NCI Meeting Explores Barriers to Drug Development for Pediatric Cancers

by Peter Mayberry, Sr. Manager of Legislative Affairs, St. Baldrick's Foundation
March 5, 2014

Why are there so few drugs available to fight childhood cancers?  Why have no new drugs been developed and approved specifically for pediatric cancers in decades?

These questions dominated the agenda on February 21 when the National Cancer Institute (NCI) convened the 64th meeting of its Director’s Consumer Liaison Group in Bethesda, Maryland. Entitled “Barriers to Drug Development in Pediatric Cancer Research,” the all-day meeting included presentations from oncologists, government scientists, physicians, and members of the childhood cancer advocacy community.

One of the most dramatic moments of the meeting occurred when a member of the advocacy panel contrasted her daughter’s treatment for AML 27 years ago with that of a young girl — also with AML — who she helped just last year. Both children received the same drugs, despite the decades that separated their diagnosis and treatment. The drugs were synthesized and approved by the FDA in the 1950s and 1960s. The advocate’s daughter suffered severe late effects from her treatment, including a secondary cancer, as well as the birth of her child born 4 months prematurely who now has severe epilepsy.

Several barriers to new drug development were identified, a primary one being almost no “market” exists for drug makers when it comes to creating drugs for kids with cancer. In terms of return on investment, pharmaceutical manufacturers get the biggest bang for the buck when they develop drugs that treat long-term, chronic ailments for large populations. But when it comes to children with cancer, the population is viewed as relatively small and treatment is short term.

Ethical issues were also raised. A lack of materials needed for research, for example, was repeatedly mentioned as a stumbling block to new drug development. According to several experts, there are not enough tumor cell samples being collected for research, due to concerns that children must undergo a biopsy to collect them. Similarly, when it comes to developing new drugs for pediatric cancers that are only diagnosed a  few times each year, it can be difficult to develop clinical trials to test the safety and effectiveness of new compounds due to the low number of affected patients.

The issue of long term effects was repeatedly discussed. Even children who are effectively treated for cancer will experience lifelong side effects due to treatment that tend to get worse with age. These can include cognitive disability, hearing and/or sight loss, stunted growth, infertility, and even premature death.

But there was good news too! Research supported by the Stand Up To Cancer-St. Baldrick’s Foundation’s Dream Team* was commended, as was the research underway within the Children’s Oncology Group that is also supported by the St. Baldrick’s Foundation.

Especially promising, according to several experts who spoke during the meeting, are experimental approaches based on a better understanding of the human genome and ways that individual immune systems can be fortified to identify, attack, and kill cancer cells. Dream Team* efforts to repeat the success rates of treating leukemia on tumor cells were also commended.

All in all, it was a bittersweet day. While there was plenty of frustration and obstacle identification, there was also genuine hope based on new research and better understanding of genetic code.

Watch the entire program online, including Peter’s presentation at the NCI meeting, beginning at the 4:49 mark in the video.

Watch the NCI Meeting

*Formerly known as the St. Baldrick’s – Stand Up to Cancer Pediatric Cancer Dream Team, this team is now the St. Baldrick’s EPICC Team (Empowering Pediatric Immunotherapies for Childhood Cancer).

Read more about St. Baldrick’s funded research: