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Tisagenlecleucel (Kymriah, cell suspension for infusion) was approved as the first gene therapy in the United States in 2017 and in the EU and many countries in 2018. The drug was originally developed by Novartis at the University of Pennsylvania. The high price of the therapy is controversial.
Mechanism of action
Tisagenlecleucel is a CAR T-cell therapy. CAR stands for (chimeric antigen receptor). It is an individualized treatment in which cytotoxic T cells are obtained from each patient’s blood. These cells are genetically engineered to target and destroy the CD19 antigen on B cells. CARs are responsible for the binding. The T cells are multiplied and administered to the patient as an infusion. CARs are fusion proteins that contain an antigen-binding domain derived from a monoclonal antibody (anti-CD19 domain). It is a cell-based cancer immunotherapy. Lentiviruses are used for gene transfer.
Indications
- Pediatric, young adult: B-cell acute lymphoblastic leukemia (B-cell ALL).
- Adults: diffuse large B-cell lymphoma (DLBCL).
Dosage
According to the professional information. The drug is administered as an intravenous infusion.
Contraindications
- Hypersensitivity
- The contraindications of chemotherapy for lymphocyte depletion should be noted
Full precautions can be found in the drug label.
Adverse effects
The most common possible adverse effects include:
- Cytokine release syndrome
- Infectious Diseases
- Hypogammaglobulinemia
- Fever
- Diarrhea
- Lack of appetite
- Hypotension
- Fatigue
- Blood count disorders
