"This work demonstrates that NALL can influence several Parkinson's disease-relevant pathways, including alpha-synuclein pathology, synaptic function, lysosomal pathways and mitochondrial proteins, suggesting broader relevance for neurodegeneration."
"NALL is an amino acid derivative of the branched-chain amino acid leucine. For decades, NALL has been used outside the U.S. to treat acute vertigo and vestibular disorders, which can cause dizziness and issues with balance. In 2024, NALL received FDA approval for the treatment of Niemann-Pick disease type C1, a rare genetic disease that causes progressive neurological symptoms and organ dysfunction, further supporting NALL's safety in humans."
Northwestern Medicine researchers identified that N-acetyl-L-leucine (NALL), an amino acid derivative of leucine, promotes neuroprotective effects in experimental Parkinson's disease models. NALL simultaneously targets multiple molecular pathways in dopaminergic neurons, including alpha-synuclein pathology, synaptic function, lysosomal pathways, and mitochondrial proteins. The compound has been used internationally for decades to treat acute vertigo and vestibular disorders, and received FDA approval in 2024 for treating Niemann-Pick disease type C1. Scientists generated dopaminergic neurons from induced pluripotent stem cells derived from patients with familial and sporadic Parkinson's disease to examine NALL's mechanisms and therapeutic potential in humans.
#parkinsons-disease-treatment #n-acetyl-l-leucine #neuroprotection #dopaminergic-neurons #fda-approved-compounds
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