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

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

View on UCSF Profiles

Cancer Center Membership

Program Member » 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.


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. Pinto NR, Applebaum MA, Volchenboum SL, Matthay KK, London WB, Ambros PF, Nakagawara A, Berthold F, Schleiermacher G, Park JR, Valteau-Couanet D, Pearson AD, Cohn SL. Advances in Risk Classification and Treatment Strategies for Neuroblastoma. J Clin Oncol. 2015 Sep 20; 33(27):3008-17.
    View on PubMed
  2. Zhou MJ, Doral MY, DuBois SG, Villablanca JG, Yanik GA, Matthay KK. Different outcomes for relapsed versus refractory neuroblastoma after therapy with (131)I-metaiodobenzylguanidine ((131)I-MIBG). Eur J Cancer. 2015 Nov; 51(16):2465-72.
    View on PubMed
  3. Anderson CP, Matthay KK, Perentesis JP, Neglia JP, Bailey HH, Villablanca JG, Groshen S, Hasenauer B, Maris JM, Seeger RC, Reynolds CP. Pilot study of intravenous melphalan combined with continuous infusion L-S,R-buthionine sulfoximine for children with recurrent neuroblastoma. Pediatr Blood Cancer. 2015 Oct; 62(10):1739-46.
    View on PubMed
  4. Huang SY, Bolch WE, Lee C, Van Brocklin HF, Pampaloni MH, Hawkins RA, Sznewajs A, DuBois SG, Matthay KK, Seo Y. Patient-Specific Dosimetry Using Pretherapy [(124)I]m-iodobenzylguanidine ([ (124)I]mIBG) Dynamic PET/CT Imaging Before [ (131)I]mIBG Targeted Radionuclide Therapy for Neuroblastoma. Mol Imaging Biol. 2015 Apr; 17(2):284-94.
    View on PubMed
  5. Parikh NS, Howard SC, Chantada G, Israels T, Khattab M, Alcasabas P, Lam CG, Faulkner L, Park JR, London WB, Matthay KK. SIOP-PODC adapted risk stratification and treatment guidelines: Recommendations for neuroblastoma in low- and middle-income settings. Pediatr Blood Cancer. 2015 Aug; 62(8):1305-16.
    View on PubMed
  6. DuBois SG, Groshen S, Park JR, Haas-Kogan DA, Yang X, Geier E, Chen E, Giacomini K, Weiss B, Cohn SL, Granger MM, Yanik GA, Hawkins R, Courtier J, Jackson H, Goodarzian F, Shimada H, Czarnecki S, Tsao-Wei D, Villablanca JG, Marachelian A, Matthay KK. Phase I Study of Vorinostat as a Radiation Sensitizer with 131I-Metaiodobenzylguanidine (131I-MIBG) for Patients with Relapsed or Refractory Neuroblastoma. Clin Cancer Res. 2015 Jun 15; 21(12):2715-21.
    View on PubMed
  7. Yanik GA, Villablanca JG, Maris JM, Weiss B, Groshen S, Marachelian A, Park JR, Tsao-Wei D, Hawkins R, Shulkin BL, Jackson H, Goodarzian F, Shimada H, Courtier J, Hutchinson R, Haas-Koga D, Hasenauer CB, Czarnecki S, Katzenstein HM, Matthay KK. (131)I-Metaiodobenzylguanidine with Intensive Chemotherapy and Autologous Stem Cell Transplantation for High-Risk Neuroblastoma. A New Approaches to Neuroblastoma Therapy (NANT) Phase II Study. Biol Blood Marrow Transplant. 2015 Apr; 21(4):673-81.
    View on PubMed
  8. DuBois SG, Allen S, Bent M, Hilton JF, Hollinger F, Hawkins R, Courtier J, Mosse YP, Matthay KK. Phase I/II study of (131)I-MIBG with vincristine and 5 days of irinotecan for advanced neuroblastoma. Br J Cancer. 2015 Feb 17; 112(4):644-9.
    View on PubMed
  9. Matthay KK, London WB, Maris J, Adamson PC, Park JR. Reply to N.-K.v. Cheung et Al. J Clin Oncol. 2014 Dec 20; 32(36):4174-5.
    View on PubMed
