Doctor Margaret MacMillian, smiling.
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We’ve made a lot of progress in pediatric oncology research, but kids with cancer still need us to do more.

- Margaret MacMillan, M.D.

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News Releases — New Discoveries and Promising Progress

U of M Develops Long-Awaited Mouse Model for Infant Leukemia

The model opens the door to further investigation into a rare and often fatal blood cancer.

Researchers at the University of Minnesota Cancer Center have produced the first genetically-engineered mouse that provides a model of infant acute lymphoblastic leukemia (ALL). The mouse model opens the door to further investigation into the biology, treatment, and possibly cure of infant acute lymphoblastic leukemia (ALL).

Laboratory Model Makes Further Research Possible

ALL is an often fatal cancer that usually strikes children from infancy to one year of age. Infant ALL currently affects about 200 babies each year in the United States, and claims as many as 60 percent before their first birthday.

An abnormally formed gene is considered to be the source of the disease. While researchers have long known the gene, the absence of a laboratory model to more intensely study the disease has frustrated researchers for nearly two decades. With an experimental model, exploring and designing treatments has been possible. "Hopefully, this mouse will help us find a cure so that in the not too distant future, a parent does not have to experience the agony of losing a baby to this cancer," said John Kersey, M.D. & Dr. Kersey led the research team and is a physician and researcher specializing in childhood cancers and director of the University's Cancer Center.

Next, Kersey and his colleagues plan to research drugs that may provide better treatment options for infant ALL.

Model Provides Hope for Child and Adult Patients

Like all leukemias, infant ALL is an acquired, not inherited, genetic disease. DNA, which contains the code for a person's genetic structure, goes through a normal process of breaking and rejoining. During this replication process, researchers believe the genetic material in a few bone marrow cells gets damaged, resulting in cancer.

"Sometimes a mistake happens in the rejoining of the DNA and the result can be leukemia," Kersey said. "In about 75 percent of infants with infant ALL, the genetic rearrangement occurs in the womb as the baby is developing. We believe the genes fuse by mistake and form the basis for infant ALL."

Adults can also get the disease. About two-thirds of them die on average within two years of diagnosis. Many adults develop this disease as a secondary cancer, having been previously treated with chemotherapy or radiation for another cancer, such as breast cancer.

Kersey collaborated with these colleagues on this research: Weili Chen, Quanzhi Li, Ph.D., Wendy Hudson, Ashish Kumar, M.D., Ph.D., and Nicole Kirchhof, all with the University of Minnesota Cancer Center. The research was funded by a grant from the National Institutes of Health (NIH) and the Children's Cancer Research Fund (CCRF).