Facing Up to the Challenges in Cell Therapy

What we asked: “Looking ahead to the next 5–10 years, what will be the key disruptors and/or what can be improved upon in the pharma industry?”

Response from: Jason Bock, CEO, CTMC

“The vast role played by the immune system across a spectrum of diseases has been the key disruptor in the biotech industry over the past decade. Biotech has developed therapeutic strategies to either activate or regulate the immune system for different purposes. For cancer treatment, checkpoint inhibitors are used to release the brakes on the immune system, while for autoimmune diseases like rheumatoid arthritis, anti-TNF medications are employed to suppress the immune response.

“Precise targeting and long serum half-life of monoclonal antibodies have been utilized as tools to modulate specific pathways of an immune response. As our understanding of the immune system has deepened, the industry has shifted to a more direct path to utilize immune cells as the effectors. The ability to directly engineer an immune response that can persist for years – or even decades – through living cells has enormous potential. The emerging field of cellular therapy will generate new therapies to treat refractory medical conditions, and could transform how healthcare is administered through the single-dose, curative potential of this modality.

“While current progress is promising, autologous cell therapy comes with challenges in chemistry, manufacturing, and control for developers. These therapies are the most complex ever developed as they are derived from each patient’s own cells. This uniqueness necessitates a complete reimagining of the traditional supply chain and economics. Since the starting material is the patient’s own cells and manufacturing occurs on demand, the relationship between the clinic and manufacturer is intertwined in such a way that the manufacturer functions like a pharmacy for cell therapies. Although the field has seen dramatic clinical efficacy responses, creating a fit-for-purpose supply chain infrastructure that meets demand at a manageable cost remains a significant hurdle.

“One approach to overcome these challenges would be the creation of key regional manufacturing hubs around the country aligned with major medical and population centers. An industrial, built-for-purpose regional “cell pharmacy” could develop locally integrated supply chain and logistics to streamline the coordination of cell collection, production, and infusion. These hubs could provide cell therapies from multiple commercial sponsors to benefit from economies of scale and drive a shift from open and manual processes to closed, automated ones. Digital, cloud-based quality systems will be essential to maintain consistency and process control across such a network. Such interconnectedness is a key differentiator from the concept of fully decentralized, localized manufacturing.

“We have a golden opportunity to transform healthcare from the continuous management of chronic conditions to a short-interaction, curative model. Cell therapies have demonstrated efficacy in illnesses ranging from terminal cancer to sickle cell disease. By leveraging what we have learned over the last decade in complex biologics manufacturing controls and applying those lessons in this new context, we can revolutionize health treatments. Future generations will likely look back in amazement at how medicine was practiced before the advent of cell therapy.”

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