We are pleased to announce more exciting news on the ALS research front coming out of our Les Turner ALS Center at Northwestern Medicine.
On February 22, Northwestern scientists, led by P. Hande Ozdinler, PhD, and Richard B. Silverman, PhD, members of the Les Turner ALS Center at Northwestern Medicine, authored a report published in Clinical and Translational Medicine (“Improving mitochondria and ER stability helps eliminate upper motor neuron degeneration that occurs due to mSOD1 toxicity and TDP‐43 pathology”), that identified the first compound that eliminates the ongoing degeneration of upper motor neurons that become diseased, which is a key contributor to ALS.
In addition to ALS, upper motor neuron degeneration also results in other motor neuron diseases, such as hereditary spastic paraplegia (HSP) and primary lateral sclerosis (PLS). So far, there has been no drug or treatment for the brain component of ALS, and no drug for HSP and PLS patients.
“Even though the upper motor neurons are responsible for the initiation and modulation of movement, and their degeneration is an early event in ALS, so far there has been no treatment option to improve their health,” said Dr. Ozdinler. “We have identified the first compound that improves the health of upper motor neurons that become diseased.”
When the newly identified compound, NU-9, was administered to mice, the upper motor neurons began to regain their health. In fact, the neurons stopped degenerating so much so that the originally diseased neurons became similar in health to the healthy, control neurons, after 60 days of treatment.
“I am very excited to find out if our hypothesis that stabilizing upper motor neurons in mice will translate to humans and NU-9 will provide hope for those inflicted with currently untreatable upper motor neuron diseases,” said Dr. Silverman.
We understand the urgent need to move forward as quickly as possible with this study. Next steps for the NU-9 compound includes a more detailed toxicology and pharmacokinetic study, which needs to be completed before any consideration can be given to conduct clinical trials in people living with ALS.
This study was launched with a pilot research grant funded by the Les Turner ALS Center at Northwestern Medicine and received funding from a variety of sources.
“We are delighted to see the outcome and potential implications of this important study resulting from the Foundation’s research program,” said Les Turner ALS Foundation CEO Andrea Pauls Backman.
To view a short video abstract that explains the study in more detail, click here.