ABSTRACT

Elemer Piros, PhD,a Istvan Petak, MD, PhD,b Attila Erdos, MD,c John Hautman, JD,a and Julianna Lisziewicz, PhDaaeMMUNITY Inc., Bethesda, Maryland, USA bOncompass Medicine Group, Budapest, HungarycCaVax Kft., Szeged, Hungary Keywords: targeted drugs, immunotherapy, vaccine, cancer, genetic biomarker, predictive biomarker, prognostic biomarker, companion diagnostics, companion treatments, personalized medicine, personalized treatments, molecular diagnostics, market opportunity, comprehensive tumor profiling, targeted immunotherapy, nanomedicine

Over the past two decades, there have been significant advances in our understanding of some of the common molecular aspects of cancer, facilitating targeted drug development for patients sharing features that are identified from their tumor. The introduction of trastuzumab in 1998 ushered in the era of personalized medicine in oncology. Trastuzumab is indicated for HER2+ breast cancer patients identified by the companion diagnostic HercepTest. For patients who do not overexpress HER2 (75-80% of the total), trastuzumab would not be beneficial at all. In a hypothetical “all comers” clinical trial, trastuzumab probably would have required many more patients to prove a statistically significant benefit. The actual registration trial of trastuzumab included only 470 patients screened by the HercepTest and took only 1.6 years to complete. Based on the response rate and frequency of HER2 amplification, it was calculated that without the pre-selection of patients, the minimum number of patients needed for the trial would have been 2200 and the trial would have taken 10 years. The “targeted” trial saved $35 million and the faster market entry led to $1.7 billion of additional revenue [2, 3].