Dr. Brent K. Young from Stanford University School of Medicine Awarded $90,000 Knights Templar Eye Foundation Grant for Neurofibromatosis Type-1 Research

Dr. Brent K. Young from Stanford University School of Medicine located in Palo Alto, California was awarded a $90,000 grant for his research entitled: Survival and regeneration of retinal ganglion cells in Neurofibromatosis type-1.

These studies aim to develop novel treatments to prevent and reverse vision loss due to vision loss in Neurofibromatosis type 1, or NF1. NF1 occurs in 1 out of every 3000 children born and frequently causes tumors around the nerve connecting the eye to the brain or the optic nerve. These tumorous growths occur before age seven and cause vision loss due to the death of the neurons, retinal ganglion cells, that travel through the optic nerve. Chemotherapy is currently the only treatment available for children experiencing NF1. While chemotherapy can reduce the tumor size, this treatment is often not recommended due to the severe side effects and low success rate (~30%). Therefore, new treatment avenues are desperately needed. Dr. Young’s focus is on developing new techniques which combine gene therapy with drug treatment to prevent retinal ganglion cell death in an animal model of NF1. He will also evaluate their effectiveness in maintaining vision. In addition, since these treatments may not work for those who have already experienced cell death, he will develop a method to replace lost retinal ganglion cells. To accomplish this, he will use cell replacement therapy, where donor cells are transplanted into a degenerated retina. Collectively this work will lay the groundwork for new therapies for children dealing with NF1.