354
Views
1
CrossRef citations to date
0
Altmetric
Special Report

The future of fibroblast growth factor receptor inhibitors and mechanisms of resistance for cholangiocarcinoma

, &
Pages 779-788 | Received 10 Feb 2023, Accepted 11 Apr 2023, Published online: 14 Apr 2023

References

  • Chmiel P, Gęca K, Rawicz-Pruszyński K, et al. FGFR inhibitors in Cholangiocarcinoma—A novel yet primary approach: where do we stand now and where to head next in targeting this axis? Cells. 2022;11(23):3929. DOI:10.3390/cells11233929
  • Goyal L, Kongpetch S, Crolley VE, et al. Targeting FGFR inhibition in cholangiocarcinoma. Cancer Treat Rev. 2021;95:102170.
  • Esnaola NF, Meyer JE, Karachristos A, et al. Evaluation and management of intrahepatic and extrahepatic cholangiocarcinoma. Cancer. 2016;122(9):1349–1369. DOI:10.1002/cncr.29692
  • Cardinale V, Semeraro R, Torrice A, et al. Intra-hepatic and extra-hepatic cholangiocarcinoma: new insight into epidemiology and risk factors. World J Gastrointest Oncol. 2010;2(11):407–416. DOI:10.4251/wjgo.v2.i11.407
  • Patel N, Benipal B. Incidence of Cholangiocarcinoma in the USA from 2001 to 2015: a US cancer statistics analysis of 50 states. Cureus. 2019;11(1):e3962.
  • Alabraba E, Joshi H, Bird N, et al. Increased multimodality treatment options has improved survival for Hepatocellular carcinoma but poor survival for biliary tract cancers remains unchanged. Eur J Surg Oncol. 2019;45(9):1660–1667. DOI:10.1016/j.ejso.2019.04.002
  • Gad MM, Saad AM, Faisaluddin M, et al. Epidemiology of Cholangiocarcinoma; United States incidence and mortality trends. Clin Res Hepatol Gastroenterol. 2020;44(6):885–893. DOI:10.1016/j.clinre.2020.03.024
  • Rizvi S, Khan SA, Hallemeier CL, et al. Cholangiocarcinoma - evolving concepts and therapeutic strategies. Nat Rev Clin Oncol. 2018;15(2):95–111. DOI:10.1038/nrclinonc.2017.157
  • Lowery MA, Ptashkin R, Jordan E, et al. Comprehensive molecular profiling of intrahepatic and extrahepatic Cholangiocarcinomas: potential targets for intervention. Clin Cancer Res. 2018;24(17):4154–4161. DOI:10.1158/1078-0432.CCR-18-0078
  • Javle M, Bekaii-Saab T, Jain A, et al. Biliary cancer: utility of next-generation sequencing for clinical management. Cancer. 2016;122(24):3838–3847. DOI:10.1002/cncr.30254
  • Zingg D, Bhin J, Yemelyanenko J, et al. Truncated FGFR2 is a clinically actionable oncogene in multiple cancers. Nature. 2022;608(7923):609–617. DOI:10.1038/s41586-022-05066-5
  • Helsten T, Elkin S, Arthur E, et al. The FGFR landscape in cancer: analysis of 4,853 tumors by next-generation sequencing. Clin Cancer Res. 2016;22(1):259–267. DOI:10.1158/1078-0432.CCR-14-3212
  • Lamarca A, Edeline J, Goyal L. How I treat biliary tract cancer. ESMO Open. 2022;7(1):100378.
  • Goyal L, Lamarca A, Strickler JH, et al. The natural history of fibroblast growth factor receptor (FGFR)-altered cholangiocarcinoma (CCA). J Clin Oncol. 2020;38(15_suppl):e16686-e. DOI:10.1200/JCO.2020.38.15_suppl.e16686
  • Jain A, Borad MJ, Kelley RK, et al. Cholangiocarcinoma with FGFR genetic aberrations: a unique clinical phenotype. JCO Precision Oncol. 2018;2018(2):1–12. DOI:10.1200/PO.17.00080
  • Cleary JM, Raghavan S, Wu Q, et al. FGFR2 extracellular domain in-frame deletions are therapeutically targetable genomic alterations that function as oncogenic drivers in Cholangiocarcinoma. Cancer Discov. 2021;11(10):2488–2505. DOI:10.1158/2159-8290.CD-20-1669
  • Ellinghaus P, Neureiter D, Nogai H, et al. Patient selection approaches in FGFR inhibitor trials—Many paths to the same end? Cells. 2022;11(19):3180. DOI:10.3390/cells11193180
  • King G, Javle M. FGFR inhibitors: clinical activity and development in the treatment of Cholangiocarcinoma. Curr Oncol Rep. 2021;23(9):108.
  • Zheng J, Zhang W, Li L, et al. Signaling pathway and small-molecule drug discovery of FGFR: a Comprehensive review. Front Chem. 2022;10:860985.
  • Voss MH, Hierro C, Heist RS, et al. A phase i, open-label, multicenter, dose-escalation study of the oral selective FGFR inhibitor debio 1347 in patients with advanced solid tumors harboring FGFR gene alterations. Clin Cancer Res. 2019;25(9):2699–2707. DOI:10.1158/1078-0432.CCR-18-1959
