OnCo
cancersCancer

Sarcomas (soft tissue, bone, GIST)

Sarcomas are dozens of rare cancers of bone and connective tissue. GIST was the first solid tumour cured-in-practice by a targeted pill; synovial sarcoma got the first TCR-T therapy.

Sarcomas are cancers of connective tissue: more than 70 subtypes of soft-tissue sarcoma (liposarcoma, leiomyosarcoma, undifferentiated pleomorphic sarcoma, synovial sarcoma, angiosarcoma and others), bone sarcomas (osteosarcoma, Ewing sarcoma, chondrosarcoma), gastrointestinal stromal tumour (GIST), and locally aggressive but non-metastasising tumours such as desmoid fibromatosis and tenosynovial giant cell tumour. Together they are about 1% of adult and 15% of childhood cancers. Because each subtype is rare, expertise concentrates in reference centres, and treatment is increasingly by histotype and genotype: FNCLCC grade, size and depth define risk in soft-tissue sarcoma; KIT/PDGFRA mutations define GIST therapy; fusion genes (SS18-SSX, EWSR1-FLI1, FUS-DDIT3) define entities and, increasingly, targets.

Surgery with negative margins remains the curative act, with limb-salvage now standard and radiotherapy improving local control in soft-tissue sarcoma. Neoadjuvant anthracycline-ifosfamide benefits high-risk localised soft-tissue sarcoma (ISG-STS 1001), and multi-agent chemotherapy cures the majority of localised osteosarcoma (MAP) and Ewing sarcoma (VDC/IE, INT-0091 and Euro Ewing 2012). Advanced soft-tissue sarcoma still depends on doxorubicin with a median survival around 18-20 months; olaratumab's failure (ANNOUNCE) showed how hard that bar is to move. Subtype-specific drugs then fill in: trabectedin and eribulin for L-sarcomas, pazopanib for non-adipocytic sarcomas, and, in GIST, the sequence imatinib (3 years adjuvant, SSGXVIII), sunitinib, regorafenib and ripretinib (INVICTUS), with avapritinib for PDGFRA D842V and ctDNA-genotype-directed selection (INSIGHT) arriving. Desmoid tumours gained their first drug in nirogacestat (DeFi, 2023) and tenosynovial giant cell tumour its second in vimseltinib (MOTION, February 2025); tazemetostat for epithelioid sarcoma was withdrawn worldwide in March 2026 over secondary blood cancers.

Sarcoma is also where solid-tumour T-cell engineering first succeeded: afamitresgene autoleucel (MAGE-A4 TCR-T) was approved in 2024 and fully approved with extension to adolescents in June 2026, and letetresgene autoleucel (NY-ESO-1) has a BLA due by end-2026. Checkpoint inhibitors help a minority (alveolar soft-part sarcoma, UPS, angiosarcoma); most sarcomas are immunologically cold. The unsolved problems are metastatic osteosarcoma and Ewing sarcoma (survival unchanged in 30 years), the HLA restriction of TCR therapies, chemoresistance of most adult subtypes, and trial feasibility in diseases with a few hundred cases a year.

State of the art today

  • Subtype-specific targeted and cellular therapies.
  • First engineered T-cell therapies for a solid tumour: afami-cel (full approval and age ≥12 in June 2026) and lete-cel (BLA due 2026) for synovial sarcoma and MRCLS.
  • GIST as a model of genotype-directed sequencing: four approved TKIs, a mutation-specific drug for D842V, and ctDNA-selected phase 3 (INSIGHT).
  • New indications for benign but destructive tumours: nirogacestat (desmoid, 2023) and vimseltinib (TGCT, 2025).
  • Neoadjuvant chemotherapy for high-risk soft-tissue sarcoma validated (ISG-STS 1001), while histotype-tailored chemotherapy was not superior.
  • Ewing sarcoma treatment unified on VDC/IE after Euro Ewing 2012; interval compression and busulfan-melphalan refine it.
  • Withdrawal of tazemetostat (March 2026) removed the only EZH2 drug from epithelioid sarcoma, a reminder that rare-disease approvals rest on thin safety data.
Who it affects

Sarcomas make up ~1% of adult cancers (~13,500 soft-tissue and ~3,900 bone sarcomas in the US per year) and ~15% of paediatric cancers, across >70 histologic subtypes.

Show survival figures (1)

Averages across everyone diagnosed, often years ago. Your stage, subtype, age, fitness and the treatment you receive matter more than the average, and the numbers are improving quickly.

  • Five-year survival is ~65% overall and ~15-30% for metastatic disease.
Group
sarcoma

Where the cases are

Cases by country · GLOBOCAN 2022
All countries →

No country-level case numbers. GLOBOCAN reports Kaposi sarcoma only; soft-tissue and bone sarcomas fall under 'other specified cancers' and cannot be separated.

Standard of care

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Localised STS

Wide excision + radiation; neoadjuvant chemotherapy for high-risk.

NCCN Guidelines: Soft Tissue Sarcoma
IMRT / IGRT (modern external beam)Proton therapyHyperthermia
Advanced

Doxorubicin ± ifosfamide; subtype-directed: imatinib, afami-cel, larotrectinib (tazemetostat was withdrawn in March 2026).

NCCN Guidelines: Soft Tissue Sarcoma
ImatinibAfamitresgene autoleucel
Localised extremity/trunk soft-tissue sarcoma, low grade

Wide resection (limb-salvage) ± radiotherapy for margins or size >5 cm; observation thereafter.

