The Significance of AMX0035 and a Fueled Hope for ALS
Once thought nearly impossible to treat, ALS has seen another step of progress with the approval of AMX0035 and a future of potential therapeutics on the way.
On King’s staging systems, ALSAQ-5 scores increased from 24.6 at stage 1 to 62.1 at stage 4, whereas for Milano-Torino Staging systems, patients’ ALSAQ-5 scores increased from 43.2 at stage 0 to 80.7 at stage 3.
Using several validated patient-reported outcome (PRO) measures, findings from a real-world survey of patients with amyotrophic lateral sclerosis (ALS) showed that the quality of life for these patients worsens as the disease continues to progress. Presented at the
In the study, 142 neurologists reported data on 880 people with ALS by completing questionnaires on patients’ demographic and clinical characteristics, including ALS Functional Rating Scale-Revised (ALSFRS-R). The ALSFRS-R was used to derive King’s staging and Milano-Torino Staging (MiToS) systems, which are well-established measures to monitor ALS disease progression. These same patients were invited to complete PRO measures including the ALS Assessment Questionnaire (ALSAQ-5), Fatigue Severity Scale (FSS), and Work Productivity and Activity Impairment (WPAI)
Senior investigator Sarah Parvanta, PhD, MPH, director ALS Focus Program,
Due to the small group size (n = 1), MiToS stage 4 was excluded. All told, PRO data were provided by up to 135 patients with ALS. All told, ALSAQ-5 scores were significantly positively correlated with both King’s and MiToS stages of disease severity. On King’s staging, ALSAQ-5 scores increased from 24.6 at stage 1 to 62.1 at stage 4, while for MiToS systems, patients’ ALSAQ-5 scores increased from 43.2 at stage 0 to 80.7 at stage 3.
Once thought nearly impossible to treat, ALS has seen another step of progress with the approval of AMX0035 and a future of potential therapeutics on the way.
The ALSAQ-5, which includes 5 questions of physical mobility, activities of daily living, eating/drinking, communication, and emotional functioning, showed a stepwise increase in impairment at each progressing stage for both King’s (r2 = 0.371; P = <.001) and MiToS (r2 = 0.193; P <.001). Significant levels of fatigue, as demonstrated by FSS scores above 40, were reported across all stages of the disease, and were significantly positively correlated with King’s (r2 = 0.234; P <.001) and non-linearly correlated with MiToS (r2 = 0.136; P = .011).
A similar study published in 2019 looked at the impact of disease progression on health-related quality of life for patients with ALS. Using a cohort of 161 individuals, ALSFRS-R score (ß = 0.75; P <.001), depression (ß = 0.08; P <.001), pain (ß = 0.07; P <.001), hopelessness (ß = 0.07; P = .001), and progression rate (ß = 0.02; P = .02) explained 76% of the ALSAQ-40 summary index variance, while progression rate alone explained 7% of the variance. The subdomains of emotional well-being, followed by activities of daily living, and finally communication and eating, were most strongly influenced by progression rate.2
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