"I was blown away by the maturity of these kids. And their parents were so finely attuned to what was going on with them. It struck me then that it would be very easy to be motivated to go to work every day," says Dr. Brown.
A decade later, Dr. Brown applies an almost child-like enthusiasm and energy to his role as director of the Kimmel Center's Pediatric Leukemia Program. "The way our body makes blood, and how that process gets disturbed to create leukemia, is something I've always found to be fascinating. One of the things that's so neat about the study of leukemia is that we know so much more about it on a genetic and molecular level than we do about solid tumors," says Dr. Brown, who explains that because leukemia invades the blood stream rather than resulting in solid tumors, researchers need only to draw blood, rather than surgically remove part of a tumor, to study it.
That's not to say leukemia is easy to fight. On the contrary, it's extraordinarily complicated as it results from not one, but several gene mutations occurring together in the same cells. Subsequently, combinations of drugs must be used against it. Dr. Brown’s lab has identified such a combination—comprised of standard chemotherapy drugs and FLT3 inhibitors (agents that prevent activity from FLT3, a gene mutation linked to certain forms of leukemia)—that may work more effectively than current therapies to kill leukemia cells. Through the Children’s Oncology Group, an international network of researchers, Dr. Brown now is leading the first clinical trials to test this combination of agents on children with leukemia.
"We're hoping that it's the first of many clinical trials to test these new agents, and that eventually they'll be part of standard treatment," Dr. Brown says.
While he's clearly excited by the prospect of developing molecularly targeted therapy that may help improve cure rates while minimizing side effects associated with therapy, Dr. Brown is also keenly aware of the grave responsibility that comes with the territory. "The trust that patients and their families show is remarkable, and something I take incredibly seriously. While I know we need new approaches to treatment, it's important that we do our homework to protect patients and expose them only to things that will help," he says.
It’s this synergy and mutual respect between families and researchers like Dr. Brown that has helped move the science of pediatric cancer—leukemia in particular—forward so quickly. “In pediatrics, over 90 percent of leukemia is treated in the context of clinical studies. The infrastructure of clinical research that has been built, with the help of families, is really unparalleled,” Dr. Brown says.
Watch Dr. Patrick Brown talk about studying childhood Leukemia