OnCo
cancersCancer

Glioma & glioblastoma

Glioblastoma is the most lethal brain tumour, barely improved since 2005. Low-grade IDH-mutant gliomas, by contrast, got their first targeted drug in 2024.

Gliomas are classified by the WHO 2021 system on molecular grounds: IDH-wild-type glioblastoma (grade 4, ~50% of gliomas, median age 65, median survival ~15 months with maximal therapy), IDH-mutant astrocytoma (grades 2-4) and 1p/19q-codeleted oligodendroglioma (better prognosis, decades of survival possible), and paediatric-type tumours including H3 K27M-mutant diffuse midline glioma (median survival ~11 months) and BRAF-altered low-grade glioma (the commonest childhood brain tumour, rarely fatal but chronically disabling). The two shared barriers are the blood-brain barrier, which excludes most drugs, and diffuse infiltration, which makes complete resection impossible.

Glioblastoma treatment has been static since 2005: maximal safe resection (improved by 5-ALA fluorescence, intraoperative MRI, and awake mapping), radiotherapy with concurrent and adjuvant temozolomide (Stupp), and tumour treating fields (EF-14). MGMT promoter methylation predicts temozolomide benefit; unmethylated patients derive little. Every major systemic trial since has failed: bevacizumab (PFS only), rindopepimut (ACT IV), nivolumab (CheckMate 143, 498, 548), depatuxizumab mafodotin (INTELLANCE-1), and many more. At recurrence, lomustine, re-resection, re-irradiation, LITT, and bevacizumab for oedema are the options, with median survival under a year. DCVax-L's externally controlled phase 3 remains contested.

Progress has come at the edges. Vorasidenib (INDIGO, approved 2024) is the first targeted therapy for grade 2 IDH-mutant glioma, delaying radiation and chemotherapy by years. Tovorafenib (2024) is the first targeted therapy for BRAF-altered paediatric low-grade glioma. Dordaviprone (August 2025) is the first drug approved for H3 K27M diffuse midline glioma. Methylation-based classification and intraoperative nanopore sequencing have transformed diagnosis. For glioblastoma itself the most promising directions are locoregional CAR-T (IL13Rα2, GD2, multi-target), focused-ultrasound and LITT-based barrier opening to deliver ADCs, radioconjugates, and chemotherapy, neoadjuvant immunotherapy with window designs, personalised neoantigen vaccines (NeoVax), and combinations built on the unmethylated-MGMT population where temozolomide adds nothing.

State of the art today

  • Vorasidenib in low-grade glioma.
  • Methylation-based diagnosis.
  • TTFields.
  • Three first-in-class targeted approvals for glioma subtypes in 2024-25: vorasidenib (IDH-mutant), tovorafenib (BRAF paediatric), dordaviprone (H3 K27M).
  • Molecular classification (WHO 2021, methylation classifier, intraoperative nanopore) now defines diagnosis.
  • Locoregional CAR-T produces objective responses in recurrent glioblastoma and DIPG, though transient.
  • Focused ultrasound opens the blood-brain barrier in humans with 4-6x higher drug delivery; efficacy trials underway.
Who it affects
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.

  • About 300,000 CNS tumours occur per year; glioblastoma median survival is about 15 months.

Where the cases are

Cases by country · GLOBOCAN 2022
All countries →

Site: Brain, central nervous system (shared total; subtype split not reported). World: 321,731 new cases, 248,500 deaths.

#CountryNew casesDeaths
1China87,49856,648
2India32,57427,990
3United States of America24,94018,545
4Brazil12,25310,998
5Russian Federation10,8928,587
6Germany7,9556,570
7France (metropolitan)6,9215,049
8Türkiye6,5116,016
9Italy6,3454,824
10United Kingdom5,8114,840

All brain and CNS tumours combined; glioblastoma is roughly half of malignant gliomas and GLOBOCAN does not report grade or IDH status.

Standard of care

9top
Glioblastoma

Resection → RT + temozolomide → TTFields; lomustine/bevacizumab at relapse.

NCCN Guidelines: Central Nervous System Cancers
Fluorescence-guided surgeryIMRT / IGRT (modern external beam)Optune / Optune Pax (TTFields)
IDH-mutant grade 2

Resection → vorasidenib or observation; RT/PCV for high-risk.

NCCN Guidelines: Central Nervous System Cancers
Vorasidenib
Diagnosis

MRI with contrast; maximal safe resection with 5-ALA and intraoperative mapping; integrated histo-molecular diagnosis with methylation classification where available.

MRIFluorescence-guided surgeryDNA methylation profiling
Glioblastoma, newly diagnosed

Radiotherapy 60 Gy (hypofractionated in elderly) with concurrent and 6 cycles adjuvant temozolomide; TTFields with maintenance temozolomide; trials for MGMT-unmethylated patients.

EORTC 26981 / NCIC CE.3 (Stupp trial)TemozolomideEF-14Optune / Optune Pax (TTFields)IMRT / IGRT (modern external beam)MGMT promoter methylation
Glioblastoma, recurrent

Re-resection or LITT if feasible; lomustine; bevacizumab for oedema/steroid sparing; re-irradiation; clinical trial (CAR-T, FUS-BBB, vaccines) strongly preferred.

Lomustine (CCNU)Bevacizumab (glioblastoma use)Laser interstitial thermal therapy (LITT)CAR-T for glioma (IL13Rα2, GD2, EGFRvIII, multi-target)Focused-ultrasound blood-brain barrier openingSBRT / SABR (stereotactic radiotherapy)
IDH-mutant grade 2 glioma

Maximal resection; vorasidenib for residual/recurrent disease (INDIGO); radiotherapy plus PCV or temozolomide for high-risk or progressive disease.

VorasidenibINDIGO
Oligodendroglioma grade 3 / astrocytoma grade 3

Radiotherapy plus PCV (RTOG 9402, EORTC 26951) or temozolomide (CATNON).

TemozolomideIMRT / IGRT (modern external beam)
H3 K27M diffuse midline glioma

Radiotherapy; dordaviprone at progression (2025); GD2 CAR-T and ONC201 first-line trials.

DordaviproneCAR-T for glioma (IL13Rα2, GD2, EGFRvIII, multi-target)
Paediatric low-grade glioma

Resection where safe; chemotherapy (carboplatin/vincristine) or tovorafenib / dabrafenib-trametinib for BRAF-altered relapsed disease; avoid radiation in young children.

Tovorafenib

Subtypes & biomarkers

top
Subtypes
  • Glioblastoma, IDH-wild-type (WHO grade 4; TERT promoter, EGFR amplification, +7/−10)
  • Astrocytoma, IDH-mutant (grades 2-4; CDKN2A/B deletion defines grade 4)
  • Oligodendroglioma, IDH-mutant and 1p/19q-codeleted (grades 2-3)
  • Diffuse midline glioma, H3 K27M-altered (including DIPG)
  • Paediatric low-grade glioma (BRAF fusion or V600E, NF1)
  • Diffuse hemispheric glioma H3 G34-mutant; infant-type hemispheric glioma (NTRK/ALK/ROS1 fusions)
Biomarkers clinicians test

Target prevalence in this cancer

Target / alterationPrevalenceSource
IDH1 / IDH2
<10% in primary glioblastoma
70-80%
cBioPortal (TCGA)
EGFR
EGFRvIII in ~25-30%
40-50%
cBioPortal (TCGA)
PRMT5 (MTAP-deleted cancers)
40%
PDGFRA
15%

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

History

16top
  1. 1926Bailey and Cushing classify gliomas

    Histologic classification that lasted, in essence, until 2016.

  2. 1978Radiotherapy proven to extend survival (BTSG)

    Whole-brain then involved-field radiation becomes standard.

    IMRT / IGRT (modern external beam)
  3. 1999Temozolomide approved (anaplastic astrocytoma)
    Temozolomide
  4. 2005Stupp regimen: temozolomide + RT
  5. 2008IDH1 mutations discovered in glioma

    Parsons/Vogelstein glioblastoma genome sequencing; reclassification follows.

    IDH1 / IDH2
  6. 2009Bevacizumab accelerated approval at recurrence

    Radiographic response without survival benefit.

    Bevacizumab (glioblastoma use)
  7. 2014AVAglio / RTOG 0825: bevacizumab no OS benefit; 5-ALA and methylation classifier emerge
    Bevacizumab (glioblastoma use)Fluorescence-guided surgeryDNA methylation profiling
  8. 2015TTFields improves OS (EF-14)
    Optune / Optune Pax (TTFields)
  9. 2016WHO 2016 integrates molecular markers; ACT IV vaccine fails
    ACT IVRindopepimut
  10. 2017CheckMate 143: immunotherapy fails at recurrence
    CheckMate 548 & CheckMate 143 & CheckMate 498
  11. 2021WHO 2021: IDH-wild-type glioblastoma defined molecularly
  12. 2022GD2 CAR-T responses in DIPG (Stanford)
    CAR-T for glioma (IL13Rα2, GD2, EGFRvIII, multi-target)
  13. 2023INDIGO: vorasidenib in grade 2 IDH-mutant glioma; DCVax-L contested publication
    INDIGODCVax-L
  14. 2024Vorasidenib approved
    Vorasidenib
  15. 2025Dordaviprone approved for H3 K27M diffuse midline glioma

    6 August 2025; first systemic therapy for the disease.

    Dordaviprone
  16. 2026ASCO 2026: NeoVax personalised vaccine immune responses; multi-target CAR-T; tovorafenib EU approval
    TovorafenibCAR-T for glioma (IL13Rα2, GD2, EGFRvIII, multi-target)Neoadjuvant immunotherapy with surgical window for glioblastoma

Pipeline

13top
Phase 3Autologous dendritic cell vaccine (tumour lysate)
DCVax-L
ApprovedSmall-molecule imipridone (ClpP agonist / DRD2 antagonist)
Dordaviprone · Modeyso
ApprovedSmall-molecule type II RAF inhibitor
Tovorafenib · Ojemda
Armoured, logic-gated & next-gen CARsBoron neutron capture therapyHyperthermiaCAR-T for glioma (IL13Rα2, GD2, EGFRvIII, multi-target)Focused-ultrasound blood-brain barrier openingLaser interstitial thermal therapy (LITT)Neoadjuvant immunotherapy with surgical window for glioblastomaFocused-ultrasound BBB opening to deliver ADCs and radioligands to gliomaPersonalised neoantigen (mRNA) vaccinesIn vivo CAR-T

Open problems

  • Blood-brain barrier.
  • Immunologically cold, heterogeneous, infiltrative.
  • No progress in glioblastoma survival in 20 years.
  • Glioblastoma median survival has not moved in 20 years; every phase 3 systemic agent since temozolomide has failed.
  • MGMT-unmethylated glioblastoma (~60%) gains almost nothing from chemotherapy and has no approved alternative.
  • Blood-brain barrier and diffuse infiltration limit delivery and resection; imaging cannot distinguish progression from pseudoprogression reliably.
  • Immunotherapy failure: low TMB, T-cell exclusion, dexamethasone, and treatment-induced lymphopenia; neoadjuvant approaches are the only signal.
  • Antigen heterogeneity and loss (EGFRvIII, IL13Rα2) undermine single-target vaccines, ADCs, and CAR-T.
  • Paediatric tumours (DIPG) have one approved drug with 22% response; durable control remains out of reach.
  • Trial design: single-arm and external-control comparisons (DCVax-L, historical vaccine data) have repeatedly misled the field.

Trials

top

Recruiting now (live from ClinicalTrials.gov)

Recruiting trials near you · live from ClinicalTrials.gov
Glioma & glioblastoma
condition: glioblastoma
Open on ClinicalTrials.gov →

Country and place are remembered in this browser only. A postcode is sent to OpenStreetMap's Nominatim service to find coordinates when you press the button; nothing else leaves your device.

Landmark trials in OnCo

ACT IVCheckMate 548 & CheckMate 143 & CheckMate 498EF-14EORTC 26981 / NCIC CE.3 (Stupp trial)ERGO2: ketogenic diet and fasting during re-irradiation of recurrent gliomaINDIGO

Expert centres

top
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

top
Bring to your appointment

Questions to ask your oncologist about Glioma & glioblastoma

Generated from this cancer's standard of care, biomarkers, and pipeline · 28 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 IDH1/2, 1p/19q codeletion, MGMT methylation, H3K27M, EGFR amplification), 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 Glioblastoma, IDH-wild-type, Astrocytoma, IDH-mutant, Oligodendroglioma, IDH-mutant and 1p/19q-codeleted.
  4. Is germline (inherited) genetic testing recommended for me or my family?
    Why: Inherited variants can change treatment and matter for relatives.

Glioblastoma

  1. For my situation (glioblastoma), which of the standard options do you recommend and why?
    Why: Guideline options include: Resection → RT + temozolomide → TTFields; lomustine/bevacizumab at relapse.
  2. Am I a candidate for Optune / Optune Pax (TTFields), and what side effects should I expect?
    Why: Knowing the expected toxicities helps you plan work, family, and supportive care.

IDH-mutant grade 2

  1. For my situation (idh-mutant grade 2), which of the standard options do you recommend and why?
    Why: Guideline options include: Resection → vorasidenib or observation; RT/PCV for high-risk.
  2. Am I a candidate for Vorasidenib, and what side effects should I expect?
    Why: Knowing the expected toxicities helps you plan work, family, and supportive care.

