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Showing posts with label News. Show all posts
Showing posts with label News. Show all posts

Saturday, March 19, 2016

Redhill Biopharma successfully meets primary endpoint in Phase III study of RHB-105 for H. pylori infection


RedHill Biopharma Ltd., an Israeli biopharmaceutical company focused on late clinical-stage, proprietary, orally-administered, small molecule drugs for inflammatory and gastrointestinal diseases, including gastrointestinal cancers, today announced positive top-line results from its Phase III study with RHB-105 for the treatment of Helicobacter pylori (H. pylori) bacterial infection. 
Top-line results from the study demonstrated 89.4% efficacy in eradicating H. pylori infection with RHB-105.
The ERADICATE Hp first Phase III study successfully met its primary endpoint of superiority over historical standard of care efficacy levels of 70%, with high statistical significance (p <0.001). No serious adverse events, new or unexpected safety issues were noted in the study.The top-line results from the RHB-105 Phase III study, demonstrating achievement of primary endpoint, were provided to RedHill by an independent third party following an independent analysis and remain subject to completion of the independent review and analysis of the underlying data, including all safety, secondary and other outcome measures, and completion of the clinical study report (CSR), expected in the third quarter of 2015.
Prof. David Graham, M.D., M.A.C.G., of the Baylor College of Medicine, a key opinion leader in the field of gastric cancer and H. pylori infection and Principal Investigator of the ERADICATE Hp study, said: “The outstanding results of the RHB-105 Phase III study, which demonstrated a 89.4% cure rate of H. pylori, are consistent with the hypothesis that this may represent a promise for a new and improved treatment for H. pylori infection, and could significantly contribute to the prevention of gastric cancer, MALT lymphoma and other gastrointestinal diseases and conditions. Given the current high levels of antibiotic resistance and treatment failures with current standard of care therapies, RHB-105 could become, if approved, a best-in-class treatment, improving and potentially saving patients’ lives.”
Ira Kalfus, M.D., RedHill’s Medical Director, added: “On the basis of the clear success of the 
ERADICATE Hp study, and the Fast-Track designation of RHB-105, we look forward to meeting with FDA to discuss the clinical and regulatory path towards marketing approval in the U.S. No new or unexpected safety issues were identified. Efficacy and safety data from this study will be submitted for presentation at an upcoming medical meeting.” Gilead Raday, RedHill’s Senior VP Corporate and Product Development, said: “We are enthusiastic about the strong Phase III top-line results of RHB-105 and its potential benefit to patients. Coupled with the QIDP designation, patent protection and expanded indication, RHB-105 should be well-positioned, if approved, for commercial success as a first-line therapy for the treatment of H. pylori infection. We would like to thank the patients, investigators and service providers who participated in this study”.
The randomized, placebo-controlled, ERADICATE Hp Phase III study was designed to evaluate the safety and efficacy of RHB-105 as a first-line treatment for confirmed H. pylori infection. A total of 118 dyspepsia patients with confirmed H. pylori infection were enrolled and treated in the ERADICATE Hp study, which was conducted in 13 clinical sites in the U.S. Subjects were randomized in a 2:1 ratio to receive either RHB-105 or a placebo, for a period of 14 days, and assessed for the eradication of H. pylori infection 28 to 35 days after completion of treatment and for the protocol-defined primary endpoint of superiority over historical standard of care efficacy levels of 70%.
RHB-105 is a new and proprietary fixed-dose oral combination therapy of two antibiotics and a proton pump inhibitor (PPI) in an all-in-one oral capsule with a planned indication for the treatment of H. pylori infection – a major cause of chronic gastritis, peptic ulcer disease, gastric cancer and mucosa associated lymphoid tissue (MALT) lymphoma. Gastric cancer is the second most common cause of cancer deaths worldwide with approximately 1 million deaths per year, of which 95% are caused by H. pylori and may be preventable. Several countries have already started, or are planning, population-based H. pylori screening and treatment programs designed to eliminate gastric cancer.
With RHB-105, RedHill is pursuing an indication of first-line treatment of H. pylori infection, regardless of ulcer status, a significantly broader indication than current standard treatments for 
H. pylori, which are typically indicated only in patients with active or recent history of duodenal ulcer disease. If approved, RHB-105 may be the first H. pylori eradication therapy to target this broader indication, which would significantly expand the potential patient population for this drug candidate. The ERADICATE Hp Phase III study is planned to be followed by a second Phase III study and additional studies may be required, subject to FDA feedback.
RHB-105 was designated by the FDA as a Qualified Infectious Disease Product (QIDP) under the Generating Antibiotic Incentives Now (GAIN) Act, which is intended to incentivize the development of new antibiotic drugs for the treatment of serious or life-threatening infections. The designation allows RedHill to benefit from Fast-Track development status for RHB-105, providing for an expedited development pathway, as well as Priority Review status, potentially leading to a shorter review time by the FDA of a New Drug Application (NDA), if filed. If approved, RHB-105 will also receive an additional five years of U.S. market exclusivity in addition to the standard exclusivity period, for a total of 8 years of market exclusivity.
The 2015 global and U.S. market potential for H. pylori eradication therapies, at current branded prices, was recently estimated at approximately $4.83 billion and $1.45 billion, respectively, and could potentially grow with increasing awareness of the health risks associated with 
H. pylori infection and the benefits of its eradication.

