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🗺️ Disease Atlas · Hematologic disorder

acute lymphoblastic leukemia

Leukemia with an acute onset, characterized by the presence of lymphoblasts in the bone marrow and the peripheral blood. It includes the acute B lymphoblastic leukemia and acute T lymphoblastic leukemia.

🔔 Follow weekly
🧬7835genes / proteins with evidence
💊306drugs and clinical candidates
🧪12+recruiting trials worldwide
✅17approved
📈70phase 3
🧬

Understand acute lymphoblastic leukemia

Cancer · Cells that grow out of control
🎓 Learn more

What it is

Leukemia is a term for cancers of the blood cells. Leukemia starts in blood-forming tissues such as the bone marrow. Your bone marrow makes the cells which will develop into white blood cells, red blood cells, and platelets. Each type of cell has a different job:

White blood cells help your body fight infection

Red blood cells deliver oxygen from your lungs to your tissues and organs

From MedlinePlus: Acute Lymphocytic Leukemia (US National Library of Medicine) ↗

🧬 Cancer: Cancer is a group of diseases in which some of the body's cells grow and divide without control, and can spread to other parts of the body.

❓ Why it happens

  • Changes (mutations) build up in the genes that tell a cell when to grow, divide and die.
  • Most of these changes happen during life: tobacco, too much sun, alcohol, some infections (such as HPV and hepatitis B and C), radiation, obesity and ageing all raise the risk.
  • Some people inherit a faulty gene that raises their risk, such as BRCA1 or BRCA2.

🫀 What it does to the body

  • Cancer cells form a lump (tumour) that can press on or invade nearby organs.
  • They can travel through the blood or lymph to start new tumours elsewhere (metastasis).
  • Blood cancers crowd out healthy blood cells in the bone marrow.

🧱 Why it is hard to cure

  • Every cancer is different, even between two people with the same type - and inside one tumour.
  • Cancer cells keep changing, so they can learn to resist a medicine that worked at first.
  • Cancer cells are the body's own cells, so killing them without harming healthy cells is hard.
  • Many cancers cause no symptoms until they have spread, and a new medicine often takes more than ten years to go from the lab to patients.

🚀 What research is trying now

  • Targeted medicines against the exact gene change in a tumour (for example EGFR, ALK or KRAS).
  • Immunotherapy that helps the immune system recognise cancer, including CAR-T cell therapy.
  • Personalised cancer vaccines built from each tumour's mutations, now in late trials.
  • Blood tests that look for tumour DNA to find cancer earlier, and AI to read scans and slides.

General information about cancer, reviewed by people; it does not describe any one person. Below: the evidence for acute lymphoblastic leukemia itself.

🤖

AI briefing

Where the science on acute lymphoblastic leukemia stands, from the evidence on this page

AI reads the targets, drugs, trials and papers below and writes where the science stands — every claim cited. It is shared with everyone who opens acute lymphoblastic leukemia.

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🎯

Top targets

Genes and proteins ranked by Open Targets evidence (0-1)
NOTCH1 notch receptor 1PROTACantibodysmall molecule🧊 AlphaFold structure ↗ 0.80
BCR BCR activator of RhoGEF and GTPasePROTACantibodysmall molecule🧊 AlphaFold structure ↗ 0.79
ABL1 ABL proto-oncogene 1, non-receptor tyrosine kinasePROTACantibodysmall molecule🧊 AlphaFold structure ↗ 0.76
BCL2 BCL2 apoptosis regulatorPROTACantibodyothersmall molecule🧊 AlphaFold structure ↗ 0.75
SF3B1 splicing factor 3b subunit 1PROTACsmall molecule🧊 AlphaFold structure ↗ 0.74
BTK Bruton tyrosine kinasePROTACantibodysmall molecule🧊 AlphaFold structure ↗ 0.74
TP53 tumor protein p53PROTACothersmall molecule🧊 AlphaFold structure ↗ 0.72
IKZF1 IKAROS family zinc finger 1PROTACsmall molecule🧊 AlphaFold structure ↗ 0.72
CHD2 chromodomain helicase DNA binding protein 2PROTAC🧊 AlphaFold structure ↗ 0.72
ATM ATM serine/threonine kinasePROTACsmall molecule🧊 AlphaFold structure ↗ 0.72
NBN nibrinPROTACsmall molecule🧊 AlphaFold structure ↗ 0.72
POLD1 DNA polymerase delta 1, catalytic subunitPROTACsmall molecule🧊 AlphaFold structure ↗ 0.70
MYD88 MYD88 innate immune signal transduction adaptorPROTACantibody🧊 AlphaFold structure ↗ 0.70
POLE DNA polymerase epsilon, catalytic subunitPROTACantibodysmall molecule🧊 AlphaFold structure ↗ 0.69
KIT KIT proto-oncogene, receptor tyrosine kinasePROTACantibodysmall molecule🧊 AlphaFold structure ↗ 0.68

Green labels: what the target is known to be druggable by. The score combines genetics, somatic mutations, drugs, expression and literature.

💊

Drugs and candidates

Every medicine linked to acute lymphoblastic leukemia, by how far it has got
Approved · 17
Asparaginase Erwinia ChrysanthemiApproved
BlinatumomabT cell surface glycoprotein CD3 cross-linking agent; B-lymphocyte antigen CD19 cross-link…Approved
Calaspargase PegolApproved
ChlorambucilApproved
ClofarabineRibonucleoside-diphosphate reductase RR1 inhibitor; DNA polymerase (alpha/delta/epsilon) …Approved
CytarabineDNA polymerase (alpha/delta/epsilon) inhibitorApproved
Show all 17
DasatinibBcr/Abl fusion protein inhibitorApproved
Dasatinib AnhydrousTyrosine-protein kinase ABL inhibitor; Platelet-derived growth factor receptor beta inhib…Approved
Inotuzumab OzogamicinCD22 binding agentApproved
MercaptopurineAmidophosphoribosyltransferase inhibitorApproved
MethotrexateDihydrofolate reductase inhibitorApproved
PegaspargaseApproved
PlerixaforC-X-C chemokine receptor type 4 partial agonistApproved
PonatinibBcr/Abl fusion protein inhibitorApproved
Ponatinib HydrochlorideBcr/Abl fusion protein inhibitorApproved
RituximabB-lymphocyte antigen CD20 binding agentApproved
ThioguanineInosine-5'-monophosphate dehydrogenase (IMPDH) inhibitorApproved
In phase 2 / 3 · 20
AclarubicinPhase 3
AllopurinolXanthine dehydrogenase inhibitorPhase 3
Antilymphocyte Immunoglobulin (Horse)Phase 3
AsparaginasePhase 3
Bortezomib26S proteasome inhibitorPhase 3
BusulfanPhase 3
Show all 20
CrisantaspasePhase 3
CyclophosphamidePhase 3
CyclosporineCyclophilin A modulatorPhase 3
DactinomycinPhase 3
DaunorubicinDNA topoisomerase II alpha inhibitorPhase 3
Daunorubicin HydrochlorideDNA topoisomerase II alpha inhibitorPhase 3
DexamethasoneGlucocorticoid receptor agonistPhase 3
DexrazoxaneDNA topoisomerase II inhibitorPhase 3
Dexrazoxane HydrochlorideDNA topoisomerase II inhibitorPhase 3
DoxorubicinDNA topoisomerase II alpha inhibitorPhase 3
Doxorubicin HydrochlorideDNA topoisomerase II alpha inhibitorPhase 3
Epirubicin HydrochloridePhase 3
EpratuzumabCD22 inhibitorPhase 3
EtoposideDNA topoisomerase II inhibitorPhase 3
Early trials · 20
Abt-751Tubulin beta inhibitorPhase 1/2
Acyclovir SodiumHuman herpesvirus 1 DNA polymerase inhibitorPhase 1/2
AspacytarabineDNA polymerase (alpha/delta/epsilon) inhibitorPhase 1/2
At-9283Tyrosine-protein kinase ABL inhibitor; Serine/threonine-protein kinase Aurora-A inhibitorPhase 1/2
BirinapantcIAP1/cIAP2 inhibitorPhase 1/2
BleximenibPhase 1/2
Show all 20
Bn-104Phase 1/2
CapivasertibSerine/threonine-protein kinase AKT inhibitorPhase 1/2
CirmtuzumabInactive tyrosine-protein kinase transmembrane receptor ROR1 inhibitorPhase 1/2
CobicistatCytochrome P450 3A inhibitorPhase 1/2
CrenigacestatPhase 1/2
Dexamethasone Phosphoric AcidGlucocorticoid receptor agonistPhase 1/2
Dexamethasone Sodium PhosphateGlucocorticoid receptor agonistPhase 1/2
DordavipronePhase 1/2
EntospletinibTyrosine-protein kinase SYK inhibitorPhase 1/2
EnzomenibPhase 1/2
Epratuzumab-Cys-TesirineB-cell receptor CD22 binding agentPhase 1/2
Forodesine HydrochloridePurine nucleoside phosphorylase inhibitorPhase 1/2
Granaticin BPhase 1/2
IdasanutlinTumour suppressor p53/oncoprotein Mdm2 inhibitorPhase 1/2
🧪

Recruiting trials

Worldwide, open to new patients now
Local Manufacture of CAR T-Cell Products for the Treatment of B-Cell Lymphoma and B-Acute Lymphoblastic Leuke…NCT05281809 · Phase 2 · 📍 PittsburghEvaluation of Treatment Efficacy According to Risk Group in Relapsed Childhood Acute Lymphoblastic LeukemiaNCT05827549 · Phase 2 · 📍 DaeguDemethylating Agents Combined With Venetoclax for High-risk T-cell Lymphoblastic Lymphoma/Leukemia Post-Trans…NCT06686108 · Phase 2 · 📍 ShanghaiCD180 CART for Relapsed or Refractory CD180 Positive Hematologic MalignanciesNCT07106749 · Phase 1 · 📍 TianjinNurture by Nature Feasibility StudyNCT07780123 · NA · 📍 FrederiksbergCAV Regimen for R/R Ph- B-ALLNCT05657652 · NA · 📍 SuzhouPediatric Orthopaedic Implant Safety & EfficacyNCT05361980 · n/a · 📍 VancouverIntestinal Microbiota Transplant Prior to Allogeneic Stem Cell Transplant (MAST) TrialNCT06355583 · Phase 2 · 📍 BirminghamLong-Term Follow-up of Patients Treated With Miltenyi Cell and Gene TherapiesNCT06508775 · NA · 📍 BerlinAnti-CRLF2-R/TSLPR Chimeric Antigen Receptor T Cells (TSLPR-CART) in Participants With Recurrent or Refractor…NCT07572136 · Phase 1 · 📍 BethesdaEfficacy and Safety of TKI Combined With Chemotherapy and Sequential CAR-T Cells in ND Adult Patients With Ph…NCT06481228 · NA · 📍 TianjinKSV01 Injection as the Therapy for Relapsed/Refractory B-Cell Acute Lymphoblastic LeukemiaNCT07246707 · Phase 1 · 📍 Hangzhou Search more trials →
📚

Newest research

Latest papers on PubMed
Fine particulate matter and minimal residual disease in childhood acute lymphoblastic leukemia from the REDIAL consortium.JNCI Cancer Spectr 2026 · PMID 42928613Successful treatment of B-cell acute lymphoblastic leukemia with SET-NUP214 rearrangement using venetoclax: A first case report a…Ann Hematol 2026 · PMID 42928399Recurrent Pleural Effusion as the Initial Presentation of T-lineage Lymphoblastic Malignancy in a Child: A Diagnostic Challenge.Cureus 2026 · PMID 42927411Allogeneic stem cells transplantation in children, adolescents and young adults previously treated by tisagenlecleucel for B-cell…Transplant Cell Ther 2026 · PMID 42926043Sequential Philadelphia-Positive Pre-B Acute Lymphoblastic Leukemia and Chronic Myeloid Leukemia With Distinct BCR::ABL1 Fusion T…Cancer Rep (Hoboken) 2026 · PMID 42924531Disruption of the interferon-gamma axis limits chimeric antigen receptor T cell efficacy against acute myeloid leukemia.bioRxiv 2026 · PMID 42922725Real-World Outcomes of Total Body Irradiation versus Chemotherapy-Based Conditioning in Pediatric Acute Lymphoblastic Leukemia.Clin Epidemiol 2026 · PMID 42921521Clinical and radiological features of posterior reversible encephalopathy syndrome in children: a retrospective study.Front Pediatr 2026 · PMID 42920612 Ask a research question in the Hypothesis Lab →
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