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.


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

  • (From 179)
  • Matthay KK, Villablanca JG, Seeger RC, Harris RE, Ramsay NK, Swift P, Shimada H, Black CT, Brodeur GM, Gerbing R, Stram DO, Reynolds CP. Treatment of high-risk neuroblastoma with intensive chemotherapy, radiotherapy, autologous bone marrow transplantation, and 13-cis-retinoic acid. N Engl J Med 1999; 341:1165-1173.
  • Chesler L, Goldenberg D, Schlieve C, Kim G, McMillan A, Kenney A, Matthay KK, Rowitch D, Weiss WA. Inhibition of PI3-kinase destabilizes Mycn protein and blocks malignant progression in neuroblastoma. Cancer Research 2006; 66:8139-8146. PMID16912192
  • Asgharzadeh S, Pique-Regi R, Wang H, Yang Y, Shimada H, Matthay K, Buckley J, Sposto R, Ortega A, Seeger RC. Gene expression profiling of metastatic neuroblastomas without MYCN amplification identifies molecular signatures associated with distinct outcomes. JNCI 2006;98:1193-1203. PMID16954472
  • Matthay KK, Quach A, Franc BL, Huberty J, Hawkins RA, Jackson H, Groshen S, Shusterman S, Yanik G, Veatch J, Brophy P, Villablanca JG, Maris JM. 131I-Metaiodobenzylguanidine (131I-MIBG) double infusion with autologous stem cell rescue for neuroblastoma: A New Approaches to Neuroblastoma Therapy (NANT) phase I study. J Clin Oncol 2009 Mar 1;27(7):1020-5. Epub 2009 Jan 26. PMID19171714
  • Matthay KK, Reynolds CP, Shimada H, Adkins SE, Haas-Kogan D, Gerbing RB, London WB, Villablanca JG. Long-term results for children with high-risk neuroblastoma treated on a randomized trial of myeloablative therapy followed by 13-cis-retinoic acid: A Children’s Oncology Group study. J Clin Oncol 2009 Mar 1;27(7):1007-13. Epub 2009 Jan 26. PMID19171716
  • Minturn JE, Evans AE, Villablanca JG, Yanik GA, Park JR, Shusterman S, Groshen S, Hellriegel ET, Bensen-Kennedy D, Matthay KK, BrodeurGM1*and Maris JM. Phase I Trial of Lestaurtinib for Children with Refractory Neuroblastoma: A New Approaches to Neuroblastoma Therapy Consortium Study. Cancer Chemotherapy & Pharmacology 2011; Feb 22. [Epub ahead of print PMID: 2134060
  • Park JR, Villablanca JG, London WB, Gerbing RB, Haas-Kogan D, Adkins ES, Attiyeh EF, Maris JM, Seeger RC, Reynolds CP, Matthay KK. Favorable outcome of high-risk stage 3 neuroblastoma with myeloablative therapy and 13-cis-retinoic acid: A report from the Children's Oncology Group. Pediatric Blood & Cancer 2009; 52:44-50. Epub ahead of print 2008 Oct 20.
  • Dubois SG, Epling CL, Teague J, Matthay KK, Sinclair E. Flow cytometric detection of Ewing sarcoma cells in peripheral blood and bone marrow. Pediatr Blood Cancer 2010; 54(1):13-8. PMID: 19711435
  • Matthay KK, Shulkin B, Ladenstein R, Michon J, Giammarile F, Lewington V, Pearson ADJ. Cohn SL. Criteria for Evaluation of Disease Extent by 123I-Metaiodobenzylguanidine Scans in Neuroblastoma. A report for the International Neuroblastoma Risk Group (INRG) Task Force. Brit J Cancer 2010; 102:1319-26. PMID: 20424613
  • Yu AL, Gilman AL, Ozkaynak MF, London WB, Kreissman S, Chen H, Smith M, Anderson B, Villablanca JG, Matthay KK, Shimada H, Grupp SA, Seeger RC, Reynolds CP, Buxton A, Reisfeld RA, Gillies SD, Cohn SL, Maris JM, Sondel PM. Chimeric Anti-GD2 Antibody with GM-CSF, IL2 and 13-cis Retinoic Acid for High-risk Neuroblastoma: A Children’s Oncology Group (COG) Phase 3 Study. N Engl J Med 2010; 363:1324-1334. PMID: 20879881
  • Baker DL, Schmidt ML, Cohn SL, Maris JM, London WB, Buxton A, Stram D, Castleberry RP, Shimada H, Sandler A, Shamberger RC, Look AT, Reynolds CP, Seeger RC, Matthay KK. Outcome after reduced therapy for intermediate-risk neuroblastoma. N Engl J Med 2010; 363:1313-1323. PMID: 20879880
  • Lee C-L, Wahnishe H, Sayre GA, Cho H, Kim H-J, Hernandez-Pampaloni JM, Dannoon SF, VanBrocklin HF, Itsara M, Weiss WA, Yang X, Haas-Kogan DA, Matthay KK, Seo Y. Radiation Dose Estimation using Preclinical Imaging with 124I-metaiodobenzylguanidine (MIBG) PET. Medical Physics 2010; 37(9):4861-7. PMID: 20964203
  • More S, Itsara M, Yang X, Geier EG, Tadano MK, Seo Y, VanBrocklin HF, Weiss WA, Mueller S, Haas-Kogan DA, DuBois SG, Matthay KK, Giacomini KM. Vorinostat increases expression of functional norepinephrine transporter in neuroblastoma in vitro and in vivo model systems. Clin Cancer Res 2011; 17:2339-2349.