DRUGapprovedsmall molecule
riluzole
Mechanism
Glutamate antagonist. Positive in SMND7 mice (2008). Only validated DiffDock hit (+0.082 at 20 poses).
Approved indications
ALS
Related claims (30)
| Type | Predicate | Conf | Source |
|---|---|---|---|
| drug target | Riluzole is a glutamate inhibitor proposed as a drug target for SMA treatment. | 59% | 14623733 |
| drug efficacy | Neuroprotective drugs as olesoxime or riluzole have shown positive effects in preclinical studies but no clear efficacy in clinical trials. | 53% | 28524214 |
| safety | Riluzole was tolerated without adverse effects or laboratory abnormalities in SMA patients. | 53% | 14623733 |
| gene expression | Riluzole treatment decreased the relative protein expression of NF-κB in spinal cord of mice (observed in mouse model). | 53% | 37879951 |
| drug efficacy | Neuroprotective drugs as riluzole have shown positive effects in preclinical studies but no clear efficacy in clinical trials. | 53% | 28524214 |
| drug efficacy | Early therapeutic efforts have focused on testing repurposed drugs such as phenylbutyrate (2), valproic acid (3), riluzole (6), hydroxyurea (7), and albuterol (9), none of which has demonstrated clinical effectiveness. | 53% | W2513799405 |
| drug efficacy | Riluzole has shown some improvement in treating SMA. | 53% | 39514016 |
| gene expression | Riluzole treatment decreased the fluorescence expression of PPIA in spinal cord of mice (observed in mouse model). | 53% | 37879951 |
| drug efficacy | Enhanced CNS biodistribution of riluzole shows promise as efficient therapeutic tool for motor neuron diseases, including amyotrophic lateral sclerosis (demonstrated in vitro and in vivo). | 53% | 40007904 |
| motor function | Riluzole did not produce measurable improvements in motor abilities in SMA patients. | 53% | 14623733 |
| drug efficacy | Riluzole, a glutamate inhibitor, has been shown to slow the rate of decline in patients with amyotrophic lateral sclerosis, another form of motor neuron disease. | 53% | 14623733 |
| gene expression | Riluzole treatment decreased fluorescence expression of PPIA in spinal cord of mice (observed in mouse model). | 53% | 37879951 |
| safety | Multifunctionalized nanoparticles significantly reduce the concentration of Riluzole in non-therapeutic organs responsible of side effects (demonstrated in vivo). | 53% | 40007904 |
| drug efficacy | Edaravone, but not riluzole, was effective in rescuing motor deficits associated with cytoplasmic TDP-43 expression and, to a lesser extent, mutant TDP-43 <sup>G294V</sup> (observed in mouse model). | 51% | 40672281 |
| neuroprotection | Riluzole treatment restored axon outgrowth caused by diminished SMN (observed in vertebrate neurons). | 51% | W2086311610 |
| drug efficacy | Edaravone, but not riluzole, was effective in rescuing motor deficits associated with cytoplasmic TDP-43 expression and, to a lesser extent, mutant TDP-43 G294V (observed in mouse model). | 51% | 40672281 |
| survival | SMA subjects in the riluzole treatment group showed longer survival compared to placebo group (3 of 7 treated patients alive at 30 months to 5 years versus 0 of 3 placebo patients alive at mean age 9 months). | 48% | 14623733 |
| pathway membership | Riluzole can be activated by SK channels. | 47% | W2086311610 |
| drug target | The pharmacological targets of riluzole include SK channels. | 47% | W2086311610 |
| drug efficacy | Riluzole ameliorated the neuromuscular defects in a C. elegans SMA model and SK channel function was required for this beneficial effect (observed in mouse model). | 45% | W2086311610 |
| drug efficacy | Riluzole extends survival by increasing the duration of symptomatic disease in the FALS model without affecting disease onset. | 45% | 9728338 |
| neuroprotection | peripheral nerve axon puncta size is partially restored after treatment with riluzole (observed in mouse model) | 45% | 39130445 |
| neuroprotection | Riluzole ameliorated the phenotype of the dnc-1 KD animals (observed in mouse model). | 45% | 31300701 |
| drug efficacy | Multifunctionalized nanoparticles show suitable characteristics for the release of Riluzole in the central nervous system (demonstrated in vitro and in vivo). | 45% | 40007904 |
| drug efficacy | Riluzole, which is an approved medication for ALS patients, ameliorated the phenotype of the dnc-1 KD animals (observed in C. elegans model). | 45% | 31300701 |
| drug efficacy | It is uncertain whether riluzole has any effect in patients with SMA type I, based on the limited available evidence (clinical trial result). | 45% | 31825542 |
| drug efficacy | The translational potential of the model was verified by predicting riluzole PK in people with spinal muscular atrophy (observed in patient cohort). | 45% | 40958536 |
