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Showing posts with label Up to Date. Show all posts
Showing posts with label Up to Date. Show all posts

Thursday, May 30, 2019

Novartis $2 million gene therapy for rare disorder is world's most expensive drug


Novartis $2 million gene therapy for rare disorder is world's most expensive drug


Swiss drugmaker Novartis on Friday won U.S. approval for its gene therapy Zolgensma for spinal muscular atrophy (SMA), the leading genetic cause of death in infants, and priced the one-time treatment at a record $2.125 million.The Food and Drug Administration approved Zolgensma for children under the age of two with SMA, including those not yet showing symptoms. The approval covers babies with the deadliest form of the inherited disease as well as those with types where debilitating symptoms may set in later.“This is potentially a new standard of care for babies with the most serious form of SMA,” said Dr. Emmanuelle Tiongson, a pediatric neurologist at Children’s Hospital Los Angeles who has provided Zolgensma to patients under an expanded access program. “The job now is trying to negotiate with insurers that this would be a long-term savings.”Novartis executives defended the price, saying that a one-time treatment is more valuable than expensive long-term treatments that cost several hundred thousand dollars a year.

Friday, August 24, 2018

Gut enzymes convert donor blood to much-needed universal type O


TORONTO — Canadian researchers believe they have found the means to convert any type of blood into universally usable group O with enzymes found in the human gut — a finding that could expand the pool of potential blood donors and make blood-matching easier and safer.
For transfusions to be safe, blood from a donor — for instance, A, B or AB types — must match that of a patient. O-type blood can be transfused into anyone and is always in high demand.
"Blood type is determined by the presence of antigens on the surface of red blood cells; type-A blood has the A antigen, B has the B antigen, AB blood has both antigens and O blood has none," said lead researcher Stephen Withers, a professor of chemistry and biochemistry at the University of British Columbia.
"Antigens can trigger an immune response if they are foreign to the body, so transfusion patients should receive either their own blood type, or type O to avoid a reaction," he said. "That's why O blood is so important."
Withers said the UBC team sampled DNA from millions of microorganisms found in various environmental samples to find one in which the desired enzymes might be found.
The researchers then turned their attention to the mucosal lining of the human gut — which contains sugars similar in structure to blood antigens — by extracting bacterial DNA from fecal samples.
"By homing in on the bacteria feeding on those sugars, we isolated the enzymes the bacteria use to pluck off the sugar molecules," said Withers, adding that researchers then used E. coli bacteria as "little factories" to produce quantities of the enzymes "and found that they were capable of performing a similar action on blood antigens.
"So we just simply add them to the red blood cells, they attach themselves to the surface of the red blood cell and then they cut the sugar off," he said Wednesday in an interview from Boston, where the research was presented at this week's American Chemical Society annual meeting.
Scientists have been studying the use of enzymes to modify blood since 1982, said Withers. "However, these new enzymes can do the job 30 times better."
The UBC team focused on transforming A-type blood into O-type. Enzymes to snip sugars off the surface of B-type blood cells had already been identified, so using both groups of enzymes together could convert AB blood to O, he noted.
Withers and his colleagues plan to apply for a patent on the newly identified enzymes and are set to work with Canadian Blood Services and the Centre for Blood Research to test them on different types of blood from a variety of donors.                 
"The next step is very much all about safety," he said. "There are further tests we need to do to make sure that in the process we've not inadvertently changed anything else on the red blood cell surface which could be deleterious to its function."
One advantage of these enzymes is that they work in whole blood, not just components of blood, meaning that donations could be quickly converted to universal type O, he suggested.
"So I could imagine that what could happen is it could be added to blood when it's donated and just left sitting there to do its conversion while this stuff is being stored."
Canadian Blood Services said 46 per cent of Canada's population has group-O blood, while 42 per cent is group A, nine per cent group B, and three per cent group AB.
"One of the main challenges with maintaining an adequate blood supply in developed countries is that the use of group O is not in proportion to the incidence of that blood group in the population," said CBS chief scientist Dr. Dana Devine, who called the blood-converting enzymes a potential "game-changer."
"This imbalance arises because group O blood can be transfused to any recipient and is used to treat patients in emergency settings when there is no time to determine the patient's actual blood type," she said in a statement. While it would be unnecessary to modify all non-O blood units, Devine said the technology will be important in settings where there are anticipated shortages of group O, including Canadian summers marked by increased motor vehicle accidents, as well as hurricane season in the Caribbean and troop deployments to combat zones.
Expanding global blood supply is critical in light of growing populations and the frequency of natural disasters, Withers agreed.

Sheryl Ubelacker, The Canadian Press

Sunday, July 22, 2018

Boys should be given HPV jab, says vaccine committee. Here’s what happens next.


