The Year in Neurology: Studies Not to Miss From 2011

Andrew N. Wilner, MD


November 23, 2011

In This Article


Neurology may no longer be cavalierly dismissed as an arcane specialty relegated to the paradigm of "Diagnose and adios." New therapies continue to appear at an increasing rate thanks to the hard work and collaboration of basic scientists and clinicians all over the world. The art of clinical drug trials, while not perfected, has progressed so that design and expectations regarding a primary endpoint have been standardized to produce reliable and credible results. The FDA is doing its job by closely examining new data, insisting that drugs meet their primary endpoint in clinical trials, and protecting the public by paying close attention to the potential for serious adverse events.

For the prevention of stroke, rivaroxaban, a factor Xa inhibitor, follows last year's approval of dabigatran as an alternative to warfarin in patients with nonvalvular atrial fibrillation. Another factor Xa inhibitor, apixaban, appears to be at least as effective as rivaroxaban, if not more so, and may soon receive FDA approval. These drugs herald the beginning of the end of the classic "Coumadin clinic" because they do not require INR monitoring. For the treatment of multiple sclerosis, at least 2 new oral drugs, BG-12 and teriflunomide, promise to decrease relapse rate and progression of disability without an increased risk for opportunistic infections or malignancies. These drugs rely on novel mechanisms of action stemming from an increased understanding of the immune system. FDA approval should be forthcoming. Although clobazam is a benzodiazepine that has been available in other countries for many years, its availability in the United States will help ameliorate the pain and distress of recurrent drop attacks in patients with Lennox-Gastaut syndrome.

Additional drugs for neurologic disorders are on the horizon. For stroke, 2 more factor Xa inhibitors are in development: edoxaban and betrixaban. One of the limitations of the new non-warfarin anticoagulants is their lack of reversibility with vitamin K, but at least 1 company is exploring a factor Xa inhibitor antidote. For epilepsy, clinical trials have been completed for perampanel, a first-in-class, noncompetitive, AMPA-type glutamate receptor antagonist. Monoclonal antibodies alemtuzumab, daclizumab, and ocrelizumab may prove to be powerful and convenient future therapies for multiple sclerosis. Cures remain elusive, but an increasing number of new treatment options will improve the quality of life for many people with diseases of the nervous system.


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