Lyxumia (lixisenatide) demonstrated cardiovascular safety in people with type 2 diabetes and high CV risk


Sanofi has announced the presentation of full results of the Phase IIIb ELIXA study, which was designed to assess the cardiovascular (CV) safety of Lyxumia® (lixisenatide) in adults with type 2 diabetes and high CV risk. As previously reported, lixisenatide met the pre-specified criterion of non-inferiority versus placebo for the composite primary endpoint of CV death, non-fatal myocardial infarction, non-fatal stroke and hospitalization for unstable angina but did not demonstrate superiority. The full results will be included in the U.S. New Drug Application for lixisenatide, which is on track to be resubmitted to the U.S. Food and Drug Administration in Q3 2015.
Additional safety findings include no signal for increased risk of heart failure, pancreatitis, pancreatic cancer or severe symptomatic hypoglycemia. Lixisenatide was generally safe and well tolerated; nausea and vomiting, which are known side effects of the GLP-1 RA class, were observed more frequently with lixisenatide.
“The importance of determining the CV safety of diabetes medicines, as set out in the FDA guidance issued in 2008, is widely recognized. People around the world are being treated with GLP-1 receptor agonists, and the CV effects were unknown,” said Dr. Marc Pfeffer, Professor of Medicine at Harvard Medical School, Senior Physician in the Division of Cardiovascular Medicine at Brigham and Women’s Hospital and Chair of the ELIXA Steering Committee. “ELIXA goes beyond the FDA guidance to deliver data related to heart failure and other insights that are not currently available for any other GLP-1 receptor agonist. Our data provide the medical community, patients and caregivers with information that will better inform them about how lixisenatide can be safely used to better control their glucose.”
“As the first completed long-term CV safety study of a GLP-1 receptor agonist, the successful ELIXA trial will be shared with health authorities around the world and provides important outcomes data that can be considered by healthcare professionals,” said Pierre Chancel, Senior Vice President, Head of Global Diabetes at Sanofi. “Sanofi is committed to developing and delivering safe and effective treatment options for people with diabetes. This study supports that important work.”
Full study results were presented during a symposium at the American Diabetes Association 75th Scientific Sessions in Boston.
Results of Analysis
Lixisenatide met the pre-specified criterion of non-inferiority versus placebo for the composite primary endpoint of MACE+: CV death, non-fatal myocardial infarction, non-fatal stroke and hospitalization for unstable angina (Hazard Ratio [95% CI]: 1.017 [0.886 to 1.168]). Since the upper bound of the 95% CI was greater than 1.0, superiority over placebo in reducing the composite primary endpoint was not met.
The CV safety of lixisenatide was also confirmed by further analyses (e.g. MACE Hazard Ratio [95% CI]: 1.02 [0.887 to 1.172]). No signal for increased risk of heart failure (HF) was observed (Hazard Ratio [95% CI]: 0.96 [0.75 to 1.23]).
Measures of non-CV safety showed pancreatitis (0.2% with lixisenatide and 0.3% with placebo), pancreatic cancer (<0.1% with lixisenatide and 0.3% with placebo), severe symptomatic hypoglycemia (0.3 events per 100 patient-years with lixisenatide; 0.6 per 100 patient-years with placebo), malignancy (2.9% with lixisenatide and 2.6% with placebo), drug-related allergic reactions (0.2% with both lixisenatide and placebo).
About ELIXA
ELIXA (Evaluation of Cardiovascular Outcomes in Patients With Type 2 Diabetes After Acute Coronary Syndrome During Treatment With Lixisenatide) is the first event-driven cardiovascular outcomes study to provide data for a glucagon-like peptide-1 receptor agonist (GLP-1 RA). ELIXA was a randomized, double-blind, parallel group trial designed to evaluate cardiovascular risk, comparing lixisenatide to placebo in a high-risk population of adults with type 2 diabetes. More than 6,000 adults with type 2 diabetes and high CV risk (i.e., patients who have recently experienced a spontaneous acute coronary syndrome event) participated in the trial. The composite primary endpoint, which was evaluated for non-inferiority and superiority, comprised cardiovascular (CV) death, non-fatal myocardial infarction, non-fatal stroke, or hospitalization for unstable angina. The global ELIXA study started in June 2010 and was completed in 2015.
About Lixisenatide
Lixisenatide is a once-daily prandial glucagon-like peptide-1 receptor agonist (GLP-1 RA) for the treatment of adult patients with type 2 diabetes mellitus. GLP-1 is a naturally-occurring peptide hormone that is released within minutes after eating a meal. It is known to suppress glucagon secretion from pancreatic alpha cells and stimulate glucose-dependent insulin secretion by pancreatic beta cells.
Lixisenatide was in-licensed from Zealand Pharma A/S (NASDAQ OMX Copenhagen: ZEAL), www.zealandpharma.com, and was approved in Europe in 2013 for the treatment of adults with type 2 diabetes mellitus to achieve glycemic control in combination with oral glucose-lowering medicinal products and/or basal insulin when these, together with diet and exercise, do not provide adequate glycemic control. Lixisenatide is currently approved in over 50 countries worldwide for the treatment of adults with type 2 diabetes, with commercial launches in most EU countries, Japan, Brazil, Mexico and other markets. Lyxumia is the proprietary name approved by the European Medicines Agency and other health authorities for the GLP-1 RA lixisenatide. Lixisenatide is an investigational product in the U.S. It will be resubmitted to the Food & Drug Administration (FDA) in the third quarter of 2015. The proprietary name in the U.S. is under consideration.

