“Personalized medicine is the big buzzword,” says Dr. Will Parsons, sitting at his desk in his office inside the TMC. Parsons, a star pediatric oncologist, was recently tapped to help Vice President Joe Biden as part of his Moonshot initiative to help cure cancer. Specializing in the treatment of brain tumors, he also has another passion, one that no doubt caught Biden’s eye: Through the federally funded BASIC3 study, he helped set up a genetic-sequencing lab to test the blood and tumors of children with cancer.
Through those tests, Parsons and the study’s other leader, Sharon Plon, a cancer geneticist and his co-director at the Cancer Genetics and Genomics Program at Texas Children’s Cancer Center, hope to find out the genetic causes of cancer and match each child with a specific drug for their specific disease. “In pediatric oncology, we’ve been trying to treat each kid in a personalized way and have been doing that based on different clinical features, or how old they are, or how far their tumor’s spread,” Parsons says. “But here, we’re really trying to learn what we can about each kid’s individual tumor, so that we can target treatments specifically that might be best for them.”
Cancer drugs have come a long way in the past few years, with scientists creating new formulas to take on specific gene mutations, with the goal of limiting negative side effects while more effectively combatting disease. But the field of research is fairly new, so studies like BASIC3, which has enrolled 280 kids and their families, will go a long way toward finding more genetic mutations, and along with that, more tailored drugs.
If the families test positive for a mutation known to increase cancer risk, TCH counselors help them decide on a course of action, including regular screenings for siblings who might be at risk. “When we’re meeting with new families, one of the first questions is always, ‘Why did this happen? Are my other kids going to get cancer?’” Parsons says. “These are totally natural questions as a parent. So the hope is that this sort of testing can help us provide answers to that.”
TCH now has a fully functioning genetic testing center that serves patients from Houston and around the country. And Parsons is spearheading a new, larger, national study that will build on BASIC3, called Pediatric MATCH. The program will enroll kids suffering from treatment-resistant cancers, sequence their genes, and match them up with clinical trials of drugs tailored to their specific needs.
“The way we’ve run trials in the past is, if we don’t know about the patient’s genetics and we have a new drug targeting a specific gene mutation, we’ll just enroll 100 kids and we’ll give it to all of them. What happens is, you get these results from the study, and it would seem a failure because it hadn’t helped almost any of the children. But if you dug through the data a little more, you’d say, ‘Wow, it didn’t work for these 95, but for this small handful, it was really awesome,’” Parsons says.
The point is to pick the needles out of the haystack. “It may be that there’s only 10 kids across the U.S. each year that have a mutation in the gene that would best respond to that drug,” he says, “but by doing this in a nationwide trial, we can hopefully find those patients and make a big difference for them.”