U.S. FDA panel backs BioMarin’s Morquio A Syndrome drug
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An experimental drug to treat Morquio A Syndrome, a rare genetic disorder that causes skeletal malformation and a variety of related lung, eye, ear and heart problems, should be approved, an advisory panel to the U.S. Food and Drug Administration concluded on Tuesday.
The 21-member panel voted overwhelmingly in favor of approval, saying the benefits of the drug, Vimizim, which is made by BioMarin Pharmaceutical Inc, outweigh its risks. The FDA is not obliged to follow the recommendations of its advisory panels but typically does so.
Morquio A Syndrome is characterized by a deficiency of an enzyme known as N-acetylgalactosamine-6-sulfatase, which causes excessive storage in the body of long chains of sugars known as glycosaminoglycans.
This build-up can lead to short stature and joint abnormalities that limit mobility and endurance. The disease can also cause hearing loss, eye problems and heart disease. Symptoms often appear before the age of five.
About 70 taken ill after California chemical plant leak
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About 70 people were taken ill after a sulphuric acid leak at a chemical company in California late on Monday, Los Angeles County Fire Department officials said.
People in the Carson area complained of throat and nose irritation and vomiting after being exposed to an “apparent sulphuric acid release in the air from a neighboring business,” Los Angeles County Fire Department spokesman Robert Diaz said.
Thirteen people were treated at local hospitals and later released, Fire Department official Phil Ulloa said. Others were treated at the scene.
Ulloa said the leak was caused by a scrubber machine malfunctioning at the Solvay chemical plant.
EU clears Roche drugs after probe into lax safety reports
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Europe’s main drugs regulator has found no new safety issues linked to Roche medicines after an investigation into the Swiss drugmaker, which was rapped last year for lax drug-safety reporting.
The European Medicines Agency (EMA) launched a probe into Roche in June 2012 after a routine inspection found it had failed to properly assess tens of thousands of cases of possible adverse drug reactions.
The investigation included 19 centrally approved drugs, several of which were for cancer. Roche is the world’s largest maker of cancer medicines, as well as producing drugs for viral infections and inflammatory diseases.
Without Sufficient Support, Community Health Centers Will Drop 1 Million Patients
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A new report by the Geiger Gibson/RCHN Community Health Foundation Research Collaborative at the George Washington University School of Public Health and Health Services (SPHHS) examines the impact of federal and state policy decisions on community health centers (CHCs) and their ability to continue providing primary care to the nation’s poorest residents. The report, “How Medicaid Expansions and Future Community Health Center Funding Will Shape Capacity to Meet the Nation’s Primary Care Needs,” estimates that under a worst-case scenario, the nation’s health centers would be forced to contract, leaving an estimated 1 million low-income people without access to health care services by the year 2020.
“Without continued support, community health centers will not be able to meet the rising demand for primary care in underserved parts of the United States,” said the lead author of the report, Leighton Ku, PhD, MPH, a professor of health policy and director of the Center for Health Policy Research at SPHHS. “Unless policymakers act now to support these centers, many low-income Americans will be left without the high-quality care that can prevent many expensive health conditions from developing in the first place.”
The authors analyzed two key policy issues - the level of federal grant funding and the expansion of Medicaid eligibility under the Affordable Care Act (ACA) - and examined the impact of various levels of support on health center capacity. The researchers found that with sufficient federal funding and Medicaid expansion in all states, health centers could nearly double their capacity by 2020. In contrast, health centers would have to curtail services and turn away patients if grant funding is limited, and Medicaid expansion is not broadened.
Innovative Experiment Aims to Boost Lung Transplants
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The pair of lungs sits inside a clear dome, gently inflating as doctors measure how well they’ll breathe if implanted into a patient who desperately needs a new set.
It’s a little-known twist of nature - your lungs can live on for a while after you die. The air left inside keeps them from deteriorating right away as other organs do.
An innovative experiment now aims to use that hour or more window of time to boost lung transplants by allowing donations from people who suddenly collapse and die at home instead of in a hospital.
