UW researchers teaming up to study blood disorders

Two researchers from the UW School of Medicine and Public Health are teaming up to better understand how a certain gene is related to leukemia and myelodysplastic syndrome.

Myelodysplastic syndrome, or MDS, is a disorder that keeps the body’s bone marrow from producing healthy blood cells.

For many patients, the disease can remain in a stable, relatively minor form for years. For others, it can progress aggressively to leukemia and then death, according to Inga Hofmann, an assistant professor of pediatrics at UW.

She and Emery Bresnick, a professor of cell and regenerative biology and director of the UW-Madison Blood Research Program, will explore the link between these diseases and regulation of the GATA-2 gene.

According to a release, it’s already known that this gene is involved with the development of MDS, but the exact nature of that relationship is less clear.

“This work is distinct from ongoing efforts in the field and has potential to transform our understanding of MDS and leukemia and create opportunities to develop new treatments,” said Bresnick, who performs research on blood-cell development at the Wisconsin Institutes for Medical Research.

Hofmann, a pediatric hematologist at UW Health, was the founding principal investigator of the Pediatric MDS and Bone Marrow Failure Registry at Boston Children’s Hospital. This resource she created will play a key role in this MDS study, as it contains samples of blood cells and bone marrow from patients all over the country.

She and Bresnick will use some of these specimens in their joint effort to determine if a mutation of the GATA-2 gene impacts various characteristics of MDS.

According to the American Cancer Society, a stem cell transplant is the only treatment which has been found to cure some MDS cases. Patients are first treated with powerful chemotherapy and radiation to kill the abnormal bone marrow cells, and then receive healthy stem cells which can produce healthy blood.

But a transplant isn’t always a perfect solution. According to the UW School of Medicine and Public Health, the risk of dangerous toxicity is high, and some patients redevelop the same issue.

That’s why this study is aiming to move toward individualized patient therapy — otherwise known as precision medicine — so that each patient with MDS can get unique treatment that takes into account things like genetics, environment and lifestyle.

The work will be supported by a two-year, $400,000 grant from the Edward P. Evans Foundation, which funds medical research relating to MDS.

–By Alex Moe