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Bridging Interventional Development Gaps

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

Traumatic brain injury (TBI) is caused by a blow to the head that leads to loss of normal function of the brain. More than 1.7 million TBIs occur each year in the United States. TBI can cause permanent disability and even death. Multiple sclerosis (MS) is a disease in which the body’s own immune system mistakenly attacks the brain and spinal cord. The symptoms of this condition include trouble seeing, muscle weakness, numbness and tingling, trouble with balance, and problems with thinking and memory. MS affects more than 2.3 million people worldwide. No cure exists for either MS or TBI. In both conditions, damage to brain cells is caused by inflammation, which is the body’s response to injury or infection. The investigators have developed a drug that blocks nerve cell damage from inflammation. For this project, the researchers will continue to study the drug to prepare it for testing in human clinical trials.

Scientific Synopsis

The investigators have synthesized short cyclic erythropoietin peptides that are free of side effects, and the lead compound (JM4) is highly effective in blocking clinical progression and inflammatory neuropathology in pre-clinical animal models of TBI and MS. Both conditions are common human neurological disorders that affect every age group with long‐term consequences, and there is a pressing medical need to bring new, effective therapeutic agents for these diseases to patients. There is no effective therapy for acute TBI.

Support from the BrIDGs program includes guidance and assistance on all aspects of the pre-clinical development process. With the support of NIH, the team is highly optimistic that it can carry this compound through phase I/II studies.

Lead Collaborators

Rutgers School of Dental Medicine, Newark, New Jersey

Peter Dowling, M.D.

Stuart Cook, M.D.

Robert Heary, M.D.

Public Health Impact

The investigators have developed a new type of small molecule intervention that is effective in animal models of TBI and MS. Development of this compound, which has no side effects, for the clinic could lead to major advances in the treatment of these two conditions.

Outcomes

Approved studies are ongoing.

Project Details

  • Synthesis of Good Manufacturing Practice (GMP) and non-GMP material
  • Formulation development
  • Pharmacokinetic/absorption, distribution, metabolism, and excretion (PK/ADME) studies
  • Investigational New Drug (IND)-directed toxicology
Last updated: 07-25-2017
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