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

Katherine K. Matthay, MD

Professor, Department of Pediatrics; Leader, Pediatric Malignancies Program, UCSF Helen Diller Family Comprehensive Cancer Center
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-present
    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. Dubois SG, Mosse YP, Fox E, Kudgus RA, Reid JM, McGovern R, Groshen S, Bagatell R, Maris JM, Twist CJ, Goldsmith KC, Granger M, Weiss B, Park JR, Macy ME, Cohn SL, Yanik GA, 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 2 Trial of Alisertib in Combination with Irinotecan and Temozolomide for Patients with Relapsed or Refractory Neuroblastoma. Clin Cancer Res. 2018 Aug 09.
    View on PubMed
  2. 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
  3. 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 Mar 30; e27023.
    View on PubMed
  4. Tolbert VP, Matthay KK. Neuroblastoma: clinical and biological approach to risk stratification and treatment. Cell Tissue Res. 2018 Mar 23.
    View on PubMed
  5. 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
  6. 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.
    View on PubMed
  7. 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 Jan; 2(1):25-34.
    View on PubMed
  8. 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 Jan 01; 24(1):189-196.
    View on PubMed
  9. 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
  10. 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 Mar; 59(3):502-508.
    View on PubMed
  11. 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
  12. Marachelian A, Villablanca JG, Liu CW, Liu B, Goodarzian F, Lai HA, Shimada H, Tran HC, Parra JA, Gallego R, Bedrossian N, Young S, Czarnecki S, Kennedy R, Weiss BD, Goldsmith K, Granger M, Matthay KK, Groshen S, Asgharzadeh S, Sposto R, Seeger RC. Expression of Five Neuroblastoma Genes in Bone Marrow or Blood of Patients with Relapsed/Refractory Neuroblastoma Provides a New Biomarker for Disease and Prognosis. Clin Cancer Res. 2017 Sep 15; 23(18):5374-5383.
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  13. Campbell K, Karski EE, Olow A, Edmondson DA, Kohlgruber AC, Coleman M, Haas-Kogan DA, Matthay KK, DuBois SG. Peripheral Blood Biomarkers Associated With Toxicity and Treatment Characteristics After 131I- Metaiodobenzylguanidine Therapy in Patients With Neuroblastoma. Int J Radiat Oncol Biol Phys. 2017 10 01; 99(2):468-475.
    View on PubMed
  14. Park JR, Bagatell R, Cohn SL, Pearson AD, Villablanca JG, Berthold F, Burchill S, Boubaker A, McHugh K, Nuchtern JG, London WB, Seibel NL, Lindwasser OW, Maris JM, Brock P, Schleiermacher G, Ladenstein R, Matthay KK, Valteau-Couanet D. Revisions to the International Neuroblastoma Response Criteria: A Consensus Statement From the National Cancer Institute Clinical Trials Planning Meeting. J Clin Oncol. 2017 Aug 01; 35(22):2580-2587.
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  15. DuBois SG, Mody R, Naranjo A, Van Ryn C, Russ D, Oldridge D, Kreissman S, Baker DL, Parisi M, Shulkin BL, Bai H, Diskin SJ, Batra V, Maris JM, Park JR, Matthay KK, Yanik G. MIBG avidity correlates with clinical features, tumor biology, and outcomes in neuroblastoma: A report from the Children's Oncology Group. Pediatr Blood Cancer. 2017 Nov; 64(11).
    View on PubMed
  16. Vo KT, Karski EE, Nasholm NM, Allen S, Hollinger F, Gustafson WC, Long-Boyle JR, Shiboski S, Matthay KK, DuBois SG. Phase 1 study of sirolimus in combination with oral cyclophosphamide and topotecan in children and young adults with relapsed and refractory solid tumors. Oncotarget. 2017 Apr 04; 8(14):23851-23861.
    View on PubMed
  17. Dinh T, Truong B, Matthay KK, DuBois SG. Comparison of Clinical Features and Outcomes in Patients With Bilateral Versus Unilateral Adrenal Neuroblastoma. J Pediatr Hematol Oncol. 2017 Mar; 39(2):108-113.
    View on PubMed
  18. Matthay KK, Maris JM, Schleiermacher G, Nakagawara A, Mackall CL, Diller L, Weiss WA. Neuroblastoma. Nat Rev Dis Primers. 2016 11 10; 2:16078.
    View on PubMed
  19. Li R, Polishchuk A, DuBois S, Hawkins R, Lee SW, Bagatell R, Shusterman S, Hill-Kayser C, Al-Sayegh H, Diller L, Haas-Kogan DA, Matthay KK, London WB, Marcus KJ. Patterns of Relapse in High-Risk Neuroblastoma Patients Treated With and Without Total Body Irradiation. Int J Radiat Oncol Biol Phys. 2017 02 01; 97(2):270-277.
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  20. Huibregtse KE, Vo KT, DuBois SG, Fetzko S, Neuhaus J, Batra V, Maris JM, Weiss B, Marachelian A, Yanik GA, Matthay KK. Incidence and risk factors for secondary malignancy in patients with neuroblastoma after treatment with (131)I-metaiodobenzylguanidine. Eur J Cancer. 2016 10; 66:144-52.
    View on PubMed

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