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Researchers Test ‘Breakthrough’ Pediatric Leukemia Treatment

Reprogrammed T-cells detect cancer

A promising method of immunotherapy to treat children with relapsed acute lymphocytic leukemia (ALL) is being investigated at the University of Wisconsin’s Carbone Cancer Center and American Family Children’s Hospital. The new trial is open to pediatric patients with relapsed/refractory ALL who have limited treatment options.

The Carbone Cancer Center is among several centers participating in a phase II study to test an immunotherapy called CTL019, which was developed by a research team at the University of Pennsylvania. Penn and Novartis have partnered to develop and commercialize targeted chimeric antigen receptor (CAR) immunotherapies for the treatment of cancers. CTL019 received the FDA’s “breakthrough therapy” designation in July 2014.

The experimental treatment uses CARs to attach to, and hunt, leukemia cells expressing a CD19 antigen by employing the study participants’ own T-cells. These cells are reprogrammed in the lab to express the CAR receptor, which may detect leukemia cells and destroy them.

“Once the T-cells are reprogrammed, the modified cells are shipped back with the special cells,” said principal investigator Dr. Christian Capitini. Both the collection of T-cells and their infusion can be outpatient procedures.

According to Capitini, the participants may develop symptoms of cytokine-release syndrome. After being infused into the patient, the CAR cells grow in massive numbers and trigger the release of inflammatory cytokine molecules.

“Symptoms include high fevers, chills, and headaches, but some very serious symptoms, like dangerous blood pressure drops or breathing difficulties, may develop,” Capitini said.

The participants must stay close to the hospital because of these rapidly developing symptoms.

“At this point, the patients are hospitalized, and some may be admitted to the intensive care unit if necessary,” Capitini said. “Patients are treated with an anti-inflammatory drug used for rheumatoid arthritis called tocilizumab. The symptoms may resolve in a matter of hours after administration of the drug.”

Follow-up care may involve monthly immunoglobulin infusions because normal B-cells are killed by the CTL019 immunotherapy.

Source: University of Wisconsin–Madison; February 24, 2015.

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