Cancer Drugs Approved in 2012

(part two)


Medullary thyroid cancer is a “rare” form of cancer that is very aggressive and deadly that has notoriously very poor response to chemotherapy. This past year cabozantinib (Cometriq) was FDA approved for metastatic disease. Cabozantinib is a tyrosine kinase inhibitor that blocks several pathways promoting cell death. The drug is oral and has as potential side effects:  perforation and hemorrhage linked to its antiangiogenic properties. Approval was expedited due to lack of effective therapies for this disease.

            In recent years numerous new drugs were developed and approved for renal cell cancer. This past year another drug was approved called axitinib. It is a tyrosine kinase inhibitor that is oral. The blood pressure needs to be monitored and blood clots can develop as major side effects.

            It is unusual to have advanced basal cell cancer usually it is recognized and surgically treated when it is relatively confined. Untreated basal cell can be locally very destructive and this past year a very effective drug for advanced disease was FDA approved by the name of vismodegib (erivedge) it is an oral drug that is a hedgehog pathway inhibitor that inhibits proliferation.  Muscular spasm and alopecia are the major side effects. The response rate and healing of the ulcerated lesions is remarkable.

            Ponatinib (iclusig) was FDA approved for the rare CML cases with T3151 mutation of BCR-ABL or for acute lymphoblastic leukemia Philadelphia chromosome positive. Ponatinib is an oral kinase inhibitor. Hypertension and cytopenias are the major side effects.  This drug is very effective in refractory leukemias.

            Of notice is the majority of the FDA cancer therapies approved in 2012 are not chemotherapeutic agents and for the most part are oral agents. We for the most part switched to targeted therapies that block pathways implicated in cell growth, rather than causing direct DNA damage typical of chemotherapeutic agents. With the change in mechanism of action of the new drugs (tyrosine kinase inhibitors / proteasome inhibitors / monoclonal antibodies / antiangeogenic), the most dreaded  side effects typical of chemotherapeutic agents have disappeared like vomiting, hair loss and bone marrow suppression and many cancers are being transformed into chronic diseases.