Nationwide Trial Seeks to Reduce Heart Damage in Pediatric Cancer Patients

Investigative drug may improve overall survival and reduce chemotherapy-induced heart damage in children with acute myelogenous leukemia.

In an upcoming trial, investigators hope to improve survival in pediatric cancer patients, and reduce chemotherapy-induced heart damage.

The trial will test a drug called CPX-351 that is designed to kill leukemia cells, while minimizing damage to the heart. The trial will include children and adolescents with relapsed acute myelogenous leukemia (AML).

Up to 30% of patients with AML who undergo chemotherapy will have late-term adverse effects that affect the heart, according to the investigators.

“You go through terrible chemotherapy, achieve remission, have a new lease on life, and then your heart fails,” said lead study author Dr Todd Cooper. “It’s not fair, and we’re determined to change this reality.”

Chemotherapy for patients who have AML is aggressive. It involves multiple anti-cancer drugs administered at a higher dose to kill the cancer cells, since AML is difficult to treat.

“The chemotherapy tends to be very intensive,” Cooper said. “Some of the most effective medicines work really well against leukemia, but the side effects, including damage to the heart can be severe.”

CPX-351, however, delivers chemotherapy in a different way than standard chemotherapy. The drugs are contained in a liposomal formulation, which is believed to be safer for the heart, according to the study. The liposome is used as a vehicle to transport the drugs into leukemia cells in the bone marrow.

“We hope that by being housed inside the liposome, that less of the chemotherapy will be deposited into the heart,” Cooper said.

Prior studies of CPX-351 have shown tremendous promise in adults, and Dr Cooper hopes to see similar success in pediatric patients.

In phase 3 trial of CPX-351 for adults with high-risk, secondary AML, there was a statistically significant improvement in overall survival compared with patients who received standard chemotherapy. CPX-351 reduced the risk of death by 31% compared with the chemotherapy drugs cytarabine and daunorubicin.

“This trial not only offers hope for more children to be cured, but for more children to live longer, leading more productive lives without late-term cardiac damage,” Cooper said. “I am thrilled to bring this therapy to children through the trial because I want kids to have access as soon as possible to these potentially lifesaving drugs.”

Although the results from the trial will not be available for a couple of years, Cooper is hopeful that CPX-351 will improve outcomes for children with relapsed AML, and potentially become a first-line treatment in the future.