A GLP-1 Analog for the Treatment of ALS
[Technical Report, Annual Report]
JOHNS HOPKINS UNIV BALTIMORE MD
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The involvement of neuroinflammation manifested by activated microglia and astrocytes in ALS is supported by a wealth of clinical and molecular evidence. In addition to producing neurotoxic cytokines, activated microglia also induce differentiation of astroglial cells into neurotoxic A1 astrocytes - direct mediators of neuronal cell death, including possibly in ALS. Therefore, development of agents that could selectively inhibit the microglial activation and A1 astrocyte formation without off-target toxicity could have profound therapeutic potential since they could be used to treat a variety of neurologic disorders for which there currently are no disease-modifying therapies. NLY01 is a GLP-1R agonist. NLY01 selectively inhibits microglial activation, and blocks induction of cytokines and A1 astrocyte formation thus, providing neuroprotection. The overall objective is to collect data for a FDA IND application for ALS. We propose to develop NLY01 for ALS its potential as a target using both ALS animal and human ALS iPSC-based models.
- Medicine and Medical Research
- Anatomy and Physiology