NCCN · Category 1 (surgery ± RT)
Limb-salvage surgery and endoprosthetic reconstructionIMRT / IGRT (modern external beam)FNCLCC grade (soft-tissue sarcoma)
Localised high-risk soft-tissue sarcoma (grade 3, >5 cm, deep)

Neoadjuvant anthracycline-ifosfamide × 3 (ISG-STS 1001) ± preoperative radiotherapy, then wide resection; regional hyperthermia with chemotherapy where available (EORTC 62961).

NCCN · Category 2A (neoadjuvant chemotherapy for h…ESMO-MCBS · A (ISG-STS 1001)
ISG-STS 1001DoxorubicinIfosfamideIMRT / IGRT (modern external beam)HyperthermiaLimb-salvage surgery and endoprosthetic reconstruction
Advanced soft-tissue sarcoma, first line

Doxorubicin 75 mg/m2 (single agent) or doxorubicin-ifosfamide for symptomatic/rapid disease (EORTC 62012: PFS but not OS benefit); histotype exceptions: trabectedin or gemcitabine-docetaxel for leiomyosarcoma, paclitaxel for angiosarcoma.

NCCN · Category 1 (anthracycline-based)
DoxorubicinIfosfamideTrabectedinANNOUNCEHistotype-tailored therapy
Advanced soft-tissue sarcoma, later lines

Trabectedin (L-sarcomas), eribulin (liposarcoma), pazopanib (non-adipocytic), gemcitabine-docetaxel, dacarbazine; pembrolizumab for alveolar soft-part sarcoma or UPS; larotrectinib for NTRK fusion; afami-cel or lete-cel for MAGE-A4/NY-ESO-1+ synovial sarcoma and MRCLS.

NCCN · Category 2A
TrabectedinPazopanibPembrolizumabAfamitresgene autoleucelLetetresgene autoleucelIGNYTE-ESO
GIST, localised

Resection; adjuvant imatinib 3 years for high risk (SSGXVIII), longer under study; none for PDGFRA D842V or SDH-deficient (imatinib-insensitive).

NCCN · Category 1 (imatinib 3 years, high-risk)ESMO-MCBS · A
ImatinibSSGXVIII/AIO (adjuvant imatinib in GIST)
GIST, advanced

Imatinib 400 mg (800 mg for exon 9) → sunitinib → regorafenib → ripretinib (INVICTUS); avapritinib for PDGFRA D842V; ctDNA KIT genotyping to choose ripretinib vs sunitinib second line (INSIGHT); surgery for oligoprogression.

NCCN · Category 1 sequence
ImatinibRipretinibINVICTUSINSIGHTAvapritinibctDNA KIT genotyping → TKI selection in GIST
Osteosarcoma

Neoadjuvant MAP (methotrexate, doxorubicin, cisplatin) → limb-salvage resection → adjuvant MAP; no benefit from adding ifosfamide-etoposide or interferon (EURAMOS-1); metastatic/relapsed: surgery of lung metastases, regorafenib/cabozantinib.

DoxorubicinLimb-salvage surgery and endoprosthetic reconstructionChildren's Oncology Group (COG)
Ewing sarcoma

Interval-compressed VDC/IE (INT-0091, AEWS0031, Euro Ewing 2012) with surgery and/or radiotherapy for local control; high-dose busulfan-melphalan for selected high-risk (Euro-EWING 99 R2); relapse: irinotecan-temozolomide, cabozantinib, trials.

INT-0091 (Ewing sarcoma)Euro Ewing 2012DoxorubicinIfosfamideProton therapy
Desmoid tumour

Active surveillance first (many regress); nirogacestat (DeFi) for progressing symptomatic disease; sorafenib alternative; surgery only for select sites; cryoablation for extra-abdominal tumours.

NCCN · Category 1 (nirogacestat)
NirogacestatDeFiThermal ablation (RFA, microwave, cryo)Intermittent or stop-and-restart nirogacestat in desmoid tumours
Tenosynovial giant cell tumour

Surgery for localised disease; vimseltinib (MOTION) or pexidartinib (REMS for hepatotoxicity) for diffuse disease not amenable to surgery.

NCCN · Category 2A
VimseltinibMOTION

Subtypes & biomarkers

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Subtypes
Biomarkers clinicians test

Target prevalence in this cancer

Target / alterationPrevalenceSource
CDK4/6
>90%
Wikipedia
EZH2
Tazemetostat withdrawn March 2026
>90%
FDA
NTRK
<1% in adult common cancers
>90%
Wikipedia
MDM2
90%
doi.org
KIT
PDGFRA in ~10%
75-80%
Wikipedia
FAP
60-90%
Wikipedia
MAGE-A4
HLA-A*02 needed for TCR-T; ~40-45% of population
60-80%
Wikipedia

How common each drug target or alteration is in this cancer. Population-level and approximate; see the target page for detail. Full matrix.

