NU-9 Update: Experimental drug repairs upper motor neurons in mouse model

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As connecting fibers between motor neurons, axons help deliver vital messages between the brain and the spinal cord. For people living with ALS, deteriorating axons cause that connection to break, contributing to paralysis and death.

Early research on NU-9, an experimental drug invented and developed by members of the Les Turner ALS Center at Northwestern Medicine, has shown the ability to lengthen the axons of diseased upper motor neurons in an SOD1 ALS mouse model. NU-9 also showed an enhanced effect when given in combination with the FDA-approved drugs riluzole and edaravone.

NU-9 was invented by Richard B. Silverman, PhD, the Patrick G. Ryan/Aon Professor at Northwestern University, and animal studies were carried out by P. Hande Ozdinler, PhD, associate professor of neurology at Northwestern’s Feinberg School of Medicine.

The research was published recently in Scientific Reports.

“For a drug to be effective, it is important for that drug to improve axon outgrowth and axon health,” said Dr. Ozdinler, the co-lead author of the study. “This is very important for connecting the brain and the spinal cord and for revitalizing the motor neuron circuitry that degenerates in patients.”

NU-9 is currently moving toward clinical trials. The investigators are carrying out animal safety studies needed for the drug to receive FDA approval and become an Investigational New Drug. Those studies include determining dose level and toxic effects.

“If everything goes well, we hope to start with healthy volunteers in a Phase 1 clinical trial early in 2023,” said Dr. Silverman, co-lead study author.

The Les Turner ALS Foundation is proud to have been an early funder of the initial work on NU-9 through the Les Turner ALS Center at Northwestern Medicine, and we look forward to the next steps for testing and development of this compound through the clinical trial process. Offering access to enrollment in multiple clinical trials and with dedicated clinical trial coordinators, the Lois Insolia ALS Clinic at the Les Turner ALS Center is Chicagoland’s first and largest multidisciplinary ALS clinic.