MTOR

Chr 1AD

mechanistic target of rapamycin kinase

Also known as: FRAP, FRAP1, FRAP2, RAFT1, RAPT1, SKS

The protein encoded by this gene belongs to a family of phosphatidylinositol kinase-related kinases. These kinases mediate cellular responses to stresses such as DNA damage and nutrient deprivation. This kinase is a component of two distinct complexes, mTORC1, which controls protein synthesis, cell growth and proliferation, and mTORC2, which is a regulator of the actin cytoskeleton, and promotes cell survival and cell cycle progression. This protein acts as the target for the cell-cycle arrest and immunosuppressive effects of the FKBP12-rapamycin complex. Inhibitors of mTOR are used in organ transplants as immunosuppressants, and are being evaluated for their therapeutic potential in SARS-CoV-2 infections. Mutations in this gene are associated with Smith-Kingsmore syndrome and somatic focal cortical dysplasia type II. The ANGPTL7 gene is located in an intron of this gene. [provided by RefSeq, Aug 2020]

OMIMResearchGenerating clinical summary…

Primary Disease Associations & Inheritance

Focal cortical dysplasia, type II, somaticMIM #607341
Smith-Kingsmore syndromeMIM #616638
AD

Clinical highlights

Gene-disease validity (ClinGen)
overgrowth syndrome and/or cerebral malformations due to abnormalities in MTOR pathway genes · ADDefinitivesufficient evidence for diagnostic panels
12
Active trials
Pubs (1 yr)
P/LP submissions
P/LP missense
LOEUF
Multiple*
Mechanism· predicted
Some data sources returned errors (1)

gnomad: TimeoutError: The operation was aborted due to timeout

Population Genetics & Constraint

Constraint data not available from gnomAD.

ClinVar Variant Classifications

0 submitted variants in ClinVar

Protein Context — Lollipop Plot

MTOR · protein map & ClinVar variants

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

VCEP specificationsBrain MalformationsReleased
Specifications ↗Panel ↗

Clinical Trials

Active and recruiting trials from ClinicalTrials.gov

Focal Cortical DysplasiaEpilepsy

Mos-FED (Mosaicism in Focal Epilepsy Cortical Dysplasia Tissue)

RECRUITING
NCT06053671Phase NAKing's College Hospital NHS TrustStarted 2023-04-09
Blood and nasal swab sampling
Lysosomal DiseasesGangliosidosisGM1

A Phase 1/2 Study of Intravenous Gene Transfer With an AAV9 Vector Expressing Human Beta-galactosidase in Type I and Type II GM1 Gangliosidosis

RECRUITING
NCT03952637Phase PHASE1, PHASE2National Human Genome Research Institute (NHGRI)Started 2019-08-19
AAV9-GLB1Abdominal ultrasoundRituximab
Non-Small Cell Lung Cancer

Phase II Umbrella Study of Novel Anti-cancer Agents in Participants With NSCLC Who Progressed on an Anti-PD-1/PD-L1 Containing Therapy

ACTIVE NOT RECRUITING
NCT03334617Phase PHASE2AstraZenecaStarted 2017-12-18
DurvalumabDanvatirsenCeralasertib
Cardiometabolic Health

How Animal and Plant Protein Shakes Change the Body's Nutrient and Health Responses

RECRUITING
NCT07121010Phase NAUniversity of PittsburghStarted 2026-03-24
Animal-Based Protein SmoothiePlant-Based Protein SmoothiePlant-Based Protein Smoothie with Leucine
Non Small Cell Lung Cancer

Metformin Plus Tyrosine Kinase Inhibitors for Treatment of Patients With Non-small Cell Lung Cancer With EGFR Mutations

RECRUITING
NCT05445791Phase PHASE3Instituto Nacional de Cancerologia de MexicoStarted 2021-07-15
Metformin HydrochloridePlacebo
Epidermal Nevi

A Pilot Study to Examine the Efficacy and Tolerability of Sirolimus on Epidermal Nevi Lesions in Patients 6 Years Old and Above.

NOT YET RECRUITING
NCT07813533Phase EARLY_PHASE1Northwestern UniversityStarted 2026-10
sirolimus topical gel 0.2%
Advanced Malignant Solid NeoplasmAnn Arbor Stage III Non-Hodgkin LymphomaAnn Arbor Stage IV Non-Hodgkin Lymphoma

Targeted Therapy Directed by Genetic Testing in Treating Pediatric Patients With Relapsed or Refractory Advanced Solid Tumors, Non-Hodgkin Lymphomas, or Histiocytic Disorders (The Pediatric MATCH Screening Trial)

ACTIVE NOT RECRUITING
NCT03155620Phase PHASE2National Cancer Institute (NCI)Started 2017-07-31
Biopsy ProcedureBiospecimen CollectionBone Marrow Aspiration and Biopsy
Lymphatic MalformationLymphatic AbnormalityLymphangioma

Institution of an Italian Registry and Biobank for Biological Sample Collection

RECRUITING
NCT06892964Phase NAFondazione Policlinico Universitario Agostino Gemelli IRCCSStarted 2024-06-27
Genetic profiling of all eligible patients
Aging

Morning-Evening Specific Longevity Food Supplement

ACTIVE NOT RECRUITING
NCT07416396Phase PHASE1University of PrimorskaStarted 2026-03-07
Morning- and evening-specific compound food supplementControl
Advanced LymphomaAdvanced Malignant Solid NeoplasmBladder Carcinoma

Targeted Therapy Directed by Genetic Testing in Treating Patients With Advanced Refractory Solid Tumors, Lymphomas, or Multiple Myeloma (The MATCH Screening Trial)

ACTIVE NOT RECRUITING
NCT02465060Phase PHASE2National Cancer Institute (NCI)Started 2015-08-17
AdavosertibAfatinibAfatinib Dimaleate
Renal Cell Carcinoma (Kidney Cancer)

Multi-omics Analysis of Renal Cell Carcinoma Mechanisms; Drug Sensitivity Testing in Patient-Derived Cell-based Microtumors

NOT YET RECRUITING
NCT07351266Cancer Institute and Hospital, Chinese Academy of Medical SciencesStarted 2026-01-25
Multi-region Tumor Sampling and Integrated Multi-omics Analysis and Microtumor PTC Drug Sensitivity Assay
Bronchiolitis Obliterans SyndromeChronic Lung Allograft DysfunctionLung Transplantation

LAM-001 in Lung Transplant Recipients With Bronchiolitis Obliterans Syndrome.

RECRUITING
NCT06018766Phase PHASE2Steven Hays, MDStarted 2023-08-17
LAM-001Placebo