Overview
Multiple endocrine neoplasia type 1 (MEN1), also known as Wermer syndrome, is a hereditary condition characterized by the development of tumors in multiple endocrine glands, most commonly the parathyroid glands, the anterior pituitary gland, and the pancreatic islet cells (enteropancreatic neuroendocrine tumors). The condition is caused by pathogenic variants in the MEN1 gene located on chromosome 11q13, which encodes the tumor suppressor protein menin. Because the disease affects hormone-producing glands, patients often experience symptoms related to hormone overproduction, including elevated calcium levels (hypercalcemia) from primary hyperparathyroidism, which is the most common and often earliest manifestation, occurring in over 90% of affected individuals by age 50. Pancreatic and duodenal neuroendocrine tumors occur in 30–70% of patients and may include gastrinomas (causing Zollinger-Ellison syndrome with peptic ulcers and diarrhea), insulinomas (causing hypoglycemia), and non-functioning tumors. Pituitary adenomas occur in 30–40% of patients, with prolactinomas being the most frequent subtype, potentially causing menstrual irregularities, galactorrhea, or visual field defects. Other associated tumors include adrenal cortical tumors, thymic and bronchial carcinoids, facial angiofibromas, collagenomas, and lipomas. There is currently no cure for MEN1. Management focuses on regular biochemical and imaging surveillance to detect tumors early, along with targeted treatment of individual tumors. Treatment options include surgical removal of tumors (such as parathyroidectomy for hyperparathyroidism), medical management of hormone excess (such as proton pump inhibitors for gastrinomas or dopamine agonists for prolactinomas), and in some cases somatostatin analogs for neuroendocrine tumors. Genetic testing and cascade screening of at-risk family members are strongly recommended to enable early detection and intervention. Lifelong surveillance beginning in childhood is essential, as some manifestations can appear as early as the first decade of life.
Also known as:
Clinical phenotype terms— hover any for plain English:
Autosomal dominant
Passed on from just one parent; each child has about a 50% chance of inheriting it
Variable
Can begin at different ages, from infancy through adulthood
FDA & Trial Timeline
10 eventsMassachusetts General Hospital — PHASE1
University Hospital, Rouen — PHASE3
AstraZeneca — PHASE1, PHASE2
Eilean Therapeutics — PHASE1
Tasca Therapeutics — PHASE1, PHASE2
F.I.R.M.O. - Fondazione Italiana Ricerca sulle Malattie dell'Osso - Ente del Terzo Settore
Arizona State University — NA
PedAL BCU, LLC — PHASE1
Alterome Therapeutics, Inc. — PHASE1
University Ghent — NA
Data sourced from FDA regulatory filings and ClinicalTrials.gov. Updated periodically.
Treatments
No FDA-approved treatments are currently listed for Multiple endocrine neoplasia type 1.
6 clinical trialsare actively recruiting — trials can provide access to cutting-edge therapies.
View clinical trials →Treatment Centers
8 centersBaylor College of Medicine Rare Disease Center ↗
Baylor College of Medicine
📍 Houston, TX
🏥 NORDStanford Medicine Rare Disease Center ↗
Stanford Medicine
📍 Stanford, CA
🔬 UDNNIH Clinical Center Undiagnosed Diseases Program ↗
National Institutes of Health
📍 Bethesda, MD
🔬 UDNUCLA UDN Clinical Site ↗
UCLA Health
📍 Los Angeles, CA
🔬 UDNBaylor College of Medicine UDN Clinical Site ↗
Baylor College of Medicine
📍 Houston, TX
🔬 UDNHarvard/MGH UDN Clinical Site ↗
Massachusetts General Hospital
📍 Boston, MA
🏥 NORDMayo Clinic Center for Individualized Medicine ↗
Mayo Clinic
📍 Rochester, MN
👤 Mayo Clinic Center for Individualized Medicine
🏥 NORDUCLA Rare Disease Day Program ↗
UCLA Health
📍 Los Angeles, CA
Travel Grants
No travel grants are currently matched to Multiple endocrine neoplasia type 1.
