Over time, the affected nerves degenerate, and many patients ultimately lose their vision. The Vision Restoration Initiative (VRI) brings together thirteen world-class NF1, ophthalmology, and neuroscience experts to develop therapies to address this clinical need. VRI is exploring three types of products:
Neuroprotection/Neuroenhancement Therapy
NF1 OPG patients who have less severe tumors or who are earlier in their disease have remaining parts of the optic nerves and the cells that make up the nerves (RGCs) that could be rescued from further damage. VRI researchers are testing and developing therapies that would not only protect these injured RGCs, but boost their vitality and performance. This would in turn result in enhanced vision for NF1 OPG patients treated with these therapies.
Exogeneous RGC Replacement Therapy
NF1 OPG patients with severe, irreversible vision loss have an insufficient number of viable RGCs to treat in order to recover a meaningful amount of vision. For these patients, VRI researchers are developing a new, healthy RGCs that will be transplanted into a patient’s eyes. A successful therapy would optimize these steps, sufficiently reconnect a patient’s eyes to the brain, and result in significant vision restoration.
Endogenous RGC Replacement Therapy
This type of therapy is similar to the exogenous RGC replacement approach. However, instead of generating new RGCs for transplantation, VRI researchers are identifying ways to stimulate cells that exist in the patient’s eye to transform into new RGCs. Then, if a sufficient number of the new RGCs regenerate their axons to the patient’s brain, they could, in theory, regain a much more significant amount of vision.