Therapeutic

RGFP996; A SPECIFIC HDAC3 INHIBITOR FOR TREATING ACUTE SPINAL CORD INJURY

There are approximately 12,500 new cases of acute160103G SCI (spinal cord injury) each year  in the United States, with that number expected to rapidly increase with the rise in the aging population. The only current treatment for SCI, albeit controversial, is the use of Methylprednisolone within the first 8 hours of injury. This medication is associated with modest positive results but substantial risk of serious side effects that include infection and avascular necrosis.

Dr. Hongyan Zou, Associate Professor in the Departments of Neurosurgery and Neuroscience at the Icahn School of Medicine, has discovered a novel treatment to improve functional recovery from acute SCI. This treatment, RGFP966, is a specific HDAC3 inhibitor that when administered in a mouse contusion SCI model within the first 2 hours of injury, leads to significant functional recovery in hind limb locomotion (BMS: Basso Mouse Scale and TMS; Toyama mouse scale tests).  This treatment also has positive neuroprotective effects on axons at the injury site by a mechanism that includes epigenetic suppression of innate immune response.

Current Development Status

  • Preclinical development

Applications

  • Acute spinal cord injury

Advantages

  • Functional recovery and neuroprotection at site of injury
  • No overt adverse effects or weight loss
  • Other animal studies report an improvement in memory upon RGFP966 treatment

Publications

  • N/A

Patent Status

  • US provisional application 62/322,967, filed on April 15, 2016

Contact

Lambert Edelmann, PhD
Business Development Director
Mount Sinai Innovation Partners | Icahn School of Medicine at Mount Sinai
Phone: 646-605-7313