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. 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.  View on PubMed
  2. 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.  View on PubMed
  3. Aboian M, Huang SY, Pampaloni MH, Hawkins RA, Huh Y, Vo K, Gustafson C, Matthay K, Seo Y 124I-MIBG PET-CT to monitor metastatic disease in children with relapsed neuroblastoma.  View on PubMed
  4. 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.  View on PubMed
  5. 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.  View on PubMed
  6. 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.  View on PubMed
  7. Sznewajs A, Pon E, Matthay KK Congenital malformation syndromes associated with peripheral neuroblastic tumors: A systematic review.  View on PubMed
  8. 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.  View on PubMed
  9. 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.  View on PubMed
  10. 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.  View on PubMed
  11. 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.  View on PubMed
  12. 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.  View on PubMed
  13. Matthay KK Interleukin 2 plus anti-GD2 immunotherapy: helpful or harmful?  View on PubMed
  14. 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.  View on PubMed
  15. Kumar P, Gill RM, Phelps A, Tulpule A, Matthay K, Nicolaides T Surveillance Screening in Li-Fraumeni Syndrome: Raising Awareness of False Positives.  View on PubMed
  16. 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.  View on PubMed
  17. Tolbert VP, Matthay KK Neuroblastoma: clinical and biological approach to risk stratification and treatment.  View on PubMed
  18. 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.  View on PubMed
  19. 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.  View on PubMed
  20. 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.  View on PubMed

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