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Re: FitzyP33 post# 327185

Tuesday, 08/24/2021 3:56:37 PM

Tuesday, August 24, 2021 3:56:37 PM

Post# of 470923
The drug works and it's only a matter of time before that's 100% proven.

https://www.researchsquare.com/article/rs-189177/v1

https://assets.researchsquare.com/files/rs-189177/v1/65385792-095a-4505-90c4-c0b85c76dbd1.pdf?c=1614110325

See below some excerpts of the FACTUAL content:

Quote:
Administration of blarcamesine to Fmr1 KO2 mice for two weeks led to correction of two key neurobehavioral phenotypes and marked improvement of a third one. Moreover, two major neuronal signaling abnormalities in mouse models of FXS, namely increased pAkt and decreased BDNF levels, were restored to WT levels in the hippocampus of Fmr1 KO2 mice. A third signaling marker, pERK was also mildly improved in the same animals

Quote:
Comparisons between Fmr1 KO2 groups demonstrated a significant reduction in total distance traveled (number of squares crossed in 3 min) by the blarcamesine-treated animals with respect to vehicle-treated mice (Student’s t-test p = 0.0006). The relevance of these changes was confirmed by comparing vehicle treated Fmr1 KO2 mice to their WT counterparts, since the former displayed an increase in the above mentioned measure of hyperactivity (p < 0.001). When all 4 mouse groups were contrasted, chronic treatment with blarcamesine significantly reduced the behavior in Fmr1 KO2 mice to levels indistinguishable from those observed in vehicle-treated WT mice (Fig. 1a).

Quote:
In conclusion, the present findings confirm the dose-dependent receptor occupancy of the S1R with blarcamesine and, combined with the therapeutic response observed at low doses in the tested preclinical model, emphasize the viability of S1R as a therapeutic target in FXS and the clinical potential of blarcamesine in FXS and other neurological disorders. Indeed, pre-clinical studies in a mouse model of Rett syndrome showed similar positive effects on multiple clinically relevant neurobehavioral phenotypes 20. Furthermore, clinical efficacy was demonstrated in a placebo-controlled Phase 2 study in Rett syndrome (NCT03758924) and previously in a smaller PK cohort of patients with this neurodevelopmental disorder 36, as well as significant cognitive improvements in a Phase 2 trial in Parkinson’s disease dementia (NCT03774459). Late-stage clinical studies of blarcamesine in adult and pediatric patients with Rett syndrome (NCT03941444, NCT04304482) and Alzheimer’s disease (NCT02756858, NCT03790709) are currently ongoing. Continued findings from these clinical studies with blarcamesine, combined with the presented data strengthens the rationale for potentially a dependable and effective treatment strategy for FXS and other neurological disorders targeting the S1R with blarcamesine.

Good luck and GOD bless,
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