Neuroblastoma edition · Most recent neuroblastoma conclusions
Last updatedMonday, November 2, 20266:43 AM ET
Today's headlineFirst trial of CAR-T cells aimed at L1CAM finds no tumor responses in 22 children (Sept 15). In Japan, a small randomized trial finds a different anti-GD2 immunotherapy mix comparable to the U.S. standard (July 23).
Most children with low- or intermediate-risk neuroblastoma do well. For high-risk disease, the search is focused on keeping it from coming back.
Cure Checker's conclusion · Oct 2026
Most recent cure information
There is no single cure for neuroblastoma, and how a child does depends mostly on their risk group. About 98% of children with low-risk disease and about 95% with intermediate-risk disease are alive five years later, often after surgery alone or modest chemotherapy. In some babies the tumor shrinks away on its own. High-risk neuroblastoma is much harder: even with very intensive treatment, about 6 in 10 children are alive at five years, and the cancer coming back is the main danger. Our view: the biggest recent gains come from immune treatments aimed at GD2, a sugar-like marker on the tumor surface, and from new ways to keep the cancer from returning.
Anti-GD2 antibodies: now part of standard care.In a 226-child randomized trial, adding the antibody dinutuximab after transplant raised 2-year event-free survival from 46% to 66%. Europe uses a close relative, dinutuximab beta.
Keeping it away: a new pill, with debate.The FDA approved eflornithine (Iwilfin) in December 2023 to lower the risk of relapse, based on a comparison with past patients rather than a randomized trial. European regulators were not convinced, and the company withdrew its EU application in July 2025.
CAR-T cells aimed at GD2: strong but still experimental.In final results from Rome (2025), 66% of children with relapsed or resistant disease responded. In children with little disease left, 68% were alive at five years. It is available only in trials.
Matching treatment to the tumor: the next step.About 1 in 10 tumors has an ALK gene change. The ALK drug lorlatinib shrank many of these tumors in early trials, and a large Children's Oncology Group trial is testing it in newly diagnosed children. Its results are not yet reported.
This is Cure Checker's editorial assessment of published evidence, not medical advice. Treatment decisions belong with your child's pediatric oncology team, ideally at a center that treats many children with neuroblastoma.
A possible missing link
Please read first. This is an AI-generated hypothesis, written by Cure Checker's AI from published research. It is speculation, not an established finding. It is not a recommendation, not a prediction that a cure exists, and not medical advice. No doctors or scientists have reviewed it, and it may be wrong. Never start, stop, change or delay any treatment because of it. Talk with your pediatric oncologist.
What looks like it is working
Targeting GD2. In a randomized trial of 226 children, adding an anti-GD2 antibody raised 2-year event-free survival from 46% to 66% (NEJM, 2010). GD2-directed CAR-T cells produced responses in 66% of children with relapsed or resistant disease (Nat Med, 2025).
Finding hidden disease. In 345 children, tumor signals left in bone marrow after early treatment went with lower 5-year event-free survival, 26.6% versus 60.4% (J Exp Clin Cancer Res, 2024).
Where the links are missing
Cells that change shape. Neuroblastoma cells can switch between two states, and the "mesenchymal" state resisted chemotherapy in the lab and was more common after treatment (Nat Genet, 2017). One lab study found this switch lowered GD2 (Nat Cancer, 2022), but another, in cells taken from patients, found these cells still carried GD2 (Front Immunol, 2024). Our inference: whether this switch helps tumors escape GD2 treatment in children is unsettled.
Escape at relapse. Cells with little or no GD2 but high levels of another marker, B7-H3, were found at relapse in 2 children (J Immunother Cancer, 2021). B7-H3 stays present in both cell states (Neuro-Oncology, 2025). Our inference: a second target might cover cells a GD2 treatment misses.
Watching the switch. In 108 children, marrow tests for mesenchymal markers found extra cases, and these markers rose before relapse (J Exp Clin Cancer Res, 2025). Our inference: we found no published study or registered trial that uses repeated tests like these during anti-GD2 treatment to decide when to add a second target, though we may have missed one.
