Grant to support a novel drug approach in hereditary spastic paraplegia awarded
Grant to support a novel drug approach in hereditary spastic paraplegia awarded
Critical Path Institute’s (C-Path) Translational Therapeutics Accelerator has awarded a $150,000 grant to Gregory Thatcher, PhD, at the University of Arizona to support innovative approaches for treating hereditary spastic paraplegia. Dr. Thatcher is collaborating with Xue-jun Li, PhD, co-director of the Regenerative Medicine and Disabilities Laboratory in the UICOMR Department of Biomedical Sciences, to advance a small molecule drug aimed at causal mechanisms of this debilitating neurological condition.
HSP refers to a group of rare, inherited neurodegenerative diseases characterized by progressive spasticity and weakness in the lower limbs. The disorders are caused by damage to the long nerve fibers that carry movement signals from the brain down the spinal cord, and their global prevalence ranges from 0.1 to 9.6 per 100,000 individuals. More than 80 gene mutations have been linked to HSP, and despite the availability of genetic testing for diagnosis and subtype classification, there are currently no disease-modifying therapies, and treatment remains limited to symptomatic management, such as antispasmodics.
Drs. Thatcher and Li are leveraging a novel drug discovery platform for small molecule Nonlipogenic ABCA1 Inducers (NLAIs) to develop therapeutics that can target upstream, disease-relevant mechanisms common across genetically diverse forms of HSP. Their approach modulates cholesterol and lipid homeostasis in neurons — pathways recognized for their role in axonal integrity and neurodegeneration.
The TRxA program at C-Path is designed to help academic researchers bridge the critical gap between basic discovery and early clinical development. In addition to financial support, awardees receive scientific and regulatory guidance to help de-risk their projects and prepare for future development stages.