CAR (Chimeric Antigen Receptor) T cell and gene therapy refers to an innovative treatment that manipulates the patient’s own T cells (immune cells) to attack malignant cancer cells. The T cells come from the patient’s blood and are re-programmed in the lab with a special receptor known as a “chimeric antigen receptor” (CAR). After the CAR receptor is added to a patient’s T cells in the lab, they can be grown in large numbers. When the newly engineered CAR T cells are infused back into the patient, they begin to search for and destroy cancer cells with a specific protein target.
CAR T cancer gene therapy is currently being studied as a treatment for many different cancer types and has been approved for different forms of leukemia and lymphoma.
Kymriah (Tisagenlecleucel) is the first CAR T cancer gene therapy to be approved by the FDA for patients. Known as a “living drug” because it is designed individually for each patient using their own T cells, Kymriah has been approved to treat young adults and children with relapsed leukemia as well as adults with diffuse large B-cell lymphoma. The approval of Kymriah has given hope to patients for future immunotherapy treatments for other types of cancers.
Many scientists are beginning to see the benefits of immunotherapy through research. ACGT Fellow, Dr. Carl June, pioneered CAR T cancer gene therapy research and led the team responsible for its FDA approval. ACGT provided early funding to Dr. June in 2004 for his immunotherapy research at the University of Pennsylvania.
This approach to engineering immune cells to treat cancer gives hope for the future and treatment of many different types of cancers. Immunotherapy for cancer treatment is based on strengthening the patient’s own immune system to kill the cancer instead of using methods like chemotherapy or radiation, which will suppress the patient’s immune system.
CAR T cancer gene therapy is capturing the attention of the medical community as a less invasive approach to treating cancer, unlike the traditional treatments of radiation, chemotherapy or surgery. Researchers are showing enthusiasm for this new type of targeted therapy as it is showing lasting results for certain cancers, with much less adverse side effects.
Patients who were enrolled in the clinical trials for Kymriah had relapsed several times and had no other options for treatment. Most of these patients are now cancer-free and leading active lives. As CAR T cancer gene therapy continues to evolve, we expect to see this type of therapy pushing the boundaries of modern medicine and changing what doctors thought was possible.