RARE DISEASE
Bronchial neuroendocrine tumor
Bronchial neuroendocrine tumor
Bronchial neuroendocrine tumor
Synonyms: Bronchial NET
Synonyms: Bronchial NET
Synonyms: Bronchial NET
Drug discovery
6
drugs
With orphan designations
Overview
Bronchial Neuroendocrine Tumors (BNETs) are rare, heterogeneous malignancies arising from pulmonary neuroendocrine cells, accounting for ~20% of lung cancers [1][2]. They encompass four subtypes: typical carcinoid (TC), atypical carcinoid (AC), large-cell neuroendocrine carcinoma (LCNEC), and small-cell lung carcinoma (SCLC) [1][16]. Symptoms include cough, hemoptysis, or obstructive pneumonia; <5% present with hormonal syndromes (e.g., carcinoid or Cushing syndrome) [1][7]. Prognosis varies widely, with 5-year survival ranging from >88% (TC) to <5% (SCLC) [1][2].
Therapies
Localized Disease: Surgical resection (lobectomy/pneumonectomy ± lymphadenectomy) [4][13].
Advanced Disease: Somatostatin analogs (symptom/tumor control), CAPTEM chemotherapy (response rate ≥30%), peptide receptor radionuclide therapy (PRRT), and targeted agents (e.g., everolimus) [3][8][18].
High-Grade Tumors: Platinum-based chemotherapy (SCLC/LCNEC); immunotherapy for mismatch repair-deficient cases [3][18].
Categories: rare endocrine diseases, rare neoplastic diseases, rare respiratory diseases
Research Papers
340 drug discovery papers about Bronchial neuroendocrine tumor, with 2 first-in-class emerging drug candidates forecasted to outperform the average preclinical success rate. Recent publications:
340 drug discovery papers about Bronchial neuroendocrine tumor, with 2 first-in-class emerging drug candidates forecasted to outperform the average preclinical success rate. Recent publications:
2026-07-23 | Small Cell Lung Cancer: From Histogenesis to a New Therapeutic Paradigm
Pulmonary neuroendocrine neoplasms (NENs) form a histogenetic continuum arising from bronchial Kulchitsky cells, ranging from well-differentiated carcinoids to highly aggressive neuroendocrine carcinomas. The 2021 WHO Classification of Thoracic Tumors maintains a morphology-based, grade-linked framework for typical carcinoid (TC), atypical carcinoid (AC), large cell neuroendocrine carcinoma (LCNEC), and small cell lung cancer (SCLC), defined by mitotic index and necrosis. Genomic data reinforce a fundamental dichotomy: carcinoids harbor low tumor mutational burden with frequent chromatin-remodeling alterations (e.g., MEN1) and wild-type TP53/RB1, whereas high-grade carcinomas are unified by concurrent TP53 and RB1 inactivation. LCNEC occupies a heterogeneous intermediate position, comprising SCLC-like (TP53/RB1 co-inactivated) and NSCLC-like (KRAS, STK11, KEAP1) subsets, explaining therapeutic variability and motivating molecularly stratified management. In SCLC, precise pathological diagnosis is essential given its immediate therapeutic implications. Immunohistochemistry — synaptophysin, chromogranin A, CD56, and INSM1 — remains indispensable, particularly in compromised small-biopsy specimens. Therapeutically, platinum-based chemotherapy combined with immunotherapy yields modest but reproducible survival gains. Molecular subtyping has uncovered lineage-based vulnerabilities, with DLL3-targeted bispecific T-cell engagers achieving the first validated subtype-specific regulatory approval. Nevertheless, clinical translation of biologically rational strategies has broadly underperformed in late-phase trials. Tumor plasticity and phenotypic switching between subtypes remain critical barriers to durable responses. Integrating histology, immunophenotype, and genomics refines diagnosis and risk stratification, yet the transition from biological hypothesis to validated precision oncology in pulmonary NENs remains incomplete.
2026-06-11 | Alternating Therapy With Osilodrostat and Etomidate in Severe Ectopic Cushing's Syndrome Complicated by Silent Bowel Perforation.
