The octapeptide NAP has femtomolar activity and is able to restore some of the anomalies caused by haploinsufficiency of the entire ADNP protein [Bassan et al., 1999; Zamostiano et al., 2001]. In cellular models, it is able to protect the cells from chemical, electrical or stress induced damage [reviewed in Gozes et al., 2005]. The presumed mode of action is NAPs ability to bind to tubulin, facilitating microtubule assembly and increasing cellular survival. Davunetide, the drug name for NAP, is a candidate for the treatment of multiple selected neurological disorders [Gozes, 2011]. Intranasal and intravenous formulations of the drug exist and both have been shown to cross the blood-brain barrier [Gozes et al., 2000a; Leker et al., 2002]. The drug has been in Phase II and even in Phase III clinical trials and appears to be well tolerated without significant side effects [Magen and Gozes, 2014]. As stated above, specific cognitive abnormalities were also ameliorated by NAP in an ADNP heterozygous knockout mouse model. While these observations may raise hope for treatment in patients with ADNP mutations, it has to
