It's one of those super technical scientific studies that typically goes unnoticed, except by experts. But the backstory is bigger than the study's narrow focus suggests. It may have implications for thousands of children worldwide who are battling cancer.
The study involves a major scientific collaboration between global rivals, the United States and China. Researchers from both countries are working together to test a new, targeted treatment for a rare form of a deadly childhood leukemia, acute lymphoblastic leukemia (ALL), caused by a specific genetic mutation.
The mutation, called the Philadelphia chromosome, is a nasty accident of nature that occurs when genes from chromosomes 9 and 22 somehow fuse.
This genetic recombination affects the blood and bone marrow, spawning a flood of immature white blood cells. The mutation causes just 3% to 5% of ALL cases, but, untreated, the outcome is "dismal," Dr. Karen Rabin, of Texas Children's Cancer Center, writes in an editorial in the Jan. 16 issue of JAMA Oncology.
In the study, published in the same journal, researchers compared two drugs, both designed to counter Philadelphia chromosome ALL. Targeted cancer therapy is, by itself, a milestone, a way to attack cancer by neutralizing the genetic defects that cause cell replication to go haywire.
Before drug treatment became available, the only hope for people with this disease was a combination of chemotherapy and bone-marrow transplantation, a devastating one-two punch that ravaged patients in a desperate attempt to save their lives.
A pioneering drug called imatinib offered the first significant remissions, improving 5-year, event-free survival to 57%, along with chemotherapy and brain radiation. The additional treatment was necessary because imatinib didn't penetrate the recesses of the brain.
Now a second-generation drug, dasatinib, shows additional promise against Philadelphia chromosome cancers. Dasatinib is more potent, penetrates more deeply into the central nervous system and prevents relapse in the brain.
In the new research, collaborators from St. Jude Children's Research Hospital in Memphis and in Chinese medical institutions compared the drugs head-to-head. Dasatinib emerged the winner.
The study, conducted from 2015 to 2018 in patients 18 and younger at 20 Chinese hospitals, was halted early by the trial's Data Safety Monitoring Board because the newer drug, dasatinib, was so much better than the older version that the study overseers wanted everyone in the trial to get the more effective drug.
In an analysis of 189 patients, 4 year event-free survival was 71% among patients taking dasatinib, versus 49% for those taking the older drug.
Only a decade ago, conducting such a trial in China would have been unthinkable. China regarded childhood cancer treatment as expensive and ineffective and did not reimburse for treatment. As a result, most children diagnosed with cancer died.
But a small pilot study, published in 2009 by Chinese researchers in collaboration with experts at St. Jude, persuaded China's minister of health that treatment is both cost-effective and can cure a substantial number of patients, says St. Jude oncologist Ching-Hon Pui, a leader of the collaboration with the Chinese Children's Cancer Group.
In 2019 -- as U.S. News reported in September in "China's Halting Leap Forward With Childhood Cancer Care", part of a series on childhood cancer care worldwide -- the government decided to provide full insurance coverage for acute lymphoblastic leukemia (ALL), which accounts for 85% of China's childhood leukemia cases.
The percentage of children who completed cancer treatment went from about 30% to 97%. As a result, the 5-year survival rate for childhood leukemia in China has climbed from around 30% to 60%, far from the 90% cure rates achieved by the United States, but getting nearer.
Consider the bigger implications of this trial as well. China has gone from a country that not long ago regarded childhood cancer with such fatalism that insurance didn't cover the disease and only a third of children completed treatment. Today, China has joined the crusade to find new weapons to treat the disease -- which has seismic implications for cancer research.
Why? The Philadelphia chromosome ALL trial offers a perfect example. It is such a rare disease the study could not have been carried out in any country but China. China's population is so big that even the rarest diseases occur more often than they do in other parts of the world, says St. Jude's Pui.
"The U.S. and Europe together don't have enough patients to do such a trial," he says. "The Chinese have an advantage. They have a lot of patients."
That's good news for children with cancer in other countries, Pui says, because drugs that work in China will work elsewhere too. "There's no boundary, he says. "Medical knowledge will be shared around the world; everybody's free to use it to help their children."
Steve Sternberg is the Assistant Managing Editor for Health Initiatives and an architect of the Healthiest Communities initiative, a population health assessment of roughly 3,000 U.S. counties. His most recent investigative projects focused on unsafe surgery in U.S. military hospitals worldwide, the dangers of doctor burnout, and preventable deaths among children undergoing complex congenital heart surgery in children's hospitals. He joined U.S. News & World Report in 2011 as the deputy health rankings editor, supporting the Best Hospitals and Best Children's Hospitals rankings. Previously, he was an investigative reporter and medical writer at USAToday from 1998 to 2011; he has also covered health and medicine at the Atlanta Journal-Constitution and the Miami Herald, and his work has appeared in The Washington Post, Mother Jones, Medical Economics, Science, The Scientist, Bioworld Today and other publications. Sternberg is a 2015 recipient of an Association of Health Care Journalist's Award for Excellence in Health Care Journalism, the Dag Hammarskjold Award for Human Rights Journalism, the American Heart Association's Howard L. Lewis Achievement Award, the National Headliner award, the Penney-Missouri award, the Global Health Council Media Award, the Kaiser Family Foundation Media Fellowship award and the John S. Knight Professional Journalism Fellowship at Stanford University, where he studied health policy and genetics in 1992. He appears as a moderator and panelist at health and journalism conferences around the U.S. A graduate of Ithaca College, he earned his masters in science writing from Johns Hopkins University. Follow him on Twitter, connect with him on LinkedIn or email him at email@example.com.