Disease · MONDO_0016318

progressive multifocal leukoencephalopathy

6 sourcesdiseases-2026-09-18-0.1.0

Cite this record

ConductScience Library. progressive multifocal leukoencephalopathy. Diseases, record MONDO_0016318. Release diseases-2026-09-18-0.1.0, 18 Sept 2026. https://conductscience.org/library/diseases/MONDO_0016318

On this page

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: progressive multifocal leukoencephalopathy; Open Targets
Open Targets ID: MONDO_0016318; Open Targets
Other names: Leukoencephalopathy, Progressive Multifocal, PML, progressive multifocal leukoencephalitis; Open Targets
Disease Ontology: DOID:643; Disease Ontology
MeSH: D007968; MeSH
NCI Thesaurus: C26815; NCI Thesaurus
Orphanet: ORPHA:217260; Orphanet
Highest stage of its drugs: Phase 2/3, for 1 of its 18 drugs, from ClinicalTrials.gov; Open Targets

Drugs18

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
PEMBROLIZUMAB	CHEMBL3137343	Antibody	Phase 2/3	ClinicalTrials.gov	2	2	2	Drugs and targets
ZIDOVUDINE	CHEMBL129	Small molecule	Phase 2	ClinicalTrials.gov	2	2	2	Drugs and targets
BRINCIDOFOVIR	CHEMBL203321	Small molecule	Phase 2	ClinicalTrials.gov	1	1	1	Drugs and targets
CYTARABINE	CHEMBL803	Small molecule	Phase 2	ClinicalTrials.gov	1	1	1	Drugs and targets
DIDANOSINE	CHEMBL1460	Small molecule	Phase 2	ClinicalTrials.gov	1	1	1	Drugs and targets
EFAVIRENZ	CHEMBL223228	Small molecule	Phase 2	ClinicalTrials.gov	1	1	1	Drugs and targets
EMTRICITABINE	CHEMBL885	Small molecule	Phase 2	ClinicalTrials.gov	1	1	1	Drugs and targets
EMTRICITABINE, (+/-)-	CHEMBL161381	Small molecule	Phase 2	ClinicalTrials.gov	1	1	1	Drugs and targets
ENFUVIRTIDE	CHEMBL525076	Protein	Phase 2	ClinicalTrials.gov	1	1	1	Drugs and targets
LOPINAVIR	CHEMBL729	Small molecule	Phase 2	ClinicalTrials.gov	1	1	1	Drugs and targets

1 to 10 of 18

See all 18 drugs

Targets19

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
CYP3A4	Cytochrome P450 3A	1	Cytochrome P450 3A inhibitor	Phase 2	https://conductscience.org/library/genes/1576	Drugs and targets
CYP3A43	Cytochrome P450 3A	1	Cytochrome P450 3A inhibitor	Phase 2	https://conductscience.org/library/genes/64816	Drugs and targets
CYP3A5	Cytochrome P450 3A	1	Cytochrome P450 3A inhibitor	Phase 2	https://conductscience.org/library/genes/1577	Drugs and targets
CYP3A7	Cytochrome P450 3A	1	Cytochrome P450 3A inhibitor	Phase 2	https://conductscience.org/library/genes/1551	Drugs and targets
IFNAR1	Interferon alpha/beta receptor	1	Interferon alpha/beta receptor agonist	Unknown	https://conductscience.org/library/genes/3454	Drugs and targets
IFNAR2	Interferon alpha/beta receptor	1	Interferon alpha/beta receptor agonist	Unknown	https://conductscience.org/library/genes/3455	Drugs and targets
PDCD1	Programmed cell death protein 1	1	Programmed cell death protein 1 inhibitor	Phase 2/3	https://conductscience.org/library/genes/5133	Drugs and targets
POLA1	DNA polymerase (alpha/delta/epsilon)	1	DNA polymerase (alpha/delta/epsilon) inhibitor	Phase 2	https://conductscience.org/library/genes/5422	Drugs and targets
POLA2	DNA polymerase (alpha/delta/epsilon)	1	DNA polymerase (alpha/delta/epsilon) inhibitor	Phase 2	https://conductscience.org/library/genes/23649	Drugs and targets
POLD1	DNA polymerase (alpha/delta/epsilon)	1	DNA polymerase (alpha/delta/epsilon) inhibitor	Phase 2	https://conductscience.org/library/genes/5424	Drugs and targets

1 to 10 of 19

See all 19 targets

Trials10

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.

