Clinical Trials

Since the NIH Clinical Center opened in 1953, clinical trials have been a key feature of the Intramural Research Program (IRP) portfolio. The program excels in the type of basic research needed to advance biomedical knowledge, providing the very foundation for health research worldwide, as well as the type of clinical research that culminates in cures and therapies.

Clinical trials performed in the IRP are distinct. Some have tested research concepts so nascent that no scientific literature existed that could support a traditional grant application. In other cases, the advances arose from years of basic and clinical research only made possible by the equipment, expertise, and research freedom available in the IRP. The following are a few examples of our clinical trials work:

• The HPV “anti-cancer” vaccine—The National Cancer Institute (NCI) Center for Cancer Research (CCR) has developed a novel vaccine technology which, in turn, has led to the first FDA-approved vaccine against cancer. CCR partnered with private industry to create two vaccines against certain cancerous strains of human papillomavirus (HPV), which could help to prevent most cases of cervical cancer, as well as genital warts and other types of genital cancer. The NCI Division of Cancer Epidemiology and Genetics (DCEG) continues this work with a Phase 3 trial in Costa Rica testing the safety and efficacy of one of these vaccines against specific deadly strains of HPV. The vaccine likely will have its major impact on the prevention of cervical cancer in developing nations as well as in underserved populations within the United States.
• Gene therapy to restore salivary gland function—Ionizing radiation is required to treat most head and neck cancer patients. Because of this, salivary glands ultimately suffer irreversible damage, leading to dry mouth (xerostomia), difficulty in swallowing (dysphagia), and severe oral infections. The National Institute of Dental and Craniofacial Research (NIDCR) developed a gene transfer strategy to treat this condition, involving the delivery of human aquaporin-1 cDNA to parotid glands. After successful animal studies, the FDA has given full approval to a clinical protocol that is currently underway to test the safety and efficacy of this strategy in adult patients with established radiation-induced parotid gland hypofunction.
• Multiple Sclerosis: treatment and new insights—Over the past eight years, the National Institute for Neurological Disorders and Stroke has conducted a series of clinical and basic studies of daclizumab in the treatment of multiple sclerosis (MS). NCI scientists initially developed daclizumab as a treatment for a rare type of leukemia. Multiple clinical studies of MS patients already on approved therapy, but with breakthrough disease activity, demonstrated an impressive and significant treatment effect. In addition, detailed examinations of the immunological events associated with treatment uncovered how daclizumab works at the cellular level which will contribute to the development of future therapies.
• Biodefense: Ebola and Marburg—Researchers at the Vaccine Research Center (VRC), which is part of the National Institute for Allergy and Infectious Diseases (NIAID), are advancing vaccines against Ebola and Marburg hemorrhagic fevers. VRC scientists have developed a vaccine approach that protects monkeys from lethal Ebola virus infection. A Phase 1 trial of the DNA vaccine, which was well tolerated and elicited promising immune responses at all doses, has been completed. VRC scientists also have developed an accelerated vaccine for Ebola that is being tested in humans; a single shot of this fast-acting, experimental Ebola vaccine successfully protected monkeys after only one month.

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