Approximately one in every five NF1 patients develops optic pathway gliomas (OPGs): tumors on nerves that connect the eyes to the brain. Over time, the affected nerves degenerate, and many patients ultimately lose their vision. Today, there are no therapies to reverse this nerve damage and restore vision in NF1 patients with OPGs. The Gilbert Family Foundation’s Vision Restoration Initiative (VRI) is dedicated to solving this unmet clinical need. Thirteen world-class NF1, ophthalmology, and neuroscience experts are collaborating as part of a scientific Dream Team to develop first-in-kind therapies to restore sight to NF1 patients. In particular, the VRI Dream Team is developing 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 aforementioned 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.
VRI Dream Team
Robert Avery, DO
Children's Hospital of Philadelphia
Petr Baranov, MD, PhD
Schepens Eye Research 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
Yuan Pan, PhD
University of Texas
MD Anderson Cancer Center
Thomas Reh, PhD
University of Washington
Michael Young, PhD
Massachusetts Eye and Ear Infirmary
Donald Zack, MD, PhD
The Johns Hopkins University
Yuan Zhu, PhD
Children’s National Health System
Robert Avery, DO
Children's Hospital of Philadelphia
Petr Baranov, MD, PhD
Schepens Eye Research 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
Yuan Pan, PhD
University of Texas
MD Anderson Cancer Center
Thomas Reh, PhD
University of Washington
Michael Young, PhD
Massachusetts Eye and Ear Infirmary
Donald Zack, MD, PhD
The Johns Hopkins University
Yuan Zhu, PhD
Children’s National Health System
VRI Advisory Board
Dennis Clegg, PhD
UC Santa Barbara
Leonard Levin, MD, PhD
McGill University
Roger Packer, MD
Children's National Health System
José-Alain Sahel, MD
University of Pittsburgh
Dennis Clegg, PhD
UC Santa Barbara
Leonard Levin, MD, PhD
McGill University
Roger Packer, MD
Children's National Health System
José-Alain Sahel, MD
University of Pittsburgh