Targeting Leukemia

Pediatric leukemia program director Patrick Brown recalls precisely the moment that he knew he wanted to make pediatric oncology a career. He was in his third year of medical school, and had just been assigned to a pediatric oncology rotation. After meeting with the first few patients, he was hooked.

“I was blown away by the maturity of these kids—and their parents were so finely attuned to what was going on with them,” says Brown. “It struck me then that it would be very easy to be mo­tivated to go to work every day.”

A decade later, Brown applies an al­most child-like enthusiasm and energy to his role as director of the Sidney Kim­mel Comprehensive Cancer 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 fascinating,” says Brown. “One of the things that’s so neat about the study of leukemia is that we know so much about it on a genetic and molecular level, and that knowledge is increasing faster and faster as new technology is developed.”

Brown explains that because leukemia often invades the blood stream, research­ers often need only to draw blood, rather than surgically remove part of a tumor, to study it. That is not to say leukemia is easy to fight. On the contrary, he adds, it is 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.

Brown’s lab identified such a com­bination—composed 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, Brown led the first clinical trials to test this combination of agents on children with leukemia.

“These were the first of many clinical trials now being done to test these new targeted strategies,” says Brown. “In the case of the FLT3 inhibitors, this com­bination has now become the standard treatment.”

While he is excited about the prospect of developing molecularly targeted and immune-based therapies to improve cure rates while minimizing side effects, Brown is also keenly aware of the grave responsibility that comes with the terri­tory. The trust that patients and their families show, he says, is something I take seriously: “While I know we need new approaches to treatment, it is im­portant that we do our homework to protect patients—to expose them only to things that we think will help, and do so in the safest way possible.”