It’s nearly a decade since the human papillomavirus (HPV) vaccine was first introduced in the UK to help protect against the virus that causes most cases of cervical cancer. But until now, it has only been routinely offered to girls.Today, the Joint Committee for Vaccination and Immunisation (JCVI) recommended that adolescent boys should now also receive the vaccine.When and how this will happen is now down to the Government. But the recommendation comes from years of mounting evidence around likely health benefits and overall cost effectiveness.

HPV and cancer

HPV is a big family of viruses. There are more than 100 different types and some are more dangerous than others. While some low-risk types cause growths like warts or verrucas, there are thirteen high-risk types that are linked to cancer.HPV is very common, with 8 in 10 people infected at some point in their life. Usually our bodies clear the infection without it causing any problems. But in some cases a lasting infection can lead to cancer.This is because the virus damages the infected cells’ DNA and causes them to start dividing out of control, setting them on the road to cancer.Thanks to the vaccination programme, many people know that HPV causes cervical cancer – in fact it’s linked to all cases of the disease in the UK. But HPV is linked to other cancers too – including anal, penis and some types of mouth and throat cancer.

Why wasn’t the HPV vaccine always available for boys?

The HPV vaccine protects against 4 types of HPV. Two are linked to cancer: HPV 16 and 18, which together cause around 7 in 10 cervical cancer cases in the UK. The vaccine also protects against HPV 6 and 11, which cause most genital warts.The vaccine has been available to girls in the UK since 2008. It was initially only recommended for girls as the strongest evidence of health benefits and cost effectiveness was for cervical cancer and genital warts.Since the vaccine was introduced, we’re starting to see HPV infections in people who have been vaccinated falling. This suggests the vaccine is preventing HPV infection and, in the future, this should prevent cervical cancers.But HPV is linked to cancers in men as well as women.Men who have sex with women will get some protection from the current vaccination programme if their partner is vaccinated. The same can’t be said for adult men who have sex with men .In 2015, the JCVI, which advises UK health departments on vaccines, recommended extending vaccination to adult men who have sex with men. This group of men are at a higher risk of anal cancer. Up to the age of 45, these men can request HPV vaccination at sexual health clinics.But up until today, the programme hadn’t been recommended for boys, as the JCVI weren’t convinced it would be cost-effective.Today’s decision brings the UK in line with other countries including the US and Australia, which already offer the vaccination to boys.

Who will be offered the vaccine?

The JCVI has recommended the vaccine for boys aged 11-13, similar to the vaccination programme for girls. HPV vaccination is most effective in people who haven’t ever had an HPV infection. And as HPV is mostly transmitted through close sexual contact, vaccination is offered at a young age when people are unlikely to have had any sexual experiences.Men above the vaccination age who don’t have sex with men won’t be offered the vaccine. But it’s important to remember that most people clear HPV infections without them causing any symptoms or problems. And for most cancers linked to HPV there are also other ways to reduce your risk through things like not smoking or drinking less alcohol.

What happens now?

The recommendation for a gender neutral vaccination programme for adolescents has been years in the making. The next step is for the Government to formally accept the recommendation and extend the programme to boys.Until it does, we won’t know the details of when and how the programme will be rolled out. Once they have accepted the recommendation, the Government must publish a plan and timetable for the roll-out.This will need to be accompanied by more details on the programme itself. When the vaccine was first offered to girls in the UK, a ‘catch-up’ programme was introduced for girls up to the age of 18, and we want the Government to do the same for boys.Finally, the programme will do nothing if people aren’t aware it’s happening. We want to see a national awareness campaign to clearly communicate about the vaccine and its potential benefits, as well as new information for parents and boys.By offering the vaccine to everyone aged 11-13, the number of cases of HPV, along with the cancers they cause, could be dramatically reduced in the future.

Friday, March 9, 2018

ACP Recommends Less-Intensive HbA1c Target for T2D No proof of benefit for targets below 7%, new guidelines say




