Targeting blood cancers

By the time he turned 16, Justin Condoluci had battled cancer for half his life. Diagnosed with acute lymphoblastic leukemia (ALL) in 2007, when he was only eight years old, the New Jersey resident had endured multiple rounds of chemotherapy and radiation treatments, a bone marrow transplant, seemingly endless trips to the doctor, and severe symptoms requiring several hospitalizations.

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Immunology researchers have been investigating T-cells—today commonly known as the “workhorses” of the immune system for their role in recognizing and killing harmful pathogens—since their discovery in 1967. If this manipulation could be controlled, various researchers theorized, it would open the door to medicines that could be uniquely personalized by relying on genetic material from each patient. Customizing the medicine to fit the patient, the researchers surmised, could reduce the potential for off-target side effects or treatment rejection. Initially, these findings prompted research into treating HIV, which remains a top public health issue in the United States.

Quickly, however, researchers around the world began applying these findings to oncology, as T-cells also play an important role in battling cancer. The genetic mutations that can lead to cancer occur frequently within our bodies, and the immune system relies on T-cells to sense these changes and destroy the affected cells before they multiply. Yet some cancer cells can trick the immune system into ignoring them, evading detection and growing unencumbered into what becomes a tumor. Researchers hypothesized if they could “train” T-cells to selectively target a protein found in certain blood cells, including cancer cells, through genetic engineering, they could reverse this potentially fatal shortcoming.

With a breakthrough clinical trial in 2011, theory became reality in a treatment known as chimeric antigen receptor T-cell therapy, or CAR T, for short. The process involves extracting a sample of a patient’s T-cells and genetically engineering them to grow artificial receptors that target many blood cancers, such as ALL. These CAR T-cells are then replicated and infused into the patient, where they can do what they do best: attack and destroy. When the treatment is successful, the engineered cells multiply in the body and, guided by the engineered receptors, identify and kill cancer cells.

The U.S. Food and Drug Administration (FDA) approved the first two CAR T treatments for patients with certain types of blood cancer in 2017. Many more CAR T treatments are in development, and experts agree the therapy has the potential to fundamentally change the field of oncology by allowing for more-targeted, personalized medicines.

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Since his CAR T treatment in 2015, Justin has been cancer-free for four years and counting, the longest remission in his life.

“I am living proof that innovation is worth it,” he says. “Now, people like me can be excited for a future—a hope I had lost during the worst parts of my battle with leukemia.”

https://www.nationalgeographic.com/science/2019/10/partner-content-targeting-blood-cancers/?