NUT midline carcinoma

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5Active trials8Specialists8Treatment centers

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UniteRare data is sourced from FDA.gov, ClinicalTrials.gov, Orphanet, OMIM, and NORD.
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Overview

NUT midline carcinoma (NMC), also called NUT carcinoma or nuclear protein in testis midline carcinoma, is a very rare and aggressive type of cancer. It is caused by a specific change in the NUT gene (also known as NUTM1), which leads to the growth of cancer cells that do not mature or develop normally. These cancer cells tend to grow very quickly and spread to nearby tissues. Despite the name 'midline,' this cancer can appear in many parts of the body, though it most often starts in the head, neck, or chest area — particularly the throat, windpipe, or lungs. It can also occur in other locations like the abdomen. Because NUT carcinoma grows so fast, symptoms often appear suddenly and worsen quickly. Common signs include a lump or mass in the neck or chest, difficulty breathing or swallowing, and pain in the affected area. The cancer can spread to lymph nodes and other organs early in its course. Treatment typically involves a combination of surgery, radiation therapy, and chemotherapy. Newer targeted therapies, especially drugs called BET bromodomain inhibitors, are being studied in clinical trials and show some promise. However, NUT carcinoma remains very difficult to treat, and outcomes are often poor despite aggressive therapy. Early and accurate diagnosis is critical to give patients the best possible chance.

Also known as:

Key symptoms:

A lump or mass in the neck, chest, or throatDifficulty breathing or shortness of breathDifficulty swallowingPersistent coughChest pain or pressureHoarse or changed voiceUnexplained weight lossFatigue and weaknessSwollen lymph nodesNasal congestion or blockageNosebleedsFever without a clear cause

Clinical phenotype terms (9)— hover any for plain English
LeukemiaHP:0001909Squamous cell carcinomaHP:0002860NeuroblastomaHP:0003006Oropharyngeal squamous cell carcinomaHP:0012182Ewing sarcomaHP:0012254Abnormal mediastinum morphologyHP:0045026PancreatoblastomaHP:0100757Pancreatic squamous cell carcinomaHP:0012142
Inheritance

Sporadic

Usually appears on its own, not inherited from a parent

Age of Onset

Variable

Can begin at different ages, from infancy through adulthood

Orphanet ↗NORD ↗

FDA & Trial Timeline

5 events
Aug 2025Cemiplimab for the Treatment of Incurable Metastatic or Unresectable NUT Carcinoma

Northwestern University — PHASE2

TrialRECRUITING
Aug 2023Testing the Safety and Efficacy of the Combination of Two Anti-cancer Drugs, ZEN003694 and Abemaciclib, for Adult and Pediatric Patients (12-17 Years) With Metastatic or Unresectable NUT Carcinoma, Breast Cancer and Other Solid Tumors

National Cancer Institute (NCI) — PHASE1

TrialRECRUITING
Jan 2023Multicenter Retrospective Cohort of Pulmonary NUT Carcinoma

Three Gorges Hospital of Chongqing University

TrialACTIVE NOT RECRUITING
Jan 2023Biology of Young Lung Cancer Study: The YOUNG LUNG Study

Dana-Farber Cancer Institute

TrialRECRUITING
Jul 2022Testing the Safety and Efficacy of the Addition of a New Anti-cancer Drug, ZEN003694, to Chemotherapy Treatment (Cisplatin and Etoposide or Carboplatin and Paclitaxel) for Adult and Pediatric Patients (12-17 Years) With NUT Carcinoma

National Cancer Institute (NCI) — PHASE1, PHASE2

TrialRECRUITING

Data sourced from FDA regulatory filings and ClinicalTrials.gov. Updated periodically.

Treatments

No FDA-approved treatments are currently listed for NUT midline carcinoma.

5 clinical trialsare actively recruiting — trials can provide access to cutting-edge therapies.

View clinical trials →

Clinical Trials

5 recruitingView all trials with filters →
Phase 21 trial
Cemiplimab for the Treatment of Incurable Metastatic or Unresectable NUT Carcinoma
Phase 2
Actively Recruiting
PI: Jochen H Lorch, MD (Northwestern University) · Sites: Chicago, Illinois · Age: 1899 yrs
Phase 11 trial
Testing the Safety and Efficacy of the Combination of Two Anti-cancer Drugs, ZEN003694 and Abemaciclib, for Adult and Pediatric Patients (12-17 Years) With Metastatic or Unresectable NUT Carcinoma, Breast Cancer and Other Solid Tumors
Phase 1
Actively Recruiting
PI: Jia Luo (Dana-Farber - Harvard Cancer Center LAO) · Sites: Los Angeles, California; Los Angeles, California +5 more · Age: 1299 yrs
Other2 trials
Multicenter Retrospective Cohort of Pulmonary NUT Carcinoma
Active
· Sites: Chongqing, Chongqing Municipality
Biology of Young Lung Cancer Study: The YOUNG LUNG Study
Actively Recruiting
PI: Pasi A Janne, MD, PhD (Dana-Farber Cancer Institute) · Sites: Boston, Massachusetts; Boston, Massachusetts +2 more · Age: 1599 yrs

