There are no effective treatments for Alzheimer’s disease (AD), a progressive brain disease that slowly destroys a person’s memory, cognitive skills and ability to carry out the simplest tasks. AD affects more than 5 million individuals in the United States and ranks as the sixth leading cause of death. The ε4 allele of the apolipoprotein-E (APOE) gene is the strongest genetic risk factor for sporadic or late-onset AD. Heterozygous carriers of the ε4 allele are at three-to-four times greater risk; homozygous carriers are at ten times greater risk.
Over 32 million Americans are living with Diabetes and newly diagnosed cases of type 1 and type 2 diabetes is increasing. A defining feature of type 2 diabetes mellitus (T2DM) is the accumulation of islet amyloid polypeptide (IAPP) fibrils in pancreatic islets. Such accumulations form amyloid plaques, referred to as islet amyloidosis. Mounting evidence suggests that islet amyloidosis plays a causative role in the development and progression of ß-cell dysfunction in T2DM.
Transformation of Weak or Non-Immunogenic Antigens to Produce an Immune Response and Therapeutic Polypeptides for the Treatment and Prevention of Cancer
A significant challenge in developing therapies for the treatment and prevention of cancer has been the discovery, selection, and exploitation of antigens.
Fatty Acid Derivatives and Their Use for the Treatment and Prevention of Autoimmune, Inflammatory, and Pain Disorders
The discovery and selection of suitable compounds for the treatment and prevention of autoimmune, inflammatory, and pain disorders is a significant challenge. Researchers at National Institute of Aging (NIA) mitigated this issue. They discovered and synthesized numerous novel fatty acid derivatives (novel small molecules) that may ameliorate these conditions and provide treatment options for these disorders. In a relevant rat model, the fatty acid derivatives developed by NIA demonstrated:
Inflammatory processes associated with the over-production of tumor necrosis-alpha (TNF-alpha), a potent activator of the immune system accompany numerous neurodegenerative diseases. TNF-alpha has been validated as a drug target with the development of the inhibitors Enbrel and Remicade (fusion antibodies) as prescription medications. Both, however, are large macromolecules that require direct injection and have limited brain access.
Melanoma is a particularly aggressive form of cancer primarily caused by over-exposure to sunlight. Although melanoma can strike at any age, the malignancy disproportionately impacts persons of advanced age, as these individuals often have decades of repeated exposure to harmful levels of ultraviolet radiation. Scientists at NIH among others have clarified the link between advanced melanoma and other malignancies and expression of SPANX-B.
The biological phenomenon of RNA interference (RNAi) has much promise for developing therapeutics to a variety of diseases. However, development of RNAi therapies remains mainly in preclinical stages largely because of difficulties in delivering small inhibitory RNAs (siRNA) and short hairpin RNAs (shRNA) into target cells. Although viral vector-based siRNA delivery systems have been widely used, their specificity and safety remain significant issues. Without a solution to these delivery problems, RNAi cannot fulfill its therapeutic promise.
Hypertension is a condition characterized by persistent high arterial blood pressure. Hypertension may have no known cause (essential or idiopathic hypertension) or may be associated with other primary diseases (secondary hypertension), and is considered a risk factor for the development of heart disease, peripheral vascular disease, stroke, and kidney disease. Preeclampsia is a similar condition that occurs in 5-10% of all pregnancies (most common in first time pregnancies) and is characterized by high blood pressure, edema, and protein in the urine. Complications of preeclampsia includ
Polymer matrix-based sustained-release formulations have gained significant interest in recent years for both parenteral and delivery of small molecules as well as proteins and other biological molecules. Compared to conventional dosage forms, these delivery systems offer improved efficacy, reduced toxicity, convenience and improved patient compliance. Colloidal carriers such as microparticles are considered a promising approach for targeting drugs to specific organs that could permit the sustained release at the target site and reduce potential side effects.
Researchers at the National Institute on Aging Laboratory of Cardiovascular Science have developed a device for externally compressing or collapsing peripheral vasculature during cardiopulmonary resuscitation (CPR) to redirect blood to the torso and head regions, thereby enhancing the likelihood of CPR success. The system includes a plurality of sleeves adapted for placement on a patient's limbs during CPR, each sleeve including at least one inflatable fluid chamber and at least one inflation