Clinical trial using dorsaVi sensors demonstrates significant reduction in back pain


New data from a cluster randomised controlled trial has demonstrated that back pain patients monitored and treated using wearable movement sensors from dorsaVi Ltd (ASX:DVL) had a significant and sustained improvement in pain and functional ability.Across all primary outcome measures, participants treated with the dorsaVi sensors with biofeedback showed a 35 % to 47 % improvement at 12 months, which were all above the threshold for clinically important difference (>30 % of baseline scores).
The study results were published online in the leading medical journal BMC Musculoskeletal Disorders.It reports on the 12 month, multicentre, cluster randomised, placebo controlled clinical trial sponsored by dorsaVi and the Victorian Government. The trial involved 112 patients – 58 in the interventional group (which received ViMove) and 54 in the control group. The results, as published in the paper, are highly positive and validate that intervention by ViMove resulted in “significant and sustained improvements in pain and activity limitation that persisted after treatment finished.”
This is the first clinical trial of its kind to investigate the effect that technology can have on the rehabilitation of low back pain (LBP). The clinical trial investigated whether changing patterns of lumbo-pelvic movement and/or posture using motion sensor biofeedback, provided by ViMove, in people with LBP would lead to reduced pain and activity limitation when compared with guidelines- based medical treatment or physiotherapy.
LBP is highly prevalent and globally is the leading cause of disability, ahead of ischaemic heart disease, chronic obstructive pulmonary disease, and other musculoskeletal disorders, including osteoarthritis. In the UK, LBP is very common, affecting 80% of the population at some point in their lives1 and the cause of 8.3 million missed work days last year2. In the past, compared with placebo or no treatment, most non-surgical treatments for non-specific LBP showed only small to moderate effects with one treatment showing little superiority over the other. In addition, short term treatment effects typically reduce over the subsequent year.
dorsaVi CEO, Dr Andrew Ronchi, said: “Medical adoption by health practitioners is based on having protocols supported by clinical evidence and published in peer reviewed journals. We are delighted with the results of the trial and that it has now been published in a peer reviewed journal.”
dorsaVi’s ViMove is a wearable sensor system that turns human movement into actionable, easily interpreted data. The sensors can track a patient’s movement at 200 frames per second. The sensors provide real-time feedback about high risk positions for patients with back pain and also encourages positive movement patterns. This data informs decisions by the treating healthcare professional to modify their patients’ daily movements, thus reducing their risk of another episode and reducing recovery time. ViMove is approved for use in Australia and Europe and cleared by the FDA under 510(k) for the United States.
About the trial
The 12 month study enrolled 112 patients at eight sites including the Austin and Epworth Hospitals. All patients reported back pain, with a vast majority (85%) classified as chronic back pain patients. All patients wore ViMove sensors and had 6-8 office visits over 10 weeks. Follow-up appointments were held at 2, 3, 6 and 12 months with their clinicians. Clinicians in the Intervention arm got access to the ViMove sensor data to guide care and their patients received real time feedback in weekly monitoring sessions. Clinicians and patients in the control group were blind to the data and were given no feedback by the ViMove device.

