ataxia

Rosolutamide (ALZ-003) is a curcumin analog, an orally active Nrf1 and Nrf2 activator.Rosolutamide modulates oxidative homeostasis, improves mitochondrial function and promotes autophagy, reduces mutant protein aggregation, and decreases intracellular mitochondrial reactive oxygen species (ROS) levels.Rosolutamide has been used in the study of neurodegeneration such as spinal cerebellar ataxia and Huntington disease. neurodegenerative diseases such as Huntington's disease. Regulates oxidative homeostasis.

Triheptanoin (IND 106011) is a fatty acid metabolic modulator. It potentially for the treatment of fatty acid oxidation disorders and GLUT1.

D-Cysteine, the D-isomer of cysteine, is a powerful inhibitor of E. coli growth and is mediated by D-amino acid oxidase to produce H2S, serving as a neuroprotective agent against cerebellar ataxia. Additionally, D-cysteine can inhibit the growth and cariogenicity of a two-species biofilm formed by Streptococcus mutans and Streptococcus hemoglobin.

AZ32 is an orally bioavailable, blood-brain barrier-penetrating ATM inhibitor with an IC50 of <6.2 nM for the ATM enzyme and an IC50 of 0.31 μM for ATM in cells.

VE-821 (ATR Inhibitor IV) is a selective ATP competitive inhibitor of ATR( Ki IC50: 13 26 nM in cell-free assays).

AZD0156 , an effective and specific inhibitors of ATM kinase, is potential antineoplastic activities and chemo- radio-sensitizing.

1. Sinapine, an alkaloid from seeds of the cruciferous species, can be used as an effective natural compound for chemo-resistance. 2. Sinapine has antioxidant and radio-protective activities.

Ceralasertib (AZD6738) is an orally active, selective ATR kinase inhibitor with an IC50 of 1 nM.

ETP-46464 is an effective and specific ATR inhibitor (IC50: 25 nM).

KU-55933 (ATM Kinase Inhibitor) is an ATM inhibitor (IC50=12.9 nM; Ki=2.2 nM) that is selective and ATP-competitive. KU-55933 induces apoptosis and has antitumor effects.

NU6027 is a potent ATR CDK inhibitor, inhibits CDK1 2, ATR and DNA-PK with Ki of 2.5 μM 1.3 μM, 0.4 μM and 2.2 μM, enter cells more readily than the 6-aminopurine-based inhibitors.

Rimtuzalcap (CAD-1883) (CAD-1883), a pioneering selective positive allosteric modulator of small-conductance calcium-activated potassium channels (SK channels), is employed in the study of movement disorders such as spinocerebellar ataxia (SCA) and essential tremor (ET).

AZ20 is an effective and specific inhibitor of ATR kinase (IC50: 5 nM, in a cell-free assay), 8-fold selectivity over mTOR.

CP-466722, an effective and reversible ATM inhibitor, does not inhibit ATR and PI3K or PIKK family members in cells.

AChE-IN-27 is a small molecule used for high-throughput assays.

1(R)-(Trifluoromethyl)oleyl alcohol is an analog of oleic acid .1It inhibits ferroptosis induced by erastin in primary fibroblasts isolated from patients with Friedreich ataxia, a neuro- and cardiodegenerative disorder characterized by loss or impaired activity of frataxin (FXN), when used at concentrations of 5, 10, or 20 μM. 1(R)-(Trifluoromethyl)oleyl alcohol (5 μM) reduces lipid peroxidation induced byFXNsiRNA knockdown in NBT human myoblasts. 1.Cotticelli, M.G., Forestieri, R., Xia, S., et al.Identification of a novel oleic acid analog with protective effects in multiple cellular models of Friedreich ataxiaACS Chem. Neurosci.11(17)2535-2542(2020)

ATR-IN-9 is a highly effective inhibitor of Ataxia-telangiectasia and RAD-3-related protein kinase (ATR). With an IC50 value as low as 10 nM, ATR-IN-9 demonstrates exceptional potency.

Potent and selective ATM kinase inhibitor (IC50 = 0.6 nM in an enzyme inhibition assay). Exhibits > 600-fold selectivity for ATM over other kinases. Inhibits ATM kinase in an in vitro cellular assay (IC50 = 0.33 μM). Potentiates the antitumor effects of the topoisomerase 1 inhibitor irinotecan in tumor bearing, immunocompromised mice. Orally bioavailable. Degorce et al (2016) Discovery of novel 3-quinoline carboxamides as potent, selective and orally bioavailable inhibitors of ataxia telangiectasia mutated (ATM) kinase. J.Med.Chem. 59 6281 PMID:27259031

TDP1 Inhibitor-2 is a potent inhibitor of TDP1 (tyrosyl-DNA phosphodiesterase 1) (IC50: 99 nM) and inhibits SCAN1 (spinal cerebellar ataxia syndrome with axonal neuropathy) (IC50: 3.5 μM).

ATR-IN-12 (Compound 5g) is a potent inhibitor of ataxia-capillaris and Rad3-related (ATR) kinase with an IC50 of 0.007 μM, making it a promising lead compound for further drug discovery studies on ATR kinases.

ATR-IN-11 (Compound Hit01) is a potent inhibitor of ataxia capillaris and Rad3-related (ATR) kinases. As a promising lead compound for developing drugs targeting the ATR kinase, a key regulatory protein in the DNA damage response (DDR) that senses replication stress (RS), ATR-IN-11 has significant potential for cancer research.

Troriluzole is an orally active glutamate modulator with anticancer activity.Troriluzole enhances the expression of excitatory amino acid transporters located in glial cells and is used in the study of spinocerebellar ataxia, Alzheimer's disease, and generalized anxiety disorder (GAD).

ATR-IN-4, a potent inhibitor of ATR (Ataxia telangiectasia mutated gene Rad 3-associated kinase), restricts the growth of DU145 prostate cancer cells and NCI-H460 lung cancer cells, with IC 50 values of 130.9 nM and 41.33 nM, respectively.

Antitumor agent-28 is a compound that specifically targets and inhibits the activity of the ataxia telangiectasia mutated (ATM) kinase. By doing so, it effectively impedes the progression of ATM-mediated diseases and demonstrates significant anti-cancer activity.