History

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  1. 1970Adjuvant chemotherapy for osteosarcoma (Jaffe, Rosen) turns a 20% survival into 60%
    Doxorubicin
  2. 1980Limb-salvage surgery shown equivalent to amputation (Rosenberg, NCI)
    Limb-salvage surgery and endoprosthetic reconstruction
  3. 1982Adjuvant radiotherapy improves local control after limb-sparing surgery (NCI randomised trial)
    IMRT / IGRT (modern external beam)
  4. 1998KIT mutations discovered in GIST (Hirota)
    KIT
  5. 2002Imatinib for GIST
    Imatinib
  6. 2002Imatinib approved for GIST: the first solid tumour controlled by a targeted pill
    Imatinib
  7. 2003INT-0091: ifosfamide-etoposide added to Ewing sarcoma therapy
    INT-0091 (Ewing sarcoma)
  8. 2007Trabectedin approved in Europe for soft-tissue sarcoma
    Trabectedin
  9. 2010EORTC 62961: regional hyperthermia with chemotherapy improves survival in high-risk STS
    Hyperthermia
  10. 2012SSGXVIII: 3-year adjuvant imatinib; pazopanib approved (PALETTE)
    SSGXVIII/AIO (adjuvant imatinib in GIST)Pazopanib
  11. 2017ISG-STS 1001: neoadjuvant anthracycline-ifosfamide for high-risk STS
    ISG-STS 1001
  12. 2019ANNOUNCE fails; olaratumab withdrawn
    ANNOUNCE
  13. 2020Ripretinib (INVICTUS) and avapritinib (D842V) approved; tazemetostat approved for epithelioid sarcoma
    RipretinibINVICTUSAvapritinib
  14. 2022Euro Ewing 2012 unifies VDC/IE; DeFi positive
    Euro Ewing 2012DeFi
  15. 2023Nirogacestat: first drug for desmoid tumours
    Nirogacestat
  16. 2024Afami-cel: first TCR-T for a solid tumour
    Afamitresgene autoleucel
  17. 2024Afami-cel: first TCR-T for a solid tumour; MOTION positive; IGNYTE-ESO reports
    Afamitresgene autoleucelMOTIONIGNYTE-ESO
  18. 2025Vimseltinib approved (14 Feb); lete-cel and Tecelra transferred to US WorldMeds
    VimseltinibLetetresgene autoleucelUS WorldMeds
  19. 2026Tazemetostat withdrawn worldwide (9 March); afami-cel full approval and age ≥12 (June); INSIGHT positive
    Afamitresgene autoleucelINSIGHTEZH2

Pipeline

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Phase 2Theranostic pair (peptide radioligand)
FAP-2286 (177Lu / 68Ga)
Phase 2TCR-T (NY-ESO-1)
Letetresgene autoleucel
ApprovedSmall-molecule kinase inhibitor (CSF1R)
Vimseltinib · Romvimza
ApprovedSmall-molecule gamma-secretase inhibitor
Nirogacestat · Ogsiveo
Carbon-ion therapyIGNYTE-ESOINSIGHTctDNA KIT genotyping → TKI selection in GISTExtending sarcoma TCR-T beyond HLA-A*02Intermittent or stop-and-restart nirogacestat in desmoid tumoursLimb-salvage surgery and endoprosthetic reconstruction

Open problems

  • Rarity limits trials.
  • Chemoresistance of most subtypes.
  • Metastatic osteosarcoma and Ewing sarcoma: survival ~20-30%, unchanged for three decades; no targeted or immune therapy has worked.
  • Advanced soft-tissue sarcoma still depends on a 1970s drug (doxorubicin); every attempt to improve first-line survival (olaratumab, evofosfamide, aldoxorubicin) failed.
  • TCR-T is limited to HLA-A*02 carriers, requires antigen screening, and is available at few centres.
  • Most sarcomas are immunologically cold; checkpoint inhibitors help only alveolar soft-part sarcoma, some UPS and angiosarcoma.
  • Rarity fragments evidence: 70+ subtypes, few randomised trials, and reliance on reference-centre expertise that is unevenly distributed.
  • Long-term survivors of childhood bone sarcoma carry anthracycline cardiotoxicity, infertility and second cancers.
  • Epithelioid sarcoma lost its only drug (tazemetostat) in 2026; INI1-loss tumours need new approaches.
  • Desmoid and TGCT therapies raise chronic-dosing questions (ovarian toxicity, cost) in non-malignant diseases.

Trials

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Recruiting now (live from ClinicalTrials.gov)

Recruiting trials near you · live from ClinicalTrials.gov
Sarcomas (soft tissue, bone, GIST)
condition: sarcoma
Open on ClinicalTrials.gov →

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Landmark trials in OnCo

ANNOUNCEDeFiEuro Ewing 2012IGNYTE-ESOINSIGHTINT-0091 (Ewing sarcoma)INVICTUSISG-STS 1001MOTIONSSGXVIII/AIO (adjuvant imatinib in GIST)VOYAGER

Expert centres

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Where the expertise is

Centres linked to this cancer in OnCo

Seeking a second opinion: ask your oncologist for a referral to a high-volume centre; most accept records and pathology by mail or telehealth. In the US, use the NCI's Find a Cancer Center tool or the nonprofit Cancer Commons, which navigates options for advanced cancers at no cost.

Questions to ask

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Bring to your appointment

Questions to ask your oncologist about Sarcomas

Generated from this cancer's standard of care, biomarkers, and pipeline · 39 questions

Newly diagnosed

  1. What is my exact diagnosis, stage, and grade, and which tests established them?
    Why: Everything else follows from an accurate stage and subtype.
  2. Which biomarkers have been tested on my tumour (for example Histologic subtype, KIT/PDGFRA, MAGE-A4 + HLA-A*02, NTRK fusions, INI1 loss), and what were the results?
    Why: These results decide eligibility for targeted therapy, immunotherapy, and trials.
  3. Which subtype is my cancer, and does that change the recommended treatment?
    Why: Recognised subtypes for this cancer include Soft-tissue sarcoma: liposarcoma, leiomyosarcoma, undifferentiated pleomorphic sarcoma, synovial sarcoma, myxofibrosarcoma, angiosarcoma, MPNST, rhabdomyosarcoma, Bone sarcoma: osteosarcoma, Ewing sarcoma, chondrosarcoma, chordoma, Gastrointestinal stromal tumour.
  4. Is germline (inherited) genetic testing recommended for me or my family?
    Why: Inherited variants can change treatment and matter for relatives.

Localised STS

  1. For my situation (localised sts), which of the standard options do you recommend and why?
    Why: Guideline options include: Wide excision + radiation; neoadjuvant chemotherapy for high-risk.

