During a year that saw them publish discoveries at a rate of more than one a day, scientists at St. Jude Children’s Research Hospital tracked down leukemia cells hiding in “sanctuary” sites near the brain, figured out why a key cancer drug sometimes stops working and uncovered tantalizing clues about a deadly, incurable brain tumor.
The breadth and impact of the 540-plus scientific papers published over the past year reflect one reason why the National Cancer Institute, during a recent review, judged St. Jude “exceptional” — the highest rating it bestows on designated comprehensive cancer centers.
“In fact, the reviewers even called us a national treasure,” said Dr. Charles Roberts, executive vice president of St. Jude.
Roberts discussed the scientific advances before an audience of St. Jude donors and supporters during a presentation called “The State of Childhood Cancer” on Wednesday, September 9. The event, part of Childhood Cancer Awareness Month, featured NBC News anchor Willie Geist.
CCAM, Roberts said, offers an opportunity to highlight the “incredible problem” of pediatric cancer, which remains the top disease-based killer of children aged 1 to 15.
Some of the most promising recent breakthroughs at St. Jude, he said, involve the most common form of childhood cancer, acute lymphoblastic leukemia. Although St. Jude advances have helped push the survival rate for ALL to 94 percent, the prognosis worsens for children who suffer relapses.
“We have advanced the treatment, but relapse remains a problem,” Roberts said. “At St. Jude, we discovered that there is what we call a sanctuary site, where leukemia cells can hide out in the fluid that surrounds the brain. Our medicines don’t get there very well.”
Researchers devised a way to deliver medicine to the cells, slashing the rate of these types of relapses by two-thirds, he said.
Another persistent problem in pediatric cancer care is drug resistance. A widely used steroid, dexamethasone, is usually very effective, Roberts said, but occasionally it “can stop working in leukemia cells, and they can become resistant.”
A St. Jude team studying the problem discovered a specific gene driving the resistance and identified a drug that reverses it.
Roberts also discussed advances in the understanding of pontine gliomas, aggressive brain tumors that usually afflict children between the ages of 5 and 9. He called them one of the most difficult cancers to treat. “No one has ever cured that.”
But research at St. Jude has pinpointed the molecular basis for the disease — knowledge that could point to a cure, Roberts said. “So we are going at it as intensely as we can because we don’t want to be in the position any longer of having to say, ‘We’ll do our best to make your child comfortable, but this one is incurable.’”
The advances, Roberts said, underscore the transformative effect of St. Jude, which, in addition to its groundbreaking scientific research, cares for children with catastrophic diseases without ever sending families a bill for treatment, travel, housing or food.
“It’s really an oasis for families in the hardest time of their lives,” he said.