Kymriah: ‘Living Drug’ Approved to Treat Childhood Leukaemia

Signalling a new era in cancer care, this week the US Food and Drug Administration approved the first treatment that genetically engineers patients’ own blood cells to fight childhood leukaemia. Kymriah is the first gene therapy to be approved for use in humans in the United States – an historic step forward.

The drug, Kymriah, is targeted against acute lymphoblastic leukaemia, an aggressive form of the disease that prevents bone marrow cells from maturing properly. Without treatment, most people with the disease would live only a few months, and whilst there are currently a number of treatments in use, Kymriah has been approved for when existing treatments fail.

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The ‘living drug’ is tailor-made to each patient, unlike conventional therapies such as surgery or chemotherapy. It’s called CAR-T, and is made by extracting white blood cells from the patient’s own blood. These cells are then genetically reprogrammed to seek out cancer cells and destroy them – once returned to the patient these cells can continue to multiple and fight disease for months.

Dr Stephan Grupp, who treated the first child with CAR-T at the Children’s Hospital of Philadelphia, said the new approach was “enormously exciting”. “We’ve never seen anything like this before,” he added.

That first patient had been near to death, but has now been cancer-free for more than five years.

In a key study that contributed to the data required for the living drug to gain approval, 63 patients were treated with Kymriah. Out of the 63 patients, 83% were in complete remission within 3 months. Some patients did relapse months later, and others are still being tracked to see how they react 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 study 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.

Kymriah was developed by Novartis Pharmaceuticals and the University of Pennsylvania. In a press release earlier this week Novartis said it would charge $475,000 for the treatment, which is made from scratch for every patient. Though they did say that there would be no charge if the patient didn’t show a response within a month. Put into perspective, for some patients, the new CAR-T therapy may replace bone marrow transplants that cost more than half a million dollars, and require a lifetime of aftercare.

Novartis plans to file for FDA approval of Kymriah for a type of non-Hodgkin lymphoma that affects adults later this year.