Drug Repurposing


Results

DrugID Drug Name Indication Highest status Target Name AD Risk Gene MOA Evidence Example Distance All Evidence
D0V6OZ CN-105 Unclearntraumatic intracerebral hemorrhage Phase 2 APOE APOE Agonist Here using human iPSC-derived cerebral organoid models, we find that APOE deletion increases alpha-synuclein (alphaSyn) accumulation accompanied with synaptic loss, reduction of GBA levels, lipid droplet accumulation and dysregulation of intracellular organelles. -8.28 more
D03SHD Myo-inositol Alzheimer disease Phase 2 APP APP Inhibitor The 5XFAD model over-express human amyloid precursor protein (APP) and presenilin 1 (PS1) harboring five familial AD mutations, which have a high APP expression correlating with a high burden and an accelerated accumulation of the 42 amino acid species of amyloid-beta. -8.09 more
D06KWU DWP-09031 Alzheimer disease Phase 1 APP APP Inhibitor The 5XFAD model over-express human amyloid precursor protein (APP) and presenilin 1 (PS1) harboring five familial AD mutations, which have a high APP expression correlating with a high burden and an accelerated accumulation of the 42 amino acid species of amyloid-beta. -8.09 more
D07VAI ALZ-801 Alzheimer disease Phase 2 APP APP Inhibitor The 5XFAD model over-express human amyloid precursor protein (APP) and presenilin 1 (PS1) harboring five familial AD mutations, which have a high APP expression correlating with a high burden and an accelerated accumulation of the 42 amino acid species of amyloid-beta. -8.09 more
D08QDF Systebryl Amyloidosis Phase 1 APP APP Inhibitor The 5XFAD model over-express human amyloid precursor protein (APP) and presenilin 1 (PS1) harboring five familial AD mutations, which have a high APP expression correlating with a high burden and an accelerated accumulation of the 42 amino acid species of amyloid-beta. -8.09 more
D09DLP Tramiprosate Alzheimer disease Phase 3 APP APP Antagonist The 5XFAD model over-express human amyloid precursor protein (APP) and presenilin 1 (PS1) harboring five familial AD mutations, which have a high APP expression correlating with a high burden and an accelerated accumulation of the 42 amino acid species of amyloid-beta. -8.09 more
D0CH6B ALZT-OP1 Alzheimer disease Phase 3 APP APP Inhibitor The 5XFAD model over-express human amyloid precursor protein (APP) and presenilin 1 (PS1) harboring five familial AD mutations, which have a high APP expression correlating with a high burden and an accelerated accumulation of the 42 amino acid species of amyloid-beta. -8.09 more
D0E8HA T-817MA Alzheimer disease Phase 2 APP APP Inhibitor The 5XFAD model over-express human amyloid precursor protein (APP) and presenilin 1 (PS1) harboring five familial AD mutations, which have a high APP expression correlating with a high burden and an accelerated accumulation of the 42 amino acid species of amyloid-beta. -8.09 more
D0G7LP Pepticlere Alzheimer disease Preclinical APP APP Inhibitor The 5XFAD model over-express human amyloid precursor protein (APP) and presenilin 1 (PS1) harboring five familial AD mutations, which have a high APP expression correlating with a high burden and an accelerated accumulation of the 42 amino acid species of amyloid-beta. -8.09 more
D0W0NU NIC5-15 Alzheimer disease Phase 2 APP APP Inhibitor The 5XFAD model over-express human amyloid precursor protein (APP) and presenilin 1 (PS1) harboring five familial AD mutations, which have a high APP expression correlating with a high burden and an accelerated accumulation of the 42 amino acid species of amyloid-beta. -8.09 more
