Our Two-Track Research Goal: For Tomorrow and Beyond

When we understood our mission to change the landscape of pediatric cancer medicine must lead to a permanent research center, it was immediately important that we fund a research chair. Thanks to our relationship with Riley Hospital, we already knew the perfect candidate: Dr. Jamie Renbarger, Riley’s section chief of Hematology/Oncology and a world-renowned scholar of both pediatric personalized medicine and pharmacogenomics, the study of how a person’s genes affect their response to drugs.

Dr. Renbarger’s expertise matches the endowment’s research goals well. The Caroline Symmes Cancer Endowment aims to conduct dual research tracks: one squarely focused on how existing cancer treatments for adults can be adapted for use in pediatrics, and the second on a molecular level, to get closer to the heart of both prevention and cure of non-CNS solid tumors through harnessing the power of the genome.

To help our donors and supporters understand how and why we think this research will succeed, we wanted to explain more about what characterizes a non-CNS solid tumor, and how the Caroline Symmes Cancer Endowment believes their unique research approach will significantly improve the treatment and prevention of these tumors.

Non-CNS? Solid?

For oncologists, one of the primary distinctions they can make about a cancer is where it originated. Cancers originating in the brain or spinal cord are classified as central nervous system tumors (CNS) while cancers forming in other areas are called non-CNS. Additionally, doctors divide cancers according to their nature; solid tumors and cancers of the blood. Unfortunately, what little funding is available for pediatric cancer research is often directed at CNS tumors and related blood cancers like leukemia. These are the most common childhood cancers, so it makes sense that funding would be directed there, but that’s also where our opportunity to make a difference arises—by providing research funding for the orphan diseases which are left out of the research umbrella.

Part 1: Transitioning Treatments

Though non-CNS solid tumors are relatively rare in children, they’re extremely common in adults. Breast cancers, lymphoma, melanoma, colon cancer, kidney cancer, bladder cancer—in adults, these non-CNS cancers are usually caused by environmental conditions, and treated with both chemotherapy and other prescriptions. Chemotherapy is also used to treat non-CNS tumors in children, and in some cases, surgery is also an option.

However, according to Dr. Renbarger, in 30% of pediatric cancer cases, chemotherapy simply isn’t adequate, and surgery either doesn’t earn a cure or simply isn’t possible. Many of the 30% of cases are non-CNS tumors whose treatment in children hasn’t been researched. By supporting Dr. Renbarger in her research, the endowment hopes to see many of the new cutting-edge medicines used in adults made suitable for children under the guidelines of the FDA. This work allows us to innovate treatment for children with orphan diseases faster, using strategies which have already found success in adults.

Part 2: Understanding the Genome

Even in adults, cancer medicines are prescribed as “one-size-fits-all,” but depending on a person’s genetics, a treatment can have very different effects. In pediatric cancer, the genome plays an even more important role. The majority of childhood cancers, both CNS and non-CNS, are the result of genetic mutations or abnormalities, not environmental conditions like in adults.

Through the research supported by the endowment, Dr. Renbarger hopes to lay the foundation of a new classification system for pediatric cancers. Rather than assigning treatments based on where the child’s cancer originated in the body, she wants to understand where the cancer originated in the gene, and try to both treat and prevent the disease at the molecular level. Imagine the ability to turn off or disable a malignant mutant gene, perhaps even before cancer develops.

This detailed, specific research will have far-reaching impacts when it is completed. Not only will we better understand the causes of cancer at a genetic level, we’ll be able to target treatments more specifically based on a patient’s genetic characteristics. In adults, some of this kind of analysis is already taking place. It’s time to harness that innovation, and the many new prescriptions available, to the benefit of children. And who better than the children whose illnesses are the least noticed and least researched?

Of course, all this research achievement is contingent upon the Caroline Symmes Cancer Endowment raising enough funds to empower Dr. Renbarger in her work. If you’re interested in contributing to that goal, learn how to help here.

Leave a Reply