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progressive multifocal leukoencephalopathy (PML) - Clinical Profile, Genes & Research | Kisho | Kisho

Rare Disease

progressive multifocal leukoencephalopathy

MONDO:0016318

Also known as:PMLprogressive multifocal leukoencephalitisleukoencephalopathy, progressive multifocal

Find Clinical TrialsTrials

Overview

Progressive multifocal leukoencephalopathy (PML) is a neurological disorder that damages the myelin that covers and protects nerves in the white matter of the brain. It is caused by the JC virus (JCV). By age 10, most people have been infected with this virus, but itrarelycauses symptoms unless the immune system becomes severely weakened.The disease occurs, rarely, in organ transplant patients; people undergoing chronic corticosteroid or immunosuppressive therapy; and individuals with cancer, such as Hodgkins disease, lymphoma, and sarcoidosis. PML is most common among individuals with acquired immune deficiency syndrome (AIDS).

TreatmentsNo approved treatments · 3 in pipeline

Clinical Trials6 registered · 4 recruiting

Diagnosis

Biomarker and diagnostic research for progressive multifocal leukoencephalopathy has been reported in the published literature.

Treatment

Compiled from FDA-approved drug records, orphan drug designations, and FDA Adverse Event Reporting System (FAERS) safety data. GeneReviews management guidance is included where available.

No approved treatments are currently available for progressive multifocal leukoencephalopathy. An additional 3 compounds hold orphan drug designation.

Orphan Drug Designations

While no drugs are FDA-approved specifically for progressive multifocal leukoencephalopathy, some of the following designated compounds may be used off-label in clinical practice. Treatment decisions should be made in consultation with a specialist familiar with this condition.

The following drugs have received orphan drug designation from the FDA for progressive multifocal leukoencephalopathy. Orphan designation reflects regulatory interest and does not indicate approval for treatment.

Brand Name	Generic Name	Sponsor

Prognosis

Estimated prevalence: Unknown (Unknown prevalence).

Research Landscape

Sourced from ClinicalTrials.gov and PubMed. Publication types are classified using NLM-curated article metadata; research type breakdowns reflect the nature of published work for this disease.

6 clinical trials registered, 4 recruiting. Interventions under study include drug therapy and other interventions. Pipeline includes 2 PHASE2, 1 PHASE1. Research is sponsored by a mix of industry and academic institutions.

NCT ID	Title	Phase	Sponsor	Status
[NCT05849467](https://clinicaltrials.gov/study/NCT05849467)

Data assembled from 5 of 12 sources · Last updated Sep 18, 2026, 9:37 PM UTC

MONDOHPOClinicalTrials.govFDAPubMedHGNCClinVarClinGenGeneReviewsOrphanetOMIMRUSP

Medical References

External Resources

Orphanet

European rare disease database

Orphanet:217260

GARD

Genetic and Rare Diseases Info Center

GARD:7468

Questions for Your Care Team

→Are any of the 6 active clinical trials a good fit for my situation?
→With 3 compounds in the orphan drug pipeline, which are furthest along in development and which clinical trials should I be aware of?
→Are there specialists or centers of excellence for this condition in my area?
→Is there a patient registry I should consider joining?

← Back to All Diseases

Research Type	Count	% of Total
Patient case studies	115	43%
Disease patterns and progression	39	15%
Research summaries	31	12%
Laboratory research	27	10%
Testing and diagnosis research	24	9%
Clinical study results	14	5%
New treatment approaches	9	3%
Other research	7	3%

Kisho

progressive multifocal leukoencephalopathy

Overview

Diagnosis

Treatment

Orphan Drug Designations

Prognosis

Research Landscape

Medical References

External Resources

Orphanet

GARD

Questions for Your Care Team

Questions for Your Care Team

Clinical Trials

Recent Publications

Latest News & Developments

Progressive multifocal leukoencephalopathy: a rare cause of neurological disorders in a patient with cirrhosis.

Bispecific Antibodies Are Associated With Progressive Multifocal Leukoencephalopathy.

Case report: rare progressive cavitary lesion in progressive multifocal leukoencephalopathy.

progressive multifocal leukoencephalopathy

Overview

Diagnosis

Treatment

Orphan Drug Designations

Prognosis

Research Landscape

Medical References

External Resources

Orphanet

GARD

Related Diseases

Questions for Your Care Team

Questions for Your Care Team

Clinical Trials

Recent Publications

Latest News & Developments

Progressive multifocal leukoencephalopathy: a rare cause of neurological disorders in a patient with cirrhosis.

Bispecific Antibodies Are Associated With Progressive Multifocal Leukoencephalopathy.

Case report: rare progressive cavitary lesion in progressive multifocal leukoencephalopathy.