Children’s Hospital Colorado research transforms the standard of care for childhood cancer

After participating in a global clinical trial, leaders at the Children’s Hospital Colorado (Children’s Colorado) Center for Cancer and Blood Disorders and the University of Colorado Cancer Center are celebrating results so transformative, they change the standard of care for treating most kids with B-cell acute lymphoblastic leukemia (B-ALL), the most common form of childhood cancer. The new therapy is less toxic than traditional chemotherapy, resulting in significantly fewer side effects like severe infections, mouth sores and bone marrow suppression, leading to improved quality of life for patients.

The findings were published in the New England Journal of Medicine and presented at the American Society of Hematology (ASH) Annual Meeting in early December. They show that adding the bi-specific T-cell engaging antibody blinatumomab to standard chemotherapy significantly improves three-year disease-free survival for children who have been newly diagnosed with B-ALL. The study was co-authored by Dr. Lia Gore, Section Head of the Center for Cancer and Blood Disorders at Children’s Colorado and Professor at University of Colorado School of Medicine, and Dr. Maureen O’Brien, Leukemia and Lymphoma Program Lead at Children’s Colorado and Professor at University of Colorado School of Medicine.

“The clinical trial closed early because the results were clear: Those not receiving the drug did significantly worse than those that were treated with blinatumomab,” said Dr. Gore. “This instantly changed the standard of care for patients at Children’s Colorado and across the nation.”

The data presented came from a phase 3 study, AALL1731, conducted by the Children’s Oncology Group (COG), the largest research organization for childhood cancer in the world. Earlier trials with blinatumomab were led by Dr. Gore and the Experimental Therapeutics Program team at Children’s Colorado. This large international trial was written by a team of COG researchers including Dr. Gore and other members of the COG acute lymphoblastic leukemia committee. The team at Children’s Colorado – including Dr. Meg Macy, Program Lead of Experimental Therapeutics Program, and Deb Schissel, Research Nurse – was instrumental to the early studies with blinatumomab and helped teach other sites around the world how to treat patients with the drug over the past 14 years. 

“We started working on the first pediatric study of blinatumomab in 2009 and Children’s Colorado helped lead the treatment of patients in the U.S. with the new protocol in 2011,” said Dr. Gore. “I feel immensely proud of the team and the work that continues to happen in this field. This research means more kids will not only survive, but they will also thrive; with this treatment, kids will do better, feel better, their lives will be less negatively impacted by cancer. There is even greater hope that they will be treated with this regimen and never relapse.”

Of the patients in the AALL1731 study who received blinatumomab, 96% were disease-free for at least three years, and they were 61% less likely to have the cancer relapse than those in the trial who did not receive the drug. This demonstrates a practice-changing breakthrough for children diagnosed with B-ALL, and blinatumomab is now being used in most patients due to these findings. Many patients who did not receive blinatumomab the first time through had it added to their treatment plans to allow them to have the same positive outcomes. 

The drug is administered via two 28-day continuous intravenous (IV) infusions and is most effective when given after the first month of standard chemotherapy, when the burden of the disease is low. The total course of treatment for childhood B-ALL takes two years. Researchers, including the team at Children’s Colorado, are working to use a form of blinatumomab that can be given as an injection under the skin without needing continuous IV infusions, which will further enhance quality of life for patients and families. Children’s Colorado will also be part of this next set of clinical trials.

“Children’s Colorado is a global leader in pediatric cancer research and treatment, and it is truly transformative to see such extraordinary results with this trial,” said Dr. Ronald J. Sokol, Chief Scientific Officer of Child Health at Children’s Colorado. “The data presented shows a significant improvement in disease-free survival for children with B-ALL and will potentially change the landscape of childhood cancer treatment for years to come.”