EDITOR'S NOTE: THIS APPROVAL IS BEYOND HUGE AS WE CAN NOW START TREATING THE ROOT CAUSE OF DISEASES RATHER THAN THEIR ORGAN (e.g. Colon Cancer)
This is a must for those interested in clinical trials of the future.
Cystic fibrosis is a rare disease that affects about 30,000 people in the United States and affects the cells that produce mucus, sweat and digestive juices. These secreted fluids are normally thin and slippery due to the movement of sufficient ions (chloride) and water in and out of the cells. People with the progressive disease have a defective cystic fibrosis transmembrane conductance regulator (CFTR) gene that can't regulate the movement of ions and water, causing the secretions to become sticky and thick. The secretions build up in the lungs, digestive tract and other parts of the body leading to severe respiratory and digestive problems, as well as other complications such as infections and diabetes.
The fight to contain the 2013-16 Ebola outbreak in West Africa was hampered by the lack of an effective treatment or vaccine. Now, according to an article published in the journal Cell (May 2017), researchers have studied the blood of an Ebola survivor, searching for human antibodies that might effectively treat not only people infected with Ebola virus, but those infected with related viruses as well. Two such antibodies have been identified that hold promise as Ebola treatments.
Despite cross-sectional evidence linking racial residential segregation to hypertension prevalence among non-Hispanic blacks, it remains unclear how changes in exposure to neighborhood segregation may be associated with changes in blood pressure. As a result, a study published on line (15 May 2017) in JAMA Internal Medicine, was performed to examine the association of changes in neighborhood-level racial residential segregation with changes in systolic and diastolic blood pressure over a 25-year period.
Last week, Target Health was honored as Dr. Jules Mitchel joined Dr. John Laschinger (FDA), for a Clinical Trials Transformation Initiative (CTTI) Webinar on CTTI's recommendations on how to design and use registries for prospective clinical trials. The Webinar attracted more than 200 participants and the questions from the attendees were all "on target." According to the CTTI website, "CTTI's recommendations for registry assessment and design can assist in making embedded clinical trials suitable for regulatory purposes. By using registries as a reusable platform for evidence generation, we can improve the efficiency of clinical trials and bring new treatments to patients faster."
Jan Evangelista Purkyne (Czech: also written Johann Evangelist Purkinje) (1787-1869) was a Czech anatomist and physiologist. He was one of the best known scientists of his time. In 1839, he coined the term ?protoplasm' for the fluid substance of a cell. His son was the painter Karel Purkyne. Such was his fame that when people from outside Europe wrote letters to him, all that they needed to put as the address was "Purkyne, Europe". He is buried in the Czech National Cemetery in Vysehrad, Prague, modern-day Czech Republic.
Uveitis is an inflammatory disease of the eye and the fifth leading cause of vision loss in the United States. Concerns about potential adverse effects of systemic corticosteroid and immunosuppressive therapy drove the development of an intraocular implant to treat uveitis locally. The fluocinolone intraocular implant, developed by Bausch & Lomb, was approved by the FDA in 2005. Early data suggested the implant was effective at controlling inflammation but had local ocular side effects. The Multicenter Uveitis Steroid Treatment Trial (MUST) was undertaken to evaluate whether the implant treatment was an improvement over systemic therapy for management of uveitis.
Enterococci bacteria are the bane of hospitals, causing thousands of multidrug-resistant infections in patients each year. Now, according to an article published in the journal Cell (11 May 2017), evidence of the bacteria's evolutionary history can be traced back to 425 million. The goal of the study was to understand why, among the vast diversity of gut flora, enterococci are so well adapted to the modern hospital environment. Results showed that based on molecular clock estimation, together with analysis of their environmental distribution, phenotypic diversity, and concordance with host fossil records, place the origins of the enterococci around the time of animal terrestrialization, 425-500 mya. Speciation appears to parallel the diversification of hosts, including the rapid emergence of new enterococcal species following the End Permian Extinction. Major drivers of speciation include changing carbohydrate availability in the host gut. Life on land would have selected for the precise traits that now allow pathogenic enterococci to survive desiccation, starvation, and disinfection in the modern hospital, foreordaining their emergence as leading hospital pathogens
An estimated 1 in every 2,500 babies in the U.S. is born with esophageal atresia. Babies with this condition cannot feed normally, and they require a feeding tube until surgery can be performed to attach the esophagus to the stomach. Most babies born with esophageal atresia also have a tracheoesophageal fistula, which also needs to be repaired surgically, since fluids from the esophagus can get into the airways and interfere with breathing.
A study recently reported the Annals of Neurology (2017;81:549-559), was performed to determine clinical and neuropathological outcomes following a clinical diagnosis of mild cognitive impairment (MCI). For the study, data were drawn from a large autopsy series (N=1,337) of individuals followed longitudinally from normal or MCI status to death, derived from 4 Alzheimer Disease (AD) Centers in the United States.
Benjamin Franklin took a great interest in health-related topics. In his day, many beliefs about health and disease were based on superstition. Franklin applied Enlightenment reasoning to his study of various afflictions and came up with some astonishingly accurate hypotheses. Here are just a few of Franklin's theories and accomplishments in the fields of health, physical fitness, and medicine.
Late infantile neuronal ceroid lipofuscinosis type 2 (CLN2), also known as tripeptidyl peptidase-1 (TPP1) deficiency, is one of a group of disorders known as neuronal ceroid lipofuscinoses (NCLs), collectively referred to as Batten disease. CLN2 disease is a rare inherited disorder that primarily affects the nervous system. In the late infantile form of the disease, signs and symptoms typically begin between ages 2 and 4. The initial symptoms usually include language delay, recurrent seizures (epilepsy) and difficulty coordinating movements (ataxia). Affected children also develop muscle twitches (myoclonus) and vision loss. CLN2 disease affects essential motor skills, such as sitting and walking. Individuals with this condition often require the use of a wheelchair by late childhood and typically do not survive past their teens. Batten disease is relatively rare, occurring in an estimated two to four of every 100,000 live births in the United States.
According to an article published in the New England Journal of Medicine (April 19, 2017), adjusting the frequency of eye screenings for people with type 1 diabetes based on their risk of severe eye problems would result in fewer eye exams at lower cost and quicker diagnosis and treatment of advanced retinopathy, which can otherwise lead to vision loss. The findings are the latest from an ongoing study funded for more than 30 years by the National Institutes of Health.
During mid-to-late gestation, neurons migrate from deep brain structures to their appointed places and organize themselves into the key working tissue of what will become the human cortex, the outer layer of the brain and seat of higher-order mental functions. This building process is complex and especially vulnerable to genetic and environmental insults that can set the stage for autism, schizophrenia, and other neurodevelopmental brain disorders.
Editor's note: The evolution of the cell is one of Science's most awesome areas of study, leading to the origins of life itself. It is beyond our comprehension, why anyone able to contribute to the funding of research, inquiring into the mystery of life, and its pathologies, which cell biology does, why anyone would not be eager to do so. Perhaps Americans should vote to require that all politicians have an education high enough to enable them to understand the worlds of science, math, technology, engineering, and all the arts (which remind us of our humanity).
Problem: How to establish a vascular system that delivers blood deep into the developing tissue. Solution: Scientists have turned to the vascular system of plants to solve a major bioengineering problem blocking the regeneration of human tissues and organs, by successfully culturing beating human heart cells on spinach leaves that were stripped of plant cells.