New Drug Dramatically Improves Survival for Children with Common Type of Leukemia

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OKLAHOMA CITY, OKLA. – Six-year-old Bentley’s diagnosis of precursor B cell acute lymphocytic leukemia (B-ALL) in 2019 sent his family into a tailspin of worry about what the future would hold for the sweet boy who loved to make everyone laugh.

It didn’t take long for the family’s anxiety to turn cautiously toward hope. Bentley was enrolled in a University of Oklahoma Health Sciences clinical trial studying whether a drug called blinatumomab, when given in conjunction with chemotherapy, would improve patients’ survival without the cancer returning. Within a month, Bentley’s leukemia was in remission, and he remains disease-free today.

“It was so scary when we received the diagnosis; I remember it like it was yesterday,” said Bentley’s mother, Jamie Wilson, who lives in Duncan with her three other children. “After learning about the clinical trial, we decided it was what we wanted to do. Bentley did really well from the start, and today, he is back to a normal 11-year-old’s life. The whole experience took a toll on us, but it also made our family stronger and more tight-knit. We’ve learned to love a little bit harder.”

The trial performed similarly well in other children at the 228 clinical trial sites around the world, so much so that the trial was halted before its completion date because of the drug’s clear benefit. Now, it will be standard practice to give blinatumomab in combination with chemotherapy to children with standard-risk B-ALL, the most common childhood cancer.

“We didn’t expect to see results like this: 96% of participants with standard-risk B-ALL who received blinatumomab and chemotherapy had not experienced a relapse of their cancer at the three-year point, compared to 87.9% of participants who only received chemotherapy. This is why research is so important – so we can continue to make these jumps forward in the quality of care. This drug is a game-changer,” said OU Health pediatric hematologist-oncologist and trial leader Rene McNall, M.D., a professor of pediatrics in the OU College of Medicine.

The trial was sponsored by Children’s Oncology Group, a National Cancer Institute-funded cooperative group. Clinical trials may be halted early when a treatment is found to be superior so that all qualifying trial participants can receive the more beneficial drug.

B-ALL occurs when immature B cells, a type of white blood cell that helps the body fight off infections, grow out of control and replace the bone marrow with multiple copies of themselves. Most children with B-ALL experience symptoms such as fever, bone pain, bruising, nosebleeds or infections as their normal blood cells are replaced by these abnormal cells. The cancer is most often diagnosed in toddlers but can be seen in all age groups, including adults.

Blinatumomab was previously studied in a clinical trial for patients whose leukemia had relapsed, which is the typical path for clinical research – first testing a drug in patients who do not have a known cure. The drug had such good results that researchers decided to create a trial for standard-risk patients.

B-ALL already had a high survival rate of about 85% for all patients diagnosed with the disease, but seeing an improvement of 10% is significant, McNall said.

“Even when we were curing 85% of kids with leukemia, we were still losing one in eight,” she said. “Now we are losing only two or three out of 100 patients. That is a huge change.”

Blinatumomab is an antibody drug with two “arms” – one arm recognizes leukemia cells, and the other recognizes T cells, which are immune cells that protect the body from disease. The drug essentially pulls both arms together so the T cells can recognize and start killing the cancer cells. As a targeted therapy that doesn’t harm healthy cells, blinatumomab comes with fewer side effects than chemotherapy.

Because the drug is given in two one-month cycles interspersed with chemotherapy, patients are connected to a continuous IV infusion and sent home with a backpack of the drug that slowly drips into a port. The arrangement involves diligence on the part of families but eliminates a lengthy hospitalization.

“This trial represents why I work in academic medicine – we want to treat our patients today, but also to conduct research for the kids who are coming next,” McNall said.