University of California San Francisco
Helen Diller Family Comprehensive Cancer Center
Katherine K. Matthay, MD

Katherine K. Matthay, MD

Professor, Department of Pediatrics, UCSF
Mildred V. Strouss Endowed Chair in Translational Research in Pediatric Oncology, UCSF

Cancer Center Program Memberships

Pediatric Malignancies

Research Summary

I am professor of pediatrics at the UCSF School of Medicine. I am the leader of the Pediatric Malignancies Program of the Helen Diller Family Comprehensive Cancer Center at UCSF and am one of the leading translational investigators in neuroblastoma therapy internationally. I have extensive experience over the last 25 years in clinical trials and translational research in neuroblastoma and pediatric oncology. I was the chair of the Neuroblastoma Strategy Group in the Children’s Cancer Group for ten years and continue on the Neuroblastoma Steering committee. In this role, I initiated several landmark randomized trials in high-risk neuroblastoma, improving outcome with myeloablative therapy and with two biologic targeted therapies. I am the Principal Investigator for the UCSF Alex Lemonade Center of Excellence for Developmental Therapeutics. I have established and lead the NCI-funded NANT clinical trials consortium for targeted therapy of neuroblastoma, now in its third 5-year cycle. I have been a leader in the development of response evaluation internationally and targeted radiotherapy with the radiopharmaceutical, metaiodobenzylguanidine (131I-mIBG). I have led multiple phase I, II, and III clinical trials for advanced neuroblastoma, as well as collaborative work in pre-clinical studies of combination targeted therapy, mechanisms of MIBG uptake, and MIBG imaging for dosimetry and prognosis. I have also collaborated closely on preclinical work in MYCN and PI3 kinase pathways.

Education

Bryn Mawr College, Bryn Mawr, PA, B.A., 1969, Chemistry
University of Pennsylvania, Philadelphia, PA, M.D., 1973, Medicine
University of Colorado, Denver, CO, Resident, 1976, Pediatrics
University of California, San Francisco, CA, Fellow, 1979, Pediatric Hematology/Oncology


Professional Experience

  • 1980-87
    Assistant Professor in Residence, Pediatrics, Univ. of Calif., San Francisco
  • 1987-93
    Associate Professor in Residence, Pediatrics, Univ. of Calif., San Francisco
  • 1991-2001
    Chair, Neuroblastoma Strategy Group, Children's Cancer Group
  • 1992-present
    Chief, Pediatric Clinical Oncology, Univ. of California, San Francisco
  • 1993-present
    Professor, Pediatrics, Univ. of California, San Francisco
  • 2000-2001; 2009-10
    Visiting Professor, Institut Curie, Paris
  • 2003-2006
    Fellowship Director, Pediatric Hematology/Oncology, Univ. of California, San Francisco
  • 2003-2013
    Division Chief, Pediatric Hematology/Oncology, Univ. of California, San Francisco
  • 2005-2019
    Leader, Pediatric Malignancies Program in the HDF Comprehensive Cancer Center

Honors & Awards

  • 1992
    American Cancer Society Scholar Award
  • 2000-01
    Bourse Henri de Rothschild for Neuroblastoma Research Award
  • 2006
    Mildred V. Strouss Endowed Chair in Translational Research
  • 2006-12
    SIOP North American continental President
  • 2007
    Hadassah Doctor of Distinction Award
  • 2012-16
    President, Advances in Neuroblastoma Research Association