Advanced

  1. For my situation (advanced), which of the standard options do you recommend and why?
    Why: Guideline options include: Doxorubicin ± ifosfamide; subtype-directed: imatinib, afami-cel, larotrectinib (tazemetostat was withdrawn in March 2026).
  2. Am I a candidate for Imatinib, Afamitresgene autoleucel, and what side effects should I expect?
    Why: Knowing the expected toxicities helps you plan work, family, and supportive care.

Localised extremity/trunk soft-tissue sarcoma, low grade

  1. For my situation (localised extremity/trunk soft-tissue sarcoma, low grade), which of the standard options do you recommend and why?
    Why: Guideline options include: Wide resection (limb-salvage) ± radiotherapy for margins or size >5 cm; observation thereafter.

Localised high-risk soft-tissue sarcoma (grade 3, >5 cm, deep)

  1. For my situation (localised high-risk soft-tissue sarcoma (grade 3, >5 cm, deep)), which of the standard options do you recommend and why?
    Why: Guideline options include: Neoadjuvant anthracycline-ifosfamide × 3 (ISG-STS 1001) ± preoperative radiotherapy, then wide resection; regional hyperthermia with chemotherapy where available (EORTC 62961).
  2. Am I a candidate for Doxorubicin, Ifosfamide, and what side effects should I expect?
    Why: Knowing the expected toxicities helps you plan work, family, and supportive care.
  3. How do the results of ISG-STS 1001 apply to someone like me?
    Why: Trial populations differ from individual patients; ask how closely you match.

Advanced soft-tissue sarcoma, first line

  1. For my situation (advanced soft-tissue sarcoma, first line), which of the standard options do you recommend and why?
    Why: Guideline options include: Doxorubicin 75 mg/m2 (single agent) or doxorubicin-ifosfamide for symptomatic/rapid disease (EORTC 62012: PFS but not OS benefit); histotype exceptions: trabectedin or gemcitabine-docetaxel for leiomyosarcoma, paclitaxel for angiosarcoma.
  2. Am I a candidate for Doxorubicin, Ifosfamide, Trabectedin, and what side effects should I expect?
    Why: Knowing the expected toxicities helps you plan work, family, and supportive care.
  3. How do the results of ANNOUNCE apply to someone like me?
    Why: Trial populations differ from individual patients; ask how closely you match.

Advanced soft-tissue sarcoma, later lines

  1. For my situation (advanced soft-tissue sarcoma, later lines), which of the standard options do you recommend and why?
    Why: Guideline options include: Trabectedin (L-sarcomas), eribulin (liposarcoma), pazopanib (non-adipocytic), gemcitabine-docetaxel, dacarbazine; pembrolizumab for alveolar soft-part sarcoma or UPS; larotrectinib for NTRK fusion; afami-cel or lete-cel for MAGE-A4/NY-ESO-1+ synovial sarcoma and MRCLS.
  2. Am I a candidate for Trabectedin, Pazopanib, Pembrolizumab or related drugs, and what side effects should I expect?
    Why: Knowing the expected toxicities helps you plan work, family, and supportive care.
  3. How do the results of IGNYTE-ESO apply to someone like me?
    Why: Trial populations differ from individual patients; ask how closely you match.

GIST, localised

  1. For my situation (gist, localised), which of the standard options do you recommend and why?
    Why: Guideline options include: Resection; adjuvant imatinib 3 years for high risk (SSGXVIII), longer under study; none for PDGFRA D842V or SDH-deficient (imatinib-insensitive).
  2. Am I a candidate for Imatinib, and what side effects should I expect?
    Why: Knowing the expected toxicities helps you plan work, family, and supportive care.
  3. How do the results of SSGXVIII/AIO (adjuvant imatinib in GIST) apply to someone like me?
    Why: Trial populations differ from individual patients; ask how closely you match.

GIST, advanced

  1. For my situation (gist, advanced), which of the standard options do you recommend and why?
    Why: Guideline options include: Imatinib 400 mg (800 mg for exon 9) → sunitinib → regorafenib → ripretinib (INVICTUS); avapritinib for PDGFRA D842V; ctDNA KIT genotyping to choose ripretinib vs sunitinib second line (INSIGHT); surgery for oligoprogression.
  2. Am I a candidate for Imatinib, Ripretinib, Avapritinib, and what side effects should I expect?
    Why: Knowing the expected toxicities helps you plan work, family, and supportive care.
  3. How do the results of INVICTUS and INSIGHT apply to someone like me?
    Why: Trial populations differ from individual patients; ask how closely you match.

Osteosarcoma

  1. For my situation (osteosarcoma), which of the standard options do you recommend and why?
    Why: Guideline options include: Neoadjuvant MAP (methotrexate, doxorubicin, cisplatin) → limb-salvage resection → adjuvant MAP; no benefit from adding ifosfamide-etoposide or interferon (EURAMOS-1); metastatic/relapsed: surgery of lung metastases, regorafenib/cabozantinib.
  2. Am I a candidate for Doxorubicin, and what side effects should I expect?
    Why: Knowing the expected toxicities helps you plan work, family, and supportive care.

Ewing sarcoma

  1. For my situation (ewing sarcoma), which of the standard options do you recommend and why?
    Why: Guideline options include: Interval-compressed VDC/IE (INT-0091, AEWS0031, Euro Ewing 2012) with surgery and/or radiotherapy for local control; high-dose busulfan-melphalan for selected high-risk (Euro-EWING 99 R2); relapse: irinotecan-temozolomide, cabozantinib, trials.
  2. Am I a candidate for Doxorubicin, Ifosfamide, and what side effects should I expect?
    Why: Knowing the expected toxicities helps you plan work, family, and supportive care.
  3. How do the results of INT-0091 (Ewing sarcoma) and Euro Ewing 2012 apply to someone like me?
    Why: Trial populations differ from individual patients; ask how closely you match.

