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.

Education

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. Ariës IM, Bodaar K, Karim SA, Chonghaile TN, Hinze L, Burns MA, Pfirrmann M, Degar J, Landrigan JT, Balbach S, Peirs S, Menten B, Isenhart R, Stevenson KE, Neuberg DS, Devidas M, Loh ML, Hunger SP, Teachey DT, Rabin KR, Winter SS, Dunsmore KP, Wood BL, Silverman LB, Sallan SE, Van Vlierberghe P, Orkin SH, Knoechel B, Letai AG, Gutierrez A. PRC2 loss induces chemoresistance by repressing apoptosis in T cell acute lymphoblastic leukemia. J Exp Med. 2018 Nov 07.
    View on PubMed
  2. Bhojwani D, Sposto R, Shah NN, Rodriguez V, Yuan C, Stetler-Stevenson M, O'Brien MM, McNeer JL, Raetz EA, Loh ML, Rheingold SR. Inotuzumab ozogamicin in pediatric patients with relapsed/refractory acute lymphoblastic leukemia. Leukemia. 2018 Sep 28.
    View on PubMed
  3. Alexander TB, Gu Z, Iacobucci I, Dickerson K, Choi JK, Xu B, Payne-Turner D, Yoshihara H, Loh ML, Horan J, Buldini B, Basso G, Elitzur S, de Haas V, Zwaan CM, Yeoh A, Reinhardt D, Tomizawa D, Kiyokawa N, Lammens T, De Moerloose B, Catchpoole D, Hori H, Moorman A, Moore AS, Hrusak O, Meshinchi S, Orgel E, Devidas M, Borowitz M, Wood B, Heerema NA, Carrol A, Yang YL, Smith MA, Davidsen TM, Hermida LC, Gesuwan P, Marra MA, Ma Y, Mungall AJ, Moore RA, Jones SJM, Valentine M, Janke LJ, Rubnitz JE, Pui CH, Ding L, Liu Y, Zhang J, Nichols KE, Downing JR, Cao X, Shi L, Pounds S, Newman S, Pei D, Guidry Auvil JM, Gerhard DS, Hunger SP, Inaba H, Mullighan CG. The genetic basis and cell of origin of mixed phenotype acute leukaemia. Nature. 2018 Sep 12.
    View on PubMed
  4. Kong G, You X, Wen Z, Chang YI, Qian S, Ranheim EA, Letson C, Zhang X, Zhou Y, Liu Y, Rajagopalan A, Zhang J, Stieglitz E, Loh M, Hofmann I, Yang D, Zhong X, Padron E, Zhou L, Pear WS, Zhang J. Downregulating Notch counteracts KrasG12D-induced ERK activation and oxidative phosphorylation in myeloproliferative neoplasm. Leukemia. 2018 Sep 11.
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  5. Kang H, Sharma ND, Nickl CK, Devidas M, Loh ML, Hunger SP, Dunsmore KP, Winter SS, Matlawska-Wasowska K. Dysregulated transcriptional networks in KMT2A- and MLLT10-rearranged T-ALL. Biomark Res. 2018; 6:27.
    View on PubMed
  6. Winter SS, Dunsmore KP, Devidas M, Wood BL, Esiashvili N, Chen Z, Eisenberg N, Briegel N, Hayashi RJ, Gastier-Foster JM, Carroll AJ, Heerema NA, Asselin BL, Gaynon PS, Borowitz MJ, Loh ML, Rabin KR, Raetz EA, Zweidler-Mckay PA, Winick NJ, Carroll WL, Hunger SP. Improved Survival for Children and Young Adults With T-Lineage Acute Lymphoblastic Leukemia: Results From the Children's Oncology Group AALL0434 Methotrexate Randomization. J Clin Oncol. 2018 Aug 23; JCO2018777250.
