OncoSignal
Live from Open Targets Platform, fetched 2026-11-11 20:50 UTC. Every row links to its source record.

NF1 neurofibromin 1

ENSG00000196712 · protein coding · Open Targets profile

Stimulates the GTPase activity of Ras. NF1 shows greater affinity for Ras GAP, but lower specific activity. May be a regulator of Ras activity. {ECO:0000269|PubMed:2121371, ECO:0000269|PubMed:8417346}.

In 12 of 15 cancers, the evidence for NF1 comes mainly from inherited variants

Each row is a cancer, strongest overall evidence first. The two dots show how strong the inherited (germline) and tumour-mutation (somatic) evidence is; the line between them is the gap.

Inherited (germline) variantsMutations in tumours (somatic)
00.250.50.751neurofibromatosis type 1neurofibromatosis type 1neurofibromatosis type 1: somatic 0.90neurofibromatosis type 1: germline 0.98neurofibromatosis-Noonan syndromeneurofibromatosis-Noonan syndromeneurofibromatosis-Noonan syndrome: germline 0.91neurofibromatosisneurofibromatosisneurofibromatosis: germline 0.89juvenile myelomonocytic leukemiajuvenile myelomonocytic leukemiajuvenile myelomonocytic leukemia: somatic 0.46juvenile myelomonocytic leukemia: germline 0.95Watson syndromeWatson syndromeWatson syndrome: germline 0.94neurofibromatosis, familial spinalneurofibromatosis, familial spinalneurofibromatosis, familial spinal: germline 0.94malignant peripheral nerve sheath tumormalignant peripheral nerve sheath tumormalignant peripheral nerve sheath tumor: somatic 0.61malignant peripheral nerve sheath tumor: germline 0.92neurofibromaneurofibromaneurofibroma: somatic 0.61neurofibroma: germline 0.89rhabdomyosarcomarhabdomyosarcomarhabdomyosarcoma: somatic 0.58rhabdomyosarcoma: germline 0.84melanomamelanomamelanoma: somatic 0.79urinary bladder carcinomaurinary bladder carcinomaurinary bladder carcinoma: somatic 0.80urinary bladder cancerurinary bladder cancerurinary bladder cancer: somatic 0.80benign neoplasmbenign neoplasmbenign neoplasm: germline 0.79nervous system benign neoplasmnervous system benign neoplasmnervous system benign neoplasm: germline 0.96autonomic nervous system neoplasmautonomic nervous system neoplasmautonomic nervous system neoplasm: germline 0.96

Scores run from 0 (no evidence) to 1 (strongest). A missing dot at 0 means Open Targets holds no evidence of that kind. Source: Open Targets Platform, fetched live.

Tractability

Small molecule
Structure with Ligand, High-Quality Pocket
Antibody
UniProt loc high conf, GO CC high conf, Human Protein Atlas loc
PROTAC
UniProt Ubiquitination, Database Ubiquitination, Half-life Data

gnomAD loss of function constraint: o/e 0.3745400011539459 (upper bound 0.4353100061416626). Low values mean loss of function is poorly tolerated, a safety flag for inhibition.

Who runs trials of these drugs

No named investigators on registry trials of NF1's approved or late-stage drugs in the last three years.

No terminated, withdrawn or suspended trials of NF1's drugs are registered

All 0 drugs and clinical candidates as a table

Each drug, stage and indication links to its Open Targets record, where the trials and approvals behind it are listed. Salt forms (for example a mesylate) are listed as Open Targets records them, as separate entries.

DrugTypeHighest stageIndicationsSource records
No drugs or clinical candidates recorded.