SMA Research Platform

Evidence graph for Spinal Muscular Atrophy

Biology-first target discovery
Christian Fischer / Bryzant Labs
14,766Targets
453Trials
84Drugs
7Datasets
6,987Sources
64,683Claims
72,052Evidence
29,649Hypotheses
DRUGphase2small molecule

fasudil

Brand names: HA-1077, Eril

Mechanism

ROCK1/2 inhibitor. Improves NMJ morphology and survival in SMNΔ7 mice (Bowerman 2012). Approved for cerebral vasospasm in Japan. Our Track 1 lead — baseline 100 ns apo-ROCK2 MD published 2026-04-12.

Approved indications

cerebral-vasospasm-japan

Related claims (50)

TypePredicateConfSource
gene expressionfasudil (30 μM) specifically increased glial cell line-derived neurotrophic factor (GDNF) among the secreted proteins at the transcription and secretion levels (in vitro).69%25185536
drug efficacyThe dual ROCK1/2 inhibitor, fasudil (10 mg/kg), reduced infarct size (mouse model).69%41704339
motor functionFasudil (ROCK inhibitor) improves the hindlimb locomotor function in ASCI rats (rat model).65%30094982
drug efficacyROCK inhibitors such as fasudil can be novel, and efficacious treatment for inflammatory neurodegenerative diseases.65%30552558
motor functionSOD1(G93A) mice benefited from oral treatment with Fasudil showing improved motor function (observed in mouse model).65%24311453
drug efficacyThe pan-ROCK inhibitor Fasudil has been clinically approved to treat hypertension, heart failure, glaucoma, spinal cord injury, and stroke (clinical).65%31302146
drug efficacyInhibition of ROCK with Y-27632 and Fasudil reduces the engulfment of neuronal debris (in vitro).65%27178562
motor functionFasudil treatment improved motor and cognitive functions in α-Syn(A53T) mice as determined by Catwalk(TM) gait analysis and novel object recognition (NOR), without apparent side effects (mouse model).65%27101974
gene expressionEnvironmental enrichment combined with fasudil downregulated ROCK, p-LIM domain kinase (LIMK)1, and p-cofilin expression (mouse model).65%33907042
neuroprotectionROCK inhibitors such as fasudil protected against dopaminergic degeneration and other neurodegenerative processes in several experimental models through inhibition of neuroinflammation and activation of survival signaling pathways.65%39915220
drug efficacyInhibition of ROCK by Fasudil may represent a useful therapeutic perspective by inhibiting microglial inflammatory responses in the CNS.65%20882700
drug efficacyThe ET-1-induced actin polymerization response was blunted by the ROCK inhibitor fasudil (rat model).65%29373038
drug efficacyFasudil treatment significantly reduced α-Syn aggregation in vitro in a H4 cell culture model as well as in a cell-free assay.65%27101974
neuroprotectionIn mice, the neuroprotective effect of Fasudil depends on the expression of PPARα induced by ROCK-PPARα-NOX axis-mediated reduction in ROS and associated oxidative damage (in vivo).65%38055403
drug efficacyThe combination therapy of fasudil (3 mg/kg i.p.) and ozagrel (10 mg/kg i.p.), which are noneffective doses, resulted in reduction of cerebral infarction (mouse model).65%21493751
drug efficacyInhibition of ROCK with Fasudil reduces the engulfment of neuronal debris (in vitro).65%27178562
drug efficacyTreatment with Fasudil (5 mg/kg rat weight/day) for 8 weeks significantly improved the motor deficits in the Cylinder and Rotarod tests (rat model).65%32568105
drug efficacyImmunohistochemical analysis revealed a significant reduction of α-Syn pathology in the midbrain of α-Syn(A53T) mice after Fasudil treatment (mouse model).65%27101974
drug efficacyChronic treatment with Fasudil induces anxiety-like behaviors that are likely the consequence of ROCK1 and/or ROCK2 inhibition (mouse model).65%31302146
drug efficacySrc inhibitor (PP2) or ROCK inhibitors (fasudil and H1152) alone could not reduce the occurrence Ator-induced ICH (zebrafish model).65%29730311
drug efficacyDeficiency of Rock1 or treatment with ROCK inhibitor Fasudil protected mice against IgE-mediated challenge in passive cutaneous anaphylaxis model (mouse model).65%26943578
gene expressionIn vitro, LIMK1, collagen I, collagen III, p-MLC, α-SMA, and p-Cofilin expression was significantly attenuated in both the fasudil-treated and untreated LIMK1 KD groups (P < 0.05).64%30861189
drug efficacyIn vitro, apoptosis was enhanced in the LIMK1 KD group treated with fasudil (P < 0.05).64%30861189
gene expressionThe expression of p110-PI3K, p-Akt, WNT1, Fzd1 and β-catenin were increased after fasudil administration (P<0.05) in MPTP-based mice model (mouse model).64%26045742
