Cell therapy 101 (part 1)Challenges of early clinical development: a ‘thumbnail sketch’

Cell & Gene Therapy Insights 2022; 8(10), 1413–1427

DOI: 10.18609/cgti.2022.207

Published: 29 November 2022
Michael D Leek

Cell therapy has long been regarded as a possible therapeutic panacea, having potential to restore structure and function of damaged and diseased tissue and whole organ systems. Historically, therapeutic targets have included treatment of diabetes, liver failure, kidney disease, CNS disorders, connective tissue disorders, and more recently, cancer. Casual observers will see cell therapies as a ‘bright new shiny toy’, a potential cure for cancer, based on some of the CAR-T data recently generated. This, however, is not the case: those of us working in the cell therapy space back in the late 80s remember the days when such products were classified as medical devices, and the first autologous and allogeneic products were starting to emerge from companies such as Advanced Tissue Sciences and Organogenesis. Many of the challenges we faced in those early days still exist. We are still grappling with logistics of shipping cell-based products from clean room to clinic, regulatory uncertainty, patient-to-patient variability with autologous approaches, donor-to-donor variation of allogeneic products, and high cost of goods. Moreover, uptake of cell therapies by established pharmaceutical companies has been slow, as cell-based products fail to achieve the requirements around scalability, reproducibility, and cost that many major corporations see as mandatory. Numerous academic and industry-based groups around the world are working on a wide variety of cell-based projects; however, not all of these groups understand the complexities of commercializing the technology from bench to bedside. In addition to gaining appropriate funding to enable progression from the laboratory to clinic, all of these companies face the challenge of navigating a complex regulatory landscape, plus transitioning to GMP manufacture. Particular issues which need to be addressed include: 

  1. Transfer from research to a clinical-grade product
  2. Manufacture product to GMP
  3. Quality testing – what needs to be done and how?
  4. What are the regulations, and how do we adhere to them?
  5. What are the challenges for commercialization? 

This article aims to address the above in a broad-brush manner, subsequent ‘Cell therapy 101’ will review specific areas in more detail.