“There aren’t enough lungs. We’re burying them,” said Dr. Thomas Egan of the University of North Carolina, Chapel Hill, who is leading the project. “It turns out your lungs don’t die when you do.”
Method to estimate LDL-C may provide more accurate risk classification
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Seth S. Martin, M.D., of the Johns Hopkins Ciccarone Center for the Prevention of Heart Disease, Baltimore, and colleagues developed a method for estimating low-density lipoprotein cholesterol (LDL-C) levels that is more accurate than the standard measure.
Low-density lipoprotein cholesterol is the primary target for treatment in national and international clinical practice guidelines. Conventionally, LDL-C is estimated by the Friedewald equation, which estimates LDL-C as (total cholesterol) - (high-density lipoprotein cholesterol [HDL-C]) - (triglycerides/5) in mg/dL. The final term assumes a fixed ratio of triglyceride levels to very low-density lipoprotein cholesterol (TG:VLDL-C) of 5:1. “Applying a factor of 5 to every individual patient is problematic given variance in the TG:VLDL-C ratio across the range of triglyceride and non-HDL-C levels,” according to background information in the study.
The researchers used a sample of lipid profiles obtained from 2009 through 2011 from 1,350,908 children, adolescents, and adults in the United States.
Drug shows early promise in treating seizures
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A study out today in the journal Nature Medicine suggests a potential new treatment for the seizures that often plague children with genetic metabolic disorders and individuals undergoing liver failure. The discovery hinges on a new understanding of the complex molecular chain reaction that occurs when the brain is exposed to too much ammonia.
The study shows that elevated levels of ammonia in the blood overwhelm the brain’s defenses, ultimately causing nerve cells to become overexcited. The researchers have also discovered that bumetanide – a diuretic drug used to treat high blood pressure - can restore normal electrical activity in the brains of mice with the condition and prevent seizures.
“Ammonia is a ubiquitous waste product of regular protein metabolism, but it can accumulate in toxic levels in individuals with metabolic disorders,” said Maiken Nedergaard, M.D., D.M.Sc., co-director of the University of Rochester Medical Center (URMC) Center for Translational Neuromedicine and lead author of the article. “It appears that the key to preventing the debilitating neurological effects of ammonia toxicity is to correct a molecular malfunction which causes nerve cells in the brain to become chemically unbalanced.”
In healthy people, ammonia is processed in the liver, converted to urea, and expelled from the body in urine. Because it is a gas, ammonia can slip through the blood-brain-barrier and make its way into brain tissue. Under normal circumstances, the brain’s housekeeping cells - called astrocytes - sweep up this unwanted ammonia and convert it into a compound called glutamine which can be more easily expelled from the brain.
Texting heart medication reminders improved patient adherence
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Getting reminder texts helped patients take their heart medicines (anti-platelet and cholesterol-lowering drugs) more regularly, according to research presented at the American Heart Association’s Scientific Sessions 2013.
In a 30-day, randomized controlled trial of 90 coronary heart disease patients, one group received customized text education messages and medication reminders; a second group got education messages only; and a third received no texts.
The text messaging groups had a 16 percent to 17 percent higher rate of taking correct doses and a higher rate of taking doses on schedule compared to the group who didn’t receive text messaging.
“There is now a major initiative to apply more innovative technologies such as mHealth, eHealth, and telehealth to effectively intervene to promote medication adherence,” said Linda Park, Ph.D., study lead author and post-doctoral fellow at San Francisco VA Medical Center in California.
Hybrid heart valve is strong, durable in early tests
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A hybrid heart valve created from thin and highly elastic mesh embedded within layers of human cells was strong and durable in a study presented at the American Heart Association’s Scientific Sessions 2013.
Researchers created a three-dimensional cell culture by coating a scaffold of nickel-titanium alloy (Nitinol), used for devices that require flexibility and motion, with layers of smooth muscle, connective tissue and lining cells. The valves performed well in a heart simulator, opening and closing under various pressures and remaining stable and strong throughout the tests.
New evidence on the biological basis of highly impulsive and aggressive behaviors
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Physical and chemical changes in the brain during development can potentially play a role in some delinquent and deviant behaviors, according to research released today. Studies looking at the underlying mechanisms that influence our ability to exercise self-control were presented at Neuroscience 2013, the annual meeting of the Society for Neuroscience and the world’s largest source of emerging news about brain science and health.
