CAR-T hype overlooks safety and cost, says analyst


The excitement surrounding the recent CAR-T pre-clinical breakthrough should be tempered by safety and cost concerns, according to an analyst at GlobalData.

The encouraging results were presented at the American Association for the Advancement of Science's annual meeting, where remission rates of over 90% were reported in terminal late-stage acute lymphoblastic leukaemia (ALL).

However, GlobalData's Oncology and Haematology Analyst Cai Xuan, PhD, has highlighted the huge estimated costs, amounting to at least $300,000 per patient.

"T-cell therapies must show curative ability in order to justify such a high price tag in comparison to existing treatment options such as stem cell transplantation, which can cost from around $100,000 to $200,000," he pointed out.

"To date, only a handful have gone ahead, in a limited number of patients, due to the high cost, as well as the long and difficult manufacturing processes involved in administering T-cell therapy. This presents a huge scalability problem which cannot easily be solved."

The solution to scalability issues surrounding CAR-T therapies may have already been addressed, however, with Cellectis' 'off-the-shelf' leukaemia candidate UCART19, which can be used in any patient.

Xuan also commented on safety concerns around CAR-T therapy: "The lack of long-term follow-up data presents the danger of additional long-term toxicities being revealed in the future."

Although having produced promising results, some recent CAR-T studies have also reported alarming safety concerns in small numbers of trial participants.

Bluebird Bio saw its stock plummet after safety concerns with its lead CAR-T candidate LentiGlobin BB305 were highlighted following a treatment trial in beta-thalassemia. Similarly, data presented from a National Cancer Institute multiple myeloma trial reported severe toxicities in the one trial participant who experienced complete remission.

The recent ALL trial, funded by Juno Therapeutics, reported a total of seven participants needing intensive care following the development of 'cytokine release syndrome'.

Linda Banks

19 February, 2016