Mass General Hospital

Healey Community Q&A Webinar: March 12, 2026 | CNM-Au8 Expanded Access Update

The Healey Community Q&A webinar on March 12, 2026 featured Dr. Jinsey Andrews presenting interim results from the NIH‑funded CNM‑AU8 expanded access program (EAP) for amyotrophic lateral sclerosis (ALS). The program targets patients ineligible for traditional clinical trials, offering them investigational therapy while simultaneously generating real‑world data on safety, efficacy, and disease biomarkers such as neurofilament light chain (NfL). Key insights include the feasibility of remote enrollment, which attracted a demographically diverse cohort—half enrolled virtually, many from high area‑deprivation index regions and geographically isolated locations like Alaska. Using the Answer ALS database, the team created propensity‑matched controls to compare outcomes, revealing that CNM‑AU8 30 mg consistently reduced NfL area‑under‑the‑curve (AUC) at weeks 24, 36, and 48, with statistically significant reductions in the overall and bulbar‑onset subgroups. Additional exploratory biomarkers, including GFAP, were also evaluated, though results remain preliminary. Dr. Andrews illustrated the neurofilament analysis with a vivid analogy: a single NfL measurement is like a snapshot of smoke, whereas AUC reflects the total smoke over the night, offering a more comprehensive view of disease activity. She highlighted a remote participant in Alaska who required air transport for home assessments, underscoring the program’s reach and patient impact. The discussion also addressed why AUC was chosen, noting its superior correlation with survival amid the dynamic fluctuations of NfL levels. The findings suggest that expanded access programs can yield supportive efficacy signals and robust biomarker data, potentially accelerating future ALS trial designs and informing regulatory deliberations. By integrating research aims into access initiatives, the NIH model builds infrastructure, validates candidate biomarkers, and expands therapeutic options for underserved ALS populations.