Molecular targeted therapy by using a drug that reduces the growth and spread of cancer cells via inhibition of a specific protein is a foundation stone of precision medicine and treatment. Recently, we could identify lead molecules that selectively inhibited proliferation and metastasis of cancer cells originating from colon, prostate, breast, and skin. These tumors are the four top cancer diseases worldwide with highest prevalence. Therefore, there is an urgent need for development of novel drugs that can cure or prolong the survival of these patients. At the initial step and as a proof of concept, we focused on metastatic melanoma as a model system. We have succeeded to identify and characterize one class of small molecules and optimized the hit compound to one derivate. This inhibitor and its derivate could effectively block the activity of its target in different human melanoma cell lines. Reduced activation of the target protein mediated by our newly discovered inhibitors resulted in slower growth, proliferation and metastasis of cancer cells both in cell culture and in a preclinical melanoma xenograft animal model. Extended toxicology experiments in healthy animals showed that our inhibitor has no major effect on the survival and health of the treated animals. Our data suggest that we succeed to generate new, safe, efficient and patentable compounds in a preclinical verification. Our future goal is to achieve positive response of our novel inhibitor in clinical trials and further using this small molecule as a new innovative therapy for cancer patients.
