Rare Diseases that People Suffer From - Term Paper Example

Summary of Articles The National Institutes of Health (NIH), which is the United States’ medical research agency, is the body that is usually in charge of many of the clinical research processes conducted on the many rare diseases that people suffer from. One of the diseases that the NIH in conjunction with other research bodies has been working on is Alzheimer’s disease. This is a disease that mostly affects elderly people whose signs and symptoms include memory loss, language problems, disorientation, mood swings and lack of self-care. Once a person is diagnosed with the disease, their estimated life span is three to nine years. There are no known medicines that completely cure the condition hence there is a need for research and trials of many different medications that could reduce the effects of the disease on the body. In working towards finding a drug that could control or eradicate Alzheimer’s, the NIH has been able to launch a portal for research on potential drugs. It supported research and experiments done on mice to test whether cancer drugs could restore brain functioning. Studies have determined that versatile proteins should be the new targets for the Alzheimer’s drugs. More research has been done to see the effect that allergy drugs have the disease and also on how drugs activate brain stem cells may reverse multiple sclerosis. NIH-led effort launches big data portal for Alzheimer’s drug On the 4th of March 2015 the NIH in conjunction with the National Institute on Aging (NIA), the Food and Drug Administration (FDA) industry, and academic scientists from other research organizations launched the data portal for Alzheimer’s drug in order to hasten the process of drug development. The opening of the portal is expected to make it easier for researchers and scientists studying the disease to be able to compare their findings and analyze other datasets. The researchers believe that this move will make it easier to develop predictive models of the disease and know the factors that lead to the development of the various signs and symptoms. The aim of the portal is to have a transparent, productive and reliable platform where different scientists and researchers can put their work and compare with the works of other scientists as they all work towards the goal of finding a drug for Alzheimer’s disease. According to Francis S. Collins, the NIH Director, the launch of the portal will enable the researchers to save time and money as well as come up with new diagnostics and effective therapies for the disease. He also says the aim of the portal was to employ open science in revolutionizing research and drug development process. The portal was developed by Sage Bionetworks and together with other scientists, bioinformatics, and drug experts will work hard and apply excellent analytical means to put together data from over two thousand post-mortem brains that have been studied. The portal is managed by the NIH, which is working together with NIA, National Institute of Neurological Disorders and Stroke, US Food and Drug Administration, four pharmaceutical companies and four non-profit groups studying Alzheimer’s. Other researchers in the program include researchers from Icahn School of Medicine, Broad Institute of MIT, Emory University, Mayo Clinic, University of California, Harvard University and the University of Florida. Repurposed Experimental Cancer Drug restores Brain Functioning Mouse Models of Alzheimer’s disease Scholars from Yale University school of Medicine led by a neurobiology researcher, neurologist and senior author of the study Stephen Strittmatter found that saracatinib, a drug initially developed and used to treat cancer could also be used to treat Alzheimer’s disease. This study was funded by the National Center for Advanced Translational Sciences, NIH, NIA, Bright Focus Foundation, Falk Medical Research Trust, and Alzheimer’s Association. It was published on 21st March and the driving force was to be able to establish new cure for Alzheimer’s as existing treatments only ease the symptoms but do not control the build-up of beta amyloid clamps in the brain. The scientists were able to establish that a protein called Fyn Kinase that is found in the brain played an important role in the working of beta amyloid to damage brain cells. Thus, they worked towards destroying this specific protein. The drug saracatinib directly targets this protein. It is an approved cancer drug that had been tried and tested in humans thus Strittmatter and his team did not need to go through the lengthy process of finding a new drug. In their experiment, they administered the drug to mice with similar symptoms as those of Alzheimer’s and noticed a change four weeks later. The mice synapse loss had been restored. Similar tests were conducted on humans to determine safety, effectiveness and ability of the human body to tolerate the drug. More tests are to be conducted on 152 participants for a year and results obtained in two years. Versatile proteins could be new target for Alzheimer’s Drugs Scientists led by Carla Shatz a renowned neurologist, professor of biology and neurobiology at Stanford University found that certain proteins that control the visual system development could be linked to the risk of Alzheimer’s in old age. While studying plasticity and focusing on ocular dominance, Dr. Shatz, and her team discovered that a protein linked to nerve cells could aid in the research on Alzheimer’s. The study was funded y the NIA, National Eye Institute, National Institute of Neurological Disorders and Stroke all part of the NIH. This specific protein is known as PirB in mice and a similar one in humans called LilrB2. Shatz and her team did an experiment on mice in order to see how exactly this protein works with the beta-amyloid protein in Alzheimer’s. They were able to find that the protein works as a receptor during the clamping process binding the amyloid proteins together in forming clumps. However when they depleted the PirB protein, they noticed that the clumping process did not occur. Generally mice experienced learning problems and memory problems in nine months but upon depletion of the PirB protein this did not happen. Since the PirB protein in mice has similar functions as the LilrB protein in humans, Dr. Shatz hopes that her team’s findings will trigger more researchers to look more into the subject. They should examine more drugs that could target blocking the functioning of this specific protein and in extension the working of the beta amyloid protein. Allergy drug inhibits Hepatitis C in Mice A study by the National Institutes of Health led by T. Jake Liang, M. D., Lead Investigator at NIH’s National Institute of Diabetes and Digestive and Kidney Diseases found that a drug usually prescribed for allergic reactions Chlorcyclizine HCl (CCZ) inhibits the Hepatitis C virus in mice. The study was conducted at the NIH campus in Bethesda Maryland and published on 8th April in Science Translation Medicine. The Hepatitis C virus causes liver cirrhosis as well as inflammation of the liver and in as much as it is a treatable disease, treatment is quite expensive. However, early diagnosis and treatment of the disease can prevent liver damage. T. Jake and his team used innovate, and throughput-screening processes and were able to establish that the use of CCZ limits the hepatitis virus activity in the body. Chlorcyclizine HCl worked by making it impossible for the virus to enter human liver cells in mice. The effects are likened to using antiviral drugs that do not have adverse side effects. The next step of the study is to find out how the use of the drugs affects humans especially when used to limit the working of the hepatitis virus. Liang cautions people to wait until it has been tested and proved that the use of the drug for the purpose of blocking entry of hepatitis c is approved before they use it for that purpose. If approved it just might be the cheaper solution to the virus. Drugs that activated brain stem cells may reverse multiple sclerosis Paul J. Tesar PhD., associate professor at Case Western Reserve School of Medicine in Cleveland, led an NIH and NINDS-funded study with an aim of finding a way to improve the body’s ability in repairing itself. They were able to identify two drugs; an antifungal and a steroid that could be used to treat multiple sclerosis. Multiple sclerosis is a condition brought about by breakdown of myelin-an insulator and transmitted between brain cells. This causes numbness, muscle weakness, vision problems and coordination and balance problems. As such, in order to reverse this body is required to repair myelin. As such, Dr. Tesar focused his study on finding a way to increase myelination. They tested mice and tried many different drugs to see if they could aid this process. While at it, they found two drugs miconazole (an antifungal) and clobetasol (a steroid that that stimulated the body to produce myelin). This opened up a new avenue for more research to be conducted on the two drugs. Dr. Tesar cautions that more research should be done before the two drugs are used for the treatment of multiple sclerosis. Works Cited Nih.gov.,. 'National Institutes Of Health (NIH)'. N.p., 2015. Web. 7 May 2015. Read More