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The treatment strategy for human disease has evolved dramatically since 2000 as a result of advances in molecular genetics, cell biology, and pharmacology. Increased understanding of molecular pathophysiology has allowed for the development of therapeutics that interact with specific molecular targets associated with disease. Agents that are the product of rational drug design, in which compounds are deliberately designed to interact with a biologic target, are often referred to as “targeted therapy” and form one of the cornerstones of “precision medicine.” The potential of targeted therapy is perhaps best exemplified with oncologic disease, but the promise of rational drug design is beginning to be seen across all fields of medicine.

Discussing all targeted therapies in medicine is beyond the scope of this chapter. Consequently, we focus on those molecular targeted therapies that have the most overlap with dermatology in either their indication or the adverse effects caused by their use (Table 194-1). Thus we review targeted therapies designed to interact with the tyrosine kinases BCR-ABL, c-KIT, PDGFR and EGFR; smoothened; histone deacetylases; and proteins of the MAP kinase pathway. We finish with a section on the future directions in melanoma targeting AKT and ERK proteins.

Of note, marketing indications, contra-indications and warnings are dynamic and constantly evolving. Thus, those covered in this chapter are current as of the writing. Please refer to the FDA product label for the most up-to-date information.

Table 194-1Molecular Target Therapies



  • The small-molecule tyrosine kinase inhibitors imatinib, nilotinib, ...

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