Community
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Start the conversation →Latest news about Multiple endocrine neoplasia type 1
Disease timeline:
New recruiting trial: BN104 in Combination With Chemotherapy or Targeted Agents for Acute Myeloid Leukemia
A new clinical trial is recruiting patients for Multiple endocrine neoplasia type 1
New recruiting trial: Study of AZD3632 Monotherapy or in Combination With Anticancer Agents in Participants With Advanced Haematologic Malignancies With KMT2Ar, NPM1m, or Other Genotypes Associated With HOX Overexpression
A new clinical trial is recruiting patients for Multiple endocrine neoplasia type 1
New recruiting trial: Study of the Value of hPG80 (Circulating Progastrin) for the Diagnosis of Neuroendocrine Tumours in Patients With an MEN1 Mutation
A new clinical trial is recruiting patients for Multiple endocrine neoplasia type 1
New recruiting trial: A Phase 1/2 Study of Bleximenib in Participants With Acute Leukemia (cAMeLot-1)
A new clinical trial is recruiting patients for Multiple endocrine neoplasia type 1
New recruiting trial: A Multi-Site Break Through Cancer Trial: Phase II Study Investigating Dual Inhibition of BCL2 and Menin in AML MRD Using the Combination of Venetoclax and Revumenib
A new clinical trial is recruiting patients for Multiple endocrine neoplasia type 1
New recruiting trial: A Phase I-II Study Investigating the All-Oral Combination of the Menin Inhibitor SNDX-5613 With Decitabine/Cedazuridine (ASTX727) and Venetoclax in Acute Myeloid Leukemia (SAVE)
A new clinical trial is recruiting patients for Multiple endocrine neoplasia type 1
New recruiting trial: A Phase II Study of the Menin Inhibitor Revumenib in Leukemia Associated With Upregulation of HOX Genes
A new clinical trial is recruiting patients for Multiple endocrine neoplasia type 1
New recruiting trial: SNDX-5613 and Gilteritinib for the Treatment of Relapsed or Refractory FLT3-Mutated Acute Myeloid Leukemia and Concurrent MLL-Rearrangement or NPM1 Mutation
A new clinical trial is recruiting patients for Multiple endocrine neoplasia type 1
New recruiting trial: Study to Assess Safety, Tolerability, PK, and PD of Multiple Doses of ZE63-0302 Administrated Orally in T2DM Patients.
A new clinical trial is recruiting patients for Multiple endocrine neoplasia type 1
New recruiting trial: Testing the Addition of an Anti-cancer Drug, SNDX-5613, to the Standard Chemotherapy Treatment (Daunorubicin and Cytarabine) for Newly Diagnosed Patients With Acute Myeloid Leukemia That Has Changes in NPM1 or MLL/KMT2A Gene
A new clinical trial is recruiting patients for Multiple endocrine neoplasia type 1
Caregiver Resources
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Family & Caregiver Grants
Financial assistance programs specifically for caregivers of rare disease patients.
Social Security Disability
Learn how rare disease patients may qualify for SSDI/SSI benefits.
Common questions about Multiple endocrine neoplasia type 1
What is Multiple endocrine neoplasia type 1?
Multiple endocrine neoplasia type 1 (MEN1), also known as Wermer syndrome, is a hereditary condition characterized by the development of tumors in multiple endocrine glands, most commonly the parathyroid glands, the anterior pituitary gland, and the pancreatic islet cells (enteropancreatic neuroendocrine tumors). The condition is caused by pathogenic variants in the MEN1 gene located on chromosome 11q13, which encodes the tumor suppressor protein menin. Because the disease affects hormone-producing glands, patients often experience symptoms related to hormone overproduction, including elevated
How is Multiple endocrine neoplasia type 1 inherited?
Multiple endocrine neoplasia type 1 follows a autosomal dominant inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.
Are there clinical trials for Multiple endocrine neoplasia type 1?
Yes — 6 recruiting clinical trials are currently listed for Multiple endocrine neoplasia type 1 on UniteRare. See the clinical trials section on this page for phase, sponsor, and site details sourced from ClinicalTrials.gov.
Which specialists treat Multiple endocrine neoplasia type 1?
11 specialists and care centers treating Multiple endocrine neoplasia type 1 are listed on UniteRare, sourced from ClinicalTrials.gov principal investigators, published research, and the NPPES NPI registry.