New Compounds For The Treatment Of Diseases Related To Protein Misfolding

Tech ID: 27261 / UC Case 2012-615-0

Summary

UCLA researchers in the Department of Neurology with an international team of scientists have developed compounds for therapeutic use in protein misfolding diseases.

Background

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.

Innovation

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.

Applications

  • Therapeutic in Alzheimer’s disease
  • Therapeutic in Parkinson’s disease
  • Therapeutic for protein misfolding disease

Advantages

  • Applicable to broad range of protein misfolding diseases
  • Prevents formation of new aggregates
  • Breaks pre-formed aggregates

Related Materials

Patent Status

Country Type Number Dated Case
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
 

Contact

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Inventors

  • Bitan, Gal

Other Information

Keywords

Protein misfolding, amyloid, therapeutics, amyloid-ß, Alzheimer’s disease, Parkinson’s disease, Type-2 diabetes

Categorized As