Novartis makes history with US approval of first “living drug” cancer therapy

September 4, 2017

Swiss multinational pharmaceutical company Novartis made history as the US Food and Drug Administration (FDA) approved its tailor-made “living drug” therapy against cancer.

Dubbed “CAR-T”, the treatment is the first to redesign a patient’s own immune system so it attacks cancer. The drug is tailor-made to each patient, unlike conventional therapies such as surgery or chemotherapy.

It is made by extracting white blood cells from the patient’s blood.The cells are then genetically reprogrammed to seek out and kill cancer. The cancer-killers are then put back inside the patient and once they find their target they multiply.

Novartis is charging US$475,000 (£367,000) for the therapy, which leaves 83% of people free of a type of blood cancer.

Dr. Scott Gottlieb, from the FDA, said: “We’re entering a new frontier in medical innovation with the ability to reprogram a patient’s own cells to attack a deadly cancer.

“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 therapy, which will be marketed as Kymriah, works against acute lymphoblastic leukemia.

Most patients respond to normal therapy and Kymriah has been approved for when those treatments fail.

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.

Out of 63 patients treated with CAR-T therapy, 83% were in complete remission within three months and long-term data is still being collected.

However, the therapy is not without risks. It can cause potentially life-threatening cytokine release syndrome from the rapid proliferation of the CAR-T cells in the body. This can be controlled with drugs.

But the potential of CAR-T technology goes beyond one type of cancer.

Dr. David Maloney, medical director of cellular immunotherapy at the Fred Hutchinson Cancer Research Center, said the FDA’s decision was a “milestone”. “We believe this is just the first of what will soon be many new immunotherapy-based treatments for a variety of cancers,” he added.

CAR-T technology has shown most promise against different blood-based cancers. However, it has struggled against “solid tumors” such as lung cancer or melanoma.

Dr. Prakash Satwani, a pediatric oncologist at Columbia University Medical, said: “The results haven’t been that great when you compare it with acute lymphoblastic leukemia, but I’m sure the technology will get better in the near future.”

Boosting the immune system is already a cornerstone of modern cancer treatment.

A range of drugs that “take the brakes off” the immune system to allow it to attack cancer more freely have already been adopted around the world.

CAR-T technology, which goes a step further and redesigns the immune system, is at a much earlier stage.

Prof Peter Johnson, the chief clinician at the charity Cancer Research UK, said the FDA’s move to approve the first genetically modified cell therapy is an exciting step forward. However, there are still a lot to learn about how to use the technology safely and who might benefit from it.


Category: Pharmaceuticals

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