Short Stabilized EPO-Peptide as Therapeutic Agents for Multiple Sclerosis and Acute Brain Trauma

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Traumatic Brain Injury and Multiple Sclerosis are common human neurological disorders that affect every age group with longterm consequences, and there is a pressing medical need to bring new, effective therapeutic agents for these diseases to patients. Full-length erythropoietin (EPO), which drives formation of red blood cells (erythropoiesis), has been shown to have beneficial effects in various preclinical models of central nervous system (CNS) diseases, including demyelinating disease, acute brain trauma, and stroke. However, use of EPO as a treatment approach for CNS diseases is limited due to the undesirable side effects of excessive erythropoiesis. The collaborators at Rutgers have synthesized short cyclic EPO peptides in an effort to reduce the major hematopoietic side effects. The lead compound (JM4) is highly effective in blocking clinical progression and inflammatory neuropathology in preclinical animal models of TBI and MS. The BrIDGs team has made progress towards the completion of the following studies on JM4: - Synthesis of non-GMP (Good Manufacturing Practice) and GMP material - Formulation development and manufacture of clinical supplies - Pharmacokinetic/absorption, distribution, metabolism, and excretion (PK/ADME) studies - Investigational New Drug (IND)-directed toxicology