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. [1] In August 2017, it became the first FDA-approved treatment that included a gene therapy step in the United States. [2]

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. [3]


The treatment was developed by Carl H. June at the University of Pennsylvania and is licensed to Novartis. [4]

In April 2017, CTL019 received breakthrough therapy designation by the US FDA for the treatment of relapsed or refractory broad-based B-cell lymphoma . [5]

In July 2017, an FDA advisory committee unanimously recommended that the agency approve it to treat B cell acute lymphoblastic leukemia . [6] [7] [8]

In August 2017 the FDA grants approval for the use of tisagenlecleucel in people with acute lymphoblastic leukemia. [9] 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. [10]


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. [6]

It uses the 4-1BB co-stimulatory domain in its CAR to improve response. [11]

Clinical trials

It has undergone a phase 2 clinical study for relapsing / remitting B cell acute lymphoblastic leukemia. [12] A frequent side effect is cytokine release syndrome (CRS). [7] [1]


  1. ^ Jump up to:b “BLA 125646 Tisagenlecleucel – Novartis FDA Briefing Document to ODAC” (PDF) .
  2. Jump up^ “FDA provides first gene therapy to the United States” . FDA – US Food & Drug Administration . US Department of Health and Human Services . Retrieved 31 August 2017 .
  3. Jump up^ FDA Approves First Gene-Altering Leukemia Treatment, Costing $ 475,000, By DENISE GRADY, New York Times, AUG. 30, 2017
  4. Jump up^ FDA Panel Recommends Approval for Gene-Altering Leukemia Treatmentby DENISE GRADY, New York Times, JULY 12, 2017
  5. Jump up^ “Novartis gets second CAR-T candidate FDA ‘breakthrough’ tag” . www.fiercebiotech.com . Fierce Biotech.
  6. ^ Jump up to:b Ledford, Heidi (12 July 2017). “Engineered cell therapy for cancer gets thumbs up from FDA advisers” . Nature . doi : 10.1038 / nature.2017.22304 .
  7. ^ Jump up to:b “FDA Panel Backs Novartis’ Pioneering New Cancer Gene Therapy” . New York Times. July 12, 2017 – via NYTimes.com.
  8. Jump up^ Stein, Rob (2017-07-12). ” ‘ Living Drug’ That Fights Cancer By Harnessing The Immune Clears Key Hurdle System” . NPR . Retrieved 2017-07-13 .
  9. Jump up^ “FDA provides first gene therapy to the United States” . FDA – Food & Drug Administration . Retrieved 6 September 2017 .
  10. Jump up^ Grady, Denise. “FDA Approves First Gene-Altering Leukemia Treatment, Costing $ 475,000” . The New York Times . Retrieved 6 September 2017 .
  11. Jump up^ “FDA Panel Unanimously Recommends Approval for Novartis’ CAR T-Cell Therapy CTL019” . GEN . GEN Genetic Engineering & Biotechnology News.
  12. Jump up^ “Determine Efficacy and Safety of CTL019 in Pediatric Patients With Relapsed and Refractory B-cell ALL” . clinicaltrials.gov .

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