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ORPHA:70573

Small cell lung cancer

medium confidenceDisorder

Also known as: SCLC

Publications

287,180

99.9th percentile

Trials

6,629

Interventional, condition-specific

Researchers

1,218

Distinct authors in sample

Gene link

—

Readiness

5/6

Stages with a signal

Clinical definition (Orphanet)

Small cell lung cancer (SCLC) is a highly aggressive malignant neoplasm, accounting for 10-15% of lung cancer cases, characterized by rapid growth, and early metastasis. SCLC usually manifests as a large hilar mass with bulky mediastinal lymphadenopathy presenting clinically with chest pain, persistent cough, dyspnea, wheezing, hoarseness, hemoptysis, loss of appetite, weight loss, and neurological and endocrine paraneoplastic syndromes. SCLC is primarily reported in elderly people with a history of long-term tobacco exposure.

How rare: 1-5 / 10 000 — about one to five people per ten thousand (still uncommon, but less ultra-rare).

Orphanet entry

Cross-references

Joined from Mondo / Orphanet. MeSH labels may enter searches; UMLS / OMIM / NCIT are stored for reference.

Additional Mondo synonyms (13)

lung oat cell carcinoma · lung small cell carcinoma · lung small cell neuroendocrine carcinoma · oat cell carcinoma of lung · oat cell carcinoma of the lung · oat cell lung carcinoma · small cell cancer of the lung, somatic · small cell carcinoma of lung · small cell carcinoma of the lung · small cell lung cancer · small cell neuroendocrine carcinoma of lung · small cell neuroendocrine carcinoma of the lung · small-cell cancer of lung

Research stages

Trial readiness signals

Where this condition sits on an open-data research pipeline — not how close a treatment is, and not medical advice. Empty stages often mean “not in these databases under this Mondo ID,” not “impossible.”

5/6 stages with a signal

An interventional trial matched this condition name on ClinicalTrials.gov — see trials below.

  1. Gene identifiedNot found

    No GenCC disease–gene assertion in this build

  2. LiteraturePresent

    287,180 matched papers (202,319 in last 10 years) Source

  3. Phenotype characterisedPresent

    1 HPO annotations (e.g. Small cell lung carcinoma) Source

  4. Animal modelPresent

    5 genotype models (Mus musculus) Source

  5. Orphan designationPartial

    2 FDA · 15 EMA designations (none yet with FDA orphan-indication approval) — e.g. Lefitolimod Source

  6. Interventional trialPresent

    6,629 matched on ClinicalTrials.gov (1450 recruiting in sample)

Biology

Genes and phenotypes

Gene–disease validity from GenCC, plus phenotypes and animal models joined from Monarch Initiative via Mondo ID — not a clinical diagnosis aid.

Do we know what causes it?

Not yet — the cause hasn't been pinned down in GenCC.

No strong gene–disease assertion joined for this Orphanet entity.

Phenotypes (Monarch / HPO)

1

Associated phenotypes · MONDO:0008433

  • Small cell lung carcinoma

Animal models (Monarch / Alliance)

5

Model associations linked to this Mondo ID

Monarch fetch 2026-07-29

Therapies

Designations, candidates, and chemicals

FDA OOPD and EMA orphan designations, Open Targets clinical candidates, and CTD chemical associations via MyDisease.info. These never change the interventional-trial headline.

Orphan designation (FDA · EMA)

17

Designations · no FDA orphan-indication approval yet

  • EMA LefitolimodTreatment of small cell lung cancer · 26/03/2021 · PositiveEMA designation
  • EMA lurbinectedin (Zepzelca)Treatment of small cell lung cancer · 26/02/2019 · PositiveEMA designation
  • EMA 7-ethyl-10-hydroxy-camptothecin;irinotecan hydrochloride trihydrateTreatment of small cell lung cancer · 24/05/2024 · PositiveEMA designation
  • EMA 2-(2-Methyl-5-nitro-1H-imidazol-1-yl)ethylsulfamideTreatment of small cell lung cancer · 22/08/2014 · PositiveEMA designation
  • EMA unknownTreatment of small cell lung cancer · 19/02/2024 · PositiveEMA designation
  • EMA N-(bromoacetyl)-3,3-dinitroazetidineTreatment of small cell lung cancer · 17/01/2018 · PositiveEMA designation
  • EMA tarlatamab (Imdylltra)Treatment of small cell lung cancer · 12/01/2024 · PositiveEMA designation
  • EMA rovalpituzumab tesirineTreatment of small cell lung cancer · 30/05/2016 · WithdrawnEMA designation

Sources: FDA OOPD · EMA orphan designations

Open Targets candidates

368

Drugs / clinical candidates · MONDO_0008433

CTD chemicals (MyDisease.info)

33 associated chemicals · 476 pathways. Therapeutic evidence is listed first when present — not a treatment recommendation.

