Parkinson's Cure Journey

Lysosome & GBA

The lane

Status
Active. Trials are running and the idea is still being tested.
Verdict
Venglustat, BIA 28-6156 and ambroxol in GBA carriers (AMBITIOUS, no cognitive benefit, preprint) failed; the ambroxol Phase 3 (ASPro-PD, reads out ~2029) and PR001 gene therapy continue.
Family
Housekeeping
Landmarks
18 in this lane, 6 more tagged with it. Advanced 3 · Open 11 · Pending 1 · Dead end 3

The verdict and status come from the project's branches.yaml and use research wording.

Landmarks in this lane

18 landmarks drawn in this lane, oldest first. Select a column heading to sort.
Chaperone-mediated autophagy2004DiscoveryStill being studied––19 Oct 20263
ATP13A2 and Kufor-Rakeb syndrome2006DiscoveryStill being studied––19 Oct 20263
GBA mutations: commonest genetic risk factor2009DiscoveryConfirmed and built on––19 Oct 20263
Ambroxol: cough medicine to first Parkinson's trials2009–2025Trial in peopleMixed or early results––22 Oct 20264
GCase and synuclein feed each other2011DiscoveryStill being studied––19 Oct 20264
GCase low in sporadic Parkinson's brains2012–2014DiscoveryStill being studied––19 Oct 20262
TFEB, the lysosome master switch2013DiscoveryStill being studied––19 Oct 20263
TMEM175: a lysosome channel2014–2026DiscoveryStill being studied––19 Oct 20264
GBA mutation severity2015–2016DiscoveryConfirmed and built on––20 Oct 20262
Dopamine oxidation damages the lysosome2017DiscoveryStill being studied––20 Oct 20263
Lysosomal storage disease genes as a group raise risk2017DiscoveryConfirmed and built on––20 Oct 20263
LRRK2 lowers GCase2019DiscoveryStill being studied––20 Oct 20263
GBA gene therapy (PR001)2020–2026Trial in peopleMixed or early results1/2–19 Oct 20263
AMBITIOUS: ambroxol in GBA carriers2022–2026Trial in peopleDid not work26519 Oct 20264
Venglustat (MOVES-PD) in GBA1 carriers2023Trial in peopleDid not work222119 Oct 20262
BIA 28-6156 (ACTIVATE) in GBA1 carriers2023–2026Trial in peopleDid not work227319 Oct 20264
GT-02287 Phase 1b (GCase modulator)2025–2026Trial in peopleMixed or early results12019 Oct 20267
ASPro-PD: ambroxol Phase 32025–2029Trial in peopleResult due Feb 2029333019 Oct 20263

Also tagged with this lane

6 landmarks drawn in another lane but tagged with this one. Select a column heading to sort.
LRRK2 and lysosome damage2018DiscoveryStill being studied––20 Oct 20264
ATP10B loss-of-function raises risk2020–2025DiscoveryStill being studied––20 Oct 20263
PSAP (prosaposin) variants2020–2021DiscoveryStill being studied––20 Oct 20263
CAP-003 intravenous GBA1 gene therapy (Capsida)2025–2026Trial in peopleDid not work2–19 Oct 20263
PreCoDe: prasinezumab in GBA carriers2025–2030Trial in peopleResult due Dec 2030212022 Oct 20265
VGN-R08b GBA1 gene therapy (Vitalgen)2026–2027Trial in peopleMixed or early results21722 Oct 20265

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