The U.S. Food and Drug Administration on Aug.30 approved the first-ever gene therapy to treat children and young adults with leukemia.

University of Michigan C.S. Mott Children's Hospital is on track to becoming a treatment center for the revolutionary "living drug" known as CAR T-cell treatment. The technique involves genetically modifying a patient's own T-cells, which then can target and kill a form of acute lymphoblastic leukemia cells.

“This is a dawn of a new and exciting era in cancer therapy,” says Gregory Yanik, M.D., clinical director of the Pediatric Blood and Marrow Transplantation Program at Mott.

“This new treatment has the potential to change the face of cancer therapy for years to come, not just in childhood acute lymphoblastic leukemia but in other cancers in which a patient’s own T-cells can be collected, genetically modified and redirected to kill a patient’s tumor. This allows us to turn patients’ own cells into a powerful weapon to fight the disease — a weapon that does not rely on chemotherapy but takes a whole new approach to attacking childhood leukemia.”

CAR T-cell treatment involves extracting millions of a patient’s own T-cells, a pivotal, disease-fighting white blood cell. The T-cells are turbocharged through bioengineering techniques that reprogram them to kill cancer cells. The technique essentially transforms the patient’s cells into what scientists call “a living drug.”

In the clinical trial, T-cells were programmed to attack a protein (called CD19) found on the surface of a common type of acute lymphoblastic leukemia. The T-cells are given to patients through an intravenous infusion that takes less than 30 minutes.

Acute lymphoblastic leukemia makes up about a fourth of cancer diagnoses among children under age 15 and is the most common childhood cancer in the U.S. Children with leukemia often respond to standard treatment, but in 15 percent of cases, cancer cells stubbornly resist treatment or patients relapse.

Doctors are quick to note that while some patients had good responses to CAR T-cell therapy, those effects have sometimes been short-lived, and the treatment can be life-threatening. Side effects can include high fevers, increased blood pressure, rapid pulse and decreased blood flow to internal organs that require stays in the Intensive Care Unit.

Novartis is the first drug company poised to offer the therapy, but it will initially limit use to a small number of medical centers trained and approved to administer the drug.

“We are pleased that our center was able to play a major role in enrolling patients for the CAR T-cell clinical trial and that we have the expertise and system in place to offer this potentially lifesaving treatment to our patients,” says C.S. Mott’s director of pediatric oncology, Rajen Mody, M.B.B.S.

“At this time, only a small fraction of patients will be considered for the new treatment,” Mody adds. “This therapy provides new hope for patients with terminal leukemia in which no other treatment has worked.”