Specialists

8 foundView all specialists →
JL
Jia Luo
Specialist
PI on 2 active trials207 NUT midline carcinoma publications
JM
Jochen H Lorch, MD
CHICAGO, IL
Specialist
PI on 1 active trial
JM
Judy Chiao, MD
Specialist
PI on 1 active trial
DM
Dawn Pinchasik, MD
PITTSBURGH, PA
Specialist
PI on 2 active trials
PP
Pasi A Janne, MD, PhD
BOSTON, MA
Specialist
PI on 2 active trials
GM
Gianni Bisogno, MD
Specialist
PI on 2 active trials
GS
Geoffrey I Shapiro
Specialist
PI on 1 active trial

Treatment Centers

8 centers
🏥 NORD

Baylor College of Medicine Rare Disease Center

Baylor College of Medicine

📍 Houston, TX

🏥 NORD

Stanford Medicine Rare Disease Center

Stanford Medicine

📍 Stanford, CA

🔬 UDN

NIH Clinical Center Undiagnosed Diseases Program

National Institutes of Health

📍 Bethesda, MD

🔬 UDN

UCLA UDN Clinical Site

UCLA Health

📍 Los Angeles, CA

🔬 UDN

Baylor College of Medicine UDN Clinical Site

Baylor College of Medicine

📍 Houston, TX

🔬 UDN

Harvard/MGH UDN Clinical Site

Massachusetts General Hospital

📍 Boston, MA

🏥 NORD

Mayo Clinic Center for Individualized Medicine

Mayo Clinic

📍 Rochester, MN

👤 Mayo Clinic Center for Individualized Medicine

🏥 NORD

UCLA Rare Disease Day Program

UCLA Health

📍 Los Angeles, CA

Travel Grants

No travel grants are currently matched to NUT midline carcinoma.

Search all travel grants →NORD Financial Assistance ↗

Community

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Latest news about NUT midline carcinoma

Disease timeline:

New recruiting trial: Testing the Safety and Efficacy of the Combination of Two Anti-cancer Drugs, ZEN003694 and Abemaciclib, for Adult and Pediatric Patients (12-17 Years) With Metastatic or Unresectable NUT Carcinoma, Breast Cancer and Other Solid Tumors

A new clinical trial is recruiting patients for NUT midline carcinoma

New recruiting trial: Biology of Young Lung Cancer Study: The YOUNG LUNG Study

A new clinical trial is recruiting patients for NUT midline carcinoma

New recruiting trial: Testing the Safety and Efficacy of the Addition of a New Anti-cancer Drug, ZEN003694, to Chemotherapy Treatment (Cisplatin and Etoposide or Carboplatin and Paclitaxel) for Adult and Pediatric Patients (12-17 Years) With NUT Carcinoma

A new clinical trial is recruiting patients for NUT midline carcinoma

New recruiting trial: Cemiplimab for the Treatment of Incurable Metastatic or Unresectable NUT Carcinoma

A new clinical trial is recruiting patients for NUT midline carcinoma

Caregiver Resources

NORD Caregiver Resources

Support, advocacy, and financial assistance for caregivers of rare disease patients.

Mental Health Support

Rare disease caregiving can be isolating. Connect with counseling and peer support.

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.

Questions for your doctor

Bring these to your next appointment

  • Q1.What specific gene fusion was found in my tumor, and does it affect my treatment options?,Are there any clinical trials for NUT carcinoma that I might be eligible for?,What is the goal of the treatment plan — to cure the cancer or to control it and manage symptoms?,Should I be seen at a specialized cancer center with experience in NUT carcinoma?,What are the most important warning signs that my condition is getting worse and when should I go to the emergency room?,How will treatment affect my ability to eat, breathe, and carry out daily activities?,Is palliative care or a supportive care team available to help manage my symptoms and quality of life?

Common questions about NUT midline carcinoma

What is NUT midline carcinoma?

NUT midline carcinoma (NMC), also called NUT carcinoma or nuclear protein in testis midline carcinoma, is a very rare and aggressive type of cancer. It is caused by a specific change in the NUT gene (also known as NUTM1), which leads to the growth of cancer cells that do not mature or develop normally. These cancer cells tend to grow very quickly and spread to nearby tissues. Despite the name 'midline,' this cancer can appear in many parts of the body, though it most often starts in the head, neck, or chest area — particularly the throat, windpipe, or lungs. It can also occur in other location

How is NUT midline carcinoma inherited?

NUT midline carcinoma follows a sporadic inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.

Are there clinical trials for NUT midline carcinoma?

Yes — 5 recruiting clinical trials are currently listed for NUT midline carcinoma on UniteRare. See the clinical trials section on this page for phase, sponsor, and site details sourced from ClinicalTrials.gov.

Which specialists treat NUT midline carcinoma?

8 specialists and care centers treating NUT midline carcinoma are listed on UniteRare, sourced from ClinicalTrials.gov principal investigators, published research, and the NPPES NPI registry.