Desmoid tumour

  1. For my situation (desmoid tumour), which of the standard options do you recommend and why?
    Why: Guideline options include: Active surveillance first (many regress); nirogacestat (DeFi) for progressing symptomatic disease; sorafenib alternative; surgery only for select sites; cryoablation for extra-abdominal tumours.
  2. Am I a candidate for Nirogacestat, and what side effects should I expect?
    Why: Knowing the expected toxicities helps you plan work, family, and supportive care.
  3. How do the results of DeFi apply to someone like me?
    Why: Trial populations differ from individual patients; ask how closely you match.

Tenosynovial giant cell tumour

  1. For my situation (tenosynovial giant cell tumour), which of the standard options do you recommend and why?
    Why: Guideline options include: Surgery for localised disease; vimseltinib (MOTION) or pexidartinib (REMS for hepatotoxicity) for diffuse disease not amenable to surgery.
  2. Am I a candidate for Vimseltinib, and what side effects should I expect?
    Why: Knowing the expected toxicities helps you plan work, family, and supportive care.
  3. How do the results of MOTION apply to someone like me?
    Why: Trial populations differ from individual patients; ask how closely you match.

Any stage

  1. Are there clinical trials I could join, for example of FAP-2286 (177Lu / 68Ga), Carbon-ion therapy, Letetresgene autoleucel, IGNYTE-ESO?
    Why: Trials are how the next standard of care is set; asking early keeps options open.
  2. Would a second opinion at a high-volume centre change anything, and can you help arrange it?
    Why: Rare or high-stakes decisions benefit from a centre that treats many similar patients.
  3. What supportive care (symptom control, nutrition, exercise, mental health, financial help) is available from the start?
    Why: Supportive care improves quality of life and helps patients complete treatment.
  4. I read that “Rarity limits trials”. How does that affect my plan?
    Why: Open problems are where trials and second opinions matter most.
  5. I read that “Chemoresistance of most subtypes”. How does that affect my plan?
    Why: Open problems are where trials and second opinions matter most.

Print this page for your appointment (your browser's print command). These prompts are for discussion; your clinical team knows your case.

Everything relevant

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Direct links plus the targets, companies, and technologies of this cancer's products.

technologies

24
Anti-angiogenic therapyCarbon-ion therapyCardio-oncologyComprehensive genomic profilingCytotoxic chemotherapyEpigenetic drugs (HDAC, DNMT, EZH2, IDH, menin, BET)FAPI PETHyperthermiaHypoxia-activated prodrugsImmune checkpoint inhibitorsIMRT / IGRT (modern external beam)Intraoperative radiotherapy (IORT)Intravesical therapy (BCG, chemotherapy, devices, gene and viral therapy)Isolated limb perfusion and infusionLimb-salvage surgery and endoprosthetic reconstructionLiquid biopsy (ctDNA)Metabolic therapy: starving the tumour of a nutrientProton therapyRadiodynamic therapy and radiosensitising nanoparticlesRadioligand therapy (beta emitters)Small-molecule kinase inhibitorsTCR-T cell therapyThermal ablation (RFA, microwave, cryo)Topoisomerase-I inhibitors (and ADC payloads)

targets

18
ALKCDK4/6CSF1REWSR1-FLI1 fusionEZH2FAPGD2 (disialoganglioside)KITMAGE-A4MDM2METNTRKPD-1PDGFRAPRAMERETROS1VEGF / VEGFR

drugs

23
ApprovedTCR-T (MAGE-A4)
Afamitresgene autoleucel · Tecelra
ApprovedSmall-molecule kinase inhibitor (PDGFRA D842V / KIT)
Avapritinib · Ayvakit
Not mapped hereSmall-molecule ALK/ROS1/MET TKI (first generation)
Crizotinib · Xalkori
ApprovedCytotoxic chemotherapy (anthracycline)
Doxorubicin · Adriamycin
Not mapped hereSmall-molecule TRK/ROS1/ALK TKI (CNS-penetrant)
Entrectinib · Rozlytrek
Not mapped hereSynthetic halichondrin B analogue (microtubule dynamics inhibitor)
Eribulin · Halaven
Phase 2Theranostic pair (peptide radioligand)
FAP-2286 (177Lu / 68Ga)
Not mapped hereNucleoside analogue (deoxycytidine)
Gemcitabine · Gemzar / Infugem
ApprovedCytotoxic chemotherapy (alkylating agent)
Ifosfamide · Ifex
ApprovedSmall-molecule kinase inhibitor (BCR-ABL, KIT, PDGFRA)
Imatinib · Gleevec
Not mapped hereSmall-molecule pan-TRK inhibitor (first generation)
Larotrectinib · Vitrakvi
Phase 2TCR-T (NY-ESO-1)
Letetresgene autoleucel
ApprovedSmall-molecule gamma-secretase inhibitor
Nirogacestat · Ogsiveo
ApprovedSmall-molecule kinase inhibitor (VEGFR/PDGFR/KIT)
Pazopanib · Votrient
ApprovedCytotoxic (liposomal anthracycline)
Pegylated liposomal doxorubicin · Doxil / Caelyx
ApprovedMonoclonal antibody (anti-PD-1)
Pembrolizumab · Keytruda / Keytruda Qlex (SC)
Not mapped hereSmall-molecule CSF1R/KIT/FLT3 inhibitor
Pexidartinib · Turalio
ApprovedSmall-molecule multi-kinase inhibitor
Regorafenib · Stivarga
ApprovedSmall-molecule kinase inhibitor (KIT/PDGFRA switch-control)
Ripretinib · Qinlock
ApprovedSmall-molecule multi-kinase inhibitor (VEGFR, PDGFR, KIT)
Sunitinib · Sutent
WithdrawnSmall-molecule EZH2 inhibitor
Tazemetostat · Tazverik
ApprovedCytotoxic (DNA minor-groove binder)
Trabectedin · Yondelis
ApprovedSmall-molecule kinase inhibitor (CSF1R)
Vimseltinib · Romvimza

