- 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, which would in turn result in enhanced vision for NF1 OPG patients treated with these therapies. Researchers are also developing strategies to enable the remaining RGCs to further overcome their OPG-inflicted damage by re-growing their axons to the brain. In theory, if a sufficient number of a patient’s remaining RGCs regenerate their axons to his/her brain, he/she would regain a much more significant amount of vision..
- 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 solution that entails generating new, healthy RGCs in the laboratory and transplanting them into the patients’ eyes. After being transplanted into the eye, the new RGCs would then be instructed to regenerate their axons to the brain. 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 aforementioned exogenous RGC replacement approach. However, instead of generating new RGCs in the laboratory, VRI researchers are identifying strategies 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, he/she could, in theory, regain a much more significant amount of vision.
VRI Dream Team
Robert Avery, DO
Children's Hospital of Philadelphia
Petr Baranov, MD, PhD
Schepens Eye Institute
Larry Benowitz, PhD
Boston Children's Hospital
Michael Fisher, MD
Children's Hospital of Philadelphia
Jeffrey Goldberg, MD, PhD
Stanford University School of Medicine
Daniel Goldman, PhD
University of Michigan
David Gutmann, MD, PhD
Washington University in St. Louis
Zhigang He, PhD
Boston Children's Hospital
Roger Packer, MD
Children's National Health System
Thomas Reh, PhD
University of Washington
Michael Young, PhD
Massachusetts Eye and Ear Infirmary
Donald Zack, MD, PhD
The Johns Hopkins University
VRI Advisory Board
Dennis Clegg, PhD
UC Santa Barbara
José-Alain Sahel, MD
University of Pittsburgh School of Medicine
Leonard Levin, MD, PhD
McGill University
Roger Packer, MD
Children's National Health System