In 2017, the National Cancer Institute and the Children’s Oncology Group joined forces to create the Pediatric MATCH (Molecular Analysis for Therapy Choice) Trial. Over 1300 children were enrolled across the United States and Australia, and the trial was one of the first clinical trials that focused specifically on pediatric cancers and the possible utilization of Targeted Therapy within pediatric cancer treatment plans. While most research arms are now closed, researchers were surprised with the amount of biomarkers that were identified. As data from this trial and other studies stemming from the MATCH Trial continue to be produced, the hope for more personalized treatment and better long-term outcomes continues to increase as more pediatric cancer research advancements are made.  

AREAS OF RESEARCH ADVANCEMENTS  

Recent research and clinical trials have focused on finding new treatment modalities that may offer more specialized approaches to cancer treatment, providing decreased side effects and increased survival rates. 

TARGETED THERAPIES & PRECISION MEDICINE:  

Targeted Therapies, sometimes referred to as  Precision Medicine, work by targeting specific mutations or abnormal proteins within the genetic profile of the specific cancer to disrupt and prevent the formation of more cancer cells. Rather than traditional cancer treatments that kill all rapidly dividing cells including healthy ones like hair follicle cells such as chemotherapy and radiation, these treatments specifically target cancer cells. Within the Pediatric MATCH clinical trial, a screening for over 160 genes associated with cancer was done to a child’s tumor sample, with hopes of delegating the child to a specific treatment arm utilizing an experimental drug able to target the identified cancer gene.  

These therapies could offer great potential as new treatments for pediatric cancer may be able to focus on providing a more personalized approach to the specific needs of the child and their cancer. Rather than determining the child’s treatment based solely on the kind or site of the child’s cancer, treatment plans can be tailored to the genetic profile of the child’s cancer with the goal of providing more effective and less toxic results compared to traditional chemotherapies.  

IMMUNOTHERAPY:  

Pediatric cancer research advancements have also been made in the realm of Immunotherapies, or treatments that utilize the body’s immune system to detect and fight cancer cells. One example is Monoclonal Antibodies, which are antibodies made in the lab that attach to cancer cell antigens and destroy them in various ways. Another is Cancer (Treatment) Vaccines, which are currently in the early stages of being studied, and are another way to help the body learn what to fight. While there are currently five FDA approved drugs that utilize this form of treatment, there are a handful of immunotherapies that may be included in the treatment of some pediatric cancers, and many currently in clinical trials.   

Another notable childhood cancer research breakthrough is a form of immunotherapy called CAR T-Cell Therapy. This therapy involves modifying and training the child’s immune system to fight their cancer. T-cells are an important part of a person’s immune system that help their body fight off germs and diseases. Car-T cells are made by collecting the child’s T-cells from their blood and modifying them in a lab to produce chimeric antigen receptors (CARs), which can target and destroy specific cancer cells. These are then infused back into the child’s bloodstream. While currently only an FDA-approved treatment option for some leukemias and lymphomas, many other clinical trials are researching its effectiveness in a variety of other pediatric cancers, such as Neuroblastoma. 

OTHER PROMISING APPROACHES:  

COMBINATION THERAPY AND THE HOPE FOR LESS TOXIC TREATMENTS:  

While the idea of combining different treatment modalities to combat cancer is not new, as new treatments continue to be developed, ongoing research on how to combine these new therapies with the current traditional treatment plans is crucial. Testing details like timing of when to administer the drugs, potential interactions with different treatments, and optimal dosages that provide the best treatment response are ongoing, with the hope that adding these new treatment forms to current regimens may provide better outcomes in the future. 

Clinical trials are also studying the possibility of reducing the dosages of certain chemotherapies and re-evaluating the need for radiation in some cancer treatments to prevent long-term side effects and reduce toxicities as well.  

INCREASED AWARENESS OF HEALTH DISPARITIES:  

Current research is also focusing on the higher rates of relapse and poorer outcomes in children from racial and ethnic minority groups, and those with a lower socioeconomic status. In addition to these poorer outcomes, children from these groups are not included in research studies and clinical trials as often when compared to children from white or wealthier backgrounds. Issues such as underrepresentation, higher costs for new testing and treatments, and lack of accessibility to clinical trials may all contribute to these issues.  

Research focusing on combating these health disparities and finding ways to ensure there is better access to care and increased availability of clinical trials and updated treatment methods is ongoing. An increased focus on incorporating social determinants of health into treatment plans and including Real World Data (such as billing information and data from electronic medical records) within research studies are current ideas to help address this issue.  

THE IMPORTANCE OF CLINICAL TRIALS:  

As pediatric cancer research advancements continue to develop, clinical trials are necessary to test their safety, potential side effects, and efficacy. Children’s participation in these trials is not only an exciting opportunity but is crucial in the development process of new treatments and protocols. Participating in these clinical trials is vital for new treatments to be accessible and utilized in the future while providing the affected child the immediate opportunity to access new treatments, new research, and the potential of receiving treatments with improved outcomes. Resources such as the National Cancer Institute (NCI), the Children’s Oncology Group, and Clinical Trials Database can offer up-to-date information about current clinical trials taking place.  

THE HOPE FOR A CURE CONTINUES:  

Whether it’s a drug training the body to fight off a child’s cancer, a personalized treatment plan providing fewer side effects, improved survival, and quality of life, or better treatment access for all children, research advancements in pediatric oncology continue to be made. There is still so much to be learned, yet with each research success, the hope for a cure for pediatric cancer is closer than it has ever been before.  

Here to Serve  

At Here to Serve, our unwavering mission is to support families with children battling cancer throughout every step of their challenging journey. As the only national organization providing comprehensive in-home support and services to families with children navigating cancer, we intimately understand the value of a strong support system. Our dedicated family care coordinators provide invaluable resources directly to families and caregivers, including access to physical, financial, and emotional options, so that you have the tools and support to face this unexpected challenge. If you or someone you know is dealing with a recent cancer diagnosis, don’t hesitate to reach out to us for tailored care and support. We are committed to building a future where all families with children going through cancer have access to the support and resources they need to focus on what matters most during this journey – caring for their loved ones.  

About the Author  

Danielle Loumagne is a Registered Nurse and Bone Marrow Transplant Certified Nurse who has worked in Pediatric Hematology/Oncology for over 13 years. As a Health Content Writer, she utilizes her experience to share deeper insights regarding what goes on within the hospital setting and strives to help her readers at Here to Serve feel more prepared and educated for their treatment and care.