OnCo
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Acute lymphoblastic leukaemia

Acute lymphoblastic leukaemia is the childhood cancer success story, and was the first disease treated with CAR-T and with a T-cell engager.

Acute lymphoblastic leukaemia is the commonest childhood cancer and a rarer, harder disease in adults. B-cell precursor ALL (85%) is classified by genetics into favourable (ETV6::RUNX1, high hyperdiploidy), intermediate, and adverse groups (KMT2A rearrangement, hypodiploidy, Ph-like/CRLF2, iAMP21, TCF3::HLF); Ph-positive (BCR::ABL1) disease accounts for a quarter of adult cases. T-ALL (15%) has its own genetics and, since 2023, its own targeted options (nelarabine, BCL-2 inhibition in trials). Measurable residual disease after induction is the strongest predictor of relapse in every subgroup.

Children are cured in over 90% with risk-adapted multi-agent chemotherapy over two to three years, and since 2024 with blinatumomab woven into consolidation (AALL1731: 3-year DFS 96% vs 88%). Adults do worse with chemotherapy alone, but three immunotherapies changed the picture: blinatumomab (E1910: 3-year OS 85% vs 68% when added frontline; standard in MRD-positive and relapsed disease), the CD22 ADC inotuzumab ozogamicin (INO-VATE: 81% remission in relapse), and CD19 CAR-T (tisagenlecleucel for patients up to 25; obe-cel for adults, 2024). Ph-positive ALL is treated with a BCR::ABL1 inhibitor (ponatinib preferred since PhALLCON, 2024) and increasingly with TKI plus blinatumomab and no chemotherapy at all (D-ALBA), while KMT2A-rearranged disease now has menin inhibitors.

Allogeneic transplant remains standard for high-risk or MRD-persistent disease but is being pushed back by MRD-negative remissions achieved with immunotherapy. The unsolved problems are CD19-negative relapse after CAR-T and blinatumomab, T-ALL relapse, Ph-like ALL, infant KMT2A-rearranged ALL, the late toxicities of paediatric therapy, and the very different outcomes for adolescents and adults treated outside specialised centres.

State of the art today

  • Immunotherapy in frontline paediatric therapy.
  • Two CD19 CAR-T products for ALL (tisagenlecleucel, obe-cel) with obe-cel showing markedly lower severe CRS and neurotoxicity; REMS removed for CAR-T in 2025.
  • Inotuzumab ozogamicin gives ~80% remission in relapse and is moving into frontline lower-intensity regimens for older adults and children.
  • MRD at 10^-5 to 10^-6 (clonoSEQ, PCR) is the organising principle for transplant, blinatumomab, and de-escalation decisions.
  • Menin inhibitors reach KMT2A-rearranged ALL, the first targeted therapy for infant leukaemia.
Show survival figures (2)

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.

  • Blinatumomab in frontline consolidation improves survival in adults (E1910) and children (AALL1731) regardless of MRD; approved June 2024.
  • Ph-positive ALL: ponatinib is the preferred TKI (PhALLCON) and chemotherapy-free TKI + blinatumomab regimens achieve 18-month survival near 95%.
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 6,500 US cases a year, 60% in children; 5-year survival above 90% in children and about 40-50% in adults, better in those under 40 treated on paediatric-style regimens.

Where the cases are

Cases by country · GLOBOCAN 2022
All countries →

Site: Leukaemia (all types) (shared total; subtype split not reported). World: 487,294 new cases, 305,405 deaths.

#CountryNew casesDeaths
1China81,94650,074
2United States of America63,14423,460
3India49,88336,871
4Germany15,1089,378
5Russian Federation14,4958,224
6Indonesia13,95910,370
7Japan13,57210,066
8France (metropolitan)13,5257,387
9Brazil11,8598,790
10Italy10,7997,197

GLOBOCAN reports leukaemia as one site; ALL is the commonest childhood cancer but a minority of adult leukaemia.

Standard of care

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Frontline

Risk-adapted chemotherapy + blinatumomab consolidation; TKI if Ph+.

Relapsed

CAR-T (tisagenlecleucel, obe-cel), inotuzumab, transplant.

Children, standard risk B-ALL

Risk-adapted multi-agent chemotherapy (induction, consolidation, interim maintenance, delayed intensification, 2-3 years maintenance) with two cycles of blinatumomab in consolidation (AALL1731, approved June 2024); intrathecal CNS prophylaxis; cranial radiation abandoned for most.

NCCN · Category 1 (blinatumomab in consolidation)
Children, high risk or MRD-positive

Intensified chemotherapy; blinatumomab for MRD persistence (AALL1331 in relapse); inotuzumab and CAR-T (tisagenlecleucel) for relapse; allogeneic transplant for very high risk or MRD persistence.

NCCN · Category 2A
Infants (<1 year), KMT2A-rearranged

Interfant-21 backbone with blinatumomab; transplant for high-risk; menin inhibitors in trials.

NCCN · Clinical trial preferred
Adolescents and young adults (15-39), Ph-negative

Paediatric-inspired regimens (CALGB 10403, GRAALL, UKALL) with asparaginase; blinatumomab consolidation (E1910); MRD-guided transplant.

NCCN · Category 1 (blinatumomab consolidation)
Adults 40-70, Ph-negative

Hyper-CVAD or age-adapted multi-agent chemotherapy with blinatumomab interleaved in consolidation (E1910); inotuzumab-containing lower-intensity regimens (Mini-hyper-CVD + InO ± blinatumomab) for older adults; MRD-driven transplant.

NCCN · Category 1 (E1910 regimen)
Ph-positive ALL, newly diagnosed

Ponatinib (PhALLCON) or dasatinib with reduced-intensity chemotherapy, or chemotherapy-free TKI + blinatumomab (D-ALBA); BCR::ABL1 PCR monitoring; transplant for MRD persistence or IKZF1-plus, increasingly omitted for MRD-negative patients.

NCCN · Category 2A (ponatinib preferred TKI)
MRD-positive after induction or consolidation

Blinatumomab (BLAST; full approval 2018) to convert to MRD negativity, then transplant or continued blinatumomab-based therapy.

NCCN · Category 1
Relapsed or refractory B-ALL

Blinatumomab (TOWER) or inotuzumab (INO-VATE) as salvage and bridge; CD19 CAR-T (tisagenlecleucel ≤25 years; obe-cel or brexucabtagene in adults) for second salvage or as definitive therapy; transplant for those not previously transplanted; CD22 or dual CAR-T for CD19-negative relapse in trials.

NCCN · Category 2A
T-cell ALL

Intensive paediatric-type chemotherapy with nelarabine for high-risk (AALL0434); relapse: nelarabine, venetoclax combinations, CD7 CAR-T and daratumumab in trials; transplant in CR2.

NCCN · Category 2A

Subtypes & biomarkers

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Subtypes
  • B-cell precursor ALL (85%): ETV6::RUNX1, high hyperdiploidy (favourable); Ph-positive BCR::ABL1 (25% of adults); Ph-like/CRLF2 (adverse, targetable in trials); KMT2A-rearranged (infants); hypodiploid, iAMP21, TCF3::HLF, DUX4/ZNF384 fusions
  • T-cell ALL (15%): early T-precursor (ETP) subtype adverse; NOTCH1 mutated majority
  • Mixed-phenotype acute leukaemia
  • Burkitt-type (mature B) leukaemia, treated as lymphoma
Biomarkers clinicians test
  • BCR-ABL (Ph+)
  • KMT2A
  • Ph-like signature
  • MRD (flow/NGS)
  • MRD by flow (10^-4) or clonoSEQ/PCR (10^-5 to 10^-6) at end of induction and consolidation
  • BCR ::ABL1 (Ph+) and transcript type
  • KMT2A rearrangement (menin inhibitor eligibility; infants)
  • Ph-like signature (CRLF2, ABL-class fusions, JAK mutations)
  • IKZF1 deletion / IKZF1-plus (adverse, especially in Ph+)
  • Hypodiploidy (<44 chromosomes) and TP53 germline testing
  • CD19 and CD22 expression and density (immunotherapy targets and escape)
  • CNS involvement at diagnosis
  • Age and white count (NCI risk in children)

Target prevalence in this cancer

Target / alterationPrevalenceSource
CD19
>95%
Wikipedia
CD22
>90%
Menin
5-10%
Wikipedia
BCR::ABL1 (Philadelphia chromosome)
~25 adults; ~3 children%
KMT2A (MLL) rearrangement
~80 in infants; 5-10 in adults%

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. 1948Farber: aminopterin remissions, birth of chemotherapy
  2. 1948Farber induces the first leukaemia remissions

    Aminopterin in children with ALL; the birth of cancer chemotherapy.

  3. 1962Combination chemotherapy and CNS prophylaxis

    Pinkel's 'total therapy' at St. Jude: multi-agent induction, cranial irradiation, maintenance; first cures.

  4. 1990Risk-adapted therapy standardised

    NCI/Rome criteria (age, white count); paediatric cooperative groups reach ~70% cure.

  5. 2000Imatinib transforms Ph-positive leukaemia

    First BCR::ABL1 inhibitor; Ph+ ALL survival doubles when added to chemotherapy.

  6. 2009MRD becomes the key risk factor

    AIEOP-BFM 2000 and UKALL 2003 show end-of-induction MRD outperforms every clinical factor; MRD-directed therapy adopted.

  7. 2012First CAR-T cures: Emily Whitehead

    CTL019 at CHOP/Penn; durable remission of refractory paediatric ALL.

  8. 2014Blinatumomab: first BiTE
  9. 2014Blinatumomab: first bispecific T-cell engager approved

    Accelerated approval in relapsed/refractory Ph-negative B-ALL.

  10. 2016INO-VATE: CD22 ADC beats chemotherapy in relapse

    CR/CRi 81% vs 29%; approval 2017.

  11. 2017Tisagenlecleucel: first CAR-T
  12. 2017Kymriah: first CAR-T approved; TOWER confirms blinatumomab OS benefit

    ELIANA-based approval for ALL up to age 25; TOWER OS 7.7 vs 4.0 months.

  13. 2018Blinatumomab approved for MRD-positive ALL

    First approval based on an MRD endpoint (BLAST).

  14. 2020D-ALBA: chemotherapy-free Ph+ ALL

    Dasatinib then blinatumomab, 18-month OS 95%.

  15. 2024Blinatumomab frontline
  16. 2024Blinatumomab frontline (E1910, AALL1731), ponatinib frontline (PhALLCON), obe-cel approved

    Immunotherapy becomes part of standard first-line therapy for adults and children; second CAR-T for adult ALL; revumenib approved for KMT2Ar leukaemia.

  17. 2025REMS removed for CAR-T; menin inhibitor combinations enter ALL trials

    FDA lifts REMS for approved CAR-T products; ziftomenib and revumenib studied in KMT2Ar ALL.

  18. 2026Subcutaneous blinatumomab and Interfant-21 progress

    Subcutaneous blinatumomab trials (including the first in mixed-phenotype leukaemia) aim to replace continuous infusion; infant trials integrate blinatumomab.

Pipeline

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Open problems

  • Adult ALL outcomes.
  • CD19-negative relapse.
  • CD19-negative relapse after blinatumomab or CAR-T; CD22 and dual-antigen CARs are early.
  • T-cell ALL has no approved immunotherapy; CD7 CAR-T (fratricide) and venetoclax combinations are experimental.
  • Ph-like ALL (CRLF2, JAK) has poor outcomes and only trial access to JAK/ABL-class inhibitors.
  • Infant KMT2A-rearranged ALL still has EFS under 50% on chemotherapy alone.
  • Continuous-infusion blinatumomab is burdensome; subcutaneous formulation not yet approved.
  • Which adults can safely skip transplant after MRD-negative immunotherapy remission is untested in randomised trials.
  • Late effects of curative paediatric therapy (neurocognitive, cardiac, second cancers) and survivorship care.
  • Adults treated outside academic centres have markedly worse survival; access to CAR-T and paediatric-inspired protocols is uneven.

Trials

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Recruiting now (live from ClinicalTrials.gov)

Recruiting trials near you · live from ClinicalTrials.gov
Acute lymphoblastic leukaemia
condition: acute lymphoblastic leukemia
Open on ClinicalTrials.gov →

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Landmark trials in OnCo

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 Acute lymphoblastic leukaemia

Generated from this cancer's standard of care, biomarkers, and pipeline · 37 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 BCR-ABL, KMT2A, Ph-like signature, MRD, MRD by flowor clonoSEQ/PCRat end of induction and consolidation), 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 B-cell precursor ALL: ETV6::RUNX1, high hyperdiploidy; Ph-positive BCR::ABL1; Ph-like/CRLF2; KMT2A-rearranged; hypodiploid, iAMP21, TCF3::HLF, DUX4/ZNF384 fusions, T-cell ALL: early T-precursorsubtype adverse; NOTCH1 mutated majority, Mixed-phenotype acute leukaemia.
  4. Is germline (inherited) genetic testing recommended for me or my family?
    Why: Inherited variants can change treatment and matter for relatives.

Frontline

  1. For my situation (frontline), which of the standard options do you recommend and why?
    Why: Guideline options include: Risk-adapted chemotherapy + blinatumomab consolidation; TKI if Ph+.
  2. Am I a candidate for Blinatumomab, and what side effects should I expect?
    Why: Knowing the expected toxicities helps you plan work, family, and supportive care.

Relapsed

  1. For my situation (relapsed), which of the standard options do you recommend and why?
    Why: Guideline options include: CAR-T (tisagenlecleucel, obe-cel), inotuzumab, transplant.
  2. Am I a candidate for Revumenib, and what side effects should I expect?
    Why: Knowing the expected toxicities helps you plan work, family, and supportive care.

Children, standard risk B-ALL

  1. For my situation (children, standard risk b-all), which of the standard options do you recommend and why?
    Why: Guideline options include: Risk-adapted multi-agent chemotherapy (induction, consolidation, interim maintenance, delayed intensification, 2-3 years maintenance) with two cycles of blinatumomab in consolidation (AALL1731, approved June 2024); intrathecal CNS prophylaxis; cranial radiation abandoned for most.
  2. Am I a candidate for Blinatumomab, 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 COG AALL1731 apply to someone like me?
    Why: Trial populations differ from individual patients; ask how closely you match.

Children, high risk or MRD-positive

  1. For my situation (children, high risk or mrd-positive), which of the standard options do you recommend and why?
    Why: Guideline options include: Intensified chemotherapy; blinatumomab for MRD persistence (AALL1331 in relapse); inotuzumab and CAR-T (tisagenlecleucel) for relapse; allogeneic transplant for very high risk or MRD persistence.
  2. Am I a candidate for Blinatumomab, Inotuzumab ozogamicin, Tisagenlecleucel, and what side effects should I expect?
    Why: Knowing the expected toxicities helps you plan work, family, and supportive care.

Infants (<1 year), KMT2A-rearranged

  1. For my situation (infants (<1 year), kmt2a-rearranged), which of the standard options do you recommend and why?
    Why: Guideline options include: Interfant-21 backbone with blinatumomab; transplant for high-risk; menin inhibitors in trials.
  2. Am I a candidate for Blinatumomab, 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 Interfant-06 apply to someone like me?
    Why: Trial populations differ from individual patients; ask how closely you match.

Adolescents and young adults (15-39), Ph-negative

  1. For my situation (adolescents and young adults (15-39), ph-negative), which of the standard options do you recommend and why?
    Why: Guideline options include: Paediatric-inspired regimens (CALGB 10403, GRAALL, UKALL) with asparaginase; blinatumomab consolidation (E1910); MRD-guided transplant.
  2. Am I a candidate for Blinatumomab, 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 ECOG-ACRIN E1910 apply to someone like me?
    Why: Trial populations differ from individual patients; ask how closely you match.

Adults 40-70, Ph-negative

  1. For my situation (adults 40-70, ph-negative), which of the standard options do you recommend and why?
    Why: Guideline options include: Hyper-CVAD or age-adapted multi-agent chemotherapy with blinatumomab interleaved in consolidation (E1910); inotuzumab-containing lower-intensity regimens (Mini-hyper-CVD + InO ± blinatumomab) for older adults; MRD-driven transplant.
  2. Am I a candidate for Blinatumomab, Inotuzumab ozogamicin, 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 ECOG-ACRIN E1910 apply to someone like me?
    Why: Trial populations differ from individual patients; ask how closely you match.

Ph-positive ALL, newly diagnosed

  1. For my situation (ph-positive all, newly diagnosed), which of the standard options do you recommend and why?
    Why: Guideline options include: Ponatinib (PhALLCON) or dasatinib with reduced-intensity chemotherapy, or chemotherapy-free TKI + blinatumomab (D-ALBA); BCR::ABL1 PCR monitoring; transplant for MRD persistence or IKZF1-plus, increasingly omitted for MRD-negative patients.
  2. Am I a candidate for Ponatinib, Dasatinib, Blinatumomab, 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 PhALLCON and D-ALBA (GIMEMA LAL2116) apply to someone like me?
    Why: Trial populations differ from individual patients; ask how closely you match.

MRD-positive after induction or consolidation

  1. For my situation (mrd-positive after induction or consolidation), which of the standard options do you recommend and why?
    Why: Guideline options include: Blinatumomab (BLAST; full approval 2018) to convert to MRD negativity, then transplant or continued blinatumomab-based therapy.
  2. Am I a candidate for Blinatumomab, and what side effects should I expect?
    Why: Knowing the expected toxicities helps you plan work, family, and supportive care.

Relapsed or refractory B-ALL

  1. For my situation (relapsed or refractory b-all), which of the standard options do you recommend and why?
    Why: Guideline options include: Blinatumomab (TOWER) or inotuzumab (INO-VATE) as salvage and bridge; CD19 CAR-T (tisagenlecleucel ≤25 years; obe-cel or brexucabtagene in adults) for second salvage or as definitive therapy; transplant for those not previously transplanted; CD22 or dual CAR-T for CD19-negative relapse in trials.
  2. Am I a candidate for Blinatumomab, Inotuzumab ozogamicin, Tisagenlecleucel 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 TOWER and INO-VATE ALL apply to someone like me?
    Why: Trial populations differ from individual patients; ask how closely you match.

T-cell ALL

  1. For my situation (t-cell all), which of the standard options do you recommend and why?
    Why: Guideline options include: Intensive paediatric-type chemotherapy with nelarabine for high-risk (AALL0434); relapse: nelarabine, venetoclax combinations, CD7 CAR-T and daratumumab in trials; transplant in CR2.
  2. Am I a candidate for Venetoclax, 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 Revumenib, Obecabtagene autoleucel, Ponatinib, Asciminib?
    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 “Adult ALL outcomes”. How does that affect my plan?
    Why: Open problems are where trials and second opinions matter most.
  5. I read that “CD19-negative relapse”. How does that affect my plan?
    Why: Open problems are where trials and second opinions matter most.

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Everything relevant

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

technologies

15

targets

11

drugs

18
ApprovedSmall-molecule allosteric ABL1 inhibitor (STAMP)
Asciminib · Scemblix
Not mapped hereEnzyme therapy
Asparaginase (pegaspargase, calaspargase pegol, Erwinia asparaginase) · Oncaspar / Asparlas / Rylaze / Erwinaze
Not mapped hereOral ROCK2 inhibitor
Belumosudil · Rezurock
ApprovedBispecific T-cell engager (CD19×CD3)
Blinatumomab · Blincyto
Not mapped hereAutologous CD19 CAR-T (CD28 costimulatory domain)
Brexucabtagene autoleucel · Tecartus
Not mapped hereAlkylating agent (oxazaphosphorine prodrug)
Cyclophosphamide · Cytoxan / Endoxan
Not mapped hereCytotoxic regimen
Cytarabine + anthracycline ('7+3')
ApprovedSmall-molecule kinase inhibitor (BCR::ABL1, SRC)
Dasatinib · Sprycel
ApprovedSmall-molecule kinase inhibitor (BCR-ABL, KIT, PDGFRA)
Imatinib · Gleevec
ApprovedADC
Inotuzumab ozogamicin · Besponsa
Not mapped hereAntifolate (DHFR inhibitor)
Methotrexate · Trexall / Otrexup / Xatmep
ApprovedCAR-T (CD19, fast off-rate)
Obecabtagene autoleucel · Aucatzyl
ApprovedSmall-molecule kinase inhibitor (pan-BCR::ABL1 incl. T315I)
Ponatinib · Iclusig
ApprovedSmall-molecule menin inhibitor
Revumenib · Revuforj
ApprovedCAR-T (CD19)
Tisagenlecleucel · Kymriah
ApprovedSmall-molecule BCL-2 inhibitor
Venetoclax · Venclexta
Not mapped hereVinca alkaloid (microtubule inhibitor)
Vincristine · Oncovin / Marqibo
ApprovedSmall-molecule menin inhibitor
Ziftomenib · Komzifti

companies

16

institutions

50
Advanced Centre for Treatment, Research and Education in CancerAll India Institute of Medical Sciences, New DelhiAmerican Society of HematologyCancer Institute (WIA), AdyarChildren's Cancer and Leukaemia GroupChildren's Cancer Hospital Egypt 57357Children's Hospital of PhiladelphiaChildren's Oncology Group (COG)Christian Medical College, VelloreECOG-ACRIN Cancer Research GroupEuropean Hematology AssociationGIMEMAGreat Ormond Street Hospital for ChildrenGroote Schuur Hospital / University of Cape TownHadassah Medical CenterHerbert Irving Comprehensive Cancer Center, Columbia UniversityHospital Clínic de Barcelona / IDIBAPSHospital de Clínicas de Porto AlegreHospital Universitari i Politècnic La FeHOVONInstituto Nacional de Câncer (INCA)Instituto Nacional de Cancerología (Colombia)Instituto Nacional de Enfermedades NeoplásicasInstituto Português de Oncologia de Lisboa Francisco GentilIRCCS Ospedale San RaffaeleKidwai Memorial Institute of OncologyKing Faisal Specialist Hospital and Research CentreKing Hussein Cancer CenterMasonic Cancer Center, University of MinnesotaNational Cancer Institute, Cairo UniversityNewcastle Cancer Centre / Northern Centre for Cancer CarePhilippine General HospitalPostgraduate Institute of Medical Education and Research, ChandigarhPrincess Máxima Center for Pediatric OncologyRamathibodi Hospital, Mahidol UniversityRuijin Hospital, Shanghai Jiao Tong UniversitySeoul St. Mary's HospitalSIOP Europe – European Society for Paediatric OncologySt. Jude Children's Research HospitalSydney Children's Hospitals Network / Children's Cancer InstituteTata Medical Center, KolkataTawam HospitalTexas Children's Cancer and Hematology CenterThe Hospital for Sick Children (SickKids)University Cancer Center Frankfurt (UCT)University College London Hospitals / UCL Cancer InstituteUniversity Hospital Düsseldorf / CIO DüsseldorfUniversity Hospitals Birmingham / University of Birmingham Cancer Research CentreUniversity of New Mexico Comprehensive Cancer CenterWorld Health Organization – Cancer Programme

pathways

1

terms

8

trials

10

pairings

3

ideas

20

collections

3

people

21

bottlenecks

5

key papers

7

Key papers

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rctNew England Journal of Medicine 2025changed practice
Children's Oncology Group AALL1731: blinatumomab added to chemotherapy for children with standard-risk B-cell ALL

AALL1731 brings immunotherapy into the front-line treatment of the commonest childhood cancer, in the group of children where most relapses were occurring despite good initial risk. Blinatumomab was approved for this use in 2024 and paediatric protocols worldwide are being amended. Whether it can allow less chemotherapy, and its effect on very low-risk children, are the next questions.

rctNew England Journal of Medicine 2024changed practice
ECOG-ACRIN E1910: adding blinatumomab to chemotherapy for adults with B-cell ALL already in MRD-negative remission

E1910 changed the standard of care for adult B-ALL: immunotherapy is now part of front-line consolidation even for patients with no detectable leukaemia, because MRD-negative by flow cytometry does not mean cured. It also demonstrated that a T-cell engager can improve overall survival in a curative setting. Chemotherapy-light or chemotherapy-free regimens built on blinatumomab and inotuzumab are the next step.

translationalNature 2023changed practice
AUGMENT-101: revumenib, the first menin inhibitor, in relapsed leukaemias driven by KMT2A rearrangement or NPM1 mutation

Revumenib proved that a transcriptional dependency, rather than a kinase, can be drugged in leukaemia, opening treatment for two genetic subgroups that together cover roughly a third of AML plus most infant ALL. It is now approved and is being combined with venetoclax-azacitidine and intensive chemotherapy in front-line trials. Single-agent remissions are often short without transplant.

translationalThe Lancet 2020
UCART19: the first gene-edited, donor-derived CAR-T cells in children and adults with relapsed B-cell ALL

The UCART19 report was the first clinical evidence that a universal, pre-manufactured CAR-T made from a donor can work, avoiding the weeks of autologous manufacturing and the problem of patients whose own T cells are too damaged. It set the template for later allogeneic programmes (including cemacabtagene autoleucel in the ALPHA studies) and for in vivo CAR generation. Short persistence and the need for deep lymphodepletion remain the central weaknesses.

translationalNew England Journal of Medicine 2018changed practice
ELIANA: the global trial that made tisagenlecleucel the first approved CAR-T therapy for children and young adults with relapsed ALL

ELIANA turned CAR-T from a single-centre experiment into a licensed product and created the regulatory and logistical template every later cell therapy has followed. For children with refractory leukaemia it offers a chance of durable remission without transplant. The trial also exposed the gaps: manufacturing failures, patients dying while waiting, and roughly half relapsing within a few years.

rctNew England Journal of Medicine 2016changed practice
INO-VATE: inotuzumab ozogamicin, a CD22 antibody-drug conjugate, versus chemotherapy for relapsed adult B-cell ALL

INO-VATE made inotuzumab a standard salvage therapy for adult ALL and a common route to transplant, and, with the TOWER trial of blinatumomab, moved adult ALL from chemotherapy-only salvage to immunotherapy. Both drugs have since been pulled into front-line regimens. The liver toxicity signal shaped how transplant conditioning is chosen after inotuzumab.

translationalNew England Journal of Medicine 2014changed practice
Maude 2014: CD19 CAR-T cells produce complete remission in 27 of 30 children and adults with relapsed ALL

This paper is the proof-of-concept for a living drug: a single infusion of a patient's own engineered T cells could eradicate leukaemia that had survived chemotherapy, transplant and antibody therapy. It defined cytokine release syndrome and its antidote, tocilizumab, and revealed antigen-loss relapse. It led directly to the first approved gene-modified cell therapy three years later.

Latest papers

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Literature trend163 papers in the last 12 months+5% vs prior 12How this is computed
Latest papers · live from Europe PMC
Open in Europe PMC

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

Connected

175top

technologies

13

targets

8

drugs

18
ApprovedSmall-molecule allosteric ABL1 inhibitor (STAMP)
Asciminib · Scemblix
Not mapped hereEnzyme therapy
Asparaginase (pegaspargase, calaspargase pegol, Erwinia asparaginase) · Oncaspar / Asparlas / Rylaze / Erwinaze
Not mapped hereOral ROCK2 inhibitor
Belumosudil · Rezurock
ApprovedBispecific T-cell engager (CD19×CD3)
Blinatumomab · Blincyto
Not mapped hereAutologous CD19 CAR-T (CD28 costimulatory domain)
Brexucabtagene autoleucel · Tecartus
Not mapped hereAlkylating agent (oxazaphosphorine prodrug)
Cyclophosphamide · Cytoxan / Endoxan
Not mapped hereCytotoxic regimen
Cytarabine + anthracycline ('7+3')
ApprovedSmall-molecule kinase inhibitor (BCR::ABL1, SRC)
Dasatinib · Sprycel
ApprovedSmall-molecule kinase inhibitor (BCR-ABL, KIT, PDGFRA)
Imatinib · Gleevec
ApprovedADC
Inotuzumab ozogamicin · Besponsa
Not mapped hereAntifolate (DHFR inhibitor)
Methotrexate · Trexall / Otrexup / Xatmep
ApprovedCAR-T (CD19, fast off-rate)
Obecabtagene autoleucel · Aucatzyl
ApprovedSmall-molecule kinase inhibitor (pan-BCR::ABL1 incl. T315I)
Ponatinib · Iclusig
ApprovedSmall-molecule menin inhibitor
Revumenib · Revuforj
ApprovedCAR-T (CD19)
Tisagenlecleucel · Kymriah
ApprovedSmall-molecule BCL-2 inhibitor
Venetoclax · Venclexta
Not mapped hereVinca alkaloid (microtubule inhibitor)
Vincristine · Oncovin / Marqibo
ApprovedSmall-molecule menin inhibitor
Ziftomenib · Komzifti

companies

8

institutions

50
Advanced Centre for Treatment, Research and Education in CancerAll India Institute of Medical Sciences, New DelhiAmerican Society of HematologyCancer Institute (WIA), AdyarChildren's Cancer and Leukaemia GroupChildren's Cancer Hospital Egypt 57357Children's Hospital of PhiladelphiaChildren's Oncology Group (COG)Christian Medical College, VelloreECOG-ACRIN Cancer Research GroupEuropean Hematology AssociationGIMEMAGreat Ormond Street Hospital for ChildrenGroote Schuur Hospital / University of Cape TownHadassah Medical CenterHerbert Irving Comprehensive Cancer Center, Columbia UniversityHospital Clínic de Barcelona / IDIBAPSHospital de Clínicas de Porto AlegreHospital Universitari i Politècnic La FeHOVONInstituto Nacional de Câncer (INCA)Instituto Nacional de Cancerología (Colombia)Instituto Nacional de Enfermedades NeoplásicasInstituto Português de Oncologia de Lisboa Francisco GentilIRCCS Ospedale San RaffaeleKidwai Memorial Institute of OncologyKing Faisal Specialist Hospital and Research CentreKing Hussein Cancer CenterMasonic Cancer Center, University of MinnesotaNational Cancer Institute, Cairo UniversityNewcastle Cancer Centre / Northern Centre for Cancer CarePhilippine General HospitalPostgraduate Institute of Medical Education and Research, ChandigarhPrincess Máxima Center for Pediatric OncologyRamathibodi Hospital, Mahidol UniversityRuijin Hospital, Shanghai Jiao Tong UniversitySeoul St. Mary's HospitalSIOP Europe – European Society for Paediatric OncologySt. Jude Children's Research HospitalSydney Children's Hospitals Network / Children's Cancer InstituteTata Medical Center, KolkataTawam HospitalTexas Children's Cancer and Hematology CenterThe Hospital for Sick Children (SickKids)University Cancer Center Frankfurt (UCT)University College London Hospitals / UCL Cancer InstituteUniversity Hospital Düsseldorf / CIO DüsseldorfUniversity Hospitals Birmingham / University of Birmingham Cancer Research CentreUniversity of New Mexico Comprehensive Cancer CenterWorld Health Organization – Cancer Programme

pathways

1

terms

8

trials

10

pairings

3

ideas

20

collections

3

people

21

bottlenecks

5

key papers

7