Gilbert Family Foundation funds clinical study to understand vision loss due to NF1 optic pathway glioma

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DETROIT, September 14, 2021 / PRNewswire / – The Gilbert Family Foundation, a private, non-profit organization created by Jennifer and Dan Gilbert to accelerate treatment of type 1 neurofibromatosis (NF1), today announced funding for a new $ 5.4 million clinical studies. The study, which will be led by researchers from the Children’s Hospital of Philadelphia cream and the Stanford University School of Medicine, aims to understand the mechanisms of vision loss caused by gliomas of the optic pathway associated with NF1, or NF1-OPG.

Almost 20% of children with NF1 will develop OPG, a tumor located along the visual pathway, which can cause permanent vision loss ranging from mild deficit to complete blindness. In April 2019, the Gilbert Family Foundation launched its Vision Restoration Initiative to fund research focused on the development of innovative therapies that repair or replace damaged visual systems caused by NF1-OPG.

“One in five patients with NF1 develop OPG, and many end up suffering from chronic vision loss, a symptom that can have devastating effects on quality of life. vision and blindness associated with NF1-OPG achieve, ”said Jennifer gilbert, co-founder of the Gilbert Family Foundation. “This study is an important milestone for our vision restoration initiative as we strive to unlock life-changing therapies and support future research.”

Using a combination of established and new visual function testing methods, this study will define the functional, structural, metabolic, and patient-reported components of NF1-OPG-induced vision loss. The findings are expected to inform the development of future therapies, validate existing disease models, and help identify new measures to assess the effectiveness of vision restoration treatments, including those developed under the Restoration Initiative. from the vision of the Gilbert Family Foundation and in preclinical animal models and human clinical trials.

The study, officially titled “Identifying Biomarkers for Visual Restoration Strategies in NF1-OPGs,” will be led by Robert Avery, DO, pediatric neuro-ophthalmologist at the Children’s Hospital of Philadelphia and assistant professor of ophthalmology and neurology at the Perelman School of Medicine at University of Pennsylvania, and Jeffrey Goldberg, MD, PhD, professor and chair of ophthalmology at Stanford University Medicine School. As members of the Gilbert Family Foundation Vision Restoration Initiative “Dream Team” The two study leaders not only have extensive experience with the techniques used in the study, but also understand the direct link and impact on the strategies developed as part of the Vision Restoration Initiative.

“Our data on test-retest variance and reliability will be essential to inform endpoint analyzes in early-phase clinical trials for new candidate therapies for neuroprotection and NF1-OPG vision restoration planned in the years to. come, “describes Goldberg. “Our novel and broad approach should identify relevant biomarkers in humans and then lead to animal testing using these biomarkers in preclinical therapeutic studies.”

This study will last around 3 years, with patient recruitment starting at the end of 2021 or early 2022. A forthcoming press release will announce the start of patient recruitment after having established the preliminaries of the project.

“As the Vision Restoration Initiative’s first comprehensive human study, this project will provide a new understanding of vision loss due to NF1-OPG,” said Avery. “We are getting closer to our goal of preventing and even reversing lifelong vision loss from these tumors.”

About the Gilbert Family Foundation
The Gilbert Family Foundation is a private non-profit foundation founded by Jennifer and Dan Gilbert to accelerate recovery from neurofibromatosis type 1 (NF1) and create economic opportunity and equity in the city of Detroit. The Gilbert Family Foundation supports groundbreaking and cutting-edge research in the hope of eradicating NF1, a genetic disease that causes tumors to grow on nerves anywhere in the body. In addition, the Foundation supports the economic stability and mobility of residents of Detroit by investing in wealth creation opportunities and removing systemic barriers such as inequitable land debt among low-income homeowners. For more information on the Gilbert Family Foundation, please visit gilbertfamilyfoundation.org.

SOURCE Gilbert Family Foundation

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http://www.gilbertfamilyfoundation.org


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