Cellular and gene therapy products (CGTs) or advanced therapy medicinal products (ATMPs) are an ingenious class of disparate research-driven biopharmaceuticals because of their potential to offer therapeutic breakthroughs for many diseases. Especially, in case of life-threatening cancers for which conventional medicine and various other modalities have not been completely effective.
The development of chimeric antigen receptor (CAR) T-cell therapy for hematological malignancies is a promising therapeutic advancement in the CGTs. There is a heightened interest in applying CAR T-cell therapy to different hematologic malignancies over the last decade. Despite the success achieved so far, significant challenges have been recognized, including the process of manufacturing CAR-T cells, emerging regulations, safety concerns, and economic and logistic burdens.
In this article, the focus is on:
- The evolution of CAR T-cell therapy, advantages over conventional therapies, autologous and allogenic CAR T-cell therapies,
- Key challenges,
- Treatment emergent adverse events,
- Routine and additional pharmacovigilance activities, and
- Measures to minimize or mitigate the risks identified.
This article also provides an insight on designations and expedited developmental schemes offered by regulatory authorities to foster and optimize the development of CGT with unmet medical needs for making the products available to the patients