Our hypothesis
Could repeated marrow or blood tests that track both cell states during anti-GD2 treatment show, months before scans can, which children's leftover cells are drifting away from GD2? If so, trials could test adding a second target such as B7-H3 while leftover disease is tiny, using a test strategy that has not been validated.
What would prove it wrong: if rising mesenchymal markers do not come before relapse in larger studies, or if tumors that relapse after anti-GD2 treatment still carry plenty of GD2.
Hypothesis written October 7, 2026. Cure Checker's AI will revisit it when major new evidence appears.
Latest studies & what they concluded
Newest first. Updated each morning as results, trial launches and papers are published.
Eye side effects of anti-GD2 antibody after a donor stem-cell transplant
A Japanese hospital looked back at 9 children with high-risk neuroblastoma who later got anti-GD2 antibody treatment. Eye side effects happened in 1 of 6 who had only a transplant of their own stem cells, and in all 3 who also had a donor (allogeneic) transplant. The authors say a donor transplant before anti-GD2 treatment may be a risk factor. With only 9 children, this is a very small look-back study, and it cannot prove cause.
Blood tests that track neuroblastoma during 131I-MIBG treatment
Using samples from 87 children in a Phase 2 trial of 131I-MIBG for relapsed or hard-to-treat neuroblastoma, researchers found tumor DNA in the blood in 44% at the start and 14% by the end of the first course. These markers tracked how much cancer was in the body, but neither was significantly linked to whether the tumor shrank. So they are not yet proven as a way to predict response.
CD47 blocker added to anti-GD2 antibody fails in mouse models
In two mouse models of chemo-resistant neuroblastoma, a CD47-blocking drug (ALX301) helped immune cells swallow tumor cells in lab dishes but did not improve tumor control or survival when added to anti-GD2 antibody, with or without chemotherapy. This is lab and animal work, not a human trial. The authors suggest it may need other partner drugs.
Higher MIBG dose before stem-cell transplant: no clear survival gain
A hospital looked back at 56 children with high-risk, MIBG-avid neuroblastoma who got radioactive MIBG as part of pre-transplant chemotherapy, at either 12 or 18 mCi/kg. Survival was about the same: 5-year overall survival was 59.5% with the lower dose and 58.2% with the higher dose. The higher dose was linked to faster blood-count recovery and more normal MIBG scans at one year (83.3% vs 46.2%). The authors say the study was small and looking back, so it cannot show which dose is best.
Dinutuximab beta in real-world use in France (QUATUOR study)
A look back at 31 children with high-risk neuroblastoma who got dinutuximab beta through France's early-access program in 2017 to 2018: 14 in first-line care and 17 with refractory or relapsed disease. Two-year event-free survival was 64.3% (first-line) and 57.5% (refractory or relapsed), and two-year overall survival was 78.6% and 80.9%. The authors call tolerability acceptable. It is a small retrospective study with no comparison group, and it was funded by the drug's maker, Recordati.
Lab study finds a possible weak spot in aggressive MYCN neuroblastoma
In cell and animal experiments, researchers found that a gene on chromosome 17q (IGF2BP1) works with MYCN to help high-risk tumors cope with stress on their DNA copying. A drug that blocks the protein CHK1 triggered immune signals and worked better with immune checkpoint drugs in the models. This is early laboratory work and has not been tested in children.
Telomere test may sort risk better in non-high-risk neuroblastoma
In 366 patients (259 not high-risk), a tumor test for telomere maintenance, a way cancer cells keep dividing, separated children with excellent outcomes from those with poor outcomes better than current molecular tests. The authors suggest it could help avoid overtreating children with favorable disease. It still needs testing in a prospective trial before use in care.
Removing the whole tumor and survival (COG ANBL0532 analysis)
In 326 children with high-risk neuroblastoma, those whose surgeons removed at least 90% of the main tumor had better 5-year event-free survival (50.3% vs 31.0%) and overall survival (61.8% vs 41.1%) than those with less removed, with no more surgical complications. This is a look back at a trial, not a test of surgery itself, and complete removal was more likely in easier-to-reach tumors.