Diagnosis

  1. For my situation (diagnosis), which of the standard options do you recommend and why?
    Why: Guideline options include: MRI with contrast; maximal safe resection with 5-ALA and intraoperative mapping; integrated histo-molecular diagnosis with methylation classification where available.

Glioblastoma, newly diagnosed

  1. For my situation (glioblastoma, newly diagnosed), which of the standard options do you recommend and why?
    Why: Guideline options include: Radiotherapy 60 Gy (hypofractionated in elderly) with concurrent and 6 cycles adjuvant temozolomide; TTFields with maintenance temozolomide; trials for MGMT-unmethylated patients.
  2. Am I a candidate for Temozolomide, Optune / Optune Pax (TTFields), 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 EORTC 26981 / NCIC CE.3 (Stupp trial) and EF-14 apply to someone like me?
    Why: Trial populations differ from individual patients; ask how closely you match.

Glioblastoma, recurrent

  1. For my situation (glioblastoma, recurrent), which of the standard options do you recommend and why?
    Why: Guideline options include: Re-resection or LITT if feasible; lomustine; bevacizumab for oedema/steroid sparing; re-irradiation; clinical trial (CAR-T, FUS-BBB, vaccines) strongly preferred.
  2. Am I a candidate for Lomustine (CCNU), Bevacizumab (glioblastoma use), and what side effects should I expect?
    Why: Knowing the expected toxicities helps you plan work, family, and supportive care.

IDH-mutant grade 2 glioma

  1. For my situation (idh-mutant grade 2 glioma), which of the standard options do you recommend and why?
    Why: Guideline options include: Maximal resection; vorasidenib for residual/recurrent disease (INDIGO); radiotherapy plus PCV or temozolomide for high-risk or progressive disease.
  2. Am I a candidate for Vorasidenib, 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 INDIGO apply to someone like me?
    Why: Trial populations differ from individual patients; ask how closely you match.

Oligodendroglioma grade 3 / astrocytoma grade 3

  1. For my situation (oligodendroglioma grade 3 / astrocytoma grade 3), which of the standard options do you recommend and why?
    Why: Guideline options include: Radiotherapy plus PCV (RTOG 9402, EORTC 26951) or temozolomide (CATNON).
  2. Am I a candidate for Temozolomide, and what side effects should I expect?
    Why: Knowing the expected toxicities helps you plan work, family, and supportive care.

H3 K27M diffuse midline glioma

  1. For my situation (h3 k27m diffuse midline glioma), which of the standard options do you recommend and why?
    Why: Guideline options include: Radiotherapy; dordaviprone at progression (2025); GD2 CAR-T and ONC201 first-line trials.
  2. Am I a candidate for Dordaviprone, and what side effects should I expect?
    Why: Knowing the expected toxicities helps you plan work, family, and supportive care.

Paediatric low-grade glioma

  1. For my situation (paediatric low-grade glioma), which of the standard options do you recommend and why?
    Why: Guideline options include: Resection where safe; chemotherapy (carboplatin/vincristine) or tovorafenib / dabrafenib-trametinib for BRAF-altered relapsed disease; avoid radiation in young children.
  2. Am I a candidate for Tovorafenib, and what side effects should I expect?
    Why: Knowing the expected toxicities helps you plan work, family, and supportive care.

Any stage

  1. Are there clinical trials I could join, for example of Armoured, logic-gated & next-gen CARs, Boron neutron capture therapy, Hyperthermia, CAR-T for glioma (IL13Rα2, GD2, EGFRvIII, multi-target)?
    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 “Blood-brain barrier”. How does that affect my plan?
    Why: Open problems are where trials and second opinions matter most.
  5. I read that “Immunologically cold, heterogeneous, infiltrative”. 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

190top

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

technologies

31
Anti-angiogenic therapyArmoured, logic-gated & next-gen CARsBoron neutron capture therapyCancer-associated thrombosis prevention and treatmentCAR-T cell therapyCAR-T for glioma (IL13Rα2, GD2, EGFRvIII, multi-target)Cytotoxic chemotherapyDNA methylation profilingEarly integrated palliative careEpigenetic drugs (HDAC, DNMT, EZH2, IDH, menin, BET)Fluorescence-guided surgeryFocused ultrasound & histotripsyFocused-ultrasound blood-brain barrier openingHyperthermiaIMRT / IGRT (modern external beam)In vivo CAR-TKetogenic diets in glioblastomaLaser interstitial thermal therapy (LITT)Magnetic nanoparticle hyperthermiaMonoclonal antibodiesMRIOff-the-shelf cancer vaccinesOptical & fluorescence imagingPersonalised neoantigen (mRNA) vaccinesPET (positron emission tomography)PET/CTProton therapySBRT / SABR (stereotactic radiotherapy)Small-molecule kinase inhibitorsSonodynamic therapyTumour treating fields (TTFields)

targets

12
B7-H3BRAFCSF1REGFRGD2 (disialoganglioside)IDH1 / IDH2MDM2PD-1PDGFRAPRMT5 (MTAP-deleted cancers)TP53VEGF / VEGFR

drugs

12
Not mapped hereFluorescence-guided surgery agent (protoporphyrin IX precursor)
5-Aminolevulinic acid (oral, for glioma surgery) · Gleolan / Gliolan
ApprovedMonoclonal antibody (anti-VEGF)
Bevacizumab · Avastin (and biosimilars)
ApprovedMonoclonal antibody (anti-VEGF)
Bevacizumab (glioblastoma use) · Avastin
Phase 3Autologous dendritic cell vaccine (tumour lysate)
DCVax-L
ApprovedSmall-molecule imipridone (ClpP agonist / DRD2 antagonist)
Dordaviprone · Modeyso
Not mapped herePET radiotracer (synthetic amino acid)
Fluciclovine F-18 · Axumin
Not mapped hereOral nitrosourea chemotherapy
Lomustine (CCNU) · Gleostine
ApprovedDevice (tumour treating fields)
Optune / Optune Pax (TTFields) · Optune
NegativePeptide vaccine (EGFRvIII)
Rindopepimut
Not mapped hereOral alkylating chemotherapy
Temozolomide · Temodar
ApprovedSmall-molecule type II RAF inhibitor
Tovorafenib · Ojemda
ApprovedSmall-molecule IDH1/2 inhibitor
Vorasidenib · Voranigo

companies

10
Blue Earth Diagnostics (Bracco)Bristol Myers SquibbDay One BiopharmaceuticalsInsightecIpsenJazz PharmaceuticalsNorthwest BiotherapeuticsNovocureRoche / GenentechServier

institutions

40
Abramson Cancer Center, University of PennsylvaniaAtrium Health Wake Forest Baptist Comprehensive Cancer CenterCancer Research UK Cambridge Centre / CRUK Cambridge InstituteCedars-Sinai CancerChildren's Cancer and Leukaemia GroupChildren's Cancer Hospital Egypt 57357City of HopeComprehensive Cancer Center Tübingen-StuttgartComprehensive Cancer Center Vienna – Medical University of Vienna / AKHDuke Cancer InstituteEdinburgh Cancer Centre / CRUK Scotland CentreEORTCErasmus MC Cancer InstituteGeneva University Hospitals (HUG)German Cancer Research Center (DKFZ)Great Ormond Street Hospital for ChildrenHeidelberg University Hospital / NCT / DKFZHolden Comprehensive Cancer Center, University of IowaHospital de Amor (Barretos Cancer Hospital)Inselspital, Bern University Hospital / University Cancer Center InselspitalJuravinski Cancer Centre / Escarpment Cancer Research InstituteLausanne University Hospital (CHUV) / Ludwig Institute LausanneMassachusetts General Hospital Cancer CenterNewcastle Cancer Centre / Northern Centre for Cancer CareO'Neal Comprehensive Cancer Center at UABOlivia Newton-John Cancer Wellness and Research CentrePrincess Máxima Center for Pediatric OncologyQIMR Berghofer Medical Research InstituteRobert H. Lurie Comprehensive Cancer Center of Northwestern UniversitySIOP Europe – European Society for Paediatric OncologyStanford Health Care / Stanford Cancer InstituteSunnybrook Odette Cancer CentreSydney Children's Hospitals Network / Children's Cancer InstituteTel Aviv Sourasky Medical CenterThe Hospital for Sick Children (SickKids)The Wistar InstituteUniversity Cancer Center Frankfurt (UCT)University Hospital Zurich / Comprehensive Cancer Center ZurichUniversity of Florida Health Cancer CenterUppsala University Hospital / Uppsala University

pathways

11
Base excision repair, PARP & alkylation damageCancer neuroscience (nerve–tumour signalling)Glutamine addictionHedgehog signallingp53 / RB / cell-cycle checkpointPD-1 / PD-L1 immune checkpoint & T-cell activationPI3K / AKT / mTORRAS / RAF / MEK / ERK (MAPK)The angiogenic switch & tumour vesselsThe blood–brain barrier & brain metastasisVEGF angiogenesis

terms

12
Accelerated approvalBlood-brain barrier (BBB)Chemoradiation (chemoradiotherapy, CRT)EGFRvIIIGradeH3 K27M (diffuse midline glioma)Hot vs cold tumoursIntrathecal therapy (lumbar puncture, Ommaya reservoir)MGMT promoter methylationNecrosisRe-irradiationTERT promoter mutation

trials

6
ACT IVCheckMate 548 & CheckMate 143 & CheckMate 498EF-14EORTC 26981 / NCIC CE.3 (Stupp trial)ERGO2: ketogenic diet and fasting during re-irradiation of recurrent gliomaINDIGO

roadmaps

2
Cell therapy roadmap: CD19 CAR-T → solid tumours → in vivo CARImmunotherapy roadmap: Coley's toxins → checkpoint inhibitors → engineered immunity

ideas

30
A billion-dollar prize for the first durable cure of a lethal metastatic cancerA permanent neutral non-profit sponsor for multi-company platform trialsA perpetual platform trial in every major cancer, funded as infrastructureA skull ultrasound implant that opens the barrier at every cycleA standard platform to get drugs into the brain: focused ultrasound plus shuttle-engineered therapeuticsA standing platform for testing new drugs with radiotherapyA standing platform trial for every major cancer, funded as infrastructureAdvance market commitments for paediatric and rare cancer drugsAttack extrachromosomal DNA, the engine of oncogene amplificationCure-focused prizes: pay for verified long-term cures, not for drugsCut the nerve supply to tumours with old drugsDeliver CAR-T cells straight into the fluid around the brainDeliver drugs and cells to the brain through the noseDesign protein degraders small enough to get into the brainDevelop drugs in children first when the target is a children's targetFocused-ultrasound BBB opening to deliver ADCs and radioligands to gliomaImplant a tiny device that tests twenty drugs inside the patient's own tumourMake bespoke mouse cancer models in weeks with in vivo gene editingMap metabolic dependencies in the patient, not the dishNeoadjuvant immunotherapy with surgical window for glioblastomaOncolytic viruses that make interleukin-12 only inside the tumourOne definitive ketogenic diet trial in glioblastoma, then stopOpen the barrier so engineered immune cells can enter the brainPartial lottery funding for good proposals in under-funded cancersPet dogs with spontaneous cancer as a bridge before human trialsRead the spinal fluid to track brain tumours without opening the skullReprogramme suppressive macrophages instead of trying to delete themRobot-placed catheters and live imaging for drug infusion into brain tumoursTreat brain metastases as a disease with its own trials programmeUltrasound-assisted blood test instead of a brain biopsy

collections

1
National Brain Tumor Society (NBTS)

people

13
Andreas von DeimlingBehnam BadieBradley E. BernsteinChristine E. BrownCrystal L. MackallEvanthia GalanisHans-Peter KiemJustin D. LathiaMichael WellerRamon E. ParsonsRobert A. FenstermakerRoger StuppStefan Pfister

bottlenecks

7
Cold tumours and the immunosuppressive microenvironmentFunding follows fashion, not burdenIncentives reward me-too drugs and marginal gainsPain relief and palliative care are unavailable to mostPreclinical models that do not predict peopleThe brain: barrier and sanctuaryTumour heterogeneity and clonal evolution

key papers

2
INDIGO: vorasidenib, the first targeted drug for IDH-mutant low-grade gliomaStupp 2005: temozolomide added to radiotherapy for newly diagnosed glioblastoma

journals

1
Neuro-Oncology

Key papers

2top
rctNew England Journal of Medicine 2023changed practice
INDIGO: vorasidenib, the first targeted drug for IDH-mutant low-grade glioma

Young adults with a grade 2 IDH-mutant glioma who have had surgery can now take a daily tablet that slows or reverses tumour growth and defers radiotherapy and chemotherapy, both of which carry long-term cognitive costs, by years. It confirms that the IDH mutation is a driver that can be targeted, not just a marker. Whether it improves survival or cognition over the long term, and whether it helps in higher-grade or previously treated tumours, is unknown.

rctNew England Journal of Medicine 2005changed practice
Stupp 2005: temozolomide added to radiotherapy for newly diagnosed glioblastoma

Patients with newly diagnosed glioblastoma who are fit and under about 70 receive six weeks of radiotherapy with daily temozolomide followed by six monthly cycles of temozolomide; this is still the backbone of treatment two decades later. Testing MGMT methylation identifies who benefits most and guides decisions in older patients. Median survival with the regimen remains only around 15-20 months, and no drug since has clearly improved on it, which is why glioblastoma is a priority for new approaches.

Latest papers

top
Literature trend17 papers in the last 12 months+89% vs prior 12How this is computed
Latest papers · live from Europe PMC
Open in Europe PMC

Query for this cancer: (TITLE:"Glioma & glioblastoma" OR ABSTRACT:"Glioma & glioblastoma") AND (treatment OR therapy OR trial OR survival OR diagnosis). Results are unfiltered search hits about Glioma & glioblastoma, not a curated reading list.

Connected

185top

Pages like this

not linked directly; found by shared links

technologies

27
Anti-angiogenic therapyArmoured, logic-gated & next-gen CARsBoron neutron capture therapyCancer-associated thrombosis prevention and treatmentCAR-T cell therapyCAR-T for glioma (IL13Rα2, GD2, EGFRvIII, multi-target)Cytotoxic chemotherapyDNA methylation profilingEarly integrated palliative careEpigenetic drugs (HDAC, DNMT, EZH2, IDH, menin, BET)Fluorescence-guided surgeryFocused ultrasound & histotripsyFocused-ultrasound blood-brain barrier openingHyperthermiaIMRT / IGRT (modern external beam)In vivo CAR-TKetogenic diets in glioblastomaLaser interstitial thermal therapy (LITT)Magnetic nanoparticle hyperthermiaMRIOff-the-shelf cancer vaccinesPersonalised neoantigen (mRNA) vaccinesProton therapySBRT / SABR (stereotactic radiotherapy)Small-molecule kinase inhibitorsSonodynamic therapyTumour treating fields (TTFields)

targets

12
B7-H3BRAFCSF1REGFRGD2 (disialoganglioside)IDH1 / IDH2MDM2PD-1PDGFRAPRMT5 (MTAP-deleted cancers)TP53VEGF / VEGFR

drugs

12
Not mapped hereFluorescence-guided surgery agent (protoporphyrin IX precursor)
5-Aminolevulinic acid (oral, for glioma surgery) · Gleolan / Gliolan
ApprovedMonoclonal antibody (anti-VEGF)
Bevacizumab · Avastin (and biosimilars)
ApprovedMonoclonal antibody (anti-VEGF)
Bevacizumab (glioblastoma use) · Avastin
Phase 3Autologous dendritic cell vaccine (tumour lysate)
DCVax-L
ApprovedSmall-molecule imipridone (ClpP agonist / DRD2 antagonist)
Dordaviprone · Modeyso
Not mapped herePET radiotracer (synthetic amino acid)
Fluciclovine F-18 · Axumin
Not mapped hereOral nitrosourea chemotherapy
Lomustine (CCNU) · Gleostine
ApprovedDevice (tumour treating fields)
Optune / Optune Pax (TTFields) · Optune
NegativePeptide vaccine (EGFRvIII)
Rindopepimut
Not mapped hereOral alkylating chemotherapy
Temozolomide · Temodar
ApprovedSmall-molecule type II RAF inhibitor
Tovorafenib · Ojemda
ApprovedSmall-molecule IDH1/2 inhibitor
Vorasidenib · Voranigo

companies

9
Bristol Myers SquibbDay One BiopharmaceuticalsInsightecIpsenJazz PharmaceuticalsNorthwest BiotherapeuticsNovocureRoche / GenentechServier

institutions

40
Abramson Cancer Center, University of PennsylvaniaAtrium Health Wake Forest Baptist Comprehensive Cancer CenterCancer Research UK Cambridge Centre / CRUK Cambridge InstituteCedars-Sinai CancerChildren's Cancer and Leukaemia GroupChildren's Cancer Hospital Egypt 57357City of HopeComprehensive Cancer Center Tübingen-StuttgartComprehensive Cancer Center Vienna – Medical University of Vienna / AKHDuke Cancer InstituteEdinburgh Cancer Centre / CRUK Scotland CentreEORTCErasmus MC Cancer InstituteGeneva University Hospitals (HUG)German Cancer Research Center (DKFZ)Great Ormond Street Hospital for ChildrenHeidelberg University Hospital / NCT / DKFZHolden Comprehensive Cancer Center, University of IowaHospital de Amor (Barretos Cancer Hospital)Inselspital, Bern University Hospital / University Cancer Center InselspitalJuravinski Cancer Centre / Escarpment Cancer Research InstituteLausanne University Hospital (CHUV) / Ludwig Institute LausanneMassachusetts General Hospital Cancer CenterNewcastle Cancer Centre / Northern Centre for Cancer CareO'Neal Comprehensive Cancer Center at UABOlivia Newton-John Cancer Wellness and Research CentrePrincess Máxima Center for Pediatric OncologyQIMR Berghofer Medical Research InstituteRobert H. Lurie Comprehensive Cancer Center of Northwestern UniversitySIOP Europe – European Society for Paediatric OncologyStanford Health Care / Stanford Cancer InstituteSunnybrook Odette Cancer CentreSydney Children's Hospitals Network / Children's Cancer InstituteTel Aviv Sourasky Medical CenterThe Hospital for Sick Children (SickKids)The Wistar InstituteUniversity Cancer Center Frankfurt (UCT)University Hospital Zurich / Comprehensive Cancer Center ZurichUniversity of Florida Health Cancer CenterUppsala University Hospital / Uppsala University

