University of California San Francisco
Helen Diller Family Comprehensive Cancer Center
Mignon Lee-Cheun Loh, MD

Mignon Lee-Cheun Loh, MD

Professor of Clinical Pediatrics, Division of Hematology Oncology, UCSF

Benioff UCSF Professorship in Children's Health; and Deborah and Arthur Ablin, MD, Endowed Chair in Pediatric Molecular Oncology, UCSF

Cancer Center Program Memberships

Pediatric Malignancies

Research Summary

My research program has focused on translating genomic and biochemical discoveries in juvenile myelomonocytic (JMML) and acute lymphoblastic leukemia (ALL) into assays and therapies that can be incorporated into clinical trials. My work in JMML has largely focused on dissecting the genomic landscape of JMML, including descriptions of PTPN11 and CBL mutations and the discovery of CBL as a new familial tumor suppressor gene. From these discoveries, I established molecular diagnostic testing for JMML in the CLIA setting at UCSF, which is now utilized in a clinical trial (#ASCT1221). Within the Children’s Oncology Group (COG), I am currently Chair of the COG ALL committee starting in April 2015 and am responsible for supervising and implementing the next generation of national ALL trials for children, adolescents, and young adults. This appointment in part stemmed from my role as a Study Co-Chair of AALL03B1, the Classification of Acute Lymphoblastic Leukemia, which over enrolled 11,200 children, and my role as Vice-Chair of Biology (2008-2015). My unique expertise is being able to straddle the physician-scientist landscape and collaborate with numerous outstanding clinicians and scientists in order to facilitate cutting edge discoveries into patient care. I am a generous collaborator and deeply believe in the concept of team science. I also have experience in developing and running early phase clinical trials for children with cancer.


Bryn Mawr College, Bryn Mawr, PA, B.A., 1987, History of Art
Columbia University College of Physicians and Surgeons, M.D., 1991, Medicine
Harvard University School of Public Health, 1999, Clinical Effectiveness
University California San Francisco Medical School, 2001, Advanced Training in Clinical Research

Professional Experience

  • 1991-1992
    Intern in Pediatrics, University of California-San Francisco, San Francisco, CA.
  • 1992-1994
    Resident in Pediatrics, The Children's Hospital, Boston, Harvard Medical School, Boston, MA.
  • 1995-1996
    Clinical Fellow, Pediatric Hematology-Oncology, The Children's Hospital, Boston and Dana-Farber Cancer Institute, Harvard Medical School, Boston, MA.
  • 1996-1998
    Research Fellow, Division of Hematology-Oncology, The Brigham and Women's Hospital, Harvard Medical School, Boston, MA.
  • 1998-1999
    Instructor, Harvard Medical School, Boston, MA
  • 1999
    Research Fellow, Brigham and Women's Hospital, Boston, MA
  • 1999-2006
    Assistant Professor of Clinical Pediatrics, University of California, San Francisco Medical School, San Francisco, CA.
  • 2006-2011
    Associate Professor of Clinical Pediatrics, University of California, San Francisco Medical School, San Francsico, CA.
  • 2011-present
    Professor of Clinical Pediatrics, Division of Hematology Oncology, San Francsico, CA.

Honors & Awards

  • 1987
    Cum Laude, Bryn Mawr College
  • 1988, 1989
    Harriet Judd Sartain Memorial Scholarship
  • 1990
    Hannah Longshore Memorial Scholarship
  • 1991
    Arnold Gold Foundation Award, Columbia University College of Physicians and Surgeons
  • 1995-1999
    Recipient, Research Training Grant (5T32HL07574), PI: Samuel Lux, MD
  • 1999-2004
    Recipient, K23 award, Mentors: D. Gary Gilliland, MD, Ph.D 1999 and Kevin M. Shannon MD, 2000-2004
  • 1999-2002
    Recipient, Child Health Research Center award, PI: Larry Shapiro, MD, PhD
  • 2007-2012
    Leukemia Lymphoma Society, Scholar in Clinical Research

