Successful use of next generation genomic sequencing (NGS)-directed therapy of clear cell carcinoma of the ovary

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 Successful use of next generation genomic sequencing (NGS)-directed therapy of clear cell carcinoma of the ovary (CCCO) with trametinib and metformin in a patient with chemotherapy-refractory disease

 Background

Clear cell carcinoma of the ovary (CCCO) represents a distinct histopathologic subtype [1] comprising 3.7–12.1 % of epithelial ovarian carcinoma (EOC). In general, CCCO has earned notoriety for being a particular challenge for management characterized by higher recurrence rates among patients with early stage disease, poor responsiveness to chemotherapy, especially platinum [2–7], de novo drug resistance, and inferior survival compared to other subtypes of EOC [8]. In light of these characteristics, many investigators have opined that CCCO deserves a unique treatment strategy as a distinct disease entity. Nevertheless, current guidelines recommend similar adjuvant treatment regimens as used for other EOC histologies....

....This patient was fortunate to gain access to a drug that is usually unavailable to ovarian cancer patients due to strict payor policies that follow the drug license to the letter. However, over-zealous regulatory behavior and wholesale restriction of physician prescribing can deprive patients of life-saving medicines. In an era when molecular insights have theranostic value that can outperform the limitations of standard therapeutic options, molecular tumor boards to determine the appropriateness of novel therapies could help patients access breakthrough approaches whose availability is hampered by the regulatory mechanism, but which are nevertheless readily available and tailor made for their individual cancers.....

 (The term “theranostics” was coined to define ongoing efforts in clinics to develop more specific, individualized therapies for various diseases, and to combine diagnostic and therapeutic capabilities into a single agent.)