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When you speak with Kimberly Stegmaier about her work as a pediatric oncologist at Children’s Hospital Boston, it is clear that she loves treating patients, particularly children with hematological cancers like leukemia. Kim devotes her time to caring for children through the in-patient oncology service yet she also maintains long-term clinical relationships with patients she has been treating since completing her pediatric hematology oncology residency.

Early on in Kim’s training she learned that to make the biggest curative impact as a physician she needed to know more about the mechanics of disease: its natural history and the biological nuances that differ between individual patients with the same disease toward the goal of finding better therapies for patients.

Many of the patients Kim treats are cured. She revels in the fact that one of her long-term pediatric patients just announced her engagement. Another is now in college. “That part is wonderful – seeing children cured and going on with their lives,” she says. In pediatrics, however, some of the cancers she treats behave more like a chronic disease requiring intermittent treatment over many years as the cancer responds but then recurs. “Moreover, there are many patients who won’t be cured with current treatment.”

Kimberly Stegmaier, MD

The memory of patients who were not cured, and her ongoing clinical care, motivates Kim as a research scientist. She directs her own laboratory at the Dana-Farber Cancer Institute. Currently a ӳ����ý associate member, her affiliation with the ӳ����ý dates back to her time as a postdoctoral fellow in the Dana-Farber laboratory of Todd Golub, ӳ����ý core member and director of the Cancer Program.

Partnering with the ӳ����ý, Kim has been taking on the challenges of finding new therapeutic targets in acute myeloid leukemia (AML). “During my clinical training as a pediatric oncologist, I was very struck by how poor the cure rates still are for children and adults with AML," Kim explains. “New approaches to treating this disease are very much needed." In particular, she has explored the use of small molecule probes to discover new vulnerabilities in AML. In AML, immature blood cells fail to develop – or differentiate – into normal cells. The consequence is a large buildup of immature blood cells in the bone marrow and a lack of healthy, functioning normal cells.

Her research seeks to better understand the reasons behind failed differentiation of AML cells and to find new therapies to promote healthy cell maturation. This week, Kim and ӳ����ý colleagues published findings of a potential new target to promote AML maturation, a protein called glycogen synthase kinase-3 alpha (GSK-3alpha). Read the paper and the ӳ����ý’s news story here.

At the time Kim joined the Golub lab, Todd was leading efforts to apply large-scale gene-expression profiling to cancer. The technique involves studying the effects of specific genes when they come in contact with particular chemicals or when they are intentionally turned on or off. In this week’s paper, the Dana-Farber/ӳ����ý team used these techniques to identify the new GSK-3 alpha target, which previously had not been identified as having a role in AML.

For Kim, caring for children with cancer serves as constant motivation for her laboratory research. “When you go back and forth between clinical care and research, you are always reminded of why you are doing what you are in the lab,” she adds. And she believes she could not be as effective in her laboratory without the insights she gains from working with patients. “Patient clinical care informs discovery,” she says. "The observation of a patient can inspire a new avenue of study in the laboratory. Likewise, my ultimate goal, like that of my colleagues at the ӳ����ý and Dana-Farber, is that the research conducted in the laboratory will translate to patient benefit in the clinic.”