In recent years, innovation in oncology drug development has been dominated by advancements in immunotherapy and stepwise progress toward the promise of precision medicine. To date, the immune checkpoint inhibitors – PD-1/PD-L1 and CTLA-4 inhibitors – have transformed the treatment landscape for certain hematologic malignancies and solid tumors and have been approved for more than 15 indications. With the approvals of tisagenlecleucel (KYMRIAH™) and axicabtagene ciloleucel (YESCARTA™) in 2017, we officially entered the era of chimeric antigen receptor (CAR) T-cell therapies and next-generation biotherapies.
Precision oncology promises a new standard of cancer care where therapies are tailored to the molecular profile of a specific tumor, in the context of a holistic view of the patient. Currently, CAR T-cell therapies are among the most powerful – and expensive – tools in oncology and one of the most-studied types of investigative products. According to the Alliance for Regenerative Medicine’s Q1 2019 Data Report, there are 642 cell therapy or gene-modified cell therapy trials underway worldwide. Accompanying this research fervor is intense interest from patients and providers seeking to gain access to these treatments. But for the full potential of precision medicine to be realized, regulatory, technical, clinical, and economic frameworks will need to evolve or be adapted to the nuances of these novel treatments.
In this white paper, we provide a brief background on cancer immunotherapy and explore key challenges which must be overcome to deliver on the promise of precision medicine in oncology.