By Kelli Anderson
The girl was an only child, with wavy dark hair, big eyes with long lashes, and a dazzling smile to match a radiant spirit. She loved Disney characters and dotted the i in her name with a little heart, as 9-year-old girls sometimes do. Michelle Monje, a Stanford MD/PhD student when she met the girl in 2002, was smitten. Twenty years later, Monje remembers how she sat alongside the girl’s mother in the family’s kitchen after the funeral as the grandmother, in tears, cooked for guests.
The girl was the first patient that Monje, MD ’04, PhD ’04, had ever seen with the rare pediatric brain cancer called diffuse intrinsic pontine glioma (DIPG). Perhaps because it strikes a few hundred children a year in the United States—usually between the ages of 4 and 11—and does its lethal work swiftly, DIPG is far less notorious than it deserves to be. It’s the leading cause of death from pediatric brain cancer. Otherwise healthy children suddenly have some clumsiness, weakness on one side or one eye that turns inward. As the tumor weaves itself around healthy tissue in a deep part of the brainstem, foreclosing the possibility of surgery, the nerves that control the head, neck and face start to fail. The typical therapy is six weeks of radiation, which might shrink the tumor, buying kids some time and giving them some function back. But the disease always returns and leads to a death of increasing paralysis, with patients unable to move, communicate, swallow, control secretions or, eventually, breathe—but still perfectly aware of everything that is happening. Patients with glioblastoma, the most common adult brain cancer, face a five-year survival rate of just 7 percent. The outlook for DIPG patients is even grimmer: The five-year survival rate is less than 1 percent. Most patients die within a year of diagnosis.
As her young patient followed DIPG’s predictably cruel trajectory, her beautiful smile fading to sadness, Monje felt grief, frustration and what would become unshakable purpose. “I had never seen anything as horrible as this disease,” she says. “I was stunned that we knew so little about it.” No one knew what mutations drove the disease or what developmental process went awry. There were no animal models, such as mice implanted with the tumor, to study. “It was a black box with no tools,” says Monje.