D0X4ZL AN-1792 Alzheimer disease Phase 2 APP APP Inhibitor The 5XFAD model over-express human amyloid precursor protein (APP) and presenilin 1 (PS1) harboring five familial AD mutations, which have a high APP expression correlating with a high burden and an accelerated accumulation of the 42 amino acid species of amyloid-beta. -8.09 more
D01EJG RG6100 Alzheimer disease Phase 2 MAPT MAPT Inhibitor Since gain-of-function mutations of the MAPT gene cause frontotemporal lobar degeneration (FTLD) characterized by deposition of tau protein, we hypothesize that MAPT duplication affecting gene dosage could also lead to disease. -8.00 more
D0I3LY PMID28766366-Compound-Scheme22Middle Unclear Patented MAPT MAPT Inhibitor Since gain-of-function mutations of the MAPT gene cause frontotemporal lobar degeneration (FTLD) characterized by deposition of tau protein, we hypothesize that MAPT duplication affecting gene dosage could also lead to disease. -8.00 more
D0I8FS BIIB092 Progressive supranuclear palsy | Alzheimer disease Phase 2 MAPT MAPT Inhibitor Since gain-of-function mutations of the MAPT gene cause frontotemporal lobar degeneration (FTLD) characterized by deposition of tau protein, we hypothesize that MAPT duplication affecting gene dosage could also lead to disease. -8.00 more
D0P8YJ PTI-80 Alzheimer disease Phase 1 MAPT MAPT Inhibitor Since gain-of-function mutations of the MAPT gene cause frontotemporal lobar degeneration (FTLD) characterized by deposition of tau protein, we hypothesize that MAPT duplication affecting gene dosage could also lead to disease. -8.00 more
D5FSV8 TRx0237 Alzheimer disease Phase 3 MAPT MAPT Inhibitor Since gain-of-function mutations of the MAPT gene cause frontotemporal lobar degeneration (FTLD) characterized by deposition of tau protein, we hypothesize that MAPT duplication affecting gene dosage could also lead to disease. -8.00 more
D05XDZ LMT-X Acute myeloid leukaemia | Alzheimer disease Phase 3 MAPT/TARDBP TARDBP Inhibitor Additionally, in Drosophila models of ALS and human neuronal cells, scientists found that overexpression of TAR DNA-binding protein 43 (TDP-43) can inhibit HSPA8 transcription, leading to the dysfunction of synaptic vesicle cycling all of which is associated with microautophagy dysfunction. -6.46 more
D05XDZ LMT-X Acute myeloid leukaemia | Alzheimer disease Phase 3 MAPT/TARDBP MAPT Inhibitor Since gain-of-function mutations of the MAPT gene cause frontotemporal lobar degeneration (FTLD) characterized by deposition of tau protein, we hypothesize that MAPT duplication affecting gene dosage could also lead to disease. -6.46 more
D01LOE LY2963016 Type-1/2 diabetes Phase 3 INS INS Agonist Results: CNN-derived image phenotypes are significantly associated with fasting metabolites related to early lipid metabolic changes as well as insulin resistance and with genetic variants mapped to candidate genes enriched for amyloid beta degradation, tau phosphorylation, calcium ion binding-dependent synaptic loss, APP-regulated inflammation response, and insulin resistance. -5.50 more
D01UYA MEDI1341 Parkinson disease Phase 1 SNCA SNCA Inhibitor We show here, in the MPP+ (1-methyl-4-phenylpyridinium ion) cell model of parkinsonism, a time- and dose-dependent increase in the hyperphosphorylation of Tau at pSer396/404 (PHF-1-reactive Tau, p-Tau), concomitant with increased accumulation of alpha-Syn, upon treatment of cells with the neurotoxin. -5.08 more
D0TU9W BIIB054 Parkinson disease Phase 2 SNCA SNCA Inhibitor We show here, in the MPP+ (1-methyl-4-phenylpyridinium ion) cell model of parkinsonism, a time- and dose-dependent increase in the hyperphosphorylation of Tau at pSer396/404 (PHF-1-reactive Tau, p-Tau), concomitant with increased accumulation of alpha-Syn, upon treatment of cells with the neurotoxin. -5.08 more
