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Possible Cures. Mystery Deaths. Daunting Costs. Can CAR-T Be Tamed?

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data this year—the first real peeks at the challenges CAR-T faces in making the leap.

Solid tumors have built more ornate defenses into their immediate environment, which makes them less penetrable to a CAR-T assault. They tend to have proteins that are also on healthy tissue, but unlike B cells that are wiped out by anti-CD19 treatments, it’s not tissue that patients can typically spare. Researchers have to choose their targets carefully. “We don’t expect first results in solid tumors as spectacular as in blood cancers,” Juno CEO Bishop said in January.

Jakobovits strikes a different tone: “If you use the right cells with the right properties we should aspire to get similar responses as in hematological cancers.”

ON THE GROUND

Administering CAR-T therapy isn’t like dispensing pills or infusing chemotherapy. Treating patients with CAR-T “requires an immense amount of planning and coordination and meetings,” says Fathi of Massachusetts General Hospital. He ticks off a long list of personnel who have to be in the loop, including cell processing experts, nurses, clinical trial coordinators, lab researchers, pharmacists, and physicians who specialize in infectious disease, pulmonary and critical care, and neurology.

While oncologists interviewed for this article believe the learning curve from one CAR-T to the next should be less steep over time, they also questioned whether the therapy will ever be straightforward enough to administer outside of major cancer centers, especially those that have experience with bone-marrow transplants.

“I don’t think it’ll be widely distributed through community for oncologists to use,” says Solomon of the Blood & Marrow Transplant Group of Georgia. “And that’s OK. The model of transplant is similar. We don’t allow just any center to do transplants.” Solomon and others would like to see the same group that sanctions transplant centers expand its purview to CAR-T therapies.

But as the Hutch’s Riddell notes, patients with solid tumors could have different responses to CAR-T than patients with blood cancers. That would make the learning curve for medical staff steeper than expected.

Meanwhile Kite says it is lining up more than 70 transplant centers, which treat nearly 90 percent of patients with the most common form of NHL, to be ready when FDA approval comes. Down the road, Kite CEO Belldegrun says he wants to move into community centers and treat patients who aren’t “the sickest of the sickest,” like those with slow-growing indolent NHL. He’s confident everyone will learn what’s necessary. A pooling of analyst estimates tabbed axicabtagene sales at $1.7 billion in 2022, according to Evaluate Pharma. (It should be noted that drug-sales projections several years ahead are far from a sure thing.)

But will insurers pay for CAR-T? Cell therapy companies will argue for the high value of putting sick people’s cancer into remission, but it’s complicated. For example, what if the CAR-T therapy extends a person’s life and makes them eligible for an expensive stem cell transplant? Kite, it should be noted, has not said how many of the 36 people whose lymphoma has disappeared in the Zuma-1 trial have subsequently undergone transplants.

What if some patients go through the cell extraction and manufacturing process only to find their modified cells aren’t having any effect? To decrease those chances, companies and academic researchers would love to find biological signals that flag when a person is more or less likely to respond to treatment—or is likely to suffer terrible side effects. But similar efforts at precision medicine in other forms of cancer immunotherapy have been slow to materialize, and aren’t likely to do so in CAR-T without much deeper data sets.

When asked if pay-for-performance schemes would make sense with CAR-T, Juno CEO Bishop says the concepts are interesting but for now face too many impediments, such as Medicare rules.

Kite CEO Belldegrun declines to discuss specifics around pricing, except to say that Kite is talking to insurers and their agents. Meanwhile, President Donald Trump is promising lower drug prices through sound bites, the pharma lobby and insurers’ agents are firing salvos at each other, and insurers are playing hardball on first-ever treatments for rare diseases. When asked about the current climate, Belldegrun seems unfazed. “It’s hard to believe that people won’t realize this is a true revolution and you can melt cancer away in a month,” he says. “If that’s not worth paying for, what’s worth paying for?”

T cell image by the National Institute of Allergy and Infectious Disease via a Creative Commons 2.0 license.

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