The Paradigm Shift in Therapeutic Intervention for ALS: Matthew Kiernan, MBBS, PhD, DSc, FRACP, FAHMS

WATCH TIME: 3 minutes

“It is a paradigm shift. A lot of this has come through understanding the disease itself. TDP43 and the connections to ALS was a relatively recent discovery in the last decade. Then, there’s been a lot of interest in ALS with the Ice Bucket Challenge and there’s been a lot of support from industry and philanthropy, and that’s driven the science through the roof. As a result, now we have a lot of compounds coming through.”

For neuromuscular disorder specialists such as Matthew Kiernan, MBBS, PhD, DSc, FRACP, FAHMS, there has been a paradigm shift in clinical intervention. One such example is Clene Nanomedicine’s investigational therapy for amyotrophic lateral sclerosis (ALS), CNM-Au8, which is an oral suspension of gold nanocrystals. In recently announced phase 2 data, the therapy showed promising results, demonstrating a 70% survival benefit among patients with ALS who entered the open-label extension of the trial (n = 36; 90% of eligible patients).¹

Data from that study, dubbed RESCUE-ALS (NCT04098406), were presented at the 2022 American Academy of Neurology (AAN) Annual Meeting, April 2-7, in Seattle, Washington, by Kiernan, who is the Bushell Chair of Neurology and professor of medicine at the Central Clinical School, as well as the codirector of Discovery and Translation at the Brain and Mind Centre of the University of Sydney and a neurologist in the Institute of Clinical Neurosciences at Royal Prince Alfred Hospital. The interim analysis of observed survival compared with estimated median survival derived from the validated European Network for the Cure of ALS prediction model, showed a significant signal for CNM-Au8 treatment on survival (HR, 0.3; P = .006, log-rank test).^1,2

Kiernan, an investigator in the trial, sat down with NeurologyLive^® to discuss the newly announced interim results being presented at the AAN meeting, offering his thoughts on the clinical takeaways from RESCUE-ALS. Additionally, he spoke to the larger progress that therapies such as CNM-Au8 represent for the field of neuromuscular medicine, suggesting that a shift in thinking has occurred, noting that the insights gained in the last decade have vaulted the field into a new era of therapy.

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REFERENCES
1. Updated Interim Data from Clene Nanomedicine’s RESCUE-ALS Open-Label Extension Study to be Presented in Emerging Science Program at 2022 AAN Annual Meeting. News release. Clene Nanomedicine. April 1, 2022.
2. Kiernan MC. Long-Term Survival of Randomized Participants Enrolled in the Phase 2 RESCUE-ALS Trial of CNM-Au8, A Cellular Energetic Catalyst. Presented at: AAN Annual Meeting; April 2-7, 2022; Abstract 007.