Hemispherx Biopharma, Inc this week released preliminary findings for the treatment of Chronic Fatigue Syndrome (CFS) with their flagship investigational drug, Ampligen®, under an expanded access program. These findings were first presented at the American Association for Chronic Fatigue Syndrome conference in Seattle, Washington in a peer-reviewed session.
The objective of this study was to review clinical endpoints including Karnofsky Performance Scores (KPS) and Cognitive Function data derived from an expanded access treatment protocol (trial AMP 511) using Ampligen® for the treatment of CFS.
Chronic Fatigue Syndrome is a symptom complex characterized by both cognitive as well as physical impairments. Although the cause of CFS remains under investigation, the presenting symptoms have been codified by the Centers for Disease Control (CDC) and prevention to assist in making a CFS diagnosis. The primary and most salient feature of CFS is persistent and pervasive fatigue and a decrease in physical performance.
Accordingly, the investigators utilized several Quality of Life (QOL) instruments, including the Karnofsky Performance Score (KPS), as well as a self-reported and physician completed instrument evaluating functionality, as endpoints assessing the clinical benefit of Ampligen®. Clinical endpoints included changes in KPS, cognitive deficit (SCL-90-R), and vitality (SF-36) during 24 to 64 weeks of therapy in this expanded access program with cost recovery (AMP 511). An activity monitor was also utilized in a subset of patients to help evaluate changes in physical activity during the study. Ampligen® dosing (200 to 400 mg twice weekly) and data collection intervals (every 8 weeks) were maintained during the study. Data from 41 severely debilitated FS patients who have received at least 16 weeks of Ampligen® treatment, an investigational drug agent, were available and included in the analyses.
The preliminary findings suggested the KPS increased from a median of 50 at Baseline before treatment to 60 by week 24. The median change in KPS from Baseline was +10 points at weeks 16 and 24. Thirteen patients were entered into an extension phase of the study and received at least 64 weeks of investigational drug Ampligen® treatment. For these thirteen severely debilitated CFS patients, KPS increased from a median of 50 at Baseline to 70 by week 64. The median change in KPS from Baseline was +20 KPS points (weeks 32 – 56) and +30 KPS points (week 64). Significant changes in cognitive deficit, vitality and activity monitor scores were also reported by week 24. The investigational drug treatment long-term was generally well tolerated and the majority of patients (>80%) entered the extension phase of the study receiving treatment beyond 24 weeks.