Pilot Trial Of Recombinant Human Growth Hormone For Remyelination In Multiple Sclerosis: Current Status And Preliminary Safety Analysis (P3.155)

OBJECTIVE: The primary aim of this single-cross-over baseline-vs.-treatment phase II clinical trial is to demonstrate disease-specific safety (since IGF-1 induces lymphocyte proliferation in vitro) and provide initial efficacy data. BACKGROUND: Multiple sclerosis (MS), an autoimmune demyelinating disease of the central nervous system, causes neurological disability among young adults. While the inflammatory process can be reduced by available medications, there is no treatment to repair the resulting demyelination. In order to improve maturation of re-myelinating oligodendrocytes in MS, recombinant human growth hormone is administered, inducing the maturation factor IGF-1. RESULTS: At the time of abstract submission, 31 patients have been screened, of whom 19 could be included and start treatment. RhGH treatment reliably induced an increase in serum IGF-1 in all treated patients; tolerability has been excellent, with mild injection site reactions in some patients. One patient suffered a transient flare-up of presumed autoimmune hepatitis, which resolved after discontinuation of rhGH. Two protocol-specified interim safety assessments (after the first four patients, and after 15 patients completed six months of rhGH treatment) revealed no increase in disease activity as assessed clinically and by monthly Gd-enhanced brain MRI. CONCLUSIONS: With excellent subjective tolerability and in the absence of safety findings in either of two interim safety analyses, the trial is being continued until the projected 30 patients have completed the study. Disclosure: Dr. Stoppe has nothing to disclose. Dr. Ettrich has nothing to disclose. Dr. Thomae has nothing to disclose. Dr. Kratzsch has nothing to disclose. Dr. Hoffmann has nothing to disclose. Dr. Hasenclever has nothing to disclose. Dr. Then Bergh has received personal compensation for activities with Biogen Idec, Novartis, Merck Serono, and Sanofi-Aventis Pharmaceuticals Inc. as a speaker or member of an advisory board. Dr. Then Bergh has received research support from Bayer Schering, Novartis and Teva Neuroscience.