This Medical Breakthrough Can Cure Cancer with Your Own Cells

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In 2008, just after she’d started kindergarten, Tori Lee was diagnosed with acute lymphoblastic leukemia (ALL), an aggressive form of blood cancer. Chemotherapy cures most children of the disease, but Tori wasn’t as lucky. A playful little girl who was doted on by her three older sisters, she “was treated with chemotherapy for about two years, and then she relapsed,” says her mother, Dana Lee. “We started a new protocol, with more intensive chemotherapy and radiation. She spent hundreds of days in the hospital.” And still the cancer held on.

With Tori growing weaker, her par­ents decided to take her to the Children’s Hospital of Philadelphia (CHOP) for several weeks of chemo in preparation for a bone marrow transplant, a complex and risky procedure. Just before the Lees were scheduled to leave for the hospital, her doctors told them that they would also collect Tori’s T cells as a backup plan: If Tori turned out to be too sick to have the transplant, she might be able to participate in an ongoing trial of a promising experimental treatment called CAR T, which takes a patient’s own immune cells and genetically reprograms them to kill cancer. CAR T had been used months earlier to cure another little girl, Emily Whitehead, with the same form of leukemia. “I reached out to the Whiteheads,” says Dana. “I was petrified to put my daughter, who’d been through multiple years of chemo, through the harsh reality of a bone marrow transplant.”


Still, deciding on CAR T therapy wasn’t easy. While Emily was doing great, several of the children who had followed her in the clinical trial at CHOP had died. Tori would be only the tenth to undergo the treatment. “We finally said, ‘All right, we want to try CAR T.’ We petitioned the study board to be included in the trial. We thought it gave her a better chance of survival” than the bone marrow transplant, says Dana.

In April 2013, doctors injected Tori with her own modified T cells. Six weeks later, her cancer was in remission. Four years on, Tori, now 14, remains cancer-free.

This past August, after 50 more patients in the trial went into remission, the FDA approved the treatment that had saved Emily and Tori. The process of genetically engineering CAR T cells, patented under the brand name ­Kymriah, is now available to other children and young adults under the age of 25 with ALL that hasn’t responded to standard treatment.

“Surgery, radiation, and chemo­therapy cure a little over half of people who develop cancer. But that means almost 600,000 Americans die of the disease every year,” says Steven A. Rosenberg, MD, PhD, chief of the surgery branch at the National Cancer ­Institute (NCI) and one of the pioneers of the effort to use the immune system to fight cancer. “With the approval of CAR T, we’re taking a first step toward a completely new approach to curing cancers that have been incurable.”