Ectopic adrenocorticotropic hormone (ACTH)-dependent Cushing's syndrome (CS), ectopic ACTH secretion (EAS) is a rare condition caused by ACTH-secreting neuroendocrine tumors (NETs), such as bronchial carcinoids. We report a 65-year-old woman with severe EAS complicated by bowel perforation. She presented with hypokalemia (K+ 2.3 mmol/L), metabolic alkalosis, resistant hypertension (180/110 mmHg), worsening diabetes (HbA1c 6.7%-9.1%), proximal muscle weakness, and 14 kg weight gain over 3 months. A silent sigmoid colon perforation required emergency resection and colostomy. Biochemical tests confirmed hypercortisolism (urine free cortisol [UFC], 1256 µg/24 h, plasma ACTH 175 pg/mL, and cortisol >40 µg/dL post-dexamethasone). Imaging identified a 2.3 cm pulmonary nodule with mild uptake on Ga-68 DOTATATE PET/CT. Bronchoscopic biopsy confirmed an ACTH-positive low-grade bronchial carcinoid tumor. Initial treatment with osilodrostat was interrupted due to acute illness and oral medication intolerance. Intravenous etomidate was employed in the ICU for rapid cortisol suppression, followed by resumption of osilodrostat after stabilization. Thoracoscopic lobectomy confirmed a low-grade carcinoid tumor (Ki-67 < 2%). Postoperatively, cortisol normalized, electrolytes stabilized, and HbA1c improved to 6.5%. This case highlights bowel perforation as a severe complication of EAS and underscores the importance of dynamic, alternating therapy with osilodrostat and etomidate, along with individualized surgical and medical management strategies.
2026-05-01 | C79-17 Bronchoscopic and Surgical Treatment of Endobronchial Carcinoid Incidentally Diagnosed in a Woman With Abdominal Pain
Abstract Introduction Endobronchial carcinoid is a rare and slow-growing neoplasm of neuroendocrine origin that arises within the bronchus. This case demonstrates the effective use of both bronchoscopic and surgical approaches in the management of endobronchial carcinoid tumors. Case Report A 58-year-old female with no significant past medical history presented with abdominal pain to the emergency department. Computed tomography (CT) of the abdomen and pelvis incidentally revealed an occlusive endobronchial mass in the basilar trunk of the right lower lobe, which was confirmed on CT chest. Upon further questioning, she endorsed a chronic, persistent post-pneumonic cough with occasional production of white sputum. The patient was scheduled for outpatient bronchoscopy. Interventional bronchoscopic recanalization demonstrated an occlusive polypoid mass. Pathology was positive for typical carcinoid cells. A staging positron emission tomography demonstrated a small persistent endobronchial nodule with no functional or anatomical evidence of metastatic disease. A right lower lobectomy is now planned with cardiothoracic surgery. Discussion Endobronchial carcinoid tumors typically originate from bronchial mucosa, particularly from the right bronchial tree. Less commonly arising from tracheobronchial cartilage. Cell types are divided into typical and atypical carcinoids - defined by mutations in genes such as p53, BCL2, or BAX. Atypical carcinoids being associated with poorer outcomes. This slow-growing neoplasm accounts for approximately 1-2% of all lung cancers and, due to its indolent nature, is commonly discovered incidentally. Approximately 25% of patients are asymptomatic at diagnosis. Symptoms include productive cough, chest pain, wheezing, hemoptysis, and recurrent post-obstructive pneumonia. As a consequence of its neuroendocrine origin, some patients may present with new-onset Cushing’s syndrome due to ectopic ACTH secretion. Surgical resection is often first-line treatment. A recent clinical trial by Neuberger et al. (2021) compared outcomes in patients treated with bronchoscopic intervention, surgery, or a combination of both. It was found that the latter resulted in a statistically significant increase in long-term survival. Therefore, as seen in our case, early diagnosis and timely intervention are critical for optimizing positive outcomes in endobronchial carcinoid. This abstract is funded by: none
2026-03-13 | Multidisciplinary management of endobronchial carcinoid tumours through a combined bronchoscopic and surgical approach.
We report the case of a 44-year-old woman with a typical carcinoid tumour of the left mainstem bronchus managed through a multidisciplinary and combined approach. Initial symptoms included haemoptysis and progressive wheezing. A subsequent bronchoscopy revealed a highly vascular, pedunculated mass nearly occluding the airway. En bloc resection using electrocautery and a cryoprobe enabled airway recanalization and diagnosis. Endobronchial ultrasound-guided transbronchial fine needle aspiration ruled out nodal disease. Histopathological examination confirmed a typical carcinoid tumour. Robotic-assisted sleeve resection was subsequently performed, preserving parenchyma and achieving negative margins. Surveillance bronchoscopy showed a well-healed anastomosis with no granulation. This case highlights the importance of bronchoscopic interventions in staging and treatment planning, as well as their role in facilitating lung-sparing surgical strategies.
