Scientific Advisory Council

Donald Small, MD PhD

Kyle Haydock Professor of Oncology
Professor of Oncology, Pediatrics, Cellular and Molecular Medicine, and Human Genetics
Director, Pediatric Oncology
Director, Johns Hopkins/National Cancer Institute Pediatric Hematology/Oncology Fellowship Program
Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins
Johns Hopkins University School of Medicine
Baltimore, MD

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Don Small received his undergraduate, MD, and PhD degrees from the Johns Hopkins University (1979, 1985). He trained in pediatrics and pediatric hematology/oncology at Hopkins. During his pediatric Hematology/Oncology fellowship, he studied the role of a number of proteins in DNA replication. He joined the Hopkins faculty in 1990, and was named the Kyle Haydock Professor of Oncology in 2003, with joint appointments in Pediatrics, Cellular and Molecular Medicine, and Human Genetics. He has served as Director of Pediatric Oncology at Johns Hopkins since 2006.

His laboratory was the first to clone the human FLT3 gene, which is the most frequently mutated gene in acute myeloid leukemia (AML). He led the team that investigated the role of FLT3 in leukemia and was the first to discover drugs capable of inhibiting the tyrosine kinase activity of FLT3. This research revealed that this class of drugs could preferentially kill leukemic cell lines and primary AML samples expressing mutant FLT3–one of the first successful molecularly targeted cancer therapies. His lab also developed a high-throughput cell-based in vitro assay that facilitated screening of a large library of kinase inhibitors and identification of several with great potency and selectivity against FLT3. His group led the first clinical trials investigating the use of a FLT3 inhibitor in adult relapsed and refractory FLT3-mutant AML, and determined how best to combine these drugs with chemotherapy. The team also helped design the first pediatric trials of FLT3 inhibitors for use in treating pediatric AML and infant ALL.

His lab continues to investigate the process of leukemic transformation, the role of FLT3 in leukemia stem cells utilizing mouse models, and signaling changes in leukemic stem cells.

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