This is a new method of treating a variety of tissue degenerative diseases by developing new, small molecule targeted therapeutic agents using existing approved drugs as carriers attached to protease inhibitors. Analogs of existing pharmaceuticals may be explored as a source for their potential as carriers of protease inhibitors of all types for their therapeutic intervention into all forms of degenerative diseases, including muscle and nerve degeneration, hearing loss, inflammatory diseases, cardiovascular disorders, pain, epilepsy, bone resorption and cancer. Calpain is a naturally occurring protease (an enzyme that digests proteins) that occurs in all cells. Under certain conditions its activity is elevated causing damage to the cell. Unregulated calpain activity has been linked to numerous central nervous system and skeletal muscle diseases. Research into the role of calpain in neurodegenerative and neuromuscular diseases has led these researchers to explore the inhibition of this protease as a therapeutic target in a variety of these neurodegenerative diseases. They are working with a small neuron-targeted protease inhibitor molecule (called Gabadur) and they have chemically linked a protease inhibitor to an analog of an existing FDA approved pharmaceutical that functions as a targeting agent to deliver Gabadur directly to the affected tissue. The advantage of this technology is that it uses already existing FDA approved pharmaceuticals as carriers to selective tissues of protease inhibitors to prevent tissue degeneration in a variety of diseases. This method utilizes the known distribution of the drug to direct such inhibitors to the affected tissue. This is a unique method of drug targeting which does not exist in current patent literature. The targeted delivery methodology of the invention advantageously increases the efficacy of a particular treatment, without invasive surgery and/or significant clinical analysis and the associated delays of making the compound commercially available.

File Number: R1557-100 

Other Information: Principal Investigator: Alfred Stracher Additional Investigators: Leo Kesner, Abraham Shulman