# Paediatric oncology roadmap: cooperative-group cures → engineered immunity → drugs developed for children first

Source: https://onco.cc/roadmaps/paediatric-oncology-roadmap/  
OnCo record `paediatric-oncology-roadmap` (Roadmap). Data CC BY-NC 4.0, attribute "Data from OnCo (onco.cc)"; commercial use needs a licence.

## TL;DR

Children's cancers were made curable by decades of cooperative trials that refined chemotherapy dose by dose. The next gains come from immunotherapy and targeted drugs built for children's cancers, from laws that stop companies skipping children, and from protecting the child who is cured from the cost of the cure.

## Summary

Childhood leukaemia was fatal in 1948 and is curable today because cooperative groups in North America and Europe ran one randomised trial after another, each refining the previous regimen. The same method made neuroblastoma, Wilms tumour, Ewing sarcoma and medulloblastoma curable in most children, and the Childhood Cancer Survivor Study then documented what those cures cost decades later: heart damage, second cancers, infertility and hearing loss.

The present era brought engineered immunity to children first. Blinatumomab, the first T-cell engager, and tisagenlecleucel, the first CAR-T ever approved (2017, after ELIANA), both started in childhood leukaemia; anti-GD2 antibodies raised cure rates in high-risk neuroblastoma. Precision medicine followed: Pediatric MATCH screened children nationally for targetable mutations, and paediatric-first drugs arrived for the commonest childhood brain tumour (tovorafenib) and for diffuse midline glioma (dordaviprone). The RACE for Children Act, in force since 2020, requires companies to study new targeted cancer drugs in children whenever the target matters in a childhood cancer, and the ACCELERATE platform brings companies, regulators and parents together to plan those studies.

The pace is set by the smallness of the market, which no incentive fully corrects; by regulatory divergence between the FDA and EMA; by late effects that are still under-recorded; and by the fact that most children with cancer live in countries where cure rates are a fraction of those in rich ones.

## Fields

- Kind: Roadmap
- Last checked: 2026-09-10

## Sources

- ACCELERATE Paediatric Strategy Forums: https://www.accelerate-platform.org/paediatric-strategy-forums/
- Childhood Cancer Survivor Study: https://ccss.stjude.org
- WHO Global Initiative for Childhood Cancer: https://www.who.int/initiatives/the-global-initiative-for-childhood-cancer

