1,570
Views
5
CrossRef citations to date
0
Altmetric
Editorial

Key challenges for drugs in clinical development for cholangiocarcinoma

ORCID Icon &
Pages 285-290 | Received 04 Dec 2020, Accepted 20 Jan 2021, Published online: 02 Feb 2021

References

  • Rizvi S, Khan SA, Hallemeier CL, et al. Cholangiocarcinoma - evolving concepts and therapeutic strategies. Nat Rev Clin Oncol. 2018;15(2):95–111.
  • Saha SK, Zhu AX, Fuchs CS, et al. Forty-year trends in cholangiocarcinoma Incidence in the U.S.: intrahepatic disease on the rise. Oncologist. 2016;21(5):594–599.
  • Banales JM, Marin JJG, Lamarca A, et al. Cholangiocarcinoma 2020: the next horizon in mechanisms and management. Nat Rev Gastroenterol Hepatol. 2020;17(9):557–588.
  • Bertuccio P, Malvezzi M, Carioli G, et al. Global trends in mortality from intrahepatic and extrahepatic cholangiocarcinoma. J Hepatol. 2019;71(1):104–114.
  • 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.
  • Shroff RT, Javle MM, Xiao L, et al. Gemcitabine, cisplatin, and nab-paclitaxel for the treatment of advanced biliary tract cancers: a phase 2 clinical trial. JAMA Oncol. 2019;5(6):824–830.
  • Lowery MA, Goff LW, Keenan BP, et al. Second-line chemotherapy in advanced biliary cancers: A retrospective, multicenter analysis of outcomes. Cancer. 2019;125(24):4426–4434.
  • Lamarca A, Palmer DH, Wasan HS, et al. ABC-06 | A randomised phase III, multi-centre, open-label study of active symptom control (ASC) alone or ASC with oxaliplatin/5-FU chemotherapy (ASC+mFOLFOX) for patients (pts) with locally advanced/metastatic biliary tract cancers (ABC) previously-treated with cisplatin/gemcitabine (CisGem) chemotherapy. J clin oncol. 2019;37(15_suppl):4003.
  • Nakamura H, Arai Y, Totoki Y, et al. Genomic spectra of biliary tract cancer. Nature Genet. 2015;47(9):1003–1010.
  • 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. 9(8): 1064–1079. 2019. .
  • 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.
  • Boscoe AN, Rolland C, Kelley RK. Frequency and prognostic significance of isocitrate dehydrogenase 1 mutations in cholangiocarcinoma: a systematic literature review. J Gastrointest Oncol. 2019;10(4):751–765.
  • Abou-Alfa GK, Macarulla T, Javle MM, et al. Ivosidenib in IDH1-mutant, chemotherapy-refractory cholangiocarcinoma (ClarIDHy): a multicentre, randomised, double-blind, placebo-controlled, phase 3 study. Lancet Oncol. 2020;21(6):796–807.
  • Lowery MA, Ptashkin R, Jordan E, et al. Comprehensive molecular profiling of intrahepatic and extrahepatic cholangiocarcinomas: potential targets for intervention. Clin Cancer Res off J Am Assoc Cancer Res. 2018;24(17):4154–4161. .
  • Subbiah V, Puzanov I, Blay JY, et al. Pan-cancer efficacy of vemurafenib in BRAF (V600)-mutant non-melanoma cancers. Cancer Discov. 2020;10(5):657–663. .
  • Kheder ES, Hong DS. Emerging targeted therapy for tumors with NTRK fusion proteins. Clin Cancer Res off J Am Assoc Cancer Res. 2018;24(23):5807–5814.
  • Churi CR, Shroff R, Wang Y, et al. Mutation profiling in cholangiocarcinoma: prognostic and therapeutic implications. PloS One. 2014;9(12):e115383.
  • 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.
  • Piha-Paul SA, Oh D-Y, Ueno M, et al. Efficacy and safety of pembrolizumab for the treatment of advanced biliary cancer: results from the KEYNOTE-158 and KEYNOTE-028 studies. International Journal of Cancer. 2020;147(8):2. .
