Vision Restoration Initiative

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. Twelve 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.

Development Pipeline for Funded Projects

Discovery
(11)
In Vivo Proof of Concept
(0)
IND Enabling
(0)
Clinical Trial Phase 1
(0)
Clinical Trial Phase 2
(0)
Clinical Trial Phase 3
(0)

Axon Regeneration and Vision Restoration in NF-1 OPG

Discovery

Factors that Promote Ganglion Cell Regeneration

Discovery

Generation and Neuroprotective Studies of NF1 Patient-derived RGCs

Discovery

Human Stem Cell-Derived RGCs for Optic Nerve Regeneration

Discovery

Neuroprotection and optic nerve regeneration in a mouse NF1 OPG model

Discovery

Tools

All Tools
Current (2)
Past (0)

Identifying Biomarkers for Visual Restoration Strategies in NF1-OPGs

Quantitative Ophthalmic Biomarkers of NF1 Associated Optic Pathway Gliomas in Phase 3 Clinical Trial

VRI Dream Team

Robert Avery, DO

Children's Hospital of Philadelphia

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

Andrew Huberman, PhD

Stanford University

School of Medicine

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

Robert Avery, DO

Children's Hospital of Philadelphia

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

Andrew Huberman, PhD

Stanford University

School of Medicine

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

Leonard Levin, MD, PhD

McGill University

José-Alain Sahel, MD

University of Pittsburgh

Yuan Zhu, PhD

Children’s National Health System

Dennis Clegg, PhD

UC Santa Barbara

Leonard Levin, MD, PhD

McGill University

José-Alain Sahel, MD

University of Pittsburgh

Yuan Zhu, PhD

Children’s National Health System