Massachusetts General Hospital launches phase II trial of BCG vaccine to reverse type 1 diabetes



FDA approval of trial testing generic vaccine announced at ADA Scientific Sessions
A phase II clinical trial testing the ability of the generic vaccine bacillus Calmette-Guérin (BCG) to reverse advanced type 1 diabetes has received approval from the U.S. Food and Drug Administration (FDA). The approval of this trial, which will shortly begin enrolling qualified patients, was announced today at the 75th Scientific Sessions of the American Diabetes Association (ADA) by Denise Faustman, MD, PhD, director of the Massachusetts General Hospital (MGH) Immunobiology Laboratory and principal investigator of the study.
The five-year trial will investigate whether repeat BCG vaccination can clinically improve type 1 diabetes in adults between 18 and 60 years of age who have small but still detectable levels of insulin secretion from the pancreas. Faustman’s research team was the first group to document reversal of advanced type 1 diabetes in mice and subsequently completed a successful phase I human clinical trial of BCG vaccination. She announced the FDA approval to launch the phase II trial during her ADA presentation, “Low Levels of C-Peptide Have Clinical Significance for Established Type 1 Diabetes.”
“We have learned a lot since the early studies in mice – not just about how BCG works but also about its potential therapeutic benefits, similar to what are being seen in trials against other autoimmune diseases,” says Faustman. “We are so grateful to all of the donors, large and small, who have made this trial possible – especially the Iacocca Foundation, which has believed in us and has been a supporter since our early days. Our goal is to complete enrollment and also to raise the remaining funds needed for the trial by the end of this year.”
A generic drug with over 90 years of clinical use and safety data, BCG is currently approved by the FDA for vaccination against tuberculosis and for the treatment of bladder cancer. The vaccine is known to elevate levels of the immune modulator tumor necrosis factor (TNF), which Faustman’s team previously showed can temporarily eliminate in both humans and mice the abnormal white blood cells responsible for autoimmune type 1 diabetes. Increased TNF levels also stimulated production of protective regulatory T cells.
In the phase I clinical trial, which was published in the August 8, 2012, issue of PLOS Medicine, two injections of BCG spaced four weeks apart led to temporary elimination of diabetes-causing T cells and provided evidence of a small, transient return of insulin secretion. The phase II clinical study will include more frequent dosing over a longer time period to determine the potential of repeat BCG vaccination to ameliorate the autoimmune state and improve clinical parameters such as HbA1c, a marker of average blood sugar control.
In the new trial, which will be double blinded and conducted at MGH, 150 adults with long-term type 1 diabetes will be randomized to receive two injections four weeks apart of either BCG or placebo and then a single injection annually for the next four years. Patients will be closely monitored over the five-year trial period. The primary outcome measure will be improved results on the HbA1c blood test, which have been shown to prevent complications.
“In the phase I clinical trial we demonstrated a statistically significant response to BCG, but our goal in phase II is to create a lasting therapeutic response,” says Faustman, an associate professor of Medicine at Harvard Medical School. “We will be working again with people who have had type 1 diabetes for many years. This is not a prevention trial; instead, we are trying to create a regimen that will treat even advanced disease. In addition to our phase I trial, we took guidance from the BCG clinical trials that are underway globally for other autoimmune diseases such as multiple sclerosis.”
Lee Iacocca, founder of the Iacocca Foundation, says, “My family and I have been fortunate to be part of this research for many years. It is incredibly exciting to be talking about curing people, not mice. I made a promise to my late wife to find a cure for type 1 diabetes. Now my family and I look forward to the continued progress and are proud to support this effort to get closer to that goal.” The Iacocca Foundation provided major funding for the phase I trial and has taken a leadership role in funding the phase II trial.
More than $19 million has been raised out of a total of $25 million needed to conduct the phase II study over the next five years. Additional information about the clinical trial, including information for potential participants and financial supporters, is available athttp://www.faustmanlab.org/ or by emailing DiabetesTrial@partners.org. Details on the trial and enrollment also are available at: https://clinicaltrials.gov/ct2/show/NCT02081326.

