paediatric

ACCELERATE is a Brussels-based forum where children's cancer doctors, drug companies, the EMA and FDA and parents agree which new cancer drugs should be tested in children and how, so that medicines developed for adults are not left untested in childhood cancers.

ACTION is the first placebo-controlled phase 3 trial ever run in diffuse midline glioma, the childhood brain-stem tumour that radiotherapy alone has never cured. It asks whether taking dordaviprone after radiotherapy lengthens life.

Acute myeloid leukaemia in children carries gene fusions rather than the mutations of ageing, is treated with four or five intensive courses of chemotherapy, and cures around two thirds of children. Adding gemtuzumab ozogamicin lowered relapse in the AAML0531 trial, and the menin inhibitor revumenib is the first targeted drug approved for the KMT2A-rearranged form common in young children.

A childhood-cancer charity started by a four-year-old patient's lemonade stand that now funds hundreds of grants and runs an open-science data lab for paediatric cancer genomics.

Led the St. Jude medulloblastoma trials that introduced molecular subgroups into risk-adapted treatment.

ATRT is an aggressive brain tumour of babies and toddlers caused by loss of a single gene, SMARCB1, part of the machinery that opens and closes DNA. Intensive chemotherapy with stem-cell rescue, and radiotherapy where age allows, now cure a meaningful share of children who once had little chance, and drugs aimed at the epigenetic consequence of SMARCB1 loss (EZH2 inhibitors) are in trials.

Burkitt lymphoma is the fastest-growing human tumour, driven by a single rearrangement that switches on the MYC gene. That speed makes it exquisitely sensitive to chemotherapy: short, intense courses, now with the antibody rituximab, cure the great majority of children in well-resourced settings. The remaining task is to bring the same cure to the African children who make up most cases.

Germ cell tumours of the brain grow near the pineal gland or above the pituitary in teenagers. The commonest kind, germinoma, is so sensitive to radiation and chemotherapy that most patients are cured; the other kinds need stronger chemotherapy and radiotherapy, and doctors measure two proteins in the blood and spinal fluid to tell them apart and to follow treatment.

The largest study of what happens to children after cancer is cured. Following tens of thousands of survivors for decades, it showed that heart damage, second cancers and other late effects were common after older treatments, and that gentler modern protocols have already halved late deaths.

Cancer in children is rare and different from adult cancer: the common types are leukaemias, brain tumours, lymphomas and embryonal tumours such as neuroblastoma and Wilms tumour, most are curable in well-resourced health systems, and the great challenge is bringing the same cures to the majority of children who live where they are not available.

Primary lung tumours in children are rare and unlike adult lung cancer. Pleuropulmonary blastoma starts as a lung cyst in infants and results from a faulty DICER1 gene that also predisposes to thyroid, ovarian and kidney tumours; removing cysts early, guided by an international registry and family gene testing, prevents progression to the aggressive solid forms.

Led the St. Jude Total Therapy studies that cure over 90% of children with leukaemia without cranial radiation.

Chordoma is a slow-growing bone cancer (a sarcoma) of the skull base and spine that arises from leftover embryonic notochord cells. Complete surgery followed by high-dose proton or carbon-ion radiotherapy controls most tumours, and the whole disease depends on a single transcription factor, brachyury, which vaccines and degraders are now trying to hit.

Adding the antibody-drug conjugate gemtuzumab ozogamicin to chemotherapy lowered the chance of relapse in children with acute myeloid leukaemia. Years after the drug had been withdrawn from the US market, this trial helped bring it back for children.

This trial asked whether children with average-risk medulloblastoma could safely receive less radiation. Shrinking the boost to the tumour bed was safe; cutting the dose to the whole brain and spine in young children was not, so 23.4 Gy remains the floor for most.

A risk-adapted Wilms tumour trial: children whose lung metastases vanished after six weeks of chemotherapy were spared lung radiation, while those with stubborn nodules or a high-risk chromosome pattern got stronger chemotherapy and did better than in the past.

Craniopharyngioma is a benign but destructive brain tumour growing from embryonic remnants beside the pituitary gland and hypothalamus. Surgery, or limited surgery plus radiotherapy, cures most people, but the price can be lifelong hormone deficiency and severe obesity. The adult (papillary) form carries a BRAF mutation and shrinks markedly with BRAF and MEK inhibitors, its first drug treatment.

London paediatric neuro-oncologist who led the trial showing that dabrafenib and trametinib work in children with BRAF V600-mutant high-grade glioma.

Diffuse midline glioma grows through the brainstem and cannot be removed surgically. A single change in a histone protein (H3 K27M) rewires how the tumour reads its DNA. Radiotherapy was long the only help; in 2025 the first drug aimed at this tumour, dordaviprone (ONC201), was approved after durable shrinkage in some patients, and GD2 CAR-T cells have produced striking early responses.