Trial	Title	Phase	Participants	Results first posted	Conditions	Drugs	Linked by	Source
NCT00746941	Study to Explore the Effect of Mefloquine in Participants With Progressive Multifocal Leukoencephalopathy (PML)	Phase 1/2	37	2014-07-31	Progressive Multifocal Leukoencephalopathy	MEFLOQUINE	Its record names the disease	ClinicalTrials.gov
NCT00001048	Comparison of Anti HIV Drugs Used Alone or in Combination With Cytosine Arabinoside to Treat Progressive Multifocal Leukoencephalopathy (PML) in HIV-Infected Patients	Phase 2	90		HIV Infections, Leukoencephalopathy, Progressive Multifocal	CYTARABINE, DIDANOSINE, ZALCITABINE, ZIDOVUDINE	Its record names the disease	ClinicalTrials.gov
NCT00002395	Safety and Effectiveness of Topotecan HCl to Treat HIV-Infected Patients With AIDS-Related Progressive Multifocal Leukoencephalopathy (PML)	Phase 2	54		HIV Infections, Leukoencephalopathy, Progressive Multifocal	TOPOTECAN	Its record names the disease	ClinicalTrials.gov
NCT06276504	Pembrolizumab in Progressive Multifocal Leukoencephalopathy (PML) in Immunocompromised Patients Without HIV Infection	Phase 2/3	33		Progressive Multifocal Leukoencephalopathy	PEMBROLIZUMAB	Its record names the disease	ClinicalTrials.gov
NCT00120367	Early Intensification of Antiretroviral Therapy Including Enfuvirtide in HIV-1-Related Progressive Multifocal Leucoencephalopathy (ANRS125)	Phase 2	30		Leukoencephalopathy, Progressive Multifocal, HIV Infections	EFAVIRENZ, EMTRICITABINE, EMTRICITABINE, (+/-)-, ENFUVIRTIDE, LOPINAVIR, RITONAVIR, TENOFOVIR	Its record names the disease	ClinicalTrials.gov
NCT00000945	A Study to Evaluate the Use of Cidofovir (an Experimental Drug) for the Treatment of Progressive Multifocal Leukoencephalopathy (PML) in AIDS Patients	Not applicable	24		HIV Infections, Leukoencephalopathy, Progressive Multifocal	CIDOFOVIR	Its record names the disease	ClinicalTrials.gov
NCT07511049	Intravenous Brincidofovir as an Antiviral for Treatment of Progressive Multifocal Leukoencephalopathy: A Pilot Study	Phase 2	24		Progressive Multifocal Leukoencephalopathy	BRINCIDOFOVIR	Its record names the disease	ClinicalTrials.gov
NCT04781309	NT-I7, a Long-Acting Recombinant IL-7 Molecule, as an Immune Reconstitution Strategy for Lymphopenia in Patients With Progressive Multifocal Leukoencephalopathy	Early phase 1	12		Progressive Multifocal Leukoencephalopathy	EFINEPTAKIN ALFA	Its record names the disease	ClinicalTrials.gov
NCT04091932	Treatment of PD-1 Inhibitor in AIDS-associated PML	Phase 2	10		Progressive Multifocal Leukoencephalopathy, AIDS	PEMBROLIZUMAB	Its record names the disease	ClinicalTrials.gov
NCT00002270	A Pilot Study of the Efficacy of Recombinant Alpha Interferon (IFN-A2b) and Zidovudine (AZT) in the Treatment of Progressive Multifocal Leukoencephalopathy (PML) Complicating HIV-1 Infection	Not applicable			HIV Infections, Leukoencephalopathy, Progressive Multifocal	INTERFERON ALFA-2B, ZIDOVUDINE	Its record names the disease	ClinicalTrials.gov

Comparisons in its trials1

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.

Comparison	Group 1, with the outcome	Group 2, with the outcome	Participants	Trial
Local Standard of Care Plus Mefloquine 250 mg vs Local Standard of Care: Participants Who Died Within 6 Months	5 of 30	2 of 17	47	NCT00746941

Source Trial comparisons

Papers from its trials2

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
23733308	RESULT	1	PubMed
21738597	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_0016318

GET https://conductscience.org/api/diseases/v1/MONDO_0016318/drugs

API reference For AI assistants