Kymriah (tisagenlecleucel, formerly CTL019) is a CAR T-cell therapy developed by Novartis for pediatric and young adult patients with acute lymphoblastic leukemia (ALL).

It is the first type of gene therapy to be approved by the U.S. Food and Drug Administration (FDA). The European Commission has not yet approved it. Novartis is also investigating Kymriah as a treatment for diffuse large B-cell lymphoma (DLBCL).

How Kymriah works

ALL is a type of blood and bone marrow cancer in which the bone marrow produces too many white blood cells or lymphocytes that do not develop correctly. The seexcess abnormal lymphocytes can build up quickly and spread to other parts of the body, causing swelling and organ damage. An excess of lymphocytes in the bone marrow can also prevent the body from making other blood cells, resulting in anemia and a reduced immune response.

Kymriah aims to genetically modify the patient’s own T-cells to target and destroy cells expressing a specific protein, CD19, that is found on the abnormal lymphocytes.

T-cells are first extracted from the patient’s blood and then genetically modified to produce a chimeric antigen receptor (CAR) that causes T-cells to target CD19. These CAR T-cells are then returned to the patient’s body, where they seek out and destroy cells expressing CD19, which includes the cancerous lymphocytes.

Kymriah in clinical trials

It has been studied in multiple clinical trials which demonstrate that the drug can successfully induce remission. The FDA approved Kymriah on Aug. 30, 2017, based on the results of a key open-label Phase 2 clinical trial (NCT02435849) called ELIANA. Kymriah is approved for cases of B-cell ALL after other standard treatments have been tried and failed.

The ELIANA trial enrolled 88 patients, ages 3 to 23, with B-cell ALL from across the United States, Canada, Europe, Australia and Japan. Kymriah induced complete remission in 83 percent of patients within three months of treatment. These results were presented at the 22nd Congress of the European Hematology Association.

The long-term safety and efficacy of the CAR T-cell treatment will be assessed in a study (NCT02445222) to monitor patients who have received Kymriah, for up to 15 years.

In April 2017, the FDA also granted Kymriah breakthrough therapy designation to treat DLBCL A Phase 2 clinical trial (NCT02445248) called JULIET is now recruiting participants to test Kymriah’s safety and efficacy in adult DLBCL patients. Novartis reported interim results from the JULIET trial, demonstrating that the primary endpoint for the first 51 patients has been met, with an overall response rate of 45 percent to Kymriah three months following treatment.

Other information

Side effects of Kymriah include a severe immune reaction called cytokine release syndrome as well as low blood pressure, oxygen deprivation and neurological complications.

Note: Lymphoma News Today is strictly a news and information website about the disease. It does not provide medical advice, diagnosis or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or other qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website.