Dinutuximab beta after relapse and survival (adjusted comparison)
Researchers compared 329 children with relapsed high-risk neuroblastoma using patient-level data and statistical matching. Those given dinutuximab beta after relapse lived longer than historical patients who never had anti-GD2 treatment (hazard ratio 0.53, 95% CI 0.35-0.81). Children who relapsed after dinutuximab beta in first-line treatment did not do better or worse than the historical group. This was not a randomized trial, so unmeasured differences between groups could affect the result.
22 children with relapsed or resistant neuroblastoma were treated. No tumors shrank, and low blood sodium limited the dose in 3. The authors say L1CAM may not be a suitable target.
Does GM-CSF add anything to anti-GD2 antibodies? (review)
No study has compared the same antibody treatment with and without GM-CSF. The reviewers say licensed treatment plans should not change on this basis and call for a randomized trial.
Japan's anti-GD2 regimen vs the U.S. standard (randomized Phase 2)
35 children. Two-year event-free survival was 80.8% with filgrastim and teceleukin versus 62.3% with the standard mix. The authors call the two "comparable" and say a larger trial is needed.
Chemo plus dinutuximab at relapse: long-term results (COG ANBL1221)
For relapsed or resistant disease treated with irinotecan, temozolomide and dinutuximab, 5-year progression-free survival was 22.6% and overall survival 54.7%.
14 children with relapsed or resistant disease. Tumors took up about half as much radiation with the second dose, and no lasting kidney damage was seen. The trial raised its first dose. Response results are not reported yet.
Lorlatinib for ALK-driven high-risk neuroblastoma (China)
25 patients. Tumors shrank in 11 of 17 who could be assessed, including all 6 newly diagnosed children who got it with chemotherapy. Lung side effects occurred when combined with other treatments. The authors call it preliminary.
39 patients. 42% made antibodies against the target, with no serious treatment-related side effects. Three-year progression-free survival was 48% in those vaccinated in good remission. There was no comparison group.
SHINE: anti-GD2 antibody with first chemo (Phase 2/3)
A multinational trial of the antibody hu14.18K322A given with first-line chemotherapy for high-risk neuroblastoma. It aims to enroll 144 children and is recruiting.
Treatment depends on the risk group. Many children with low-risk disease need only surgery or careful watching. High-risk disease needs months of intensive treatment, and that is where most new research is focused.
High-risk · after transplant · COG ANBL0032 · SIOPEN HR-NBL1
Antibodies latch onto GD2 on tumor cells and call in the immune system. In the U.S. trial, 2-year event-free survival rose from 46% to 66%. Pain during infusions is common and is managed in hospital.
Eflornithine (Iwilfin)
Approved · U.S.
High-risk · after antibody treatment · FDA Dec 13, 2023
A pill taken for up to two years to lower the risk of relapse. Compared with similar past patients, the risk of relapse was about half. It was not a randomized trial, and the EU application was withdrawn in 2025.
Naxitamab (Danyelza)
Accelerated approval · U.S.
Relapsed or resistant disease in bone or marrow · FDA Nov 25, 2020
Another anti-GD2 antibody, given with GM-CSF. In the main study, 45% of patients responded. Continued approval depends on a confirmatory trial.
GD2 CAR-T cells (GD2-CART01)
Promising · Phase 1/2
Bambino Gesù Children's Hospital, Rome · Nature Medicine 2025
A child's own immune cells are engineered to attack GD2. 66% responded, and results were best in children with little disease left. Side effects included cytokine release syndrome, mostly mild.
Lorlatinib for ALK-changed tumors
Early signal · trials
Children's Oncology Group ANBL1531 · not approved for neuroblastoma
An ALK-blocking pill. In an early trial, tumors shrank in 30% of children taking it alone and 63% with chemo. A large trial in newly diagnosed children is still running, and lung problems have been reported when it is given with antibody treatment.
Chemo plus antibody at relapse
Promising · Phase 2
BEACON-Immuno (Europe) · COG ANBL1221 (U.S.)
Adding an anti-GD2 antibody to chemotherapy for relapsed disease. In a randomized European trial, responses were 30.2% versus 18.2%, and the time before the cancer grew again was longer.
Standard treatment today
Treatment
For
What it does
Careful watching
Some babies with small or favorable tumors
Regular scans and tests. Some tumors in babies shrink away without treatment.
