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Neuroblastoma |
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The information provided in this review is intended for general informational purposes only. It is
not intended to be a substitute for professional medical advice, diagnosis, or treatment.
Always seek the advice of your physician or other qualified healthcare provider with any
questions you may have regarding a medical condition. Feel free to share this information page
with them.
The inclusion of clinical trials in this review does not imply endorsement or recommendation by
MyChild’sCancer. The decision to participate in a clinical trial should be made in consultation
with your healthcare provider and after carefully considering the risks and potential benefits
associated with the trial.
Childhood Cancer Name
Neuroblastoma
Biology
Neuroblastoma is a disease in which malignant (cancer) cells form in neuroblasts (immature
nerve tissue) in the adrenal glands, neck, chest, or spinal cord.
It is a type of childhood cancer that originates in immature nerve cells, typically in the adrenal
glands but can also occur in other areas of the body such as the abdomen, chest, or pelvis. It
predominantly affects infants and young children and is one of the most common solid tumors
found in this age group.
The symptoms of neuroblastoma can vary widely depending on the size and location of the
tumor. Common signs may include abdominal swelling, pain, a lump or mass, unexplained
weight loss, fatigue, and changes in bowel or bladder habits. However, some cases may be
asymptomatic and only discovered incidentally during routine medical exams or imaging tests.
Characteristics
- Usually begins in infancy; most often diagnosed between birth and age five.
- May be found incidentally on prenatal ultrasound.
- By diagnosis, it has often metastasized to lymph nodes, bone, bone marrow, liver, and
skin; in adolescents, also the lungs and brain. - Sometimes caused by inherited gene mutations.
- In babies <1.5 years old, prognosis is significantly better.
Cancer Localization
Nervous System, Other
Childhood Cancer Diagnostic Method
Biopsy analysis, Blood test
Neuroblastoma Risk Groups
1. Low-Risk Neuroblastoma: Localized tumors with favorable biological features. Often
regress spontaneously or respond well to surgery/observation.
2. Intermediate-Risk Neuroblastoma: May have regional spread but lack high-risk
markers. Treatment involves surgery, chemotherapy, and sometimes radiation.
3. High-Risk Neuroblastoma: Aggressive, often metastatic tumors. Require intensive
multimodal treatment.
4. Relapsed Neuroblastoma: Particularly challenging, requiring personalized combinations
of therapies.
Treatment Options
Treatment for neuroblastoma depends on several factors including the child’s age, the risk-
stratification, whether it has spread to other parts of the body, and the presence of any genetic
mutations. The choice of treatment is often tailored to each individual case to achieve the best
possible outcome while minimizing side effects. Families should consult with pediatric
oncologists and multidisciplinary teams specializing in neuroblastoma to determine the most
appropriate treatment approach for their child.
Below are common treatment modalities used in the management of neuroblastoma:
Surgery:
Surgery is often the first step in treating low or intermediate-risk neuroblastoma, aiming to
remove as much of the tumor as possible.
Chemotherapy:
Chemotherapy is a type of cancer treatment that uses drugs to destroy cancer cells. These drugs
work by targeting rapidly dividing cells, which are characteristic of cancer cells. Chemotherapy
can be given in various forms, including orally (as pills or capsules) or intravenously (through a
vein). In the context of neuroblastoma treatment, chemotherapy is often administered in cycles
over several months. The specific drugs used, and the duration of treatment depend on factors
such as the child’s age, the stage and aggressiveness of the cancer, and whether the tumor has
spread to other parts of the body.
Chemotherapy may be used before surgery (neoadjuvant chemotherapy) to shrink the tumor and
make it easier to remove, or after surgery (adjuvant chemotherapy) to kill any remaining cancer
cells and reduce the risk of recurrence. In cases where the tumor cannot be completely removed
with surgery, chemotherapy may be used to help control the growth of the cancer.
Radiation Therapy:
Radiation therapy uses high-energy beams to target and kill cancer cells. It may be used before or
after surgery, or in cases where the tumor cannot be completely removed. Advanced techniques
in radiation therapy, such as intensity-modulated radiation therapy (IMRT), proton therapy, and
stereotactic radiosurgery, allow for more precise delivery of radiation to tumor sites while
minimizing damage to surrounding healthy tissues.
Stem Cell Transplant:
High-dose chemotherapy can damage the bone marrow, where blood cells are made. Stem cell
transplant, also known as bone marrow transplant, may be used to replace damaged cells.
Immunotherapy:
Immunotherapy is a type of treatment that helps the body’s immune system fight cancer. For
neuroblastoma, commonly used monoclonal antibodies include Dinutuximab (Unituxin),
Dinutuximab beta (Qarziba), and Naxitamab (Danielza).
Differentiated Therapies:
Differentiated therapies aim to induce the maturation or differentiation of neuroblastoma cells
into more benign, less aggressive forms. One of the primary agents used in differentiated
therapies is retinoic acid, a derivative of vitamin A.
Targeted Therapies:
Targeted therapies focus on specific molecular targets or pathways involved in the growth and
progression of neuroblastoma. Examples include drugs targeting ALK (anaplastic lymphoma
kinase) mutations, such as crizotinib and lorlatinib, as well as other molecularly targeted agents
and small molecule inhibitors.
