Tragedy, Determination and Chance – The Story of CAR-T Therapy

In 2010, Emily Whitehead was diagnosed with acute lymphoblastic leukemia at age 5. We would like to share her story. Emily, her parents’ most precious treasure, faced an unusual situation.

After 2 chemotherapy sessions, she developed a severe deep soft tissue infection (necrotizing fasciitis) in both legs and nearly lost them. Despite this difficult treatment, 16 months later her disease recurred. This time, a stem cell transplant was said to be necessary, but before this even more difficult treatment, her family sought a second opinion and approached the Children’s Hospital of Pennsylvania. They had heard that the University of Pennsylvania was developing a new treatment called CART-19, genetically modifying immune T cells to attack cancer cells. Unfortunately, for this new treatment, clinical trials had not yet been opened by the FDA . Emily had to return to her former hospital. Her leukemia cells doubled every day. She received a more intensive chemotherapy regimen lasting 3 weeks, but showed no improvement.

Later, the clinical trial opened and Emily became the first child to receive CART-19 treatment. Ultimately, she achieved a complete response to CAR-T treatment and is now 12; she is both growing up and helping advance a series of studies that enabled her survival.

In another study involving 63 children diagnosed with leukemia, cancer cells were completely eliminated in %83 of patients within 3 months.

Following successful results, in August 2017, the FDA approved Novartis’s CAR-T treatment tisagenlecleucel for relapsed or treatment-resistant (refractory) acute lymphoblastic leukemia.

What Is CAR-T Cell Therapy and How Is It Performed?

CAR-T cell therapy, an abbreviation of “Chimeric Antigen Receptors,” is a fully personalized form of treatment. The patient’s T cells are genetically modified to attack cancer cells. T cells are first separated from a blood sample taken from the patient. In the laboratory, genes enabling them to recognize cancer cells are transferred into the T cells. The genetically modified T cells are then returned to the patient. Producing this patient-specific cancer immunotherapy drug takes approximately 22 days.

Because living cells are introduced into the body, this treatment is also called a living drug .

Although CAR-T therapies first succeeded in blood and bone marrow (hematological) cancers, they are also thought potentially effective for solid (organ) tumors.

In oncology, the most important development of 2018 was selected by the American Society of Clinical Oncology (ASCO) as CAR-T cellular immunotherapy, a living cancer drug. CAR-T cell therapy is virtually the pinnacle of immune system treatments in oncology...

The video below shows how CAR-T treatment is prepared and administered.

Related Topic

Details of the studies leading to CAR-T therapy’s FDA approval and its side effects