Working Faster Than the Speed of ALS to Transform Neurodegenerative Disease Research

The Story Behind the Speed of ALS

Dr. Ho’s dedication to ALS research is as much about compassion as it is about science. During the interview, she spoke about Brooke Eby, an ALS advocate who has become an inspiration for many, including Dr. Ho.

“She [Brooke Eby] said that she has to live at the speed of ALS because her disease might kill her in two to five years. So, here’s a young woman in her early thirties who now wants to cram as much of the richness and fullness of life experience into two to five years. At Clene I take this challenge seriously: let’s develop a drug for ALS patients faster than the speed of ALS,” expressed Dr. Ho.

ALS is a brutal disease, often leaving patients with only a few years to live after diagnosis. Dr. Ho’s reflections on this reality underscore the importance of accelerating the development of new treatments. With heartfelt determination, she shared, “Let’s not let another person be told that there’s nothing we can do for you. Let’s instead be able to look them in the eye and say, we’re doing everything we can for you.”

XTALKS CLINICAL EDGE: Issue 3 — Interview with Clene Nanomedicine

Xtalks Clinical Edge is a magazine for clinical research professionals and all who want to be informed about the latest trends and happenings in clinical trials. This magazine immerses you in a world where industry leaders, patient advocates and top researchers converge to provide the most insightful perspectives on clinical trials.

Gold Nanocrystals — Insights into Nanomedicine

Central to Clene’s efforts is CNM-Au8, an oral suspension containing gold nanocrystals. This investigational drug, with completed Phase II trials, is being studied as a potential treatment for ALS, MS and PD. CNM-Au8 works by improving the health of mitochondria — the energy producers in our cells — while also reducing harmful oxidative stress and lowering levels of misfolded proteins. These actions help protect neurons, the cells that are most affected by these diseases.

Dr. Ho elaborated on the mechanism of action of CNM-Au8, explaining how gold nanocrystals, when engineered with clean surfaces, exhibit extraordinary catalytic and therapeutic activities.

“With CNM-Au8, we would expect to see activation of some mitochondrial markers,” Dr. Ho explained. “We would see higher levels of NAD+, and higher levels of the NAD+ to NADH ratio, just to give some examples. And we’d also like to see a decrease in toxic oxidative markers and an activation of an antioxidative response.”

Research suggests that NAD+, a molecule involved in energy production, plays a role in the progression of ALS. By boosting NAD+ levels and improving mitochondrial health, CNM-Au8 could offer a new approach to slowing the progression of neurodegenerative diseases.