Neurodegenerative diseases and nerve injury are among the most debilitating medical conditions. While current genetically-engineered growth factor proteins have showed promise, these protein-based drugs are very difficult to administer. Vertex Pharmaceuticals is developing small molecule, orally administered compounds that promote nerve growth in central and peripheral nerve injury. Data supporting the nerve regeneration activity of VA-10,367, a representative from a bank of compounds designed by Vertex, will be presented at the 27th Annual Society for Neuroscience in New Orleans, Louisiana.
Dr. Joseph Madsen of Children's Hospital in Boston will present results obtained in a spinal cord injury model. Dr. Madsen's study showed that VA-10,367 has the ability to enhance the recovery of function following spinal cord injury. The model showed that two days after injury, rats receiving the compound showed enhanced functional recovery. There is also biological evidence that the compound enhances the recovery after spinal cord injury through effects on neuronal regeneration. These are the first results showing that neurophilin compounds have the potential to regenerate nerves and restore functional activity in a model of spinal cord injury.
Dr. Bruce Gold of the Oregon Health Sciences University will present results obtained in a peripheral nerve injury model. In this model there is a controlled injury to the sciatic nerve that rendered the rat unable to walk. Following oral administration of VA-10,367 the days to onset of foot movement and walking were accelerated compared to the control. Microscopic analysis of nerves from the injured rats demonstrated that VA-10,637 enhanced the regeneration of injured nerves leading to recovery of function.