The Pediatric Early Phase-Clinical Trial Network (PEP-CTN): How Cookies for Kids’ Cancer is Helping to Pave the Way for Breakthroughs in Pediatric Cancer Treatment

Childhood Cancer Awareness | Childhood Cancer Research | Clinical Trials | Pediatric Early Phase-Clinical Trial Network

Did you know that there are more than a dozen major types of childhood cancers, and each is as unique as the child fighting it? Pediatric cancer is not a singular disease, remains the leading cause of death by disease in children in the U.S. and two thirds of childhood cancer survivors suffer long-term side effects as a result of the treatments they receive.

The Children's Oncology Group (COG), a leading research cooperative dedicated to improving the survival and quality of life for children and adolescents with cancer, plays a crucial role in advancing pediatric cancer care. COG has more than 100 active clinical trials open at any given time. These trials include front-line treatment for many types of childhood cancers, studies aimed at determining the underlying biology of these diseases, and trials involving new and emerging treatments, supportive care, and survivorship.

COG has treated more children with cancer than any other organization in history and has been responsible for many of the improvements in treatment and cure rates over the past 40 years. One of COG's most significant initiatives is the Pediatric Early Phase-Clinical Trial Network (PEP-CTN). This network is a collaborative effort involving 42 premier pediatric oncology centers across the United States, Canada, and Australia. It is designed to accelerate the development of novel therapies for children with cancer through efficient and innovative clinical trials.

Cookies for Kids’ Cancer is the largest philanthropic supporter of PEP-CTN which, simply put, makes Phase I treatments available for more kids. We match dollar for dollar the funding the National Cancer Institute (NCI) provides for EVERY CHILD enrolled at the 42 participating core and non-core centers which are located across the country. For seven years of our funding, we actually doubled the amount the NCI funds.

The Importance of Phase I Clinical Trials in Pediatric Cancer

The PEP-CTN prioritizes the development of treatments for difficult-to-treat childhood cancers, where treatment options are often limited. Phase I clinical trials are the first step in bringing new cancer treatments to patients. These trials aim to:

Determine safe dosage: The primary objective is to find the highest dose of a new treatment that can be given to a patient without causing unacceptable side effects.

Assess a treatment's effects on the body: Phase I trials carefully monitor how the treatment is absorbed, distributed, metabolized, and excreted from the body. This information is crucial for understanding how the therapy works and for designing future studies.

Identify potential side effects: By closely monitoring patients, researchers can identify and characterize any side effects associated with a therapy.

The Role of the PEP-CTN - Kids Don’t Have Time to Wait

At Cookies for Kids’ Cancer we aim to fund the most promising research that will make it from a lab to a child’s bedside in the shortest amount of time possible. Kids don’t have time to wait.

The PEP-CTN plays a pivotal role in conducting these critical early-phase trials by leveraging a standardized approach which reduces the time it takes to bring new therapies to children. By fostering strong collaborations between researchers, clinicians, and pharmaceutical companies, they’re able to facilitate the rapid development and testing of promising new therapies while tailoring treatments to the characteristics of each child's cancer. The network includes a diverse range of institutions with expertise in various aspects of pediatric oncology, including medical oncology, surgery, radiation oncology, and supportive care.

Your Dollars at Work - PEP-CTN Achievements

With your help, since 2012, support from Cookies for Kids’ Cancer has gone to more than 40 institutions for nearly 1,000 children who enrolled in early phase studies. The PEP-CTN has already made significant contributions to the field of pediatric oncology, including:

Developing novel targeted therapies: The network has played a key role in the development of targeted therapies, which aim to specifically attack cancer cells while minimizing harm to healthy tissues. This includes therapies that target specific genetic mutations and pathways involved in cancer development.

Improving the treatment of difficult to treat cancers: The PEP-CTN has focused on developing treatments for difficult-to-treat childhood cancers, such as neuroblastoma, rhabdomyosarcoma, and medulloblastoma, where treatment options are often limited.

Advancing immunotherapy: The network has been at the forefront of immunotherapy research in pediatric oncology, exploring the use of immune system-boosting therapies to fight cancer.

The Importance of Philanthropic Investment

Awareness, support and above all, philanthropic investment are crucial for the success of the PEP-CTN and the continued advancement of pediatric cancer research. Together, we can play a vital role in improving the outcomes for children with cancer.

At Cookies for Kids’ Cancer, our vision is that no family is ever told, “We have run out of options.” The Children's Oncology Group's PEP-CTN represents significant advancement in the fight against pediatric cancer and brings us as many steps closer to realizing that vision. By fostering collaboration, prioritizing innovation, and focusing on the needs of children, the network is paving the way for a future where all children with cancer can have access to the best possible treatments and achieve long-term survival and optimal quality of life.

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Institutions* that have received funding from Cookies for Kids’ Cancer
in conjunction with PEP-CTN include:

*Twenty-one sites are designated as core sites located within the US and participate in all PEP-CTN trials, and 21 non-core member sites in the U.S., Canada and Australia that participate in selected trials. All PEP-CTN sites are selected through a rigorous peer review process.