In the past few days, Novartis published further encouraging data suggesting that the treatment that saved Emily is on course to become one of the first so-called CAR-T therapies on the market.
CAR-T is shorthand for the genetically engineered chimeric antigen receptors T-cells which these therapies deploy to hunt and destroy cancer.
The most advanced CAR-T therapies developed by Novartis, Juno and Kite are so-called “Autologous” treatments which must be personalised for each patient.
The modified cells are then reinfused into the patient’s blood, where they proliferate.
Analysts have estimated the treatments are likely to cost up to $300,000 per patient – potentially making them among the most expensive therapies on the market at a time when high drug costs are facing increasing scrutiny in the USand Europe.
It is developing a so-called “Allogeneic“, or universal, CAR-T therapy made from donor cells that can be used by any patient.
Ré Choulika, co-founder and chief executive of Cellectis, says the autologousCAR-T therapies are “a service not a product” and will be too expensive for widespread adoption.
“A $1m therapy is not my idea of healthcare,” he says.
Investors seem impressed: shares in Cellectis have more than doubled this year and Pfizer, the US drug group, has struck a deal to license the product in theUS. However, Usman Azam, head of cell and gene therapy for Novartis, says it is too early to declare the success of Cellectis’s approach based on a single patient compared with the much larger body of evidence behind autologous therapies.
James Noble, chief executive of Adaptimmune, a UK cell therapy company, says technology has moved on since the Dendreon failure, including the ability to freeze the cells before and after they are re-engineered.