Targeting Gaps in Pediatric Cancer Science

“Nobody can cover the entire playing field in childhood cancer research,” says Robert Arceci, MD, PhD, chair of the scientific advisory committee at St. Baldrick's Foundation in Monrovia, CA. “St. Baldrick's tries to fill gaps that other agencies are not addressing, such as training promising translational physician-scientists, and to fund the most impactful science available.”

Since becoming an independent foundation in 2005, St. Baldrick's has given more than $127 million in grants for pediatric cancer research, says Arceci, who is also codirector of the Ronald A. Matricaria Institute of Molecular Medicine at Phoenix Children's Hospital in Arizona.

The foundation claims to fund more in childhood cancer research grants than any organization other than the federal government, raising money primarily through events in which family and friends gather donations by pledging to shave their heads in solidarity with children affected by cancer. (The first such going-bald event, held on St. Patrick's Day in 2000, inspired the name “Baldrick.”)

In addition to its awards for young scientists and doctors working in pediatric oncology research, this year St. Baldrick's Foundation began to fund training for international scholars in low- and middle-income countries.

Among its larger projects, in April 2013, St. Baldrick's announced it would partner with Stand Up to Cancer to support a Pediatric Cancer Dream Team, a $14.5 million, 4-year project to develop immunogenomics-based advances for children with cancer. “Progress in immunotherapy has been a bit of a roller coaster,” Arceci remarks. “This is a superb team of people who bring all of their complementary skills together to try to move the bar further.”

Overall, St. Baldrick's has awarded over $24 million in grants this year, including $6.1 million to the Children's Oncology Group (COG), a cooperative research group with about 200 member institutions that treat more than 90% of the kids diagnosed with cancer in the United States.

“There's no other group that can do phase III randomized clinical trials in pediatrics like the COG, but it is not sufficiently funded by the NIH to do the work that it needs to do,” says Arceci. St. Baldrick's has given over $46 million to the COG since 2005.

Many St. Baldrick's grants to individual investigators focus on new ideas for targeted therapies.

“The concept of individualized medicine has attracted so much of the medical oncology world, and yet it's in children that this challenge might be most effectively addressed, because of the less complex mutational load of many pediatric cancers,” Arceci points out. “We need to create individualized approaches to rigorously test treatments in children with cancer. The future has arrived; let's stop talking about it.”

The foundation also maintains a strong focus on research related to treatment side effects, survivorship issues, and healthcare delivery for children and adolescents, Arceci emphasizes.

“One supportive-care epidemiology study, for instance, is addressing the much higher incidence of relapse with acute lymphoblastic leukemia among patients of Hispanic background than of non-Hispanic background, which may be due in part to compliance for oral medication during those 2 to 3 years of maintenance therapy,” he says. “If it's a matter of taking pills on a regular schedule, that should be fixable, but nobody has really done it. You can say this isn't cloning genes, but it's just as important, if it has a real impact.”

For more news on cancer research, visit Cancer Discovery online at http://CDnews.aacrjournals.org.