Surgery
Low risk, and part of other plans
Removes the tumor. For many low-risk children it is the only treatment needed.
Chemotherapy
Intermediate risk
Several cycles, usually before surgery. Recent trials have safely reduced the amount many children need.
Induction chemo + surgery
High risk
Several drug combinations over months to shrink the tumor, then surgery.
Tandem stem cell transplant + radiation
High risk
Two rounds of high-dose chemo, each followed by the child's own stored stem cells, then radiation to the tumor site.
Anti-GD2 antibody + GM-CSF + isotretinoin
High risk
Immune treatment and a vitamin A-related drug to clear leftover cells.
Eflornithine
High risk, U.S.
Pills for up to two years after antibody treatment to lower the risk of relapse.
Natural remedies & supplements
No supplement, herb or diet cures neuroblastoma or prevents relapse. Some can interfere with chemotherapy or with isotretinoin, which is part of standard care. We rate each by the strength of human evidence. Always check with your child's oncology team or pharmacist first.
Good human evidenceObservational / mixedLab only, or possible harm
Claims you'll see online
Nutrition support with a dietitian
Poor nutrition is common during childhood cancer treatment. A Cochrane review found limited, low-quality evidence that extra feeds help children gain weight. This is part of supportive care, not a tumor treatment.
Exercise & play
Studies in children with leukemia show some fitness gains, but the effects are "not yet convincing." No neuroblastoma-specific trials exist. Ask what is safe during treatment.
Turmeric / curcumin
Neuroblastoma research on curcumin is lab-only. Lab studies suggest it may blunt some chemotherapy drugs, and it changes how the liver processes medicines.
Cannabis / CBD oil
NCI says evidence for cannabis as a cancer treatment in children is limited to a few case reports. It may help nausea or appetite, but CBD oils can change how chemo works.
Use with caution
Vitamin A supplements
Isotretinoin is related to vitamin A. Its FDA label (written for acne) warns against vitamin A supplements because of added toxicity. In neuroblastoma, isotretinoin already causes high calcium in about 1 in 5 children.
High-dose antioxidants & IV vitamin C
Not FDA-approved to treat cancer, with no studies in children. In adults, antioxidant supplements during chemo or radiation have been linked to worse outcomes.
St. John's wort
Cut the active form of irinotecan, a chemo drug used in neuroblastoma, by 42% in a small study. The effect can last weeks after stopping.
Probiotics
Rare bloodstream infections have been reported in people with cancer. A pediatric oncology guideline advises against routine use to prevent C. difficile infection.
Mistletoe
Adult studies have major weaknesses, and there are none in children. NCI says it can't be recommended outside well-designed trials.
Be careful: clinics and websites selling "cures" for childhood cancer can be very costly and may delay treatment that works. The FDA warns that claims like "treats all forms of cancer" or "more effective than chemotherapy" are red flags. Antineoplastons (the Burzynski clinic), once promoted for children's cancers, are not FDA-approved for any disease; the FDA barred new child patients from the program in 2012, and it has been on full FDA hold since 2016. Bring anything you're considering to your child's oncology team, and use the trial registries below to find real trials.
Where to get care and support
Centers recognized for expertise in this condition, and communities of families living with it.
Where people find expert care
St. Jude Children's Research HospitalMemphis, Tennessee, USA
An NCI-designated Comprehensive Cancer Center and a member of the NANT neuroblastoma research consortium. Its neuroblastoma program studies GD2 antibodies given with chemotherapy and lists open neuroblastoma trials.
Children's Hospital of Philadelphia, Neuroblastoma Developmental Therapeutics ProgramPhiladelphia, Pennsylvania, USA
A NANT member site whose team cares for children with high-risk neuroblastoma when standard treatments are no longer working, and has studied neuroblastoma for more than 25 years.
These are centers recognized for expertise in this condition, not a ranking, and Cure Checker does not endorse or rate any facility. Outcomes depend on the individual. Ask your doctor which center fits your situation.
Support groups & communities
You don't have to face your child's neuroblastoma alone. These groups connect parents, caregivers and survivors with people who understand. Most are free.
