July 22, 2019
Cell therapy and gene therapy — three summer reads that “get it”
In cell therapy, gene therapy, and personalized cancer vaccines, most media coverage understandably focuses on the medicine — the amazing science behind it, and the transformative treatment options being created for patients. But as the field of personalized therapeutics expands, a few authors are catching on to the fact that other innovations — including new enabling digital technologies — are also required.
This summer, we’re taking note of three pieces by writers who “get” the full extent of transformation required in advanced therapies. All are worth a read this summer.
Along with great behind-the-scenes looks at the science, and moving medical narratives from doctors and patients, this piece also examines cell therapy manufacturing processes and innovations that will reduce cost:
“But nowhere in these lessons did I encounter the Japanese term kaizen—the continuous improvement of a manufacturing process to its leanest, most efficient form. It would have been a worthwhile lesson. Engineers in the world of industrial manufacturing obsess about this. But as doctors, as medical scientists and inventors, we are taught to think about curing deadly diseases or about creating new systems of care. We want to battle the mortal coil, not the plastic coil. We want to close the gaps in access to medical care, not the gaps between hooks in the operating room. We give priority to proofs of principle, not to the particularities of production. Yet, if the newest generations of therapies are to succeed at scale, it may be the small skirmishes that determine the outcome of the larger war. For cellular therapy to reach the masses, its innovators cannot ignore the most trivial-seeming details of the human and material factors of the manufacturing process. Perhaps we need a change in our culture, or even in our vocabulary. In Cleveland, as in operating theatres around the world, the clinicians were in yarak. The new generation of medical care will be enabled by the ceaseless demands of kaizen.”
Vineti is working hard to help drive kaizen for cell therapy and gene therapy every day! (And if you’d rather listen than read, the online version of the New Yorker article includes a link to an audio version at the top.)
Reflecting the growing significance of cell therapies in many areas of oncology, these powerhouse partners in cancer research teamed up on this thoughtful white paper, with some key focus areas:
- Opportunities to accelerate early discovery through IND flexibility
- Opportunities to accelerate the optimization of cell products during late stage development and post licensure
- Establishing a scientific development consortium to share fundamental data and/or expedite investigational product development and testing processes
- Establishing agreed upon standard technologies to facilitate technology transfer between academic innovators and industry GMP producers
That last point, related to standardization, is central to Vineti’s work. We help lead the Standards Coordinating Body, and the drive towards standardization shapes the development of our software platform for cell and gene therapies. So we took extra note of the following recommendation in the FOCR/PICI white paper, and look forward to the discussion!
“Regulatory guidance for new cell therapy digital platform: The digital platform is a unique and critical aspect of cell therapy manufacturing, and various components such as Chain of Identity (COI) must be described in the BLA. It will behoove the field to develop regulatory guidance akin to regulating the manufacturing facility where it would be inspectable at any time but operational changes under controlled procedures are allowed.“
“We Weren’t Prepared for CAR-T: Lessons from an Industry Executive,” by Louis Garguilo, Chief Editor of Outsourced Pharma, in Cell & Gene.
This overview of a talk by Joanne Beck. Executive Vice President of Pharmaceutical Development and Operations at Celgene, gives great insights into many of the challenges in CAR-T cell therapies, including these:
“I feel the biopharma industry was far behind on the process and analytical development and manufacturing technology side with CAR-T,” she says evenly. “So we’re scrambling. If you look at where we should be to make CAR-T accessible and affordable, we don’t have enough automation, the analytics is behind, and we haven’t developed ready-to-use data systems. The supply chain is still immature – we’re struggling with the patient and cell journey. “
The article addresses many different challenges in CAR-T, and sums things up with the ultimate reason we’re all working towards solutions: “After all, patients are waiting.”
Looking for another great read? Here’s our article on commercial-phase lessons for clinical-phase success in cell and gene therapies, written together with advanced therapies expert Simon Ellison. Thank you!
Here at Vineti, our software platform supports personalized cell therapy treatments for patients every day. We’re working to industrialize and scale personalized therapies ,and help this entire field grow. If you’d like to see our solution in action, please contact us to learn more or schedule a demo.