Federal Project Grant Award Summary

Hugo W. Moser Research Institute at Kennedy Krieger, Inc. received a $445,500 Project Grant award from the National Institute of Neurological Disorders and Stroke (NINDS) under the Extramural Research Programs in the Neurosciences and Neurological Disorders program (CFDA 93.853), effective June 16, 2026 through May 31, 2028. The research project investigates drug repurposing strategies for X-linked adrenoleukodystrophy (ALD), a severe neurological disorder caused by mutations in the ABCD1 gene that impair the peroxisomal import of very long chain fatty acids. The funded work will explore whether cystic fibrosis transmembrane conductance regulator (CFTR) modulator drugs—developed for cystic fibrosis treatment and previously approved by the FDA—can be repurposed to restore aldehyde dehydrogenase protein (ALDP) function in ALD patients carrying missense variants at homologous residue positions.

The project directly addresses the critical therapeutic gap in ALD treatment by testing the hypothesis that ALD patient cells with specific missense variants will respond to CFTR corrective drugs, leveraging the sequence homology between ABCD1 and CFTR within nucleotide-binding domains. Deliverables include experimental validation of CFTR modulators' ability to reduce very long chain fatty acid (VLCFA) accumulation in patient-derived primary cells, providing a novel translational pathway to restore protein function in a devastating genetic disorder that currently lacks adequate therapeutic options for affected populations.