Oral 23rd International Society of Magnetic Resonance Conference 2023

Revealing the mechanism of potent SARM1 inhibition by small molecules (#175)

Yun Shi 1 , Jeffrey D. Nanson 2 , Weixi Gu 2 , Tamim Mosaiab 1 , Veronika Masic 1 , Eduardo Vasquez 1 , Mohammad Kawsar Manik 2 , Sulin Li 2 , Bostjan Kobe 2 , Thomas Ve 1
  1. Institute for Glycomics, Griffith University, Southport, QLD, Australia
  2. School of Chemistry and Molecular Biosciences, University of Queensland, Brisbane, QLD, Australia

SARM1 (sterile alpha and TIR motif containing 1) is a key executioner of axon degeneration and a promising therapeutic target for neurodegenerative disorders. It catalyzes the cleavage of the essential metabolite nicotinamide adenine dinucleotide (NAD+) in neurons, leading to axon degeneration and eventual neuronal death. High-throughput screening had identified 5-iodo-isoquinoline, a small molecule with only 11 heavy atoms, as a potent inhibitor of SARM1 (IC50 = 75 nM). Employing NMR-based enzymatic assays, we demonstrated that 5-iodo-isoquinoline undergoes a base-exchange reaction with the nicotinamide base of NAD+, and that the product, namely 1AD, is the bona fide inhibitor. We further uncovered the double-displacement catalytic mechanism of the base-exchange reaction. In addition, our NMR-based assays showed that similar base-exchange reactions can occur with other pyridine and isoquinoline derivatives, and that base-exchange activity is likely to be common among TIR motif containing proteins. While some base-exchange products have become valuable chemical probes for structural biology, this mechanism is also being leveraged to develop further inhibitors of SARM1 and axon degeneration. 

  1. Shi et al. Molecular Cell (2022), 82, 1-17. Manik et al. Science (2022), 377, eadc8969.