recombinant nerve growth factor R100E

Recombinant nerve growth factor R100E (rhNGF-R100E) is a genetically engineered variant of human nerve growth factor (NGF) designed to provide neuroprotective and neuroregenerative benefits without the significant pain-inducing side effects typically associated with wild-type NGF. The R100E mutation (arginine to glutamic acid at position 100) was identified in patients with Hereditary Sensory and Autonomic Neuropathy type V (HSAN V), who exhibit a loss of pain perception but maintain normal cognitive and motor functions. This mutation significantly reduces the protein's affinity for the p75 neurotrophin receptor (p75NTR) and its ability to activate nociceptive pathways, while maintaining its potent agonist activity at the Tropomyosin receptor kinase A (TrkA) receptor. Developed by the Fondazione Policlinico Universitario Agostino Gemelli IRCCS, rhNGF-R100E is primarily being investigated for the treatment of optic pathway gliomas (OPG) in pediatric patients, where it aims to prevent or reverse vision loss by supporting the survival of retinal ganglion cells.

Other names

painless nerve growth factorpainless NGFhuman nerve growth factor R100E