A Global View of ALL

A standard of care since the 1970s, this dex-vincristine combo also has repercussions that extend far beyond the treatment phase. The problems include personality and mood disturbance; sleep problems; metabolic syndrome, which is linked to heart disease and Type 2 diabetes; and peripheral neuropathy, a type of nerve damage. 

“The side effects from the dexamethasone and vincristine are not negligible,” says St. Jude oncologist Hiroto Inaba, MD, PhD, principal investigator of Total Therapy 17, a St. Jude clinical trial for ALL.

As a result, Pui — and an esteemed group of researchers in China — decided to look into the status quo and possibly change it.

Collaboration is key

In the U.S., children with ALL have a survival rate of 90%. Yet, children in low- and middle-income countries, such as China, fare far worse.

In 2018, St. Jude Global embarked on a mission to change that scenario. A signed accord and research partnerships with China count among its many achievements.

Even before the global program began, Pui had been working for decades to improve outcomes for children with leukemia in China. Since 2006, he has spearheaded an annual teaching conference on childhood cancer in Asia. And, thanks to his efforts, ALL became the first childhood disease covered by the Chinese New Medical Insurance Policy—providing hope to thousands of underprivileged children in China.

Today, Pui serves as medical director of the St. Jude Global China Program. Under the hospital’s new strategic plan, Pui will continue to play an important role in improving treatment and research of front-line ALL studies in collaboration with the Chinese Children’s Cancer Group.

Lifting the burden

St. Jude children and families love Pui. He’s more than willing to do a magic trick or tell a good joke — or a joke good enough to elicit a groan — to make connections and quell treatment anxiety.

Understandably, some patients and families dread steroid pulses. Pui wondered if prolonged treatment with these drugs was necessary. 

To find out, he collaborated with researchers in China to conduct a clinical trial with 6,108 patients — the largest randomized trial in pediatric leukemia history.

The scientists found that the dex-vincristine therapy could be safely eliminated in low-risk patients beyond the first year of treatment, effectively quashing toxic side effects linked to this regimen.

“Many parents will be very happy,” Pui says.

Additional studies will be needed to determine if this regimen can be omitted for patients in standard-risk groups.

“The weeks that they get this medicine are terrible,” Pui says. But the burden may be lifted for a substantial number of patients and families.