Understanding the impact of changes in specific prefrontal regions during brain development could lead to new treatments and earlier interventions for disorders in which impulsivity plays a key factor. The research may have implications for understanding and dealing with aggressive and troublesome behaviors.
Today’s new findings show that:
Common genetic pathway could be conduit to pediatric tumor treatment
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Investigators at Johns Hopkins have found a known genetic pathway to be active in many difficult-to-treat pediatric brain tumors called low-grade gliomas, potentially offering a new target for the treatment of these cancers.
In laboratory studies, researchers found that the pathway, called mammalian target of rapamycin (mTOR), was highly active in pediatric low-grade gliomas, and that mTOR activity could be blocked using an experimental drug, leading to decreased growth of these tumors.
“We think mTOR could function as an Achilles heel,” says study co-author Eric Raabe, M.D., Ph.D., an assistant professor of pediatrics, oncology and pathology at the Johns Hopkins Kimmel Cancer Center. “It drives cancer growth, but when mTOR is inhibited, the tumor falls apart.” The work was described Nov. 7 in the journal Neuro-Oncology.
Overall, brain tumors affect more than 4,000 children each year in the U.S., and they are the leading cause of cancer deaths in children, according to Raabe. Low-grade gliomas are the most common group of tumors of the central nervous system in children. Current treatments for these tumors include surgery and chemotherapy, which often cause significant side effects. Many of these tumors are located in areas like the optic pathway, where they can’t be easily removed by surgery without causing damage, including blindness. In addition to vision loss, some of Raabe’s patients have endured paralysis or learning problems as a result of the tumor or treatment. “Even though these tumors are considered ‘low grade’ and not particularly aggressive, many patients suffer severe, life-altering symptoms, so we desperately need better therapies,” says Raabe.
Sexual function dramatically improves in women following bariatric surgery, Penn study finds
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The first study to look extensively at sexual function in women who underwent bariatric surgery found that significant improvements in overall sexual function, most reproductive hormones and in psychological status were maintained over two years following surgery. Women reporting the poorest quality of sexual function prior to surgery saw the most dramatic improvements one year after surgery, on par with women who reported the highest quality of sexual function prior to surgery. The new report by researchers with the Perelman School of Medicine at the University of Pennsylvania appears in the November 4 edition of JAMA Surgery.
More than half of women who seek bariatric surgery report signs of sexual dysfunction and, consequentially, psychological stress.
“For many people, sex is an important part of quality of life. The massive weight losses typically seen following bariatric surgery are associated with significant improvements in quality of life,” said the study’s lead author David Sarwer, PhD, professor of Psychology in Psychiatry and Surgery in the Perelman School of Medicine at the University of Pennsylvania. “This is one of the first studies to show that women also experience improvements in their sexual functioning and satisfaction, as well as significant improvements in their reproductive hormones.”
Researchers followed 106 women with an average Body Mass Index of 44.5 who underwent bariatric surgery (85 had gastric bypass and 21 had gastric banding procedures). Following surgery, the women lost an average of 32.7 percent of their original body weight after the first year, and 33.5 percent at the end of the second year.
Genetic rarity rules in wild guppy population, study finds
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When it comes to choosing a mate, female guppies don’t care about who is fairest. All that matters is who is rarest.
Florida State University Professor Kimberly A. Hughes in the Department of Biological Science has a new study just published in the journal Nature that is the first to demonstrate a female preference for rare males using an experiment in a wild population, rather than a laboratory setting.
This study of genetic differences in male guppies is relevant to understanding variation in humans as well as in other organisms, Hughes said.
Hughes and her longtime collaborators studied guppies in Trinidad and found that male guppies with rare color patterns mated more - and lived longer - than the common males. The males’ color variations are genetic and not due to diet or temperature. And the males’ actual appearance didn’t matter to the females, who are tan in color and do the choosing of mates.
“No matter which color pattern we made rare in any group, they mated more and had more offspring,” Hughes said.
