Innovations in stroke clinical trial design

The pace of development of new drugs and devices for the treatment of stroke is dependent upon the rate that promising compounds can be tested in humans. While this final stage of drug development is the product of years of preclinical work, it is a critical step not only because these trials are extremely expensive and take many years to complete but also because poorly designed clinical trials can derail a promising therapy or promote an ineffective one. Historically, the stroke field has suffered from a poor track record in drug development. The only approved therapy for acute stroke is intravenous tPA (N Engl J Med 333:1581-87). Stroke preventive therapies include antithrombotic agents and statins (Eur Heart J. 2008;29:1082-3; BMJ. 2002;324:71-86; N Engl J Med. 2006;355:549-59). There are no proven therapies for stroke recovery. There are numerous obstacles inherent to developing therapies for stroke, such as the need to deliver drug to the affected blood vessels and injured brain tissue, the narrow window for intervention, and the heterogeneity of the patient population.