Neuroblastoma is a childhood cancer that develops from immature nerve cells, most often appearing in the adrenal glands or along the spine. It's the most common cancer in infants and accounts for about 6% of childhood cancers. Treatment depends heavily on risk group — some tumors disappear on their own while high-risk neuroblastoma requires intensive chemotherapy, surgery, radiation, immunotherapy, and stem cell transplant.
What's actually going on in research
Trials are testing new immunotherapy combinations, targeted drugs for ALK mutations and other genetic changes, radioligand therapies that deliver radiation directly to tumor cells, and ways to make standard chemotherapy more effective while reducing long-term harm. Researchers are also studying how to identify which low-risk tumors can be safely watched rather than treated.
Immunotherapy combinations
Dinutuximab, an antibody targeting the GD2 protein on neuroblastoma cells, is now standard for high-risk disease. Trials are testing it with checkpoint inhibitors and cellular therapies to improve responses.
Targeted therapies
About 15% of neuroblastomas have ALK gene mutations. Drugs like lorlatinib and ensartinib that block ALK are showing activity in early studies and may offer less toxic treatment than chemotherapy alone.
Radioligand therapy
Drugs that attach radioactive particles to molecules that seek out neuroblastoma cells are being tested. MIBG therapy is already used in some centers, and newer agents are in trials.
What to know before you search
Eligibility typically depends on risk group (low, intermediate, or high), prior treatments, tumor genetics including MYCN amplification and ALK mutations, and whether the disease is newly diagnosed or relapsed.
What types of trials are currently open
- Immunotherapy trials — Testing antibodies, cellular therapies, and combinations that help the immune system attack neuroblastoma cells expressing GD2 or other targets.
- Targeted therapy trials — Testing drugs that block specific genetic changes in the tumor, such as ALK inhibitors for ALK-mutated neuroblastoma.
- Chemotherapy trials — Testing new drug combinations or schedules to improve cure rates while reducing side effects and late complications.
- Radiation trials — Testing radioligand therapies and new radiation approaches to kill tumor cells while sparing normal tissue.
- Observation studies — Following children with low-risk tumors to learn which can be safely watched without immediate treatment.
Recently added Neuroblastoma trials
Haploidentical Donor Cytokine-Induced Memory-Like Natural Killer Cells (CIML-NK) for Relapsed & Refractory Neuroblastoma
The goal of this study is to demonstrate that cytokine-induced memory-like natural killer cells (CIML-NK cells) can be generated from donor cells and infused safely into patients with relapsed or refractory neuroblastoma during dinutuximab-based therapy.
Phase I Study of Becotatug Vedotin for Safety and Efficacy in EGFR-Positive Pediatric Relapsed/Refractory or Metastatic Solid Tumors
There is a significant unmet medical need for effective therapies for pediatric relapsed/refractory solid tumors. EGFR is highly and stably expressed in multiple pediatric solid tumor subtypes, and adult Phase I data of Becotatug Vedotin demonstrated a manageable safety profile and promising antitumor activity in EGFR-positive advanced solid tumors.This is a multicenter, non-randomized, single-arm, open-label Phase I clinical trial sponsored by Sun Yat-sen University Cancer Center (SYSUCC). The trial evaluates the safety, tolerability, pharmacokinetics (PK), immunogenicity, and preliminary efficacy of Becotatug Vedotin-an EGFR-targeted antibody-drug conjugate (ADC)-in pediatric patients with EGFR-positive relapsed/refractory or metastatic solid tumors.
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