The National Institute on Aging (NIA) has allocated approximately $7.3 million to further research on NU-9, an experimental drug developed by Northwestern University. The funding aims to explore the drug's potential effectiveness in treating neurodegenerative diseases beyond amyotrophic lateral sclerosis (ALS), such as Alzheimer's disease and frontotemporal degeneration.
NU-9, invented by chemist Richard B. Silverman, targets underlying mechanisms common to several neurodegenerative diseases rather than just addressing symptoms. Silverman collaborated with neurologist P. Hande Ozdinler in developing the compound. Both are affiliated with Northwestern University and are members of the Les Turner ALS Center and the Chemistry of Life Processes Institute.
“NU-9 acts on some of the shared causes of neurodegeneration, so we thought it may have a broader impact beyond ALS,” said Ozdinler, who is leading the new NIA-funded project. She noted that ALS, FTD, and Alzheimer's disease share cellular issues like mitochondrial dysfunction and brain inflammation.
In previous studies using mouse models for ALS, NU-9 improved neuron health significantly after 60 days of treatment. The drug showed promise in lengthening axons and improving mitochondria and endoplasmic reticulum health within neurons.
Silverman founded Akava Therapeutics to conduct necessary safety studies for NU-9, now known as AKV9, aiming for approval from the U.S. Food and Drug Administration (FDA). The FDA granted Investigational New Drug status last year, allowing Phase I trials to assess safety in healthy human subjects.
Ozdinler likened protein aggregates in neurodegenerative diseases to "garbage inside a house," explaining that NU-9 helps clean these aggregates out of cells, potentially restoring neuron health.
For this expanded research effort funded by NIA, William Klein from Northwestern's Weinberg College has joined Silverman and Ozdinler’s team. Klein brings expertise in Alzheimer’s disease research.
“It is a long process — possibly 10 to 12 years — to discover and bring a new drug to the market,” Silverman stated. “But this drug has us very excited and hopeful about its potential.”
The project titled “Investigations of the protein aggregation inhibitor NU-9 and its derivatives: Mode of action and impact on Alzheimer’s disease and related dementias” is supported under grant number 2R01AG061708-06 from NIH's National Institute on Aging.
Silverman, Ozdinler, Klein, along with Northwestern University itself hold financial interests in Akava Therapeutics through equities or royalties.