Novartis adds to CAR-T's luster with more excellent cancer data

A new cancer treatment from Novartis ($NVS) and its partners at the University of Pennsylvania came through in another round of early studies, posting stellar results and burnishing the promise of therapies that train the immune system to bear down on tumor cells.

The drug, CTL019, left 92% of patients cancer-free in a leukemia study and performed well against lymphomas in clinical trial results presented at the annual American Society of Hematology (ASH) meeting, stirring hopes that the treatment can change the standard of care for blood cancers.

Novartis' candidate is part of a new class of therapies that work by removing T cells from a patient's blood and equipping them with targeting mechanisms called chimeric antigen receptors (CARs), which seek out and bind to proteins expressed by cancer cells. The resulting CAR-equipped T cells, or CAR-Ts, are infused back into the patient, at which point they home in on cancers and attack them as they would any commonplace infection.

In the first trial presented at ASH, investigators administered CTL019 to 39 children with the rare and deadly acute lymphocytic leukemia, finding that 36 of them, or 92%, experienced a complete response after infusion. After 6 months, 70% of the responders remained in remission, researchers said, and 75% were still alive.

In a separate study, CTL019 charted a 100% response rate and 80% complete response rate in 5 patients with follicular lymphoma, and the treatment shrunk the tumors of 5 out of 11 patients with diffuse large B cell lymphoma, with 36% showing no signs of disease at three months, researchers said.

And, in a 24-patient Phase II study on chronic lymphocytic leukemia, CTL019 notched a 42% response rate, eliciting a complete response among half of those subjects. In a 9-month follow-up, researchers noted a 68% rate of overall survival.

In each study, some or all of the patients experienced cytokine release syndrome (CRS), a bodily response to cell therapy that results in flu-like symptoms and, in some cases, death. Novartis reported three CRS-related deaths tied to CTL019, noting that each had a separate infection and that the severity of the syndrome tends to correspond to disease burden.

On the whole, the researchers are heralding CTL019's latest results as further evidence of the transformational power of CAR-T therapies for blood cancers, and Novartis is barreling toward commercialization as it pours money into Penn and prepares its breakthrough-designated treatment for a pivotal study.

"Innovation in the cellular therapy field is accelerating right now," Usman Azam, Novartis' head of cell and gene therapies, said in a statement. "When we see the response patients have to CTL019 when they have few options left, it's incredibly inspiring. Novartis will leverage our facility in Morris Plains, the first FDA-approved Good Manufacturing Practices quality site for a cell therapy, and the multicenter study for CTL019 in collaboration with the University of Pennsylvania, to broaden the reach of this therapy to additional patients in the clinical setting."

Meanwhile, Novartis and Penn are competing in clinic and courtroom alike with Juno Therapeutics, a well-funded upstart with a CAR-T platform of its own and promising data to match. And the technology's promise has brought a host of contenders to the table, including oncology heavyweight Celgene ($CELG), which reached out to gene therapy pioneer bluebird bio ($BLUE) to get to work in the space, and Santa Monica, CA's Kite Pharma ($KITE), which grossed more than $140 million in a June IPO on the strength of its approach to T cell modification. Latecomers Pfizer ($PFE) and Johnson & Johnson ($JNJ) have also shouldered into CAR-T with high-dollar deals earlier this year.

- read Penn's announcement
- here's Novartis' statement

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