In cell and gene therapies, access and cost limit the use of these treatments. “Historically, the genesis of cell therapy and later cell and gene therapy emerged in academic centers combining scientific discoveries of the immune system and implementing these discoveries in clinical trials to address unmet medical needs, particularly in cancer and infectious diseases,” says Øystein Åmellem, PhD, director of cell therapy at Thermo Fisher Scientific. “Today, decentralization of cell and gene therapy is emerging as a response to the high cost of such therapies, but also to reduce logistical complexity, risk, and time that ultimately will give patients and their families better healthcare.”
So far, decentralizing the manufacturing of cell and gene therapies gains more attention than traction. “Decentralization of cell and gene therapy is a hot topic discussed in conferences and publications and thus a strategy that most players in this industry are discussing,” Åmellem notes. “There are nonprofit organizations, governmental sponsors, and a few industrial companies that sit in the driver’s seat.” Despite the clinical studies underway, Åmellem points out that “as always with innovation in the healthcare industry, someone needs to demonstrate a sustainable business model for decentralized cell and gene therapy before the majority follows.”
Manufacturing cell and gene therapies closer to the patients makes many steps easier. “The vision is a scenario without the need for the cost and time associated with airplanes and, for many countries, the complexity of customs clearance,” Åmellem explains. “In autologous cell and gene therapy, the time required to manufacture the personalized drug ready for re-injection—referred to as vein-to-vein time—ranges between three and four weeks.” Decentralized manufacturing could cut that time in half or more.
Beyond the cost in money and time, the vein-to-vein time impacts a patient’s experience or even being eligible for a treatment. “This period can be daunting for the patients awaiting treatment, and vein-to-vein time can make cell and gene therapies unsuitable for patients with rapidly progressing disease,” Åmellem says.
Making this leap to decentralization, though, depends on technology, such as using modular equipment. “All decentralization manufacturing needs robust systems that are easy to transfer from a centralized process development and validation site to all the decentralized drug manufacturing sites,” Åmellem explains. Those systems should be closed, automated, and flexible. “The cell and gene therapy industry is in rapid evolution towards high volume—such as in various allogeneic drug platforms—and miniaturization—for instance in autologous CAR-T therapies,” Åmellem says. “So, flexibility is required for development of the next generation of cell and gene therapy drugs.”
In essence, decentralization depends on ecosystem-wide changes. “I believe we are at a crossroads in the cell and gene therapy industry,” Åmellem says. “The need for a modular approach will continue to benefit the industry related to the flexibility and scalability required to support the evolution of cell and gene therapies.” Developing the best technologies, though, will depend on “partnerships across the industry,” Åmellem says.
As Åmellem concludes: “The vision is that decentralization will make cell and gene therapies more attainable for more patients from an accessibility, cost, and time perspective.” Making that transition, though, will take more development and time.