## Connected records

- collections: [Alex's Lemonade Stand Foundation (ALSF)](https://onco.cc/collections/alexs-lemonade-stand/), [St. Baldrick's Foundation](https://onco.cc/collections/st-baldricks/)
- ideas: [18F-MFBG PET replacing 123I-MIBG scintigraphy](https://onco.cc/ideas/idea-mfbg-pet-replaces-mibg/), [A paediatric palliative care team in every childhood cancer unit](https://onco.cc/ideas/idea-acc-paediatric-palliative-in-every-childhood-unit/), [Advance market commitments for paediatric and rare cancer drugs](https://onco.cc/ideas/idea-fund-amc-paediatric-rare/), [An international consortium pooling the outcome of every treated child with cancer](https://onco.cc/ideas/idea-data-paediatric-rwe-consortium/), [Automatic reciprocity of orphan and rare-paediatric designations between regulators](https://onco.cc/ideas/idea-reg-orphan-designation-reciprocity/), [Cash for transport and food to stop families abandoning curative treatment](https://onco.cc/ideas/idea-acc-abandonment-stipends/), [Degraders for the fusion proteins that drive childhood sarcomas](https://onco.cc/ideas/idea-bio1-fusion-tf-degraders/), [Develop drugs in children first when the target is a children's target](https://onco.cc/ideas/idea-bio2-paediatric-first-development/), [Escrow a share of adult revenue until the paediatric study is done](https://onco.cc/ideas/idea-fund-paediatric-deferral-escrow/), [GD2 CAR-T as consolidation in high-risk neuroblastoma](https://onco.cc/ideas/idea-gd2-car-t-frontline-consolidation/), [Guaranteed-quality childhood cancer medicines free of charge in 50 countries](https://onco.cc/ideas/idea-acc-childhood-medicines-platform-scale/), [Make paediatric combination studies part of every relevant adult cancer drug approval](https://onco.cc/ideas/idea-tr2-paediatric-combo-prea/), [One global paediatric cancer development plan instead of separate FDA and EMA plans](https://onco.cc/ideas/idea-reg-single-paediatric-plan/), [Protect hearing from cisplatin in adults as we now do in children](https://onco.cc/ideas/idea-moon-hearing-protection-cisplatin/)
- roadmaps: [Cell therapy roadmap: CD19 CAR-T → solid tumours → in vivo CAR](https://onco.cc/roadmaps/cell-therapy-roadmap/), [Recovery and rejuvenation roadmap: cure is not enough → survivorship gets a name → the cohorts that measured the cost → exercise proven as treatment → biological ageing measured and sold → repair, if anyone funds it](https://onco.cc/roadmaps/rejuvenation-roadmap/), [Supportive care and survivorship roadmap: making treatment bearable → proving it extends life → caring for tens of millions afterwards](https://onco.cc/roadmaps/survivorship-roadmap/)
- cancers: [Acute lymphoblastic leukaemia](https://onco.cc/cancers/all-leukemia/), [Diffuse midline glioma, H3 K27-altered (including DIPG)](https://onco.cc/cancers/dipg-dmg/), [Neuroblastoma (paediatric)](https://onco.cc/cancers/neuroblastoma/), [Paediatric low-grade glioma](https://onco.cc/cancers/paediatric-low-grade-glioma/), [Wilms tumour (nephroblastoma)](https://onco.cc/cancers/wilms-tumor/)
- technologies: [CAR-T cell therapy](https://onco.cc/technologies/car-t/), [CAR-T for glioma (IL13Rα2, GD2, EGFRvIII, multi-target)](https://onco.cc/technologies/glioma-car-t/), [Comprehensive genomic profiling](https://onco.cc/technologies/cgp/), [Late deaths after childhood cancer, what causes them, and the proof that gentler treatment worked](https://onco.cc/technologies/rejuv-paed-late-mortality/), [MIBG imaging and 131I-MIBG therapy](https://onco.cc/technologies/mibg-theranostics/), [Oncofertility and fertility preservation](https://onco.cc/technologies/fertility-preservation/), [Patient-derived xenografts](https://onco.cc/technologies/pdx-models/), [Survivorship care and late-effects surveillance](https://onco.cc/technologies/survivorship-care-plan/), [Tandem autologous transplant](https://onco.cc/technologies/tandem-transplant/)
- drugs: [131I-MIBG (iobenguane I-131) therapy](https://onco.cc/drugs/i131-mibg/), [Asparaginase (pegaspargase, calaspargase pegol, Erwinia asparaginase)](https://onco.cc/drugs/asparaginase/), [Blinatumomab](https://onco.cc/drugs/blinatumomab/), [Dabrafenib + trametinib](https://onco.cc/drugs/dabrafenib-trametinib/), [Dexrazoxane](https://onco.cc/drugs/dexrazoxane/), [Dinutuximab (ch14.18) / dinutuximab beta](https://onco.cc/drugs/dinutuximab/), [Dordaviprone](https://onco.cc/drugs/dordaviprone/), [Larotrectinib](https://onco.cc/drugs/larotrectinib/), [Mercaptopurine](https://onco.cc/drugs/mercaptopurine/), [Methotrexate](https://onco.cc/drugs/methotrexate/), [Mifamurtide](https://onco.cc/drugs/mifamurtide/), [Selumetinib](https://onco.cc/drugs/selumetinib/), [Sodium thiosulfate (otoprotectant)](https://onco.cc/drugs/sodium-thiosulfate/), [Tisagenlecleucel](https://onco.cc/drugs/tisagenlecleucel/), [Tovorafenib](https://onco.cc/drugs/tovorafenib/), [Vincristine](https://onco.cc/drugs/vincristine/), [Vorasidenib](https://onco.cc/drugs/vorasidenib/)
- companies: [Children's Oncology Group (COG)](https://onco.cc/companies/childrens-oncology-group/), [Day One Biopharmaceuticals](https://onco.cc/companies/day-one-biopharmaceuticals/), [Innovative Therapies for Children with Cancer (ITCC)](https://onco.cc/companies/itcc/), [Y-mAbs Therapeutics](https://onco.cc/companies/y-mabs/)
- institutions: [ACCELERATE](https://onco.cc/institutions/accelerate-platform/), [European Medicines Agency](https://onco.cc/institutions/ema/), [FDA Oncology Center of Excellence](https://onco.cc/institutions/fda-oce/), [SIOP Europe (European Society for Paediatric Oncology)](https://onco.cc/institutions/siop-europe/), [St. Jude Children's Research Hospital](https://onco.cc/institutions/st-jude/)
- terms: [Accelerated approval](https://onco.cc/terms/accelerated-approval/), [Orphan drug](https://onco.cc/terms/orphan-drug/), [RACE for Children Act](https://onco.cc/terms/race-for-children-act/)
- trials: [ACTION](https://onco.cc/trials/action-dmg/), [Childhood Cancer Survivor Study (CCSS)](https://onco.cc/trials/ccss/), [COG AALL1731](https://onco.cc/trials/aall1731/), [COG AAML0531](https://onco.cc/trials/aaml0531/), [COG ACNS0331](https://onco.cc/trials/acns0331/), [COG ANBL0032](https://onco.cc/trials/anbl0032/), [COG ANBL0532](https://onco.cc/trials/anbl0532/), [COG ANBL1531](https://onco.cc/trials/anbl1531/), [COG AREN0533](https://onco.cc/trials/aren0533/), [ELIANA](https://onco.cc/trials/eliana/), [EURAMOS-1](https://onco.cc/trials/euramos-1/), [FIREFLY-1](https://onco.cc/trials/firefly-1/), [GD2-CART01 (Bambino Gesù phase 1/2)](https://onco.cc/trials/gd2-cart01/), [INT-0091 (Ewing sarcoma)](https://onco.cc/trials/int-0091/), [Inter-B-NHL Ritux 2010](https://onco.cc/trials/inter-b-nhl-ritux-2010/), [Interfant-06](https://onco.cc/trials/interfant-06/), [NCI-COG Pediatric MATCH (APEC1621)](https://onco.cc/trials/pediatric-match/), [SIOPEN HR-NBL1](https://onco.cc/trials/hr-nbl1/), [TADPOLE (CDRB436G2201)](https://onco.cc/trials/tadpole/), [TOWER](https://onco.cc/trials/tower/)
- bottlenecks: [Incentives reward me-too drugs and marginal gains](https://onco.cc/bottlenecks/b-incentive-misalignment/), [Most of the world has almost no cancer care](https://onco.cc/bottlenecks/b-global-access/), [Rare and paediatric cancers without markets](https://onco.cc/bottlenecks/b-rare-cancers/), [Regulatory divergence between regions](https://onco.cc/bottlenecks/b-regulatory-fragmentation/), [Survivorship and late effects are neglected](https://onco.cc/bottlenecks/b-survivorship/), [Trials enrol too few, too slowly](https://onco.cc/bottlenecks/b-trial-enrolment/)

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