No Child Left Behind

Childhood cancer garners sympathy from all, but drug development from few.

Not too long after her 15th birthday, Callie Caylor finished a high school basketball game with the feeling that her heart was gripped in a vise. Eventually, her breathing found its normal cadence, the pain quieted, and the Texas high school freshman was given a diagnosis of asthma.

Life went as usual the next few weeks. Then one morning after a track meet, the chest pain was so excruciating Callie could not lift herself out of bed. She would soon find out that her 120-pound frame carried a tumor in her torso the size of a football.

Doctors at Children’s Medical Center Dallas—a little over an hour from her hometown—diagnosed Callie with Ewing’s sarcoma, a rare malignancy arising from the bone, which disproportionately affects children and young adults. Treatments melted away her cancer, and for more than a year, Callie returned to a teenager’s universe of school, movies with friends, and as always, athletics.

Until the day a scan found a dime-sized spot of tumor in her remaining lung. That is when Callie’s parents made a surprising discovery: their daughter’s choices for backup treatment came from a shallow pool. In the race to develop better drugs to treat cancer, children have often been left at the starting gate.

The problem, at least in dollar terms, is that most children are healthy. In the health consumer market, big patients simply overpower little ones. In any given year, more than one million adults will learn they have cancer, but less than 13,000 children under age 19 will get the same diagnosis.

To makes things worse, at least for the bottom line, the common cancers—breast, prostate, colon, and lung—occur rarely, if at all, in minors, who tend to suffer leukemias and brain, bone, connective tissue, and nervous system malignancies. For drug companies wanting and needing robust sales, and even taking into account orphan product allowances, children’s cancers are often too infrequent to be profitable.

Donna Caylor, Callie’s mother, understands the business sense, but asks government decision makers and pharmaceutical executives to consider one other reality. “They need to be in those hospitals and looking at those children,” she says.

Not just her daughter. Two-thirds of all medications used in kids—drugs for any condition—have not been tested and labeled for pediatric use, according to data reported last year. (One in five adult prescriptions across conditions is off-label.) But oncology may feel the deepest urgency, since cancer remains the most fatal illness of childhood.

“It’s a real problem getting pharmaceuticals specifically for children,” says Jessica Boklan, MD, of Phoenix Children’s Hospital. Mostly, they get the hand-medowns. “It’s pure economics.”

Beyond economics, she says, the logistical and ethical demands of clinical trials have also worked against children. The small number of patients, especially for some of the more deadly cancers, makes it more difficult for researchers to gather enough participants for trials with statistical muscle. Also, ethical guidelines have historically discouraged the testing of experimental medications in children. How does all this play out? One study last year in the Archives of Internal Medicine reported that 93 percent of children with cancer receive at least one drug not approved for pediatric use.

Talk about this article with other patients, caregivers, and advocates in the Childhood Cancers CURE discussion group.
CURE wants to hear from you! We are inviting you to Share Your Story with the readers of CURE. Submit your personal experience with cancer by visiting Share Your Story
Not yet receiving CURE in your mailbox? Sign up to receive CURE Magazine by visiting