Therapy - immunomodulation - Part IIFriday, September 30, 2005, 15:30 - 17:00
GEMSEP1: A new drug candidate for secondary progressive form of multiple sclerosisM. Geffard, C. Tranchant, M. Fleury, S. Wiertlewski, A.-M. Guennoc, M.-P. Dabadie (Pessac, Strasbourg, Nantes, Tours, Cenon, F)
Background: GEMSEP1 is a new drug candidate developed as an oral treatment for Multiple Sclerosis (MS). Being a combination of functional polypeptides, fatty acids, antioxidants, free radicals scavengers and amino acids individually linked to linear poly-lysines (PL, Molecular Weight about 23,000 Daltons), GEMSEP1 is a multi action low dose drug candidate with potential synergistic immunomodulator effect in Secondary Progressive MS (SPMS). An open label and multicenter trial was conducted to evaluate safety and tolerability as primary end point in patients with SPMS.
Methods: 22 patients (age [32 - 62]) with active SPMS (i.e. worsened Expanded Disability Status Scale (EDSS) of at least 1 point in the last 24 months) and EDSS score [3 - 6.5] were included by 3 centres. Patients were treated with 3.2 ml (i.e. 0.96mg of active substance) of GEMSEP1 daily (sublingual administration) for 6 months. Evaluation by investigators was recorded through 5 visits from inclusion date. A fasting blood sample was performed before and at the end of the treatment : complete blood count, platelets, bilirubin, ASAT/ALAT, gammaGT, alkaline phosphatases, total cholesterol, HDL-cholesterol, LDL-cholesterol, apolipoproteins A1 and B, triglycerides, glycemia and creatinine. EDSS was assessed at baseline and final visit.
Results: The analysis of adverse events (AEs) did not show evidence for any potential organ effect : incidence of AE generally remained under 20% for most organ systems. Incidence of AE related to “central and peripheral nervous system” was 30%, which included cases of disease progression .Incidence of AE related to “body as a whole/general disorders” was 23%, which included cases of influenza-like symptoms, fatigue (frequently seen in MS), hot flushes and leg pain.. None of these AE was linked to the product according to investigators and no serious adverse event was reported during the trial. No biological abnormality nor abnormal change was reported in laboratory tests. EDSS score remained unchanged in 55% of the patients (12/22), improved in 18% (4/22) and worsened in 27% (6/22).
Conclusions: This first pilot study suggests a good safety profile of GEMSEP1 in human.
These preliminary biological and clinical results are encouraging for further development of GEMSEP1 toward a phase IIb trial.