Solving Kids' Cancer UK
Family support
Families · UK
A family support team giving emotional support, practical guidance and information to families affected by neuroblastoma, with pages for each risk group, relapse and bereavement.
Before you join: support groups share experiences, not medical advice. Check anything about treatment with your child's oncology team. Some posts describe hard outcomes, so read at your own pace. Think about your child's privacy, because Reddit and many forums are public, and never share passwords or financial details. Cure Checker isn't affiliated with these groups.
How other countries treat neuroblastoma
The backbone of chemotherapy, surgery, transplant and radiation is similar worldwide. The differences are in immunotherapy and maintenance: the U.S. uses dinutuximab and eflornithine, Europe and the UK use dinutuximab beta, and Japan uses dinutuximab with different helper drugs.
Who has approved what
Treatment
U.S.
EU
UK
Canada
Japan
China
Australia
Switz.
DinutuximabUnituxin, anti-GD2
2015
Withdrawn 2017
—
2018
2021
—
—
—
Dinutuximab betaQarziba, anti-GD2
—
2017, exceptional
NICE 2018
—
—
2021, conditional
—
—
NaxitamabDanyelza, anti-GD2
2020, accelerated
—
—
—
—
2022, conditional
—
—
EflornithineIwilfin / Ifinwil, maintenance
2023
Withdrawn 2025
—
—
—
—
2025
2025
2022 approved (year)Review conditional, under review, or limited fundingRejected refused or withdrawn— not approved, or no approval on record
Available abroad, not in the U.S.
Dinutuximab beta (Qarziba)
EU · UK · China
EU authorisation May 8, 2017 · NICE Aug 22, 2018
Europe's anti-GD2 antibody, used after transplant and for relapsed disease. It has no FDA approval. The European trial showed that adding IL-2 brought more side effects and no benefit, so it is given without it.
Japan approved dinutuximab for use after high-dose chemotherapy, given with filgrastim and teceleukin instead of the U.S. helper drugs. A 2026 randomized trial found the two approaches comparable.
Japan approves dinutuximab. China conditionally approves dinutuximab beta.
China conditionally approves naxitamab.
U.S. approves eflornithine to lower relapse risk. GD2 CAR-T results from Rome appear in NEJM.
Australia and Switzerland approve eflornithine. The EU application is withdrawn.
Randomized trials in Europe and Japan refine antibody treatment. New relapse trials open.
What causes neuroblastoma
Neuroblastoma is a cancer of very young nerve cells, called neuroblasts, that normally help build the body's automatic nervous system before birth. It most often starts in an adrenal gland, above a kidney. Nothing parents did or didn't do causes it. There are no known lifestyle or environmental causes, and only 1 to 2% of cases run in families.
Step 1 · The start
Begins before birth
Origin: neural crest cells, which become the nerves that run the body automatically
Timing: gene studies show it can begin as early as the first trimester of pregnancy
Age: median age at diagnosis is 17 months, and 90% are found before age 5
Not caused by: anything parents did. No clear environmental cause is known.
Step 2 · The tipping point
Cells don't finish maturing
MYCN: extra copies in 16–25% of tumors and 40–50% of high-risk tumors
ALK: changed in about 10% of tumors, and the main cause of the rare inherited form
Others: TERT rearrangements, 11q loss, and ATRX changes, which are more common in older children and teens
Step 3 · The damage
How it shows up
A lump: most often in the belly, sometimes in the chest, neck or pelvis
Spread: often to lymph nodes, bone, bone marrow, liver or skin by diagnosis
Signs: bone pain, a swollen belly, bruising around the eyes, bluish skin lumps in babies
Babies: some tumors in infants shrink away on their own
Step 4 · The result
Risk group shapes the outlook
Five-year survival by risk group, from the NCI
Risk depends on age, stage, MYCN, how the cells look under the microscope, and chromosome changes
High-risk disease can return, sometimes years later
1983–1984, MYCN: researchers found a gene copied up to 140 times in neuroblastoma cells. A year later, extra copies were found in half of advanced tumors and none of the early-stage ones studied. It is still one of the most important risk markers.