companies

17
AdaptimmuneBayerDaiichi SankyoDeciphera Pharmaceuticals (Ono)EisaiEli Lilly (incl. Loxo)ImmaticsIpsenJohnson & JohnsonMerck & Co. (MSD)NovartisPfizer (incl. Seagen)PharmaMarRoche / GenentechSanofiSpringWorks Therapeutics (Merck KGaA)US WorldMeds

institutions

31
A.C. Camargo Cancer CenterCentre Léon BérardCentre Oscar LambretChildren's Cancer and Leukaemia GroupChildren's Cancer Hospital Egypt 57357Children's Oncology Group (COG)Chris O'Brien LifehouseEORTCErasmus MC Cancer InstituteFondazione IRCCS Istituto Nazionale dei TumoriGarvan Institute of Medical Research / Kinghorn Cancer CentreGunma University Heavy Ion Medical CenterGustave RoussyInstitut BergoniéInstituto Português de Oncologia de Lisboa Francisco GentilIRCCS Istituto Romagnolo per lo Studio dei Tumori 'Dino Amadori' (IRST)Leiden University Medical CenterMaria Skłodowska-Curie National Research Institute of OncologyMD Anderson Cancer CenterMemorial Sloan Kettering Cancer CenterOslo University Hospital – The Norwegian Radium HospitalPeking University Cancer HospitalPrincess Máxima Center for Pediatric OncologyQST Hospital (National Institutes for Quantum Science and Technology)SIOP Europe – European Society for Paediatric OncologySociety of Surgical OncologySydney Children's Hospitals Network / Children's Cancer InstituteTel Aviv Sourasky Medical CenterThe Ohio State University Comprehensive Cancer Center – James Cancer Hospital and Solove Research InstituteThe Royal MarsdenWest German Cancer Center (WTZ), University Hospital Essen

pathways

4
p53 / RB / cell-cycle checkpointSWI/SNF chromatin remodellingThe cell-cycle engine (cyclins & CDKs)The p53 network (guardian of the genome)

terms

12
Anthracyclines (doxorubicin, epirubicin)Dose-dense and metronomic chemotherapyFNCLCC grade (soft-tissue sarcoma)Histotype-tailored therapyHLA-A*02:01 restrictionLi-Fraumeni syndrome (germline TP53)Limb-salvage surgeryObjective response rate (ORR)Progression-free survival (PFS)RetroperitoneumSarcoma (tissue type)Wide local excision

trials

11
ANNOUNCEDeFiEuro Ewing 2012IGNYTE-ESOINSIGHTINT-0091 (Ewing sarcoma)INVICTUSISG-STS 1001MOTIONSSGXVIII/AIO (adjuvant imatinib in GIST)VOYAGER

pairings

2
Caution: cumulative anthracycline dose and the heartctDNA KIT genotyping → TKI selection in GIST

ideas

37
A funded expert second opinion for every new high-stakes or rare cancer diagnosisA non-profit pharmaceutical company for the cancers markets ignoreA same-week expert second opinion for every rare cancer diagnosisA survivorship passport app for adolescent and young adult survivorsAdvance market commitments for paediatric and rare cancer drugsAn international consortium pooling the outcome of every treated child with cancerAn open model bank for the rare tumours nobody has models forAn open organoid bank for cancers too rare to have modelsAttack extrachromosomal DNA, the engine of oncogene amplificationDegraders for the fusion proteins that drive childhood sarcomasDevelop drugs in children first when the target is a children's targetEscrow a share of adult revenue until the paediatric study is doneExtending sarcoma TCR-T beyond HLA-A*02FAP theranostics as a pan-cancer stromal strategyGroup trials by broken mechanism, not by organ or single mutationGrow immune command posts inside tumoursImplant a tiny device that tests twenty drugs inside the patient's own tumourInhaled immune therapy to make the lung hostile to arriving tumour cellsIntermittent or stop-and-restart nirogacestat in desmoid tumoursLet adolescents from age 12 into adult trials when the cancer biology is the sameLet patients themselves donate their records and samples for ultra-rare cancersMandatory national virtual tumour boards for rare and complex cancersMilestone-based venture philanthropy with royalties recycled into the pipelineOne global rare cancer network with n-of-1 and Bayesian trial frameworksOne legal framework for pooling rare cancer data across bordersOne standing umbrella trial for all rare cancers in a countryOpen-source drug discovery to clinical proof of concept for neglected cancersPartial lottery funding for good proposals in under-funded cancersPatent-free open-source development of repurposed and off-patent cancer drugsPay a prize for rare cancer drugs instead of hoping for a marketPet dogs with spontaneous cancer as a bridge before human trialsRandomise inside the registry that already follows every patientTest drugs on the patient's own cancer cells when there is no trial to joinThe pathology lab triggers a trial referral the day a rare cancer is diagnosedTrial-in-a-box: a preconfigured protocol kit any hospital can open for a rare cancerTurn one tumour into a vaccine to treat all the othersWhole-body MRI plus blood DNA surveillance for people with Li-Fraumeni syndrome

people

15
Alessandro GronchiBrigitte C. WidemannCharles D. BlankeChristopher R. VakocCrystal L. MackallDouglas S. HawkinsJean-Yves BlayKate GrangerMin-Hee RyuOlivier DelattrePeter W. T. PistersShunji TakahashiStefan FröhlingTerry FoxYoon-Koo Kang

bottlenecks

3
Rare and paediatric cancers without marketsThe undruggable driversWrong doses

key papers

2
SPEARHEAD-1: afami-cel, the first engineered T-cell receptor therapy approved for a solid tumour, in synovial sarcomaSPEARHEAD-1: afamitresgene autoleucel, the first engineered T-cell receptor therapy approved for a solid tumour, in synovial sarcoma

