Targeting Fibroblast Growth Factor Receptor Pathway: Precision Medicine for Biliary Cancer and Beyond

被引：2

作者：

Uson Jr, Pedro Luiz Serrano ^{[1
,2
]}

Borad, Mitesh J. ^{[1
,3
,4
,5
,6
]}

机构：

[1] Mayo Clin, Dept Med, Div Hematol & Oncol, Scottsdale, AZ USA

[2] Hosp Israelita Albert Einstein, Ctr Personalized Med, Sao Paulo, Brazil

[3] Mayo Clin, Ctr Individualized Med, Rochester, MN USA

[4] Mayo Clin, Dept Mol Med, Rochester, MN USA

[5] Mayo Clin, Canc Ctr, Phoenix, AZ USA

[6] Mayo Clin, 5777 Mayo Blvd, Phoenix, AZ 85054 USA

来源：

SEMINARS IN LIVER DISEASE | 2023年 / 43卷 / 02期

关键词：

cholangiocarcinoma; FGFR2; biliary cancers; pemigatinib; infigratinib; futibatinib; FGFR INHIBITOR; PHASE-I; CHOLANGIOCARCINOMA; EFFICACY; RESISTANCE; MUTATIONS; DERAZANTINIB; FUTIBATINIB; RLY-4008; BGJ398;

D O I：

10.1055/a-2049-3149

中图分类号：

R57 [消化系及腹部疾病];

学科分类号：

摘要：

Fibroblast growth factor receptor 2 (FGFR2) inhibitors are now being included in the treatment guidelines of multiple countries for patients with advanced cholangiocarcinoma (CCA). Activation of the FGF-FGFR pathway is related to proliferation and tumor progression. Targeting the FGF-FGFR pathway is effective and can yield durable responses in patients with CCA harboring FGFR2 fusions or rearrangements. In this review article, we address molecules and clinical trials evaluating FGFR inhibitors in advanced CCA. We will further discuss identified mechanisms of resistance and the strategies to overcome it. The incorporation of next-generation sequencing in advanced CCA and circulating tumor DNA on disease progression will unveil mechanisms of resistance and improve the development of future clinical trials and more selective drugs and combinations.

引用

页码：218 / 225

页数：8

共 46 条

[1] Pemigatinib for previously treated, locally advanced or metastatic cholangiocarcinoma: a multicentre, open-label, phase 2 study [J].

Abou-Alfa, Ghassan K. ;

Sahai, Vaibhav ;

Hollebecque, Antoine ;

Vaccaro, Gina ;

Melisi, Davide ;

Al-Rajabi, Raed ;

Paulson, Andrew S. ;

Borad, Mitesh J. ;

Gallinson, David ;

Murphy, Adrian G. ;

Oh, Do-Youn ;

Dotan, Efrat ;

Catenacci, Daniel, V ;

Van Cutsem, Eric ;

Ji, Tao ;

Lihou, Christine F. ;

Zhen, Huiling ;

Feliz, Luis ;

Vogel, Arndt .

LANCET ONCOLOGY, 2020, 21 (05) :671-684

[2] A pilot study of Pan-FGFR inhibitor ponatinib in patients with FGFR-altered advanced cholangiocarcinoma [J].

Ahn, Daniel H. ;

Uson Junior, Pedro Luiz Serrano ;

Masci, Peter ;

Kosiorek, Heidi ;

Halfdanarson, Thorvardur R. ;

Mody, Kabir ;

Babiker, Hani ;

DeLeon, Thomas ;

Sonbol, Mohamad Bassam ;

Gores, Gregory ;

Smoot, Rory ;

Bekaii-Saab, Tanios ;

Mahipal, Amit ;

Mansfield, Aaron ;

Tran, Nguyen H. ;

Hubbard, Joleen M. ;

Borad, Mitesh J. .

INVESTIGATIONAL NEW DRUGS, 2022, 40 (01) :134-141

[3] Evaluation of NUC-1031: a first-in-class ProTide in biliary tract cancer [J].

Arora, Mansi ;

Bogenberger, James M. ;

Abdelrahman, Amro ;

Leiting, Jennifer L. ;

Chen, Xianfeng ;

Egan, Jan B. ;

Kasimsetty, Aradhana ;

Lenkiewicz, Elzbieta ;

Malasi, Smriti ;

Uson, Pedro Luiz Serrano ;

Nagalo, Bolni Marius ;

Zhou, Yumei ;

Salomao, Marcela A. ;

Kosiorek, Heidi E. ;

Braggio, Esteban ;

Barrett, Michael T. ;

Truty, Mark J. ;

Borad, Mitesh J. .

CANCER CHEMOTHERAPY AND PHARMACOLOGY, 2020, 85 (06) :1063-1078

[4] Phase I, first-in-human study of futibatinib, a highly selective, irreversible FGFR1-4 inhibitor in patients with advanced solid tumors [J].