  • 4-demethoxy-3'-deamino-3'-aziridinyl-4'-methylsulfonyldaunorubicin · therapeutic
  • 4'-chlorodiazepam · therapeutic
  • ABT-737 · therapeutic
  • AVI protocol · therapeutic
  • Carboplatin · therapeutic
  • Cisplatin · therapeutic
  • Cyclophosphamide · therapeutic
  • Doxorubicin · therapeutic
  • ECHO protocol · therapeutic
  • Epirubicin · therapeutic
  • Etoposide · therapeutic
  • fosbretabulin · therapeutic

Pathways: Purine metabolism; Lysine degradation; Arginine and proline metabolism; Inositol phosphate metabolism; Metabolic pathways; EGFR tyrosine kinase inhibitor resistance; Endocrine resistance; Platinum drug resistance

MyDisease.info · MONDO:0008433

Literature

Is anyone studying this?

287,180

287,180 papers — among the better-studied rare conditions, though still a fraction of common-disease literature (breast cancer: over 700,000). Median papers in the last 10 years for a rare disease in this dataset (publications denominator n=3967) is 59.

287,180 papers since the earliest indexed year in this search — median last-10-year count for a rare disease in this dataset is 59 (publications denominator n=3967).

202,319 in the last 10 years · medium confidence · 99.9th percentile (publications denominator)

Phrase hits: 286,653 · MeSH hits: 1,233

Open Europe PMC search

Who's working on it?

1,218

Distinct author names in 200 sampled papers — named people below.

Who's working on it?

People publishing on this condition (sampled Europe PMC records). Affiliation is the most recent found in that sample.

  1. 01
    Liu X12 papers · 2026

    Changchun University of Chinese Medicine Jilin Ginseng Academy Changchun China.

    Papers in Europe PMC
  2. 02
    Wang Y8 papers · 2026

    Cancer Center, Beijing Friendship Hospital, Capital Medical University, Beijing, China.

    Papers in Europe PMC
  3. 03
    Wang J7 papers · 2026

    Department of Thoracic Surgery, Peking University People's Hospital, Beijing, 100044, China; Thoracic Oncology Institute, Peking University People's Hospital, Beijing, 100044, China; Research Unit of Intelligence Diagnosis and Treatment in Early Non-small Cell Lung Cancer, Chinese Academy of Medical Sciences, 2021RU002, Peking University People's Hospital, Beijing, 100044, China; Institute of Advanced Clinical Medicine, Peking University, Beijing, 100191, China. Electronic address: jwangmd@pku.edu.cn.

    Papers in Europe PMC
  4. 04
    Zhang J7 papers · 2026

    Radiology Department, Ningbo No.2 Hospital, Ningbo, 315010, China.

    Papers in Europe PMC
  5. 05
    Li M6 papers · 2026

    Department of Radiotherapy, Third Affiliated Hospital of Kunming Medical University (Yunnan Cancer Hospital, Yunnan Cancer Center), Kunming, 650000 China.

    Papers in Europe PMC
  6. 06
    Zhang Y6 papers · 2026

    Takeda Pharmaceuticals America, Inc., Lexington, MA, USA.

    Papers in Europe PMC
  7. 07
    Chen Y5 papers · 2026

    Department of Respiratory and Critical Care Medicine, Affiliated Hospital of Shaoxing University, Shaoxing, China.

    Papers in Europe PMC
  8. 08
    Wang X5 papers · 2026

    Dalian Medical University, Dalian, 116027, China.

    Papers in Europe PMC
  9. 09
    Chen J4 papers · 2026

    Department of Thoracic Surgery and Oncology, The First Affiliated Hospital of Guangzhou Medical University, State Key Laboratory of Respiratory Disease, National Clinical Research Center for Respiratory Disease, Guangzhou Institute of Respiratory Health, Guangzhou, China.

    Papers in Europe PMC
  10. 10
    Chen Z4 papers · 2026

    State Key Laboratory of Respiratory Disease, National Clinical Research Center for Respiratory Disease, Guangzhou Institute of Respiratory Health, The First Affiliated Hospital of Guangzhou Medical University, Guangzhou 510120, China.

    Papers in Europe PMC

Clinical research

Is a treatment being tested?

6,629

interventional trials for this specific condition

6,629 interventional trials matched this specific condition name; 1450 currently recruiting in our sample.

