NU 2018-085
Inventors
Punett Opal
Samuel I. Stupp*
Short Description
A novel cost-effective neuropeptide with VEGF activity, less immunogenicity and great stability.
Background
Beyond its canonical role as an angiogenic factor, vascular endothelial growth factor (VEGF) has recently been implicated in several neurodegenerative diseases such a Spinocerebellar Ataxia Type 1 (SCA1). SCA1 pathology has been associated with abnormally low VEGF and conversely improved with exogenous VEGF treatment. However, despite its promising therapeutic efficacy, treatment with exogenous VEGF as clinical therapy remains challenging as recombinant VEGF is prohibitively expensive, highly immunogenic and biologically unstable.
Abstract
To address the pressing need for an affordable, bio-stable and non-immunogenic VEGF neurodegeneration therapy, Northwestern researchers have designed a new amphilic VEGF-mimetic peptide called nano-VEGF. This novel compound potently mimics endogenous VEGF biological activity. Nano-VEGF effectively engages with VEGF receptors to initiate robust downstream signaling in vitro. Proof of concept in vivo treatment with nano-VEGF resulted in both functional improvements in SCA1 mice and decreased neuropathology, even when treatment occured during advanced disease. The stabilized recombinant nano-VEGF peptide is retained in the brain parenchyma for up to four weeks following delivery, before eventually biodegrading by design. Finally, the development of nano-VEGF reveals a novel low cost strategy for generating prohibitively expensive and biologically unstable therapeutic neuropeptides such as nerve growth factor (NGF) and brain derived neurotrophic factor (BDNF), which are essential for the treatment and management of diverse neurological diseases.
Applications
Advantages
Publications
Hu YS et al., Self-assembling vascular endothelial growth factor nanoparticles improve function in spinocerebellar ataxia type 1. Brain. Feb 1, 2019
IP Status
US Patent pending (17/292,265)