While amyloid plaques have long been a hallmark of Alzheimer’s Disease (AD), and measuring amyloid deposition has been shown to corelate with disease progress, there is little direct evidence that these plaques are the causative defect in AD. In addition, it is well understood that many genes in addition to amyloid that are normally expressed in the brain have reduced expression, or are silenced in AD. A growing number of researchers are now focused on understanding the roll of these down-regulated genes in AD, but most agree that therapeutic approaches that reset the regulation and expression of these genes will be necessary for an effective treatment of the disease. Fortem has identified and is developing novel small molecule drugs that function as a regulators of gene expression and have shown significant activity in animal models of cognitive decline. Researchers at Cedars Sinai led by our scientific founder, Dr. Keith Black, and our scientific advisor, Dr. Maya Koronyo, have reproducibly demonstrated significant improvement in cognitive performance in a transgenic animal model of cognitive decline as measured by a battery of established cognition tests. Impressively, these improvements are observed within days of treatment with a single dose of the drug and persist for upwards of several months. Repeat dosing extends the period of improved cognitive performance. In addition to demonstrating marked improvement in cognitive performance, we have shown correlation with the up-regulation of genes involved in learning and cognition, neurite formation and protein expression in the animals – all correlating with the observed improvement in cognitive performance testing and neuro-histological changes in these animals. These studies provide an important understanding of the mechanism of action of our lead drug.

Fortem is currently performing IND-enabling pre-clinical studies in preparation for clinical studies.