CAR-T cells are complex products, and translating them from basic and preclinical research to clinical trials and commercialization can be challenging. Understanding the regulations and developing a protocol that puts safety first—at every stage of development—are important first steps in bringing promising CAR-T cell therapies to the patients who need them most.
CAR-T Cell Therapies: Navigating Regulations and Managing Toxicities in Clinical Trials
Immuno-gene therapeutics are transforming the therapeutic landscape of cancer therapy. Last year’s approvals of the first two chimeric antigen receptor (CAR)-T cell therapies—tisagenlecleucel (marketed as Kymriah™) and axicabtagene ciloleucel (marketed as Yescarta™)—heralded a new era of precision oncology.
With more than 300 CAR-T cell clinical trials ongoing, we have seen only the tip of the iceberg.1 While CAR-T cell therapies have demonstrated dramatic efficacy in hematological malignancies, these treatments are associated with serious toxicities. As the number of clinical trials involving CAR-T cell therapies in both hematologic malignancies and solid tumors increases, there is an urgent need for sponsors of these trials—many of them small to midsize biotech or specialty pharma companies—to fully understand the regulations and safety considerations surrounding these novel treatments.
Understanding the Regulatory Landscape
Clinical trials of immuno-gene therapeutics are becoming increasingly common, and regulatory guidelines are steadily evolving to keep pace with advances in technology. As part of the study planning process, it is critical for sponsors of gene therapy technologies to gain a detailed understanding of the regulatory environment in every country where the study may be conducted.