RECOMBINANT CNTF FOR TREATMENT OF NEUROPATHIES

CILIARY NEUROTROPHIC FACTOR (CNTF) IS A POTENT POLYPEPTIDE HORMONE WHOSE ACTIONS APPEAR TO BE RESTRICTED TO THE NERVOUS SYSTEM. CNTF HAS BEEN OBSERVED TO PROMOTE SURVIVAL, NEUROTRANSMITTER SYNTHESIS AND NEURITE OUTGROWTH IN VARIOUS SYMPATHETIC, SENSORY AND PARASYMPA- THETIC NEURONS AT FEMTOMOLAR TO LOW PICOMOLAR CONCENTRATIONS. THUS CNTF HAS POTENTIAL CLINICAL APPLICATIONS IN THE TREATMENT OF A NUMBER OF DIFFERENT NEUROPATHIES AND NEURODEGENERATIVE DISORDERS IN MAN AS WELL AS IN THE TREATMENT OF TRAUMATIC NERVE INJURY. IT IS THE GOAL OF THIS PROJECT TO ASSESS THIS POTENTIAL BY FIRST PROVIDING A SOURCE OF HUMAN CNTF USING RECOMBINANT DNA METHODS (PHASE I) AND THEN USING THIS SOURCE TO ESTABLISH EFFICACY IN SPECIFIC ACUTE ANIMAL MODELS OF PERIPHERAL NEUROPATHIES (PHASE II) AND, IF INDICATED, IN HUMAN CLINICAL TRIALS (PHASE III).