Susan L. Cohn, M.D., chair of the St. Baldrick’s Scientific Advisory Committee and world renowned neuroblastoma expert, explains what neuroblastoma is and how St. Baldrick’s research is contributing to better outcomes for patients.
What is Neuroblastoma?
Neuroblastoma is a rare type of childhood cancer that develops in nerve tissue outside the central nervous system. Most commonly, it starts in the adrenal glands, located above the kidneys, but it can also form in nerve tissue near the spine. This cancer type primarily affects children under the age of 5 and is the most common extracranial solid tumor in children.
Despite its name, neuroblastoma is not a brain tumor. Every year, around 750 new cases are diagnosed in children under 15 in North America. The median age of diagnosis is just 2 years old, with over 95% of cases diagnosed in children under 10. The behavior varies widely—children with low- and intermediate-risk cases often respond well to treatment, but those with high-risk neuroblastoma face a much more challenging prognosis.
Signs and Symptoms of Neuroblastoma
The symptoms depend on where the tumor is located. Unfortunately, the signs are often similar to more common childhood illnesses, which can lead to delays in diagnosis. Key symptoms to watch for include:
- Abdominal mass or swelling
- Bone pain
- Bruising around the eyes
- Constipation
- Weakness or fatigue
- Irritability
- Fevers
- Anemia or bruising due to low blood counts
If your child experiences persistent or unusual symptoms, it’s important to consult a healthcare provider for further evaluation.
Neuroblastoma cell seen in nerve tissue.
Advances in Research and Treatment
Research in neuroblastoma treatment has advanced significantly over the past 30 years, particularly for high-risk neuroblastoma. More intensive therapies have improved survival rates, but these treatments often come with serious short- and long-term side effects, impacting the quality of life for survivors.
Key Research Highlights:
- Improving Treatment Outcomes for High-Risk Neuroblastoma
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- Recent studies supported by St. Baldrick’s Foundation and conducted by the Children’s Oncology Group have led to significant breakthroughs:
- Reduced Therapy for Intermediate-Risk Neuroblastoma: Using a biology- and response-based algorithm, researchers have safely reduced treatment intensity for some patients while maintaining positive outcomes. Learn more.
- Improved Survival with Tandem Stem Cell Transplant: A landmark study showed that children with high-risk neuroblastoma treated with tandem autologous stem cell transplants had better outcomes than those receiving a single transplant. Read the study.
- Recent studies supported by St. Baldrick’s Foundation and conducted by the Children’s Oncology Group have led to significant breakthroughs:
- Understanding Long-Term Effects of Treatment
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- Dr. Tara Henderson, MD, MPH, is leading a survivorship study called Late Effects After High-Risk Neuroblastoma (LEAHRN). This research focuses on the long-term impacts of modern therapies, including immunotherapy and high-dose chemotherapy, to better inform future treatments and follow-up care for survivors.
- Global Collaboration Through Data Sharing
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- St. Baldrick’s has established the International Neuroblastoma Risk Group (INRG) Data Commons, which houses data on more than 20,000 neuroblastoma patients worldwide. This resource has been instrumental in research that is reshaping risk classification and treatment approaches.
- Additionally, the Pediatric Cancer Data Commons (PCDC), led by Dr. Samuel Volchenboum, is integrating clinical, genomic, and imaging data from institutions around the globe to accelerate pediatric cancer research.
“Funding from St. Baldrick’s has been crucial in advancing these collaborative, data-driven efforts,” says Dr. Volchenboum. “By supporting the PCDC, St. Baldrick’s is helping researchers worldwide access and analyze critical data to improve outcomes for children with neuroblastoma.”
New Research and Future Directions
Ongoing studies continue to push the boundaries of neuroblastoma treatment and survivorship care. The LEAHRN study, for example, is enrolling 5-year survivors of high-risk neuroblastoma diagnosed after 2000 to assess the long-term effects of modern therapies. These findings will play a key role in designing future clinical trials and improving long-term care for survivors.
St. Baldrick’s Foundation is also leading efforts to democratize pediatric cancer data, enabling researchers to collaborate on a global scale. By combining resources and expertise, the goal is to improve treatment strategies, reduce toxicities, and ultimately increase survival rates for children with neuroblastoma.
New Research and Future Directions
Neuroblastoma research is advancing rapidly, providing hope for improved outcomes and a better quality of life for survivors. Thanks to global initiatives and organizations like St. Baldrick’s Foundation, scientists are uncovering innovative ways to fight this challenging pediatric cancer. Together, these efforts are paving the way for a brighter future for children.
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Learn about one of our recent Research Outcomes dealing with Neuroblastoma:
Research Outcomes: Unlocking Hope
