Using a revolutionary new treatment option, Stanford Health Care is now treating certain blood cancers in patients who have failed to improve with chemotherapy, radiation therapy, or stem cell transplant.

Called CAR T-cell therapy, the treatment involves removing immune cells from the patient’s blood, engineering them in a laboratory to improve their cancer-fighting power, then reinfusing them into the patient. The technique is giving hope to people who had been told they were running out of options.

“It’s exceeding anything I could ever have imagined two years ago,” said David Miklos, MD, PhD, associate professor of medicine at the Stanford University School of Medicine and CAR T-cell therapy expert at Stanford Health Care. “We hijack the patient’s T cells, making a chimeric molecule.”

Just last month, a new CAR T-cell therapy was approved by the FDA and is now available to select patients with a certain type of hematologic cancer. Stanford Health Care is the only Northern California health system to offer the treatment.

CAR T-cell therapy, a form of immunotherapy, is a relatively new weapon against cancer. Physicians have known that our immune systems protect us against cancer, but until recently didn’t know how to employ that knowledge. With CAR T-cell therapy, clinicians remove T cells, a kind of white blood cell, from the patient. In a laboratory, they then manipulate the cells with a virus that causes them to produce chimeric antigen receptors, or CARs, which in turn attack the tumor cells.

“We supercharge the immune system,” Miklos said, adding that the engineered cells stay in the body, fighting cancer “potentially forever.”

Meanwhile, the patient undergoes chemotherapy to reduce the white blood cell count, which encourages the T cells, once reinfused, to multiply. Nine days after the T cells are removed, the patient receives the infusion. Within a week, the engineered cells increase from 2 million to 20 billion and begin attacking the cancer.

During the infusion and for several days afterward, patients stay in the hospital so they can be monitored for side effects. Most patients experience what feels like a severe flu — a sign that their immune systems are revving up. Some also develop neurotoxicity, which can affect mental functioning. To treat these conditions, physicians slow down the expansion of the CAR T cells.

Physicians are currently using CAR T-cell therapy to treat diffuse large B-cell lymphoma and acute lymphoblastic leukemia. Miklos said they hope to treat other forms of cancer, eventually even solid tumors such as those found in breast and lung cancer, in the near future.

Only patients who have failed to improve after traditional cancer treatments are receiving the therapy for now, but Miklos said the goal is to find treatments that can replace chemotherapy. “Imagine that this is the beginning of no longer depending on chemo for treating patients,” he said. “It’s a game changer.”

For more information about CAR T-cell therapy, please also visit:

• CAR-T Immunotherapy Q&A
• New cell therapy for relapsed leukemia patients