Mignon Lee-Cheun Loh, MD

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

Professor of Clinical Pediatrics, Division of Hematology Oncology, UCSF; Co-Leader, Pediatric Malignancies Program, UCSF Helen Diller Family Comprehensive Cancer Center

Cancer Center Program Membership

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.

Research Funding

  • July 1, 2015 - June 30, 2020 - Center for Precision Medicine in Leukemia (CPML), Co-Principal Investigator. Sponsor: NIH/NIGMS, Sponsor Award ID: P50GM115279
  • September 24, 2007 - July 31, 2015 - International Symposium on Juvenile Myelomonocytic Leukemia (JMML), Co-Principal Investigator. Sponsor: NIH/NCI, Sponsor Award ID: R13CA132568
  • September 30, 2010 - September 29, 2013 - Identifying the spectrum of genetic alterations in high risk ALL, Co-Principal Investigator. Sponsor: NIH/NCI, Sponsor Award ID: RC4CA156329
  • September 30, 2009 - December 31, 2012 - Targeted Therapies for Childhood Acute Lymphoblastic Leukemia, Co-Principal Investigator. Sponsor: NIH/NCI, Sponsor Award ID: RC2CA148529


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

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. Liu Y, Yang W, Smith C, Cheng C, Karol SE, Larsen EC, Winick N, Carroll WL, Loh ML, Raetz EA, Hunger SP, Winter SS, Dunsmore KP, Devidas M, Yang JJ, Evans WE, Jeha S, Pui CH, Inaba H, Relling MV Class II HLA Variants Associate with Risk of Pegaspargase Hypersensitivity.  View on PubMed
  2. Mattano LA, Devidas M, Maloney KW, Wang C, Friedmann AM, Buckley P, Borowitz MJ, Carroll AJ, Gastier-Foster JM, Heerema NA, Kadan-Lottick NS, Matloub YH, Marshall DT, Stork LC, Loh ML, Raetz EA, Wood BL, Hunger SP, Carroll WL, Winick NJ Favorable Trisomies and ETV6-RUNX1 Predict Cure in Low-Risk B-Cell Acute Lymphoblastic Leukemia: Results From Children's Oncology Group Trial AALL0331.  View on PubMed
  3. Clinical characteristics and outcomes of B-ALL with ZNF384 rearrangements: a retrospective analysis by the Ponte di Legno Childhood ALL Working Group.  View on PubMed
  4. Brown PA, Ji L, Xu X, Devidas M, Hogan LE, Borowitz MJ, Raetz EA, Zugmaier G, Sharon E, Bernhardt MB, Terezakis SA, Gore L, Whitlock JA, Pulsipher MA, Hunger SP, Loh ML Effect of Postreinduction Therapy Consolidation With Blinatumomab vs Chemotherapy on Disease-Free Survival in Children, Adolescents, and Young Adults With First Relapse of B-Cell Acute Lymphoblastic Leukemia: A Randomized Clinical Trial.  View on PubMed
  5. Tran TH, Nguyen JV, Stecula A, Akutagawa J, Moorman AV, Braun BS, Sali A, Mullighan CG, Shah NP, Dai Y, Devidas M, Roberts KG, Smith CC, Loh ML The EBF1-PDGFRB T681I mutation is highly resistant to imatinib and dasatinib in vitro and detectable in clinical samples prior to treatment.  View on PubMed
  6. Brown PA, Kairalla JA, Hilden JM, Dreyer ZE, Carroll AJ, Heerema NA, Wang C, Devidas M, Gore L, Salzer WL, Winick NJ, Carroll WL, Raetz EA, Borowitz MJ, Small D, Loh ML, Hunger SP FLT3 inhibitor lestaurtinib plus chemotherapy for newly diagnosed KMT2A-rearranged infant acute lymphoblastic leukemia: Children's Oncology Group trial AALL0631.  View on PubMed
  7. Pikman Y, Tasian SK, Sulis ML, Stevenson K, Blonquist TM, Apsel Winger B, Cooper TM, Pauly M, Maloney KW, Burke MJ, Brown PA, Gossai N, McNeer JL, Shukla NN, Cole PD, Kahn JM, Chen J, Barth MJ, Magee JA, Gennarini L, Adhav AA, Clinton CM, Ocasio-Martinez N, Gotti G, Li Y, Lin S, Imamovic A, Tognon CE, Patel T, Faust HL, Contreras CF, Cremer A, Cortopassi WA, Garrido Ruiz D, Jacobson MP, Dharia NV, Su A, Robichaud AL, Saur Conway A, Tarlock K, Stieglitz E, Place AE, Puissant A, Hunger SP, Kim AS, Lindeman NI, Gore L, Janeway KA, Silverman LB, Tyner JW, Harris MH, Loh ML, Stegmaier K Matched Targeted Therapy for Pediatric Patients with Relapsed, Refractory or High-risk Leukemias: A Report from the LEAP Consortium.  View on PubMed
  8. Rau RE, Dai Y, Devidas M, Rabin KR, Zweidler-McKay P, Angiolillo A, Schore RJ, Burke MJ, Salzer WL, Heerema NA, Carroll AJ, Winick NJ, Hunger SP, Raetz EA, Loh ML, Wood BL, Borowitz MJ Prognostic impact of minimal residual disease at the end of consolidation in NCI standard-risk B-lymphoblastic leukemia: A report from the Children's Oncology Group.  View on PubMed