NightstaRx and The University of Alberta announce the start of the first Canadian gene therapy study to treat Choroideremia



NightstaRx Ltd (“Nightstar”), the biopharmaceutical company specialising in bringing therapies for retinal dystrophies to patients, announces that the University of Alberta, has begun enrolling and dosing subjects in a Phase II clinical trial of the Company’s gene therapy for the treatment of choroideremia (CHM). This gene therapy approach uses a viral vector known as adeno-associated virus (AAV) to deliver a wild-type copy of the Rab-escort protein 1 (REP-1) gene (AAV2-REP1) into cells of the eye.
The trial, which is sponsored by the University of Alberta, is an open label study involving a total of 6 male patients, who will each receive a single dose of AAV2-REP1 via a sub retinal injection.
Choroideremia is an inherited X-linked recessive disease which inevitably causes blindness. It is caused by mutations to the CHM gene which encodes Rab-escort protein 1 and affects approximately 1 in 50,000 people. The first symptom of the condition is usually an impairment of night vision which often occurs in early childhood. This is followed by progressive narrowing of the field of vision, as well as a decrease in the ability to see details, culminating in blindness, most commonly in late adulthood. No effective treatment currently exists.
David Fellows, CEO of Nightstar said:
“Gene therapy treats genetic diseases at the molecular level by correcting what is wrong with defective genes. We are broadening our pipeline of products in development and are leading the way in the development of an effective gene therapy treatment for choroideremia. This new study, sponsored by the University of Alberta, is another step forward in the development of AAV2-REP1. We have been granted Orphan Drug Designation for the product in the United States and Europe and the data to date has shown very promising results.”
Ian MacDonald, Professor of Ophthalmology and Visual Sciences, University of Alberta commented:
“We are very excited to be working with Nightstar and to have treated our first choroideremia patient. Choroideremia is a devastating condition for individuals and families, but we believe our new gene therapy will arrest any further deterioration of vision and will provide long lasting benefit. The next challenge will be making this therapy available to all individuals with the condition as soon as we possibly can.”
About Choroideremia
Choroideremia is an inherited disorder that leads to progressive loss of vision due to degeneration of the choroid and retina which is caused by a lack of Rab Escort Protein-1 (REP-1) and occurs almost exclusively in males. The first symptoms occur in childhood, with night blindness being the most common first symptom. As the disease progresses, there is loss of peripheral vision or ‘tunnel vision’, and later a loss of central vision. Progression of the disease continues throughout the individual’s life, although both the rate and the degree of visual loss can vary, even within the same family. There is currently no treatment or cure for this disease.


More than 95% of the global population has at least one health problem, study finds


The largest and most in-depth study of global health trends to date finds that more than 95% of the world’s population has health problems, with more than a third of us experiencing five or more conditions.

The results of the Global Burden of Disease Study 2013 (GBD 2013) were recently published inThe Lancet.Lead study author Prof. Theo Vos, of the Institute of Health Metrics and Evaluation at the University of Washington in Seattle, and colleagues set out to calculate up-to-date estimates of disease and injury incidence and prevalence among 188 countries between 1990 and 2013, using data from more than 35,000 sources.
The team also sought to estimate the number of disability-adjusted life years (DALYS) – that is, the number of healthy years lost due to illness – over the 23-year period.In total, the researchers were able to provide global estimates of incidence and prevalence for 301 acute and chronic diseases, and they were able to assess the effects of 2,337 health consequences that result from at least one of these disorders.
Prof. Vos and colleagues found that in 2013, only 1 in 20 people (4.3%) around the globe had no health problems, meaning more than 95% of us had one or more illnesses.
The researchers found that 2.3 billion people worldwide – more than a third of us – had at least five health conditions in 2013. Over the 23-year study period, the number of people with 10 or more health conditions rose by 52%.
The number of people with multiple illnesses increased with age. In 2013, 36% of children in developed countries aged 0-4 years had no illnesses, compared with only 0.03% of adults aged 80 and older.
The number of healthy years lost due to illness increased from 21% in 1990 to 31% in 2013, and the number of years lived with disability (YLD) rose from 537.6 million in 1990 to 764.8 million in 2013.
The researchers attribute the increase in YLD over the 23-year period to population growth and aging. They found that the main drivers for YLD were musculoskeletal disorders, mental illnesses, substance abuse disorders, neurological conditions and chronic respiratory disorders.

