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Clinical Trials for Pediatric Cancer

What are clinical trials?

Clinical trials are carefully designed research studies to test an experimental treatment in a group of patients with a certain disease. An experimental treatment is one that has not been fully tested to be part of standard medical care. Often, these are newly developed medicines or existing medicines that are used in a different disease or in a different group of patients. Studies may also look at ways to make surgery, radiation, or other therapies more effective using other methods, devices or equipment. Sometimes, clinical trials test different doses, schedules, or combinations of treatments.

Graphic with statistic cited from the Children's Oncology Group that says, "Each year, more than 60% of young people with cancer are treated in a clinical research study."

Clinical trials are an important part of finding cures for children with cancer.

Scientists and doctors are always looking for better treatments with fewer side effects. Clinical trials are a very important part of finding cures and promoting long-term health and quality of life for children with cancer.

In childhood cancer, clinical trials can test ways to:

Doctor holding clipboard with patient

Sometimes, clinical trials test different doses, schedules, or combinations of treatments.

Clinical trials versus usual medical care

It is important to understand how clinical trials are different from usual medical care. The goal of research is to improve how diseases are treated in the future. A very specific research protocol or plan has to be followed for an entire group of patients. In standard medical care, the benefit of treatment is known, and doctors make care decisions based on individual patient needs. In a clinical trial, the treatment is experimental, and the benefits and side effects are not fully known. Sometimes, patients may receive usual medical care together with an experimental treatment. In other trials, patients may receive only the experimental therapy. The methods will vary based on the research protocol and the phase of the clinical trial.

Patient parents and doctors fighting pediatric cancer together

Theresa Beech, President of Because of Daniel, with Rosie Kaplan, MD, the Head of Tumor Microenvironment and Metastasis Section at the National Cancer Institute share how research inspired by her training as a Space Mission Design Engineer at NASA led to planning for new clinical trials.

Reviewed: June 2018