2025-08-21 | Preoperative Embolization of a Pulmonary Carcinoid Tumor with Unusual Arterial Supply: A Case Report
Background Pulmonary carcinoid tumors are rare neuroendocrine neoplasms that carry a risk of bleeding. We report a case of a tumor with unusual arterial supply from multiple sources including collateral from the renal artery, successfully managed with preoperative embolization followed by surgical resection. Case presentation: A 39-year-old woman with no significant medical history presented with chronic cough. Chest radiography revealed an opacity in the right hilar region. Computed tomography (CT) showed a mass obstructing the right middle lobe bronchus. 18F-fluorodeoxyglucose positron emission tomography (FDG-PET) demonstrated no abnormal uptake in the lesion. However, a Ga-68 DOTATATE somatostatin receptor scan (DOTA-PET) showed intense tracer uptake in the mass. Bronchoscopy identified a reddish endobronchial lesion completely occluding the middle lobe bronchus and causing distal collapse. Biopsy was deferred due to the lesion’s hypervascular appearance. With a presumptive diagnosis of carcinoid tumor, a multidisciplinary team planned for surgical resection. To mitigate intraoperative bleeding, preoperative angiographic evaluation was performed. Digital subtraction angiography (DSA) revealed that the blood supply originated from the right bronchial artery, right internal mammary artery and right renal artery. All identified feeding arteries were selectively catheterized and embolized with coils. The patient underwent a right middle lobectomy. Intraoperative blood loss was minimal, and the tumor was resected completely. Pathology confirmed carcinoid tumor. At two-year follow-up, the patient remains asymptomatic. Conclusions This case highlights a rare arterial supply to a lung tumor and demonstrates that preoperative embolization of multiple feeding vessels can facilitate safe resection of a hypervascular tumor. Awareness of vascular anomalies and multidisciplinary approach were key to successful management.
2026-07-23 | Small Cell Lung Cancer: From Histogenesis to a New Therapeutic Paradigm
Pulmonary neuroendocrine neoplasms (NENs) form a histogenetic continuum arising from bronchial Kulchitsky cells, ranging from well-differentiated carcinoids to highly aggressive neuroendocrine carcinomas. The 2021 WHO Classification of Thoracic Tumors maintains a morphology-based, grade-linked framework for typical carcinoid (TC), atypical carcinoid (AC), large cell neuroendocrine carcinoma (LCNEC), and small cell lung cancer (SCLC), defined by mitotic index and necrosis. Genomic data reinforce a fundamental dichotomy: carcinoids harbor low tumor mutational burden with frequent chromatin-remodeling alterations (e.g., MEN1) and wild-type TP53/RB1, whereas high-grade carcinomas are unified by concurrent TP53 and RB1 inactivation. LCNEC occupies a heterogeneous intermediate position, comprising SCLC-like (TP53/RB1 co-inactivated) and NSCLC-like (KRAS, STK11, KEAP1) subsets, explaining therapeutic variability and motivating molecularly stratified management. In SCLC, precise pathological diagnosis is essential given its immediate therapeutic implications. Immunohistochemistry — synaptophysin, chromogranin A, CD56, and INSM1 — remains indispensable, particularly in compromised small-biopsy specimens. Therapeutically, platinum-based chemotherapy combined with immunotherapy yields modest but reproducible survival gains. Molecular subtyping has uncovered lineage-based vulnerabilities, with DLL3-targeted bispecific T-cell engagers achieving the first validated subtype-specific regulatory approval. Nevertheless, clinical translation of biologically rational strategies has broadly underperformed in late-phase trials. Tumor plasticity and phenotypic switching between subtypes remain critical barriers to durable responses. Integrating histology, immunophenotype, and genomics refines diagnosis and risk stratification, yet the transition from biological hypothesis to validated precision oncology in pulmonary NENs remains incomplete.
2026-06-11 | Alternating Therapy With Osilodrostat and Etomidate in Severe Ectopic Cushing's Syndrome Complicated by Silent Bowel Perforation.
