Researchers receive $2 million to pursue targets in rare pediatric cancer

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Indiana University School of Medicine researcher Reuben Kapur, PhD, Frieda and Albrecht Kipp Professor of Pediatrics, has received more than $2 million to identify new targets in the rare pediatric blood cancer known as juvenile myelomonocytic leukemia or JMML. Kapur, who conducts his research at the Herman B Wells Center for Pediatric Research and the IU Simon Cancer Center, will use the funds from the National Heart, Lung and Blood Institute to support his research over the next five years.

JMML is a rare pediatric leukemia that makes up about 1 to 2 percent of all pediatric blood cancers. It occurs when cancerous white blood cells proliferate and prevent healthy blood cells from functioning properly, which can lead to infection and organ failure. Most cases of JMML are diagnosed in children under the age of 4.

If left untreated, JMML can progress rapidly. But treatment options are scarce, and the cancerous cells are often resistant to chemotherapies. Instead, the most common treatment for JMML is a stem cell transplant, which is often successful in curing the disease. Still, about half of the patients who receive a stem cell transplant experience a relapse of the disease.

To better understand why relapse is so common, Kapur and his team of investigators hope to build on their previous work to characterize the bone marrow microenvironment and its interaction with different cellular mutations that are associated with JMML. Ultimately, their work will shed new light on JMML development and progression, and help to identify new ways to target treatment—especially for the 50 percent of children and their families who are devastated by relapse.

Reuben Kapur, PhD, is the Frieda and Albrecht Kipp Professor of Pediatrics, director of the Hematologic Malignancies and Stem Cell Biology research program at the Wells Center and co-director of the Hematopoiesis, Hematologic Malignancies and Immunology research program at the IU Simon Cancer Center.

The project referenced above is titled Targeting Novel Pathways in JMML, project number 1R01HL146137-01, with funds awarded by the National Heart, Lung and Blood Institute.

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