Selected Publications

  1. Stieglitz E, Mazor T, Olshen AB, Geng H, Gelston LC, Akutagawa J, Lipka DB, Plass C, Flotho C, Chehab FF, Braun BS, Costello JF, Loh ML. Genome-wide DNA methylation is predictive of outcome in juvenile myelomonocytic leukemia. Nat Commun. 2017 Dec 19; 8(1):2127.
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  2. Liu Y, Easton J, Shao Y, Maciaszek J, Wang Z, Wilkinson MR, McCastlain K, Edmonson M, Pounds SB, Shi L, Zhou X, Ma X, Sioson E, Li Y, Rusch M, Gupta P, Pei D, Cheng C, Smith MA, Auvil JG, Gerhard DS, Relling MV, Winick NJ, Carroll AJ, Heerema NA, Raetz E, Devidas M, Willman CL, Harvey RC, Carroll WL, Dunsmore KP, Winter SS, Wood BL, Sorrentino BP, Downing JR, Loh ML, Hunger SP, Zhang J, Mullighan CG. The genomic landscape of pediatric and young adult T-lineage acute lymphoblastic leukemia. Nat Genet. 2017 Aug; 49(8):1211-1218.
    View on PubMed
  3. Tran TH, Shah AT, Loh ML. Precision Medicine in Pediatric Oncology: Translating Genomic Discoveries into Optimized Therapies. Clin Cancer Res. 2017 Sep 15; 23(18):5329-5338.
    View on PubMed
  4. Yoshimi A, Balasis ME, Vedder A, Feldman K, Ma Y, Zhang H, Lee SC, Letson C, Niyongere S, Lu SX, Ball M, Taylor J, Zhang Q, Zhao Y, Youssef S, Chung YR, Zhang XJ, Durham BH, Yang W, List AF, Loh ML, Klimek V, Berger MF, Stieglitz E, Padron E, Abdel-Wahab O. Robust patient-derived xenografts of MDS/MPN overlap syndromes capture the unique characteristics of CMML and JMML. Blood. 2017 Jul 27; 130(4):397-407.
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  5. Kruth KA, Fang M, Shelton DN, Abu-Halawa O, Mahling R, Yang H, Weissman JS, Loh ML, Müschen M, Tasian SK, Bassik MC, Kampmann M, Pufall MA. Suppression of B-cell development genes is key to glucocorticoid efficacy in treatment of acute lymphoblastic leukemia. Blood. 2017 Jun 01; 129(22):3000-3008.
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  6. Reshmi SC, Harvey RC, Roberts KG, Stonerock E, Smith A, Jenkins H, Chen IM, Valentine M, Liu Y, Li Y, Shao Y, Easton J, Payne-Turner D, Gu Z, Tran TH, Nguyen JV, Devidas M, Dai Y, Heerema NA, Carroll AJ, Raetz EA, Borowitz MJ, Wood BL, Angiolillo AL, Burke MJ, Salzer WL, Zweidler-McKay PA, Rabin KR, Carroll WL, Zhang J, Loh ML, Mullighan CG, Willman CL, Gastier-Foster JM, Hunger SP. Targetable kinase gene fusions in high-risk B-ALL: a study from the Children's Oncology Group. Blood. 2017 Jun 22; 129(25):3352-3361.
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  7. Frismantas V, Dobay MP, Rinaldi A, Tchinda J, Dunn SH, Kunz J, Richter-Pechanska P, Marovca B, Pail O, Jenni S, Diaz-Flores E, Chang BH, Brown TJ, Collins RH, Uhrig S, Balasubramanian GP, Bandapalli OR, Higi S, Eugster S, Voegeli P, Delorenzi M, Cario G, Loh ML, Schrappe M, Stanulla M, Kulozik AE, Muckenthaler MU, Saha V, Irving JA, Meisel R, Radimerski T, Von Stackelberg A, Eckert C, Tyner JW, Horvath P, Bornhauser BC, Bourquin JP. Ex vivo drug response profiling detects recurrent sensitivity patterns in drug-resistant acute lymphoblastic leukemia. Blood. 2017 Mar 16; 129(11):e26-e37.
