En Español | Members Area

Policy Papers

BACKGROUNDER: SELECTED MEDICINES IN DEVELOPMENT FOR NEUROLOGIC DISORDERS

Alzheimer’s disease: One in 10 Americans over 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 it inhibits the degradation of the neurotransmitter acetylcholine. A report by researchers at the National Institutes of Health indicates that the medicine may be able not only to improve the symptoms of Alzheimer’s patients but may also interfere with the progression of the disease.

Glioblastoma (Brain Cancer): Brain cancers are among the most difficult cancer to treat. About 40,900 primary brain tumors are diagnosed in the U.S. each year, and more than 17,000 of these are high-grade gliomas, a highly invasive form of cancer that sends cancerous cells throughout the brain and spinal cord. A potential weapon against this disease is a new drug that singles out and destroys the malignant cells without harming the healthy cells.

Insomnia: More than one-half of adults in the United States report trouble sleeping a few nights each week, while 35 percent of the adult population report they have experienced insomnia every night or almost every night within the past year. One potential medicine binds more selectively than currently marketed products at the specific subtype of GABA-A receptors within the brain believed to be responsible for promoting sleep.

Parkinson’s disease: Approximately 500,000 to one million Americans suffer from Parkinson’s disease, and 50,000 new cases are diagnosed each year. A potential new medicine for Parkinson’s is a novel cell therapy that utilizes normal human cells attached to microcarriers to enhance brain levels of dopamine, the neurotransmitter deficient in Parkinson’s patients. The cells, retinal pigment epithelial cells, produce L-DOPA, a natural precursor of dopamine. The microcarriers, to which the cells are attached, enable long-term cell survival. The medicine is injected into the brain regions lacking dopamine using a surgical technique called sterotactic injection, which does not require general anesthesia.

Spinal Cord Injury:
An estimated 11,000 new spinal cord injuries occur each year in the United States. One potential medicine for chronic spinal cord injury (SCI) improves impulse conduction in nerve fibers in which the insulating layer of the spinal cord, called myelin, has been damaged. The medicine has shown to improve spasticity in people with chronic SCI. This medicine is also being tested in multiple sclerosis patients to improve walking ability and muscle strength.

Stroke: Stroke is the third leading cause of death in the United Sates after heart disease and cancer. Each year, about 700,000 Americans experience a new or recurrent stroke. One potential medicine has shown to preserve function and brain tissue, which is irreversibility damaged after acute stroke, when it is administered immediately after the onset of permanent ischemic in a model of acute stroke.