What is Chimeric Antigen Receptor (CAR) T Cell and Gene Therapy?

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.

FDA has Approved Three CAR T cell therapies for Blood Cancers

A huge advancement in cancer immunotherapy treatments occurred in 2017 when the FDA approved the first-ever cancer gene therapy treatment KYMRIAH® (tisagenlecleucel), a CAR T cell therapy, to treat pediatric leukemia. This treatment, pioneered by Dr. Carl H. June, MD, and his team at the University of Pennsylvania with funding support from ACGT, uses CAR T cell therapy and genetically reprograms the patient’s immune cells to fight the cancer. The patient’s T cells are removed and genetically engineered in a lab. They are grown in large numbers with one objective: to kill the cancer cell. This is known as a “living therapy” with more than an 80 percent effective rate in patients. Super T cells are created and infused back in the body where they will multiply and attack the cancer cells, making the treatment even more effective.

Later in 2017, the FDA approved YESCARTA® (axicabtagene ciloleucel), also a CAR T cell therapy, the second gene therapy approved by the FDA and the first for certain types of non-Hodgkin lymphoma (NHL).

In June 2020, FDA approved a third CAR-T therapy called TECARTUS™ for relapsed/refractory mantle cell lymphoma.

  • Learn more about KYMRIAH on the Kite Pharma Patient Website
  • Learn more about YESCARTA on the Novartis Patient Website
  • Learn more about TECARTUS on the Kite Pharma Patient Website

What are the Benefits of CAR T Cancer Therapy?

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.

Watch Video

ACGT Fellow and Scientific Advisory Council Member, Dr. Carl H. June, MD, talks about making the next generation of CAR T therapies more potent and safer for patients. These include combination therapies for solid tumors and treatments with less toxic side effects. “Cure is a big word,” he remarks. The goal with cell and gene therapies was never to shoot “layups” but rather to score “three-pointers.”

Dr. Carl H. June, MD, 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 and again in 2008 for his immunotherapy research at the University of Pennsylvania.


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