Childhood Cancers Pediatric Research and Clinical Trials

The team at Dana-Farber/Boston Children’s Cancer and Blood Disorders Center improves care in many ways. Some changes come from scientific research into childhood cancer and blood disorders. Understanding diseases deeply—even at the cellular or molecular level—leads to new drugs and therapies. Other improvements come from moments spent at the bedside, when doctors and nurses see opportunities to improve current treatment methods. Learn more at Dana-Farber/Boston Children’s.

Clinical trials

Dana-Farber/Boston Children's Cancer and Blood Disorders Center participates the Children's Oncology Group (COG), a consortium of cancer treatment centers across the United States, Canada and other countries that conduct studies of nearly every kind of pediatric cancer. Our participation in the COG gives children with cancer unparalleled access to the newest clinical trials. We also participate in several other consortia, such as Therapeutic Advances in Childhood Leukemia (TACL), Pediatric Oncology Experimental Therapeutics Investigators' Consortium (POETIC), New Approaches to Neuroblastoma Therapy (NANT) and the DFCI Pediatric Leukemia Consortium.

If your child has progressive or recurrent disease, she may be eligible for a number of experimental therapies available through COG or other groups, or through one of our independent clinical investigators.

Read about our innovative research on the Cancer Care Center page.

Frequently Asked Questions (FAQ) about Clinical Trials

Q: What is a clinical trial?

A: A clinical trial is a research study that uses volunteers to test new methods of screening, prevention, diagnosis or treatment of a disease. These studies are designed to answer important questions about cancer diagnosis, treatment and prevention. If enrolling in a clinical trial is an option, your child's primary oncologist will explain what the treatment involves, its goals and objectives, and answer any questions you have about enrolling on the study.

Q: What is the purpose of a clinical trial?

A: Most clinical trials are designed to ask a question that hasn't been answered yet such as, "Does a certain medicine cure a disease, or make some of the symptoms go away?" The results help us learn which treatments are currently most effective for certain diseases.

For cancer in particular, medical research is very important. Clinical trials test the efficacy of new medications, evaluate new approaches designed to minimize long-term effects, and provide treatments to preserve quality of life during and after treatment.

Q: Why should my child enroll in a clinical trial?

A: Most pediatric cancers are treated at major medical institutions, which use state-of-the-art treatments. Many of these treatments are based on clinical trials. Clinical trials rely on scientific findings to investigate the newest medications and most advanced procedures to treat childhood cancer.

Doctors recommend what they believe will be the best treatment. In many cases, your child may receive the best care possible by following a plan outlined in a clinical trial.

Q: What if I want to remove my child from a clinical trial?

A: Participation in clinical trials is completely voluntary. Before we begin treating your child in the trial, we will fully explain all parts of the treatment plan and fill out formal consent forms. You may remove your child from the clinical trial at any time.

Q: Are there long-term benefits of clinical trials?

A: Clinical trials are important in the long-term treatment of childhood cancer, and discoveries made years ago through clinical trials are now the standard of care for many of the curable cancers. By participating in a trial, your child will help us develop improved treatments for future children with the same condition.

Q: How can I obtain additional information?

A: To help patients and families understand more about clinical trials, Dana-Farber Cancer Institute produced an informational video, called "Entering a Clinical Trial: is it right for you?" The video explains how to evaluate, enroll and participate in a cancer clinical trial. If the video does not answer your question, our doctors and nurses are available to address your concerns.

Types of clinical trials

How are clinical trials categorized?

Clinical trials are categorized as Phase III, II or I. Classification depends on the purpose of the study and determines the total number of patients who may receive treatment on that clinical trial. Newly-diagnosed patients are often treated using a Phase III trial.

Phase III studies compare the new treatment to a standard (current, state-of-the-art) treatment. At the beginning of the study, doctors don't know if the new treatment is better than the standard treatment. Phase III trials typically enroll hundreds of patients, and are carried out in treatment centers nationwide. If a drug successfully completes the Phase III level of testing, the pharmaceutical company that makes it can apply for FDA approval for this use.

In cases when the standard of care or Phase III treatment is not effective, your child's health care team may recommend a Phase II trial.

Phase II studies investigate whether the new treatment works for a specific type of cancer. They also continue to evaluate the safety of the treatment. It is important to realize that it is not yet known whether a Phase II drug will be effective against your cancer. Phase II studies generally enroll no more than 100 people.

If the standard of care, or a Phase III or Phase II treatment does not deliver positive results, your child's primary oncologist may suggest enrolling your child on a Phase I study.

Phase I studies primarily evaluate:

  • the safety of a new drug
  • how much and how often the drug needs to be given
  • the side effects of the new drug are

This is the first level of testing for a new drug or a known drug given in a new way, but is generally based on laboratory testing or the safe dose of a similar drug given to other people. Very few patients are enrolled on Phase I trials and they are available only through select programs.

Two important aspects of Phase I trials are that:

  • Medications used in pediatric cancer clinical trials have usually been tested in adult trials before they are used in children. However, because your child might be one of the first pediatric patients in the world to receive the new drug, the side effects in children may not be known.
  • Although this drug has shown promise in the laboratory or in adults, it is not known if it will work against your child’s cancer.

If our physicians recommend enrolling your child on any clinical trial, they will explain its purpose, benefits, possible side effects and answer any questions you or your child have. You will also have to sign a consent form before any treatment begins.

Learn more about pediatric cancer clinical trials

For many children with rare or hard-to-treat conditions, clinical trials provide new options.