Treatment Protocols
The different approaches for neuroblastoma treatment:
SIOPEN (International Society of Pediatric Oncology Europe Neuroblastoma): A
collaborative network of pediatric oncologists, researchers, and healthcare professionals
dedicated to advancing the understanding and treatment of neuroblastoma. SIOPEN aims to
improve outcomes for children with neuroblastoma by conducting clinical trials, sharing knowledge and expertise, and promoting collaboration among institutions and healthcare providers across Europe and beyond.
For Low-Risk Neuroblastoma:
Treatment often involves observation or minimal intervention, as many low-risk tumors may
spontaneously regress without treatment. In cases where intervention is necessary, treatment may
include surgery to remove the tumor. SIOPEN protocols may emphasize surveillance and careful
monitoring to detect any signs of disease progression or recurrence.
For High-Risk Neuroblastoma:
SIOPEN protocols aim to maximize treatment effectiveness while minimizing the risk of
recurrence and long-term side effects. These protocols often involve multimodal approaches that
include surgery, chemotherapy, radiation therapy, autologous stem cell transplant, and
immunotherapy. Multimodal approaches target different aspects of the disease, including the
primary tumor, metastatic sites, and residual cancer cells.
COG (Children’s Oncology Group) Protocols:
Conducts clinical trials primarily in North America (along with Canada and Australia) and
develops treatment protocols for pediatric cancers, including neuroblastoma. COG protocols
often incorporate a combination of chemotherapy, surgery, radiation therapy, stem cell transplant,
and immunotherapy.
NANT (New Approaches to Neuroblastoma Therapy) Consortium:
A collaborative group of researchers and institutions focused on developing novel therapies for
patients with relapsed or refractory neuroblastoma. NANT conducts clinical trials to evaluate
new drugs, targeted therapies, and immunotherapies.
Emerging and Expiremental Techniques
Emerging Technique: Liquid Biopsy for Disease Monitoring
High-risk neuroblastoma presents challenges in disease monitoring and treatment due to tumor
heterogeneity and difficult-to-access tumor locations.
Liquid biopsy emerges as a promising non-
invasive technique for monitoring disease progression and detecting therapeutic targets.
Researchers at the Children’s Hospital of Philadelphia (CHOP) investigated the use of liquid biopsies to monitor tumor changes in high-risk neuroblastoma patients.
Liquid biopsy involves analyzing fragments of tumor DNA called circulating tumor DNA (ctDNA) in the blood to detect mutations, even in difficult-to-access tumor locations.
Serial ctDNA profiling provides real-time genomic data, enabling personalized treatment
approaches and improved disease surveillance.
Benefits of Liquid Biopsy in Neuroblastoma:
- Offers insights into genetic characteristics of the tumor, aiding in treatment decision-making.
- Facilitates monitoring of disease progression and recurrence, potentially guiding adjustments in therapy.
- Particularly beneficial in cases where traditional tissue biopsy is challenging, such as in young children or tumors located in difficult-to-access areas.
Collaborative Efforts:
MyChild’sCancer collaborates with pediatric experts like Prof. Yaël P. Mossé from CHOP’s Cancer Center to advance second opinion programs and promote innovative and individualized therapeutic strategies. Through this collaboration, families were introduced to the possibility of undergoing a liquid biopsy, influencing treatment strategies in real-world cases.
Emerging Therapy – CAR-T Cell Therapy for Neuroblastoma
Researchers at CHOP have developed innovative chimeric antigen receptor T (CAR-T) therapy that reprograms a child’s own immune T cells to attack neuroblastoma cells.
In particular, the newer GPC2-CAR-T trial has demonstrated early signs of safety and anti-tumor activity in children with relapsed or refractory neuroblastoma.
Unlike earlier GD2-CAR-T trials, the GPC2 approach does not require strict disease-burden criteria, making it accessible to a broader range of patients.
Benefits of CAR-T Therapy in Neuroblastoma:
- Targets tumor cells more precisely, potentially sparing healthy tissues.
- May offer options for children with relapsed or treatment-resistant disease.
- Demonstrates encouraging early clinical activity with a favorable safety profile in Phase 1 studies.
- Provides access to cutting-edge immunotherapy research, with the potential to expand options for relapsed/refractory cases
These recommendations have now been echoed across several independent consultations with international experts, including Dr. Yaël Mossé at CHOP, underscoring their growing importance in the treatment landscape for high-risk or relapsed neuroblastoma. Families interested in these options should discuss them with their oncology team and consider seeking a specialized second opinion.
Parent Tips and Testimonials
- Babies <1.5 years old often have a very good prognosis; disease behaves differently than
in older children. - Harvest bone marrow and stem cells as early as possible, as relapse is common and future
collection may not be possible. - Always prepare for relapse as it can occur suddenly.
- Stay updated on new clinical trials, even during remission.
- MSKCC in New York offers advanced antibody therapies (3F8). Check if similar options
are available locally.
Link to Testimonial:
