ATM

Chr 11ADSomaticAR

ATM serine/threonine kinase

Also known as: AT1, ATA, ATC, ATD, ATDC, ATE, TEL1, TELO1

The ATM protein is a cell cycle checkpoint kinase that phosphorylates key downstream proteins including p53, BRCA1, and CHK2 to control DNA damage response and maintain genome stability. Biallelic mutations cause ataxia-telangiectasia, an autosomal recessive disorder, while heterozygous mutations confer dominant susceptibility to breast cancer. The pathogenic mechanism involves dominant-negative effects for cancer predisposition and loss of function for ataxia-telangiectasia.

OMIMResearchSummary from RefSeq, OMIM, UniProt, Mechanism
LOFmechanismAD/Somatic/ARLOEUF 0.715 OMIM phenotypes
Clinical SummaryATM
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Gene-Disease Validity (ClinGen)
ataxia telangiectasia · ARDefinitive

Definitive — sufficient evidence for diagnostic panels

2 total gene-disease associations curated

Population Constraint (gnomAD)
Low constraint (pLI 0.00) — loss-of-function variants are relatively tolerated in the population.
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Clinical Trials
12 active or recruiting trials — potential therapeutic options may be available

Population Genetics & Constraint

gnomAD v4 — loss-of-function & missense intolerance

Tolerant — LoF & missense variants common in population
LoF Constraint
0.71LOEUF
pLI 0.000
Z-score 4.82
OE 0.60 (0.510.71)
Tolerant

Typical tolerance to LoF variation

Missense Constraint
1.10Z-score
OE missense 0.92 (0.880.96)
1411 obs / 1532.7 exp
Tolerant

Mild missense constraint

Observed / Expected Ratios
LoF OE0.60 (0.510.71)
00.351.4
Missense OE0.92 (0.880.96)
00.61.4
Synonymous OE1.05
01.21.6
LoF obs/exp: 103 / 171.0Missense obs/exp: 1411 / 1532.7Syn Z: -0.86
Curated Mechanism (G2P)Gene2Phenotype (DDG2P) ↗
definitiveATM-related cancerLOFAD
definitiveATM-related ataxia-telangiectasiaLOFAR
DN
0.6552th %ile
GOF
0.4381th %ile
LOF
0.3261th %ile

This gene has evidence for multiple mechanisms of pathogenicity (dominant-negative and loss-of-function). Both the Badonyi & Marsh prediction and the broader genomic evidence point to dominant-negative as the predominant mechanism. Different variants in this gene may act through different mechanisms — interpret in context of the specific variant.

DNprediction above median · 1 literature citation
LOF1 literature citation

Note: In-silico variant effect predictors (SIFT, PolyPhen, REVEL, CADD) may underestimate pathogenicity of missense variants in genes with GOF or DN mechanisms. Consider functional evidence and clinical context.

Literature Evidence

DNDominant negative ATM mutations in breast cancer familiesPMID:11830610
LOFThis study examined the cancer incidence of individuals with heterozygous pathogenic variants in over 120 families with ataxia telangiectasia (A-T). The cancer incidence in adult blood relatives of A-T individuals were compared to their adult spouses. The adjusted cancer rate ratio was 1.6 for malesPMID:3574400

Predictions from Badonyi M, Marsh JA. PLoS ONE. 2024;19(8):e0307312.

ClinVar Variant Classifications

0 submitted variants in ClinVar

Protein Context — Lollipop Plot

ATM · protein map & ClinVar variants

Showing all ClinVar variants across the protein. Search a specific variant to highlight its position.

