[Updated, 12/5/16, 4 p.m. See below.] If you’re following cancer immunotherapy, you might feel a bit swamped by this week’s wave of clinical data from experimental programs that turn living T cells into revved-up cancer fighters.
We’re updating our ongoing cancer immunotherapy trial resource with American Society of Hematology results, plus a few others we’ve dug up since we launched the resource in October. The idea is to provide a top-line look at how people have fared with the growing array of products, most of them still experimental, which have attracted so much attention but come with caveats as well.
No T cell therapy has been approved, although Kite Pharma (NASDAQ: KITE) and Novartis (NYSE: NVS) could get a green light in 2017 for CAR-T treatments of non-Hodgkin lymphoma and pediatric acute lymphoblastic leukemia (ALL), respectively. Both said this weekend they hope to have applications to the FDA in the first quarter.
We’ll continue to update our T cell chart with ASH news today and tomorrow.
—[new item] Kite dribbled out a few more tidbits on its Zuma-1 trial for NHL and said it had begun its FDA application process. The firm also lumped together early results from its Zuma-3 and Zuma-4 trials in ALL. Of 11 measurable patients, nine had a complete response (CR). Of 13 enrolled, one patient died of a severe immune reaction called cytokine release syndrome (CRS); five had serious CRS and five had serious neurological side effects, Kite reported. All the “Zuma” studies are using Kite’s main product KTE-C19, which is now called axicabtagene ciloleucel.
—Two more deaths recently halted the “Rocket” trial (NCT02535364) for adults with ALL. The treatment, JCAR015, is the most advanced from Juno Therapeutics (NASDAQ: JUNO), but the trial has been halted for a second time this year, bringing the number of deaths in the trial to five.
—Eager for good CAR-T news after the Juno deaths and Novartis’s decision this summer to dissolve its gene and cell therapy division, investors bid up Bluebird Bio (NASDAQ: BLUE) on a first look at data from nine multiple myeloma patients. Two other early CAR-T myeloma programs have not fared as well. (NCT02546167, NCT02658929, NCT02215967)
—Novartis and affiliates reported data from a trial (NCT02435849) that the healthcare giant hopes will lead to regulatory approval for its CAR-T program CTL-019 in pediatric ALL. Data from ELIANA, as the trial is known, showed 41 of 50 patients going into complete remission; 25 of the 41 stayed in remission six months after receiving the cells. Novartis says it will take its case to the FDA in early 2017.
—[new item] Novartis also has positioned a trial dubbed JULIET as a springboard to approval for CTL-019 in non-Hodgkin lymphoma. (NCT02445248) But Novartis did not present JULIET data at ASH. Instead, Novartis’s collaborators at Penn presented separate data from the same patient population. Through late July, seven of 13 people had a response to the treatment after three months. Five of them were in complete remission. None of the patients with CRs had relapsed. A Novartis spokeswoman said JULIET was ongoing and the company would present data at an unspecified later date.
—There were updates from two trials using specialized T cells to help patients after bone marrow transplants. One, from Bellicum Therapeutics (NASDAQ: BLCM), ups to 35 the number of kids with immune diseases who remain disease free after a transplant from a half-matched donor. (NCT02065869) The second, affiliated with Juno, is from a Fred Hutchinson Cancer Research Center trial using a variant of T-cell therapy called TCR. The patients had a type of acute myeloid leukemia (AML) that put them at high risk of relapse post-transplant; 12 of 12 have remained cancer-free. (NCT01640301)
(The Hutch investigator Aude Chapuis told Xconomy that a study of the TCR therapy in AML patients who don’t need an immediate transplant is starting soon. The trial will use engineered versions of the patients’ own T cells, not donor cells.)
—Phase 1 trial data was reported by Belgian cell therapy developer Celyad. (NCT02203825)
—The Baylor College of Medicine presented data in 2015 and 2016 from three early studies of CAR-T cells against cancers of the brain, blood, and sarcoma. (NCT01109095, NCT00902044, NCT00881920) The brain and sarcoma data are some of the first for T cell therapy against solid tumors. It is unclear when the next updates are due.
Photo of William Coley, father of immunotherapy, via the Wellcome Trust.