New type 2 diabetes guidelines from the American College of Physicians (ACP) recommend less-intensive blood sugar control for most patients, with a glycated hemoglobin (HbA1c) target between 7% and 8%.
"Studies have not consistently shown that intensive glycemic control to HbA1c levels below 7% reduces clinical microvascular events, such as loss or impairment of vision, end-stage renal disease, or painful neuropathy, or reduces macrovascular events and death," said first author Amir Qaseem, MD, PhD, ACP vice president for clinical policy, and colleagues.
To develop the new recommendations, the authors evaluated six sets of current guidelines from other organizations and reviewed five important clinical trials on which those guidelines are based. The updated guidance, published online in Annals of Internal Medicine, offers four key statements:
  • Statement 1: Clinicians should personalize goals for glycemic control in patients with type 2 diabetes on the basis of a discussion of the benefits and harms of pharmacotherapy, patients' preferences, patients' general health and life expectancy, treatment burden, and costs of care
  • Statement 2: Clinicians should aim to achieve an HbA1c level between 7% and 8% in most patients with type 2 diabetes
  • Statement 3: Clinicians should consider de-intensifying pharmacologic therapy in patients with type 2 diabetes who achieve HbA1c levels less than 6.5%
  • Statement 4: Clinicians should treat patients with type 2 diabetes to minimize symptoms related to hyperglycemia and avoid targeting an HbA1c level in patients with a life expectancy less than 10 years due to advanced age (80 years or older), residence in a nursing home, or chronic conditions (such as dementia, cancer, end-stage kidney disease, or severe chronic obstructive pulmonary disease or congestive heart failure) because the harms outweigh the benefits in this population
Qaseem et al reviewed and scored currently available guidelines using the AGREE II(Appraisal of Guidelines for Research and Evaluation II) instrument. This tool asks 23 questions about central aspects of recommendations, including their scope and purpose, stakeholder involvement, rigor of development, clarity of presentation, applicability, and editorial independence. The authors scored each guideline independently, and then compared the scores.
The two lowest-scoring guidelines were from the American Association of Clinical Endocrinologists/American College of Endocrinology (AACE/ACE) and the American Diabetes Association (ADA). Both scored lowest on stakeholder involvement, applicability, editorial independence, and scientific rigor.
"Several factors were important in considering guideline quality," the authors said. "For example, although many guidelines described benefits, adverse effects, and the strength and limitations of evidence or linked the evidence to the recommendation, they often inadequately described how they had considered or weighted these factors in developing the final recommendations. The guidelines frequently relied on selective reporting of studies or outcomes and focused on relative versus absolute effects and asymptomatic surrogate measures rather than patient-centered health outcomes."
The five clinical trials reviewed were:
  • ACCORD (Action to Control Cardiovascular Risk in Diabetes)
  • ADVANCE (Action in Diabetes and Vascular Disease: Preterax and Diamicron Modified Release Controlled Evaluation)
  • VADT (Veterans Affairs Diabetes Trial)
  • Both UKPDS (United Kingdom Prospective Diabetes Study) trials
"Collectively, these trials showed that treating to targets of 7% or less compared with targets around 8% did not reduce death or macrovascular events over about 5 to 10 years of treatment but did result in substantial harms, including but not limited to hypoglycemia," the authors wrote. "No trials show that targeting HbA1c levels below 6.5% in diabetic patients improves clinical outcomes, and pharmacologic treatment to below this target has substantial harms."
The ACP recommendation to de-escalate therapy in patients who achieve HbA1c levels less than 6.5% is notably different from other guidelines, said David Lam, MD, of Icahn School of Medicine at Mount Sinai in New York City, in a statement. "This may change how some providers care for patients who are able to achieve this level of control. However, it is important to also consider what potential impact the subsequent increase in HbA1C level may have on the patient's quality of life and their perception of overall health."
The recommendation against a specific HbA1C target in patients with limited life expectancy and multiple comorbidities is another difference, he continued. "While many providers likely already adjust their A1C goals in this subset of patients, this guideline may further change the care in this group of patients."
Qaseem and co-authors concluded: "The ACP believes that clinicians should reevaluate HbA1c levels and revise treatment strategies on the basis of changes in the balance of benefits and harms due to changed costs of care and patient preferences, general health, and life expectancy."