  • Kim RD, Chung V, Alese OB, et al. A phase 2 multi-institutional study of nivolumab for patients with advanced refractory biliary tract cancer. JAMA Oncology. 2020;6(6):888–894. .
  • Marabelle A, Fakih MG, Lopez J, et al. Association of tumour mutational burden with outcomes in patients with select advanced solid tumours treated with pembrolizumab in KEYNOTE-158. Ann Oncol. 2019;30(Suppl. 5):v477–v478.
  • Le DT, Durham JN, Smith KN, et al. Mismatch repair deficiency predicts response of solid tumors to PD-1 blockade. Science. 2017;357(6349):409–413. .
  • Jusakul A, Cutcutache I, Yong CH, et al. Whole-genome and epigenomic landscapes of etiologically distinct subtypes of cholangiocarcinoma. Cancer Discov. 2017;7(10):1116–1135.
  • Job S, Rapoud D, Dos Santos A, et al. Identification of four immune subtypes characterized by distinct composition and functions of tumor microenvironment in intrahepatic cholangiocarcinoma. Hepatology. 2019;25.
  • Provenzano PP, Cuevas C, Chang AE, et al. Enzymatic targeting of the stroma ablates physical barriers to treatment of pancreatic ductal adenocarcinoma. Cancer Cell. 2012;21(3):418–429.
  • Ma C, Han M, Heinrich B, et al. Gut microbiome-mediated bile acid metabolism regulates liver cancer via NKT cells. Science. 2018;360:6391.
  • Hwang JP, Feld JJ, Hammond SP, et al. Hepatitis B virus screening and management for patients with cancer prior to therapy: ASCO provisional clinical opinion update. J clin oncol. 2020;38(31):3698–3715.
  • Valle JW, Bai L-Y, Orlova R, et al. Ramucirumab (RAM) or merestinib (MER) or placebo (PL) plus gemcitabine (GEM) and cisplatin (CIS) as first-line treatment for advanced or metastatic biliary tract cancer (BTC): A randomized, double-blind, phase II study. J clin oncol. 2020;38(4_suppl):477.
  • Phelip JM, Edeline J, Blanc JF, et al. Modified FOLFIRINOX versus CisGem first-line chemotherapy for locally advanced non resectable or metastatic biliary tract cancer (AMEBICA)-PRODIGE 38: study protocol for a randomized controlled multicenter phase II/III study. Dig Liver Dis. 2019;51(2):318–320.
  • Burd A, Schilsky RL, Byrd JC, et al. Challenges and approaches to implementing master/basket trials in oncology. Blood Adv. 2019;3(14):2237–2243.
  • FDA. Rare diseases: natural history studies for drug development; draft guidance for industry; availability. Office of the commissioner, office of clinical policy and programs, office of orphan products development, et al., editors. Silver Spring, MD: U.S. Department of Health and Human Services, Food and Drug Administration, Center for Drug Evaluation and Research (CDER), Center for Biologics Evaluation and Research (CBER), Office of Orphan Products Development (OOPD). 2019: 1–19. https://www.fda.gov/media/122425/download2019
  • Zill OA, Greene C, Sebisanovic D, et al. Cell-Free DNA next-generation sequencing in pancreatobiliary carcinomas. Cancer Discov. 2015;5(10):1040–1048.
  • Peraldo-Neia C, Scatolini M, Grosso E, et al. Assessment of a high sensitivity method for identification of IDH1 R132x mutations in tumors and plasma of intrahepatic cholangiocarcinoma patients. Cancers (Basel). 2019;11:4.
  • Shen N, Zhang D, Yin L, et al. Bile cellfree DNA as a novel and powerful liquid biopsy for detecting somatic variants in biliary tract cancer. Oncol Rep. 2019;42(2):549–560.

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.