Progressive multifocal leukoencephalopathy: current treatment options and future perspectives

Progressive multifocal leukoencephalopathy (PML) is a rare but debilitating and frequently fatal viral disease of the central nervous system, primarily affecting individuals with chronically and severely suppressed immune systems. The disease was relatively obscure until the outbreak of HIV/AIDS, wh...

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Main Authors: Dejan Pavlovic, Andriani C. Patera, Fredrik Nyberg, Marianne Gerber, Maggie Liu
Format: Article
Language:English
Published: SAGE Publishing 2015-11-01
Series:Therapeutic Advances in Neurological Disorders
Online Access:https://doi.org/10.1177/1756285615602832
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author Dejan Pavlovic
Andriani C. Patera
Fredrik Nyberg
Marianne Gerber
Maggie Liu
author_facet Dejan Pavlovic
Andriani C. Patera
Fredrik Nyberg
Marianne Gerber
Maggie Liu
author_sort Dejan Pavlovic
collection DOAJ
description Progressive multifocal leukoencephalopathy (PML) is a rare but debilitating and frequently fatal viral disease of the central nervous system, primarily affecting individuals with chronically and severely suppressed immune systems. The disease was relatively obscure until the outbreak of HIV/AIDS, when it presented as one of the more frequent opportunistic infections in this immune deficiency syndrome. It attracted additional attention from the medical and scientific community following the discovery of significant PML risk associated with natalizumab, a monoclonal antibody used for treatment of relapsing–remitting multiple sclerosis. This was followed by association of PML with other immunosuppressive or immunomodulating drugs. PML is currently untreatable disease with poor outcomes, so it is a significant concern when developing new immunotherapies. Current prophylaxis and treatment of PML are focused on immune reconstitution, restoration of immune responses to JC virus infection, and eventual suppression of immune reconstitution inflammatory syndrome. This approach was successful in reducing the incidence of PML and improved survival of PML patients with HIV infection. However, the outcome for the majority of PML patients, regardless of their medical history, is still relatively poor. There is a high unmet need for both prophylaxis and treatment of PML. The aim of this review is to discuss potential drug candidates for prophylaxis and treatment of PML with a critical review of previously conducted and completed PML treatment studies as well as to provide perspectives for future therapies.
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spelling doaj.art-87e76bac22ff447696ffa65c8b34381f2022-12-22T01:53:15ZengSAGE PublishingTherapeutic Advances in Neurological Disorders1756-28561756-28642015-11-01810.1177/1756285615602832Progressive multifocal leukoencephalopathy: current treatment options and future perspectivesDejan PavlovicAndriani C. PateraFredrik NybergMarianne GerberMaggie Liu Progressive multifocal leukoencephalopathy (PML) is a rare but debilitating and frequently fatal viral disease of the central nervous system, primarily affecting individuals with chronically and severely suppressed immune systems. The disease was relatively obscure until the outbreak of HIV/AIDS, when it presented as one of the more frequent opportunistic infections in this immune deficiency syndrome. It attracted additional attention from the medical and scientific community following the discovery of significant PML risk associated with natalizumab, a monoclonal antibody used for treatment of relapsing–remitting multiple sclerosis. This was followed by association of PML with other immunosuppressive or immunomodulating drugs. PML is currently untreatable disease with poor outcomes, so it is a significant concern when developing new immunotherapies. Current prophylaxis and treatment of PML are focused on immune reconstitution, restoration of immune responses to JC virus infection, and eventual suppression of immune reconstitution inflammatory syndrome. This approach was successful in reducing the incidence of PML and improved survival of PML patients with HIV infection. However, the outcome for the majority of PML patients, regardless of their medical history, is still relatively poor. There is a high unmet need for both prophylaxis and treatment of PML. The aim of this review is to discuss potential drug candidates for prophylaxis and treatment of PML with a critical review of previously conducted and completed PML treatment studies as well as to provide perspectives for future therapies.https://doi.org/10.1177/1756285615602832
spellingShingle Dejan Pavlovic
Andriani C. Patera
Fredrik Nyberg
Marianne Gerber
Maggie Liu
Progressive multifocal leukoencephalopathy: current treatment options and future perspectives
Therapeutic Advances in Neurological Disorders
title Progressive multifocal leukoencephalopathy: current treatment options and future perspectives
title_full Progressive multifocal leukoencephalopathy: current treatment options and future perspectives
title_fullStr Progressive multifocal leukoencephalopathy: current treatment options and future perspectives
title_full_unstemmed Progressive multifocal leukoencephalopathy: current treatment options and future perspectives
title_short Progressive multifocal leukoencephalopathy: current treatment options and future perspectives
title_sort progressive multifocal leukoencephalopathy current treatment options and future perspectives
url https://doi.org/10.1177/1756285615602832
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