Neuroblastoma Treatment and Clinical Trials

Neuroblastoma treatment advancements through clinical trials

Over the years, many research studies (also known as clinical trials) have been conducted to understand the best ways to treat neuroblastoma. From these clinical trials, oncologists have established current standards for neuroblastoma treatment. Find more information on the current neuroblastoma treatment options.

Clinical trials may provide your child the opportunity to receive cutting-edge treatment. Children with neuroblastoma are living longer today because of past clinical trials that showed new treatments were safe and effective.

Evolution through Clinical Trials

Learn why clinical trials are important in neuroblastoma and why they may be an option for your child.

Evolution of high-risk neuroblastoma treatment

For example, in patients with high-risk neuroblastoma, the survival at diagnosis is reported to be about 50%.¹ Clinical trials have shown that adding additional agents to the standard therapy improves survival in patients with treatment-responsive disease. The figure below highlights the evolution of high-risk neuroblastoma treatment through various studies. Of note, these studies represent specific patient populations, and these results cannot be extrapolated as a whole. It’s important to discuss with your child’s doctor what their specific prognosis and treatment will be.

Evolution of high-risk neuroblastoma treatment

To find out more about the clinical trials that inform the figure above, you can read the articles using the links below.

References: 1. DuBois SG, Bagatell R. Improving outcomes in children with high-risk neuroblastoma: the role of randomized trials. J Clin Oncol. 2021. doi:10.1200/JCO.21.01066. 2. Matthay KK, Villablanca JG, Seeger RC, et al; for the Children's Cancer Group. Treatment of high-risk neuroblastoma with intensive chemotherapy, radiotherapy, autologous bone marrow transplantation, and 13-cis-retinoic acid. N Engl J Med. 1999;341(16):1165-1173. 3. Park JR, Kreissman SG, London WB, et al. Effect of tandem autologous stem cell transplant vs single transplant on event-free survival in patients with high-risk neuroblastoma: a randomized clinical trial. JAMA. 2019;322(8):746-755.

Introduction to clinical trials

Clinical trials are research studies that test new ways to improve treatments in a group of people with a certain disease. In neuroblastoma, clinical trials often focus on new treatments to see if they are safer or more effective than what is currently available.

Clinical trials follow strict standards and are completely voluntary

Clinical trials follow strict rules set by the Food and Drug Administration (FDA). These rules make sure that clinical trials are as safe as possible
Participation in clinical trials is completely voluntary and you may choose to withdraw your child from a clinical trial at any time if you change your mind

Clinical trials are broken out by phases

Neuroblastoma clinical trials happen in phases. Goals for each trial depend on its phase and your child’s diagnosis.

The goal for a specific clinical trial will depend on the phase it's in.

Phase 1: The purpose of a phase 1 clinical trial is to find a safe dose for the treatment being tested
Phase 2: The purpose of a phase 2 clinical trial is to see how the treatment affects the body and fights cancer
Phase 3: A phase 3 clinical trial generally tests the standard treatment against a new alternative treatment. The goal for this type of trial is to see if the new alternative treatment helps to increase cure rates, decrease side effects, or limit late effects of treatment

Clinical trials follow strict standards and are completely voluntary

Clinical trials may vary based on location

Different hospitals are associated with different research organizations, so some clinical trials may only be available at certain hospitals. Learn more about the different research groups in neuroblastoma.

Find detailed information on the different phases of a clinical trial.

The decision to explore clinical trials or choose the current standard treatment is one that should be made along with your child’s oncologist. Because every neuroblastoma diagnosis is unique, your child’s oncologist can help you find the treatment best tailored to meet the needs of your child.