Doctor discussing treatment options with a patient.
Doctor discussing treatment options with a patient. Photo courtesy: 2019 Fotolia

Cancer treatment is in the midst of a seismic shift: With the emergence of targeted therapies, we’re now in an era of more personalized, precision medicine—giving cancer patients more options, and more hope, than ever before.

A new crop of potentially life-saving drugs is radically changing what the future of cancer care looks like— and improving outcomes for patients suffering from some of the most prevalent cancers in the United States erdafitinib (Balversa®; Janssen Pharmaceutical Companies of Johnson & Johnson), the first-ever personalized treatment for bladder cancer, is one of these drugs.

A new targeted therapy for adult patients with locally advanced or metastatic bladder cancer (that has a type of susceptible genetic alteration known as FGFR3 or FGFR2), erdafitinib has shown great promise in early clinical trials— so much so, in fact, that erdafitinib has been granted an Accelerated Approval from the U.S. Food and Drug Administration (FDA).

Accelerated Approval
The decision to grant Accelerated Approval is based on clinical trial data showing that the drug is thought to predict a clinical benefit to patients.  Accelerated Approval is a ‘fast track’ of sorts that enables the FDA to approve drugs for serious conditions in order to fill an unmet medical need.

And in this case, the need is certainly there: Bladder cancer is the sixth most common cancer in the U.S., with 81,000 new cases diagnosed each year, and 17,000 deaths annually. The most common type of bladder cancers arises from the transitional epithelium and are called transitional cell or urothelial carcinomas. Typically, the patient’s choice  of treatment will depend on the invasion of the muscle layer of the bladder, and has traditionally involved surgery, chemotherapy and/ or radiation treatment.

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Immunotherapy is also increasingly used in advanced stages, but until now there have been no approved targeted agents for bladder cancer. The way physicians approach the disease is rapidly transforming though— and a number of different targets have been identified in bladder cancer, including: RAS, PIK3CA,TSC1 (a regulator of mTOR pathway activation), TP53, FGFR, FGFR2, and FGFR3.

Clinical trials
Erdafitinib, which acts specifically on FGR2 and FGR3, was studied in a clinical trial that included 87 patients. All participants in the trial had locally advanced or metastatic bladder cancer, with FGFR3 or FGFR2 genetic alterations, and had previously been treated with chemotherapy to little or no avail. The overall response rate to erdafitinib in these patients was 32.2%, with 2.3% of patients having a complete response and nearly 30% having a partial response.

Tailoring treatment in accordance with the patient’s precise genetic mutation or biomarker is quickly becoming the new standard in cancer care. The use of targeted therapies in bladder cancer is still an emerging field of research— but the apparent success of erdafitinib showcases the promise they may hold.

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