  10. Thampi S, Matthay KK, Boscardin WJ, Goldsby R, DuBois SG. Clinical Features and Outcomes Differ between Skeletal and Extraskeletal Osteosarcoma. Sarcoma. 2014; 2014:902620.
    View on PubMed
  11. Gustafson WC, Meyerowitz JG, Nekritz EA, Chen J, Benes C, Charron E, Simonds EF, Seeger R, Matthay KK, Hertz NT, Eilers M, Shokat KM, Weiss WA. Drugging MYCN through an allosteric transition in Aurora kinase A. Cancer Cell. 2014 Sep 8; 26(3):414-27.
    View on PubMed
  12. Vo KT, Matthay KK, Neuhaus J, London WB, Hero B, Ambros PF, Nakagawara A, Miniati D, Wheeler K, Pearson AD, Cohn SL, DuBois SG. Clinical, biologic, and prognostic differences on the basis of primary tumor site in neuroblastoma: a report from the international neuroblastoma risk group project. J Clin Oncol. 2014 Oct 1; 32(28):3169-76.
    View on PubMed
  13. Zinter MS, DuBois SG, Spicer A, Matthay K, Sapru A. Pediatric cancer type predicts infection rate, need for critical care intervention, and mortality in the pediatric intensive care unit. Intensive Care Med. 2014 Oct; 40(10):1536-44.
    View on PubMed
  14. Meany HJ, London WB, Ambros PF, Matthay KK, Monclair T, Simon T, Garaventa A, Berthold F, Nakagawara A, Cohn SL, Pearson AD, Park JR. Significance of clinical and biologic features in Stage 3 neuroblastoma: a report from the International Neuroblastoma Risk Group project. Pediatr Blood Cancer. 2014 Nov; 61(11):1932-9.
    View on PubMed
  15. Baggott C, Cooper BA, Marina N, Matthay KK, Miaskowski C. Symptom assessment in pediatric oncology: how should concordance between children's and parents' reports be evaluated? Cancer Nurs. 2014 Jul-Aug; 37(4):252-62.
    View on PubMed
  16. Polishchuk AL, Li R, Hill-Kayser C, Little A, Hawkins RA, Hamilton J, Lau M, Tran HC, Strahlendorf C, Lemons RS, Weinberg V, Matthay KK, DuBois SG, Marcus KJ, Bagatell R, Haas-Kogan DA. Likelihood of bone recurrence in prior sites of metastasis in patients with high-risk neuroblastoma. Int J Radiat Oncol Biol Phys. 2014 Jul 15; 89(4):839-45.
    View on PubMed
  17. Kang MH, Villablanca JG, Bender JL, Matthay KK, Groshen S, Sposto R, Czarnecki S, Ames MM, Reynolds CP, Marachelian A, Maurer BJ. Probable fatal drug interaction between intravenous fenretinide, ceftriaxone, and acetaminophen: a case report from a New Approaches to Neuroblastoma (NANT) Phase I study. BMC Res Notes. 2014; 7:256.
    View on PubMed
  18. Morgenstern DA, London WB, Stephens D, Volchenboum SL, Hero B, Di Cataldo A, Nakagawara A, Shimada H, Ambros PF, Matthay KK, Cohn SL, Pearson AD, Irwin MS. Metastatic neuroblastoma confined to distant lymph nodes (stage 4N) predicts outcome in patients with stage 4 disease: A study from the International Neuroblastoma Risk Group Database. J Clin Oncol. 2014 Apr 20; 32(12):1228-35.
    View on PubMed
  19. Mossé YP, Deyell RJ, Berthold F, Nagakawara A, Ambros PF, Monclair T, Cohn SL, Pearson AD, London WB, Matthay KK. Neuroblastoma in older children, adolescents and young adults: a report from the International Neuroblastoma Risk Group project. Pediatr Blood Cancer. 2014 Apr; 61(4):627-35.
    View on PubMed
  20. Kersten E, Scanlan P, Dubois SG, Matthay KK. Current treatment and outcome for childhood acute leukemia in Tanzania. Pediatr Blood Cancer. 2013 Dec; 60(12):2047-53.
    View on PubMed

Go to UCSF Profiles, powered by CTSI