D4N2JV PRX002 Parkinson disease Phase 2 SNCA SNCA Inhibitor We show here, in the MPP+ (1-methyl-4-phenylpyridinium ion) cell model of parkinsonism, a time- and dose-dependent increase in the hyperphosphorylation of Tau at pSer396/404 (PHF-1-reactive Tau, p-Tau), concomitant with increased accumulation of alpha-Syn, upon treatment of cells with the neurotoxin. -5.08 more
DIW37N NPT200-11 Multiple system atrophy | Parkinson disease Phase 1 SNCA SNCA Inhibitor We show here, in the MPP+ (1-methyl-4-phenylpyridinium ion) cell model of parkinsonism, a time- and dose-dependent increase in the hyperphosphorylation of Tau at pSer396/404 (PHF-1-reactive Tau, p-Tau), concomitant with increased accumulation of alpha-Syn, upon treatment of cells with the neurotoxin. -5.08 more
D06WPM Coprexa Neurological disorder Phase 3 SOD1 SOD1 Inhibitor KEY FINDINGS: The results showed that apoptosis and reactive oxygen species levels significantly increased in Abeta25-35-treated cells, whereas the mRNA and protein levels of Nrf2, SOD and HO-1 decreased. -5.06 more
D0D9NZ Methoxyestradiol Unclear Phase 2 SOD1 SOD1 Inhibitor KEY FINDINGS: The results showed that apoptosis and reactive oxygen species levels significantly increased in Abeta25-35-treated cells, whereas the mRNA and protein levels of Nrf2, SOD and HO-1 decreased. -5.06 more
D0I1CQ BIIB067 Amyotrophic lateral sclerosis Phase 3 SOD1 SOD1 Inhibitor KEY FINDINGS: The results showed that apoptosis and reactive oxygen species levels significantly increased in Abeta25-35-treated cells, whereas the mRNA and protein levels of Nrf2, SOD and HO-1 decreased. -5.06 more
D0T8MA ATN-224 Solid tumour/cancer Phase 2 SOD1 SOD1 Inhibitor KEY FINDINGS: The results showed that apoptosis and reactive oxygen species levels significantly increased in Abeta25-35-treated cells, whereas the mRNA and protein levels of Nrf2, SOD and HO-1 decreased. -5.06 more
D09AYM MABp1 Colorectal cancer | Type-2 diabetes | Acne vulgaris | Atopic dermatitis | Hidradenitis suppurativa | Plaque psoriasis | Pyoderma gangreUnclearsum | Peripheral vascular disease Phase 3 IL1A IL1A Inhibitor Subsequently, we exposed NGC0211 cells directly to AD-related factors like amyloid-beta peptides (Abeta40/42) or activated astrocyte-conditioned medium (Abeta40/42/IL-1beta/TNFalpha-treated) and evaluated biochemical stress markers, cell death indicators, cell proliferation marker (Ki67), and hmNGF release. -4.51 more
D0I9XH Celastrol Motor neurone disease Preclinical TNF/IL1B TNF Suppressor Subsequently, we exposed NGC0211 cells directly to AD-related factors like amyloid-beta peptides (Abeta40/42) or activated astrocyte-conditioned medium (Abeta40/42/IL-1beta/TNFalpha-treated) and evaluated biochemical stress markers, cell death indicators, cell proliferation marker (Ki67), and hmNGF release. -4.25 more
D0I9XH Celastrol Motor neurone disease Preclinical TNF/IL1B IL1B Suppressor In oxidatively-stressed neural cells and rodents' brains, cytosolic oxidatively-damaged mitochondrial DNA accumulated and increased antiviral and inflammatory proteins, including cleaved caspase-1, interleukin-1beta, and interferon-beta. -4.25 more
D0F7DS PMID26161698-Compound-18 Unclear Patented GSK3B/CDK5 GSK3B Inhibitor We further showed that miR-219-5p could mediate a decrease in the protein levels of tau-tubulin kinase 1 (TTBK1) and glycogen synthase kinase 3beta (GSK-3beta) by directly binding to their 3'-untranslated region, thereby promoting the phosphorylation of tau in SH-SY5Y Cells. -4.03 more