Selected Publications

  1. Twist CJ, Schmidt ML, Naranjo A, London WB, Tenney SC, Marachelian A, Shimada H, Collins MH, Esiashvili N, Adkins ES, Mattei P, Handler M, Katzenstein H, Attiyeh E, Hogarty MD, Gastier-Foster J, Wagner E, Matthay KK, Park JR, Maris JM, Cohn SL. Maintaining Outstanding Outcomes Using Response- and Biology-Based Therapy for Intermediate-Risk Neuroblastoma: A Report From the Children's Oncology Group Study ANBL0531. J Clin Oncol. 2019 Aug 06; JCO1900919.
    View on PubMed
  2. Sznewajs A, Pon E, Matthay KK. Congenital malformation syndromes associated with peripheral neuroblastic tumors: A systematic review. Pediatr Blood Cancer. 2019 Jul 02; e27901.
    View on PubMed
  3. Tolbert VP, Dvorak CC, Golden C, Vissa M, El-Haj N, Perwad F, Matthay KK, Vo KT. Risk Factors for Transplant-Associated Thrombotic Microangiopathy after Autologous Hematopoietic Cell Transplant in High-Risk Neuroblastoma. Biol Blood Marrow Transplant. 2019 Jun 12.
    View on PubMed
  4. Campbell K, Shyr D, Bagatell R, Fischer M, Nakagawara A, Nieto AC, Brodeur GM, Matthay KK, London WB, DuBois SG. Comprehensive evaluation of context dependence of the prognostic impact of MYCN amplification in neuroblastoma: A report from the International Neuroblastoma Risk Group (INRG) project. Pediatr Blood Cancer. 2019 Aug; 66(8):e27819.
    View on PubMed
  5. Braunstein SE, London WB, Kreissman SG, Villablanca JG, Davidoff AM, DeSantes K, Castleberry RP, Murray K, Diller L, Matthay K, Cohn SL, Shulkin B, von Allmen D, Parisi MT, Van Ryn C, Park JR, La Quaglia MP, Haas-Kogan DA. Role of the extent of prophylactic regional lymph node radiotherapy on survival in high-risk neuroblastoma: A report from the COG A3973 study. Pediatr Blood Cancer. 2019 Jul; 66(7):e27736.
    View on PubMed
  6. Oldridge DA, Truong B, Russ D, DuBois SG, Vaksman Z, Mosse YP, Diskin SJ, Maris JM, Matthay KK. Differences in Genomic Profiles and Outcomes between Thoracic and Adrenal Neuroblastoma. J Natl Cancer Inst. 2019 Feb 21.
    View on PubMed
  7. Seo Y, Huh Y, Huang SY, Hernandez-Pampaloni JM, Hawkins RA, Gustafson WC, Vo KT, Matthay KK. Technical Note: Simplified and practical pretherapy tumor dosimetry - A feasibility study for 131 I-MIBG therapy of neuroblastoma using 124 I-MIBG PET/CT. Med Phys. 2019 Feb 13.
    View on PubMed
  8. Twist CJ, Naranjo A, Schmidt ML, Tenney SC, Cohn SL, Meany HJ, Mattei P, Adkins ES, Shimada H, London WB, Park JR, Matthay KK, Maris JM. Defining Risk Factors for Chemotherapeutic Intervention in Infants With Stage 4S Neuroblastoma: A Report From Children's Oncology Group Study ANBL0531. J Clin Oncol. 2019 Jan 10; 37(2):115-124.
    View on PubMed
  9. Matthay KK. Interleukin 2 plus anti-GD2 immunotherapy: helpful or harmful? Lancet Oncol. 2018 Dec; 19(12):1549-1551.
    View on PubMed
  10. DuBois SG, Mosse YP, Fox E, Kudgus RA, Reid JM, McGovern R, Groshen S, Bagatell R, Maris JM, Twist CJ, Goldsmith K, Granger MM, Weiss B, Park JR, Macy ME, Cohn SL, Yanik G, Wagner LM, Hawkins R, Courtier J, Lai H, Goodarzian F, Shimada H, Boucher N, Czarnecki S, Luo C, Tsao-Wei D, Matthay KK, Marachelian A. Phase II Trial of Alisertib in Combination with Irinotecan and Temozolomide for Patients with Relapsed or Refractory Neuroblastoma. Clin Cancer Res. 2018 Dec 15; 24(24):6142-6149.
    View on PubMed
  11. Kumar P, Gill RM, Phelps A, Tulpule A, Matthay K, Nicolaides T. Surveillance Screening in Li-Fraumeni Syndrome: Raising Awareness of False Positives. Cureus. 2018 Apr 24; 10(4):e2527.
    View on PubMed
