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FDA approves a 'historic' new type of leukemia treatment

The FDA approved a "historic" new cancer treatment for kids with leukemia, but it carries a big price tag.
/ Source: NBC News

The Food and Drug Administration has approved a new kind of cancer treatment in what it called a “historic action”.

The new treatment is the first U.S. approval of a treatment for genetic modification, and its maker promises some unprecedented pricing options— including offering it for free if it doesn’t work quickly.

But for those it does help, the new treatment, called Kymriah, comes with a giant price tag: $475,000 per treatment.

Novartis, its maker, thinks it will cure most patients with a single treatment. In a very small trial, 83 percent of children who got the treatment went into remission within three months.

It’s an approach called CAR-T, short for chimeric antigen receptor T-cell therapy. It’s approved for a very specific type of cancer in a specific group of patients — B-cell acute lymphoblastic leukemia (ALL) in patients under the age of 25 who have not been helped by other treatments or who have relapsed. It can also be effective with other types of cancer.

The new leukemia drug reprograms a patient’s own cells to attack the deadly cancer, possibly in a single treatment.
The new leukemia drug reprograms a patient’s own cells to attack the deadly cancer, possibly in a single treatment.

“We’re entering a new frontier in medical innovation with the ability to reprogram a patient’s own cells to attack a deadly cancer,” said FDA Commissioner Dr. Scott Gottlieb. “New technologies such as gene and cell therapies hold out the potential to transform medicine and create an inflection point in our ability to treat and even cure many intractable illnesses.”

The treatment is complicated and can be dangerous. Immune cells called T-cells are taken from each patient, genetically altered to target the leukemia cells, and then reinfused back into the patient.

“We’ve never seen anything like this before and I believe this therapy may become the new standard of care for this patient population,” said Dr. Stephan Grupp, director of the Cancer Immunotherapy Frontier Program at Children’s Hospital of Philadelphia (CHOP).

“ALL is a cancer of the bone marrow and blood, in which the body makes abnormal lymphocytes. The disease progresses quickly and is the most common childhood cancer in the U.S.,” the FDA said.

“The FDA is requiring that hospitals and their associated clinics that dispense Kymriah be specially certified,” it added. And it will require Novartis to follow up carefully to take note of bad reactions to the treatment.

The National Cancer Institute estimates that 3,100 people 20 and younger are diagnosed with ALL each year.

Novartis said it made a deal with the federal government's Center for Medicare and Medicaid Services that it would not charge for patients who did not respond within the first month.

Taxpayers helped

Patient advocacy groups complained.

“While Novartis’ decision to set a price at $475,000 per treatment may be seen by some as restraint, we believe it is excessive. Novartis should not get credit for bringing a $475,000 drug to market and claiming they could have charged people a lot more,” said David Mitchell, Founder and President of Patients For Affordable Drugs.

American taxpayers invested over $200 million in CAR-T’s discovery.
American taxpayers invested over $200 million in CAR-T’s discovery.

“But let’s remember, American taxpayers invested over $200 million in CAR-T’s discovery. To date, Novartis has not acknowledged the significance of taxpayers’ investment, and the company declined to detail its own investment."

Some experts noted that a bone marrow stem cell transplant, a common leukemia treatment, can cost at least that much and this approach may eventually be a replacement.