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.

Research Funding

  • August 5, 1999 - May 31, 2023 - Cancer Center Support Grant , Co-Investigator . Sponsor: NIH, Sponsor Award ID: P30CA082103
  • July 6, 2000 - May 31, 2015 - Biology and Therapy of High Risk Neuroblastoma , Co-Investigator . Sponsor: NIH/NCI, Sponsor Award ID: P01CA081403
  • December 1, 1981 - March 31, 2007 - PEDIATRIC CLINICAL RESEARCH CENTER , Co-Investigator . Sponsor: NIH, Sponsor Award ID: M01RR001271
  • June 20, 2003 - May 31, 2006 - 131 I-MIBG Escalating Dose Rapid Sequence Double Infusi* , Principal Investigator . Sponsor: NIH, Sponsor Award ID: R21CA097758


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

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. Rosenberg MI, Greenstein E, Buchkovich M, Peres A, Santoni-Rugiu E, Yang L, Mikl M, Vaksman Z, Gibbs DL, Reshef D, Salovin A, Irwin MS, Naranjo A, Ulitsky I, de Alarcon PA, Matthay KK, Weigman V, Yaari G, Panzer JA, Friedman N, Maris JM. Polyclonal lymphoid expansion drives paraneoplastic autoimmunity in neuroblastoma. Cell Rep. 2023 08 29; 42(8):112879.  View on PubMed
  2. Streby KA, Parisi MT, Shulkin BL, LaBarre B, Bagatell R, Diller L, Grupp SA, Matthay KK, Voss SD, Yu AL, London WB, Park JR, Yanik GA, Naranjo A. Impact of diagnostic and end-of-induction Curie scores with tandem high-dose chemotherapy and autologous transplants for metastatic high-risk neuroblastoma: A report from the Children's Oncology Group. Pediatr Blood Cancer. 2023 Aug; 70(8):e30418.  View on PubMed
  3. Goldsmith KC, Park JR, Kayser K, Malvar J, Chi YY, Groshen SG, Villablanca JG, Krytska K, Lai LM, Acharya PT, Goodarzian F, Pawel B, Shimada H, Ghazarian S, States L, Marshall L, Chesler L, Granger M, Desai AV, Mody R, Morgenstern DA, Shusterman S, Macy ME, Pinto N, Schleiermacher G, Vo K, Thurm HC, Chen J, Liyanage M, Peltz G, Matthay KK, Berko ER, Maris JM, Marachelian A, Mossé YP. Lorlatinib with or without chemotherapy in ALK-driven refractory/relapsed neuroblastoma: phase 1 trial results. Nat Med. 2023 05; 29(5):1092-1102.  View on PubMed
  4. Campbell K, Groshen S, Evans AC, Wilson S, Sebastian A, Loots GG, Marachelian A, Armant M, Pal S, Haas-Kogan DA, Park JR, Granger M, Matthay KK, Coleman MA, DuBois SG. Modulation of Radiation Biomarkers in a Randomized Phase II Study of 131I-MIBG With or Without Radiation Sensitizers for Relapsed or Refractory Neuroblastoma. Int J Radiat Oncol Biol Phys. 2023 04 01; 115(5):1115-1128.  View on PubMed
  5. Park JR, Villablanca JG, Hero B, Kushner BH, Wheatley K, Beiske KH, Ladenstein RL, Baruchel S, Macy ME, Moreno L, Seibel NL, Pearson AD, Matthay KK, Valteau-Couanet D. Early-phase clinical trial eligibility and response evaluation criteria for refractory, relapsed, or progressive neuroblastoma: A consensus statement from the National Cancer Institute Clinical Trials Planning Meeting. Cancer. 2022 Nov 01; 128(21):3775-3783.  View on PubMed
  6. Desai AV, Gilman AL, Ozkaynak MF, Naranjo A, London WB, Tenney SC, Diccianni M, Hank JA, Parisi MT, Shulkin BL, Smith M, Moscow JA, Shimada H, Matthay KK, Cohn SL, Maris JM, Bagatell R, Sondel PM, Park JR, Yu AL. Outcomes Following GD2-Directed Postconsolidation Therapy for Neuroblastoma After Cessation of Random Assignment on ANBL0032: A Report From the Children's Oncology Group. J Clin Oncol. 2022 Dec 10; 40(35):4107-4118.  View on PubMed
  7. Qiu B, Matthay KK. Advancing therapy for neuroblastoma. Nat Rev Clin Oncol. 2022 08; 19(8):515-533.  View on PubMed
  8. Vo KT, DuBois SG, Neuhaus J, Braunstein SE, Weil BR, Naranjo A, Irtan S, Balaguer J, Matthay KK. Pattern and predictors of sites of relapse in neuroblastoma: A report from the International Neuroblastoma Risk Group (INRG) project. Pediatr Blood Cancer. 2022 09; 69(9):e29616.  View on PubMed