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  7. Wong JC, Bryant V, Lamprecht T, Ma J, Walsh M, Schwartz J, Del Pilar Alzamora M, Mullighan CG, Loh ML, Ribeiro R, Downing JR, Carroll WL, Davis J, Gold S, Rogers PC, Israels S, Yanofsky R, Shannon K, Klco JM. Germline SAMD9 and SAMD9L mutations are associated with extensive genetic evolution and diverse hematologic outcomes. JCI Insight. 2018 Jul 25; 3(14).
    View on PubMed
  8. Roberts KG, Reshmi SC, Harvey RC, Chen IM, Patel K, Stonerock E, Jenkins H, Dai Y, Valentine M, Gu Z, Zhao Y, Zhang J, Payne-Turner D, Devidas M, Heerema NA, Carroll AJ, Raetz EA, Borowitz MJ, Wood BL, Mattano LA, Maloney KW, Carroll WL, Loh ML, Willman CL, Gastier-Foster JM, Mullighan CG, Hunger SP. Genomic and outcome analyses of Ph-like ALL in NCI standard-risk patients: a report from the Children's Oncology Group. Blood. 2018 Jul 11.
    View on PubMed
  9. Gagne AL, Maguire JA, Gandre-Babbe S, Chou ST, Tasian SK, Loh ML, Weiss MJ, Gadue P, French DL. Generation of a human Juvenile myelomonocytic leukemia iPSC line, CHOPi001-A, with a mutation in CBL. Stem Cell Res. 2018 Jul 05; 31:157-160.
    View on PubMed
  10. Milani G, Matthijssens F, Van Loocke W, Durinck K, Roels J, Peirs S, Thénoz M, Pieters T, Reunes L, Lintermans B, Vandamme N, Lammens T, Van Roy N, Van Nieuwerburgh F, Deforce D, Schwab C, Raimondi S, Dalla Pozza L, Carroll AJ, De Moerloose B, Benoit Y, Goossens S, Berx G, Harrison CJ, Basso G, Cavé H, Sutton R, Asnafi V, Meijerink J, Mullighan C, Loh M, Van Vlierberghe P. Genetic characterization and therapeutic targeting of MYC-rearranged T cell acute lymphoblastic leukaemia. Br J Haematol. 2018 Jun 25.
    View on PubMed
  11. Tasian SK, Casas JA, Posocco D, Gandre-Babbe S, Gagne AL, Liang G, Loh ML, Weiss MJ, French DL, Chou ST. Mutation-specific signaling profiles and kinase inhibitor sensitivities of juvenile myelomonocytic leukemia revealed by induced pluripotent stem cells. Leukemia. 2018 Jun 08.
    View on PubMed
  12. Slayton WB, Schultz KR, Kairalla JA, Devidas M, Mi X, Pulsipher MA, Chang BH, Mullighan C, Iacobucci I, Silverman LB, Borowitz MJ, Carroll AJ, Heerema NA, Gastier-Foster JM, Wood BL, Mizrahy SL, Merchant T, Brown VI, Sieger L, Siegel MJ, Raetz EA, Winick NJ, Loh ML, Carroll WL, Hunger SP. Dasatinib Plus Intensive Chemotherapy in Children, Adolescents, and Young Adults With Philadelphia Chromosome-Positive Acute Lymphoblastic Leukemia: Results of Children's Oncology Group Trial AALL0622. J Clin Oncol. 2018 May 29; JCO2017767228.
    View on PubMed
  13. Perez DR, Nickl CK, Waller A, Delgado-Martin C, Woods T, Sharma ND, Hermiston ML, Loh ML, Hunger SP, Winter SS, Chigaev A, Edwards B, Sklar LA, Matlawska-Wasowska K. High-Throughput Flow Cytometry Identifies Small-Molecule Inhibitors for Drug Repurposing in T-ALL. SLAS Discov. 2018 May 01; 2472555218774248.