  • Mazzaferro V, El-Rayes BF, Droz Dit Busset M, et al. Derazantinib (ARQ 087) in advanced or inoperable FGFR2 gene fusion-positive intrahepatic cholangiocarcinoma. Br J Cancer. 2019;120(2):165–171. DOI:10.1038/s41416-018-0334-0
  • Javle MM, Abou-Alfa GK, Macarulla T, et al. Efficacy of derazantinib in intrahepatic cholangiocarcinoma patients with FGFR2 mutations or amplifications: interim results from the phase 2 study FIDES-01. J Clin Oncol. 2022;40(4_suppl):427. DOI:10.1200/JCO.2022.40.4_suppl.427
  • Rizzo A. Targeted therapies in advanced Cholangiocarcinoma: a focus on FGFR inhibitors. Med (Kaunas). 2021;57(5):458.
  • Park JO, Feng Y-H, Chen Y-Y, et al. Updated results of a phase IIa study to evaluate the clinical efficacy and safety of erdafitinib in Asian advanced cholangiocarcinoma (CCA) patients with FGFR alterations. J Clin Oncol. 2019;37(15_suppl):4117. DOI:10.1200/JCO.2019.37.15_suppl.4117
  • Loriot Y, Schuler MH, Iyer G, et al. Tumor agnostic efficacy and safety of erdafitinib in patients (pts) with advanced solid tumors with prespecified fibroblast growth factor receptor alterations (FGFRalt) in RAGNAR: interim analysis (IA) results. J Clin Oncol. 2022;40(16_suppl):3007. DOI:10.1200/JCO.2022.40.16_suppl.3007
  • Pant S, Schuler MH, Iyer G, et al. Efficacy and safety of erdafitinib in adults with cholangiocarcinoma (CCA) with prespecified fibroblast growth factor receptor alterations (FGFRalt) in the phase 2 open-label, single-arm RAGNAR trial: expansion cohort results. J Clin Oncol. 2023;41(4_suppl):610. DOI:10.1200/JCO.2023.41.4_suppl.610
  • Javle M, Roychowdhury S, Kelley RK, et al. Infigratinib (BGJ398) in previously treated patients with advanced or metastatic cholangiocarcinoma with FGFR2 fusions or rearrangements: mature results from a multicentre, open-label, single-arm, phase 2 study. Lancet Gastroenterol Hepatol. 2021;6(10):803–815. DOI:10.1016/S2468-1253(21)00196-5
  • Rizzo A, Ricci AD, Brandi G. Futibatinib, an investigational agent for the treatment of intrahepatic cholangiocarcinoma: evidence to date and future perspectives. Expert Opin Investig Drugs. 2021;30(4):317–324.
  • Sootome H, Fujita H, Ito K, et al. Futibatinib is a novel irreversible FGFR 1-4 inhibitor that shows selective antitumor activity against FGFR-Deregulated tumors. Cancer Res. 2020;80(22):4986–4997. DOI:10.1158/0008-5472.CAN-19-2568
  • Goyal L, Meric-Bernstam F, Hollebecque A, et al. Futibatinib for FGFR2-rearranged intrahepatic Cholangiocarcinoma. N Engl J Med. 2023;388(3):228–239. DOI:10.1056/NEJMoa2206834
  • Subbiah V, Iannotti NO, Gutierrez M, et al. FIGHT-101, a first-in-human study of potent and selective FGFR 1-3 inhibitor pemigatinib in pan-cancer patients with FGF/FGFR alterations and advanced malignancies. Ann Oncol. 2022;33(5):522–533. DOI:10.1016/j.annonc.2022.02.001
  • Abou-Alfa GK, Sahai V, Hollebecque A, et al. Pemigatinib for previously treated, locally advanced or metastatic cholangiocarcinoma: a multicentre, open-label, phase 2 study. Lancet Oncol. 2020;21(5):671–684. DOI:10.1016/S1470-2045(20)30109-1
  • Zugman M, Botrus G, Pestana RC, et al. Precision medicine targeting FGFR2 genomic alterations in advanced Cholangiocarcinoma: current state and future perspectives. Front Oncol. 2022;12:860453.
  • Schram AM, Kamath SD, El-Khoueiry AB, et al. First-in-human study of highly selective FGFR2 inhibitor, RLY-4008, in patients with intrahepatic cholangiocarcinoma and other advanced solid tumors. J Clin Oncol. 2021;39(15_suppl):TPS4165-TPS. DOI:10.1200/JCO.2021.39.15_suppl.TPS4165
  • Hollebecque A, Borad M, Goyal L, et al. Efficacy of RLY-4008, a highly selective FGFR2 inhibitor in patients (pts) with an FGFR2-fusion or rearrangement (f/r), FGFR inhibitor (FGFRi)-naïve cholangiocarcinoma (CCA): ReFocus trial. 2022 2/9/23]; Available from: https://oncologypro.esmo.org/meeting-resources/esmo-congress/efficacy-of-rly-4008-a-highly-selective-fgfr2-inhibitor-in-patients-pts-with-an-fgfr2-fusion-or-rearrangement-f-r-fgfr-inhibitor-fgfri-naiv.
