Examining IGFBP7 as a potential therapeutic target in people with ALS.

A single nucleotide variant in an insulin-like growth factor (IGFBP7) promotor, which reduces IGFBP7 levels in brain, was previously associated with an ALS "reversal" phenotype. This raises the question of whether IGFBP7 might be a therapeutic target in ALS. Here, we use a combinatorial analysis to show that IGFBP7-Antisense (AS1) is associated with resistance to ALS. In ALS patients' blood, we demonstrate increased IGFPB7 protein relative to healthy controls. In ALS patients' spinal cords and iPS-derived motor neurons, we demonstrate increased IGFBP7 mRNA levels relative to healthy controls. These four new analyses support IGFBP7 as a possible therapeutic target in ALS.