Ganaxolone

• CAT Number: R019134
• CAS Number: 38398-32-2
• Molecular Formula: C22H36O2
• Molecular Weight: 332.5
• Purity: ≥95%
• Target: GABA Receptor
• Solubility: Soluble in DMSO > 10 mM
• Storage: Store at +4C
• InChI: InChI=1S/C22H36O2/c1-14(23)17-7-8-18-16-6-5-15-13-20(2,24)11-12-21(15,3)19(16)9-10-22(17,18)4/h15-19,24H,5-13H2,1-4H3/t15-,16-,17+,18-,19-,20+,21-,22+/m0/s1
• InChIKey: PGTVWKLGGCQMBR-FLBATMFCSA-N
• SMILES: C[C@@]12[C@](CC[C@]3([H])[C@]2([H])CC[C@@]4(C)[C@@]3([H])CC[C@@H]4C(C)=O)([H])C[C@](C)(O)CC1

Ganaxolone (CAT: R019134), also known as CCD 1042 and C1042, is a central nervous system (CNS)-selective GABAA modulator with well-established actions on specific targets within the brain. It is recognized for its anxiolytic and anticonvulsant properties. Ganaxolone has shown efficacy in protecting against seizures in various animal models, including the pentylenetetrazol, 6 Hz, and amygdala kindling models. Its mechanism of action involves acting as a positive allosteric modulator of the GABAA receptor, enhancing the inhibitory effects of gamma-aminobutyric acid (GABA).

Reference

1:Randomized, double-blind, placebo-controlled phase 2 study of ganaxolone as add-on therapy in adults with uncontrolled partial-onset seizures. Sperling MR, Klein P, Tsai J.Epilepsia. 2017 Apr;58(4):558-564. doi: 10.1111/epi.13705. Epub 2017 Feb 23. PMID: 28230252 2:Effects of the synthetic neurosteroid ganaxolone on seizure activity and behavioral deficits in an Angelman syndrome mouse model. Ciarlone SL, Wang X, Rogawski MA, Weeber EJ.Neuropharmacology. 2017 Apr;116:142-150. doi: 10.1016/j.neuropharm.2016.12.009. Epub 2016 Dec 13. PMID: 27986596 3:Evaluation of the neuroactive steroid ganaxolone on social and repetitive behaviors in the BTBR mouse model of autism. Kazdoba TM, Hagerman RJ, Zolkowska D, Rogawski MA, Crawley JN.Psychopharmacology (Berl). 2016 Jan;233(2):309-23. doi: 10.1007/s00213-015-4115-7. Epub 2015 Nov 3. PMID: 26525567 Free PMC Article4:Effect of nucleus accumbens shell infusions of ganaxolone or gaboxadol on ethanol consumption in mice. Ramaker MJ, Strong-Kaufman MN, Ford MM, Phillips TJ, Finn DA.Psychopharmacology (Berl). 2015 Apr;232(8):1415-26. doi: 10.1007/s00213-014-3777-x. Epub 2014 Oct 25. PMID: 25342197 Free PMC Article5:Ganaxolone improves behavioral deficits in a mouse model of post-traumatic stress disorder. Pinna G, Rasmusson AM.Front Cell Neurosci. 2014 Sep 11;8:256. doi: 10.3389/fncel.2014.00256. eCollection 2014. PMID: 25309317 Free PMC Article6:A potential effect of ganaxolone in an animal model of infantile spasms. Yum MS, Lee M, Ko TS, Velíšek L.Epilepsy Res. 2014 Nov;108(9):1492-500. doi: 10.1016/j.eplepsyres.2014.08.015. Epub 2014 Sep 2. PMID: 25219352 7:GABA transport and neuroinflammation are coupled in multiple sclerosis: regulation of the GABA transporter-2 by ganaxolone. Paul AM, Branton WG, Walsh JG, Polyak MJ, Lu JQ, Baker GB, Power C.Neuroscience. 2014 Jul 25;273:24-38. doi: 10.1016/j.neuroscience.2014.04.037. Epub 2014 May 9. PMID: 24814730 8:Differences in the reinstatement of ethanol seeking with ganaxolone and gaboxadol. Ramaker MJ, Ford MM, Phillips TJ, Finn DA.Neuroscience. 2014 Jul 11;272:180-7. doi: 10.1016/j.neuroscience.2014.04.065. Epub 2014 May 9. PMID: 24814021 Free PMC Article9:Effect of ganaxolone and THIP on operant and limited-access ethanol self-administration. Ramaker MJ, Strong MN, Ford MM, Finn DA.Neuropharmacology. 2012 Sep;63(4):555-64. doi: 10.1016/j.neuropharm.2012.05.007. Epub 2012 May 18. PMID: 22613838 Free PMC Article10:Alteration of ethanol drinking in mice via modulation of the GABA(A) receptor with ganaxolone, finasteride, and gaboxadol. Ramaker MJ, Ford MM, Fretwell AM, Finn DA.Alcohol Clin Exp Res. 2011 Nov;35(11):1994-2007. doi: 10.1111/j.1530-0277.2011.01551.x. Epub 2011 Jun 7. PMID: 21649668 Free PMC Article