Nicole Gularte was 26 years old and straight out of graduate school when she was diagnosed with acute lymphoblastic leukemia, also known as ALL, in 2010. This type of fast-moving blood cancer causes the bone marrow to produce too many immature white blood cells, called lymphocytes, which can spread the disease to other parts of the body. If left untreated, it can turn deadly within months.
Determined to keep fighting, Gularte decided to try a different tactic: enroll in an experimental treatment known as CAR-T therapy, which uses her own immune cells that have been reengineered to find and kill the cancer.
For CAR-T, doctors extract a patient’s T cells and send them to a lab where they’re carefully reengineered and multiplied so they can sniff out the cancer cells and mark them for destruction. Then the manipulated cells are shipped back to the patient’s treatment location, where they’re injected back into the patient’s bloodstream so they can do their work. The therapy, developed by doctors at Children’s Hospital of Philadelphia, gained fame after it saved a young girl named Emily Whitehead in 2012. With guidance and encouragement from Emily, her family and the Emily Whitehead Foundation, Gularte received her own modified cells a year ago, and she’s now cancer-free. “I have officially survived acute lymphoblastic leukemia eight times,” she wrote on her blog.
The good news is that stories like Whitehead’s and Gularte’s could soon become more common. In August, the U.S. Food Drug Administration approved the first cell therapy in the United States for the treatment of pediatric and young-adult patients with a form of ALL. “We’re entering a new frontier in medical innovation with the ability to reprogram a patient’s own cells to attack a deadly cancer,” said FDA commissioner Scott Gottlieb.
Top and above: “[If] these therapies move to a first-line treatment, then the number of patients will go up dramatically into the hundreds of thousands, if not more,” says GE Healthcare’s Phil Vanek. Images credit: CCRM.
But cell therapy is a complex process that involves more than manufacturing a pill. It requires a setup that resembles a biotech factory. “Cell therapy has the potential to cure everything from cancer to diabetes,” says Phil Vanek, general manager for cell therapy growth strategy at GE Healthcare. “But we need to make it affordable and scalable.”