Elsevier

Biological Psychiatry

Volume 79, Issue 9, 1 May 2016, Pages 710-712
Biological Psychiatry

Commentary
N-Methyl-D-Aspartate Receptors, Ketamine, and Rett Syndrome: Something Special on the Road to Treatments?

https://doi.org/10.1016/j.biopsych.2016.03.1045Get rights and content

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Acknowledgments and Disclosures

David Katz’s laboratory is supported by grants from the National Institutes of Health (Grant No. NS087132), Rett Syndrome Research Trust, and AstraZeneca.

David Katz serves on the Scientific Advisory Board of the Rett Syndrome Research Trust, has been a consultant for SAGE Therapeutics during the past year, and is a founding advisor to ArRETT Neurosciences, a company seeking to develop treatments for Rett syndrome. Frank Menniti is founder, former Chief Scientific Officer, and stockholder of

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    A natural history study has provided deep insights into the Rett Syndrome classification and the various clinical phenotypes (Neul et al., 2014; Kaufmann et al., 2016; Katz et al., 2016a; Tarquinio et al., 2017, 2018; Buchanan et al., 2019; Stallworth et al., 2019). Unfortunately, despite major scientific breakthroughs, most clinical trials have failed, and the currently available therapies offer only very limited success (Percy, 1946; Leoncini et al., 2011; Pozzo-Miller et al., 2015; Katz et al., 2016a, 2016b; Nissenkorn et al., 2017; Smith-Hicks et al., 2017; MacKay et al., 2018; Singh and Santosh, 2018; Gomathi et al., 2020). Thus, the devastating breathing abnormalities, like most other symptoms, are largely resistant to pharmacological treatment.

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    Treatment of Mecp2-mutant mice with recombinant IGF1 protein reverses behavioral and synaptic defects (Castro et al., 2014; Tropea et al., 2009). This, combined with promising in vitro data showing beneficial effects of IGF1 in RTT patient iPSc-derived neurons (de Souza et al., 2017; Williams et al., 2014) has led to numerous clinical trials targeting IGF1 (Katz et al., 2016; O'Leary et al., 2018). As one example, recombinant human IGF1 (Mecasermin) treatment correlated with marked improvements in disease severity, social and cognitive measures in RTT patients versus untreated patients (Pini et al., 2016).

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