US clears breakthrough gene therapy for childhood leukemia – ABC News
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Opening a fresh era in cancer care, U.S. health officials on Wednesday approved a breakthrough treatment that genetically engineers patients’ own blood cells into an army of assassins to seek and ruin childhood leukemia.
The Food and Drug Administration called the approval historic, the very first gene therapy to hit the U.S. market. Made from scrape for every patient, it’s one of a wave of “living drugs” under development to fight extra blood cancers and other tumors, too.
Novartis Pharmaceuticals set the price for its one-time infusion of so-called “CAR-T cells” at $475,000, but said there would be no charge for patients who didn’t demonstrate a response within a month.
“This is a brand fresh way of treating cancer,” said Dr. Stephan Grupp of Children’s Hospital of Philadelphia, who treated the very first child with CAR-T cell therapy — a lady who’d been near death but now is cancer-free for five years and counting. “That’s enormously titillating.”
CAR-T treatment uses gene therapy technics not to fix disease-causing genes but to turbocharge T cells, immune system soldiers that cancer too often can evade. Researchers filter those cells from a patient’s blood, reprogram them to harbor a “chimeric antigen receptor” or CAR that zeroes in on cancer, and grow hundreds of millions of copies. Returned to the patient, the revved-up cells can proceed multiplying to fight disease for months or years.
It’s a entirely different way to corset the immune system than popular immunotherapy drugs called “checkpoint inhibitors” that treat a multiplicity of cancers by helping the assets’s natural T cells better spot tumors. CAR-T cell therapy gives patients stronger T cells to do that job.
“We’re coming in a fresh frontier in medical innovation with the capability to reprogram a patient’s own cells to attack a deadly cancer,” said FDA Commissioner Scott Gottlieb.
The very first CAR-T version, developed by Novartis and the University of Pennsylvania, is approved for use by several hundred patients a year who are despairingly ill with acute lymphoblastic leukemia, or ALL. It strikes more than Three,000 children and youthfull adults in the U.S. each year and while most get through, about fifteen percent relapse despite today’s best treatments.
In a key investigate of sixty three advanced patients, eighty three percent went into remission soon after receiving the CAR-T cells. Importantly, it’s not clear how long that benefit lasts: Some patients did relapse months later. The others still are being tracked to see how they fare long-term.
Still, “a far higher percentage of patients go into remission with this therapy than anything else we’ve seen to date with relapsed leukemia,” said Dr. Ted Laetsch of the University of Texas Southwestern Medical Center, one of the explore sites. “I wouldn’t say we know for sure how many will be cured yet by this therapy. There certainly is a hope” that some will be.
Most patients suffered side effects that can be grueling, even life-threatening. An immune overreaction called “cytokine release syndrome” can trigger high fevers, plummeting blood pressure and in severe cases organ harm, side effects that require sophisticated care to help patients without blocking the cancer attack. The FDA designated a treatment for those side effects Wednesday.
“This is remarkable technology,” said Dr. Mikkael Sekeres of the American Society of Hematology. But, he cautioned that CAR-T “isn’t a panacea.”
Among concerns, sometimes leukemia can develop resistance, and sometimes patients worsen while waiting for their fresh cells, said Sekeres, who directs the Cleveland Clinic’s leukemia program and wasn’t involved with CAR-T testing.
“Unluckily leukemia grows so rapidly that it can evade even the smartest of our technologies,” he added.
To better ensure patient safety, the FDA is requiring Novartis to suggest CAR-T therapy only through medical centers specially trained and certified to treat the complicated treatment. Novartis expects to have thirty two centers around the country, mostly in large cities, running by year’s end, with the very first twenty suggesting care within the next month.
Patients’ collected immune cells will be frozen and shipped to a Novartis factory in Fresh Jersey that creates each dose, a process the company says should take about three weeks. The $475,000 price tag doesn’t include the cost of needed hospitalizations, travel to a certified hospital and other expenses.
On a conference call Wednesday, Novartis executives said the company is working with the Medicaid program and private insurers and expects broad coverage, and will suggest some financial assistance with such things as copay and travel costs. But they didn’t promise all patients would be able to get the therapy.
For some patients, the fresh CAR-T therapy might substitute bone marrow transplants that cost more than half a million dollars, noted Grupp, who led the Novartis probe.
“I don’t want to be an apologist for high drug prices in the U.S.,” Grupp stressed. But if it’s the last treatment they need, “that’s a truly significant one-time investment in their wellness, especially in kids who have a entire lifetime ahead of them.”
“This is a turning point in the fight” against ALL, said Penn’s Dr. Carl June, who pioneered the therapy.
But he and other researchers say thousands more patients eventually may benefit. Kite Pharma’s similar CAR-T brand, developed by the National Cancer Institute, is expected to win approval later this year to treat aggressive lymphoma, and Juno Therapeutics and other companies are studying their own versions against blood cancers including numerous myeloma.
Scientists around the country also are attempting to make CAR-T therapies that could fight more common solid tumors such as brain, breast or pancreatic cancers — a firmer next step.
“Albeit narrow in scope, today’s FDA ruling is a milestone,” said Dr. David Maloney of the Fred Hutchinson Cancer Research Center in Seattle, whose team has worked with Juno and is researching CAR-T in a multiplicity of cancers. “Approvals are an significant step, but they’re just the beginning.”
AP Medical Writer Linda A. Johnson in Trenton, Fresh Jersey, contributed to this report.
This Associated Press series was produced in partnership with the Howard Hughes Medical Institute’s Department of Science Education. The AP is solely responsible for all content.