Clinical Research in ALS, the Potential of CNM-Au8 in the Care Landscape: James Berry, MD, MPH


The director of the Massachusetts General Hospital ALS Care Center discussed the importance of effective disease-modifying therapies for ALS and the potential of CNM-Au8 in the management of the disease. [WATCH TIME: 2 minutes]

WATCH TIME: 2 minutes

“I think it's important to underline how critical clinical research is in a disease like ALS. Certainly some things that we work on don't work, other things look like they do have benefit. For the community at large, it's a real opportunity to develop drugs that can be hugely beneficial for a population of people that do really need our help.”

In clinical studies, investigational agent CNM-Au8 (Clene Nanomedicine) has demonstrated an ability to restore energy in brain cells and reduce energetic dysregulation, which plays an important role in the disease progression of amyotrophic lateral sclerosis (ALS). Therefore, directly addressing energetic dysregulation in patients with ALS using therapies like CNM-Au8, a gold nanocrystal suspension, could potentially shift the paradigm of care in the field.1

James Berry, MD, MPH, director of the Massachusetts General Hospital ALS Care Center, recently presented findings of the survival analyses of CNM-Au8 from both the RESCUE-ALS trial (NCT04098406) and the HEALEY ALS Platform trial (NCT04297683; Regimen C) at the 2023 American Academy of Neurology (AAN) Annual Meeting, April 22-27, in Boston, Massachusetts.2 Findings from RESCUE-ALS revealed a significant survival benefit with CNM-Au8, resulting in more than a 70% decrease in risk of death (5 vs. 14 events, log-rank HR = 0.291; 95% CI, 0.118-0.718, P = .0115). HEALEY-ALS showed more than a 90% reduction in risk of death alone or death/permanently assisted ventilation (PAV) at week 24 for the 30mg dose, but not the 60 mg dose when adjusted for baseline imbalances (PAV and death, 4 vs. 1, P = .028, death only, 3 vs. 1, P = .057; unadjusted for multiple comparisons).

Berry sat down with NeurologyLive® in an interview at AAN 2023 to talk about effective disease-modifying therapies for ALS and the potential ability to transform the management of the disease. He also talked about the potential benefits of having a panel of different treatments that have disease-modifying effects for patients with ALS. Furthermore, Berry explained the importance of clinical research in addressing the unmet needs of patients with ALS and the ALS community.

Click here for more coverage from AAN 2023.

1. Jiang, J., Wang, Y., & Deng, M. (2022). New developments and opportunities in drugs being trialed for amyotrophic lateral sclerosis from 2020 to 2022. Frontiers in pharmacology, 13, 1054006.
2. Berry J, Maragakis N, Paganoni S, et al. Evidence for Survival Benefit in ALS with CNM-Au8 Treatment Across Three Study Populations. Presented at: 2023 AAN Annual Meeting; April 22-27, Boston, Massachusetts.
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