Mignon Lee-Cheun Loh, MD

Mignon Lee-Cheun Loh, MD

Professor of Clinical Pediatrics, Division of Hematology Oncology, UCSF

Deborah and Arthur Ablin Endowed Chair in Pediatric Molecular Oncology, UCSF

Phone: (415) 476-3831 (appts)
Box 0106, UCSF
San Francisco, CA 94143-0106

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Cancer Center Membership

Program Member » Hematopoietic Malignancies» 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, Taylor-Weiner AN, Chang TY, Gelston LC, Wang YD, Mazor T, Esquivel E, Yu A, Seepo S, Olsen SR, Rosenberg M, Archambeault SL, Abusin G, Beckman K, Brown PA, Briones M, Carcamo B, Cooper T, Dahl GV, Emanuel PD, Fluchel MN, Goyal RK, Hayashi RJ, Hitzler J, Hugge C, Liu YL, Messinger YH, Mahoney DH, Monteleone P, Nemecek ER, Roehrs PA, Schore RJ, Stine KC, Takemoto CM, Toretsky JA, Costello JF, Olshen AB, Stewart C, Li Y, Ma J, Gerbing RB, Alonzo TA, Getz G, Gruber TA, Golub TR, Stegmaier K, Loh ML. The genomic landscape of juvenile myelomonocytic leukemia. Nat Genet. 2015 Oct 12.
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  2. Jones CL, Gearheart CM, Fosmire S, Delgado-Martin C, Evensen NA, Bride K, Waanders AJ, Pais F, Wang J, Bhatla T, Bitterman DS, de Rijk SR, Bourgeois W, Dandekar S, Park E, Burleson TM, Madhusoodhan PP, Teachey DT, Raetz EA, Hermiston ML, Müschen M, Loh ML, Hunger SP, Zhang J, Garabedian MJ, Porter CC, Carroll WL. MAPK signaling cascades mediate distinct glucocorticoid resistance mechanisms in pediatric leukemia. Blood. 2015 Nov 5; 126(19):2202-12.
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  3. Karol SE, Yang W, Van Driest SL, Chang TY, Kaste S, Bowton E, Basford M, Bastarache L, Roden DM, Denny JC, Larsen E, Winick N, Carroll WL, Cheng C, Pei D, Fernandez CA, Liu C, Smith C, Loh ML, Raetz EA, Hunger SP, Scheet P, Jeha S, Pui CH, Evans WE, Devidas M, Mattano LA, Relling MV. Genetics of glucocorticoid-associated osteonecrosis in children with acute lymphoblastic leukemia. Blood. 2015 Oct 8; 126(15):1770-6.
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  4. Tasian SK, Loh ML, Hunger SP. Childhood acute lymphoblastic leukemia: Integrating genomics into therapy. Cancer. 2015 Oct 15; 121(20):3577-90.
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  5. Borowitz MJ, Wood BL, Devidas M, Loh ML, Raetz EA, Salzer WL, Nachman JB, Carroll AJ, Heerema NA, Gastier-Foster JM, Willman CL, Dai Y, Winick NJ, Hunger SP, Carroll WL, Larsen E. Prognostic significance of minimal residual disease in high risk B-ALL: a report from Children's Oncology Group study AALL0232. Blood. 2015 Aug 20; 126(8):964-71.
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  6. Xu H, Zhang H, Yang W, Yadav R, Morrison AC, Qian M, Devidas M, Liu Y, Perez-Andreu V, Zhao X, Gastier-Foster JM, Lupo PJ, Neale G, Raetz E, Larsen E, Bowman WP, Carroll WL, Winick N, Williams R, Hansen T, Holm JC, Mardis E, Fulton R, Pui CH, Zhang J, Mullighan CG, Evans WE, Hunger SP, Gupta R, Schmiegelow K, Loh ML, Relling MV, Yang JJ. Inherited coding variants at the CDKN2A locus influence susceptibility to acute lymphoblastic leukaemia in children. Nat Commun. 2015; 6:7553.
