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Our Research Projects

The INADcure Foundation works in collaboration with scientific advisors to identify the most promising INAD research projects for funding. We are currently partnering with top scientists and labs across the country who are working on the next breakthroughs for INAD.

Partners-01

Grantee: Dr. Hugo Bellen
Bellen Lab at Baylor College of Medicine
Location: Houston, Texas

Gene Therapy & Compound Screening

Bellen Lab at Baylor College of Medicine

Dr. Hugo Bellen is an Investigator of the Howard Hughes Medical Institute and Distinguished Service Professor at Baylor College of Medicine (BCM) in the Departments of Molecular and Human Genetics and Neuroscience. Dr. Bellen’s current research focuses on elucidating pathogenic mechanisms of neurodevelopmental and neurodegenerative diseases.

The INADcure Foundation is currently funding his lab to Develop AAV-based gene therapy strategies for Infantile Neuroaxonal Dystrophy (INAD) in mice models. We have also awarded a grant to his lab to screen compounds on fruit flies and on cells of affected children in hopes of identifying potential treatments to stop or slow the progression of the disease.

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Grantee: The New York Stem Cell Foundation
Location: New York, New York

Partners-01

Grantee: Dr. Hugo Bellen
Bellen Lab at Baylor College of Medicine
Location: Houston, Texas

Gene Editing / Cell Therapy Initiative

The New York Stem Cell Foundation in partnership with Baylor College of Medicine

The New York Stem Cell Foundation (NYSCF) and the INADcure Foundation share an interest in using stem cell-based models of human disease to advance cures for rare diseases. The INADcure Foundation recently expanded its partnership with NYSCF to explore the possibility of developing a cellular therapy for INAD patients using gene-corrected neurons. In collaboration with CRISPR pioneer Dr. Feng Zhang of MIT, NYSCF has successfully gene-edited a patient iPSC line to correct their PLA2G6 mutation. The next step is differentiating this line into neurons, to verify that the gene edit successfully reverses disease phenotypes. If a proof of concept is established, it will open doors to pursue a cell replacement therapy.

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Grantee: The New York Stem Cell Foundation
Location: New York, New York

INAD BioBank

The New York Stem Cell Foundation

The INADcure Foundation has created an INAD biobank in partnership with the New York Stem Cell Foundation Research Institute. A biobank is a type of biorepository that stores patient-specific biological samples for use in research. Establishing this biobank allows for research ready patient and unaffected family member cells to be easily accessible and delivered to scientists. To date, we have collected skin and blood samples from 8 patients/famiiies.

Are you a researcher who is interested in our samples?
Click here to see the INADcure Repository

WUSTL_Medicine

Grantee: Paul T. Kotzbauer, MD, PhD
Assistant Professor at Department of Neurology, Washington University School of Medicine
Location: St. Louis, Missouri, United States

Gene Therapy Initiative

Washington University | Saint Louis, Missouri

The goal of the INAD gene therapy research project is to develop treatments for impaired PLA2G6 function, the underlying cause of Infantile Neuroaxonal Dystrophy (INAD). Gene therapy is a revolutionary approach to treating genetic diseases. It involves a one-time delivery of a normal copy of the defective gene which ideally causes the body to naturally produce the missing enzyme, which is what is causing the neurodegeneration in INAD. It is the most promising ‘cure’ for our children. The primary objective of the current project is to determine the safety and efficacy of gene therapy approaches in the INAD mouse model, focusing on understanding key requirements for delivering the PLA2G6 gene to the central and peripheral nervous system.

Grantee: NBIA Research Group
at Oregon Health and Science University
Location: Portland,Oregon
Project status: Currently funding 2nd year of a 5-year study

PLAN Ready Natural History Study

NBIA Research Group | Oregon Health and Science University

The purpose of this study is to help researchers better understand the natural history of PLAN/INAD, meaning how symptoms appear and change over time. By studying individuals with INAD the NBIA Research Group at OHSU hopes to identify disease markers that can be used in future clinical tria ls. A disease marker is any symptom or measurement that happens reliably in a disease, changes predictably with disease progression, and becomes “better” with successful treatment. Natural history studies provide data that serve as the foundation for future drug trial, are required by the FDA, and can hold up a study from starting if natural history data is not sufficient. Supporting this work is essential in order to ensure the rapid advancement through the FDA’s clinical trial application process when potential new drugs or treatments (like gene therapy) are ready for clinical trials.

All of the research projects funded by the INADcure Foundation are reviewed by our Scientific Medical Advisory Board (SMAB). Our team of experts provide scientific review of research proposals to ensure that all research projects funded by the Foundation are scientifically sound and have potential to make an impact within the /NAO research community. In add ition, the SMAB provides insight, scientific direction, and expertise to the Foundation on an ongoing basis.

For more information about any of these initiatives, please contact us at info@INADcure.org.