Acute and chronic neurodegenerative disorders such as Parkinson's disease, stroke, head trauma, spinal cord injury, Huntington's, Alzheimer's and ALS affect millions of people worldwide. Currently there are only few effective treatments and no cures for these diseases. In general, therapies focus on inhibiting pathogenic processes, but promoting beneficial coping and restorative responses could be equally or even more effective.
Proneuron develops therapies aiming at neuronal tissue restoration by employing the body's own machinery of protection and repair via the immune system. The body routinely employs the immune system to protect itself against intruding microorganisms (such as bacteria and viruses) by attacking and eliminating them. In cases of tissue damage, the immune system plays a major role in wound healing and regeneration by eliminating toxic elements, clearance of dead cells and secretion of trophic and growth factors needed for repair and renewal. In the CNS, however, the common belief has been that the immune activity should be limited. In fact, in the degenerating CNS, such as in Parkinson's disease, immune activity is considered to be an element of the events leading to disease progression and thus should be inhibited. Yet, anti-inflammatory agents have failed to show any significant benefit in clinical trials.
Recent research has shown that the inflammatory response in the damaged brain is a complex of both toxic and reparative responses. This new understanding has led to the concept of "protective autoimmunity" formulated by Professor Michal Schwartz at The Weizmann Institute of Science in Israel as a new approach for treating neurodegenerative diseases. It was shown that selective boost of the "wanted" immune response can circumvent the "unwanted" inflammatory-associated degeneration and improves functional recovery in animal models of acute and chronic degenerative diseases. Proneuron, using this approach, has developed PN277 as a lead product that has been found to have significant immune-mediated neurorestorative activities in several preclinical models for acute and chronic neurodegenerative conditions, including spinal cord injury and stroke.
PN277 has the potential to significantly benefit those suffering from Parkinson's disease. The purpose of this project is to validate this immune-based therapy as a disease-modifying clinical approach and to advance the pre-clinical development of PN277 as a therapy for Parkinson's disease.