Gilbert Family Foundation Commits $11 Million to Combat Vision Loss in NF1 Patients

Source: The Gilbert Family Foundation

The Gilbert Family Foundation today announced the Vision Restoration Initiative, the Foundation’s second research initiative working to accelerate the development of therapies for neurofibromatosis type 1 (NF1).

“We recognized the importance of connecting top researchers from across the country to work collaboratively in finding a cure for NF1,” said Dan Gilbert, Cofounder of the Gilbert Family Foundation. “Under this newest initiative, the foundation is providing funds and, more importantly, a platform to bring together scientific leaders and their multidisciplinary expertise. We are invested in bringing some of the brightest minds together with the kinds of talent and capital that is needed to beat the devastating effects of neurofibromatosis.”

NF1 causes tumors to grow on the optic nerves connecting the eyes to the brain in an estimated 20% of patients, causing many of them to lose their sight. In an effort to reverse this vision loss, the foundation will allocate more than $11 million in research grants to develop innovative therapies that either repair or replace the damaged optic nerves.

The foundation utilized a rigorous and hands-on approach to assemble a “dream team” of ophthalmology, neuroscience, and NF1 experts from across the United States.

“I am excited to join the team that the Gilbert family has put together with the goal of discovering and optimizing therapeutic candidates for vision protection and restoration, and to translate these to patients with NF1 and optic tumors,” said Jeffrey Goldberg, MD, PhD, at the Stanford University School of Medicine.

“I am extremely grateful to the Gilbert family for their investment in this important area of NF1 research,” said David H. Gutmann, MD, PhD, at Washington University in St. Louis. “The ability to work with some of the finest scientists in the world and collectively focus our energies on vision protection represents an unprecedented opportunity to find new treatments for individuals with NF1 optic gliomas.”

The team will focus on developing three types of products:

Neuroprotection/Neuroenhancement Therapy. Therapy designed to prevent further vision loss in NF1 patients with mild vision loss. Researchers will work to develop therapies that protect the optic nerve from further damage, while boosting its vitality and performance.

Exogenous Cell Replacement Therapy: Aimed at returning sight to NF1 patients with significant vision loss. Researchers will work to generate new, healthy cells for transplantation into patients’ eyes. These transplanted cells could then regenerate the optic nerve.

Endogenous Cell Replacement Therapy: Also aimed at returning sight to NF1 patients with significant vision loss. However, instead of generating new cells for transplantation, researchers will work to develop therapies that stimulate the patient’s eyes to generate new cells that would regenerate the optic nerve.

For more information on the Gilbert Family Foundation, visit: gilbertfamilyfoundation.org

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