Gene discovery could revolutionize the treatment of blood disorders

Investigators from the University’s School of Medicine have found that a protein called NF-Ya activates several genes known to regulate the development of hematopoietic stem cells (HSC), or blood-producing stem cells, in bone marrow.

Leukemia and related diseases are created by replicating two HCSs cells and no differentiated cells at the point of division of an original HCS cell. Meanwhile, conditions like bone-marrow failure occur because the body runs out of HSCs because the second scenario of two differentiated cells and no HCSs occur at division. The identification of the gene master that regulates the outcome of the division process brings the possibility of preventing unwanted divisions one step closer.

According to the researchers, knowing the details of this pathway may one day lead to new treatments for such blood diseases as leukemia, as well as a better understanding of how HSCs work in the context of bone-marrow and peripheral-stem-cell transplantation.

“We want to figure out how this process is normally regulated in the body, so that we can learn to control it for therapeutic purposes,” said lead investigator Dr Stephen Emerson, associate director of Clinical Research for Penn’s Abramson Cancer Center and chief of the division of Hematology-Oncology.

“For some clinical purposes, we might want to shift the balance so that we can grow more stem cells, for those who need them. Conversely, for patients in whom this process has gone awry, such as acute leukemia, we might block the regulatory gene to shift the balance of self-renewal versus differentiation so that all the immature, leukemic cells differentiate and die.”