Advocacy

Curing Childhood Cancer in the 21st Century: It’s Time for a Kid-centric Approach

by Lionel Chow, M.D., Ph.D.
June 25, 2014

Kids are not simply small adults. They need targeted research for targeted cures. You can help — speak up for kids with cancer today.

Lionel Chow, M.D., Ph.D.

Lionel Chow, M.D., Ph.D., is a St. Baldrick’s Scholar studying high-grade gliomas, a particularly aggressive and deadly class of brain tumors in children.

Over the past four decades, we have seen dramatic gains in long-term survival and cure rates in nearly all forms of childhood cancer. Thanks to the collaborative spirit of pediatric oncologists across the world and the invaluable participation of pediatric patients in clinical trials, the five-year survival rate for all childhood cancers combined currently sits at about 80%.

Despite these advances, cancer remains the leading cause of non-accidental death in children and adolescents in the United States. Even more disconcerting is the fact that all childhood cancers receive less than 4% of the total amount of research funding from the National Cancer Institute.

While the FDA has approved 88 new drugs to treat adult cancers since January 2000, only three have been approved for use in the pediatric age group. This rather thin record of drug development for pediatric cancers underscores that the driving force for drug discovery in the pharmaceutical industry is the larger market potential of common adult cancers.

But more importantly, these numbers hint at the fundamental differences that exist between childhood and adult cancers.

Cancer in children is not the same as cancer in adults. First and foremost, many pediatric cancers are unique to children because they originate in cells that are only found in the fetus and in early childhood.

While the FDA has approved 88 new drugs to treat adult cancers since January 2000, only three have been approved for use in the pediatric age group.

But even in cancer types that do have adult counterparts, such as acute lymphoblastic leukemia (ALL), kids and adults respond differently to therapy because their bodies are different, and because the biology of the cancer cells is fundamentally different.

Learn more about cancer in children >

Researchers have known for years that there are genetic mutations in childhood leukemia that are not found in adult leukemia. And now, with advances in genomics and the development of powerful DNA and RNA sequencing technologies, more striking differences between childhood and adult cancers continue to be revealed.

In general, childhood cancers tend to have considerably fewer mutations than adult cancers, and many of the mutations found in childhood cancers aren’t found in adult cancers at all. In a particularly revealing example, more than half of children with glioblastoma are now known to carry a certain mutation that is never found in adult glioblastoma or any other type of childhood or adult cancer.

But even as several teams of dedicated pediatric cancer researchers work toward an understanding of how these mutations lead to glioblastoma in children, there will be little or no interest from the pharmaceutical industry because it’s not relevant to a large enough number of patients.

Research specifically designed to investigate pediatric cancers and their driving mutations needs to be encouraged and supported.

The path toward continued improvements in the care and treatment of children with cancer is clear. Research specifically designed to investigate pediatric cancers and their driving mutations needs to be encouraged and supported.

Private foundations like St. Baldrick’s play a critical role in addressing the funding gap. Aside from raising money and providing much-needed childhood cancer research funding, St. Baldrick’s and its supporters can also be an important and unified voice to advocate on behalf of children with cancer.

We continue to see exciting developments in pediatric cancer research, thanks in part to the efforts of the St. Baldrick’s Foundation and its supporters. However, translating these findings into improvements in patient outcomes will require a renewed commitment and focus on childhood cancer research at the national level.

There simply needs to be more federal funding of pediatric cancer research.

Be a voice for children with cancer. Join St. Baldrick’s advocacy network, Speak Up for Kids’ Cancer.

Speak Up for Kids’ Cancer

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