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Donald Small, M.D., Ph.D.

Director, Division of Pediatric Oncology
Professor of Oncology


Appointment Phone


Main Location

The Johns Hopkins Hospital

Out-of-State & International Patients +
Out of State Patients

Call 410-464-6641 (8a.m. to 6p.m., EST, Mon-Fri)

Learn more about our out-of-state patient services »

International Patients

Call +1-410-502-7683 (7a.m. to 6p.m., EST, Mon-Fri)

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  • Director, Division of Pediatric Oncology
  • Kyle Haydock Professor of Oncology
  • Professor of Oncology
  • Professor of Pediatrics

Centers & Institutes



The Johns Hopkins Hospital

Appointment Phone: 443-287-6997

600 N. Wolfe Street
Charlotte R. Bloomberg Childrens Center
Baltimore, MD 21287 map
Phone: 410-614-0994
Fax: 410-955-8897


Acute Lymphoblastic Leukemia, Acute Myeloid Leukemia, Chronic Myeloid Leukemia , Hodgkin's Disease , Leukemia, Medical Oncology, Myelodysplastic Syndrome, Non-Hodgkin's Lymphoma, Pediatric Oncology

Research Interests

Development of leukemia and lymphoma and FLT3 mutations.


Don Small received his undergraduate, and then M.D. and Ph.D. degrees from the Johns Hopkins University in 1979 and 1985. His Ph.D. research was conducted with Bert Vogelstein in the Oncology Department and his postdoctoral research with Tom Kelly in the Molecular Biology and Genetics Department. He trained in pediatrics and pediatric hematology/oncology at Johns Hopkins and joined the faculty in 1990 where he moved up the ranks and is the Kyle Haydock Professor of Oncology with joint appointments in Pediatrics and Cellular and Molecular Medicine and Human Genetics. He has been the Director of Pediatric Oncology since 2006.
His laboratory was the first to clone the human FLT3 gene that is the most frequently mutated gene in acute myeloid leukemia (AML) and results in very poor chances of cure for these patients. They investigated its role in leukemia and were the first to discover drugs able to inhibit the tyrosine kinase activity of FLT3. His laboratory showed that this class of drugs would preferentially kill leukemic cell lines and primary AML samples expressing mutant FLT3. Thus, this is one of the first examples of molecularly targeted therapy for cancer. They developed a high-throughput cell-based in vitro assay that enabled them to screen a large library of kinase inhibitors and find 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 to best combine these drugs with chemotherapy. They also helped design the first pediatric trials of FLT3 inhibitors in pediatric AML and infant ALL.
His lab continues to study the process of leukemic transformation and the role of FLT3 in leukemia stem cells through the generation of mouse models and by studying signaling changes in these cells. more

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C-Answers - Pediatric and Childhood Cancer

    Additional Information

  • Education +


    • Johns Hopkins University School of Medicine / MD (1985)


    • Johns Hopkins University School of Medicine / Pediatrics (1987)


    • Johns Hopkins University School of Medicine (1990)
  • Research & Publications +

    Clinical Trial Keywords: FLT3 inhibitors, lymphoma, leukemia

    Selected Publications

    Brown, P.; Levis, M.; McIntyre, E.; Griesemer, M.; Small, D. Combinations of the FLT3 inhibitor CEP-701 and chemotherapy synergistically kill infant and childhood MLL-rearranged ALL cells in a sequence-dependent manner. Leukemia. 2006 Aug;20(8):1368-1376.

    Kim, K.T.; Levis, M.; Small, D. Constitutively activated FLT3 phosphorylates BAD partially through pim-1. Br J Haematol. 2006 Sep;134(5):500-509.

    Knapper, S.; Burnett, A.K.; Littlewood, T.; Kell, W.J.; Agrawal, S.; Chopra, R.; Clark, R.; Levis, M.J.; Small, D. A phase 2 trial of the FLT3 inhibitor lestaurtinib (CEP701) as first-line treatment for older patients with acute myeloid leukemia not considered fit for intensive chemotherapy. Blood. 2006 Nov 15;108(10):3262-3270.

    Levis, M.; Brown, P.; Smith, B.D.; Stine, A.; Pham, R.; Stone, R.; Deangelo, D.; Galinsky, I.; Giles, F.; Estey, E.; Kantarjian, H.; Cohen, P.; Wang, Y.; Roesel, J.; Karp, J.E.; Small, D. Plasma inhibitory activity (PIA): a pharmacodynamic assay reveals insights into the basis for cytotoxic response to FLT3 inhibitors. Blood. 2006 Nov 15;108(10):3477-3483.

