Revolutionary Gene-Editing Cancer Treatment Gets Green Light In The US For The First Time

Scanned electron micrograph of a human T-cell from the immune system of a healthy donor. NIAID (National Institute of Allergy and Infectious Diseases)

The US Food and Drug Administration (FDA) has just approved a pioneering cancer treatment that reprogrammes the patient's own white blood cells to hunt down cancer.

Novartis, the Swiss pharmaceutical company who helped develop the treatment, announced they are “so proud to be part of this historic moment in cancer treatment.”

However, the treatment does not come cheap. Novartis is charging $475,000 for the life-saving treatment.

The “living drug”, known as Kymriah (or tisagenlecleucel), is a one-time personalized treatment that uses the power of gene therapy. The patient’s T-cells (a type of white blood cell) are shipped off to a lab where they are genetically redesigned to include a new gene that contains a specific protein. Armed with this protein, the T-cells are directed to target and kill leukemia cells. These modified cells are then reintroduced back into the patient by an intravenous infusion.

The FDA said the approval for the first gene therapy available in the US was “history in action.” The treatment will now be available for patients 25 years of age and under with the aggressive type of blood cancer, B-cell acute lymphoblastic leukemia, who have failed to respond to other treatments.

“Kymriah is a first-of-its-kind treatment approach that fills an important unmet need for children and young adults with this serious disease,” Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research, said in a statement.  

“Not only does Kymriah provide these patients with a new treatment option where very limited options existed, but a treatment option that has shown promising remission and survival rates in clinical trials."

So far, the treatment has proved to be incredibly effective. Clinical trials of the “living drug” showed 83 percent of patients who received the treatment were free of cancer within three months.

Although this move has been widely met with optimism, the treatment’s heavy price tag has also attracted its fair share of criticism and left many people pointing towards the "completely broken" drug pricing system in the US.

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

“As a cancer patient, the potential of CAR-T is exciting and I applaud FDA’s approval. It means hope for hundreds of children and their families, and we’re all excited at the potential for cancer patients," he added. "But let’s remember, American taxpayers invested over $200 million in CAR-T’s discovery."

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