Tisagenlecleucel , marketed as Kymriah , is a treatment for B-cell acute lymphoblastic leukemia which uses the body’s own cells to fight cancer ( adoptive cell transfer ).
T cells from a person with cancer are removed, genetically engineered to make a specific T-cell receptor that reacts to cancer, and transferred back to the person. The T cells are engineered to target a protein called CD19 that is common on B cells . A chimeric T cell receptor (” CAR-T “) is expressed on the surface of the cell.
It was invented and initially developed at the University of Pennsylvania; Novartis completed development, obtained FDA approval, and markets treatment.  In August 2017, it became the first FDA-approved treatment that included a gene therapy step in the United States. 
It is administered in a single treatment, which will cost $ 475,000. Novartis says this is cheaper than some bone marrow transplants. Novartis says it will not charge people who do not respond. 
The treatment was developed by Carl H. June at the University of Pennsylvania and is licensed to Novartis. 
In April 2017, CTL019 received breakthrough therapy designation by the US FDA for the treatment of relapsed or refractory broad-based B-cell lymphoma . 
In July 2017, an FDA advisory committee unanimously recommended that the agency approve it to treat B cell acute lymphoblastic leukemia .   
In August 2017 the FDA grants approval for the use of tisagenlecleucel in people with acute lymphoblastic leukemia.  According to Novartis, the treatment will be given to specific medical centers where these patients have been trained to respond to this type of treatment. 
In a 22-day process, the treatment is customized for each person. T cells are purified from blood cells, which are then modified by a virus that inserts a gene into the cells’ genome . The gene encodes a chimaeric antigen receptor(CAR) that targets leukemia cells. 
It uses the 4-1BB co-stimulatory domain in its CAR to improve response. 
It has undergone a phase 2 clinical study for relapsing / remitting B cell acute lymphoblastic leukemia.  A frequent side effect is cytokine release syndrome (CRS).  
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