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(Bloomberg) -- It’s a choice no doctor ever wants to make: which patient will get a chance to live, and which will not.

That is a decision oncologists may soon confront when the first two products in a revolutionary new way to treat cancer reach the U.S. market this year.

Unlike pills or chemotherapy, the therapy known as CAR-T can’t be stockpiled. Because it’s a bespoke treatment that involves re-engineering a patient’s cells in a specialized lab, the logistics of getting CAR-T to the thousands of Americans who will be eligible as soon as this year are uniquely daunting. Some blood cancer patients who face imminent death won’t get it in time, at least initially, forcing doctors to make difficult decisions.

“God, it’s awful,’’ said one of the founding fathers of CAR-T, Carl June from the University of Pennsylvania, tearing up at the memory of children he was unable to save. “I can’t tell you how bad.’’

CAR-T has the potential to become a blockbuster for the manufacturers of the first drugs, Swiss giant Novartis AG and Kite Pharma Inc. of California, with annual sales reaching $1 billion within five years based on analysts’ estimates. In the shorter term, the drugmakers face three major challenges to get their treatments to patients once approved by the U.S. Food and Drug Administration: a weeks-long, complex manufacturing process; potentially lethal side effects; and the limited number of medical centers that will be ready for CAR-T at launch time -- fewer than 50.

Novartis told investors last month that its treatment will target about 49,000 blood cancer patients with the most common form of non-Hodgkin lymphoma, worldwide. The first indication to get approved, in October, will be for an ultra-rare form of relapsed leukemia that develops in about 2,200 children and young adults each year.

Novartis said it has by now produced more than 250 CAR-T cocktails across various clinical studies. While that’s still a fraction of the eligible population, the drugmaker said it’s taking the necessary steps to scale up.

“We are confident we will be able to meet the needs of patients,’’ Novartis said by email.

Kite said its approach will be deliberately prudent, with a “sure and steady’’ introduction of its product. About 7,400 U.S. patients will be eligible for its initial approval in aggressive non-Hodgkin lymphoma, expected in November.

Waiting Lists

Waitlists for clinical trials are already lengthy.

Jimmy Boyd, a 67-year-old warehouse manager from Louisiana, was diagnosed with lymphoma in March 2016. After radiation and multiple rounds of chemotherapy failed, his doctors signed him up for CAR-T in three hospitals across the U.S. It took six months for the first slot to open up, a Kite trial at MD Anderson Cancer Center in Houston.

It’s easy to understand why doctors are eager to try CAR-T with Boyd’s deadly type of malignancy as fast as they can. The median survival for patients at his stage of diffuse large B-cell lymphoma is only four months after all other treatments fail. yet in early CAR-T studies from Novartis and Kite, more than half of such patients saw their tumors shrink, and in about a third of the cases, cancer disappeared after three to six months.

CAR-T treatments are living drugs, engineered by genetically altering infection-fighting T-cells to destroy cancer. The process entails drawing a patient’s blood; extracting the T-cells; inserting a gene that will enable them to identify tumors as targets; then infusing the cancer-killing compound back into the patient at a specialized medical center.

Boyd will soon return to Houston to get his infusion. If it works, he could be back to his hobby, fishing sea bass, in a month. “I feel real optimistic,” he said. “I really do.”

Infusion Centers

The complexity of production was underscored in the early results from a Novartis trial last month. Of the 141 patients enrolled, 85 received the CAR-T cocktail. Novartis was unable to produce cells for nine patients, and 16 died before they could be infused.

The company said it has since updated its Morris Plains, New Jersey, processing plant and had a success rate of 97 percent for the last 30 patients enrolled. The Basel-based drugmaker expects a turnaround of about 22 days at approval time.

Time isn’t the only hurdle. Another roadblock is the lack of qualified infusion centers. Medical teams need to learn how to identify potentially deadly side effects including cytokine release syndrome, an inflammatory response to treatment that has killed several patients, swelling in the brain, and infections. Even experienced hospitals like Fox Chase Cancer Center in Northeast Philadelphia took months to get ready.

“In a general oncology unit, it’s probably very difficult, impossible or unsafe to do these types of cell therapy,” said Henry Fung, vice chairman of hematological malignancies at the 113-year-old center, one of the nation’s first cancer hospitals. “We need to have the whole team ready before we will start it."

Novartis will start with 30 to 35 accredited sites, while Santa Monica-based Kite plans to have 10 locations at first.

Kite Chief Commercial Officer Shawn Tomasello said the uniqueness of CAR-T requires caution.

“Typically with the launch of a new product, I would be in a ‘fast and furious, immediate revenue up and to the right as fast as you can’ mode,” she said. “This is not the place to employ that strategy.”

To contact the reporters on this story: Michelle Fay Cortez in Minneapolis at mcortez@bloomberg.net, Caroline Chen in San Francisco at cchen509@bloomberg.net.

To contact the editors responsible for this story: Drew Armstrong at darmstrong17@bloomberg.net, Cecile Daurat, John Lauerman

©2017 Bloomberg L.P.

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