chimeric antigen receptor T-cell therapy, or “CAR-T” therapy for short, has stirred excitement across the scientific community because of its potential not to just treat a broad range of cancers, but reduce them to undetectable levels. With a treatment licensed exclusively to Juno, Sloan-Kettering, for instance, reported some impressive results from a small trial earlier this year: of 16 acute lymphoblastic leukemia (ALL) patients treated with a CAR-T therapy, 88 percent of them were effectively cured.
It’ll be a long time, of course, before oncologists and patients know whether the procedures prove safe and effective. Sloan-Kettering, for instance, had to suspend some CAR-T clinical trials for a short time because two patients died during treatment. Though the trials have been restarted, the incidence of cytokine release syndrome—a toxic immune response that can occur when a cancer treatment acts rapidly on its target—will be closely watched.
“We’re working on a number of approaches that we think have the potential to ameliorate it, or maybe even eliminate it,” Bishop says, though he declined to be more specific.
The temporary trial stoppage highlighted just how new the field is, and how much has to be accomplished before CAR-T therapy is a real option for cancer patients.
Yet despite the many hurdles to come, the tantalizing potential of these CAR-T treatments has led to some big investments across the sector. Some of the other entrants: Carl June’s group at the University of Pennsylvania, which is collaborating with Novartis; Summit, NJ-based Celgene (NASDAQ: [[ticker:CELG]]), via a deal with Cambridge, MA-based gene therapy company Bluebird Bio (NASDAQ: [[ticker:BLUE]]); and Santa Monica, CA-based Kite Pharma (NASDAQ: [[ticker:KITE]]), which recently banked $128 million in one of the biggest biotech IPOs of the year. Pfizer also recently cut a deal with Paris-based Cellectis to tap into its CAR-T work.
The field has also spawned a big legal feud between Juno and Carl June’s group. UPenn and St. Jude Children’s Research Hospital had been battling over IP in the CAR-T field for a few years already; when St. Jude’s won a CAR-T patent in 2013, Juno licensed it and promised to cover most of the costs of its legal fight with Penn. Novartis has likewise stepped in on Penn’s side. That case is ongoing. Documents show a trial is set to begin in late February 2015. Bishop declined to comment on the status of the case, only saying “that process continues.”
According to Bishop, Juno is currently enrolling patients in three different clinical trials for different B-cell malignancies, including adult and pediatric ALL, and Non-Hodgkins Lymphoma. The three drug candidates Juno is testing in these studies are known as JCAR014, JCAR015, and JCAR017.
Juno is also developing therapies that use a second approach, so-called “high-affinity T-cell receptors,” or TCR, which aim for specific proteins inside tumor cells, rather than on their surface. But its CAR-T efforts have moved more quickly. The three drug candidates Juno is testing in trials so far are all CAR-T therapies, not TCR. Bishop declined to comment on the status of its TCR program except to say “a detailed update” will come by the end of the year.
The cash will help with those development efforts and presumably add to Juno’s fast-growing operation as well. Bishop declined to give a specific headcount, but he says new people are joining the company “pretty much every week.” That’s positive news for Seattle’s biotech scene, which took a big hit last week with the layoffs Amgen announced.
“It’s a very good thing for the region,” Nelsen says.
