The pilot study was conducted at Emory University Hospital to evaluate the safety, tolerability and preliminary efficacy of Spheramine and included six patients with moderate to advanced Parkinson’s disease. In this pilot study, Spheramine was administered in the most affected side of the patient’s brain using a needle and MRI guidance. The study’s primary efficacy measure was the improvement in the motor score of the Unified Parkinson’s disease Rating Scale (UPDRS). Secondary efficacy variables included patient-reported quality of life measures.
Results of the study showed long-term improvement of symptoms and, importantly, significant clinical improvements were noted in mobility – an average of 44% improvement from baseline at 48 months in UPDRS motor scores. Significant clinical improvements were also seen in patient-reported quality of life scores, 23% improvement from baseline at 48 months. There were no Spheramine-related serious adverse events reported.
Based on the positive one-year results seen in the open-label pilot study, Titan and its partner Bayer Schering Pharma initiated a multicenter, double-blind, randomized, sham surgery-controlled study (STEPS) to further evaluate the safety and efficacy of Spheramine. This study completed enrollment with 71 patients last year, and top-line efficacy results are expected to be available in third quarter of 2008.