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  8. Karol SE, Larsen E, Cheng C, Cao X, Yang W, Ramsey LB, Fernandez CA, McCorkle JR, Paugh SW, Autry RJ, Lopez-Lopez E, Diouf B, Jeha S, Pui CH, Raetz EA, Winick NJ, Carroll WL, Hunger SP, Loh ML, Devidas M, Evans WE, Yang JJ, Relling MV. Genetics of ancestry-specific risk for relapse in acute lymphoblastic leukemia. Leukemia. 2017 Jun; 31(6):1325-1332.
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  9. Liu Y, Fernandez CA, Smith C, Yang W, Cheng C, Panetta JC, Kornegay N, Liu C, Ramsey LB, Karol SE, Janke LJ, Larsen EC, Winick N, Carroll WL, Loh ML, Raetz EA, Hunger SP, Devidas M, Yang JJ, Mullighan CG, Zhang J, Evans WE, Jeha S, Pui CH, Relling MV. Genome-Wide Study Links PNPLA3 Variant With Elevated Hepatic Transaminase After Acute Lymphoblastic Leukemia Therapy. Clin Pharmacol Ther. 2017 Jan 16.
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  10. Tran TH, Loh ML. Ph-like acute lymphoblastic leukemia. Hematology Am Soc Hematol Educ Program. 2016 Dec 02; 2016(1):561-566.
    View on PubMed
  11. Liu W, Yu WM, Zhang J, Chan RJ, Loh ML, Zhang Z, Bunting KD, Qu CK. Inhibition of the Gab2/PI3K/mTOR signaling ameliorates myeloid malignancy caused by Ptpn11 (Shp2) gain-of-function mutations. Leukemia. 2017 Jun; 31(6):1415-1422.
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  12. Gu Z, Churchman M, Roberts K, Li Y, Liu Y, Harvey RC, McCastlain K, Reshmi SC, Payne-Turner D, Iacobucci I, Shao Y, Chen IM, Valentine M, Pei D, Mungall KL, Mungall AJ, Ma Y, Moore R, Marra M, Stonerock E, Gastier-Foster JM, Devidas M, Dai Y, Wood B, Borowitz M, Larsen EE, Maloney K, Mattano LA, Angiolillo A, Salzer WL, Burke MJ, Gianni F, Spinelli O, Radich JP, Minden MD, Moorman AV, Patel B, Fielding AK, Rowe JM, Luger SM, Bhatia R, Aldoss I, Forman SJ, Kohlschmidt J, Mrózek K, Marcucci G, Bloomfield CD, Stock W, Kornblau S, Kantarjian HM, Konopleva M, Paietta E, Willman CL, Loh ML, Hunger SP, Mullighan CG. Genomic analyses identify recurrent MEF2D fusions in acute lymphoblastic leukaemia. Nat Commun. 2016 11 08; 7:13331.
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  13. Dong L, Yu WM, Zheng H, Loh ML, Bunting ST, Pauly M, Huang G, Zhou M, Broxmeyer HE, Scadden DT, Qu CK. Leukaemogenic effects of Ptpn11 activating mutations in the stem cell microenvironment. Nature. 2016 11 10; 539(7628):304-308.
    View on PubMed
