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
  • January 1, 1978 - November 30, 2002 - CHILDRENS CANCER GROUP , Principal Investigator . Sponsor: NIH, Sponsor Award ID: U10CA017829
  • June 1, 1985 - November 30, 1992 - TREATMENT OF LEUKEMIA BY ANTIBODY-DIRECTED LIPOSOMES , Principal Investigator . Sponsor: NIH, Sponsor Award ID: R01CA039448

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
  • 2018
    Lifetime Achievement award, Advances in Neuroblastoma Research Association
  • 2023
    Lifetime Achievement award, International Society of Pediatric Oncology

Selected Publications

  1. Chesler L, Schlieve C, Goldenberg DD, Kenney A, Kim G, McMillan A, Matthay KK, Rowitch D, Weiss WA. Editor's Note: Inhibition of Phosphatidylinositol 3-Kinase Destabilizes Mycn Protein and Blocks Malignant Progression in Neuroblastoma. Cancer Res. 2024 Oct 01; 84(19):3311.  View on PubMed
  2. Kumar P, Koach J, Nekritz E, Mukherjee S, Braun BS, DuBois SG, Nasholm N, Haas-Kogan D, Matthay KK, Weiss WA, Gustafson C, Seo Y. Aurora Kinase A inhibition enhances DNA damage and tumor cell death with 131I-MIBG therapy in high-risk neuroblastoma. EJNMMI Res. 2024 Jun 13; 14(1):54.  View on PubMed
  3. Barr EK, Naranjo A, Twist CJ, Tenney SC, Schmidt ML, London WB, Gastier-Foster J, Adkins ES, Mattei P, Handler MH, Matthay KK, Park JR, Maris JM, Desai AV, Cohn SL. Long-term follow-up of patients with intermediate-risk neuroblastoma treated with response- and biology-based therapy: A report from the Children's Oncology Group study ANBL0531. Pediatr Blood Cancer. 2024 Aug; 71(8):e31089.  View on PubMed
  4. Kumar P, Willard VW, Embry L, Naranjo A, LaBarre B, Matthay KK, de Alarcon PA. Late cognitive and adaptive outcomes of patients with neuroblastoma-associated opsoclonus-myoclonus-ataxia-syndrome: A report from the Children's Oncology Group. Pediatr Blood Cancer. 2024 Jul; 71(7):e31039.  View on PubMed
  5. Huang M, Fang W, Farrel A, Li L, Chronopoulos A, Nasholm N, Cheng B, Zheng T, Yoda H, Barata MJ, Porras T, Miller ML, Zhen Q, Ghiglieri L, McHenry L, Wang L, Asgharzadeh S, Park J, Gustafson WC, Matthay KK, Maris JM, Weiss WA. ALK upregulates POSTN and WNT signaling to drive neuroblastoma. Cell Rep. 2024 03 26; 43(3):113927.  View on PubMed
  6. Kumar P, Koach J, Nekritz E, Mukherjee S, Braun BS, DuBois SG, Nasholm N, Haas-Kogan D, Matthay KK, Weiss WA, Gustafson C, Seo Y. Aurora Kinase A inhibition enhances DNA damage and tumor cell death with 131I-MIBG therapy in high-risk neuroblastoma. Res Sq. 2024 Jan 18.  View on PubMed
  7. Hogarty MD, Ziegler DS, Franson A, Chi YY, Tsao-Wei D, Liu K, Vemu R, Gerner EW, Bruckheimer E, Shamirian A, Hasenauer B, Balis FM, Groshen S, Norris MD, Haber M, Park JR, Matthay KK, Marachelian A. Phase 1 study of high-dose DFMO, celecoxib, cyclophosphamide and topotecan for patients with relapsed neuroblastoma: a New Approaches to Neuroblastoma Therapy trial. Br J Cancer. 2024 Mar; 130(5):788-797.  View on PubMed
  8. Chang D, Oh T, Roland JL, Matthay KK, Vo KT, Edwards CS, Sun PP, Auguste KI, Gupta N. Pediatric neuroblastoma with intraspinal extension: the role of surgical management. J Neurosurg Pediatr. 2024 Mar 01; 33(3):245-255.  View on PubMed
  9. Batra V, Gikandi A, Pawel B, Martinez D, Granger MM, Marachelian A, Park JR, Maris JM, Vo KT, Matthay KK, DuBois SG. Norepinephrine transporter and vesicular monoamine transporter 2 tumor expression as a predictor of response to 131 I-MIBG in patients with relapsed/refractory neuroblastoma. Pediatr Blood Cancer. 2024 Jan; 71(1):e30743.  View on PubMed
  10. 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
  11. 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
  12. 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
  13. 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
  14. 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 11 01; 128(21):3775-3783.  View on PubMed
  15. 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 12 10; 40(35):4107-4118.  View on PubMed
  16. Qiu B, Matthay KK. Advancing therapy for neuroblastoma. Nat Rev Clin Oncol. 2022 08; 19(8):515-533.  View on PubMed
  17. 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
  18. 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 Feb 01; 197(2):101-112.  View on PubMed
  19. 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
  20. 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

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