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Review
. 2016 Jul;38(1):3-15.
doi: 10.3892/ijmm.2016.2620. Epub 2016 May 31.

FGFR inhibitors: Effects on cancer cells, tumor microenvironment and whole-body homeostasis (Review)

Affiliations
Review

FGFR inhibitors: Effects on cancer cells, tumor microenvironment and whole-body homeostasis (Review)

Masaru Katoh. Int J Mol Med. 2016 Jul.

Abstract

Fibroblast growth factor (FGF)2, FGF4, FGF7 and FGF20 are representative paracrine FGFs binding to heparan-sulfate proteoglycan and fibroblast growth factor receptors (FGFRs), whereas FGF19, FGF21 and FGF23 are endocrine FGFs binding to Klotho and FGFRs. FGFR1 is relatively frequently amplified and overexpressed in breast and lung cancer, and FGFR2 in gastric cancer. BCR-FGFR1, CNTRL-FGFR1, CUX1-FGFR1, FGFR1OP-FGFR1, MYO18A-FGFR1 and ZMYM2-FGFR1 fusions in myeloproliferative neoplasms are non-receptor-type FGFR kinases, whereas FGFR1-TACC1, FGFR2-AFF3, FGFR2-BICC1, FGFR2-PPHLN1, FGFR3-BAIAP2L1 and FGFR3-TACC3 fusions in solid tumors are transmembrane-type FGFRs with C-terminal alterations. AZD4547, BGJ398 (infigratinib), Debio-1347 and dovitinib are FGFR1/2/3 inhibitors; BLU9931 is a selective FGFR4 inhibitor; FIIN-2, JNJ-42756493, LY2874455 and ponatinib are pan-FGFR inhibitors. AZD4547, dovitinib and ponatinib are multi-kinase inhibitors targeting FGFRs, colony stimulating factor 1 receptor (CSF1R), vascular endothelial growth factor (VEGF)R2, and others. The tumor microenvironment consists of cancer cells and stromal/immune cells, such as cancer-associated fibroblasts (CAFs), endothelial cells, M2-type tumor-associating macrophages (M2-TAMs), myeloid-derived suppressor cells (MDSCs) and regulatory T cells. FGFR inhibitors elicit antitumor effects directly on cancer cells, as well as indirectly through the blockade of paracrine signaling. The dual inhibition of FGF and CSF1 or VEGF signaling is expected to enhance the antitumor effects through the targeting of immune evasion and angiogenesis in the tumor microenvironment. Combination therapy using tyrosine kinase inhibitors (FGFR or CSF1R inhibitors) and immune checkpoint blockers (anti-PD-1 or anti-CTLA-4 monoclonal antibodies) may be a promising choice for cancer patients. The inhibition of FGF19-FGFR4 signaling is associated with a risk of liver toxicity, whereas the activation of FGF23-FGFR4 signaling is associated with a risk of heart toxicity. Endocrine FGF signaling affects the pathophysiology of cancer patients who are prescribed FGFR inhibitors. Whole-genome sequencing is necessary for the detection of promoter/enhancer alterations of FGFR genes and rare alterations of other genes causing FGFR overexpression. To sustain the health care system in an aging society, a benefit-cost analysis should be performed with a focus on disease-free survival and the total medical cost before implementing genome-based precision medicine for cancer patients.

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Figures

Figure 1
Figure 1
Receptor tyrosine kinase (RTK) signaling cascades and RTK inhibitors. (A) RTK signaling cascades. Multiple RTKs, such as fibroblast growth factor receptors (FGFRs), CSF1R and VEGFR2, redundantly activate the RAS-ERK, PI3K-AKT, IP3-Ca2+ and DAG-PKC signaling cascades. (B) Representative tyrosine kinase inhibitors (TKIs) and their targets.
Figure 2
Figure 2
Receptor tyrosine kinase (RTK) superfamily. Phylogenetic tree of 54 human RTKs are shown. *, 18 RTKs in the Oncomine Comprehensive Panel (137). RTKs are classified into the EGFR, FGFR, INSR ROR and EPH groups. FGFR1, FGFR2, FGFR3, FGFR4, CSF1R and VEGFR2, shown in red, belong to the FGFR group.
Figure 3
Figure 3
Fibroblast growth factor receptor (FGFR) alterations in human cancer. FGFR genes are activated in human cancer as a result of gene amplification, coding mutation and gene fusion. FGFR gene fusions are further classified into two groups. Type 1 FGFR fusions in hematological malignancies encode non-transmembrane-type FGFR kinases. Type 2 FGFR fusions in solid tumors encode transmembrane-type FGFRs with C-terminal substitution to the region of fusion partners.
Figure 4
Figure 4
Small-molecule fibroblast growth factor receptor (FGFR) inhibitors and FGFRs. (A) Small-molecule FGFR inhibitors. Enzymatic IC50 values for FGFRs and other substrates are listed. (B) Alignment of the tyrosine kinase domain of FGFR1, FGFR2, FGFR3 and FGFR4. Amino-acid position is shown on both sides of the alignment. Amino-acid residues conserved in all members of FGFRs are shown by asterisk, whereas amino-acid residues conserved in FGFR1, FGFR2 and FGFR3 but not in FGFR4 are shown by sharp. FGFR4 is relatively divergent from FGFR1, FGFR2 and FGFR3. Tyrosine residue in the hinge region (Y563 in FGFR1, Y566 in FGFR2 and Y557 in FGFR3) is substituted to C552 in FGFR4, which divergence is involved in the selectivity of FGFR inhibitors for FGFR1/2/3 and FGFR4.
Figure 5
Figure 5
Selection of fibroblast growth factor receptor (FGFR) inhibitor for precision medicine. (A) Classification of FGFR inhibitors based on substrate specificities. *, FIIN-2 is for experimental use only. **, Severe adverse effects of ponatinib have been reported in patients with chronic myeloid leukemia. (B) Flow-chart for the choice of FGFR inhibitor in clinic. MPN, myeloproliferative neoplasm; Amp, gene amplification; OverE, overexpression; Fus, fusion. FGFR1/2/3 inhibitors and pan-FGFR inhibitors are applicable for human cancer with genetic alteration in FGFR1, FGFR2 or FGFR3. FGFR1/2/3 inhibitors are not the choice for patients with heart diseases, whereas pan-FGFR inhibitors are not the choice for patients with liver dysfunction. FGFR4 inhibitor is applicable for HCC with genetic alteration in FGF19.
Figure 6
Figure 6
Cancer therapy targeting tumor microenvironment. Cancer cells, cancer-associated fibroblasts (CAFs), endothelial cells, myeloid-derived suppressor cells (MDSCs), tumor-associating macrophages of M2 type (M2-TAMs) and regulatory T cells are representative components of tumor microenvironment. Interactions between cancer cells and stromal/immune cells are involved in almost all steps of carcinogenesis. CSF1 signaling through CSF1R induces proliferation and differentiation of MDSCs and M2-TAMs. Dual inhibition of fibroblast growth factor receptor (FGFR) and CSF1R/VEGFR2 is expected to increase antitumor effects through targeting immune evasion and angiogenesis in the tumor microenvironment. Combination of FGFR/CSF1R inhibitor targeting cancer cells and stromal/immune cells and anti-PD-1/CTLA-4 monoclonal antibody targeting regulatory T cells and de-repressing CD8+ T cells may be a promising choice for cancer patients.
Figure 7
Figure 7
Genome-based precision medicine for cancer patients. Amp, amplification; Fus, fusion; Mut, mutation. Patients with breast cancer (red), gastric cancer (green), lung cancer (blue) and other cancers (yellow) are reorganized into the groups of cancer patients with specific genetic alterations. Patients with fibroblast growth factor receptor (FGFR), EGFR, HER2, ALK and RET alterations are prescribed FGFR inhibitor, EGFR inhibitor, HER2 monoclonal antibody (mAb), ALK inhibitor and RET inhibitor, respectively. However, at present, there is no targeted therapy for cancer patients with MYC Amp, RHOA Mut, TP53 Mut, ASXL1 Mut, etc.

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