
Malcolm Smith, M.D., Ph.D.
Dr. Smith is Associate Branch Chief, Pediatrics, in the Cancer Therapy Evaluation Program (CTEP), NCI. Dr. Smith has been a member of CTEP since 1990 and during his years at CTEP has focused on developing NCI’s preclinical and clinical research programs for children with cancer. Dr. Smith serves as an NCI scientific liaison to childhood cancer researchers in the Children’s Oncology Group (COG), focusing primarily on hematologic malignancies and brain cancers. He also serves as the Program Director for the Pediatric Early Phase Clinical Trials Network (PEP-CTN) and the Pediatric Brain Tumor Consortium (PBTC). He serves as a project scientist for the Pediatric Preclinical in Vivo Testing (PIVOT) Program. Dr. Smith was a co-leader of the NCI Moonshot Fusion Oncoproteins in Childhood Cancers Implementation Team and is a member of the NCI team coordinating the Targeting Fusion Oncoproteins in Childhood Cancer (TFCC) Network. Dr. Smith is the author or co-author of more than 240 publications and 25 book chapters related to childhood cancer research, and he serves on the NCI PDQ Pediatric Editorial Board.
Selected Publications
Lessons learned from 20 years of preclinical testing in pediatric cancers. Smith MA, Houghton PJ, Lock RB, Maris JM, Gorlick R, Kurmasheva RT, Li XN, Teicher BA, Chuang JH, Dela Cruz FS, Dyer MA, Kung AL, Lloyd MW, Mossé YP, Stearns TM, Stewart EA, Bult CJ, Erickson SW.Pharmacol Ther. 2024 Dec;264:108742. doi: 10.1016/j.pharmthera.2024.108742. Epub 2024 Nov 5.
International Consensus on Minimum Preclinical Testing Requirements for the Development of Innovative Therapies For Children and Adolescents with Cancer. Vassal G, Houghton PJ, Pfister SM, Smith MA, Caron HN, Li XN, Shields DJ, Witt O, Molenaar JJ, Colombetti S, Schüler J, Stancato LF.Mol Cancer Ther. 2021 Aug;20(8):1462-1468. doi: 10.1158/1535-7163.MCT-20-0394. Epub 2021 Jun 9.PMID: 34108262
A Menin-MLL Inhibitor Induces Specific Chromatin Changes and Eradicates Disease in Models of MLL-Rearranged Leukemia. Krivtsov AV, Evans K, Gadrey JY, Eschle BK, Hatton C, Uckelmann HJ, Ross KN, Perner F, Olsen SN, Pritchard T, McDermott L, Jones CD, Jing D, Braytee A, Chacon D, Earley E, McKeever BM, Claremon D, Gifford AJ, Lee HJ, Teicher BA, Pimanda JE, Beck D, Perry JA, Smith MA, McGeehan GM, Lock RB, Armstrong SA.Cancer Cell. 2019 Dec 9;36(6):660-673.e11. doi: 10.1016/j.ccell.2019.11.001.PMID: 31821784
The genomic landscape of pediatric acute lymphoblastic leukemia. Brady SW, Roberts KG, Gu Z, Shi L, Pounds S, Pei D, Cheng C, Dai Y, Devidas M, Qu C, Hill AN, Payne-Turner D, Ma X, Iacobucci I, Baviskar P, Wei L, Arunachalam S, Hagiwara K, Liu Y, Flasch DA, Liu Y, Parker M, Chen X, Elsayed AH, Pathak O, Li Y, Fan Y, Michael JR, Rusch M, Wilkinson MR, Foy S, Hedges DJ, Newman S, Zhou X, Wang J, Reilly C, Sioson E, Rice SV, Pastor Loyola V, Wu G, Rampersaud E, Reshmi SC, Gastier-Foster J, Guidry Auvil JM, Gesuwan P, Smith MA, Winick N, Carroll AJ, Heerema NA, Harvey RC, Willman CL, Larsen E, Raetz EA, Borowitz MJ, Wood BL, Carroll WL, Zweidler-McKay PA, Rabin KR, Mattano LA, Maloney KW, Winter SS, Burke MJ, Salzer W, Dunsmore KP, Angiolillo AL, Crews KR, Downing JR, Jeha S, Pui CH, Evans WE, Yang JJ, Relling MV, Gerhard DS, Loh ML, Hunger SP, Zhang J, Mullighan CG.Nat Genet. 2022 Sep;54(9):1376-1389. doi: 10.1038/s41588-022-01159-z. Epub 2022 Sep 1.PMID: 36050548
The B7-H3-Targeting Antibody-Drug Conjugate m276-SL-PBD Is Potently Effective Against Pediatric Cancer Preclinical Solid Tumor Models. Kendsersky NM, Lindsay J, Kolb EA, Smith MA, Teicher BA, Erickson SW, Earley EJ, Mosse YP, Martinez D, Pogoriler J, Krytska K, Patel K, Groff D, Tsang M, Ghilu S, Wang Y, Seaman S, Feng Y, Croix BS, Gorlick R, Kurmasheva R, Houghton PJ, Maris JM.Clin Cancer Res. 2021 May 15;27(10):2938-2946. doi: 10.1158/1078-0432.CCR-20-4221. Epub 2021 Feb 22.PMID: 33619171
Additional links to Scientific Publications
Dr. Smith’s ORCID number is 0000-0001-9880-9876.