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It’s one of the most effective cancer treatments so far. And one of the most expensive cancer drugs ever.
That’s why cancer specialists are both excited about so-called CAR-T cell therapy -- and worried about what it portends for the affordability of cancer care.
Dr. Peter Bach, who studies drug pricing, says the price tag for two recently approved CAR-T cell drugs -- one is $373,000 for a single dose, the other $475,000 -- are benchmarks on the road to ever-higher cancer drug price tags.
“The one truth about cancer drugs is that prices are always higher than they were the year before,” says Bach, director of the Drug Pricing Lab at Memorial Sloan-Kettering Cancer Center in New York. “Now, every time we get a new drug that costs more, it signals that you can get away with charging even more.”
The drugs’ sticker price is only the beginning. It doesn’t include pre-treatment tests and monitoring, costly tests such as PET scans to monitor the patients’ response, and sophisticated management of toxic side effects, which sometimes require administration of an antidote that itself costs tens of thousands of dollars a dose.
The total package can easily exceed a half-million dollars, and some estimate it might add up to $750,000 or more. At this rate, if all the patients who might be helped by the new $373,000 drug got it, the national cost could be $5.6 billion -- more than the entire health budgets of many nations. And that's for only about 7,500 patients.
CAR-T cell therapy gives new urgency to a long-simmering question: Is there any ceiling on cancer drug prices?
But there’s a parallel truth about new cancer treatments that dramatically raise the chances of longer life and perhaps even cure. Once they’re approved, desperate patients and eager doctors start lining up to get them. And, understandably, no one wants to say no.
'A Huge Leap'
“This is a game-changing technology,” says Dr. Jeffrey Barnes of Massachusetts General Hospital, which began moving CAR-T cell therapy from purely research to mainstream status over the past few weeks. The therapy reengineers patients’ own blood cells so they can lead an attack on cancer cells.
“To take someone who has a chance of improvement in their disease of 5 percent and be able to offer them 75 or 80 percent…it’s a huge leap,” Barnes says.
Barnes is alluding to a landmark study on CAR-T cell therapy published last month in the New England Journal of Medicine. Among 101 patients with an advanced blood cancer called B-cell lymphoma who got CAR-T cells, four out of five saw their cancers retreat. That happens in only one in 20 patients who get older treatments.
Most striking, more than half the CAR-T cell patients saw their lymphomas disappear completely. And more than a year later, 42 percent were still apparently cancer-free.
“The idea in theory isn’t just control, it’s permanent control,” Barnes says. “If you can harness this power and these cells can persist, you might be able to take an incurable disease and make it a curable disease.”
“Cure” isn’t a word that cancer specialists use with abandon, especially in talking about patients with advanced disease that’s beyond all other treatment.
Barbara Kearney, a 71-year-old retired medical technician, reached for that hope. Last month she became the very first Mass. General patient, and one of the first in the nation, to get CAR-T cell therapy outside of a research study. She was diagnosed with B-cell lymphoma in 2006.
She had already gone through four different treatment regimens, including a bone marrow transplant. None of them worked.
When Kearney’s doctor told her about CAR-T cell therapy last fall, she was eager to try it, despite a high probability of serious side effects that can be life-threatening.
“I’m not afraid of dying at all,” she said in an interview in her Dorchester apartment in late November. “My thing is to help people become aware and let them know that, you know, there’s still a chance.”
The MGH doctors extracted cells from Kearney’s bloodstream and sent them to a California lab where technicians did some fancy genetic engineering. It caused a distinctive protein to appear on the surface of the cancerous blood cells. The marker protein acts as a beacon for immune cells to home in on and destroy the cancer cells. The protein is called a chimeric antigen receptor, or CAR. The immune cells are called T-cells -- thus CAR-T cell therapy.
The California lab, owned by a company called Gilead Sciences, grew up millions of Kearney’s custom-made CAR-T cells and shipped them back to Boston. On Dec. 11, MGH doctors dripped a clear liquid containing 160 million CAR-T cells into Kearney’s veins.
For 48 hours nothing happened. Then Kearney developed a fever -- a common side-effect that signals the stepped-up immune activity caused by the CAR-T cells. But unfortunately, Barnes says, by the time she got the infusion, her aggressive lymphoma had already invaded her lungs and liver.
The hyperactive immune activity caused Kearney's blood pressure to fluctuate wildly, damaging her kidneys. She suffered confusion and other neurologic symptoms, as two-thirds of CAR-T patients do. Ultimately she developed multiple organ failure.
“Despite heroic efforts, she passed away on Dec. 29,” Barnes says.
Kearney's story shows that not even high-priced therapy comes with any guarantees.
Forging Ahead Despite Uncertainties
The Mass. General doctors are disappointed, but not deterred. They've done five more CAR-T infusions in the past few weeks. And more last-ditch patients like Kearney are lined up to get CAR-T treatments there, at the Dana-Farber Cancer Institute, and at 15 other hospitals around the nation.
Mara Bloom, director of the Mass. General Cancer Center, says the hospital isn’t sure how CAR-T cell therapy is going to be paid for. Hospital officials are negotiating with insurers on a case-by-case basis. (Kearney’s insurer said it would cover her treatment.) Medicare, the most important cancer payer, must by law cover FDA-approved cancer drugs, but the program has yet to say how much it will pay.
Some hospitals are holding back from treating CAR-T cell patients because of reimbursement uncertainties. But Bloom says “we can’t wait,” given many patients’ deteriorating status.
This material was prepared specially for the WORLD HEALTH NEWS project by journalist John Bates.
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