drug efficacyIn vitro, cell migration was attenuated in the LIMK1 KD group treated with fasudil (P < 0.05).64%30861189
drug efficacyEnvironmental enrichment combined with fasudil treatment (mouse model) can ameliorate memory dysfunction through inhibition of the hippocampal ROCK/cofilin pathway, alteration of the dynamic distribution of F-actin, and inhibition of neuron...60%33433459
drug efficacyFasudil treatment ameliorated memory deficits in APP/PS1 Tg mice, accompanied by the reduction of Aβ deposition and Tau protein phosphorylation, the decrease of BACE and the increase of PSD-95, as well as inhibition of TLRs-NF-κB-MyD88 infl...60%27401064
neuroprotectionThe combination treatment of fasudil and ozagrel exhibits additive effects for neuroprotection after MCAO (mouse model).59%21493751
drug efficacyFasudil significantly reduced the number of newly affected muscles compared to placebo in a dose dependent manner over different thresholds at day 90 (clinical trial result).59%40950480
survivalSOD1(G93A) mice benefited from oral treatment with Fasudil showing prolonged survival (observed in mouse model).59%24311453
drug efficacyInhibition of ROCK with Fasudil attenuates the production of the inflammatory mediator nitric oxide during stimulation with lipopolysaccharide (in vitro).59%27178562
drug efficacyFasudil exhibited multitarget therapeutic effect in APP/PS1 Tg mice (mouse model).59%27401064
drug efficacyThe ROCK inhibitor, fasudil, prevented Aβo-induced actin stabilization, synaptic impairment, and synaptic loss by blocking cofilin phosphorylation (in vitro).59%30341179
neuroprotectionFasudil has neuroprotective effects against ischemic spinal cord injury in rabbits.59%20141964
drug efficacySmall molecule ROCK inhibitors, including belumosudil and fasudil, ameliorate the characteristic mitochondrial defects in cultured FA iPSCs, neurons, cardiomyocytes, and FA patient primary fibroblasts (in vitro).59%40404357
drug targetFasudil hydrochloride is a potent, broadly selective Rho-associated coiled-coil-containing protein kinase (ROCK) inhibitor (in vitro).59%40253509
drug efficacyThe ROCK inhibitor Fasudil has shown symptomatic and disease-modifying effects in Parkinson's disease (PD) models (in vitro) and (in vivo).59%38419648
drug targetFasudil, next to its effects mediated by ROCK-inhibition, directly interacts with α-Syn and attenuates α-Syn pathology.59%27101974
neuroprotectionROCK inhibition by Fasudil, protecting neurons and mobilizating neural stem cells, might represent a useful therapeutic perspective for various neurological disorders characterized by neuron death.59%19447168
drug efficacyFasudil treatment was accompanied by reduced Aβ deposition, Tau protein phosphorylation, and BACE expression, as well as increased PSD-95 expression in hippocampus (mouse model).59%27401064
neuroprotectionFasudil, a Rho kinase inhibitor, has neuroprotection in the 1-methyl-4-phenyl-1,2,3,6-tetrahydropyridine (MPTP)-based Parkinson's disease (PD) (mouse model).59%26045742
drug targetFasudil is a small molecule inhibitor of Rho-associated kinase (ROCK).59%39424560
pathway membershipThe present study highlights a significant role of ROCK/p-Akt/eNOS pathway in the protective effects of fasudil and simvastatin on neurotoxicity and mitochondrial dysfunction induced by 3-NP (in rat model).59%26169112
drug efficacyFasudil, a clinically approved ROCK inhibitor, suppressed rough eye phenotype and mitigated pathogenic tau levels by inducing autophagic pathways in a Drosophila model of tauopathy (mouse model).59%26818518
drug efficacyHydroxyfasudil (HF) inhibited infiltration of CD4<sup>+</sup> T cells and CD68<sup>+</sup> macrophages in the brain in the cuprizone (CPZ) mouse model.59%30660624
drug efficacyInhibition of ROCK with Y-27632 and Fasudil attenuates the production of the inflammatory mediator nitric oxide during stimulation with lipopolysaccharide (in vitro).59%27178562
neuroprotectionFasudil-induced neuroprotection and/or neurogenesis are mediated partially through astrocyte-derived G-CSF (in vitro).59%19447168
motor functionIn the MPTP-mouse model of PD, the administration of Fasudil improved the motor function recovery.59%25908255
gene expressionCombined intervention of MSCs and Fasudil further increased the expression of BDNF and GDNF compared with the delivery of MSCs alone (mouse model).59%27573128
drug efficacyFasudil inhibits gene expression profiles of motor and autophagic cellular cascades and inflammatory/angiogenic responses (in vitro).59%25750613

