Development of Herceptin

A common frustration of drug development, particularly in oncology, is that relatively few scientifically sound ideas become translated into clinically useful drugs. For example, the realization that cancer frequently arises and is maintained by specific molecular alterations is now a well-established tenet of cell biology. However, the reduction of these scientific principles into clinical practice has resulted in relatively few newly developed therapeutic entities. One example is Herceptin, which was approved by the U.S. FDA in September 1998 for the treatment of women with metastatic breast cancer whose tumors overexpress the HER2 protein. Several features of Herceptin make it a unique therapeutic agent. First, it has the “designer” quality in that only women whose tumors contain a specific molecular alteration are eligible for therapy. Second, Herceptin is a biological agent with properties and features that vastly distinguish it from conventional cytotoxic chemotherapeutic drugs. In this review, we will highlight some of the key scientific elements in the discovery and the development of Herceptin.