Sarcomas (soft tissue, bone, GIST) · What is coming

Extending sarcoma TCR-T beyond HLA-A*02

Intermittent or stop-and-restart nirogacestat in desmoid tumours

A funded expert second opinion for every new high-stakes or rare cancer diagnosis

A non-profit pharmaceutical company for the cancers markets ignore

A same-week expert second opinion for every rare cancer diagnosis

A survivorship passport app for adolescent and young adult survivors

Advance market commitments for paediatric and rare cancer drugs

An international consortium pooling the outcome of every treated child with cancer

An open model bank for the rare tumours nobody has models for

An open organoid bank for cancers too rare to have models

Attack extrachromosomal DNA, the engine of oncogene amplification

Degraders for the fusion proteins that drive childhood sarcomas

Develop drugs in children first when the target is a children's target

Escrow a share of adult revenue until the paediatric study is done

FAP theranostics as a pan-cancer stromal strategy

Group trials by broken mechanism, not by organ or single mutation

Grow immune command posts inside tumours

Implant a tiny device that tests twenty drugs inside the patient's own tumour

Inhaled immune therapy to make the lung hostile to arriving tumour cells

Let adolescents from age 12 into adult trials when the cancer biology is the same

Let patients themselves donate their records and samples for ultra-rare cancers

Mandatory national virtual tumour boards for rare and complex cancers

Milestone-based venture philanthropy with royalties recycled into the pipeline

One global rare cancer network with n-of-1 and Bayesian trial frameworks

One legal framework for pooling rare cancer data across borders

One standing umbrella trial for all rare cancers in a country

Open-source drug discovery to clinical proof of concept for neglected cancers

Partial lottery funding for good proposals in under-funded cancers

Patent-free open-source development of repurposed and off-patent cancer drugs

Pay a prize for rare cancer drugs instead of hoping for a market

Pet dogs with spontaneous cancer as a bridge before human trials

Randomise inside the registry that already follows every patient

Test drugs on the patient's own cancer cells when there is no trial to join

The pathology lab triggers a trial referral the day a rare cancer is diagnosed

Trial-in-a-box: a preconfigured protocol kit any hospital can open for a rare cancer

Turn one tumour into a vaccine to treat all the others

Whole-body MRI plus blood DNA surveillance for people with Li-Fraumeni syndrome

What is comingSarcomas (soft tissue, bone, GIST)

What is coming

In development

Drugs in phase 3 · 6

Drugs in phase 2 · 3

Drugs at an unstated stage · 1

Technologies being tested · 8

Trials under way · 23

Trials reported · 28

Combinations being explored · 2

Ideas not yet in a trial · 37

Open problems and what is being done

What changed