難病「進行性核上性麻痺」で脳内タウ蓄積が速く進む人ほど症状も速く進行することを実証 ~同一患者の追跡で初めて解明、タウ蓄積を抑える治療が症状進行の抑制につながる根拠に~

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2026-09-05 量子科学技術研究開発機構

進行性核上性麻痺(PSP)患者を約1年間追跡し、脳内の異常タウ蓄積の進行速度と症状悪化の関係を世界で初めて生体脳で実証した。量子科学技術研究開発機構(QST)などの研究グループは、独自開発のタウPET薬剤florzolotau(18F)を用いて、PSP患者26名を初回と約1年後に撮像。淡蒼球でタウ蓄積が増加するとともに、前頭頭頂葉や小脳白質へ蓄積が広がることを確認した。特に典型的なリチャードソン症候群18名では、淡蒼球におけるタウ蓄積の増加量がPSP Rating Scaleの症状進行と強く相関し、タウの蓄積速度が速い患者ほど症状も速く進行した。これにより、タウPETによる経時的変化がPSPの病態進行を客観的に評価する画像バイオマーカーとなる可能性が示された。タウ蓄積を標的とする治療薬の開発や臨床試験での薬効評価への活用が期待される。

難病「進行性核上性麻痺」で脳内タウ蓄積が速く進む人ほど症状も速く進行することを実証 ~同一患者の追跡で初めて解明、タウ蓄積を抑える治療が症状進行の抑制につながる根拠に~
図1.PSPに特徴的なタウ蓄積領域

<関連情報>

時空間的なタウ蓄積と進行性核上性麻痺への臨床的影響:縦断的フロルゾロタウ(18F)陽電子放出断層撮影 Spatiotemporal Tau Accumulation and its Clinical Impact on Progressive Supranuclear Palsy: Longitudinal Florzolotau (18F) Positron Emission Tomography

Yoshikazu Chishiki MD, MEng, Kenji Tagai MD, PhD, Yuko Kataoka MD, PhD, Ryoji Goto MD, Kenta Osawa MD, Asaka Oyama MSc, Hideki Matsumoto DDS, PhD …
Movement Disorders  Published: 04 September 2026
DOI:https://doi.org/10.1002/mds.70468

Abstract

Background
Florzolotau (18F) positron emission tomography (florzolotau PET) enables high-contrast in vivo detection of four-repeat tau pathology in progressive supranuclear palsy (PSP), but whether longitudinal tau imaging reflects disease progression remains unclear.

Objectives
To explore longitudinal tau accumulation using florzolotau PET and evaluate its association with clinical progression in PSP.

Methods
Twenty-six patients with PSP (18 Richardson’s syndrome [PSP-RS], 8 non-RS) and 10 age- and sex-matched healthy controls (HCs) underwent florzolotau PET, magnetic resonance imaging, and clinical assessments at baseline and after 1 year. Regional standardized uptake value ratios (SUVRs) were extracted across 57 regions of interest. Partial least squares (PLS) multivariate analyses revealed regions with elevated baseline SUVR and longitudinal increase (ΔSUVR) in PSP relative to HCs, alongside regions where ΔSUVR was associated with changes in PSP Rating Scale scores.

Results
At baseline, tau deposition was most prominent in the globus pallidus (GP) and midbrain in patients with PSP. Longitudinal tau increases were observed in the GP, frontoparietal cortex, and cerebellar white matter, and minimal progression was observed in the midbrain. GP tau accumulation exhibited the strongest association with clinical progression in the PLS model among PSP-RS and a univariate correlation (Spearman’s ρ = 0.688, P = 0.002).

Conclusions
This study provides in vivo evidence of the spatiotemporal progression of tau pathology in PSP. In the GP, tau accumulation emerges early and continues to increase with clinical deterioration. These findings support the utility of florzolotau PET for monitoring disease progression and as a biological outcome measure in tau-targeted therapeutic trials. © 2026 The Author(s). Movement Disorders published by Wiley Periodicals LLC on behalf of International Parkinson and Movement Disorder Society.

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