Historic study involving Simmons Cancer Center shows effectiveness of CAR-T therapy

A historic study involving researchers from UT Southwestern's Harold C. Simmons Comprehensive Cancer Center demonstrates the effectiveness of CAR-T therapy, which uses genetically modified immune cells to treat acute lymphoblastic leukemia (ALL) in children and young adults. The research appears in the New England Journal of Medicine.

"While most children with ALL respond well to chemotherapy, the patients in this trial were patients whose cancer had returned, and they desperately needed an alternative. It was gratifying to be part of this pioneering effort using a genetically modified version of the patient's T-cells to attack their cancer cells, and to see such positive results for so many patients," said Dr. Ted Laetsch, Assistant Professor of Pediatrics with the Simmons Cancer Center at UT Southwestern, which is recognizing its 75th anniversary this year. Dr. Laetsch served as the lead investigator for the only clinical trial site in the Southwest for the CAR-T trial.

The global trial treated a total of 75 young patients who had acute lymphoblastic leukemia (ALL) that was resistant to treatment. Of the 75 patients who were treated, 81 percent went into remission following treatment, a high number for any treatment. Based on these promising early results, the FDA gave approval to CAR-T therapy for ALL patients 25 years old and younger in late August.

Dr. Laetsch is now providing CAR-T treatment for young ALL patients whose leukemia did not respond to therapy or whose cancer relapsed more than once at the Pauline Allen Gill Center for Cancer and Blood Disorders at Children's Health. UT Southwestern physicians will shortly be treating other types of blood and bone marrow cancers with CAR-T therapy, short for Chimeric Antigen Receptor T-cell therapy.

"It is very gratifying to see children who previously did not have any good options, respond so well to this new therapy and continue to do well," Dr. Laetsch said. "This is a new frontier in cancer treatment."

Dr. Larry Anderson, Associate Professor of Internal Medicine, is currently enrolling adult patients in a clinical trial of a CAR-T therapy for multiple myeloma. In early 2018, UT Southwestern will join a select group of medical centers providing CAR-T therapy for patients with large B-cell lymphoma.

CAR-T is an innovative immunotherapy that uses a re-engineered version of the patient's immune cells as a living drug. The process involves taking the patient's T-cells, a type of white blood cell in the body's immune system, and sending them to a commercial laboratory. There, the cells are primed to specifically recognize cancer cells, much like T-cells in the body are primed to recognize a virus during an infection. The activated T-cells are then returned to the patient's blood, where they attack the cancer cells. Each dose of this CAR-T living drug is unique and specific to the patient.

Acute lymphoblastic leukemia is a cancer of the blood in which the bone marrow makes too many white blood cells. With approximately 3,500 new cases a year in children, ALL is the most common childhood cancer, according to the National Cancer Institute. Childhood ALL typically responds well to traditional chemotherapy, but in cases where the cancer does not respond to initial treatment, or in which the cancer returns, subsequent rounds of chemotherapy are effective less than half the time, Dr. Laetsch said.

Adult multiple myeloma patients interested in participating in the CAR-T multiple myeloma trial at UT Southwestern can call 214-645-HOPE (214-645-4673) and ask for Julie Zuckerman.