Neuroblastoma is a cancer of immature nerve cells that mostly affects children under five. It accounts for about 15% of childhood cancer deaths. Treatment depends on risk group — low-risk tumors may need no treatment at all, while high-risk cases require surgery, chemotherapy, stem cell transplant, immunotherapy, and radiation.
What's actually going on in research
Trials are testing new immunotherapy combinations, targeted drugs that exploit neuroblastoma's genetic vulnerabilities, cellular therapies including CAR T-cells and natural killer cells, and ways to make existing treatments less toxic. Research focuses heavily on high-risk disease and on preventing relapse, which remains common and often fatal.
GD2-targeted immunotherapy
Dinutuximab, an antibody targeting GD2 on neuroblastoma cells, is now standard for high-risk patients and has improved survival. Trials are testing newer GD2 antibodies with fewer side effects and combining them with other immune-boosting drugs.
Targeted therapy for ALK mutations
About 14% of neuroblastomas have ALK gene changes that can be targeted with drugs like lorlatinib. Trials are testing whether these drugs work better than chemotherapy for children whose tumors have these mutations.
Cellular therapy
CAR T-cells and natural killer cells engineered to attack GD2 are in early trials. These living therapies might reach cancer cells that hide from antibodies and chemotherapy.
What to know before you search
Eligibility depends on risk group, whether the tumor is newly diagnosed or relapsed, prior treatments received, and specific genetic features of the tumor like MYCN amplification or ALK mutations.
What types of trials are currently open
- Treatment trials — Testing new drug combinations, immunotherapies, or targeted drugs to improve cure rates for high-risk neuroblastoma or reduce treatment intensity for lower-risk disease.
- Relapse trials — Testing treatments for neuroblastoma that has come back after initial therapy, including novel immunotherapies and cellular therapies.
- Stem cell transplant trials — Studies comparing different transplant approaches or testing whether some children can skip transplant.
- Supportive care trials — Testing ways to reduce pain, neuropathy, hearing loss, and other side effects of neuroblastoma treatment.
- Observational studies — Following children with neuroblastoma to understand how tumors behave, which genetic features predict outcomes, and long-term effects of treatment.
Recently added Neuroblastoma trials
Naxitamab Enabled Transplant-Free Study in High-Risk Neuroblastoma
A prospective, multicenter, clinical trial in participants with high-risk neuroblastoma that have completed induction therapy, evaluating the feasibility, efficacy, and toxicity of using anti-GD2 chemo-immunotherapy (naxitamab/irinotecan/temozolomide/GM-CSF) as consolidation followed by standard radiation, then standard anti-GD2 immunotherapy, and 2 years of DFMO maintenance.
Prospective Multicenter Study of the Genomic, Transcriptomic, and Biological Profile of Patients With Neuroblastoma
This is an investigator-initiated, multicenter, prospective, non-profit study. Patients who provide informed consent to participate will be asked to provide a peripheral blood sample, a fresh tumor tissue sample (obtained by biopsy), and/or a bone marrow sample. All patients with neuroblastoma at diagnosis, with relapsed disease, or with refractory disease may participate, provided that the diagnosis and disease status are established by the treating physician in accordance with national clinical practice and by the participating study center. Enrolled patients will be reported and registered by means of a paper-based notification form sent by email to the Coordinating Center, which will assign a unique identification code to each patient. At the time of notification, clinical and treatment-related data will be collected using a paper-based Case Report Form (CRF). Biological material (peripheral blood, bone marrow, and/or tumor biopsy samples) must be centralized at the Laboratory of Experimental Oncology Therapies, IRCCS Istituto Giannina Gaslini, Genoa, Italy, in accordance with the procedures described in the study protocol. Samples will be processed and analyzed as specified in the protocol. The results of the initial analysis performed at CEINGE, Naples, will be communicated by the CEINGE Laboratory to the Coordinating Center. A multidisciplinary meeting will then be held with a panel of experts, including oncologists, biologists, pharmacists, and infectious disease specialists. Following this meeting, an individual report will be prepared for each patient. The report will include the results of Whole Exome Sequencing (WES) or Cancer Gene Panel (CGP) analysis performed on the patient's DNA, together with any potential therapeutic recommendations based on the molecular alterations identified. The report will be sent to the physician responsible for the individual patient. For patients who provide additional consent by signing the appropriate informed consent form, biological material may also be used for the following purposes: i) Immunophenotyping, with particular focus on GD2 and B7-H3 expression in tumor biopsy samples and in neuroblastoma cells infiltrating the bone marrow; ii) High-throughput screening of libraries comprising readily available anticancer drugs; iii) Generation of in vitro and in vivo tumor models, including primary cell lines and spheroids in vitro, and murine models such as Patient-Derived Xenografts (PDX) and Cell Patient-Derived Xenografts (CPDX) at the Coordinating Center, as well as zebrafish models at San Martino-IST, Genoa, and/or Città della Speranza, Padua.
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