Clinical Trials For Children With Cancer

By: Ramamoorthy Nagasubramanian, MD

WHY DO CHILDHOOD CANCER CLINICAL TRIALS MATTER?

Simply put, clinical trials save lives. In fact, childhood cancer research and clinical trials have fueled one of the great success stories in modern medicine — a pediatric cancer survival rate of more than 80%. Despite these advances, cancer is still the leading cause of death by disease in children. What’s more, all childhood cancers combined receive only 4% of U.S. federal cancer research funding. We have more work to do.

HOW HAVE CLINICAL TRIALS IMPROVED CANCER CARE IN KIDS?

Treating cancer in kids is not the same as adults. We’re looking at not only treating the disease today, but also protecting children from treatment side effects for optimal development, health, and quality of life for decades to come. Pediatric cancer clinical trials have helped us develop safer, more effective treatments for kids and strategies to better manage symptoms and side effects. Thanks to advances in cancer clinical trials and research, a child with cancer today compared to even 10 years ago has a much different experience through treatment and beyond.

WHAT DO I NEED TO KNOW ABOUT CHILDHOOD CANCER CLINICAL TRIALS?

Clinical trials, which are available at leading pediatric cancer centers, offer a level of care that kids can’t get everywhere. One of the largest clinical trial networks is the Children’s Oncology Group (COG). Funded by the National Cancer Institute, and part of the larger National Clinical Trials Network, the COG involves more than 200 hospitals and research centers in the U.S. and abroad and offers more than 100 clinical trials. There are also other networks and consortiums related to specific cancers. Membership in groups like the COG is important because childhood cancer is so rare, and we can make a larger impact when we come together.

WHAT ARE THE TYPES AND PHASES OF CLINICAL TRIALS?

Clinical trials can test treatments, preventions, diagnostics and screenings, as well supportive care aimed at improving outcomes and quality of life. They also study how genes influence diseases and patterns of disease across groups of people. Phase 1 trials are smaller and are the first to study safety, dosage and delivery on human subjects. Phase 2 trials study effectiveness. Phase 3 trials are much larger and test if treatments work better or have fewer side effects than current best practices. The whole process can take 10 years or more.

WHY DO PARENTS OR GUARDIANS ENROLL CHILDREN IN CLINICAL TRIALS?

There are many reasons. For some, especially those with rare disease, clinical trials may provide the most advanced, latest care available. This can mean a better chance for kids to live and live longer, and why many choose to enroll in Phase 1 and 2 trials. Some participate for the close, constant health monitoring.

Others may participate for the potential for treatment cost savings.

WHAT ARE THE BARRIERS TO CHILDHOOD CANCER CLINICAL TRIAL ENROLLMENT?

Compared to adults, cancer in children is rare. About 16,000 children under age 20 are diagnosed with cancer in the U.S. each year. Sometimes, strict eligibility requirements mean fewer participants can enroll. Lack of awareness, finances, logistics and even cultural differences can also affect enrollment. Pediatric cancer centers with large clinical trial participation actively invest in resources to reduce these barriers, so that more kids can have a better chance to live.

HOW CAN I HELP?

Clinical trial enrollment directly affects outcomes for kids with cancer. The more children we can enroll, the faster we can advance promising treatments so that kids everywhere have access to lifesaving care. As a trusted care provider, you can help raise awareness about the importance, and particularly the availability, of childhood cancer clinical trials in our area. Talk to your parents about the benefits of participating and refer them to a pediatric cancer center with active involvement in large cancer research networks.

WHAT ARE WAYS TO IMPROVE OPPORTUNITIES FOR CLINICAL TRIALS?

It’s unacceptable that society’s most underserved children — from racial and ethnic minority groups — are more likely to die of childhood cancer than their non-minority peers due to lack of access to care. So, Nemours Children’s Health decided to do something about it.

To create a more equitable care environment for patients, we’ve worked intentionally to identify and minimize all barriers to research and clinical trial participation. To measure the impact of these efforts, our program tracks and reports weekly on three essential data elements:

1. Total clinical trial enrollments

2. Clinical trial enrollments across racial and ethnic groups and compared to population estimates, and

3. Screen failures (eligible but not enrolled) across racial and ethnic groups, as well as primary language spoken.

Our objective is to enroll all eligible patients in available clinical trials. We measure our program against national benchmarks published each year in the COG Report Card. What we’ve learned along the way is that by understanding the factors that contribute to underrepresentation in research and clinical trials, clinicians can create targeted strategies to increase engagement.

For example, by translating our research consent forms into different languages common in our catchment areas, we’ve elimi- nated patient screening failures due to language barriers. This is especially relevant considering that half of our clinical trial enrollments identify as Hispanic or non-white, a significant proportion of whom required consent in a language other than English.

Our Center for Cancer and Blood Disorders ranks second in the U.S. for accruals to pediatric clinical trials, enrolling patients on 291 trials in 2021.

Offering hundreds of cancer clinical trials each year, and we are among the national leaders in participation in symptom management and prevention research.

Today, the Nemours Children’s Center for Cancer and Blood Disorders boasts the nation’s second largest pediatric clinical trial program of its kind — and the largest in Florida. We’re one of only two pediatric programs nationwide to be recognized as a National Cancer Institute Community Oncology Research Program (NCORP)

Ramamoorthy Nagasubramanian, MD, is chief of Hematology/Oncology at Nemours Children’s Hospital, Florida. Dr. Naga has been caring for Nemours Children’s hematology/oncology patients since 2007. He did his early medical training in India at Apollo Hospitals. He moved to Chicago for a residency in pediatrics and completed a double fellowship in pediatric hematology/oncology and clinical pharmacology at University of Chicago hospitals. Dr. Naga is the principal investigator of clinical trials in pediatric cancer, sickle cell anemia and hemophilia. Call (407) 650-7715 or visit Nemours.org to find out more or schedule an appointment. 