pathways

11
Base excision repair, PARP & alkylation damageCancer neuroscience (nerve–tumour signalling)Glutamine addictionHedgehog signallingp53 / RB / cell-cycle checkpointPD-1 / PD-L1 immune checkpoint & T-cell activationPI3K / AKT / mTORRAS / RAF / MEK / ERK (MAPK)The angiogenic switch & tumour vesselsThe blood–brain barrier & brain metastasisVEGF angiogenesis

terms

12
Accelerated approvalBlood-brain barrier (BBB)Chemoradiation (chemoradiotherapy, CRT)EGFRvIIIGradeH3 K27M (diffuse midline glioma)Hot vs cold tumoursIntrathecal therapy (lumbar puncture, Ommaya reservoir)MGMT promoter methylationNecrosisRe-irradiationTERT promoter mutation

trials

6
ACT IVCheckMate 548 & CheckMate 143 & CheckMate 498EF-14EORTC 26981 / NCIC CE.3 (Stupp trial)ERGO2: ketogenic diet and fasting during re-irradiation of recurrent gliomaINDIGO

roadmaps

2
Cell therapy roadmap: CD19 CAR-T → solid tumours → in vivo CARImmunotherapy roadmap: Coley's toxins → checkpoint inhibitors → engineered immunity

ideas

30
A billion-dollar prize for the first durable cure of a lethal metastatic cancerA permanent neutral non-profit sponsor for multi-company platform trialsA perpetual platform trial in every major cancer, funded as infrastructureA skull ultrasound implant that opens the barrier at every cycleA standard platform to get drugs into the brain: focused ultrasound plus shuttle-engineered therapeuticsA standing platform for testing new drugs with radiotherapyA standing platform trial for every major cancer, funded as infrastructureAdvance market commitments for paediatric and rare cancer drugsAttack extrachromosomal DNA, the engine of oncogene amplificationCure-focused prizes: pay for verified long-term cures, not for drugsCut the nerve supply to tumours with old drugsDeliver CAR-T cells straight into the fluid around the brainDeliver drugs and cells to the brain through the noseDesign protein degraders small enough to get into the brainDevelop drugs in children first when the target is a children's targetFocused-ultrasound BBB opening to deliver ADCs and radioligands to gliomaImplant a tiny device that tests twenty drugs inside the patient's own tumourMake bespoke mouse cancer models in weeks with in vivo gene editingMap metabolic dependencies in the patient, not the dishNeoadjuvant immunotherapy with surgical window for glioblastomaOncolytic viruses that make interleukin-12 only inside the tumourOne definitive ketogenic diet trial in glioblastoma, then stopOpen the barrier so engineered immune cells can enter the brainPartial lottery funding for good proposals in under-funded cancersPet dogs with spontaneous cancer as a bridge before human trialsRead the spinal fluid to track brain tumours without opening the skullReprogramme suppressive macrophages instead of trying to delete themRobot-placed catheters and live imaging for drug infusion into brain tumoursTreat brain metastases as a disease with its own trials programmeUltrasound-assisted blood test instead of a brain biopsy

collections

1
National Brain Tumor Society (NBTS)

people

13
Andreas von DeimlingBehnam BadieBradley E. BernsteinChristine E. BrownCrystal L. MackallEvanthia GalanisHans-Peter KiemJustin D. LathiaMichael WellerRamon E. ParsonsRobert A. FenstermakerRoger StuppStefan Pfister

bottlenecks

7
Cold tumours and the immunosuppressive microenvironmentFunding follows fashion, not burdenIncentives reward me-too drugs and marginal gainsPain relief and palliative care are unavailable to mostPreclinical models that do not predict peopleThe brain: barrier and sanctuaryTumour heterogeneity and clonal evolution

key papers

2
INDIGO: vorasidenib, the first targeted drug for IDH-mutant low-grade gliomaStupp 2005: temozolomide added to radiotherapy for newly diagnosed glioblastoma

journals

1
Neuro-Oncology