cancer, the pediatric cancer drug market is often well below the radar screen, and typically it has not made business sense to invest in research and development for these cancers. Many drugs developed for adults have been found effective in children, in large part because most have a generalized affinity for cancer cells. The dark side of this characteristic is that noncancerous tissue may be damaged in the process, causing the well-known adverse effects of anticancer drugs. But in fact, the biological and clinical characteristics of nearly all childhood cancers differ substantially from adult cancers. Over the past few years, differences at the molecular level have been documented for all the major childhood cancers, and herein lies the promise: the molecular abnormalities represent a place to start searching for drug “targets.” Cutting-edge science notwithstanding, market forces are not sufficient to drive the process and bring to the bedside new drugs for children with cancer. Because so much of the technical capacity for drug discovery and development for pediatric cancers already exists—much of it supported by the National Institutes of Health (NIH) intramurally or extramurally, as well as in the private for-profit sector—it is possible that these drugs could emerge from an alternate pathway. Specifically, a “public– private partnership” could knit together the pieces in a virtual research and development (R&D) network. Networks such as this are relatively new, but are working well for cystic fibrosis, tuberculosis, malaria, and other neglected tropical diseases. The resources already in place for pediatric cancers are poised for this development.

Recommendation 1: A new public–private partnership, involving government, industry, academic and other research institutions, advocacy groups, philanthropies, and others, should be formed to lead pediatric cancer drug discovery and development.

R&D specifically for childhood cancers is not the only way to improve treatment. New drugs in development for adult cancers may prove useful for children, including some that, in the final analysis, fail to provide significant benefit to adults. This poses a dilemma for companies that would never be able to recoup development costs if a full-scale effort resulted only in a drug for children. At whatever stage a product is in development, if this is the case, the government should consider taking over the development process, either directly or through funding external work.

Recommendation 2: The National Cancer Institute should assume responsibility as the developer of last resort for agents that show promise only in children if companies decide not to proceed with full-scale development.

Even when an agent is shown effective in adults, historically, long delays have intervened between the time testing is begun in adults and in children. Because there are relatively few children with these cancers, clini-

The National Academies of Sciences, Engineering, and Medicine
500 Fifth St. N.W. | Washington, D.C. 20001

Copyright © National Academy of Sciences. All rights reserved.
Terms of Use and Privacy Statement