Purpose: Genetic and preclinical studies have implicated FGFR signaling in the 
pathogenesis of adenoid cystic carcinoma (ACC). Dovitinib, a suppressor of FGFR 
activity, may be active in ACC.Experimental Design: In a two-stage phase II 
study, 35 patients with progressive ACC were treated with dovitinib 500 mg 
orally for 5 of 7 days continuously. The primary endpoints were objective 
response rate and change in tumor growth rate. Progression-free survival, 
overall survival, metabolic response, biomarker, and quality of life were 
secondary endpoints.Results: Of 34 evaluable patients, 2 (6%) had a partial 
response and 22 (65%) had stable disease >4 months. Median PFS was 8.2 months 
and OS was 20.6 months. The slope of the overall TGR fell from 1.95 to 0.63 on 
treatment (P < 0.001). Toxicity was moderate; 63% of patients developed grade 
3-4 toxicity, 94% required dose modifications, and 21% stopped treatment early. 
An early metabolic response based on 18FDG-PET scans was seen in 3 of 15 
patients but did not correlate with RECIST response. MYB gene translocation was 
observed and significantly correlated with overexpression of MYB but did not 
correlate with FGFR1 phosphorylation or clinical response to 
dovitinib.Conclusions: Dovitinib produced few objective responses in patients 
with ACC but did suppress the TGR with a PFS that compares favorably with those 
reported with other targeted agents. Future studies of more potent and selective 
FGFR inhibitors in biomarker-selected patients will be required to determine 
whether FGFR signaling is a valid therapeutic target in ACC. Clin Cancer Res; 
23(15); 4138-45. ©2017 AACR.