Product characterisation key to successful CAR T therapies, Autolus
Last year saw the US approvals of the first two chimeric antigen receptor (CAR) T-cell therapies: Novartis’ Kymriah (tisagenlecleucel) and Kite/Gilead’s Yescarta (axicabtagene ciloleucel).
Many, including the US FDA, heralded this as the beginning of “a new frontier in medical innovation” but patient groups have hit out at their list prices: $475,000 (€389,000) for the single treatment of Kymriah and $373,000 for Yescarta.
And speaking at last month’s Cell Therapy Manufacturing & Gene Therapy Congress in Amsterdam, Jim Faulkner, SVP and head of manufacturing at UK-based CAR T-cell therapy developer Autolus, said it was imperative to reduce the costs to allow such therapies to succeed.
“We’ve raised the promise of cell therapy and shown what can be done, yet behind that are price tags that are eye watering and issues around our ability to supply the market and actually deliver these products,” he told delegates.
“It is absolutely imperative that we take what is now beyond a promise into something that’s a reality for patients.”
Understanding the biology
The CAR T production experience so far has been variable, difficult to scale and expensive, “so we are not in a great place right now,” he added. Ideally, companies need to bring about a good commercial process consisting of a cheap, robust, predictable and scalable platform making consistent product.
“At the heart of these products are T Cells, and understanding these T Cells – what they do, how they behave ex vivo, how you can manipulate them – is absolutely critical for you to be able to produce a quality product.”
And while he said there are other factors which will aid successful CAR T commercialisation and reduced prices – having the right safety profile, clinical efficacy, facility and logistics network – he said understanding the product and the biology is core.
Without adequate characterisation, it is not possible to know what the product is, how process changes impact the product, and how to monitor and control the process parameters, he argued.
“If you do not understand what the product is that you are trying to make, you cannot design the process to try and make that product. It’s a very circular argument.”