  9. Matthijssens F, Sharma ND, Nysus M, Nickl CK, Kang H, Perez DR, Lintermans B, Van Loocke W, Roels J, Peirs S, Demoen L, Pieters T, Reunes L, Lammens T, De Moerloose B, Van Nieuwerburgh F, Deforce DL, Cheung LC, Kotecha RS, Risseeuw MDP, Van Calenbergh S, Takarada T, Yoneda Y, van Delft FW, Lock RB, Merkley SD, Chigaev A, Sklar LA, Mullighan CG, Loh ML, Winter SS, Hunger SP, Goossens S, Castillo EF, Ornatowski W, Van Vlierberghe P, Matlawska-Wasowska K RUNX2 regulates leukemic cell metabolism and chemotaxis in high-risk T cell acute lymphoblastic leukemia.  View on PubMed
  10. Advani AS, Larsen E, Laumann K, Luger SM, Liedtke M, Devidas M, Chen Z, Yin J, Foster MC, Claxton D, Coffan K, Tallman MS, Appelbaum FR, Erba H, Stone RM, Hunger SP, McNeer JL, Loh ML, Raetz E, Winick N, Carroll W, Larson RA, Stock W Comparison of CALGB 10403 (Alliance) and COG AALL0232 toxicity results in young adults with acute lymphoblastic leukemia.  View on PubMed
  11. Angiolillo AL, Schore RJ, Kairalla JA, Devidas M, Rabin KR, Zweidler-McKay P, Borowitz MJ, Wood B, Carroll AJ, Heerema NA, Relling MV, Hitzler J, Lane AR, Maloney KW, Wang C, Bassal M, Carroll WL, Winick NJ, Raetz EA, Loh ML, Hunger SP Excellent Outcomes With Reduced Frequency of Vincristine and Dexamethasone Pulses in Standard-Risk B-Lymphoblastic Leukemia: Results From Children's Oncology Group AALL0932.  View on PubMed
  12. Hecht A, Meyer JA, Behnert A, Wong E, Chehab F, Olshen A, Hechmer A, Aftandilian C, Bhat R, Choi SW, Chonat S, Farrar JE, Fluchel M, Frangoul H, Han JH, Kolb EA, Kuo DJ, MacMillan ML, Maese L, Maloney KW, Narendran A, Oshrine B, Schultz KR, Sulis ML, Van Mater D, Tasian SK, Hofmann WK, Loh ML, Stieglitz E Molecular and phenotypic diversity of CBL-mutated juvenile myelomonocytic leukemia.  View on PubMed
  13. den Boer ML, Cario G, Moorman AV, Boer JM, de Groot-Kruseman HA, Fiocco M, Escherich G, Imamura T, Yeoh A, Sutton R, Dalla-Pozza L, Kiyokawa N, Schrappe M, Roberts KG, Mullighan CG, Hunger SP, Vora A, Attarbaschi A, Zaliova M, Elitzur S, Cazzaniga G, Biondi A, Loh ML, Pieters R Outcomes of paediatric patients with B-cell acute lymphocytic leukaemia with ABL-class fusion in the pre-tyrosine-kinase inhibitor era: a multicentre, retrospective, cohort study.  View on PubMed
  14. Schwab CJ, Roberts KG, Boer JM, Gohring G, Steinemann D, Vora A, Macartney C, Hough RE, Thorn Z, Dillon R, Escherich G, Cazzaniga G, Schlegelberger B, Loh ML, Den Boer ML, Moorman AV, Harrison CJ SSBP2-CSF1R is a Recurrent Fusion in B-lineage Acute Lymphoblastic Leukaemia with Diverse Genetic Presentation and Variable Outcome.  View on PubMed
  15. Schönung M, Meyer J, Nöllke P, Olshen AB, Hartmann M, Murakami N, Wakamatsu M, Okuno Y, Plass C, Loh ML, Niemeyer CM, Muramatsu H, Flotho C, Stieglitz E, Lipka DB International Consensus Definition of DNA Methylation Subgroups in Juvenile Myelomonocytic Leukemia.  View on PubMed
  16. Yang W, Devidas M, Liu Y, Smith C, Dai Y, Winick N, Hunger SP, Loh ML, Raetz EA, Larsen E, Carroll WL, Winter SS, Dunsmore KP, Mattano LA, Relling MV, Karol SE Genetics of osteonecrosis in pediatric acute lymphoblastic leukemia and general populations.  View on PubMed
  17. Saint Fleur-Lominy S, Evensen NA, Bhatla T, Sethia G, Narang S, Choi JH, Ma X, Yang JJ, Kelly S, Raetz E, Harvey RC, Willman C, Loh ML, Hunger SP, Brown PA, Getz KM, Meydan C, Mason CE, Tsirigos A, Carroll WL Evolution of the Epigenetic Landscape in Childhood B Acute Lymphoblastic Leukemia and Its Role in Drug Resistance.  View on PubMed
  18. Zhang H, Liu AP, Devidas M, Lee SHR, Cao X, Pei D, Borowitz M, Wood B, Gastier-Foster JM, Dai Y, Raetz E, Larsen E, Winick N, Bowman WP, Karol S, Yang W, Martin PL, Carroll WL, Pui CH, Mullighan CG, Evans WE, Cheng C, Hunger SP, Relling MV, Loh ML, Yang JJ Association of GATA3 polymorphisms with minimal residual disease and relapse risk in childhood acute lymphoblastic leukemia.  View on PubMed
  19. Lee SHR, Qian M, Yang W, Diedrich JD, Raetz E, Yang W, Dong Q, Devidas M, Pei D, Yeoh A, Cheng C, Pui CH, Evans WE, Mullighan CG, Hunger SP, Savic D, Relling MV, Loh ML, Yang JJ Genome-wide association study of susceptibility loci for TCF3-PBX1 acute lymphoblastic leukemia in children.  View on PubMed
  20. Teachey DT, Hunger SP, Loh ML Optimizing Therapy in the Modern Age: Differences in Length of Maintenance Therapy in Acute Lymphoblastic Leukemia.  View on PubMed

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