Aktuelle Therapiestudien im Bereich der atypischen Parkinson Syndrome

Zusammenfassung

Bei den atypischen Parkinson Syndromen handelt es sich um eine Gruppe neurodegenerativer Erkrankungen, die klinisch durch eine akinetisch-rigide Symptomatik mit zusätzlichen nicht-motorischen Symptomen gekennzeichnet sind. Anhand ihrer Pathologie, dem zugrundeliegenden fehlgefalteten Protein, lassen sich diese in Synucleinopathien (Multisystematrophie und Demenz vom Lewy-Body-Typ) und Tauopathien (progressive supranukleäre Blickparese und Kortikobasale Degeneration) einteilen. Dabei existieren bisher keine kausalen Therapiemöglichkeiten, in den meist rasch progredienten Krankheitsverlauf einzugreifen. Symptomatische Therapien besitzen nur einen kurzfristigen, häufig unbefriedigenden Effekt. In den letzten Jahren konnten durch präklinische Forschung Pathomechanismen in der Krankheitsentstehung charakterisiert werden. Dabei wurden Substanzen entwickelt, die beispielsweise die pathologische Protein-Aggregation oder in die Verbreitung der Pathologie verhindern und im Tiermodell einen Einfluss auf das Fortschreiten der Krankheit zeigen konnten. Dabei wurden erste klinische Studien, welche in die Entstehung der zugrundeliegenden Pathologie eingreifen, durchgeführt; viele befinden sich aktuell in der Rekrutierung oder Planung. In folgendem Artikel stellen wir die aktuellen Entwicklungen im Gebiet der atypischen Parkinson Syndrome vor und präsentieren die aktuellen Studien.

Abstract

Atypical Parkinson syndromes are a heterogeneous group of neurodegenerative diseases which present with parkinsonism and other non-motor symptoms. On the basis of the underlying pathology, namely the abnormal aggregation of the proteins alpha-synuclein or tau, atypical Parkinson syndromes can be divided into synucleinopathies (multiple system atrophy, Lewy body dementia) and tauopathies (progressive supranuclear palsy, corticobasal degeneration). Currently there are no effective treatments to slow down disease progression available. Medications which help to manage the symptoms show only temporary and insufficient efficacy. In recent years, preclinical research identified essential steps in the pathogenesis of the diseases. Treatments which inhibit pathological protein aggregation and its spreading were developed and showed promising results in animal models. First clinical trials of causal treatments targeting the underlying pathomechanism have been finished; several trials are recruiting patients or being planned at the moment. In the following article we present the latest developments regarding the causal therapy of atypical Parkinson syndromes and the current clinical trials.

Schlüsselwörter

Atypische Parkinson Syndrome - PSP - MSA - kausale Therapien

Key words

Atypical Parkinson Syndromes - PSP - MSA - causal therapies

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