US-based biopharmaceutical firm Neurotrope has started a Phase IIa clinical trial to evaluate Bryostatin, a potent modulator of the enzyme protein kinase C epsilon (PKCe), as a treatment for patients with Alzheimer's disease (AD).
The company said that in preclinical in-vivo models, this effect has been shown to play a major role in slowing or reversing AD and restore cognition, memory and motor skills.
Top-line results from the Phase IIa trial are expected to be reported by the end of the first quarter of 2015.
Neurotrope co-chairmen and co-chief executive officers Charles Ramat and Paul Freiman said: "Advancing the clinical development of bryostatin in patients with AD is a significant milestone for Neurotrope.
"We believe bryostatin may restore synaptic structures and functions damaged by AD, leading to improvements in cognition and memory, thereby improving quality of life in AD patients.
"With 36 million people worldwide estimated to have AD, there is significant commercial potential for a new therapeutic that is effective in delaying the progression of the disease."
A total of 15 patients will be enrolled in the randomised, double-blind, placebo-controlled, single dose Phase IIa trial, out of which ten will be randomised to receive bryostatin by injection while the remaining five will receive a matching placebo control.
The primary objective of the trial will be to evaluate the safety and tolerability of a single dose of bryostatin in the treatment of AD patients.
The trial's secondary objectives include the preliminary evaluation of the efficacy of a single dose of bryostatin in the treatment of patients with AD, its pharmacokinetics and pharmacodynamics and to correlate the changes in PKCe with plasma levels of bryostatin and with improvement in cognitive function.
Bryostatin, a natural product produced by a marine invertebrate organism called Bugula neritina, is isolated from organic matter harvested from the ocean.
Image: In the left normal brain, in the right brain of a person with Alzheimer's disease. Photo courtesy of Garrondo.