Off-Target Findings (238)

T1: 6T3: 200T4: 11untiered: 21
TargetRoleTierBoltz-2 iPTMChai-1 iPTMVerdict
GAP43SMAT10.880Primary SMA hit
NCALDSMAT10.820Primary SMA hit
PFN2SMAT10.677Primary SMA hit
STMN1SMAT10.676Primary SMA hit
CHRNGion_channelSMAT10.554Primary SMA hit
CFL1SMAT10.501Primary SMA hit
CHEK1kinaseOFF-TARGETT30.979Selectivity flag (off-target substrate)
GRK4kinaseOFF-TARGETT30.978Selectivity flag (off-target substrate)
DRD4gpcrOFF-TARGETT30.978Selectivity flag (off-target substrate)
PRKG1kinaseOFF-TARGETT30.977Selectivity flag (off-target substrate)
PRKCAkinaseOFF-TARGETT30.976Selectivity flag (off-target substrate)
MAPK7kinaseOFF-TARGETT30.975Selectivity flag (off-target substrate)
AURKCkinaseOFF-TARGETT30.975Selectivity flag (off-target substrate)
GRK1kinaseOFF-TARGETT30.975Selectivity flag (off-target substrate)
CAMK2GkinaseOFF-TARGETT30.971Selectivity flag (off-target substrate)
AMHR2kinaseOFF-TARGETT30.970Selectivity flag (off-target substrate)
AURKBkinaseOFF-TARGETT30.968Selectivity flag (off-target substrate)
GPR42gpcrOFF-TARGETT30.966Selectivity flag (off-target substrate)
MAPK10kinaseOFF-TARGETT30.964Selectivity flag (off-target substrate)
NTRK1kinaseOFF-TARGETT30.964Selectivity flag (off-target substrate)
HRH2gpcrOFF-TARGETT30.963Selectivity flag (off-target substrate)
PAK2kinaseOFF-TARGETT30.958Selectivity flag (off-target substrate)
AAK1kinaseOFF-TARGETT30.957Selectivity flag (off-target substrate)
GPR65gpcrOFF-TARGETT30.951Selectivity flag (off-target substrate)
PRKD3kinaseOFF-TARGETT30.948Selectivity flag (off-target substrate)
DRD1gpcrOFF-TARGETT30.947Selectivity flag (off-target substrate)
ITKkinaseOFF-TARGETT30.946Selectivity flag (off-target substrate)
KITkinaseOFF-TARGETT30.944Selectivity flag (off-target substrate)
FGFR1kinaseOFF-TARGETT30.942Selectivity flag (off-target substrate)
ADORA2BgpcrOFF-TARGETT30.942Selectivity flag (off-target substrate)
ULK3kinaseOFF-TARGETT30.941Selectivity flag (off-target substrate)
PDGFRAkinaseOFF-TARGETT30.940Selectivity flag (off-target substrate)
TGFBR1kinaseSMAT30.939Selectivity flag (off-target substrate)
MAP3K3kinaseOFF-TARGETT30.937Selectivity flag (off-target substrate)
MAST4kinaseOFF-TARGETT30.937Selectivity flag (off-target substrate)
RHOgpcrOFF-TARGETT30.934Selectivity flag (off-target substrate)
STK17AkinaseOFF-TARGETT30.934Selectivity flag (off-target substrate)
PDPK1kinaseOFF-TARGETT30.934Selectivity flag (off-target substrate)
CDC42BPAkinaseOFF-TARGETT30.933Selectivity flag (off-target substrate)
ACVR2AkinaseOFF-TARGETT30.932Selectivity flag (off-target substrate)
ADORA3gpcrOFF-TARGETT30.931Selectivity flag (off-target substrate)
FFAR4gpcrOFF-TARGETT30.926Selectivity flag (off-target substrate)
HCRTR2gpcrOFF-TARGETT30.925Selectivity flag (off-target substrate)
RIPK2kinaseOFF-TARGETT30.923Selectivity flag (off-target substrate)
QRFPRgpcrOFF-TARGETT30.923Selectivity flag (off-target substrate)
CCR3gpcrOFF-TARGETT30.923Selectivity flag (off-target substrate)
SYKkinaseOFF-TARGETT30.922Selectivity flag (off-target substrate)
GALR2gpcrOFF-TARGETT30.919Selectivity flag (off-target substrate)
ADRB3gpcrOFF-TARGETT30.919Selectivity flag (off-target substrate)
HTR5AgpcrOFF-TARGETT30.918Selectivity flag (off-target substrate)

Showing top 50 of 238 targets.

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