Bahleda, R. ;

Meric-Bernstam, F. ;

Goyal, L. ;

Tran, B. ;

He, Y. ;

Yamamiya, I ;

Benhadji, K. A. ;

Matos, I ;

Arkenau, H-T .

ANNALS OF ONCOLOGY, 2020, 31 (10) :1405-1412

[5] Multicenter Phase I Study of Erdafitinib (JNJ-42756493), Oral Pan-Fibroblast Growth Factor Receptor Inhibitor, in Patients with Advanced or Refractory Solid Tumors [J].

Bahleda, Rastislav ;

Italiano, Antoine ;

Hierro, Cinta ;

Mita, Alain ;

Cervantes, Andres ;

Chan, Nancy ;

Awad, Mark ;

Calvo, Emiliano ;

Moreno, Victor ;

Govindan, Ramaswamy ;

Spira, Alexander ;

Gonzalez, Martha ;

Zhong, Bob ;

Santiago-Walker, Ademi ;

Poggesi, Italo ;

Parekh, Trilok ;

Xie, Hong ;

Infante, Jeffrey ;

Tabernero, Josep .

CLINICAL CANCER RESEARCH, 2019, 25 (16) :4888-4897

[6]

Bekaii-Saab T S., 2020, J CLIN ONCOL

[7] The clinical landscape of cell-free DNA alterations in 1671 patients with advanced biliary tract cancer [J].

Berchuck, J. E. ;

Facchinetti, F. ;

DiToro, D. F. ;

Baiev, I ;

Majeed, U. ;

Reyes, S. ;

Chen, C. ;

Zhang, K. ;

Sharman, R. ;

Uson, P. L. S., Jr. ;

Maurer, J. ;

Shroff, R. T. ;

Pritchard, C. C. ;

Wu, M-J ;

Catenacci, D. V. T. ;

Javle, M. ;

Friboulet, L. ;

Hollebecque, A. ;

Bardeesy, N. ;

Zhu, A. X. ;

Lennerz, J. K. ;

Tan, B. ;

Borad, M. ;

Parikh, A. R. ;

Kiedrowski, L. A. ;

Kelley, R. K. ;

Mody, K. ;

Juric, D. ;

Goyal, L. .

ANNALS OF ONCOLOGY, 2022, 33 (12) :1269-1283

[8] Efficacy of derazantinib in intrahepatic cholangiocarcinoma (iCCA) patients with FGFR2 fusions, mutations or amplifications [J].

Borad, M. ;

Javle, M. ;

Shaib, W. L. ;

Mody, K. ;

Bergamo, F. ;

Harris, W. P. ;

Damjanov, N. ;

Macarulla, T. ;

Brandi, G. ;

Masi, G. ;

Busset, M. Droz Dit ;

Boncompagni, A. ;

Dimova-Dobreva, M. ;

Engelhardt, M. ;

Saulay, M. ;

Halfdanarson, T. R. ;

Knox, J. ;

Abou-Alfa, G. K. ;

Personeni, N. ;

Mazzaferro, V. .

ANNALS OF ONCOLOGY, 2022, 33 (07) :S567-S568

[9]

Borad M J., 2020, J CLIN ONCOL S

[10] Integrated Genomic Characterization Reveals Novel, Therapeutically Relevant Drug Targets in FGFR and EGFR Pathways in Sporadic Intrahepatic Cholangiocarcinoma [J].

Borad, Mitesh J. ;

Champion, Mia D. ;

Egan, Jan B. ;

Liang, Winnie S. ;

Fonseca, Rafael ;

Bryce, Alan H. ;

McCullough, Ann E. ;

Barrett, Michael T. ;

Hunt, Katherine ;

Patel, Maitray D. ;

Young, Scott W. ;

Collins, Joseph M. ;

Silva, Alvin C. ;

Condjella, Rachel M. ;

Block, Matthew ;

McWilliams, Robert R. ;

Lazaridis, Konstantinos N. ;

Klee, Eric W. ;

Bible, Keith C. ;

Harris, Pamela ;

Oliver, Gavin R. ;

Bhavsar, Jaysheel D. ;

Nair, Asha A. ;

Middha, Sumit ;

Asmann, Yan ;

Kocher, Jean-Pierre ;

Schahl, Kimberly ;

Kipp, Benjamin R. ;

Fritcher, Emily G. Barr ;

Baker, Angela ;

Aldrich, Jessica ;

Kurdoglu, Ahmet ;

Izatt, Tyler ;

Christoforides, Alexis ;

Cherni, Irene ;

Nasser, Sara ;

Reiman, Rebecca ;

Phillips, Lori ;

McDonald, Jackie ;

Adkins, Jonathan ;

Mastrian, Stephen D. ;

Placek, Pamela ;

Watanabe, Aprill T. ;

LoBello, Janine ;

Han, Haiyong ;

Von Hoff, Daniel ;

Craig, David W. ;

Stewart, A. Keith ;

Carpten, John D. .

PLOS GENETICS, 2014, 10 (02)

← 1 2 3 4 5 →