| drug efficacy | Riluzole prodrugs are useful in treating spinal muscular atrophy. | 44% | AU2022202163B2 |
| survival | In a small randomized controlled study, three of seven children treated with riluzole were still alive at the ages of 30, 48 and 64 months, whereas all three children in the placebo group died; but the difference was not statistically signi... | 41% | 22513939 |
| drug target | Riluzole prodrugs are relevant for spinal muscular atrophy. | 36% | AU2022202163B2 |
Off-Target Findings (246)
T1: 5T3: 224T4: 14untiered: 3
| Target | Role | Tier | Boltz-2 iPTM | Chai-1 iPTM | Verdict |
|---|---|---|---|---|---|
| GAP43 | SMA | T1 | 0.824 | — | Primary SMA hit |
| CHRNGion_channel | SMA | T1 | 0.662 | — | Primary SMA hit |
| NCALD | SMA | T1 | 0.658 | — | Primary SMA hit |
| PFN2 | SMA | T1 | 0.577 | — | Primary SMA hit |
| STMN1 | SMA | T1 | 0.560 | — | Primary SMA hit |
| PTK6kinase | OFF-TARGET | T3 | 0.985 | — | Selectivity flag (off-target substrate) |
| BTKkinase | OFF-TARGET | T3 | 0.984 | — | Selectivity flag (off-target substrate) |
| ADORA1gpcr | OFF-TARGET | T3 | 0.983 | — | Selectivity flag (off-target substrate) |
| HTR1Agpcr | OFF-TARGET | T3 | 0.982 | — | Selectivity flag (off-target substrate) |
| FGRkinase | OFF-TARGET | T3 | 0.981 | — | Selectivity flag (off-target substrate) |
| GRK2kinase | OFF-TARGET | T3 | 0.972 | — | Selectivity flag (off-target substrate) |
| HTR2Cgpcr | OFF-TARGET | T3 | 0.972 | — | Selectivity flag (off-target substrate) |
| FRKkinase | OFF-TARGET | T3 | 0.964 | — | Selectivity flag (off-target substrate) |
| MAP3K3kinase | OFF-TARGET | T3 | 0.964 | — | Selectivity flag (off-target substrate) |
| CHEK1kinase | OFF-TARGET | T3 | 0.962 | — | Selectivity flag (off-target substrate) |
| HTR1Egpcr | OFF-TARGET | T3 | 0.962 | — | Selectivity flag (off-target substrate) |
| DRD2gpcr | OFF-TARGET | T3 | 0.957 | — | Selectivity flag (off-target substrate) |
| PTGFRgpcr | OFF-TARGET | T3 | 0.955 | — | Selectivity flag (off-target substrate) |
| EPHA1kinase | OFF-TARGET | T3 | 0.955 | — | Selectivity flag (off-target substrate) |
| GRK1kinase | OFF-TARGET | T3 | 0.952 | — | Selectivity flag (off-target substrate) |
| ADORA2Bgpcr | OFF-TARGET | T3 | 0.952 | — | Selectivity flag (off-target substrate) |
| DRD1gpcr | OFF-TARGET | T3 | 0.950 | — | Selectivity flag (off-target substrate) |
| LGR5gpcr | OFF-TARGET | T3 | 0.949 | — | Selectivity flag (off-target substrate) |
| TGFBR1kinase | SMA | T3 | 0.948 | — | Selectivity flag (off-target substrate) |
| LTB4R2gpcr | OFF-TARGET | T3 | 0.944 | — | Selectivity flag (off-target substrate) |
| SYKkinase | OFF-TARGET | T3 | 0.942 | — | Selectivity flag (off-target substrate) |
| OPRD1gpcr | OFF-TARGET | T3 | 0.941 | — | Selectivity flag (off-target substrate) |
| TACR3gpcr | OFF-TARGET | T3 | 0.941 | — | Selectivity flag (off-target substrate) |
| MRGPRX1gpcr | OFF-TARGET | T3 | 0.940 | — | Selectivity flag (off-target substrate) |
| PRKCAkinase | OFF-TARGET | T3 | 0.939 | — | Selectivity flag (off-target substrate) |
| KITkinase | OFF-TARGET | T3 | 0.938 | — | Selectivity flag (off-target substrate) |
| EPHA5kinase | OFF-TARGET | T3 | 0.938 | — | Selectivity flag (off-target substrate) |
| S1PR2gpcr | OFF-TARGET | T3 | 0.936 | — | Selectivity flag (off-target substrate) |
| AAK1kinase | OFF-TARGET | T3 | 0.935 | — | Selectivity flag (off-target substrate) |
| KSR1kinase | OFF-TARGET | T3 | 0.933 | — | Selectivity flag (off-target substrate) |
| BDKRB1gpcr | OFF-TARGET | T3 | 0.932 | — | Selectivity flag (off-target substrate) |
| ACVR2Akinase | OFF-TARGET | T3 | 0.929 | — | Selectivity flag (off-target substrate) |
| PRKCEkinase | OFF-TARGET | T3 | 0.923 | — | Selectivity flag (off-target substrate) |
| GIPRgpcr | OFF-TARGET | T3 | 0.921 | — | Selectivity flag (off-target substrate) |
| KISS1Rgpcr | OFF-TARGET | T3 | 0.918 | — | Selectivity flag (off-target substrate) |
| SCTRgpcr | OFF-TARGET | T3 | 0.916 | — | Selectivity flag (off-target substrate) |
| MAP3K13kinase | OFF-TARGET | T3 | 0.916 | — | Selectivity flag (off-target substrate) |
| PDPK1kinase | OFF-TARGET | T3 | 0.916 | — | Selectivity flag (off-target substrate) |
| PAK1kinase | OFF-TARGET | T3 | 0.915 | — | Selectivity flag (off-target substrate) |
| GPR55gpcr | OFF-TARGET | T3 | 0.915 | — | Selectivity flag (off-target substrate) |
| MINK1kinase | OFF-TARGET | T3 | 0.913 | — | Selectivity flag (off-target substrate) |
| CCRL2gpcr | OFF-TARGET | T3 | 0.912 | — | Selectivity flag (off-target substrate) |
| CHRNB2ion_channel | OFF-TARGET | T3 | 0.912 | — | Selectivity flag (off-target substrate) |
| RIPK2kinase | OFF-TARGET | T3 | 0.909 | — | Selectivity flag (off-target substrate) |
| PAK2kinase | OFF-TARGET | T3 | 0.908 | — | Selectivity flag (off-target substrate) |
Showing top 50 of 246 targets.