Ectopic adrenocorticotropic hormone (ACTH)-dependent Cushing's syndrome (CS), ectopic ACTH secretion (EAS) is a rare condition caused by ACTH-secreting neuroendocrine tumors (NETs), such as bronchial carcinoids. We report a 65-year-old woman with severe EAS complicated by bowel perforation. She presented with hypokalemia (K+ 2.3 mmol/L), metabolic alkalosis, resistant hypertension (180/110 mmHg), worsening diabetes (HbA1c 6.7%-9.1%), proximal muscle weakness, and 14 kg weight gain over 3 months. A silent sigmoid colon perforation required emergency resection and colostomy. Biochemical tests confirmed hypercortisolism (urine free cortisol [UFC], 1256 µg/24 h, plasma ACTH 175 pg/mL, and cortisol >40 µg/dL post-dexamethasone). Imaging identified a 2.3 cm pulmonary nodule with mild uptake on Ga-68 DOTATATE PET/CT. Bronchoscopic biopsy confirmed an ACTH-positive low-grade bronchial carcinoid tumor. Initial treatment with osilodrostat was interrupted due to acute illness and oral medication intolerance. Intravenous etomidate was employed in the ICU for rapid cortisol suppression, followed by resumption of osilodrostat after stabilization. Thoracoscopic lobectomy confirmed a low-grade carcinoid tumor (Ki-67 < 2%). Postoperatively, cortisol normalized, electrolytes stabilized, and HbA1c improved to 6.5%. This case highlights bowel perforation as a severe complication of EAS and underscores the importance of dynamic, alternating therapy with osilodrostat and etomidate, along with individualized surgical and medical management strategies.
2026-05-01 | C79-17 Bronchoscopic and Surgical Treatment of Endobronchial Carcinoid Incidentally Diagnosed in a Woman With Abdominal Pain
Abstract Introduction Endobronchial carcinoid is a rare and slow-growing neoplasm of neuroendocrine origin that arises within the bronchus. This case demonstrates the effective use of both bronchoscopic and surgical approaches in the management of endobronchial carcinoid tumors. Case Report A 58-year-old female with no significant past medical history presented with abdominal pain to the emergency department. Computed tomography (CT) of the abdomen and pelvis incidentally revealed an occlusive endobronchial mass in the basilar trunk of the right lower lobe, which was confirmed on CT chest. Upon further questioning, she endorsed a chronic, persistent post-pneumonic cough with occasional production of white sputum. The patient was scheduled for outpatient bronchoscopy. Interventional bronchoscopic recanalization demonstrated an occlusive polypoid mass. Pathology was positive for typical carcinoid cells. A staging positron emission tomography demonstrated a small persistent endobronchial nodule with no functional or anatomical evidence of metastatic disease. A right lower lobectomy is now planned with cardiothoracic surgery. Discussion Endobronchial carcinoid tumors typically originate from bronchial mucosa, particularly from the right bronchial tree. Less commonly arising from tracheobronchial cartilage. Cell types are divided into typical and atypical carcinoids - defined by mutations in genes such as p53, BCL2, or BAX. Atypical carcinoids being associated with poorer outcomes. This slow-growing neoplasm accounts for approximately 1-2% of all lung cancers and, due to its indolent nature, is commonly discovered incidentally. Approximately 25% of patients are asymptomatic at diagnosis. Symptoms include productive cough, chest pain, wheezing, hemoptysis, and recurrent post-obstructive pneumonia. As a consequence of its neuroendocrine origin, some patients may present with new-onset Cushing’s syndrome due to ectopic ACTH secretion. Surgical resection is often first-line treatment. A recent clinical trial by Neuberger et al. (2021) compared outcomes in patients treated with bronchoscopic intervention, surgery, or a combination of both. It was found that the latter resulted in a statistically significant increase in long-term survival. Therefore, as seen in our case, early diagnosis and timely intervention are critical for optimizing positive outcomes in endobronchial carcinoid. This abstract is funded by: none
2026-03-13 | Multidisciplinary management of endobronchial carcinoid tumours through a combined bronchoscopic and surgical approach.
We report the case of a 44-year-old woman with a typical carcinoid tumour of the left mainstem bronchus managed through a multidisciplinary and combined approach. Initial symptoms included haemoptysis and progressive wheezing. A subsequent bronchoscopy revealed a highly vascular, pedunculated mass nearly occluding the airway. En bloc resection using electrocautery and a cryoprobe enabled airway recanalization and diagnosis. Endobronchial ultrasound-guided transbronchial fine needle aspiration ruled out nodal disease. Histopathological examination confirmed a typical carcinoid tumour. Robotic-assisted sleeve resection was subsequently performed, preserving parenchyma and achieving negative margins. Surveillance bronchoscopy showed a well-healed anastomosis with no granulation. This case highlights the importance of bronchoscopic interventions in staging and treatment planning, as well as their role in facilitating lung-sparing surgical strategies.