Sunday, July 17, 2016

Novel Therapeutic Advances for Management of Diabetes Mellitus




Diabetes mellitus (DM) has become one of the most challenging health problems in the 21st century. It is a serious public health problem globally, but the good news is that various advances are being made in prevention, detection, and treatment of diabetes. Though prevention is always better than cure, yet sometimes prevention is not possible and the cure seems to take ages. The interest to find a possible cure for diabetes has eventually led to exploration of various new scientific areas of research.
Volanesorsen
Volanesorsen (formerly ISIS-APOCIIIRx)is an antisense drug in development intended to treat patients with severely high triglycerides either as a single agent or in combination with other triglyceride-lowering agents.Elevation in triglycerides has always increased the risk of cardiovascular disorders as well as type 2 diabetes mellitus (T2DM). Researchers from the University of Pennsylvania have found the therapeutic effect of lipid-lowering medication sin improving insulin sensitivity in people with T2DM. Volanesorsen is an oral medication that can help to control the blood sugar levels. Itis believed to improve the insulin sensitivity and significantly decrease the patients’ overall glycated hemoglobin (HbA1c). After taking the medication for 12 weeks, researchers found that adult T2DM patients receiving volanesorsen experienced 69% reduction in triglycerides, and 57% improvement in whole-body insulin sensitivity.Researchers concluded that decrease in triglycerides was strongly related to improved insulin sensitivity and improved HbA1c.
PEGylated Lispro
PEGylated lispro (LY2605541) has been developed by the addition of polyethylene glycol (PEG) to insulin lispro, leading to its extended half-life. Early phase evaluations of PEGyalted lispro confirm its non-inferiority to glargine with a potential weight advantage when used in T2DM. Since insulin therapy is anabolic and its initiation is commonly associated with weight gain, an insulin preparation associated with a beneficial weight profile would represent a particularly valuable therapeutic entity.
Pre-clinical studies indicate that LY2605541 has low mitogenic potency, exerting a preferential hepatic effect on glucose homeostasis. However, LY2605541 appears to cause elevated transaminases and derangement of lipid profiles. On the basis of these initial observations, LY2605541 requires further extensive clinical evaluation to fully assess its risk/benefit profile in the management ofdiabetes.
Islet Transplantation
A novel technology has been developed to help treat type 1 diabetes mellitus (T1DM) and potentially negate the need for insulin injections. Islets are organoids that produce multiple hormones, including insulin, and donated islets are already treating severe cases of T1DMeffectively. Islet transplantation can transform the lives of T1DM patients, and may result in long-term freedom from insulin injections with excellent glucose control.
Natural Products
Plants possessing antidiabetic properties may be suitable as an adjunct to the existing therapies or as a prospective source of new hypoglycemic compounds. Many new bioactive drugs isolated from plants demonstrate antidiabetic activity equal to known oral hypoglycemic agents such as tolbutamide and chlorpropamide. Several plant species with terpenoids, flavonoids, phenols, coumarins, and other bioactive constituents have shown their potency in reducing blood glucose levels. Plants likeAllium sativum(Liliaceae),Gymnema sylvestre(Asclepiadaceae),Murraya koenigii(Rutaceae),Allium cepa(Liliaceae),Withania somnifera (Solanaceae), andFerula foetida (Umbelliferae) have exhibited antidiabetic properties when assessed in experimental models of diabetes. These traditional approaches might prove to bebeneficial in treatment of diabetic complications.
Great strides have been made clinically in the management of diabetes. Extensive research may lead to discovery of newer and better options for diabetes. With new technologies revolutionizing the treatment possibilities, the search for an effective medication is not far ahead.
Turacoz Healthcare Solutions aims to create awareness for such healthcare issues like diabetes mellitus which pose a serious threat to people, irrespective of race, gender, or age. We consider it our corporate responsibility to share the knowledge related to therapeutic research and development, and keep you updated with the recent medical advancements in the healthcare industry.“Being aware of a disease and its deleterious consequences”is the first-step towards its prevention. So stay healthy and stay long.

Saturday, March 19, 2016

FIRST PATIENT IN THE WORLD BRIDGED TO A SUCCESSFUL HEART TRANSPLANT VIA A TOTAL ARTIFICIAL HEART.

A petite 44-year-old woman has become the first patient in the world to be bridged to a successful heart transplant, that is, to go from needing a transplant to receiving one, with an experimental Total Artificial Heart designed for smaller patients.  The UCLA patient received a successful heart transplant at Ronald Reagan UCLA Medical Center, thanks to the smaller Total Artificial Heart.
The team explain that the 50cc SynCardia temporary Total Artificial Heart is a smaller investigational version of the larger 70cc SynCardia heart, which was approved for use in people awaiting a transplant by the Federal Food and Drug Administration in 2004 and has been used by more than 1,440 patients worldwide.  They go on to add that the 50cc device is designed to be used by smaller patients, including most women, some men and many adolescents, with end-stage biventricular heart failure, where both sides of the heart are failing to pump enough blood to sustain the body. The device provides mechanical support until a donor heart can be found.
Nemah Kahala, a wife and mother of five, was transferred to UCLA from Kaiser Permanente Los Angeles Medical Center in March. She was suffering from restrictive heart muscle disease and in critical condition. Her heart failure was so advanced that repair surgery and other mechanical assist devices could not help.  Nemah was placed on a life support system called extra corporal membrane oxygenation, however, the team note that this only works for about 10 days before a person’s organs begin to deteriorate.
With the clock ticking, doctors needed to buy time by replacing Nemah’s failing heart with an artificial heart while she waited for a heart transplant. Her chest cavity was too small for her to receive the larger 70cc artificial heart. However, under a one-time emergency use permitted under FDA guidelines, her doctors were able to implant the experimental 50cc device.
Her surgeons explain that Mrs. Kahala’s condition was deteriorating so rapidly that she would have not survived while waiting for a transplant. The team were therefore grateful to have this experimental technology available to save her life and help bridge her to a donor heart.
The researchers state that the artificial heart provides an immediate and safe flow of blood to help vital organs recover faster and make patients better transplant candidates.  After the two-hour surgery to implant the artificial heart, Nemah remained hospitalized in the intensive care unit and eventually began daily physical therapy to help make her stronger for transplant surgery.
Two weeks after the total artificial heart surgery, she was strong enough to be placed on the heart transplant list. After a week of waiting, a donor heart was found.
In addition to the high-tech medicine that kept the patient alive, Mrs. Kahala and her family have exemplified how a solid support system that includes loved ones and a compassionate medical team practicing what we at UCLA have termed ‘Relational Medicine’ plays an important role in surviving a medical crisis.
Since 2012, the UCLA Heart Transplant Program has implanted eight 70cc SynCardia Total Artificial Hearts. UCLA also participated in the clinical study of a 13.5-pound Freedom portable driver, a backpack-sized device that powers the artificial heart, allowing the patient to leave the hospital , that received FDA approval on June 26, 2014.
Nemah was discharged from UCLA on April 18. She is grateful to be home in Riverside with her family, who own a grocery store in the city of Orange.

