FDA approves breakthrough treatment for dire cancers

FDA approves breakthrough treatment for dire cancers

FDA approves breakthrough treatment for dire cancers

In a trial of Kymriah, 83 percent of 63 patients with hard-to-treat disease were in remission within three months, according to Novartis.

The new treatment involves removing immune cells called T cells from a patient's blood and giving them a gene for a protein called a chimeric antigen receptor, or vehicle, that directs the T cells to target leukemia cells. It transforms a patient's immune system cells to fight a form of leukemia.

The FDA issued a historic decision today in approving the first gene therapy in the United States to treat certain pediatric and young adult patients with refractory B-cell precursor acute lymphoblastic leukemia (ALL) or who are in second or later relapse. Kymriah is intended for people with B-cell ALL whose cancer hasn't responded to or has returned after treatment, the FDA said.

"This is a brand new way of treating cancer", said Dr Stephan Grupp of Children's Hospital of Philadelphia, who treated the first child with CAR-T cell therapy - a girl who had been close to death, but now is cancer-free for five years and counting.

It's too soon to tell how widely applicable CAR-T will be - it's especially suited to leukemia since infusing patients with T-cells is relatively simple.

Kymriah, which is also known as CTL019, is a type of CART-T therapy that involves immune cells being extracted and genetically modified to destroy cancer before it returns to the body.

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The drug has shown promising remission and survival rates in clinical trials, the FDA said.

The one-time treatment has a boxed warning for cytokine release syndrome or CRS, a life-threatening side effect that can cause blood pressure to drop dangerously low.

However, Kymriah comes with the potential for severe side effects. According to the release, other adverse events include serious infections, hypotension, acute kidney injury, fever, and hypoxia, with most symptoms appearing within 22 days after tisagenlecleucel.

A spokeswoman for Switzerland-based Novartis declined to say how much the drug will cost, though analysts have estimated a price of $500,000 or more.

Other genetic therapies for cancer are also in the research pipeline. It is the first gene therapy of its kind to hit the US market.

Researchers filter the cells from a patient's blood, reprogramme them with a "chimeric antigen receptor" that targets cancer, and grow hundreds of millions of copies. More than half were children and teens.

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