D0F7DS PMID26161698-Compound-18 Unclear Patented GSK3B/CDK5 CDK5 Inhibitor Inhibition of miR-132 in primary human neurons and neural cells leads to increased NOS1 levels and triggers excessive production of nitric oxide, followed by aberrant S-nitrosylation (SNO) of specific proteins associated with neurodegeneration and tau pathology, such as cyclin-dependent kinase 5, dynamin-related protein 1, and glyceraldehyde-3-phosphate dehydrogenase. -4.03 more
D00EHX TT-301 Alzheimer disease Phase 1 IL1B IL1B Inhibitor In oxidatively-stressed neural cells and rodents' brains, cytosolic oxidatively-damaged mitochondrial DNA accumulated and increased antiviral and inflammatory proteins, including cleaved caspase-1, interleukin-1beta, and interferon-beta. -4.03 more
D07NSU Glucosamine Osteoarthritis Approved IL1B IL1B Inhibitor In oxidatively-stressed neural cells and rodents' brains, cytosolic oxidatively-damaged mitochondrial DNA accumulated and increased antiviral and inflammatory proteins, including cleaved caspase-1, interleukin-1beta, and interferon-beta. -4.03 more
D0N1FS Diacerein Unclear Phase 4 IL1B IL1B Inhibitor In oxidatively-stressed neural cells and rodents' brains, cytosolic oxidatively-damaged mitochondrial DNA accumulated and increased antiviral and inflammatory proteins, including cleaved caspase-1, interleukin-1beta, and interferon-beta. -4.03 more
D0R4ZT Gallium nitrate Hypercalcaemia Approved IL1B IL1B Inhibitor In oxidatively-stressed neural cells and rodents' brains, cytosolic oxidatively-damaged mitochondrial DNA accumulated and increased antiviral and inflammatory proteins, including cleaved caspase-1, interleukin-1beta, and interferon-beta. -4.03 more
D03DPZ INK128 Breast cancer | Multiple myeloma Phase 2 MTOR MTOR Inhibitor Specifically, we found that autophagy inhibition increases the accumulation of the C-terminal fragments of TDP-43, whereas inhibition of mTOR, a key protein kinase involved in autophagy regulation, reduces the 25-kDa C-terminal fragment accumulation and restores TDP-43 localization. -3.91 more
D03FCF Novolimus Artery steUnclearsis Approved MTOR MTOR Inhibitor Specifically, we found that autophagy inhibition increases the accumulation of the C-terminal fragments of TDP-43, whereas inhibition of mTOR, a key protein kinase involved in autophagy regulation, reduces the 25-kDa C-terminal fragment accumulation and restores TDP-43 localization. -3.91 more
D03LJR Sirolimus Organ transplant rejection | Multiple myeloma | Dutch elm disease Approved MTOR MTOR Inhibitor Specifically, we found that autophagy inhibition increases the accumulation of the C-terminal fragments of TDP-43, whereas inhibition of mTOR, a key protein kinase involved in autophagy regulation, reduces the 25-kDa C-terminal fragment accumulation and restores TDP-43 localization. -3.91 more
D08PSC BI 860585 Solid tumour/cancer Phase 1 MTOR MTOR Inhibitor Specifically, we found that autophagy inhibition increases the accumulation of the C-terminal fragments of TDP-43, whereas inhibition of mTOR, a key protein kinase involved in autophagy regulation, reduces the 25-kDa C-terminal fragment accumulation and restores TDP-43 localization. -3.91 more
D0ES1Q Temsirolimus Renal cell carciUnclearma Approved MTOR MTOR Inhibitor Specifically, we found that autophagy inhibition increases the accumulation of the C-terminal fragments of TDP-43, whereas inhibition of mTOR, a key protein kinase involved in autophagy regulation, reduces the 25-kDa C-terminal fragment accumulation and restores TDP-43 localization. -3.91 more
D0H7XL OSI-027 Renal cell carciUnclearma Phase 2 MTOR MTOR Inhibitor Specifically, we found that autophagy inhibition increases the accumulation of the C-terminal fragments of TDP-43, whereas inhibition of mTOR, a key protein kinase involved in autophagy regulation, reduces the 25-kDa C-terminal fragment accumulation and restores TDP-43 localization. -3.91 more