UCLA researchers in the Department of Neurology with an international team of scientists have developed compounds for therapeutic use in protein misfolding diseases.
Protein misfolding is the underlying cause of a number of neurodegenerative diseases such as Alzheimer’s disease (AD) and Parkinson’s disease (PD) as well systemic diseases such as Type-2 diabetes and prion diseases. In each of these diseases, a protein adopts an altered conformation composed of β-sheets and is found in large deposits that are toxic to cells. For example, AD patients are known to have large β-sheet rich deposits composed of the protein, amyloid-β. Currently, these diseases have no therapeutics and with a global aging population present a huge financial burden.
UCLA researchers have developed novel compounds based on the β-sheet rich structure adopted by the proteins in the aggregated state. The small molecules bind the β-sheet structure and suppress their formation. They also disassemble pre-formed aggregates robustly. The small molecules have shown to be neuro-protective against amyloid-β induced aggregation and toxicity in cultured cells.
|United States Of America||Issued Patent||8,481,494||07/09/2013||2012-615|
|European Patent Office||Published Application||EP2493859||09/05/2012||2012-615|
Protein misfolding, amyloid, therapeutics, amyloid-ß, Alzheimer’s disease, Parkinson’s disease, Type-2 diabetes