New childhood leukemia protocol is ‘tremendous win’

A new treatment protocol for the most common form of childhood cancer has “instantly” changed how doctors treat pediatric leukemia — and provided a first-in-decades boost for the long-term survival and health of young patients.

“This happens once in several generations,” said Dr. Lia Gore of Children’s Hospital Colorado. Gore co-authored a paper published earlier this month describing the effects of the treatment.

“We’ve now moved this outcome so that the vast majority of children with this type of leukemia will have a 96% cure rate,” she said.

The breakthrough, described in a New England Journal of Medicine paper and presented at a conference in mid-December, is called blinatumomab, an immunotherapy drug delivered to patients over a 28-day IV drip. (Gore said one of her patients surfed sand dunes while wearing a backpack holding their medicine.)

The treatment teaches the immune system to recognize and destroy leukemia cells, which are otherwise invisible to the body’s disease-fighting T-cells. Its success not only improves survivor rates but also cuts down on the need for toxic treatments like chemotherapy, which can have life-altering impacts for the growing share of patients who beat the disease.

For more than a decade, researchers, co-led by Gore’s team at Children’s, had studied the impact of the medication on B-cell acute lymphoblastic leukemia, the most common type of childhood cancer. It afflicts roughly 5,000 American kids each year, Gore said. Over the summer, an oversight committee then began analyzing the latest study’s early data. That set examined blinatumomab’s impact on children between the ages of 1 and 10 who were newly combating the disease.

Some children in the study were not getting blinatumomab so that researchers could gauge whether it worked better than the standard chemotherapy approach, which seeks to kill all of the body’s fast-growing cells — including, but not only, cancer cells.

The data analyzed by the committee showed that patients who received the new treatment fared so much better than their peers that researchers determined it would be unethical to continue withholding the medication from any of the study’s subjects.

The findings were the most significant advancement in the field since at least the early 1970s, Gore said.

As a result, physicians treating this common type of leukemia have begun incorporating the new protocol into their treatment strategies. It has fundamentally realigned the established standard of care for a disease that was typically fatal just 50 years ago.

A similar study, also published earlier this year, showed promise for adults with leukemia.

“For this field, there’s not much better you can get, truly, than 97% (as a cure rate) for any sort of cancer,” said Dr. John Molina, a physician at the Leukemia and Myeloid Disorders Program at the Cleveland Clinic. He also has studied B-cell acute lymphoblastic leukemia. “… It’s incredibly important, because most patients with leukemia will eventually die because of relapsed disease.

“If we’re able to cure patients upfront, we drastically decrease the relapse risk.”

Not only that, but the use of blinatumomab will also improve the overall quality of life for children recovering from leukemia, Gore said. While survival rates have improved for pediatric leukemia over the last several decades, the previous treatment approach often had long-term impacts on children’s health, from weakened bones and hip or knee replacements to neurological problems.

“If I have a 3-year-old who’s being treated for (acute lymphoblastic leukemia) and they develop a lifelong toxicity and side effects, we might be talking about 80 years of that,” Gore said.

But blinatumomab will mean less chemotherapy and other intensive treatments, which in turn will lessen the long-term price of beating leukemia.

“That’s a tremendous win,” she said.

Because survival rates and treatment have improved so much already, Molina said, researchers and providers had wondered if they were approaching a ceiling with the prevailing treatment protocol driven by chemotherapy.

But the findings shatter that ceiling.

Blinatumomab was first identified by German researchers, Gore said. In 2011, researchers began studying it in children whose leukemia had returned. Gore was the North American principal investigator for the 2011 study, she said, and Children’s was a lead hospital for the trial.

In the years since, providers have introduced it earlier and earlier in treatment, culminating in the results published earlier this year.

The Food and Drug Administration authorized blinatumomab in 2017, Molina said. In light of the latest results, the FDA has now authorized the treatment as a front-line therapy for new patients, too.

Blinatumomab, which trains the immune system’s T-cells to recognize a specific protein on leukemia cells, can also be used against other forms of leukemia that have the same protein.

“This is wave of the future,” Molina said. “We’ll be using more immunotherapy earlier in therapy, and hopefully having less patients that do relapse.”