High Risk / Refractory Acute Myeloid Leukemia

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Innovative Research for High-Risk & Relapsed Leukemias

Leukemia is a major area of research at Cincinnati Children’s. Research in the Cancer and Blood Diseases Institute is focused on the development of new therapies for children with very high-risk and relapsed forms of the disease.

Novel blood and marrow transplant regimens

Researchers at Cincinnati Children’s have developed and now lead a series of clinical research studies involving new approaches to blood and marrow transplantation for children with leukemia and related disorders.

We offer an extensive portfolio of new types of blood and marrow transplant regimens incorporating new approaches to chemotherapy. The goal of these approaches is to eliminate leukemia cells and develop new anti-infection therapies.

We are also working to develop novel approaches to donor selection  to potentially harness the immune system to destroy remaining leukemia cells in the recipient.

Molecularly targeted chemotherapy

Researchers are investigating new types of molecularly targeted chemotherapy regimens that block the signaling pathways that cancer cells use to grow and spread.

Understanding the causes of leukemia

Other research initiatives focus on understanding the causes of leukemia, particularly in children with Fanconi anemia and Down syndrome. This research is critical to improving the outcome in these high-risk groups of children. In addition, many of the insights gained from studying leukemia in these children have important relevance for our efforts to advance the cure of leukemias in children who do not have these disorders.

Cincinnati Children’s researchers have recently discovered genes important to understanding which children with Down syndrome are most at risk for developing leukemia. The laboratory of John P. Perentesis, MD, FAAP, is studying potential causes for the marked 20-fold increase in leukemia risk in children with Down syndrome.

Working to provide “tailored” therapies

A series of clinical research studies is examining the important role of genetically determined differences in chemotherapy drug metabolism. The research also is exploring how susceptibility to side effects influences the outcome of current chemotherapy regimens for childhood leukemia. This work has implications for the potential development of future personalized, or “tailored,” approaches to therapy.


  • More About Our Research

    Learn more about our world-class research on cancer in the Division of Oncology.