Ependymomas grow from the cells lining the fluid spaces of the brain and spinal cord, mostly in children under five. Removing the whole tumour followed by focused radiotherapy controls most cases; molecular groups defined in 2021 behave differently, with posterior fossa group A relapsing often, and there is no approved drug.

The largest osteosarcoma trial ever run, across four cooperative groups on two continents. Neither adding interferon for good responders nor adding ifosfamide and etoposide for poor responders improved outcomes, so three-drug MAP chemotherapy remained the standard and the field turned to new biology.

Ewing sarcoma is a bone and soft-tissue cancer of teenagers driven by a single fusion gene, EWSR1-FLI1. Intensive chemotherapy with surgery or radiation cures most localised cases; disease that has spread at diagnosis, and relapse, remain hard to treat, and no drug against the fusion protein itself has yet succeeded.

FIREFLY-1 showed that a once-weekly pill, tovorafenib, shrinks most relapsed childhood low-grade gliomas driven by BRAF changes, including the common KIAA1549-BRAF fusion that older BRAF drugs could not treat safely. It led to the first approval of a drug for this disease.

Germ cell tumours arise from the cells meant to become eggs or sperm and can appear in the gonads, lower back, chest or brain. They are among the most curable childhood cancers because they respond to cisplatin chemotherapy and release blood markers that make monitoring easy. The work now is to cure with less: surgery alone for low-risk tumours, gentler platinum drugs, and protecting hearing.

Memphis paediatric neuro-oncologist who led the study showing that the Hedgehog inhibitor vismodegib helps only adults and older children whose medulloblastoma belongs to the SHH subgroup, an early example of subgroup-directed brain tumour therapy.

Group 3 and group 4 medulloblastoma are the two commonest forms of this cerebellar brain tumour and the ones without a druggable driver. Group 3 strikes young children, often with extra copies of MYC and spread through the spinal fluid; group 4 affects older boys. Both get surgery, craniospinal radiotherapy and chemotherapy; trials showed the radiation dose cannot be cut for young children.

H3 K27M is a single change in a histone that defines diffuse midline glioma, including the brain-stem tumour DIPG. In 2025 dordaviprone became the first drug approved for tumours carrying it.

Hepatoblastoma is a childhood liver cancer, mostly of toddlers, cured in most standard-risk cases with cisplatin chemotherapy and surgery, including liver transplant when the tumour cannot be cut out. Sodium thiosulfate given after cisplatin halves the permanent hearing loss cisplatin causes, and became the first approved otoprotectant in 2022.

High-risk childhood leukaemia means a child aged ten or over, a very high white cell count, T-cell disease, spread to the brain or testes, or adverse genetics, and it is treated with longer and more intensive chemotherapy. Most children are still cured; the T-cell form gained the drug nelarabine after the AALL0434 trial, and cranial radiotherapy has been dropped for almost everyone.

High-risk neuroblastoma has spread widely in a child over 18 months old or carries extra copies of the MYCN gene. Treatment lasts about 18 months and uses every tool: chemotherapy, surgery, high-dose chemotherapy with stem cell rescue, radiotherapy, and the anti-GD2 antibody dinutuximab, which raised survival in ANBL0032; eflornithine, given afterwards, was approved in 2023 to lower relapse.

The international society of doctors and scientists who study histiocytic disorders; its LCH trials, run since the 1990s, set the worldwide standard for treating Langerhans cell histiocytosis in children.

Leukaemia diagnosed in the first year of life is a different disease from leukaemia in older children: most cases carry a broken KMT2A gene and respond poorly to chemotherapy, and fewer than half of infants were cured for twenty years. One course of the immune drug blinatumomab after induction raised two-year disease-free survival from about half to over 80 percent in a pilot study.

IMT is a rare tumour, grouped with the sarcomas, of spindle cells mixed with inflammatory cells, most often in the lung or abdomen of children and young adults. Surgery cures most, and about half carry an ALK gene fusion, so the ALK-blocking pill crizotinib is approved for those that cannot be removed, one of the first targeted approvals for a childhood solid tumour.

Dutch paediatric oncologist who led the study showing that adding one course of blinatumomab to chemotherapy sharply improves survival for babies with KMT2A-rearranged leukaemia.

Europe's network of children's hospitals that run the first trials of new cancer drugs in children, so that European children can access experimental medicines close to home.

Adding the antibody rituximab to intensive chemotherapy in children with high-risk Burkitt and related lymphomas cut treatment failures by about two-thirds, making an already curable disease more so. It is the model of a joint European-North American children's cancer trial.

Intermediate-risk neuroblastoma sits between the tumours that go away on their own and the high-risk disease that needs everything. A few cycles of moderate chemotherapy followed by surgery cure most children, and trials have spent twenty years showing how few cycles are enough.

Paris paediatric neuro-oncologist who led HERBY, the trial that showed adding bevacizumab does not help children with high-grade glioma, and who leads European trials in diffuse midline glioma.