2025-08-21 | Preoperative Embolization of a Pulmonary Carcinoid Tumor with Unusual Arterial Supply: A Case Report
Background Pulmonary carcinoid tumors are rare neuroendocrine neoplasms that carry a risk of bleeding. We report a case of a tumor with unusual arterial supply from multiple sources including collateral from the renal artery, successfully managed with preoperative embolization followed by surgical resection. Case presentation: A 39-year-old woman with no significant medical history presented with chronic cough. Chest radiography revealed an opacity in the right hilar region. Computed tomography (CT) showed a mass obstructing the right middle lobe bronchus. 18F-fluorodeoxyglucose positron emission tomography (FDG-PET) demonstrated no abnormal uptake in the lesion. However, a Ga-68 DOTATATE somatostatin receptor scan (DOTA-PET) showed intense tracer uptake in the mass. Bronchoscopy identified a reddish endobronchial lesion completely occluding the middle lobe bronchus and causing distal collapse. Biopsy was deferred due to the lesion’s hypervascular appearance. With a presumptive diagnosis of carcinoid tumor, a multidisciplinary team planned for surgical resection. To mitigate intraoperative bleeding, preoperative angiographic evaluation was performed. Digital subtraction angiography (DSA) revealed that the blood supply originated from the right bronchial artery, right internal mammary artery and right renal artery. All identified feeding arteries were selectively catheterized and embolized with coils. The patient underwent a right middle lobectomy. Intraoperative blood loss was minimal, and the tumor was resected completely. Pathology confirmed carcinoid tumor. At two-year follow-up, the patient remains asymptomatic. Conclusions This case highlights a rare arterial supply to a lung tumor and demonstrates that preoperative embolization of multiple feeding vessels can facilitate safe resection of a hypervascular tumor. Awareness of vascular anomalies and multidisciplinary approach were key to successful management.
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Drug Discovery Landscape
6 orphan drug designations for Bronchial neuroendocrine tumor.
6 orphan drug designations for Bronchial neuroendocrine tumor.
Drug | Therapy type | Regulator | Orphan designation | Approval | Sponsor |
|---|---|---|---|---|---|
Humanised IgG1 monoclonal antibody against delta-like ligand 3, octakis(thioether) with N-[6-(2-mercapto-5-pyrimidinyl)-1-oxo-5-hexyn-1-yl]-L-valyl-N6,N6-dipropyl-L-lysyl-N-[[3-[(7S)-7-ethyl-7,8,11,13-tetrahydro-7-hydroxy-8,11-dioxo-10H-1,3-dioxolo[4,5-g]pyrano[3',4':6,7]indolizino[1,2-b]quinolin-14-yl]propoxy]methyl]glycinamide | antibodies | EMA | 2026-05-20 | — | PPD Bulgaria EOOD |
Atigotatug, nivolumab | antibodies | EMA | 2026-03-25 | — | Bristol-Myers Squibb Pharma EEIG |
Humanised IgG1 monoclonal antibody against SEZ6, conjugated to (2S)-2-(2-bromoacetamido)-N-[(2S)-1-({3-[(7S)-7-ethyl-7-hydroxy-8,11-dioxo-7,8,11,13-tetrahydro-2H,10H-[1,3]dioxolo[4,5-g]pyrano[3',4':6,7]indolizino[1,2-b]quinolin-14-yl]bicyclo[1.1.1]pentan-1-yl}amino)-1-oxopropan-2-yl]-3-methylbutanamide | antibodies | EMA | 2026-03-25 | — | AbbVie Deutschland GmbH & Co. KG |
Humanised bispecific monoclonal antibody against programmed cell death 1 ligand 1 and tumor necrosis factor receptor superfamily member 9 | — | EMA | 2026-01-09 | — | Voisin Consulting Life Sciences |
Human IgG1 monoclonal antibody against B7-H3, conjugated to N-((2R,10S)-10-Benzyl-2-cyclopropyl-1-(((1S,9S)-9-ethyl-5-fluoro-9-hydroxy-4-methyl-10,13-dioxo-2,3,9,10,13,15-hexahydro-1H,12H-benzo[de]pyrano[3',4':6,7]indolizino[1,2-b]quinolin-1-yl)amino)-1,6,9,12,15-pentaoxo-3-oxa-5,8,11,14-tetraazahexadecan-16-yl)-6-(2,5-dioxo-2,5-dihydro-1H-pyrrol-1-yl)hexanamide | antibodies | EMA | 2025-10-22 | — | GlaxoSmithKline Trading Services Limited |
IgG-like T-cell engager binding to DLL3 and CD3 | antibodies | EMA | 2024-08-21 | — | Boehringer Ingelheim International GmbH |
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