Project Grant F32NS148709

Grant Award Summary

The National Institute of Neurological Disorders and Stroke (NINDS) awarded The Trustees of Columbia University in the City of New York $227,640 under the Extramural Research Programs in the Neurosciences and Neurological Disorders program (CFDA 93.853) for a two-year project running from November 1, 2026 through October 31, 2028. The research investigates CCR5 (C-C chemokine receptor type 5) as a combination therapy target for spinal muscular atrophy (SMA), a devastating neuromuscular disorder caused by deficiency of the SMN (survival motor neuron) protein. While three SMN-restoring therapies have been approved for SMA, many patients continue to experience persistent motor deficits. This project aims to explore how targeting neuroinflammation—specifically the CCR5-dependent neuroimmune signaling axis—could enhance treatment outcomes when combined with existing therapies.

The research will examine the role of reactive microglia and astrocytes in motor neuron synaptic dysfunction and test whether CCR5 antagonism can reduce pro-inflammatory signaling and improve motor circuit function in SMA mouse models. By addressing the understudied neuroinflammatory component of SMA pathogenesis, this project seeks to develop therapeutic strategies that complement existing SMN-restoring approaches and better address persistent motor deficits in SMA patients, particularly those treated at later disease stages where current interventions yield limited recovery.