Novartis/Penn's customized T cell wows ASH with stellar leukemia data

Back in the summer of 2011, University of Pennsylvania investigator Carl June grabbed headlines around the world with the news that a personalized immune cell therapy had wiped out all signs of leukemia in two of three patients in a tiny study he had mounted to test its potential. A year later, the pharma giant Novartis ($NVS) had stepped in to license the technology, pumping $20 million into the pact to push it along. Late last year the company snagged Dendreon's ($DNDN) shuttered manufacturing site in New Jersey to make the therapy. And over the weekend, June was back with another impressive round of results for a much larger group of patients in what is fast becoming a race to the finish line with one of the most promising new leukemia therapies in the clinic.

A total of 19 of 22 pediatric patients with lethal cases of acute lymphoblastic leukemia experienced complete remissions after being treated with the CAR T therapy, CTL019, a T cell engineering to target cancer cells that express the CD19 protein. Five of those patients later relapsed, though one had developed tumors that did not express CD19. And the first patient treated in that study is still in remission after 20 months.

Among 32 adult patients with chronic lymphocytic leukemia, 7 experienced a complete remission and 15 responded to the therapy. The data were discussed at the annual meeting of the American Society of Hematology in New Orleans.

Dendreon's Provenge helped highlight the potential of personalized cancer therapies, as well as the difficulty of marketing them in the face of easier-to-use rivals. But Novartis believes it has a breakthrough in its hands that can radically alter the field. To make this treatment, physicians extract T cells from patients, which are then genetically modified to hunt down and destroy its cancer target. But it also divides once it encounters its target, multiplying into a host that can overwhelm the cancer.

"These CTL019 data at ASH reinforce that CAR therapy has the potential to change the treatment paradigm for those suffering from various types of leukemia," June said in a statement. Novartis added that there is a lot of potential for this therapy, as CD19 is associated with a number of B-cell malignancies including ALL, CLL, diffuse large B-cell lymphoma, follicular lymphoma and mantle cell lymphoma.

June told Bloomberg over the weekend that the plan now is to set up multicenter studies for this treatment that can begin next year.  

Two of the three original leukemia patients June tested this on are still alive.

As EP Vantage reports this morning, Novartis isn't alone in the CAR T field. The NCI has been working with Kite Pharma on one such program while other investigators are also active in the CD19 field.

- here's the release
- here's the Bloomberg report 
- here's the EP Vantage report
- more coverage on ASH from FiercePharma