The SynCardia Total Artificial Heart, at left, and a human heart, at right. Credit: SynCardia 2015


Source:  UCLA university of California 

THE TOP TEN HEALTH INNOVATIONS OF 2014.



A great year for health innovations with exciting discoveries, verification, validations and breakthroughs.  The big disease areas are still cancer, heart disease, diabetes and Alzheimer’s, however, it’s the methodology of detection, imaging, and the descriptive diagnostics that have been innovated this year.  In some cases these disciplines and techniques bleed through to multiple disease areas as other teams pick up the technology.  A common thread running through all of these areas however, is the all important biomarker.  And with each newly discovered biomarker comes the benchmark and standardisation. A new receptor, more inroads in stem cell technology and regenerative medicine, all leading back to control and revamping of our own DNA and a big push in immunology.
We saw the definitive breakthrough that is precision genetics with the evolution of personalized medicine into precision medicine; becoming more precise and honed, moving into master regulator genes as opposed to the confusing multiple loci and the personal immunity simulation of the past.  The medical community theorizing and validating that the mass of loci being targeted were in fact the resulting cascade of these precisely hit master genes.  This big push in genetics with researchers giving clarification, gaining a clear direction in sequencing and beginning to classify epigenetic methylation.
With epigenetic methylation research teams are now beginning to map and classify cancer via the epigenome. Reaching the source of all disease, that moment of mutation, the moment of epigenetic methylation; that clear crystalline moment when disease is born.  Medical teams around the world have summised that all disease comes from nuclei, is ordered by the gene and transcribed through the MiRNA; still an important area, to the moment of epigenetic methylation.  The moment that our DNA is changed forever and the cascade of disease-causing loci and proteins begin.
Health innovators have also taken a step back even further in the source and began to chip away at the facade of neurogenetics.  The genetic code held in our brain, the regulator of not only diseases and disorders of the brain but also  disease within the body.  This leads on nicely to the great inroads being made in neuroinnovations, standardised and medicalised, this area holds many answers to the questions the medical community have been asking for decades.  When we finally map every circuit, every pathway, all neurogenetics, it is expected to lead to the man-made regulation of all disease.  And with neuroscience this need not always be invasive.
Neuroinnovations have been astounding this year with multiple validated studies medicalising diagnostics, representing disease of the brain in terms in which all disease should be presented, in scans, in blood work, in genetic terms.  We have also witnessed a big drive to uncover more about the role the lesser-known cells, such as Astrocytes, play in the brain as well as the surrounding microglia in the hope this will lead to a crack in the looming blood-brain-barrier.  And most importantly the medical community has been given more control over the disorders we now understand to be more than emotive figments of imagination; more control to help with tangible and very real mental disorders which have now been imaged, seen with the naked eye in blood work and designated the correct biomarkers.
So, what were the biggest healthinnovations of 2014? What did health innovators concur on this year?  Which disease areas, disciplines and techniques were of the most interest to health innovators across the globe and were seen as the biggest breakthroughs, raising the most interest?
So here you go health innovators, here are the Top 10 Healthinnovations of 2014 based on the total number of views and shares:
1.  At number one we have the Karolinska Institutet of Sweden, whose breakthrough
neuroscientific human study lays the foundation for neurogenetic study in neuroplasticity.  This study identified the cells responsible for the superior plasticity of the human brain, the oligodendrocyte otherwise known as myelin.  Here we saw researchers modulate myelin production, with potential to fight TBI, MS, enhance plasticity, enhance brain processes, memory, fight dementia, whilst opening a door to neuroprotection.  Neurologically-wise this study has it all.  We are seeing a sharp increase in non-neuronal studies in the brain with researchers investigating the role of these lesser-known brain cells.  The number one study also provides detailled knowledge on the brain cell in question, oligodendrocytes.  They were able to establish, through, histological studies that at birth most oligodendrocytes are immature. The study then goes on to tell us what age we are when oligodendrocytes reach maturity and their turnover rate thereafter. The researchers were even able to carbon-date the cells and determine their age which is why this is the most viewed and shared Healthinnovations of 2014.
2.  At number two we have is the National University of Singapore of Singapore, who found a new type of immune cell that is expected to help in the development of a future treatment for Multiple Sclerosis (MS).  The study also explores the role of the all-important interleukin pathway, a very hot area at the moment, in the immune system.  The team found that a new type of immune T helper cells named TH-GM cells play a crucial role in the immune system and pathogenesis of neuronal
inflammation. The team showed that STAT5, a member of the STAT family of proteins, programs TH-GM and initiates the immune response to an auto-antigen in responding to a signal from an interleukin, IL-7, causing neuroinflammation, pathogenesis and damage in the central nervous system. Thus blocking IL-7 or STAT5 would provide a significant therapeutic benefit for Multiple Sclerosis (MS).
3.  Coming in next at number three St Vincent’s Hospital and the Victor Chang Cardiac Research Institute of Australia, who transplanted an already dead heart in a world’s first.  This breakthrough firmly pushed the human race out of the age of regeneration hurtling into the aeon of reanimation.  Surgeon’s developed a technique which means hearts that had been still for 20 minutes can be resuscitated and
transplanted into a patient.  The technique involves donor hearts being transferred to a portable machine known as a ‘heart in a box’ in which they were placed in a preservation solution, resuscitated and kept warm.  So far three people have received hearts in this way, with this novel transplant system expected to save 30 percent more lives by providing more hearts, which were in the past deemed unsuitable and/or too starved of oxygen to transplant.
