Broadley, S.Vanags, D.Williams, B.Johnson, B.Feeney, D.Griffiths, L.Shakib, S.Brown, G.Coulthard, A.Mullins, P.Kneebone, C.2009-10-282009-10-282009Multiple Sclerosis Journal, 2009; 15(3):329-3361352-45851477-0970http://hdl.handle.net/2440/51600<h4>Background</h4>Chaperonin 10 (Cpn10) is a mitochondrial molecule involved in protein folding. The aim of this study was to determine the safety profile of Cpn10 in patients with multiple sclerosis (MS).<h4>Methods</h4>A total of 50 patients with relapse-remitting or secondary progressive MS were intravenously administered 5 mg or 10 mg of Cpn10 weekly for 12 weeks in a double-blind, randomized, placebo controlled, phase II trial. Clinical reviews, including Expanded Disability Status Scale and magnetic resonance imaging (MRI) with Gadolinium, were undertaken every 4 weeks. Stimulation of patient peripheral blood mononuclear cells with lipopolysaccharide ex vivo was used to measure the in vivo activity of Cpn10.<h4>Results</h4>No significant differences in the frequency of adverse events were seen between treatment and placebo arms. Leukocytes from both groups of Cpn10-treated patients produced significantly lower levels of critical proinflammatory cytokines. A trend toward improvement in new Gadolinium-enhancing lesions on MRI was observed, but this difference was not statistically significant. No differences in clinical outcome measures were seen.<h4>Conclusions</h4>Cpn10 is safe and well tolerated when administered to patients with MS for 3 months, however, a further extended phase II study primarily focused on efficacy is warranted.enchaperonin 10clinical trialheat shock proteinsmultiple sclerosis [41]randomizedcontrolled (CONSORT agreement)treatmentJournal article002009036910.1177/13524585080991410002644681000092-s2.0-6184915037539150Shakib, S. [0000-0002-7199-5733]