Clinical trials are prospective biomedical or behavioral research studies on human participants designed to answer specific questions about biomedical or behavioral interventions, including new treatments and known interventions that warrant further study and comparison. Clinical trials generate data on dosage, safety and efficacy. They are conducted only after they have received health authority/ethics committee approval in the country where approval of the therapy is sought. These authorities are responsible for vetting the risk/benefit ratio of the trial—their approval does not mean the therapy is 'safe' or effective, only that the trial may be conducted.
Biogen Inc. is an American multinational biotechnology company based in Cambridge, Massachusetts, United States specializing in the discovery, development, and delivery of therapies for the treatment of neurological diseases to patients worldwide. Biogen operates in Argentina, Brazil, Canada, China, France, Germany, Hungary, India, Italy, Japan, Mexico, Netherlands, Poland, Sweden, and Switzerland.
Rosiglitazone is an antidiabetic drug in the thiazolidinedione class. It works as an insulin sensitizer, by binding to the PPAR in fat cells and making the cells more responsive to insulin. It is marketed by the pharmaceutical company GlaxoSmithKline (GSK) as a stand-alone drug or for use in combination with metformin or with glimepiride. First released in 1999, annual sales peaked at approximately $2.5-billion in 2006; however, following a meta-analysis in 2007 that linked the drug's use to an increased risk of heart attack, sales plummeted to just $9.5-million in 2012. The drug's patent expired in 2012.
Rimonabant (also known as SR141716; trade names Acomplia, Zimulti) is an anorectic antiobesity drug approved in Europe in 2006 but was withdrawn worldwide in 2008 due to serious psychiatric side effects; it was never approved in the United States. Rimonabant is an inverse agonist for the cannabinoid receptor CB1 and was first-in-class for clinical development.
In clinical trials, a surrogate endpoint is a measure of effect of a specific treatment that may correlate with a real clinical endpoint but does not necessarily have a guaranteed relationship. The National Institutes of Health (USA) defines surrogate endpoint as "a biomarker intended to substitute for a clinical endpoint".
An approved drug is a medicinal preparation that has been validated for a therapeutic use by a ruling authority of a government. This process is usually specific by country, unless specified otherwise.
The Infectious Diseases Society of America (IDSA) is a medical association representing physicians, scientists, and other healthcare professionals who specialize in infectious diseases. It was founded in 1963 and is based in Arlington, Virginia. As of 2018, IDSA had more than 11,000 members from across the United States and nearly 100 other countries on six different continents. IDSA's purpose is to improve the health of individuals, communities, and society by promoting excellence in patient care, education, research, public health, and prevention relating to infectious diseases. It is a 501(c)(6) organization.
Flibanserin, sold under the brand name Addyi, is a medication approved for the treatment of pre-menopausal women with hypoactive sexual desire disorder (HSDD). The medication improves sexual desire, increases the number of satisfying sexual events, and decreases the distress associated with low sexual desire. The most common side effects are dizziness, sleepiness, nausea, difficulty falling asleep or staying asleep and dry mouth.
Kenneth L. Davis is the executive vice chairperson of the board of trustees at the Mount Sinai Health System in New York City, and an American author and medical researcher who developed the Alzheimer's Disease Assessment Scale, the most widely used tool to test the efficacy of treatments for Alzheimer's disease designed specifically to evaluate the severity of cognitive and noncognitive behavioral dysfunctions characteristic to persons with Alzheimer's disease. His research led to four of the first five FDA-approved drugs for Alzheimer's.
The United States Food and Drug Administration (FDA) initiated the FDA Accelerated Approval Program in 1992 to allow faster approval of drugs for serious conditions that fill an unmet medical need. The faster approval relies on use of surrogate endpoints. Drug approval typically requires clinical trials with endpoints that demonstrate a clinical benefit, such as increased survival for cancer patients. Drugs with accelerated approval can initially be tested in clinical trials that use a surrogate endpoint, or something that is thought to predict clinical benefit. Surrogate endpoints typically require less time, and in the case of a cancer patient, it is much faster to measure a reduction in tumor size, for example, than overall patient survival.
Aducanumab, sold under the brand name Aduhelm, is a monoclonal antibody designed to treat Alzheimer's disease. It is a monoclonal antibody that targets aggregated forms (plaque) of amyloid beta (Aβ) found in the brains of people with Alzheimer's disease to reduce its buildup. It was developed by Biogen and Eisai. Aducanumab is given via intravenous infusion.
Encenicline is a selective partial agonist of the α7 nicotinic receptor. It was in phase III clinical trials for the treatment of cognitive impairment in schizophrenia, but failed to meet the study endpoints in 2016.
Scott Gottlieb is an American physician, investor, and author who served as the 23rd commissioner of the Food and Drug Administration (FDA) from May 2017 until April 2019. He is presently a senior fellow at the conservative think tank the American Enterprise Institute (AEI), a partner at the venture capital firm New Enterprise Associates (NEA), a member of the board of directors of drug maker Pfizer, Inc and gene sequencing company Illumina, Inc., and a contributor to the cable financial news network CNBC and the CBS News program Face the Nation. An elected member of the National Academy of Medicine, Gottlieb is the author of The New York Times best selling book Uncontrolled Spread on the COVID-19 pandemic and the national security vulnerabilities that it revealed. His forthcoming book, The Miracle Century: Making Sense of the Cell Therapy Revolution, traces the scientific achievements that propelled progress in cell therapies.
Amyloid-related imaging abnormalities (ARIA) are abnormal differences seen in magnetic resonance imaging of the brain in patients with Alzheimer's disease. ARIA is associated with anti-amyloid drugs, particularly human monoclonal antibodies such as aducanumab. There are two types of ARIA: ARIA-E and ARIA-H. The phenomenon was first seen in trials of bapineuzumab.
Milton Packer is an American cardiologist who is known for his clinical research concerning heart failure.
Susan S. Ellenberg is an American statistician specializing in the design of clinical trials and in the safety of medical products. She is a professor of biostatistics, medical ethics and health policy in the Perelman School of Medicine at the University of Pennsylvania. She was the 1993 president of the Society for Clinical Trials and the 1999 President of the Eastern North American Region of the International Biometric Society.
Trudie Lang is a Professor of Global Health Research at the University of Oxford. She specialises in clinical trials research capacity building in low-resource setting, and helped to organise the trial for the drug brincidofovir during the 2014 Ebola virus outbreak.
Sodium oligomannate is a mixture of oligosaccharides isolated from the marine algae Ecklonia kurome that is used in China as a treatment for Alzheimer's disease (AD).
Dalzanemdor is experimental drug being investigated for the treatment of neurological disorders and cognitive impairment. It acts as a positive allosteric modulator of the NMDA receptor, whose activity is essential for learning, memory, and cognition. Dalzanemdor is an analog of the neurosteroid 24S-hydroxycholesterol.
Anti-amyloid drugs, also known as anti-amyloid antibodies (AAA), are a class of monoclonal antibodies developed to treat Alzheimer's disease. The first drug in the class to be developed, in the early 2000s, is bapineuzumab, but it did not show effectiveness in later-stage trials. The first drug to be approved by the US Food and Drug Administration (FDA) is aducanumab—in 2021.
