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

  • July 6, 2000 - May 31, 2015 - Biology and Therapy of High Risk Neuroblastoma, Co-Investigator. Sponsor: NIH/NCI, Sponsor Award ID: P01CA081403
  • August 5, 1999 - September 29, 2012 - Cancer Center Support Grant, Co-Investigator. Sponsor: NIH/NCI, Sponsor Award ID: P30CA082103
  • December 1, 1981 - September 29, 2006 - PEDIATRIC CLINICAL RESEARCH CENTER, Co-Investigator. Sponsor: NIH/NCRR, Sponsor Award ID: M01RR001271
  • June 20, 2003 - May 31, 2006 - 131 I-MIBG Escalating Dose Rapid Sequence Double Infusi*, Principal Investigator. Sponsor: NIH/NCI, Sponsor Award ID: R21CA097758

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

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, 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 Jul 16; JCO2100703.  View on PubMed
  2. 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 Oct; 68(10):e29117.  View on PubMed
  3. 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 Apr 15; 27(8):2179-2189.  View on PubMed
  4. 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
  5. 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
  6. 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
  7. 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
  8. 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
  9. 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
  10. Sznewajs A, Pon E, Matthay KK. Congenital malformation syndromes associated with peripheral neuroblastic tumors: A systematic review. Pediatr Blood Cancer. 2019 10; 66(10):e27901.  View on PubMed
  11. 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 10; 25(10):2031-2039.  View on PubMed
  12. 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 08; 66(8):e27819.  View on PubMed
  13. 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 07; 66(7):e27736.  View on PubMed
  14. 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 May; 46(5):2477-2486.  View on PubMed
  15. 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 01 10; 37(2):115-124.  View on PubMed
  16. Matthay KK. Interleukin 2 plus anti-GD2 immunotherapy: helpful or harmful? Lancet Oncol. 2018 12; 19(12):1549-1551.  View on PubMed
  17. 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 12 15; 24(24):6142-6149.  View on PubMed
  18. 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
  19. 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 07; 65(7):e27023.  View on PubMed
  20. 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

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