4.  At number four is the Hebrew University of Jerusalem of Israel and the Max Dellbruck Institute of Germany.  The team not only discovered how the recently discovered circular RNA is formed they also identified a key muscular dystrophy link.  Unlike all other known RNAs, this molecule is circular, and is labelled circular RNA.  The team found that circRNAs not only compete with normal
RNAs, but the body actually produces them at the expense of normal RNA. The researchers observed that circRNAs play an important role in brain function, and likely in brain disease.  In addition, the researchers identified the protein ‘muscleblind’ as a factor involved in circRNA biogenesis, and showed that muscleblind can enhance and regulate the production of a subset of circular RNAs.  Importantly, defects in muscleblind function are known to cause a severe degenerative disease called myotonic dystrophy. Characterized by progressive muscle wasting and weakness, this is the most common form of muscular dystrophy that begins in adulthood.
5.  At the midway mark, at number five, we have the University of California, Los Angeles (UCLA), of the United States of America.  The team utilized a novel technology called electric field-induced release and measurement (EFIRM) to test lung cancer patients’ saliva for epidermal growth factor receptor (EGFR) gene mutations, a sign of lung cancer, which can be treated by medication
such as thymidine kinase inhibitors. The total detection time is less than 10 minutes and only requires a small saliva sample.  EFIRM is a multiplexible electrochemical sensor which uses electrode chips to enable exosomes in saliva to rapidly release molecular constituents (DNA, RNA and proteins) while simultaneously detecting any mutations in tumour-causing DNA sequences.  So here is a cancer detection study hitting all the marks, precision genetics, non-invasive testing, epigenetic mutation/methylation markers for cancer and a new spectra method. And of course that all important new biomarker.
6. Coming in at number six we have a multi-centre study led by the University of California of the United States of America.  This study reminded the medical community how important verification
and building on initial discovery is in making past findings standardised for usage in mass clinical settings.  Here the team provided the first evidence of the medium- to long-term safety and tolerability of transplanting human embryonic stem cells (hESCs) in humans.   This important benchmark also provided set hESCs dosages when treating human macular degeneration with stem cell therapy.
7.  At number seven we have the University of Connecticut, of the United States of America.   The team found a new way to identify protein mutations in cancer cells to develop the first precision medical vaccine to treat patients with ovarian cancer.  This vaccine is so precise that it can recognise tiny differences and mistakes on a cancer cell’s surface epitopes, thus allowing the cancer ‘vaccine’ to attack and kill the cancer cell.  In this way the
surrounding healthy cells with normal epitope sequences are ignored and left intact.  These subtle epitope mutations come from incorrect epigenetic methylation when cancer cells proliferate, making this a best-in-class when it comes to precision genetics and medicine.
8.  Nearing the end of this great list we have the Lund University of Sweden, McGill University of Canada and theHeart and Stroke Foundation of Canada at number eight.  This large-scale study  linked the genetic predisposition to elevated low-density lipoprotein cholesterol (LDL-C) to aortic
valve calcium and narrowing of the aortic valve.  This study was also valuable due to the fact there are 35,000 participants providing strong, instant validation for this new discovery.  Any team in the future will find validation an easy job with this study giving it an easy path into clinical procedure.  The data suggests that, in addition to the established risks for myocardial infarction and other vascular diseases, increases in low-density lipoprotein cholesterol are also associated with increased risk for aortic stenosis.  The team now plan to investigate whether intervention to reduce low-density lipoprotein cholesterol could prevent aortic valve disease.
9.  At number nine of the Top Ten Health innovations of 2014  we have the Salk Institute and Harvard Medical School of the United States of America.  Very few studies assimilate the spinal
chord and brain as one entity let alone map new spinal chord-to-brain neural mechanisms and receptors for chronic pain.  The researchers set out to precisely identify the spinal neurons involved in these circuits. They deciphered the role each of the two neuronal cell types, pain receptors and touch receptors, play in the processing of pain signals in the dorsal horn, the location where the sensory neurons connect with the spinal chord. The findings of this study are expected to help find new targets and treatments for the people who suffer from chronic pain as well as allodynia, fibromyalgia and nerve damage caused by diseases such as diabetes, cancer and autoimmune disorders as well as physical trauma.  As new technology and diagnostics become more available to the neuroscientific community these rich neurobiological studies, whereby more vital neuroanatomy is discovered, are greatly anticipated and received.
10. And at number ten we have Boston University of the United States of America and the United States Army, with a genetically-based breakthrough for Ebola.  One of the greatest concerns about this killer disease is the conjecture over the time of contagion.  The general consensus is that
Ebola only becomes contagious once a fever breaks with opponents stating that this is only because the fever is the onset of vomiting, unexplained bleeding and/or diarrhea, ie. excessive bodily fluid-loss.  Thus the existing argument is that although the incubation period of Ebola can be as long as three weeks, this excessive loss of Ebola-infected bodily fluid grossly raises the level of potential contamination and infection.  The researchers put paid to this argument by providing an RNA-based assay which can distinguish between different hemorrhagic fevers, including Marburg (Ebola cousin) and Lassa before the person becomes symptomatic, at the point the virus enters the blood stream.  As the test includes the cousin of the Ebola hemorrhagic virus, the Marburg hemorrhagic virus, it is hoped that the test can be tweaked to include Ebola.