  • Krook MA, Reeser JW, Ernst G, et al. Fibroblast growth factor receptors in cancer: genetic alterations, diagnostics, therapeutic targets and mechanisms of resistance. Br J Cancer. 2021;124(5):880–892. DOI:10.1038/s41416-020-01157-0
  • Bai A, Meetze K, Vo NY, et al. GP369, an FGFR2-IIIb-specific antibody, exhibits potent antitumor activity against human cancers driven by activated FGFR2 signaling. Cancer Res. 2010;70(19):7630–7639. DOI:10.1158/0008-5472.CAN-10-1489
  • Sommer A, Kopitz C, Schatz CA, et al. Preclinical Efficacy of the auristatin-based antibody-drug conjugate BAY 1187982 for the treatment of FGFR2-positive solid tumors. Cancer Res. 2016;76(21):6331–6339. DOI:10.1158/0008-5472.CAN-16-0180
  • Kim SB, Meric-Bernstam F, Kalyan A, et al. First-in-human phase i study of aprutumab ixadotin, a fibroblast growth factor receptor 2 antibody-drug conjugate (BAY 1187982) in patients with advanced cancer. Target Oncol. 2019;14(5):591–601. DOI:10.1007/s11523-019-00670-4
  • Hollebecque A, Silverman I, Owens S, et al. 720P - Comprehensive genomic profiling and clinical outcomes in patients (pts) with fibroblast growth factor receptor rearrangement-positive (FGFR2+) cholangiocarcinoma (CCA) treated with pemigatinib in the fight-202 trial. Ann Oncol. 2019;30:v276.
  • Lau DK, Jenkins L, Weickhardt A. Mechanisms of acquired resistance to fibroblast growth factor receptor targeted therapy. Cancer Drug Resist. 2019;2(3):568–579.
  • Kitano S, Yamamoto T, Taketo MM. Novel parameter for cancer chemosensitivity to fibroblast growth factor receptor inhibitors. Cancer Sci. 2022;113(11):4005–4010.
  • Wu Q, Zhen Y, Shi L, et al. EGFR inhibition potentiates FGFR inhibitor therapy and overcomes resistance in FGFR2 fusion-positive Cholangiocarcinoma. Cancer Discov. 2022;12(5):1378–1395. DOI:10.1158/2159-8290.CD-21-1168
  • Varghese AM, Patel JAA, Janjigian YY, et al. Non-invasive detection of acquired resistance to FGFR inhibition in patients with cholangiocarcinoma harboring FGFR2 alterations. J Clin Oncol. 2019;37(15_suppl):4096. DOI:10.1200/JCO.2019.37.15_suppl.4096
  • Goyal L, Saha SK, Liu LY, et al. Polyclonal secondary FGFR2 mutations drive acquired resistance to FGFR inhibition in patients with FGFR2 fusion–positive Cholangiocarcinoma. Cancer Discovery. 2017;7(3):252–263. DOI:10.1158/2159-8290.CD-16-1000
  • Varghese AM, Patel J, Janjigian YY, et al. Noninvasive detection of polyclonal acquired resistance to FGFR inhibition in patients with Cholangiocarcinoma harboring FGFR2 alterations. JCO Precis Oncol. 2021;5(5):44–50. DOI:10.1200/PO.20.00178
  • Goyal L, Shi L, Liu LY, et al. TAS-120 overcomes resistance to ATP-Competitive FGFR inhibitors in patients with FGFR2 fusion-positive intrahepatic Cholangiocarcinoma. Cancer Discov. 2019;9(8):1064–1079. DOI:10.1158/2159-8290.CD-19-0182
  • Primrose JN, Fox RP, Palmer DH, et al. Capecitabine compared with observation in resected biliary tract cancer (BILCAP): a randomised, controlled, multicentre, phase 3 study. Lancet Oncol. 2019;20(5):663–673. DOI:10.1016/S1470-2045(18)30915-X
  • Valle J, Wasan H, Palmer DH, et al. Cisplatin plus gemcitabine versus gemcitabine for biliary tract cancer. N Engl J Med. 2010;362(14):1273–1281. DOI:10.1056/NEJMoa0908721
  • Oh D-Y, Ruth He A, Qin S, et al. Durvalumab plus gemcitabine and cisplatin in advanced biliary tract cancer. NEJM Evidence. 2022;1(8):EVIDoa2200015. DOI:10.1056/EVIDoa2200015

Reprints and Corporate Permissions

Please note: Selecting permissions does not provide access to the full text of the article, please see our help page How do I view content?

To request a reprint or corporate permissions for this article, please click on the relevant link below:

Academic Permissions

Please note: Selecting permissions does not provide access to the full text of the article, please see our help page How do I view content?

Obtain permissions instantly via Rightslink by clicking on the button below:

If you are unable to obtain permissions via Rightslink, please complete and submit this Permissions form. For more information, please visit our Permissions help page.