Gene therapy for cancer as FDA approves Novartis’ CAR-T gene therapy costing $475,000 for ALL

Prof Dr,DRAM,HIV /AIDS,HEPATITIS ,SEX DISEASES & WEAKNESS expert,New Delhi,India, +917838059592

First time in USA ,A Gene therapy to treat cancer by using patients own blood cell has been approved. Novartis AG’s CAR-T cell therapy was approved by the Food and Drug Administration on Wednesday, making it the first gene therapy to be available in the U.S.Novartis’ NVS, +1.38%  Kymriah was approved for young people up to age 25 with a form of acute lymphoblastic leukemia (ALL).The list price is $475,000 for one-time treatment, Novartis said.

        There is a lot of criticism regarding such high price of this drug but as Cancer drugs are evolved after much research spending hugh money so The company demanded that if such price is not provided in futue no company will invest in such cancer research.Therefore,The company planned that to price the therapy, Kymriah, “based on the clinical outcomes achieved,” and specifically only allow for payment when ALL patients “respond to Kymriah by the end of the first month.” The company plans to work with the Centers for Medicare and Medicaid Services to implement the pricing strategy.

    CAR-T, or chimeric antigen receptor T-cell therapy, uses a patient’s immune T-cells and re-engineers them to better fight cancer.As such, each dose of Kymriah is customized to the individual patient’s T-cells through genetic modification.Kymriah treatment also has the possibility of serious side effects, including cytokine release syndrome (CRS), which consists of high fever and flu-like symptoms and can be life-threatening, and neurological events, which are also life-threatening.

              It’s called CAR-T, for chimeric antigen receptor T-cell, the scientific name of the genetically altered white cells.patients’ own white blood cells are removed, genetically engineered, and reinserted, a process that, for the Novartis product, takes 22 days. 

                  Kymriah is a special medicine. It created a national stir when, in an early trial at the University of Pennsylvania, it saved a young girl named Emily Whitehead who was near death. In a clinical trial, 83% of children with acute lymphoblastic leukemia who got the medicine went into remission. Based on earlier studies, one would expect that about 60% would remain in remission after a year, says Stephen Group of the University Pennsylvania, who led Novartis’ study.

  Gilead, the top maker of drugs for HIV and hepatitis C, would purchase Kite Pharma, a biotech firm that is Novartis’ biggest rival, for $11 billion. Kite is developing a similar treatment, but for adults with another blood cancer called diffuse large b-cell lymphoma (DLBCL).