Treatment with the drug erdafitinib, an inhibitor of FGFR, is effective for advanced urinary tract cancer with alteration in the FGFR 2 or 3 genes. This is indicated by the conclusions of an international study published in the New England Journal of Medicine, directed by the University of Texas, MD Anderson Cancer Center of the United States, where Jesús García-Donas, head of the Gynecological and Genitourinary Tumors Unit of the Clara Campal Integral Cancer Center HM CIOCC, has worked.
The phase II study involved 210 selected patients from more than 2,200 candidates in 14 countries, with a duration of 3 years. The authors have confirmed that the medication is effective in 40 percent of patients by blocking the FGFR protein.
After doing the genetic study of the candidate patients to be included in the clinical trial, the work began with a first stage of finding the correct dose and determining the appropriate dosage of the drug. Next, a second phase was completed to establish its effectiveness and safety.
77 percent of cases in which the advanced ureter or bladder tumor had a mutation of the FGFR 2 or 3 gene experienced a decrease in the size of metastases and up to 40 percent achieved a significant reduction, according to the evaluation criteria RECIST
Targeted oral therapy also achieved a 59 percent response in patients in whom immunotherapy had previously failed. Therefore, experts consider that it could be a viable option for these cases that need alternative treatments. Arlene Siefker-Radtke, principal investigator of the study, has indicated that “patients have been desperately in need of alternative strategies, especially when a large number cannot tolerate current standards of care.” “We are very pleased to see a 40 percent response rate in patients treated in this clinical trial. It not only worked well in patients with lymph node metastases, but also in patients with a high volume and a very aggressive disease, ”he said.
First FDA-directed targeted therapy
The results of the trial, in which 99 patients with metastatic or non-surgically resectable urothelial cancer and alterations verified in the FGFR3 gene participated, led to the approval of erdafitinib in April by the American Medicines Agency (FDA), which converted it in the first targeted therapy approved to treat patients with advanced bladder cancer.
A phase III trial is currently being conducted to analyze the efficacy of erdafitinib in relation to chemotherapy or pembrolizumab control point blocker inhibitor in patients with metastatic urothelial cancer and FGFR3 mutations.