Clinical Trials for Pediatric Cancer at MSK Kids

Clinical Trials for Pediatric Cancer at MSK Kids

MSK Medical Oncologist Nai-Kong Cheung, MD, PhD

MSK neuroblastoma specialist Nai-Kong Cheung is one of many researchers studying better ways to treat pediatric cancers and achieve long-term survival.

Finding New Hope for Pediatric Cancers, One Child at a Time

Families come to MSK Kids from across the country — and the world — for exceptional care. Many also come for something more: The opportunity for their child to participate in a clinical trial, also known as a research study, of a new treatment. Thirty-five percent of young people treated at MSK take part in a clinical trial at some point during their care here.

Scientists at MSK Kids are conducting more than 100 clinical trials to better understand childhood cancers and blood disorders and find new ways to treat them. Clinical trials are the reason why the cure rate for childhood cancers has risen from practically zero in the 1960s to more than 80 percent today.

We know that understanding clinical trials can be challenging. We’ve created these pages to help clarify the process so you can decide if a clinical trial is right for your family. Your doctor will let you know if your child is eligible to participate in one.

What is a clinical trial?

Did you know that nearly every cancer treatment used today exists because it was first studied in a clinical trial? Today’s clinical trials are leading to the cancer treatments of tomorrow.

There are different types of clinical trials. Some offer a chance to try a promising new treatment that hasn’t yet been used in children with cancer. Some offer hope when standard treatments aren’t working. Others use a child’s tumor sample to try to better understand childhood cancer. And sometimes a clinical trial looks at a treatment that is just as effective as another but may have fewer side effects.

Why Clinical Trials Are So Important

Breakthroughs in cancer treatment don’t happen overnight. They evolve after years of research in the laboratory, then in small groups of patients, and then in larger groups of patients. Sometimes a clinical trial shows that a drug doesn’t work as well as expected. However, along the way, the researchers learn something new that helps them understand childhood cancer more. If your child’s doctor recommends a clinical trial, it is because we think your child would benefit from a new cancer treatment that we hope is better than current treatments.

From Bench to Bedside: Getting New Treatments to Children Faster

MSK has a robust Early Drug Development Service. Their mission is to speed up the development of new treatments at their earliest stage: phase I clinical trials. These studies evaluate the safety of a new treatment and determine the best dose for patients. Through the relationship between MSK Kids and the Early Drug Development Service, we can get new drugs to children from the lab to a child’s bedside faster.

Your Child’s Safety Is Our #1 Priority

We understand that you may feel anxious or scared about your child receiving a treatment that hasn’t been tried before. You can rest assured that lots of research takes place in the laboratory, and sometimes in adult patients, before we begin evaluating new treatments in young patients. Before enrolling your child in one of these important studies, we take these steps:

  • First, a dedicated Pediatric Protocol Steering Committee (PSC) reviews each pediatric clinical trial to make sure that the trial design is safe and appropriate for children. For example, the PSC ensures that imaging exams, blood draws, and clinical tests specifically consider children’s needs.
  • Second, our Research Council meets to determine if the trial is scientifically appropriate — in other words, they ensure the trial has been designed to provide important and usable knowledge.
  • Once the Research Council gives its approval, our Institutional Review Board (IRB) reviews the trial to make sure that patients’ health, safety, and rights are protected. The IRB includes representatives from the community in addition to different departments at MSK. This provides a variety of viewpoints before the trial is approved.
  • Finally, your child’s doctor and the research team match children and clinical trials together. They do so very carefully, following a list of eligibility criteria that determine which children are and are not eligible to participate in a trial. We aim to choose the clinical trial that is best for each patient, as well as the most appropriate patients for each clinical trial. If your child is not chosen for a clinical trial, it is for his or her benefit. We do all we can to match your child with the safest, most effective cancer therapy options.

Making the Decision to Enroll in a Clinical Trial

If your child’s doctor tells you about a clinical trial that may benefit him or her, the decision to participate is entirely up to you and your family. No one will ever force you to enroll your child in a clinical trial. We do all we can to help you understand the trial so you can make an informed decision. Here’s how it works:

  • A member of our research team will explain everything you need to know about the trial: its goals, what treatment your child will receive, how the treatment is given, how long your child will be in the trial, and possible side effects. They will also discuss the alternative treatments available should you decide not to participate in the trial. This process is called informed consent.
  • Participation is completely voluntary, and if you change your mind, it’s OK to withdraw from the trial at any time.
  • Your consideration to participate in a clinical trial is very much appreciated. The knowledge we gain will benefit families around the world.

The new drug larotrectinib (Vitrakvi®) changed Rihanna’s life. Learn about one family’s clinical trial experience.