  14. Zhang J, McCastlain K, Yoshihara H, Xu B, Chang Y, Churchman ML, Wu G, Li Y, Wei L, Iacobucci I, Liu Y, Qu C, Wen J, Edmonson M, Payne-Turner D, Kaufmann KB, Takayanagi SI, Wienholds E, Waanders E, Ntziachristos P, Bakogianni S, Wang J, Aifantis I, Roberts KG, Ma J, Song G, Easton J, Mulder HL, Chen X, Newman S, Ma X, Rusch M, Gupta P, Boggs K, Vadodaria B, Dalton J, Liu Y, Valentine ML, Ding L, Lu C, Fulton RS, Fulton L, Tabib Y, Ochoa K, Devidas M, Pei D, Cheng C, Yang J, Evans WE, Relling MV, Pui CH, Jeha S, Harvey RC, Chen IL, Willman CL, Marcucci G, Bloomfield CD, Kohlschmidt J, Mrózek K, Paietta E, Tallman MS, Stock W, Foster MC, Racevskis J, Rowe JM, Luger S, Kornblau SM, Shurtleff SA, Raimondi SC, Mardis ER, Wilson RK, Dick JE, Hunger SP, Loh ML, Downing JR, Mullighan CG. Deregulation of DUX4 and ERG in acute lymphoblastic leukemia. Nat Genet. 2016 Dec; 48(12):1481-1489.
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  15. Tasian SK, Teachey DT, Li Y, Shen F, Harvey RC, Chen IM, Ryan T, Vincent TL, Willman CL, Perl AE, Hunger SP, Loh ML, Carroll M, Grupp SA. Potent efficacy of combined PI3K/mTOR and JAK or ABL inhibition in murine xenograft models of Ph-like acute lymphoblastic leukemia. Blood. 2017 Jan 12; 129(2):177-187.
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  16. Sherborne A, Lavergne V, Yu K, Lee L, Davidson P, Mazor T, Smirnov IV, Horvai AE, Loh M, Dubois SG, Goldsby R, Neglia JP, Hammond S, Robison LL, Wustrack R, Costello JF, Nakamura A, Shannon KM, Bhatia S, Nakamura JL. Somatic and germline TP53 alterations in second malignant neoplasms from pediatric cancer survivors. Clin Cancer Res. 2016 Sep 28.
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  17. Rodriguez V, Kairalla J, Salzer WL, Raetz EA, Loh ML, Carroll AJ, Heerema NA, Wood BL, Borowitz MJ, Burke MJ, Asselin BL, Devidas M, Winick NJ, Carroll WL, Hunger SP, Dreyer ZE. A Pilot Study of Intensified PEG-Asparaginase in High-risk Acute Lymphoblastic Leukemia: Children's Oncology Group Study AALL08P1. J Pediatr Hematol Oncol. 2016 Aug; 38(6):409-17.
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  18. Rau RE, Carroll AJ, Heerema NA, Arland L, Carroll WL, Winick NJ, Raetz EA, Loh ML, Yang W, Relling MV, Dai Y, Devidas M, Hunger SP. Klinefelter syndrome and 47,XYY syndrome in children with B cell acute lymphoblastic leukaemia. Br J Haematol. 2016 Jul 19.
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  19. Zaliova M, Moorman AV, Cazzaniga G, Stanulla M, Harvey RC, Roberts KG, Heatley SL, Loh ML, Konopleva M, Chen IM, Zimmermannova O, Schwab C, Smith O, Mozziconacci MJ, Chabannon C, Kim M, Frederik Falkenburg JH, Norton A, Marshall K, Haas OA, Starkova J, Stuchly J, Hunger SP, White D, Mullighan CG, Willman CL, Stary J, Trka J, Zuna J. Characterization of leukemias with ETV6-ABL1 fusion. Haematologica. 2016 Sep; 101(9):1082-93.
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  20. Liu C, Yang W, Devidas M, Cheng C, Pei D, Smith C, Carroll WL, Raetz EA, Bowman WP, Larsen EC, Maloney KW, Martin PL, Mattano LA, Winick NJ, Mardis ER, Fulton RS, Bhojwani D, Howard SC, Jeha S, Pui CH, Hunger SP, Evans WE, Loh ML, Relling MV. Clinical and Genetic Risk Factors for Acute Pancreatitis in Patients With Acute Lymphoblastic Leukemia. J Clin Oncol. 2016 Jun 20; 34(18):2133-40.
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