New myelodysplastic syndrome research now underway at the University of Minnesota

The largest study of its kind to identify environmental, lifestyle and genetic factors that may increase a person’s risk of developing myelodysplastic syndrome (MDS) is currently underway at the Masonic Cancer Center, University of Minnesota. Leading the study is Julie Ross, PhD, professor and director of the University of Minnesota Medical School’s Division of Pediatric Epidemiology and Clinical Research and member of the Masonic Cancer Center Carcinogenesis and Chemoprevention and Prevention and Etiology research programs.

With a $2.5 million grant from the National Cancer Institute, researchers are comparing data from 700 patients who have MDS and from 700 without, looking for clues as to why some patients with MDS develop leukemia and some do not. Approximately one-third of patients with MDS eventually develop acute myeloid leukemia (AML).

MDS is a group of bone marrow stem-cell disorders that affects up to 50 per 100,000 people over the age of 70 in the United States. Minnesota has one of the highest MDS rates in the country.

Risk factors for MDS include chemotherapy or radiation therapy; exposure to certain chemicals, including tobacco smoke, pesticides and industrial chemicals; and exposure to heavy metals, such as lead and mercury. Diagnosis often involves excluding other diseases, as MDS manifests itself with fatigue, anemia, recurrent infections and bleeding that won’t stop. MDS can also lead to an increased risk of cancer, especially leukemia. A bone marrow aspiration and biopsy are often used for a definitive diagnosis.

The only known cure for MDS is a bone marrow stem cell transplant during which defective cells are destroyed and replaced with healthy, donated cells. Due to the older age at which MDS is often diagnosed and the intensity of performing a stem cell transplant, however, this treatment is not appropriate for many MDS patients. The goals of treatment then focus on preventing and treating complications, improving the quality of life and prolonging survival. Current non-curative treatments for MDS include the following:

  • Blood transfusions to replace red, white or platelet cells
  • Medications to increase the number of blood cells, stimulate existing cells to mature, suppress a patient’s immune system and/or lessen the need for transfusion

In March 2012, as an expansion of this work on MDS in Minnesota, Ross and Masonic Cancer Center physician Jeffrey Miller, MD, along with Mayo physicians Mark Litzow, MD and Aref Al-Kali, MD, (as four co-principal investigators) were awarded a $1.35 million grant from the Minnesota Partnership to tie together some new avenues of research at both institutions. The MDS partnership grant will fund pilot projects to:

  • Identify genes that may be important in both MDS development and progression to leukemia. If certain genes can be identified, it may help to develop predictors that can signal who is most at risk of developing leukemia.
  • Test new drugs that contain MDS antibodies to see if those drugs help reduce the number of MDS cells. If these drugs show promise, they could potentially be developed into new therapies for patients with MDS.
  • Develop a new clinical trial testing the feasibility of enhancing a patient’s own immune system by giving them natural killer cells from healthy people to reduce the number of MDS cells.

All of these approaches will have an impact on MDS and could provide important lessons that could be applicable to other cancers.

Masonic Cancer Center investigators working with Ross on the five-year NIH-funded MDS study include Erica Warlick, MD; Adina Cioc, MD; Betsy Hirsch, PhD and Jenny Poynter, PhD Masonic Cancer Center investigators working with Ross and Miller on the MDS partnership award include Warlick; Daniel Weisdorf, MD; David Largaespada, PhD; Dan Vallera, PhD, and Michael Burke, MD. Together, the team is collaborating with Mayo investigators Litzow; Al-Kali; Mrinal Patnaik, MD; James Cerhan, MD, PhD; Eric Klees, PhD; Nicole Hoppman-Chaney; and the Minnesota Department of Health.

For more information on the MDS research study, please contact or 1-866-434-9879.