Thomas G. Brott, M.D., performed the first population-based study of carotid endarterectomy (CEA). He was the principal designer of the National Institutes of Health (NIH) Stroke Scale. Dr. Brott and his team tested the tissue plasminogen activator (tPA) enzyme alteplase, a first-in-human treatment for acute ischemic stroke. Dr. Brott was the principal investigator of the first large prospective trial to identify natural history and outcomes during the first minutes and hours of intracerebral hemorrhage (ICH).
For prevention of ischemic stroke, Dr. Brott was the principal investigator for the Carotid Revascularization Endarterectomy Versus Stenting Trial (CREST), which demonstrated comparable and excellent safety and efficacy for carotid endarterectomy and carotid artery stenting. Currently, he leads the CREST-2 trial comparing the safety and efficacy of carotid revascularization plus intensive medical treatment to that of intensive medical treatment alone for patients with 70% or higher asymptomatic carotid artery stenosis.
Carotid artery disease
- The population study of carotid endarterectomy showed a troubling rate of complications for CEA, and the results were an important justification for NIH-funded clinical trials of CEA for symptomatic and asymptomatic patients, the North American Symptomatic Carotid Endarterectomy Trial (NASCET) and the Asymptomatic Carotid Atherosclerosis Study (ACAS), respectively.
- The CREST trial results, published in The New England Journal of Medicine, are among the foundations of clinical practice guidelines in the U.S. and Europe. The results were the basis for the U.S. Food and Drug Administration (FDA) approval of carotid stenting of symptomatic and asymptomatic patients with the Acculink and Accunet carotid stent systems.
- The CREST-2 results, expected to be presented and published in 2026, will be the foundation of treatment for patients with severe CAD, defined as stenosis of 70% or higher. Comparable trials have not been completed for the preceding 20 years.
Ischemic and hemorrhagic stroke
- The first-in-human studies of alteplase provided initial safety and efficacy data that guided the design of the phase 3 National Institute of Neurological Disorders and Stroke tPA Stroke Trial. Dr. Brott and his team were the leading enrollers in that trial, the results of which led to FDA licensure of alteplase, the first drug deemed a safe and effective treatment for acute ischemic stroke.
- Dr. Brott and his colleagues then formulated the first combined approach to stroke therapy; this intravenous endovascular strategy is now the standard in patients who have a visible thrombus identified as the cause of their strokes.
- The ICH study demonstrated that major intracranial bleeding continues over the first minutes and hours after ICH onset in more than 30% of patients and that ICH growth is linked to clinical deterioration. The occurrence of ongoing bleeding and its link to clinical deterioration has been the foundation of clinical trials of treatments for ICH.
Significance to patient care
Dr. Brott's studies and clinical trials in carotid artery disease impact patient care at clinics and hospitals throughout the U.S. and many other clinics and hospitals worldwide. The NIH Stroke Scale has become the standard measurement of neurological deficit for patients with stroke and in clinical trials worldwide; it is embedded in EPIC and other electronic medical record systems. Patients with ischemic and hemorrhagic stroke worldwide are examined with the NIH Stroke Scale, imaged with computerized tomography or magnetic resonance imaging (where available), and treated with methods pioneered by Dr. Brott and his colleagues.
- Eugene and Marcia Applebaum Professor of Neurosciences #1, Mayo Clinic, 2009.
- James C. and Sarah K. Kennedy Dean of Research at Mayo Clinic in Florida, Mayo Clinic, 2008-2009.