Sensitive Blood Test May Help Rule Out Heart Attack



WEDNESDAY Oct. 7, 2015, 2015 — A new, highly sensitive blood test may help doctors quickly rule out heart attack for almost two-thirds of people who seek emergency room treatment for chest pain, a new study suggests.
Researchers said their findings could potentially reduce unnecessary hospital admissions and substantially lower health-care costs. “Until now, there were no quick ways to rule out a heart attack within the emergency department,” said the study’s lead author, Dr. Anoop Shah, from the University of Edinburgh in Scotland.
“Over the last two decades, the number of hospital admissions due to chest pain has tripled. The overwhelming majority of these patients do not have a heart attack,” Shah said. Assessing a possible heart attack requires lengthy stays in the ER or hospitalization for repeat testing, the study authors pointed out.
The new test is more sensitive than the standard version, Shah’s team said. It can detect far lower blood levels of troponin, a protein released when heart muscle is damaged. The more damage that occurs, the higher blood levels of troponin will be. A slight increase in troponin suggests some damage has occurred, while very high levels indicate a person has had a heart attack, the researchers explained.
Using this new test, doctors could potentially double the number of low-risk patients able to be safely discharged from the emergency room, the researchers reported in the Oct. 8 issue ofThe Lancet.”Use of this approach is likely to have major benefits for both patients and health-care providers,” Shah said in a journal news release.
For the study, the researchers measured troponin levels in more than 6,000 patients admitted to the hospital with chest pain, and assessed their risk for heart attack and death from heart attack within 30 days.
The investigators found that 61 percent of the patients with a troponin level below 5 ng/L (nanograms per liter of blood) were at very low risk of heart attack and could have been discharged early, regardless of age, gender, and risk factors for heart disease. One year out, these patients had a three times lower risk of heart attack and cardiac death than those with higher troponin levels, the researchers said.
The authors of an accompanying editorial in the journal said patient follow-up will be needed to validate use of this test in routine practice.”Trials are needed to assess the safety and effectiveness of clinical pathways that involve no further testing for such patients,” wrote Martin Than from Christchurch Hospital, New Zealand, and colleagues.


