Disease · MONDO_0001437

pulmonary alveolar proteinosis

6 sourcesdiseases-2026-09-18-0.1.0

Cite this record

ConductScience Library. pulmonary alveolar proteinosis. Diseases, record MONDO_0001437. Release diseases-2026-09-18-0.1.0, 18 Sept 2026. https://conductscience.org/library/diseases/MONDO_0001437

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Disease

Names and IDs as Open Targets gives them. Its IDs in MeSH, Orphanet, the Disease Ontology and the NCI Thesaurus are those Open Targets states for it.

Name: pulmonary alveolar proteinosis; Open Targets
Open Targets ID: MONDO_0001437; Open Targets
Other names: Alveolar proteinosis, Detection of PAS-positive extracellular material in broncho-alveolar lavage, Intraalveolar phospholipid accumulation; Open Targets
Disease Ontology: DOID:12120; Disease Ontology
MeSH: D011649; MeSH
NCI Thesaurus: C85037; NCI Thesaurus
Highest stage of its drugs: Phase 3, for 1 of its 9 drugs, from ClinicalTrials.gov; Open Targets

Drugs9

The drugs Open Targets' clinical reports study for the disease, the highest stage first, then the most trial reports. The stage is Open Targets', the highest any of its sources reports for the pair, and each row names the sources that report it.

Drug	ChEMBL ID	Type	Open Targets' stage	Stage reported by	Reports	Of them trials	Trials shown here	Source
MOLGRAMOSTIM	CHEMBL2109134	Protein	Phase 3	ClinicalTrials.gov	5	5	1	Drugs and targets
SARGRAMOSTIM	CHEMBL1201670	Protein	Phase 2	ClinicalTrials.gov	6	6	3	Drugs and targets
REGRAMOSTIM	CHEMBL2107881	Protein	Phase 2	ClinicalTrials.gov	4	4	1	Drugs and targets
RITUXIMAB	CHEMBL1201576	Antibody	Phase 2	ClinicalTrials.gov	2	2	2	Drugs and targets
ASCORBIC ACID	CHEMBL196	Small molecule	Phase 1/2	ClinicalTrials.gov	1	1	0	Drugs and targets
FOLIC ACID	CHEMBL1622	Small molecule	Phase 1/2	ClinicalTrials.gov	1	1	1	Drugs and targets
METHIONINE	CHEMBL42336	Small molecule	Phase 1/2	ClinicalTrials.gov	1	1	1	Drugs and targets
PYRIDOXINE	CHEMBL1364	Small molecule	Phase 1/2	ClinicalTrials.gov	1	1	1	Drugs and targets
VITAMIN B12	CHEMBL2375172		Phase 1/2	ClinicalTrials.gov	1	1	1	Drugs and targets

Targets4

The targets ChEMBL's mechanism records give for the disease's drugs, through Open Targets, the most drugs first. A mechanism record says what a drug acts on, not that the target causes the disease.

Target	Protein	Its drugs that act on it	Mechanisms	Highest stage of those drugs	Gene in Genes	Source
CSF2RA	Granulocyte-macrophage colony-stimulating factor receptor	1	Granulocyte-macrophage colony-stimulating factor receptor agonist	Phase 2	https://conductscience.org/library/genes/1438	Drugs and targets
CSF2RB	Granulocyte-macrophage colony-stimulating factor receptor	1	Granulocyte-macrophage colony-stimulating factor receptor agonist	Phase 2	https://conductscience.org/library/genes/1439	Drugs and targets
CSF3R	Granulocyte colony-stimulating factor receptor	1	Granulocyte colony stimulating factor receptor agonist	Phase 2	https://conductscience.org/library/genes/1441	Drugs and targets
MS4A1	B-lymphocyte antigen CD20	1	B-lymphocyte antigen CD20 binding agent	Phase 2	https://conductscience.org/library/genes/931	Drugs and targets

Trials7

Trials an Open Targets clinical report links to the disease, posted results first, then the largest. A trial is shown when the release's checks confirm its link to the disease and Drugs and targets shows its link to one of the disease's drugs. 3 more are not shown, since the checks do not confirm them.

