Lucia Notterpek, Ph.D., professor and senior associate dean for research at the w88 login, Reno School of Medicine (UNR Med), has been awarded anInnovation Grants to Nurture Initial Translational Efforts(IGNITE) grant from the National Institute of Neurological Disorders and Stroke (NINDS) for over M to further research into treatments for demyelinating neuropathies—debilitating disorders that currently have no cure.
Running from January 1, 2025, to December 31, 2027, the three-year grant will support Notterpek and her co-investigators Thomas Gould, Ph.D., and Nicole Procacci, Ph.D., to examine how a novel drug could help restore myelin, the protective sheath around nerves that is critical for proper nerve function. Damage to myelin, whether from genetic disorders or injury, can lead to progressive pain, muscle weakness and disability, as seen in conditions such as multiple sclerosis and several peripheral nerve system diseases.
“Myelin, a lipid enriched insulator produced by glial cells, is essential for nerve signals to travel efficiently,” said Notterpek. “When myelin deteriorates, or becomes abnormal due to genetic conditions, patients experience significant neurological symptoms, yet there are currently no disease-modifying treatments available.”
Notterpek’s research focuses on cholesterol’s role in myelin production. Her team recently discovered that a protein called PMP22 plays a key role in regulating cholesterol access in Schwann cells, the cells responsible for creating myelin in the peripheral nervous system. Mutations in the PMP22 gene, which affect cholesterol distribution, are responsible for over 60% of inherited peripheral neuropathies.
To address this, Notterpek’s team is collaborating with Artery Therapeutics, Inc., a biotechnology company that has developed a promising drug candidate, CS6253. Originally designed for Alzheimer’s disease, this drug has shown potential in early studies for correcting cholesterol mislocalization in Schwann cells, potentially restoring myelin production and improving nerve function.
“Our preliminary research indicates that this drug can help Schwann cells regain proper cholesterol balance, which is crucial for repairing damaged myelin,” Notterpek explained. “Since CS6253 cleared investigational new drug (IND) status and Phase 1 safety studies in healthy volunteers, we could be one step closer to a much-needed treatment for patients with neuropathy, if our upcoming studies confirm our preliminary promising findings.”
With rigorous testing planned in specialized animal models, the next phase of this research will determine whether CS6253 can safely and effectively restore nerve function. If successful, the drug could rapidly move to clinical trials, offering new hope to those suffering from demyelinating neuropathies.
“This grant allows us to push forward with groundbreaking work that could transform the way we treat these conditions,” said Notterpek. “We’re excited about the potential impact for patients who currently have very few options.”
