Gordon B. Mills, MD, PhD, chairman, Department of Systems Biology, chief, Section of Molecular Therapeutics, Professor of Medicine and Immunology at the University of Texas MD Anderson Cancer Center, discusses examples of recent and unexpected responses to medications in diseases that did not have a treatment.

1. Crizotinib (Xalkori), a recently FDA approved drug that targets the EML4-ALK translocation in non-small cell lung cancer, has shown remarkable responses including a disease control rate of 74% and a demonstrated 50% and 61% median objective response rate.
2. Rapamycin analogs have demonstrated endometrial benefits in over 50% of patients.
3. BRAF targeted therapies, such as Vemurafenib (Zelboraf), which is approved for BRAFV600E-positive unresectable or metastatic melanoma, have demonstrated remarkable progress, including a 74% reduction in disease progression.

In order to drive this type of therapy to the next level the first step will be to construct combinatory therapies. The field will begin to evolve from a short-term response that benefits a lot of patients, to a long-term durable response, and finally a cure.