S4–04–03: Protecting the brain by nasal peptide delivery: From concept to the clinic

Gozes, Illana; Shiryaev, Natalia; Jouroukhim, Yan; Pinhasov, Albert; Ginzburg, Irith; Rosenmann, Hanna

doi:10.1016/j.jalz.2006.05.286

Cited by 2 publications

(1 citation statement)

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“… 46 In this respect, davunetide was able to compensate for katanin-induced defective axonal branching in the presence of reduced tau expression. In vivo confirmation comes from several animal models in which davunetide reduces the level of phosphorylated tau in models of AD, 47 , 48 tauopathy 33 , 49 and ADNP deficiency. 50 …”

Section: Davunetide: Review Of Pharmacology Mode Of Actionmentioning

confidence: 96%

Critical appraisal of the role of davunetide in the treatment of progressive supranuclear palsy

Gold¹,

Lorenzl²,

Stewart³

et al. 2012

NDT

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Progressive supranuclear palsy (PSP) is a rare neurodegenerative disease characterized by the accumulation of tau protein aggregates in the basal ganglia, brainstem and cerebral cortex leading to rapid disease progression and death. The neurofibrillary tangles that define the neuropathology of PSP are comprised of aggregated 4R tau and show a well-defined distribution. Classically, PSP is diagnosed by symptoms that include progressive gait disturbance, early falls, vertical ophthalmoparesis, akinetic-rigid features, prominent bulbar dysfunction and fronto-subcortical dementia. There are currently no effective therapies for the treatment of this rapidly degenerating and debilitating disease. Davunetide is a novel neuroprotective peptide that is thought to impact neuronal integrity and cell survival through the stabilization of microtubules. Preclinical activity in models of tauopathy has been translated to clinical studies, demonstrating pharmacologic activity that has supported further development. Davunetide’s efficacy and tolerability are being tested in a placebo-controlled study in PSP patients, making it the most advanced drug candidate in this indication. This review examines the disease characteristics of PSP, the rationale for treating PSP with davunetide and assesses some of the challenges of clinical trials in this patient population.

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