors with cells that are a sufficient genetic match to the patient's cells is often difficult, and transplant patients can be left vulnerable to a variety of problems, including transplant rejection and graft-versus-host disease.
The use of hematopoietic stem cells derived from a patient's own induced pluripotent stem cells (iPS cells) offers a potential new treatment option without the risk of rejection, but researchers have struggled to produce these cells. In vitro Blood must be collected in sufficient quantity and purity to be reinfused into patients for clinically effective treatments, a challenge that stems in part from distinguishing which cells are mature enough to become the right type to form adult blood cells.
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Recently, researchers have identified specific genetic markers expressed by these desirable cell types. Through specific culture methods, they can induce the expression of these genes in iPS cell populations, allowing the cells to develop into blood stem cell types suitable for transplantation. Utilizing this technology, the team at MCRI developed a large cohort of hematopoietic stem cells that closely resemble those found in human fetuses. The researchers tested these engineered cells in immune-deficient mice and demonstrated that they could develop into functional bone marrow comparable to the levels observed in umbilical cord blood cell transplants. Furthermore, the engineered cells could survive freezing and thawing before infusion, a critical logistical step in the human transplant process.
“Perfecting stem cell methods that mimic the development of normal blood stem cells in the body will help us understand and develop personalized treatments for a range of blood disorders, including leukemia and bone marrow failure,” explained co-author Ed Stanley (MCRI).
The team hopes to move their lab-grown cells into Phase 1 trials within the next five years to test their safety when transplanted into humans.
