Prana Alzheimer's disease data features at world leading
conference
Professor Rudolph Tanzi presents results from testing PBT2 in the
“Alzheimer’s in a Dish Model” at the Alzheimer’s Association International
Conference in Toronto, Canada.
MELBOURNE, July 27th 2016: Professor Rudolph Tanzi, Founding Scientist and Chief
Scientific Advisor for Prana Biotechnology, presented results obtained from testing
PBT2, Prana’s lead candidate for Huntington and Alzheimer’s diseases, at the
Alzheimer’s Association International Conference (AAIC) in Toronto, Canada on July
26, 2016.
The presentation is entitled: “Reconstructing Alzheimer Amyloid and Tau Pathology in
3D Cell Cultures Derived from Human Stem Cells.”
In October, 2014, Professor Tanzi and his colleague Dr. Doo Yeon Kim of
Massachusetts General Hospital/Harvard Medical School reported in the
journal Nature that they successfully recreated Alzheimer’s disease pathology in an
organoid consisting of human stem-cell derived neurons grown in 3D cultures. The
landmark disease model, awarded with the Smithsonian 2015 American Ingenuity
Award, exhibited beta-amyloid plaque deposition, neurofibrillary tangles and neuronal
cell death, all major hallmarks of Alzheimer's disease. The ‘Alzheimer’s-in-a-Dish
Model’ provided the first proof of concept that beta-amyloid is sufficient to trigger
neurofibrillary tangle formation.
Since that time, the inventors have been expanding and further validating the model
for drug screening. At the AAIC 2016 meeting, Professor Tanzi reported testing results
with PBT2 in the ‘Alzheimer’s-in-a-Dish Model’. He found that treatment of the 3D
model cells with PBT2 significantly reduced levels of both phospho-tau (p-tau)
aggregates and Aβ42 fibrils when compared to controls, also visible with immuno-
staining. PBT2 also led to modest improvements in neuronal cell viability in the model.
Professor Tanzi reported that PBT2 testing in the 3D model resulted in dose-related,
statistically significant reductions in p-tau (40 to 56%) and soluble Aβ42 (31 to 51%).
PBT2 testing also resulted in statistically significant reductions in p-tau/total tau and
insoluble Aβ42 ranging from 34% to 37% and 31% to 46%, respectively.
PBT2 comes from a library of over 2,000 compounds which Prana is evaluating
separately for various indications. The 3D Alzheimer’s model adds to the body of
evidence that PBT2 significantly reduces both p-tau and Aß42.
Based on Prana’s prior pre-clinical and clinical testing and these new results, PBT2
appears to carry great potential for targeting both the proteins at the root of
Alzheimer’s; Aß42 and p-tau. p-tau also plays a role in other neurodegenerative
disorders, such as Huntington disease.
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