Tisagenlecleucel
KymriahRCSB PDB 7URV · 3D model
Drag to rotate. Scroll or pinch to zoom.
Your own T-cells are removed, genetically reprogrammed to recognize and attack cells carrying a marker called CD19, then infused back in.
How it works
Doctors first draw blood from the patient and separate out the T-cells, the immune system's hunter cells. Those T-cells are sent to a lab, where a modified virus inserts new genetic instructions that teach them to spot a protein called CD19 on certain cancerous B-cells. The re-engineered cells are grown into large numbers and frozen for shipping. After a short course of chemotherapy to make room for the new cells, the CAR-T cells are infused back into the bloodstream, where they multiply and destroy CD19-marked cells.
What it’s used for
Used for children and young adults (up to age 25) with a form of leukemia that hasn't responded to treatment or has come back at least twice, and for adults with certain lymphomas that have relapsed after at least two prior rounds of treatment.
- Mechanism
- Autologous anti-CD19 CAR-T cell therapy
- Drug class
- CAR-T cell therapy
- Therapeutic area
- Oncology (B-cell malignancies)
The first approved cell therapy engineered from a patient's own immune system
Kymriah wasn't just a new drug — it was a new kind of medicine entirely. Instead of giving a patient a manufactured compound, doctors take a patient's own immune cells out of their body, genetically reprogram them in a lab to recognize and attack their specific leukemia, and infuse them back in. It was the first therapy like this the FDA had ever approved for anyone, for any disease — the first "living drug." It opened the door for the wave of cell and gene therapies that followed, including Casgevy, also on this site, the first CRISPR-based treatment ever approved.
Pivotal trial
A single-arm, multicenter, international phase 2 trial in children and young adults with relapsed or refractory B-cell ALL, supporting approval with an overall remission rate above 80% within three months of infusion.