Positive clinical outcomes in two phase 2a studies: ADAS‐COG in Alzheimer’s and MDS‐UPDRS in Parkinson’s patients plus markers of toxic cascade that leads to nerve cell death

Abstract

AbstractBackgroundOverexpression of neurotoxic proteins drives dysregulation of axonal transport, inflammation, nerve cell death, and loss of function. By inhibiting translation of amyloid precursor protein, tau and alpha‐synuclein our drug has been shown in animal models to restore axonal transport, lower inflammation and protect nerve cells and to restore full function in 7 animal models ‐ Down Syndrome [Chen 2020], Alzheimer’s [Teich 2018], stroke [Turcato 2018], traumatic brain injury [Chesselet], Parkinson’s [Kuo 2019], frontotemporal dementia [Davies], and acute glaucoma [Sundstrom]. We showed safety in over 130 healthy and sick people [Maccecchini 2012].MethodsWe are conducting a 2‐part double‐blind, placebo‐controlled phase 2a study in 14 AD and 14 PD patients, collected blood, CSF, conducted safety evaluations and psychometric tests. The patients were treated for 25 days. At the end of the study again safety, psychometric tests, blood and CSF. The plasma and CSF samples are being analyzed for AD and PD specific biomarkers, markers of axonal transport, inflammation, neurodegeneration and synapse health. The second part of the study is enrolling an additional 40 patients with the same study design.ResultsIn AD patients, we are reporting statistically significant improvements in a number of ADAS‐Cog variables: Number of Cancellations, Word Recall, Commands, Spoken Language, Remembering Test Instructions, and ADAS‐Cog 11. In PD patients, we also see statistically significant improvements in speed, coordination and motor function. The first step of the toxic cascade to have been measured is inflammation. Specific markers measured are: YKL40, sTREM2 and GFAP. The statistical significance holds whether the levels are compared within at 0 and at 25 days or between placebo treated and Posiphen treated at 25 days.ConclusionsWe showed that our drug improves cognition in AD and motor function in PD. The PD data is substantiated by the lowering of inflammatory markers. We are in the process of showing that by lowering more than one neurotoxic protein, our drug reverses the toxic cascade that leads to nerve cell death and loss of function. We still have a lot of markers to measure in CSF and plasma and expect to finish by July.