Today's pediatric cancer treatments are the result of more than 70 years of research. This progress continues today in the groundbreaking clinical trials that seek to push cure rates higher and minimize the side effects of treatment. Clinical trials are an important part of treatment options for today's children with cancer — and are key to finding the most effective treatments for tomorrow's children with cancer.
That's why these families said “yes” to participating in a clinical trial.
Hear their voices. Meet their children.
Emily was 4 when she was diagnosed with high-risk neuroblastoma. Since participating in a clinical trial that involved back-to-back stem cell transplants, Emily celebrates being cancer-free by playing soccer and hanging out with her sister and two dogs.
Read Emily's story
In 2016, a large cancerous tumor on his hip left Jesus barely able to walk. When surgery wasn't an option, he enrolled in a clinical trial of a targeted drug that dramatically shrunk his tumor. Now, Jesus is able to live an active, pain-free life.
Read Jesus's story
Nothing could stop Allison from dancing or showing off her gymnastic skills — not even seven tumors on her brain and spine. She danced her way through a ground-breaking clinical trial that used precision medicine to match her tumors’ genetic mutation to a drug that targets that exact mutation.
Read Allison's story
Drs. Diller, Kieran and Janeway treated Emily, Allison and Jesus and led their innovative clinical trials. From confirming that two stem cell transplants are better than one to matching a tumor’s genetic abnormality to its most effective drug, find out how these clinical trials are shaping the future of cancer treatment.
Learn more about our clinical trials
Steven DuBois, MD, MS, director of the Advancing Childhood Cancer Therapies Clinic at Dana-Farber/Boston Children's Cancer and Blood Disorders Center, answers questions about clinical trials. Read the entire Q&A here.
Clinical trials are systematic, scientific investigations of new drugs or therapies for a specific disease. Through clinical trials, we have taken many fatal childhood cancers and turned them into diseases that now have cure rates of over 90 percent.
After decades of focusing on various chemotherapy agents and combinations to improve outcomes, much of today's focus is on precision medicine, immunotherapies and other novel approaches that we hope will deliver less toxic and more effective treatment than chemotherapy.
When we hear about a new drug or combination that has not yet been tested in children but shows promise in laboratory research or adult trials, we must first determine the best dose to use in children. What are the side effects? How is it absorbed into the blood? These are the goals of a Phase 1 trial.
Once a pediatric dose is established, we move to a Phase 2 trial. Here the aim is to study a similar group of patients treated with the same dose of the medicine and determine how likely their cancers are to respond to the new therapy.
If Phase 2 produces promising results, we then move into a Phase 3 trial for newly diagnosed patients to see if adding the new drug or adjusting the regimen upfront cures more patients and/or reduces toxicity. This is usually a large, randomized trial. A successful Phase 3 trial can help establish a new standard of care.
Whatever the phase of the clinical trial, our job is to provide families with the information they need to make an informed decision about their child's care, and once they have made a decision, to provide them with the physician, nursing, and psycho-social support they need to see them through this difficult time.
In 1947, Dr. Sidney Farber, the Boston Children's Hospital pathologist who founded Dana-Farber Cancer Institute, achieved the first successful remission of childhood leukemia using chemotherapy. In one of the great medical advances of the 20th century, many pediatric cancers became curable, thanks to the clinical research that built on this breakthrough. The revolution that Sidney Farber launched continues in today's clinical trials seeking even more effective ways to treat children with cancer.
Learn more about our history