Cell Notes: Unraveling the Complexities of Cell Therapy: MOA, Potency and Efficacy Explored

May 24, 2024

In the rapidly evolving field of cell therapy, the journey from concept to cure is filled with scientific and regulatory challenges. A recent article in the Journal of Translational Medicine by Simon et al, sheds light on the intricate landscape of cell therapy product (CTP) development. The article, titled “Mechanism of action, potency and efficacy: considerations for cell therapies,” serves as a valuable resource for professionals navigating these complexities.

The authors delve into the heart of CTPs, discussing the critical importance of understanding the mechanism of action (MOA). This is the foundation upon which the efficacy and potency of a therapy are built. However, the article demonstrates that the MOA for many FDA-approved CTPs remains a mystery, a testament to the enigmatic nature of biological systems.

Potency, a measure of the activity of a CTP, is another focal point of the discussion. The authors introduce a framework to clarify the relationships between MOA, potency, and efficacy, which are often muddled in the development process. They highlight a significant hurdle--- the lack of correlation between potency test results and clinical outcomes. This gap underscores the need for more predictive and accurate tests that can bridge laboratory findings with real-world therapeutic success.

The article also discusses the design of clinical trials, which are focused on safety and efficacy. The authors argue that there may be unrealistic expectations that clinical trials will determine the MOA or validate a potency test. This may not be impossible, but it certainly is not the norm, and suggests a need to reframe what we expect to learn from clinical trials.

The inherent complexity of CTPs, with their various interactions and behaviors, adds layers of difficulty in pinpointing a singular MOA or developing straightforward potency tests. This complexity is not just a scientific challenge but also a regulatory one, as it impacts communication and expectations between product development groups and regulatory bodies.

The insights shared by the authors highlight the importance of improved methodologies emphasizing the need for a deeper understanding of biological mechanisms at play and for more robust and reliable testing procedures that can keep up with innovative treatments being developed. Please feel free to share your thoughts on potency via email or in the comments on LinkedIn.