Fact Sheets
Alzheimer’s Disease: One in 10 Americans over age 65 and nearly half of those over 85 suffer from Alzheimer’s disease. A potential medicine for Alzheimer’s disease has two mechanisms. It blocks the production of beta amyloid protein, which forms plaques in the brains of Alzheimer’s patients and is thought to be the cause of Alzheimer’s, and the medicine also inhibits the degradation of the neurotransmitter acetylcholine, which is thought to improve memory. A report by National Institutes of Health researchers indicates that the medicine may improve Alzheimer’s symptoms and may also interfere with the progression of the disease.
Diabetes: Diabetes affects about 16 million Americans. About half of all diabetes cases occur in people older than age 55, and more than 8.6 million Americans age 60 and older have diabetes. Uncontrolled diabetes can lead to kidney failure, blindness, amputations and premature death. A new, first-in-class medicine in development has been shown in clinical trials to significantly improve long-term glycemic control. The medicine blocks an enzyme (DPP-4) from inactivating a specific hormone (GLP-1) and thereby increases levels of the GLP-1 hormone which stimulates insulin production and reduces glucagon secretion. By increasing levels of GLP-1, the new medicine could improve the imbalance between insulin and glucose that causes type 2 diabetes. Protection of the pancreas and its ability to produce insulin is an additional potential long-term benefit of this medicine.
Macular Degeneration: Age-related macular degeneration (AMD) is the leading cause of irreversible visual impairment in the elderly. Early or late-stage AMD affects about 3.6 million American over age 70. AMD can either be wet or dry. In wet AMD, new blood vessels grow beneath the retina and leak blood and fluid, causing disruption and retinal dysfunction and creating blind spots. A new medicine in development for the wet form of AMD is a humanized therapeutic antibody fragment designed to bind and inhibit VEGF (vascular endothelial growth factor), a protein that plays a critical role in the creation of new blood vessels in the eye. The medicine works by blocking new blood vessel growth and leakiness. In early clinical trials, 94 percent of patients treated with the medicine experienced stable or improved visual acuity.
Parkinson’s Disease: Approximately one million Americans suffer from Parkinson’s disease (PD), and each year as many as 60,000 new cases are diagnosed – one every nine minutes. Currently available treatments for PD can diminish the symptoms of the disease but do not replace lost nerve cells or halt the disease’s progression. A nervous system growth factor gene therapy being studied aims to deliver the growth factor, glial cell line derived neurotrophic factor (GDNF), into the brain to prevent degeneration of nerve cells and the loss of the neurotransmitter dopamine. Early studies are providing hope that this new research will result in new treatments for PD.
Rheumatoid Arthritis: Rheumatoid arthritis affects 2.1 million Americans, most of whom are women. In this autoimmune disorder, a person’s joints become painful, swollen, stiff, and, in severe cases, deformed. One medicine currently in clinical trials targets immune cells, B cells, which are believed to play a role in the progress of the disease, by binding to the CD20 antigen on the cell’s surface. The medicine then recruits the body’s natural defenses to destroy the marked B cells. Not all healthy B cells have the CD20 antigen, allowing healthy B cells to regenerate after treatment and return to normal levels within several months. According to recent trial results, use of this medicine could improve the symptoms of rheumatoid arthritis.

