Establishing Rationale for the Clinical Development of Cell Therapy Products: Consensus between Risk and Benefit

Abstract

Despite long-term research achievements, the development of cell therapy (CT) products remains challenging. This is because the risks experienced by the subject and therapeutic effects in the clinical trial stage are unclear due to the various uncertainties of CT when administered to humans. Nevertheless, as autologous cell products for systemic administration have recently been approved for marketing, CT product development is accelerating, particularly in the field of unmet medical needs. The human experience of CT remains insufficient compared with other classes of pharmaceuticals, while there are countless products for clinical development. Therefore, for many sponsors, understanding the rationale of human application of an investigational product based on the consensus and improving the ability to apply it appropriately for CT are necessary. Thus, defining the level of evidence for safety and efficacy fundamentally required for initiating the clinical development and preparing it using a reliable method for CT. Furthermore, the expertise should be strengthened in the design of the first-in-human trial, such as the starting dose and dose-escalation plan, based on a sufficiently acceptable rationale. Cultivating development professionals with these skills will increase the opportunity for more candidates to enter the clinical development phase.

Assessment of the Therapeutic Effectiveness of Glutathione-Enhanced Mesenchymal Stem Cells in Rat Models of Chronic Bladder Ischemia-Induced Overactive Bladder and Detrusor Underactivity

“…MSC-based therapy has gained significant attention as a potential treatment strategy for intractable disorders, including OAB and DUA (30). To achieve successful outcomes in clinical research, it is essential to comprehend the dis- ease-specific characteristics of stem cells and select the most suitable candidates, while also improving their functionality (31). Our previous studies shed light on the activation of genes associated with the inflammatory response and oxidative stress in bladder tissue with CBI-induced DUA (7), underscoring the significance of selecting stem cells with an enhanced antioxidant capacity to improve therapeutic efficacy.…”