The Gilbert Family Foundation in Detroit, a private foundation established by Dan and Jennifer Gilbert to accelerate a cure for neurofibromatosis type one (NF1) and build economic opportunity and equity in the city of Detroit, has announced $23 million in grants to fund research projects as part of the organization’s vision restoration initiative (VRI) and brain tumor initiative (BTI).
Five grants totaling more than $7 million will be invested into BTI, and nearly $16 million will be invested into 13 research projects supporting VRI.
“NF1 is a complex and unpredictable disease that affects 1 in 3,000 people around the world,” says Jennifer Gilbert, co-founder of the Gilbert Family Foundation. “Since our son Nick’s diagnosis, we’ve witnessed first-hand how this disease impacts the lives of NF1 patients and their families.
“By launching the brain tumor initiative and expanding the vision restoration initiative, we hope to accelerate the discovery of therapeutic options for NF1 patients and ultimately eradicate brain and optic pathway tumors for good.”
BTI focuses on identifying and developing therapeutic strategies to effectively treat NF1-associated transformed gliomas. Gliomas are the most common primary central nervous system (CNS) tumors in NF1 patients, usually involving optic pathways in children.
These tumors can spontaneously transform into higher grade tumors, causing greater risk to NF1 patients. BTI seeks to conduct studies to provide critical characterization of NF1 transformed gliomas and identify the most promising treatments for patients.
The BTI researchers include Dr. John Maris of the Children’s Hospital of Philadelphia, Dr. Chelsea Kotch of the Children’s Hospital of Philadelphia, Thomas De Raedt of the Children’s Hospital of Philadelphia, and Dr. David Gutmann of Washington University in St. Louis.
“The primary aim of our studies is to increase our understanding of the biology and behavior of glioma in patients with neurofibromatosis type 1,” says Koch. “Through large-scale retrospective and prospective analyses of clinical and molecular data, we will determine risk factors for tumor development and evaluate the approaches to and outcomes of treatment; this knowledge will ultimately improve the care of patients with NF1 who develop brain tumors.”
The Gilbert Family Foundation’s expanded support for VRI seeks to address a common yet unmet need for NF1 patients. Approximately one in every five NF1 patients develop 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.
The VRI researchers include Dr. Jeffrey Goldberg of the Stanford University School of Medicine, Dr. Yuan Pan of Anderson Cancer Center, Dr. Donald Zack of John Hopkins University, Larry Benowitz of Boston Children’s Hospital, Zhigang He of Boston Children’s Hospital, Dr. David Gutmann of Washington University in St. Louis, Thomas Reh of the University of Washington, Daniel Goldman of the Regents of the University of Michigan, Dr. Peter Baranov of Schepens Eye Research Institute Inc., Michael Young of Schepens Eye Research Institute, Yuan Zhu of the Children’s National Medical Center, Dr. Robert Avery of the Children’s Hospital of Philadelphia, and Dr. Michael Fisher of the Children’s Hospital of Philadelphia.
“Our team hopes to expand on the findings we made in earlier phases, with an increased emphasis on several potentially translatable strategies for neuroprotection and vision restoration in NF1,” says Zhigang He, researcher at Boston Children’s Hospital.
“This funding will help us identify new targets for RGC protection, test the protective and regenerative effects of positive hits, develop a gene therapy approach to restore NF1-like GAP function in NF, and develop myelination-promoting treatments for restoring vision in NF1 test subjects.”
This new round of commitments brings the Gilbert Family Foundation’s total investment into curing NF1 to more than $95 million.
In addition to BTI and VRI, the Gilbert Family Foundation also includes the Gene Therapy Initiative (GTI), which is focused on developing curative therapies that address the underlying genetic abnormalities in NF1 patients.
Dan and Jennifer Gilbert also raise money for NF1 research through NF Forward, which hosts an annual gala called The beNeFit in Detroit each November. This year’s beNeFit is the 10th annual gala. In total, over $45 million dollars have been raised for the Children’s Tumor Foundation and NF Forward through the event. More information can be found here.