Research Summary

Dr. Katherine Matthay is the chief of Pediatric Hematology-Oncology at UCSF Benioff Children's Hospital and has been focused for more than 20 years on translational research in neuroblastoma, a deadly childhood cancer. At UCSF, she has developed and led multiple trials using a unique targeted radiopharmaceutical, 131I-MIBG for neuroblastoma.

Dr. Matthay also leads the UCSF Pediatric Malignancies Program and an NIH funded Phase I New Approaches to Neuroblastoma Therapy consortium (NANT) for treatment of relapsed and resistant neuroblastoma.

Research Funding

  • August 5, 1999 - May 31, 2028 - 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. Davini M, Naranjo A, Chen L, Bagatell R, DuBois SG, Irwin MS, Goldsmith KC, Hogarty MD, Matthay KK. Factors Impacting Overall Survival Post-Relapse in High-Risk Neuroblastoma: Children's Oncology Group Outcomes From 2000 to 2019. Pediatr Blood Cancer. 2026 Sep; 73(9):e70520. View on PubMed
  2. Lerman BJ, Matthay KK, Hollinger F, Black C, Lazar AA, Pampaloni M, DuBois SG, Vo KT. Clinical and Biological Features of Response in Resistant Neuroblastoma to 131I-Metaiodobenzylguanidine Radiotherapy in the Anti-GD2 Immunotherapy Era. Pediatr Blood Cancer. 2026 Aug; 73(8):e70385. View on PubMed
  3. Cohn SL, London WB, Schleiermacher G, Moreno L, Ambros IM, Ambros PF, Bagatell R, Popovic MB, Beiske KH, Berthold F, Birz S, Brisse HJ, Brodeur GM, Brock PR, Burchill S, Eggert A, Federico SM, Fischer M, Furner BT, Hero B, Machin D, Kamijo T, Matthay KK, Nakagawara A, Naranjo A, Pötschger U, Valteau-Couanet D, Watkins MT, Irwin MS, Volchenboum SL, Park JR, Pearson ADJ. International neuroblastoma risk group consortium: a model of networking for rare cancers. J Natl Cancer Inst. 2026 Apr 01; 118(4):579-588. View on PubMed
  4. Wang Y, Huh Y, Matthay KK, Vo KT, Pampaloni MH, DuBois SG, Seo Y. Simplified dosimetry using two-time-point kinetic modeling of 124I-MIBG PET for 131I-MIBG therapy in neuroblastoma. Med Phys. 2026 Mar; 53(3):e70388. View on PubMed
  5. Papyan R, Hoveyan J, Guscott M, Lowe J, Villanueva G, Streby KA, Tadevosyan E, Demirtshyan E, Hylton J, Tamamyan G, Lam CG, Matthay K, Howard SC. The Price of Survival: Global Inequities in Anti-GD2 Immunotherapy for Neuroblastoma. Pediatr Blood Cancer. 2026 Apr; 73(4):e70152. View on PubMed
  6. London WB, Villanueva G, Shyr D, van Heerden J, Fuentes-Alabi S, Saab R, Harrison D, Papyan R, Kim Nguyen HT, Noun D, Kambugu J, Rascon J, Garami M, Guo D, Kao P, Naranjo A, Park JR, Cohn SL, Rodriguez-Galindo C, Matthay KK. Adaptive Clinical Neuroblastoma Risk Groups-Tailoring Treatment in Low- and Middle-Income Countries: An International Neuroblastoma Risk Group Project. JCO Glob Oncol. 2025 12; 11:e2500349. View on PubMed
  7. Cash T, Marachelian A, DuBois SG, Chi YY, Baregamyan A, Groshen SG, Jonus HC, Shamirian A, Crowley M, Goodarzian F, Acharya PT, Pawel B, Erbe AK, Shahi A, Zaborek J, Kennedy E, Asgharzadeh S, Villablanca JG, Pinto N, Weiss BD, Mosse YP, Desai AV, Macy ME, Granger M, Vo KT, Sondel PM, Matthay KK, Park JR, Goldsmith KC. Phase I Study of 131I-Metaiodobenzylguanidine With Dinutuximab ± Vorinostat for Patients With Relapsed or Refractory Neuroblastoma: A New Approaches to Neuroblastoma Therapy Trial. J Clin Oncol. 2025 08; 43(22):2490-2501. View on PubMed
  8. 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
  9. 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
  10. 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
  11. 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
  12. 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
  13. 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
  14. 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 03; 130(5):788-797. View on PubMed
  15. 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 03 01; 33(3):245-255. View on PubMed
  16. 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
  17. 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
  18. 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 08; 70(8):e30418. View on PubMed
  19. 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
  20. 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

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