    Piloto, O.; Nguyen, B.; Huso, D.; Kim, K.T.; Li, Y.; Witte, L.; Hicklin, D.J.; Brown, P.; Small, D. IMC-EB10, an anti-FLT3 monoclonal antibody, prolongs survival and reduces nonobese diabetic/severe combined immunodeficient engraftment of some acute lymphoblastic leukemia cell lines and primary leukemic samples. Cancer Res. 2006 May 1;66(9):4843-4851.

    Radomska, H.S.; Basseres, D.S.; Zheng, R.; Zhang, P.; Dayaram, T.; Yamamoto, Y.; Sternberg, D.W.; Lokker, N.; Giese, N.A.; Bohlander, S.K.; Schnittger, S.; Delmotte, M.H.; Davis, R.J.; Small, D.; Hiddemann, W.; Gilliland, D.G.; Tenen, D.G. Block of C/EBP alpha function by phosphorylation in acute myeloid leukemia with FLT3 activating mutations. J Exp Med. 2006 Feb 20;203(2):371-381.

    Baldwin, B.R.; Li, L.; Tse, K.F.; Small, S.; Collector, M.; Whartenby, K.A.; Sharkis, S.J.; Racke, F.; Huso, D.; Small, D. Transgenic mice expressing Tel-FLT3, a constitutively activated form of FLT3, develop myeloproliferative disease. Leukemia. 2007 Apr;21(4):764-771.

    Brown, P.; McIntyre, E.; Rau, R.; Meshinchi, S.; Lacayo, N.; Dahl, G.; Alonzo, T.A.; Chang, M.; Arceci, R.J.; Small, D. The incidence and clinical significance of nucleophosmin mutations in childhood AML. Blood. 2007 Aug 1;110(3):979-985.

    Kim, K.T.; Baird, K.; Davis, S.; Piloto, O.; Levis, M.; Li, L.; Chen, P.; Meltzer, P.; Small, D. Constitutive Fms-like tyrosine kinase 3 activation results in specific changes in gene expression in myeloid leukaemic cells. Br J Haematol. 2007 Sep;138(5):603-615.

    Li, L.; Piloto, O.; Kim, K.T.; Ye, Z.; Nguyen, H.B.; Yu, X.; Levis, M.; Cheng, L.; Small, D. FLT3/ITD expression increases expansion, survival and entry into cell cycle of human haematopoietic stem/progenitor cells. Br J Haematol. 2007 Apr;137(1):64-75.

    Piloto, O.; Wright, M.; Brown, P.; Kim, K.T.; Levis, M.; Small, D. Prolonged exposure to FLT3 inhibitors leads to resistance via activation of parallel signaling pathways. Blood. 2007 Feb 15;109(4):1643-1652.

    Li, L.; Piloto, O.; Nguyen, H.B.; Greenberg, K.; Takamiya, K.; Racke, F.; Huso, D.; Small, D. Knock-in of an internal tandem duplication mutation into murine FLT3 confers myeloproliferative disease in a mouse model. Blood. 2008 Apr 1;111(7):3849-3858.

    Sallmyr, A.; Fan, J.; Datta, K.; Kim, K.T.; Grosu, D.; Shapiro, P.; Small, D.; Rassool, F. Internal tandem duplication of FLT3 (FLT3/ITD) induces increased ROS production, DNA damage and misrepair: implications for poor prognosis in AML. Blood. 2008 Jan 11.

    Small, D. Targeting FLT3 for the treatment of leukemia. Seminars in hematology. 2008 Jul;45(3 Suppl 2):S17-21.

    Whartenby, K.A.; Small, D.; Calabresi, P.A. FLT3 inhibitors for the treatment of autoimmune disease. Expert Opin Investig Drugs. 2008 Nov;17(11):1685-1692.

    Pratz, K.W.; Cortes, J.; Roboz, G.J.; Rao, N.; Arowojolu, O.; Stine, A.; Shiotsu, Y.; Shudo, A.; Akinaga, S.; Small, D.; Karp, J.E.; Levis, M. A pharmacodynamic study of the FLT3 inhibitor KW-2449 yields insight into the basis for clinical response. Blood. 2009 Apr 23;113(17):3938-3946.

    Schafer, E.; Irizarry, R.; Negi, S.; McIntyre, E.; Small, D.; Figueroa, M.E.; Melnick, A.; Brown, P. Promoter hypermethylation in MLL-r infant acute lymphoblastic leukemia: biology and therapeutic targeting. Blood. 2010 Jun 10;115(23):4798-4809.
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