August 24, 2008, ALK: a study showed that inherited ALK gene changes explain most families with neuroblastoma. ALK also turned out to be changed in some non-inherited tumors, which led to ALK-blocking drug trials.
March 27, 2023, before birth: a genetic study found that neuroblastoma of every type can start forming as early as the first trimester of pregnancy, and that how long a tumor has been evolving relates to how it behaves.
Find a clinical trial
Live numbers and the newest entries from the world's main trial registries and medical databases. Most of this loads fresh each time you open the page.
Where neuroblastoma trials are recruiting right now
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Drug trials with no U.S. site
Newest published clinical trials
PubMed · U.S. National Library of Medicine
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Newest Cochrane systematic reviews
Cochrane Database of Systematic Reviews, via PubMed
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Newest trials in the EU registry
EU Clinical Trials Information System
Newest UK & international registrations
ISRCTN registry
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FDA records for the medicines in this report
Medicine
Brand
FDA application
Original approval
From Drugs@FDA via FDA's openFDA service. Some older brands are missing from FDA's open data, and newly approved drugs can take a few weeks to appear. An approval may be for a different disease than this one.
Search every registry yourself
The WHO's ICTRP gathers trials from registries worldwide, including China, Japan, India, Iran, Australia and Brazil. Cochrane CENTRAL and OpenMD don't allow automated access, so they're linked here for you to search directly.
Financial help for families living with neuroblastoma
These charities give money or pay bills for families of children with neuroblastoma or another childhood cancer. Funds are limited and rules vary, so contact each one directly. Your hospital's social worker can often apply for you.
Neuroblastoma organizations
Solving Kids' Cancer UK: Big Love Fund wellbeing grants
Grants
Children and young people up to 30 with neuroblastoma · UK and Ireland · no means test
Up to £250 a year for activities, equipment, experiences or items that support the child's physical or emotional wellbeing, during treatment or within 5 years after it ends.
Children diagnosed with cancer under 21 · any U.S. hospital
Pays creditors directly for costs caused by the diagnosis, such as uncovered prescriptions, treatment travel, and mortgage or rent. Once per 12 months, twice in total. Apply through the hospital social worker.
Parents of children under 16 with cancer, and young people 16–24 · UK
Helps with treatment travel, accommodation, food and living costs, household bills and equipment. The family's Young Lives vs Cancer social worker applies. No amount is stated.
Before you apply: Cure Checker does not run, verify or endorse these organizations, and being listed is not a promise of help. Funds are limited and rules change, so confirm eligibility and details with each organization. Be careful of anyone who asks for payment up front or for your bank login to 'release' a grant.
Sources
Everything this report is based on, grouped by section. Checked October 7, 2026.
Survival figures are 5-year overall survival from NCI's PDQ summary and can't predict any one child's outcome. The share of childhood cancers (6–10%) and cancer deaths (12–15%) is not used on this page because only one older review gave it. Approvals: Canada's approval date is from the company announcement (Health Canada's site was down); China's dates are from company or trade-press announcements, not the regulator. We could not confirm dates for Qarziba in Australia or eflornithine in Israel, and did not check South Korea, so these are not shown. Eflornithine's U.S. approval rests on a comparison with past patients, not a randomized trial. Lorlatinib and MIBG results from COG ANBL1531 have not been published. Natural remedies: almost no studies are specific to neuroblastoma; most evidence comes from adults or other childhood cancers. The isotretinoin label cited is the acne label. The Burzynski hold history comes from the company's own SEC filing. Missing-link hypothesis: whether the mesenchymal cell state lowers GD2 is contested, and one large study found GD2 loss did not change survival. Expert care centers: NCI designation and NANT membership describe each center as a whole, not neuroblastoma results; Children's Hospital Los Angeles (NANT's operations center) is left out because its site blocked automated checks. Support groups: the Facebook group run by the Neuroblastoma Children's Cancer Society could not be checked automatically. Financial help: checked October 7, 2026. Only the Solving Kids' Cancer UK grants are specific to neuroblastoma. Family Reach and CancerCare had no open childhood-cancer fund that day, HealthWell's pediatric fund was closed and does not cover neuroblastoma, and the PAN Foundation and Patient Advocate Foundation sites blocked automated checks, so they are not listed.