- French biotech firm Cellectis announced a major breakthrough on Thursday as an 11-month-old baby girl with fatal, refractory leukemia was apparently cleared of her cancer two months after treatment with the company's experimental CAR-T therapy.
- Baby girl Layla was given the treatment, UCART19, by scientists at London's Great Ormond Street Hospital under compassionate use protocol after doctors had all but given up on her chances of survival.
- Cellectis is one of several major firms working on hotly-anticipated CAR-T therapies, alongside Novartis, Kite Pharma, and Juno.
Thursday was quite a day for Cellectis and its T-cell treatment, which the company says stands out against Novartis, Kite, and Juno's experimental therapies due to its universality (the other three firms are all working on therapies that would need to be highly personalized for individual patients).
According to Reuters, doctors were ecstatic. "Her leukemia was so aggressive that such a response is almost a miracle," said Paul Veys, a professor and director of bone marrow transplant at GOSH who led the team treating Layla. "As this was the first time that the treatment had been used, we didn't know if or when it would work, so we were over the moon when it did."
Now, to reign in the excitement a bit: These are extraordinarily preliminary results, just two months out after treatment. And as the biopharma industry has come to learn time and time again, initial positive results from exciting therapies can fade over the course of time, so there's still a lot more observation and wider-scale clinical testing to be done before UCART19 can be hailed as a game-changer.
If future trials do show that Cellectis' therapy holds the promise of universal administration to patients, then the company may truly have a significant advantage over its competitors. As BioPharma Dive has previously reported, the next major challenge for companies focusing on CAR-T is large-scale production due to the massive costs associated with personalized treatments. Per-patient production costs for these therapies can range from $50,000 to $100,000, which pushes the cost of therapy up to at least $200K to $300K for a large company like Novartis, and as high as $450,000 for smaller companies.