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  7. Fernandez CA, Smith C, Yang W, Mullighan CG, Qu C, Larsen E, Bowman WP, Liu C, Ramsey LB, Chang T, Karol SE, Loh ML, Raetz EA, Winick NJ, Hunger SP, Carroll WL, Jeha S, Pui CH, Evans WE, Devidas M, Relling MV. Genome-wide analysis links NFATC2 with asparaginase hypersensitivity. Blood. 2015 Jul 2; 126(1):69-75.
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  8. Loh ML, Tasian SK, Rabin KR, Brown P, Magoon D, Reid JM, Chen X, Ahern CH, Weigel BJ, Blaney SM. A phase 1 dosing study of ruxolitinib in children with relapsed or refractory solid tumors, leukemias, or myeloproliferative neoplasms: A Children's Oncology Group phase 1 consortium study (ADVL1011). Pediatr Blood Cancer. 2015 Oct; 62(10):1717-24.
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  9. Chen Z, Shojaee S, Buchner M, Geng H, Lee JW, Klemm L, Titz B, Graeber TG, Park E, Tan YX, Satterthwaite A, Paietta E, Hunger SP, Willman CL, Melnick A, Loh ML, Jung JU, Coligan JE, Bolland S, Mak TW, Limnander A, Jumaa H, Reth M, Weiss A, Lowell CA, Müschen M. Signalling thresholds and negative B-cell selection in acute lymphoblastic leukaemia. Nature. 2015 May 21; 521(7552):357-61.
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  10. Ma X, Edmonson M, Yergeau D, Muzny DM, Hampton OA, Rusch M, Song G, Easton J, Harvey RC, Wheeler DA, Ma J, Doddapaneni H, Vadodaria B, Wu G, Nagahawatte P, Carroll WL, Chen IM, Gastier-Foster JM, Relling MV, Smith MA, Devidas M, Guidry Auvil JM, Downing JR, Loh ML, Willman CL, Gerhard DS, Mullighan CG, Hunger SP, Zhang J. Rise and fall of subclones from diagnosis to relapse in pediatric B-acute lymphoblastic leukaemia. Nat Commun. 2015; 6:6604.
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  11. Geng H, Hurtz C, Lenz KB, Chen Z, Baumjohann D, Thompson S, Goloviznina NA, Chen WY, Huan J, LaTocha D, Ballabio E, Xiao G, Lee JW, Deucher A, Qi Z, Park E, Huang C, Nahar R, Kweon SM, Shojaee S, Chan LN, Yu J, Kornblau SM, Bijl JJ, Ye BH, Mark Ansel K, Paietta E, Melnick A, Hunger SP, Kurre P, Tyner JW, Loh ML, Roeder RG, Druker BJ, Burger JA, Milne TA, Chang BH, Müschen M. Self-Enforcing Feedback Activation between BCL6 and Pre-B Cell Receptor Signaling Defines a Distinct Subtype of Acute Lymphoblastic Leukemia. Cancer Cell. 2015 Mar 9; 27(3):409-25.
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  12. Winter SS, Dunsmore KP, Devidas M, Eisenberg N, Asselin BL, Wood BL, Leonard Rn MS, Murphy J, Gastier-Foster JM, Carroll AJ, Heerema NA, Loh ML, Raetz EA, Winick NJ, Carroll WL, Hunger SP. Safe integration of nelarabine into intensive chemotherapy in newly diagnosed T-cell acute lymphoblastic leukemia: Children's Oncology Group Study AALL0434. Pediatr Blood Cancer. 2015 Jul; 62(7):1176-83.
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  13. Diouf B, Crews KR, Lew G, Pei D, Cheng C, Bao J, Zheng JJ, Yang W, Fan Y, Wheeler HE, Wing C, Delaney SM, Komatsu M, Paugh SW, McCorkle JR, Lu X, Winick NJ, Carroll WL, Loh ML, Hunger SP, Devidas M, Pui CH, Dolan ME, Relling MV, Evans WE. Association of an inherited genetic variant with vincristine-related peripheral neuropathy in children with acute lymphoblastic leukemia. JAMA. 2015 Feb 24; 313(8):815-23.