3D Protein StructureAlphaFold

Clinical Trials

Active and recruiting trials from ClinicalTrials.gov

Advanced Solid TumorsEwing SarcomaHepatocellular Carcinoma (HCC)

A Phase 1/1B Study of ST-01156, a Small Molecule RBM39 Degrader, in Patients With Advanced Solid Malignancies

RECRUITING
NCT07197554Phase PHASE1SEED Therapeutics, Inc.Started 2025-12-01
ST-01156
Malignant Neoplasm of BreastBreast Cancer

Comprehensive Analysis of Predictors of the Treatment With Pembrolizumab and Olaparib in Patients With Unresectable or Metastatic HER2 Negative Breast Cancer and a Deleterious Germline Mutation or a Homologous Recombination Deficiency (COMPRENDO

ACTIVE NOT RECRUITING
NCT05033756Phase PHASE2Institut fuer FrauengesundheitStarted 2022-07-30
Pembrolizumab Injection [Keytruda]Olaparib Oral Tablet [Lynparza]
Solid TumorMetastatic Cancer

A Study of RP-3500 in Combination With Standard Radiation Therapy in People With Solid Tumor Cancer

ACTIVE NOT RECRUITING
NCT05566574Phase PHASE1, PHASE2Memorial Sloan Kettering Cancer CenterStarted 2022-09-30
RP-3500External Beam Radiotherapy (EBRT)
Pancreatic CancerPancreatic Ductal AdenocarcinomaPDAC

A Prospective Registry for Patients at High-Risk for Pancreatic Cancer

RECRUITING
NCT06151223Mayo ClinicStarted 2021-07-13
Bio-specimen Collection: BloodBio-specimen Collection: Pancreatic JuiceMRI
Metastatic Prostate Cancer

High Dose Testosterone for ATM, CDK12 or CHEK2 Altered Prostate Cancers

RECRUITING
NCT05011383Phase PHASE2VA Office of Research and DevelopmentStarted 2021-08-31
High dose testosterone
Metastatic Solid TumorBRCA1 MutationBRCA2 Mutation

Combination Therapy in Cancers With Mutations in DNA Repair Genes

RECRUITING
NCT05694715Phase PHASE1University of California, San FranciscoStarted 2023-05-23
NiraparibIrinotecan
Prostate Cancer

PROMISE Registry: A Prostate Cancer Registry of Outcomes and Germline Mutations for Improved Survival and Treatment Effectiveness

RECRUITING
NCT04995198Prostate Cancer Clinical Trials ConsortiumStarted 2021-05-03
Solid TumorAdvanced Solid TumorMetastatic Cancer

KPMNG Study of MOlecular Profiling Guided Therapy Based on Genomic Alterations in Advanced Solid Tumors II

RECRUITING
NCT05525858Seoul National University Bundang HospitalStarted 2022-09-28
AlectinibAtezolizumabErlotinib
BRCA MutationPALB2 Gene MutationDuctal Carcinoma in Situ

Low Dose TamOxifen and LifestylE Changes for bReast cANcer prevenTion

ACTIVE NOT RECRUITING
NCT06033092Phase PHASE2European Institute of OncologyStarted 2024-06-21
Tamoxifen 10 mg TabletIntermittent caloric restrictionStep counter Device
Polycythemia VeraEssential ThrombocythaemiaMyelofibrosis

Prevalence Of Germline Gene Mutations In Patients With Myeloproliferative Neoplasms With Family History

NOT YET RECRUITING
NCT06923670Phase NAFondazione Policlinico Universitario Agostino Gemelli IRCCSStarted 2025-05-21
NGS testingNGS analysis for mutations in genes involved in familial predisposition to hematological malignancies
Breast CancerTriple Negative Breast Neoplasms

NordicTrip, a Translational Study of Preoperative Chemotherapy in TNBC

ACTIVE NOT RECRUITING
NCT04335669Phase PHASE3Lund University HospitalStarted 2019-12-20
epirubicin, cyclophosphamide, paclitaxel, carboplatin, pembrolizumabepirubicin, cyclophosphamide, capecitabine, paclitaxel, carboplatin, pembrolizumab
Pancreatic Cancer, ATM, BRCA, Hereditary Cancer

Pancreatic Cancer Registry: For Any Person With a Personal or Family History

RECRUITING
NCT02886247Johns Hopkins UniversityStarted 1994-06
Clinical Literature
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