Key papers

2top
translationalThe Lancet 2024changed practice
SPEARHEAD-1: afami-cel, the first engineered T-cell receptor therapy approved for a solid tumour, in synovial sarcoma

Afami-cel showed that T cells can be redirected against an intracellular cancer antigen, something CAR-T cannot do, and produced meaningful responses in a rare sarcoma with few options. It opens a path to TCR-T against other shared antigens and neoantigens. Restriction to one HLA type and one antigen, plus complex manufacturing, means it will help a small, defined group.

rctThe Lancet 2024changed practice
SPEARHEAD-1: afamitresgene autoleucel, the first engineered T-cell receptor therapy approved for a solid tumour, in synovial sarcoma

Patients with advanced synovial sarcoma, a rare cancer of young adults with few effective drugs, now have an approved cell therapy that produces responses lasting about a year in a substantial minority, if their tissue type and tumour antigen match. It proves that engineered T cells can work against a solid tumour when a good target is present, which had been elusive. It is not a cure for most, requires specialised centres, and only a minority of patients are eligible.

Latest papers

top
Literature trend1,178 papers in the last 12 months-2% vs prior 12How this is computed
Latest papers · live from Europe PMC
Open in Europe PMC

Query for this cancer: (TITLE:"Sarcomas" OR ABSTRACT:"Sarcomas" OR TITLE:"soft tissue, bone, GIST" OR ABSTRACT:"soft tissue, bone, GIST") AND (treatment OR therapy OR trial OR survival OR diagnosis). Results are unfiltered search hits about Sarcomas (soft tissue, bone, GIST), not a curated reading list.

Connected

179top

Pages like this

not linked directly; found by shared links

technologies

17
Carbon-ion therapyCardio-oncologyCytotoxic chemotherapyEpigenetic drugs (HDAC, DNMT, EZH2, IDH, menin, BET)HyperthermiaHypoxia-activated prodrugsIMRT / IGRT (modern external beam)Intraoperative radiotherapy (IORT)Isolated limb perfusion and infusionLimb-salvage surgery and endoprosthetic reconstructionLiquid biopsy (ctDNA)Metabolic therapy: starving the tumour of a nutrientProton therapyRadiodynamic therapy and radiosensitising nanoparticlesSmall-molecule kinase inhibitorsTCR-T cell therapyThermal ablation (RFA, microwave, cryo)

targets

13
CDK4/6CSF1REWSR1-FLI1 fusionEZH2FAPGD2 (disialoganglioside)KITMAGE-A4MDM2NTRKPDGFRAPRAMEVEGF / VEGFR

drugs

23
ApprovedTCR-T (MAGE-A4)
Afamitresgene autoleucel · Tecelra
ApprovedSmall-molecule kinase inhibitor (PDGFRA D842V / KIT)
Avapritinib · Ayvakit
Not mapped hereSmall-molecule ALK/ROS1/MET TKI (first generation)
Crizotinib · Xalkori
ApprovedCytotoxic chemotherapy (anthracycline)
Doxorubicin · Adriamycin
Not mapped hereSmall-molecule TRK/ROS1/ALK TKI (CNS-penetrant)
Entrectinib · Rozlytrek
Not mapped hereSynthetic halichondrin B analogue (microtubule dynamics inhibitor)
Eribulin · Halaven
Phase 2Theranostic pair (peptide radioligand)
FAP-2286 (177Lu / 68Ga)
Not mapped hereNucleoside analogue (deoxycytidine)
Gemcitabine · Gemzar / Infugem
ApprovedCytotoxic chemotherapy (alkylating agent)
Ifosfamide · Ifex
ApprovedSmall-molecule kinase inhibitor (BCR-ABL, KIT, PDGFRA)
Imatinib · Gleevec
Not mapped hereSmall-molecule pan-TRK inhibitor (first generation)
Larotrectinib · Vitrakvi
Phase 2TCR-T (NY-ESO-1)
Letetresgene autoleucel
ApprovedSmall-molecule gamma-secretase inhibitor
Nirogacestat · Ogsiveo
ApprovedSmall-molecule kinase inhibitor (VEGFR/PDGFR/KIT)
Pazopanib · Votrient
ApprovedCytotoxic (liposomal anthracycline)
Pegylated liposomal doxorubicin · Doxil / Caelyx
ApprovedMonoclonal antibody (anti-PD-1)
Pembrolizumab · Keytruda / Keytruda Qlex (SC)
Not mapped hereSmall-molecule CSF1R/KIT/FLT3 inhibitor
Pexidartinib · Turalio
ApprovedSmall-molecule multi-kinase inhibitor
Regorafenib · Stivarga
ApprovedSmall-molecule kinase inhibitor (KIT/PDGFRA switch-control)
Ripretinib · Qinlock
ApprovedSmall-molecule multi-kinase inhibitor (VEGFR, PDGFR, KIT)
Sunitinib · Sutent
WithdrawnSmall-molecule EZH2 inhibitor
Tazemetostat · Tazverik
ApprovedCytotoxic (DNA minor-groove binder)
Trabectedin · Yondelis
ApprovedSmall-molecule kinase inhibitor (CSF1R)
Vimseltinib · Romvimza