More information
The American Heart Association describes the symptoms of heart attack.

Insight into cancer resistance in elephants could aid human treatment !!!!



They are the largest land animals in the world, weighing up to 14,000 pounds and standing up to 4 meters tall. Given their size, elephants should be highly susceptible to cancer – they have at least 100 times more cells than humans – but they rarely develop the disease. In a new study, researchers shed light on the mechanisms behind elephants’ resistance to cancer – information that could fuel knowledge on cancer resistance in humans.
Study leader Dr. Joshua D. Schiffman, of the University of Utah School of Medicine, and colleagues publish their findings in JAMA.
Theoretically, an animal’s cancer risk should increase with their size and lifespan; the bigger an animal is, the more cells they have, which should increase the rate of cell division and susceptibility to gene mutations.
In 1975, however, a study by Dr. Richard Peto, of the University of Oxford in the UK, challenged this notion. He observed that cancer incidence across species is not dependent on an animal’s size or lifespan – a theory that is now hailed “Peto’s Paradox.”
A good example of this theory is the disparity in cancer incidence between humans and elephants; despite elephants being significantly larger than humans, their risk for cancer is much lower.
Previous research has suggested that specific molecular mechanisms in elephants protect them against cancer, though Dr. Schiffman and colleagues note that such mechanisms are poorly understood.
For this latest study, the team set out to learn more about the disparities in cancer mortality rates across different mammals, with a specific focus on elephants, and to shed light on possible mechanisms that induce cancer resistance in different species.
ELEPHANTS HAVE MULTIPLE COPIES OF KEY TUMOR-SUPPRESSOR GENE
The researchers assessed information on disease and cause of death for 36 mammalian species, including African or Asian elephants.
The genomes of all species were assessed, as well as the activity of peripheral blood lymphocytes – a type of white blood cell – among elephants, healthy humans and patients with a disease called Li-Fraumeni syndrome (LFS), a rare inherited condition that greatly increases the risk for cancer. This was to assess response to DNA damage.
Overall, the researchers found that cancer mortality rates did not increase with the size or lifespan of a mammal. For example, the cancer mortality rate for elephants was only 4.8%, compared with an 11-25% cancer mortality rate in humans.
The team also revealed that elephants possess at least 20 copies of a major tumor-suppressor gene called TP53, while healthy humans only have one copy, with two alleles (gene variants) inherited from each parent. People with LFS only inherit one functioning allele of the TP53 gene, according to the team, putting them at a 90-100% lifetime risk for cancer.
The researchers explain that the TP53 gene plays a key role in the response to DNA damage by triggering a form of cell death called apoptosis via the p53 protein. Compared with human lymphocytes, the researchers found that elephant lymphocytes were subject to p53-induced apoptosis at higher rates.
Based on their findings, the team suggests the additional copies of the TP53 gene and increased p53-induced apoptosis in elephants have evolved to protect them against cancer.
The authors write:
“Compared with other mammalian species, elephants appeared to have a lower-than-expected rate of cancer, potentially related to multiple copies of TP53. Compared with human cells, elephant cells demonstrated increased apoptotic response following DNA damage.
These findings, if replicated, could represent an evolutionary-based approach for understanding mechanisms related to cancer suppression.”
HUMAN CANCER VULNERABILITY LIKELY DOWN TO MODERN LIFESTYLE FACTORS
In an editorial linked to the study, Mel Greaves, PhD, of the UK’s Institute of Cancer Research, says the theory that elephants may be protected against cancer due to the acquisition of multiple copies of the TP53 gene seems “plausible.”
However, Greaves notes that it is unclear what implications the findings have for cancer in humans. “Perhaps the main message from this innovative investigation is to bring into focus the question of why humans appear to be so ill-adapted to cancer, given the average size and life span,” he speculates.
“The human genome is replete with footprints of positive selection in the not-too-distant historical past. Humans may have acquired, in one particular respect, an extra cancer suppressor gene variant early on in evolutionary history approximately 1.8 million years ago,” Greaves continues.
He points out, however, that modern humans are particularly vulnerable to cancer, which is more down to lifestyle factors – such as smoking – that are not seen in other animals. “These behaviors are relatively recently acquired by humans, over a few hundred years, and the risks they impart far exceed prior and otherwise effective cancer suppressor mechanisms that were inherited from primate ancestors,” explains Greaves.

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Hi,I,m Basim from Canada I,m physician and I,m interested in clinical research feild and web development.you are more welcome in our professional website.all contact forwarded to basimibrahim772@yahoo.com.


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