    View on PubMed
  14. Churchman ML, Qian M, Te Kronnie G, Zhang R, Yang W, Zhang H, Lana T, Tedrick P, Baskin R, Verbist K, Peters JL, Devidas M, Larsen E, Moore IM, Gu Z, Qu C, Yoshihara H, Porter SN, Pruett-Miller SM, Wu G, Raetz E, Martin PL, Bowman WP, Winick N, Mardis E, Fulton R, Stanulla M, Evans WE, Relling MV, Pui CH, Hunger SP, Loh ML, Handgretinger R, Nichols KE, Yang JJ, Mullighan CG. Germline Genetic IKZF1 Variation and Predisposition to Childhood Acute Lymphoblastic Leukemia. Cancer Cell. 2018 May 14; 33(5):937-948.e8.
    View on PubMed
  15. Place AE, Pikman Y, Stevenson KE, Harris MH, Pauly M, Sulis ML, Hijiya N, Gore L, Cooper TM, Loh ML, Roti G, Neuberg DS, Hunt SK, Orloff-Parry S, Stegmaier K, Sallan SE, Silverman LB. Phase I trial of the mTOR inhibitor everolimus in combination with multi-agent chemotherapy in relapsed childhood acute lymphoblastic leukemia. Pediatr Blood Cancer. 2018 Mar 30; e27062.
    View on PubMed
  16. Place AE, Goldsmith K, Bourquin JP, Loh ML, Gore L, Morgenstern DA, Sanzgiri Y, Hoffman D, Zhou Y, Ross JA, Prine B, Shebley M, McNamee M, Farazi T, Kim SY, Verdugo M, Lash-Fleming L, Zwaan CM, Vormoor J. Accelerating drug development in pediatric cancer: a novel Phase I study design of venetoclax in relapsed/refractory malignancies. Future Oncol. 2018 Mar 29.
    View on PubMed
  17. Burns MA, Liao ZW, Yamagata N, Pouliot GP, Stevenson KE, Neuberg DS, Thorner AR, Ducar M, Silverman EA, Hunger SP, Loh ML, Winter SS, Dunsmore KP, Wood B, Devidas M, Harris MH, Silverman LB, Sallan SE, Gutierrez A. Hedgehog pathway mutations drive oncogenic transformation in high-risk T-cell acute lymphoblastic leukemia. Leukemia. 2018 Mar 20.
    View on PubMed
  18. Tran TH, Harris MH, Nguyen JV, Blonquist TM, Stevenson KE, Stonerock E, Asselin BL, Athale UH, Clavell LA, Cole PD, Kelly KM, Laverdiere C, Leclerc JM, Michon B, Schorin MA, Welch JJG, Reshmi SC, Neuberg DS, Sallan SE, Loh ML, Silverman LB. Prognostic impact of kinase-activating fusions and IKZF1 deletions in pediatric high-risk B-lineage acute lymphoblastic leukemia. Blood Adv. 2018 Mar 13; 2(5):529-533.
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  19. Dvorak CC, Satwani P, Stieglitz E, Cairo MS, Dang H, Pei Q, Gao Y, Wall D, Mazor T, Olshen AB, Parker JS, Kahwash S, Hirsch B, Raimondi S, Patel N, Skeens M, Cooper T, Mehta PA, Grupp SA, Loh ML. Disease burden and conditioning regimens in ASCT1221, a randomized phase II trial in children with juvenile myelomonocytic leukemia: A Children's Oncology Group study. Pediatr Blood Cancer. 2018 Mar 12.
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  20. Gupta S, Devidas M, Loh ML, Raetz EA, Chen S, Wang C, Brown P, Carroll AJ, Heerema NA, Gastier-Foster JM, Dunsmore KP, Larsen EC, Maloney KW, Mattano LA, Winter SS, Winick NJ, Carroll WL, Hunger SP, Borowitz MJ, Wood BL. Flow-cytometric vs. -morphologic assessment of remission in childhood acute lymphoblastic leukemia: a report from the Children's Oncology Group (COG). Leukemia. 2018 02 23.
    View on PubMed

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