Meanwhile, this week Roche secured FDA approval to use its arthritis drug Actemra (tocilizumab) as a treatment for CAR-T-induced CRS.
USA regulators approved a revolutionary new cancer treatment from Novartis AG on Wednesday, the first in a group of coming therapies that offer breakthrough cures for dire cases as well as stratospheric costs.
The new treatment is the first US approval of a treatment for genetic modification, and its maker promises some unprecedented pricing options- including offering it for free if it doesn't work quickly.
About 3,100 people under the age of 20 are diagnosed with ALL each year in the United States.
Kymriah will carry a boxed warning because of the treatment's potential to cause deadly side effects, including neurological complications and what's known as cytokine release syndrome, a systemic reaction triggered by the destruction of the cancer cells.
The patient's T-cells are collected and sent to a manufacturing centre where they are genetically modified to include a new gene that contains a specific protein (a chimeric antigen receptor or CAR) that directs the T-cells to target and kill leukaemia cells that have a specific antigen (CD19) on the surface. When the cells are modified they are put back into the patient's body to get rid of cancer cells.
"[The] approval is transformational for pediatric cancer patients and cancer research overall".
The procedure is personalized for the individual.
Novartis points out that some groups think the company could actually have charged more for Kymriah.
The company plans to make Kymriah available to about 30 hospitals by the end of the year.
While Kymriah's price tag will grab headlines, it pales in comparison with some other gene therapy treatments.
It also comes with some serious potential risks.
Kymriah does have side effects, causing cytokine release syndrome, a systemic response to the proliferation of auto T-cells that can cause flu-like symptoms such as a high fever, and neurological events.
Kymriah, marketed by Novartis, was granted several special designations, including "fast track", "priority review", and "breakthrough therapy" consideration. If you know anyone who has cancer, that's exciting news. Even so, the approval has industry experts and cancer doctors excited.
For years, the foundations of cancer treatment were surgery, chemotherapy, and radiation therapy. The researchers have done outstanding work.
Those arguments didn't assuage David Mitchell, founder of Patients for Affordable Drugs, and Jamie Love, director nonprofit Knowledge Ecology International, who believe the drug should be more affordable given that taxpayers funded early research.
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