  12. Pinto N, DuBois SG, Marachelian A, Diede SJ, Taraseviciute A, Glade Bender JL, Tsao-Wei D, Groshen SG, Reid JM, Haas-Kogan DA, Reynolds CP, Kang MH, Irwin MS, Macy ME, Villablanca JG, Matthay KK, Park JR. Phase I study of vorinostat in combination with isotretinoin in patients with refractory/recurrent neuroblastoma: A new approaches to Neuroblastoma Therapy (NANT) trial. Pediatr Blood Cancer. 2018 Jul; 65(7):e27023.
    View on PubMed
  13. Tolbert VP, Matthay KK. Neuroblastoma: clinical and biological approach to risk stratification and treatment. Cell Tissue Res. 2018 05; 372(2):195-209.
    View on PubMed
  14. Villablanca JG, Ji L, Shapira-Lewinson A, Marachelian A, Shimada H, Hawkins RA, Pampaloni M, Lai H, Goodarzian F, Sposto R, Park JR, Matthay KK. Predictors of response, progression-free survival, and overall survival using NANT Response Criteria (v1.0) in relapsed and refractory high-risk neuroblastoma. Pediatr Blood Cancer. 2018 May; 65(5):e26940.
    View on PubMed
  15. Kondapavulur S, Cooke DL, Kao A, Amans MR, Alexander M, Darflinger R, Dowd CF, Higashida RT, Damato B, Halbach VV, Matthay KK, Hetts SW. Estimation of intra-arterial chemotherapy distribution to the retina in pediatric retinoblastoma patients using quantitative digital subtraction angiography. Interv Neuroradiol. 2018 Apr; 24(2):214-219.
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  16. de Alarcon PA, Matthay KK, London WB, Naranjo A, Tenney SC, Panzer JA, Hogarty MD, Park JR, Maris JM, Cohn SL. Intravenous immunoglobulin with prednisone and risk-adapted chemotherapy for children with opsoclonus myoclonus ataxia syndrome associated with neuroblastoma (ANBL00P3): a randomised, open-label, phase 3 trial. Lancet Child Adolesc Health. 2018 01; 2(1):25-34.
    View on PubMed
  17. Erbe AK, Wang W, Carmichael L, Kim K, Mendonça EA, Song Y, Hess D, Reville PK, London WB, Naranjo A, Hank JA, Diccianni MB, Reisfeld RA, Gillies SD, Matthay KK, Cohn SL, Hogarty MD, Maris JM, Park JR, Ozkaynak MF, Gilman AL, Yu AL, Sondel PM. Neuroblastoma Patients' KIR and KIR-Ligand Genotypes Influence Clinical Outcome for Dinutuximab-based Immunotherapy: A Report from the Children's Oncology Group. Clin Cancer Res. 2018 01 01; 24(1):189-196.
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  18. Ladenstein R, Lambert B, Pötschger U, Castellani MR, Lewington V, Bar-Sever Z, Oudoux A, Sliwinska A, Taborska K, Biassoni L, Yanik GA, Naranjo A, Parisi MT, Shulkin BL, Nadel H, Gelfand MJ, Matthay KK, Park JR, Kreissman SG, Valteau-Couanet D, Boubaker A. Validation of the mIBG skeletal SIOPEN scoring method in two independent high-risk neuroblastoma populations: the SIOPEN/HR-NBL1 and COG-A3973 trials. Eur J Nucl Med Mol Imaging. 2018 Feb; 45(2):292-305.
    View on PubMed
  19. Yanik GA, Parisi MT, Naranjo A, Nadel H, Gelfand MJ, Park JR, Ladenstein RL, Poetschger U, Boubaker A, Valteau-Couanet D, Lambert B, Castellani MR, Bar-Sever Z, Oudoux A, Kaminska A, Kreissman SG, Shulkin BL, Matthay KK. Validation of Postinduction Curie Scores in High-Risk Neuroblastoma: A Children's Oncology Group and SIOPEN Group Report on SIOPEN/HR-NBL1. J Nucl Med. 2018 03; 59(3):502-508.
    View on PubMed
  20. Campbell K, Gastier-Foster JM, Mann M, Naranjo AH, Van Ryn C, Bagatell R, Matthay KK, London WB, Irwin MS, Shimada H, Granger MM, Hogarty MD, Park JR, DuBois SG. Association of MYCN copy number with clinical features, tumor biology, and outcomes in neuroblastoma: A report from the Children's Oncology Group. Cancer. 2017 Nov 01; 123(21):4224-4235.
    View on PubMed

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