Data as of 11 September 2026 · last trial check 10 August 2026

6,629 interventional trials — more than 77.2% of diseases in the trials denominator have none at all (5501 of 7126; this disease is at the 100th percentile).

medium confidence · 100th percentile (trials denominator)

Recruiting interventional trials

From the matched ClinicalTrials.gov set

6,629 interventional trials matched after quoted-phrase search and title/condition post-filter.

Observational and natural-history studies

1,043 observational studies match this condition. These do not test a treatment and are not counted in the interventional-trial headline, but they are genuine research: natural-history work often defines the endpoints needed for a future rare-disease trial, and families may be able to enroll.

Recruiting or not-yet-recruiting

Open the complete matched search on ClinicalTrials.gov

Other registries (secondary)

Broader net from EU CTIS, ISRCTN, and ICTRP when available — deduped against ClinicalTrials.gov IDs already counted above. Dual-model LLM relevance gates what we keep. These rows are not added to the interventional headline.

raw 127 · after dedupe 127 · already on CT.gov 0 · kept 0 · parent 0 · uncertain 127 · dropped 0 · fetched 2026-07-30

Source notes: ictrp: Error: ICTRP public search unavailable (WHO portal is SPA-only; SOAP needs partnership). Tried: https://apps.who.int/tri

No secondary-registry studies passed dual-model relevance for this condition name (after dedupe).

Uncertain / not reviewed (127)

Where to find support

Condition-specific patient organisations, when Orphanet lists them, are on the disease’s Orphanet page. We also link umbrella groups that support undiagnosed and ultra-rare families.

Orphanet entry for Small cell lung cancer — check Associations / patient organisations on that page.

India — NPRD

Last verified 2026-07-26

This ORPHAcode is not on our curated NPRD list (direct or Mondo-parent match). That does not decide clinical eligibility; families in India should ask a notified Centre of Excellence about current coverage.

Hand-curated for this project. ORPHAcode mappings are best-effort and may be incomplete or imprecise for umbrella categories. Parent (Mondo) matches mean the policy lists a broader category — confirm eligibility with a Centre of Excellence. Financial entitlements summarised from public policy statements and may change. This is not official government guidance.

How we counted this

Europe PMC query (preferred label + any corrected label + Orphanet and Mondo exact synonyms, stoplisted; unioned with resolved MeSH labels when available). UMLS / OMIM / NCIT cross-references are stored on the overview but are not added to the query string.

"Small cell lung cancer" OR "lung oat cell carcinoma" OR "lung small cell carcinoma" OR "lung small cell neuroendocrine carcinoma" OR "oat cell carcinoma of lung" OR "oat cell carcinoma of the lung" OR "oat cell lung carcinoma" OR "small cell cancer of the lung, somatic" OR "small cell cancer of lung, somatic" OR "small cell carcinoma of lung" OR "small cell carcinoma of the lung" OR "small cell neuroendocrine carcinoma of lung" OR "small cell neuroendocrine carcinoma of the lung" OR "small-cell cancer of lung" OR "small-cell cancer of the lung"

Run this search on Europe PMC

MeSH descriptor terms unioned into the query: Small Cell Lung Carcinoma

ClinicalTrials.gov query (quoted phrases + MeSH via query.cond, plus recall-expansion terms when used):

"Small cell lung cancer" OR "lung oat cell carcinoma" OR "lung small cell carcinoma" OR "lung small cell neuroendocrine carcinoma" OR "oat cell carcinoma of lung" OR "oat cell carcinoma of the lung" OR "oat cell lung carcinoma" OR "small cell cancer of the lung, somatic" OR "small cell cancer of lung, somatic" OR "small cell carcinoma of lung" OR "small cell carcinoma of the lung" OR "small cell neuroendocrine carcinoma of lung" OR "small cell neuroendocrine carcinoma of the lung" OR "small-cell cancer of lung" OR "small-cell cancer of the lung" OR "Small Cell Lung Carcinoma"

Interventional trials matched via: both, phrase, mesh (mesh = registered under a MeSH descriptor no name phrase would catch; recall-expansion = gene / selected parent terms used only for trials).

Study-type breakdown: 6629 interventional · 1043 observational · 41 expanded access. Only interventional studies enter the trial headline.

Query health: ok — strategies attempted: phrase, mesh; with hits: phrase, mesh

Run this search on ClinicalTrials.gov

Synonyms dropped by stoplist: SCLC

Confidence reasoning

  • Preferred label is multi-word and distinctive
  • 1 synonym(s) dropped by stoplist (may under-count)
  • No label/synonym collisions with other diseases in this corpus

Ingested 2026-07-27T01:33:39.953Z