Trial	Title	Phase	Participants	Results first posted	Conditions	Drugs	Linked by	Source
NCT01511068	Inhaled Granulocyte-Macrophage Colony Stimulating Factor (GM-CSF) in Hereditary Pulmonary Alveolar Proteinosis (PAP)	Phase 2	2	2020-09-01	Hereditary Pulmonary Alveolar Proteinosis	REGRAMOSTIM, SARGRAMOSTIM	Its record names the disease	ClinicalTrials.gov
NCT02835742	Pulmonary Alveolar Proteinosis GM-CSF Inhalation Efficacy Trial in Japan	Phase 2	78		Pulmonary Alveolar Proteinosis, Autoimmune	SARGRAMOSTIM	Its record names the disease	ClinicalTrials.gov
NCT00552461	Prospective Trial of Rituximab for Primary Pulmonary Alveolar Proteinosis	Phase 2	10		Pulmonary Alveolar Proteinosis, Primary Disease	RITUXIMAB	Its record names the disease	ClinicalTrials.gov
NCT03006146	Evaluation of a Single Dose of Inhaled Sargramostim in Patients With Autoimmune Pulmonary Alveolar Proteinosis	Phase 1	10		Autoimmune Pulmonary Alveolar Proteinosis	SARGRAMOSTIM	Its record names the disease	ClinicalTrials.gov
NCT01842386	Rituximab for Anti-cytokine Autoantibody-Associated Diseases	Phase 1	7		Pulmonary Alveolar Proteinosis (PAP), Severe Mucocutaneous Candidiasis	RITUXIMAB	Its record names the disease	ClinicalTrials.gov
NCT06431776	Inhaled Molgramostim in Pediatric Participants With Autoimmune Pulmonary Alveolar Proteinosis (aPAP).	Phase 3	5		Autoimmune Pulmonary Alveolar Proteinosis	MOLGRAMOSTIM	Its record names the disease	ClinicalTrials.gov
NCT03887169	Administration of Methionine in Patients With Pulmonary Alveolar Proteinosis by Mutation of the MARS Gene.	Phase 1/2	3		Pulmonary Alveolar Proteinosis, Mutation Ala393Thr of the MARS Gene, mutationSer567Leu of the MARS Gene	FOLIC ACID, METHIONINE, PYRIDOXINE, VITAMIN B12	Its record names the disease	ClinicalTrials.gov

Comparisons in its trials0

Comparisons Trial comparisons calculated in the trials shown here, most participants first. A comparison in one of these trials does not always compare a treatment for the disease, so read the two groups.

None in this release.

Papers from its trials3

The papers the trials shown here list on ClinicalTrials.gov as their results (RESULT) or as drawn from them (DERIVED), results first, newest first.

PMID	Listed as	Trials listing it	Source
34503986	RESULT	1	PubMed
31483963	DERIVED	1	PubMed
21478218	DERIVED	1	PubMed

Data dates

The date of each source this release read.

Open Targets: 2026-09-18; Open Targets
Drug links and their checks, from Drugs and targets: 2026-09-18; Drugs and targets
ClinicalTrials.gov, from Trials: 2026-11-12; Trials
Comparisons, from Trial comparisons: 2026-03-23; Trial comparisons
PubMed, for the checks: 2026-11-06; PubMed
MeSH, for the checks: 2026-11-10; MeSH

Sources 6

ClinicalTrials.gov's posted results, through CTTI's AACTFreely available. Acknowledgement requested
ChEMBL, through Open TargetsCC BY-SA 3.0
ClinicalTrials.govNo license stated
NLM's MeSHNLM's terms
Open TargetsCC0 1.0
PubMedNLM's terms

Each source's terms

ClinicalTrials.gov's posted results, through CTTI's AACTClinicalTrials.gov data via Aggregate Analysis of ClinicalTrials.gov (AACT), CTTI. CTTI makes its materials freely available and requests acknowledgement. https://aact.ctti-clinicaltrials.org/documentation/299
ChEMBL, through Open TargetsCC BY-SA 3.0, for the parts from ChEMBL. Read on 8 Nov 2026 at https://platform-docs.opentargets.org/licence
ClinicalTrials.govnone stated
NLM's MeSHNLM's
Open TargetsCC0 1.0. Open Targets asks that users cite its latest paper: Buniello et al. 2025, Open Targets Platform: facilitating therapeutic hypotheses building in drug discovery, Nucleic Acids Research 53(D1):D1467, PMID 39657122. Read on 8 Nov 2026 at https://platform-docs.opentargets.org/licence
PubMedNLM's terms

ClinicalTrials.gov's data carry an international copyright outside the United States: https://clinicaltrials.gov/about-site/terms-conditions.

For software

Read this disease from software.

The record and each of its lists are JSON, twenty rows a page. No key.

GET https://conductscience.org/api/diseases/v1/MONDO_0001437

GET https://conductscience.org/api/diseases/v1/MONDO_0001437/drugs

API reference For AI assistants