FDA panel recommends approval of gene altering therapy to fight cancer

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Recently a Food and Drug Administration panel unanimously recommended the agency approve a cutting-edge cancer therapy. 

According to the New York Times, Novartis presented its data to the panel, and is one of several drug companies looking to create a “living drug” through a treatment called CAR T-cell therapy -- the first-ever treatment that genetically alters a patient’s own cells to fight cancer. Next month, Kite Pharma is expected to get an answer from the FDA as well. 

One expert called it the “most exciting thing I’ve seen in my lifetime.” 

So what is it? 

CAR T-cell treatment is a process where millions of a patient’s T-cells are removed and genetically engineered. Think of T-cells as the soldiers of the immune system -- fighting off foreign invaders dangerous to the body. However, they can’t win the fight against cancer. 

Scientists take these “soldiers” out through the blood and give them an upgrade. By isolating the patient’s T-cells, researchers tweak the outside of each cell by adding a receptor called CAR (chimeric antigen receptor). The modified T-cells are then infused back into the body -- No longer your average immune soldier, but rather a group of immune Special Forces designed to find and kill cancer cells.

These new T-cells capable of killing thousands of cancer cells, and a single turbocharged cell can destroy up to 100,000 cells. 

According to Bloomberg.com, “The Novartis medicine awaits clearance for a severe form of cancer in children and young adults known as acute lymphoblastic leukemia, while Kite’s first target is diffuse large B-cell lymphoma, which afflicts a larger group of people.” Since leukemia is a cancer of the blood, doctors have been focusing on that cancer, as well as other types of leukemias, before tackling cancers in other parts of the body.” 

However, this medical breakthrough doesn’t come without risks. Although no one died as a result of the therapy in the Novartis trials -- several people have died in other trials from cerebral edema, a condition in which excessive fluid causes the brain to swell. 

Even successful cases have had their drawbacks. In 2012, then 6-year-old Emily Whitehead was the first child ever to undergo the treatment, and suffered from severe side effects such as horrible fevers, crashing blood pressure, and lung congestion. She nearly died, but emerged cancer free and was at the FDA/ Novartis panel as an advocate for its approval. 

Like all medical visits, there is the financial cost to consider as well. Analysts estimate that the very personalized, one-time treatment could cost more than $300,000. However that sticker shock might fade when considering how the costs of years of current treatments and hospital stays can add up. 

There is still a long way to go in the fight against cancers, but if the FDA officially approves CAR T-cell treatments as it is expected to -- It could be a game changer.

 

Watch the video to see how a world without cancer could be just around the corner. 

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