  9. Evans AC, Setzkorn T, Edmondson DA, Segelke H, Wilson PF, Matthay KK, Granger MM, Marachelian A, Haas-Kogan DA, DuBois SG, Coleman MA. Peripheral Blood Transcript Signatures after Internal 131I-mIBG Therapy in Relapsed and Refractory Neuroblastoma Patients Identifies Early and Late Biomarkers of Internal 131I Exposures. Radiat Res. 2022 02 01; 197(2):101-112.  View on PubMed
  10. Matthay KK, Hylton J, Penumarthy N, Khattab M, Soh SY, Nguyen HTK, Alcasabas AP, Fawzy M, Saab R, Khan MS, Ghandour K, Chantada G, Parikh NS, Faulkner L, Lam CG, Howard SC. Global Neuroblastoma Network: An international multidisciplinary neuroblastoma tumor board for resource-limited countries. Pediatr Blood Cancer. 2022 04; 69(4):e29568.  View on PubMed
  11. Fishel Ben Kenan R, Polishchuk AL, Hawkins RA, Braunstein SE, Matthay KK, DuBois SG, Haas-Kogan DA. Anatomic patterns of relapse and progression following treatment with 131 I-MIBG in relapsed or refractory neuroblastoma. Pediatr Blood Cancer. 2022 02; 69(2):e29396.  View on PubMed
  12. DuBois SG, Granger MM, Groshen S, Tsao-Wei D, Ji L, Shamirian A, Czarnecki S, Goodarzian F, Berkovich R, Shimada H, Villablanca JG, Vo KT, Pinto N, Mosse YP, Maris JM, Shusterman S, Cohn SL, Goldsmith KC, Weiss B, Yanik GA, Twist CJ, Irwin MS, Haas-Kogan DA, Park JR, Marachelian A, Matthay KK. Randomized Phase II Trial of MIBG Versus MIBG, Vincristine, and Irinotecan Versus MIBG and Vorinostat for Patients With Relapsed or Refractory Neuroblastoma: A Report From NANT Consortium. J Clin Oncol. 2021 11 01; 39(31):3506-3514.  View on PubMed
  13. Weiss BD, Yanik G, Naranjo A, Zhang FF, Fitzgerald W, Shulkin BL, Parisi MT, Russell H, Grupp S, Pater L, Mattei P, Mosse Y, Lai HA, Jarzembowski JA, Shimada H, Villablanca JG, Giller R, Bagatell R, Park JR, Matthay KK. A safety and feasibility trial of 131 I-MIBG in newly diagnosed high-risk neuroblastoma: A Children's Oncology Group study. Pediatr Blood Cancer. 2021 10; 68(10):e29117.  View on PubMed
  14. Yu AL, Gilman AL, Ozkaynak MF, Naranjo A, Diccianni MB, Gan J, Hank JA, Batova A, London WB, Tenney SC, Smith M, Shulkin BL, Parisi M, Matthay KK, Cohn SL, Maris JM, Bagatell R, Park JR, Sondel PM. Long-Term Follow-up of a Phase III Study of ch14.18 (Dinutuximab) + Cytokine Immunotherapy in Children with High-Risk Neuroblastoma: COG Study ANBL0032. Clin Cancer Res. 2021 04 15; 27(8):2179-2189.  View on PubMed
  15. Temple WC, Vo KT, Matthay KK, Balliu B, Coleman C, Michlitsch J, Phelps A, Behr S, Zapala MA. Association of image-defined risk factors with clinical features, histopathology, and outcomes in neuroblastoma. Cancer Med. 2021 04; 10(7):2232-2241.  View on PubMed
  16. Lavergne V, Sabnis A, Tupule A, Davidson PR, Kline C, Matthay K, Nicolaides T, Goldsby R, Braunstein S, Fogh SE, Sneed PK, Menzel P, Nakamura A, DuBois SG, Haas-Kogan DA, Nakamura JL. Germline MUTYH Mutation in a Pediatric Cancer Survivor Developing a Secondary Malignancy. J Pediatr Hematol Oncol. 2020 10; 42(7):e647-e654.  View on PubMed
  17. Aboian MS, Huang SY, Hernandez-Pampaloni M, Hawkins RA, VanBrocklin HF, Huh Y, Vo KT, Gustafson WC, Matthay KK, Seo Y. 124I-MIBG PET/CT to Monitor Metastatic Disease in Children with Relapsed Neuroblastoma. J Nucl Med. 2021 01; 62(1):43-47.  View on PubMed
  18. 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 11 01; 111(11):1192-1201.  View on PubMed
  19. Chévez-Barrios P, Eagle RC, Krailo M, Piao J, Albert DM, Gao Y, Vemuganti G, Ali MJ, Khetan V, Honavar SG, O'Brien J, Leahey AM, Matthay K, Meadows A, Chintagumpala M. Study of Unilateral Retinoblastoma With and Without Histopathologic High-Risk Features and the Role of Adjuvant Chemotherapy: A Children's Oncology Group Study. J Clin Oncol. 2019 11 01; 37(31):2883-2891.  View on PubMed
  20. 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 12 01; 37(34):3243-3255.  View on PubMed

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