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UCL Queen Square Institute of Neurology

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Translational Clinical Trials

Dr. Raj Kapoor

At the moment there are no proven treatments for progressive disability in multiple sclerosis. We have been active in defining the pathophysiology of progression, and have translated some of these findings into designing and implementing proof of concept clinical trials of neuroprotection. A phase II trial of sodium channel blockade with lamotrigine was completed in 2010.

We have now substantiated the concept of neuroprotection with sodium channel blockade in a recent trial in which patients with acute optic neuritis were treated with phenytoin, which reduced the loss of the retinal nerve fibre layer significantly when compared with placebo. The protocol for this trial can be seen at

  • ¬†Neuroprotection with Phenytoin in Optic Neuritis: Trial Protocol v3.0 (pdf)

We also led the UK arm of the phase III trial of natalizumab to prevent disability in secondary progressive MS.

Currently we are contributing to a number of international efforts to speed the development of treatments for progressive MS. These include the Progressive MS Alliance, which includes a number of MS Societies around the world, and the MS Outcomes Assessment Consortium, a collaboration between the US MS Society, the Critical Path Institute, and other key stakeholders, to define a new clinical outcome measure for use in phase III trials.

Overall, our objective is to translate findings in basic models of neuroinflammatory and neurodegenerative disease into well designed proof of concept clinical trials, with effective outcome measurements, in order to accelerate the discovery of novel therapies in MS.