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  14. Maude SL, Dolai S, Delgado-Martin C, Vincent T, Robbins A, Selvanathan A, Ryan T, Hall J, Wood AC, Tasian SK, Hunger SP, Loh ML, Mullighan CG, Wood BL, Hermiston ML, Grupp SA, Lock RB, Teachey DT. Efficacy of JAK/STAT pathway inhibition in murine xenograft models of early T-cell precursor (ETP) acute lymphoblastic leukemia. Blood. 2015 Mar 12; 125(11):1759-67.
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  15. Niemeyer CM, Loh ML, Cseh A, Cooper T, Dvorak CC, Chan R, Xicoy B, Germing U, Kojima S, Manabe A, Dworzak M, De Moerloose B, Starý J, Smith OP, Masetti R, Catala A, Bergstraesser E, Ussowicz M, Fabri O, Baruchel A, Cavé H, Zwaan M, Locatelli F, Hasle H, van den Heuvel-Eibrink MM, Flotho C, Yoshimi A. Criteria for evaluating response and outcome in clinical trials for children with juvenile myelomonocytic leukemia. Haematologica. 2015 Jan; 100(1):17-22.
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  16. Suryani S, Bracken LS, Harvey RC, Sia KC, Carol H, Chen IM, Evans K, Dietrich PA, Roberts KG, Kurmasheva RT, Billups CA, Mullighan CG, Willman CL, Loh ML, Hunger SP, Houghton PJ, Smith MA, Lock RB. Evaluation of the In Vitro and In Vivo Efficacy of the JAK Inhibitor AZD1480 against JAK-Mutated Acute Lymphoblastic Leukemia. Mol Cancer Ther. 2015 Feb; 14(2):364-74.
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  17. Stieglitz E, Ward AF, Gerbing RB, Alonzo TA, Arceci RJ, Liu YL, Emanuel PD, Widemann BC, Cheng JW, Jayaprakash N, Balis FM, Castleberry RP, Bunin NJ, Loh ML, Cooper TM. Phase II/III trial of a pre-transplant farnesyl transferase inhibitor in juvenile myelomonocytic leukemia: A report from the Children's Oncology Group. Pediatr Blood Cancer. 2015 Apr; 62(4):629-36.
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  18. Perez-Andreu V, Roberts KG, Xu H, Smith C, Zhang H, Yang W, Harvey RC, Payne-Turner D, Devidas M, Cheng IM, Carroll WL, Heerema NA, Carroll AJ, Raetz EA, Gastier-Foster JM, Marcucci G, Bloomfield CD, Mrózek K, Kohlschmidt J, Stock W, Kornblau SM, Konopleva M, Paietta E, Rowe JM, Luger SM, Tallman MS, Dean M, Burchard EG, Torgerson DG, Yue F, Wang Y, Pui CH, Jeha S, Relling MV, Evans WE, Gerhard DS, Loh ML, Willman CL, Hunger SP, Mullighan CG, Yang JJ. A genome-wide association study of susceptibility to acute lymphoblastic leukemia in adolescents and young adults. Blood. 2015 Jan 22; 125(4):680-6.
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  19. Baughn LB, Biegel JA, South ST, Smolarek TA, Volkert S, Carroll AJ, Heerema NA, Rabin KR, Zweidler-McKay PA, Loh M, Hirsch B. Integration of cytogenomic data for furthering the characterization of pediatric B-cell acute lymphoblastic leukemia: a multi-institution, multi-platform microarray study. Cancer Genet. 2015 Jan-Feb; 208(1-2):1-18.
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  20. Salzer WL, Jones TL, Devidas M, Dreyer ZE, Gore L, Winick NJ, Sung L, Raetz E, Loh ML, Wang CY, De Lorenzo P, Valsecchi MG, Pieters R, Carroll WL, Hunger SP, Hilden JM, Brown P. Decreased induction morbidity and mortality following modification to induction therapy in infants with acute lymphoblastic leukemia enrolled on AALL0631: A report from the children's oncology group. Pediatr Blood Cancer. 2015 Mar; 62(3):414-8.
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