In 2009, gang members in a Maryland prison were caught using their smartphones to approve targets for robberies and even to order seafood and cigars.
Palifermin, a human keratinocyte growth factor (KGF) recombinant protein, is the only FDA approved targeted therapy for preventing oral mucositis in bone-marrow transplant patients (4% of the at-risk population), but it has no effect on existing mucositis (Sonis, 2010).
Currently, sorafenib is the only approved targeted therapy for patients with advanced HCC.
All currently approved targeted therapies for metastatic RCC (sunitinib, sorafenib, temsirolimus, everolimus, bevacizumab, pazopanib and axitinib) lack FGFR activity [ 19].
The results of clinical trials show that approved targeted therapies for rare disease subtypes are not universally effective.
Sorafenib (Nexavar), a multiple kinase inhibitor, was approved by the US Food and Drug Administration for the treatment of advanced HCC in 2007, and is the first clinically approved targeted drug therapy for HCC.
The majority of clinical trials investigating novel agents have been negative and the only approved targeted-therapy is bevacizumab, for which reliable predictive biomarkers still elude us.
As mentioned earlier, HIF-1 plays a significant role in HCC chemotherapy resistance and thereby limits the efficiency of sorafenib, which is the only approved targeted small molecular drug for HCC therapy.
Patients eligible for FDA approved targeted therapies (erlotinib, afatinib, crizotinib, and ceritinib) are those diagnosed with advanced stage lung adenocarcinomas with activating mutations in the EGFR gene or EML4-ALK fusions [2, 13, 19 22].
For prospective anti-cancer targets, the expression parameters correlate with the extent of clinical development, being higher for FDA approved targets (black bars) as compared with the mix of target and non-targets (striped bars, "cancer-related" category).