Led the COG renal tumour committee that used biology to tailor Wilms tumour treatment.

Neuroblastoma geneticist whose lab found the ALK mutations and immunotherapy targets now in paediatric trials.

Leads the COG Hodgkin lymphoma committee and the trials bringing brentuximab and nivolumab to children with the disease.

Paediatric oncologist who chaired COG AALL0434, the largest trial ever run in childhood T-cell leukaemia, which showed that adding nelarabine improves disease-free survival.

Langerhans cell histiocytosis is a disorder in which a small group of immune cells with a faulty growth signal (most often a BRAF mutation) pile up in bone, skin, pituitary or organs. It ranges from a single bone lesion that heals after biopsy to a life-threatening disease of infants. A year of gentle chemotherapy cures most children, and BRAF or MEK inhibitors rescue those with resistant disease.

The Histiocyte Society's third international trial showed that treating multisystem Langerhans cell histiocytosis for a full year, rather than six months, roughly halves the chance of the disease coming back, while adding methotrexate added nothing but toxicity.

Low-risk neuroblastoma is the form found in infants and young children whose tumour has not spread beyond its site or, in the special stage MS pattern, has spread only to the liver, skin and a little marrow. Many of these tumours shrink and disappear on their own, so treatment is surgery, or simply watching, and almost every child survives.

Medulloblastoma is the most common malignant childhood brain tumour, arising in the cerebellum. Surgery, radiation to the whole brain and spine, and chemotherapy cure about 70%, at a heavy cost to thinking and growth; treatment is now being tailored to four molecular subgroups so that the low-risk children get less.

Neurosurgeon-scientist who defined the four molecular subgroups of medulloblastoma that now guide therapy.

Founded cancer neuroscience and led the GD2 CAR-T trial that produced the first regressions of diffuse midline glioma.

Multisystem Langerhans cell histiocytosis is the severe form of this rare histiocytosis, in which the abnormal cells involve several organs at once, most dangerously the liver, spleen and bone marrow of infants. It is treated with a year of vinblastine and prednisone, with stronger drugs or BRAF-targeted tablets for children who do not respond quickly; survival is now high but late effects remain.

MYCN amplification, more than four times the normal copy number of the gene on FISH, marks the most aggressive fifth of neuroblastomas and puts a child in the high-risk group whatever their age or stage. No drug targets it; it decides how much treatment is given.

Developed the anti-GD2 antibodies 3F8 and naxitamab that treat relapsed neuroblastoma.

Pediatric MATCH was the first nationwide precision-medicine trial for children: every child with a relapsed solid tumour could have their tumour sequenced and, if a matching drug existed, join a trial arm for it. It proved the plumbing works, even though most single drugs given alone did little.

Neuroblastoma is a childhood nerve-cell cancer where anti-GD2 antibodies and, recently, GD2 CAR-T have improved survival in high-risk disease.

NUT carcinoma is a fast-growing cancer of the midline of the body driven by a single fused gene, BRD4-NUTM1, that locks cells in an immature state. Chemotherapy and surgery rarely control it for long, but drugs that block the BET proteins the fusion depends on have produced responses and are the focus of trials.

Osteosarcoma is the most common bone cancer, mostly in teenagers. Chemotherapy plus surgery cures about two-thirds when it has not spread; because no new drug has beaten that chemotherapy in a large trial in 30 years, the next gains are being sought in cellular therapy against GD2, HER2 and B7-H3.

High-grade gliomas in children look like adult glioblastoma under the microscope but are driven by different genes, so they are now classified separately. Surgery and radiotherapy remain the mainstay and chemotherapy adds little; the real gains are in small subsets with a targetable gene change, such as BRAF V600E tumours and the fusion-driven tumours of infants.

Paediatric low-grade gliomas are slow-growing brain tumours driven almost always by a single overactive signal, the MAPK pathway, most often through a BRAF gene change. Because the switch is known, pills that block it (dabrafenib with trametinib, and tovorafenib) now shrink tumours far more often than chemotherapy, and children are increasingly spared radiation to the developing brain.

Ph-like leukaemia behaves like Philadelphia chromosome-positive leukaemia, with the same kind of overactive growth signalling, but lacks the BCR::ABL1 gene itself. It is caused by a scattered set of gene fusions and mutations, many of them blockable by existing kinase pills such as dasatinib or ruxolitinib, and it is now screened for at diagnosis so those drugs can be tried.

Philadelphia chromosome-positive leukaemia carries the same faulty BCR::ABL1 gene as chronic myeloid leukaemia. Until 2000 most children with it needed a bone marrow transplant; adding the targeted pill imatinib to chemotherapy, and then dasatinib, means most are now cured without one.

A US law that makes drug companies test new targeted cancer drugs in children whenever the drug's target matters in a childhood cancer, instead of letting them skip children because their cancers are rare.