companies

9
AdaptimmuneDeciphera Pharmaceuticals (Ono)ImmaticsJohnson & JohnsonNovartisPharmaMarSanofiSpringWorks Therapeutics (Merck KGaA)US WorldMeds

institutions

31
A.C. Camargo Cancer CenterCentre Léon BérardCentre Oscar LambretChildren's Cancer and Leukaemia GroupChildren's Cancer Hospital Egypt 57357Children's Oncology Group (COG)Chris O'Brien LifehouseEORTCErasmus MC Cancer InstituteFondazione IRCCS Istituto Nazionale dei TumoriGarvan Institute of Medical Research / Kinghorn Cancer CentreGunma University Heavy Ion Medical CenterGustave RoussyInstitut BergoniéInstituto Português de Oncologia de Lisboa Francisco GentilIRCCS Istituto Romagnolo per lo Studio dei Tumori 'Dino Amadori' (IRST)Leiden University Medical CenterMaria Skłodowska-Curie National Research Institute of OncologyMD Anderson Cancer CenterMemorial Sloan Kettering Cancer CenterOslo University Hospital – The Norwegian Radium HospitalPeking University Cancer HospitalPrincess Máxima Center for Pediatric OncologyQST Hospital (National Institutes for Quantum Science and Technology)SIOP Europe – European Society for Paediatric OncologySociety of Surgical OncologySydney Children's Hospitals Network / Children's Cancer InstituteTel Aviv Sourasky Medical CenterThe Ohio State University Comprehensive Cancer Center – James Cancer Hospital and Solove Research InstituteThe Royal MarsdenWest German Cancer Center (WTZ), University Hospital Essen

pathways

4
p53 / RB / cell-cycle checkpointSWI/SNF chromatin remodellingThe cell-cycle engine (cyclins & CDKs)The p53 network (guardian of the genome)

terms

12
Anthracyclines (doxorubicin, epirubicin)Dose-dense and metronomic chemotherapyFNCLCC grade (soft-tissue sarcoma)Histotype-tailored therapyHLA-A*02:01 restrictionLi-Fraumeni syndrome (germline TP53)Limb-salvage surgeryObjective response rate (ORR)Progression-free survival (PFS)RetroperitoneumSarcoma (tissue type)Wide local excision

trials

11
ANNOUNCEDeFiEuro Ewing 2012IGNYTE-ESOINSIGHTINT-0091 (Ewing sarcoma)INVICTUSISG-STS 1001MOTIONSSGXVIII/AIO (adjuvant imatinib in GIST)VOYAGER

pairings

2
Caution: cumulative anthracycline dose and the heartctDNA KIT genotyping → TKI selection in GIST

ideas

37
A funded expert second opinion for every new high-stakes or rare cancer diagnosisA non-profit pharmaceutical company for the cancers markets ignoreA same-week expert second opinion for every rare cancer diagnosisA survivorship passport app for adolescent and young adult survivorsAdvance market commitments for paediatric and rare cancer drugsAn international consortium pooling the outcome of every treated child with cancerAn open model bank for the rare tumours nobody has models forAn open organoid bank for cancers too rare to have modelsAttack extrachromosomal DNA, the engine of oncogene amplificationDegraders for the fusion proteins that drive childhood sarcomasDevelop drugs in children first when the target is a children's targetEscrow a share of adult revenue until the paediatric study is doneExtending sarcoma TCR-T beyond HLA-A*02FAP theranostics as a pan-cancer stromal strategyGroup trials by broken mechanism, not by organ or single mutationGrow immune command posts inside tumoursImplant a tiny device that tests twenty drugs inside the patient's own tumourInhaled immune therapy to make the lung hostile to arriving tumour cellsIntermittent or stop-and-restart nirogacestat in desmoid tumoursLet adolescents from age 12 into adult trials when the cancer biology is the sameLet patients themselves donate their records and samples for ultra-rare cancersMandatory national virtual tumour boards for rare and complex cancersMilestone-based venture philanthropy with royalties recycled into the pipelineOne global rare cancer network with n-of-1 and Bayesian trial frameworksOne legal framework for pooling rare cancer data across bordersOne standing umbrella trial for all rare cancers in a countryOpen-source drug discovery to clinical proof of concept for neglected cancersPartial lottery funding for good proposals in under-funded cancersPatent-free open-source development of repurposed and off-patent cancer drugsPay a prize for rare cancer drugs instead of hoping for a marketPet dogs with spontaneous cancer as a bridge before human trialsRandomise inside the registry that already follows every patientTest drugs on the patient's own cancer cells when there is no trial to joinThe pathology lab triggers a trial referral the day a rare cancer is diagnosedTrial-in-a-box: a preconfigured protocol kit any hospital can open for a rare cancerTurn one tumour into a vaccine to treat all the othersWhole-body MRI plus blood DNA surveillance for people with Li-Fraumeni syndrome

people

15
Alessandro GronchiBrigitte C. WidemannCharles D. BlankeChristopher R. VakocCrystal L. MackallDouglas S. HawkinsJean-Yves BlayKate GrangerMin-Hee RyuOlivier DelattrePeter W. T. PistersShunji TakahashiStefan FröhlingTerry FoxYoon-Koo Kang

bottlenecks

3
Rare and paediatric cancers without marketsThe undruggable driversWrong doses

key papers

2
SPEARHEAD-1: afami-cel, the first engineered T-cell receptor therapy approved for a solid tumour, in synovial sarcomaSPEARHEAD-1: afamitresgene autoleucel, the first engineered T-cell receptor therapy approved for a solid tumour, in synovial sarcoma