Researchers from the Cincinnati Children’s Hospital Medical Center and the University of Cincinnati (UC), led by Jose Cancelas, MD, PhD, and David Williams, MD, found that a group of proteins known as the RAC GTPase family plays a significant role in regulating the location and movement of stem cells in bone marrow.

The researchers discovered that by inhibiting RAC GTPase activity in mice, they were able to “instruct” stem cells to move from their home in the bone marrow and into the blood stream, where they can easily be collected. They achieved this using a drug, developed by Cincinnati Children’s faculty member Yi Zheng, PhD, known as NSC23766.

Their findings are reported in the Aug. 6 edition of the scientific journal Nature Medicine.

Dr. Cancelas, lead author of the report, is director of research at UC’s Hoxworth Blood Center. Dr. Williams, the senior author, heads experimental hematology at Cincinnati Children’s.

“Our findings demonstrate that RAC GTPase proteins are essential for injected stem cells to migrate into the correct location in the bone marrow,” said Dr. Williams.

Researching the location of and the factors involved in stem cell regeneration is important to the development of new therapeutic tools in stem cell therapy, said Dr. Cancelas.

Adult stem cell transplantation, or bone marrow transplantation, is used during the treatment of cancer and genetic blood diseases, such as sickle cell anemia, to restore blood cell formation in bone marrow that has been damaged by high-dose chemotherapy or radiation therapy. It has also shown promise in animal studies for possible treatment of organ damage, such as that seen in heart disease and degenerative diseases like Parkinson’s.

The research team also included Andrew Lee, Rethinasamy Prabhakar, PhD, and Keith Stringer, MD, PhD. Their work was supported by grants from the National Institutes of Health and the National Blood Foundation.

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