Disability rates not declining as fast as death rates

The leading causes of health loss did not change much between 1990 and 2013, according to the researchers.
They found that in 2013, musculoskeletal disorders, such as low back pain and arthritis, and mental and substance use disorders – particularly anxiety, depression and drug and alcohol use disorders – accounted for almost 50% of all health loss globally.
Low back pain and major depression were ranked as two of the top 10 disability contributors in 2013 among every country. These conditions caused more health loss than chronic obstructive pulmonary disease (COPD), diabetes and asthma combined, according to the results.
The team found the leading causes of disability and health loss varied by regions. Falls, for example, were found to be the second leading cause of disability in 11 of 13 countries in central Europe, while anxiety disorders were more prominent causes of disability in Caribbean regions.
Past war conflict was identified as the main contributor to health loss in Cambodia, Nicaragua, Rwanda, while ranking as the second leading cause of health loss in Vietnam.
Perhaps one of the most important findings was that rates of disability as a result of health problems are not declining as rapidly as death rates from such conditions. The team points to diabetes as an example; while diabetes rates rose by 43% between 1990 and 2013, death rates from the condition only increased by 9%.
“The fact that mortality is declining faster than non-fatal disease and injury prevalence is further evidence of the importance of paying attention to the rising health loss from these leading causes of disability, and not simply focusing on reducing mortality,” says Prof. Vos, adding:
“Large, preventable causes of health loss, particularly serious musculoskeletal disorders and mental and behavioral disorders, have not received the attention that they deserve. Addressing these issues will require a shift in health priorities around the world, not just to keep people alive into old age, but also to keep them healthy.”
The Bill and Melinda Gates Foundation funded the study.

McMaster researchers test fecal transplantation to treat ulcerative colitis



Two new studies led by researchers from the Farncombe Family Digestive Health Research Institute at McMaster University show that transplantation of fecal matter may be a useful tool in the fight against ulcerative colitis (UC).
Ulcerative colitis is a chronic, debilitating inflammatory bowel condition characterized by symptoms including bloody stools, diarrhea, abdominal pain, weight loss and malnutrition. It results from the development of abnormal immune responses to the normal bacteria in the digestive tract. It is difficult to treat and standard therapy doesn’t always work.
There is currently great interest in treating UC with fecal microbiota transplantation (FMT), which involves transplanting gut fecal bacteria from healthy people into patients with UC.
A study recently published in Inflammatory Bowel Diseases found that UC can be controlled by the type of bacteria that inhabits the gut. The study was led by Elena Verdu, an associate professor of medicine with the Michael G. DeGroote School of Medicine.
“Our animal research provides insight that selected bacterial groups, involved in gut health, are important for protecting the colon against injury and inflammation,” said Verdu.
Along the same theme, in research published on June 29, 2015 in Gastroenterology, professor of medicine Paul Moayyedi and his team explored the safety and efficacy of FMT by conducting a placebo-controlled, randomized trial. They found that “FMT induces remission in a significantly greater percentage of patients with active UC than placebo,” the authors wrote.
“Our study in patients with ulcerative colitis is the first randomized trial of fecal microbiota transplantation in adults with ulcerative colitis and shows that this therapy may work,” said Moayyedi. “The effect of fecal transplant seems to be dependent on the sort of bacteria that is in the donor stool, which fits with the observations of Dr. Verdu’s animal study.”
In Verdu’s study, mice were given gut bacteria from patients with severe UC and the effects were compared to those produced in mice that were given bacteria from a healthy person. The results identified a reduced amount of the bacterial families that are important for gut health in the feces of patients with severe colitis.
Second, they found that when mice were given these bacteria and then exposed to a toxin that causes gut injury, the resulting inflammation was higher in the mice with UC bacteria than in mice with bacteria from the healthy person, in whom the beneficial bacterial groups were abundant.
“The study also showed that the same protective effect could be achieved using the fecal material from the healthy person as with specific groups of bacteria that were isolated from the ‘healthy’ fecal matter,” said Verdu. “This suggests that specific combinations of beneficial bacteria extracted from healthy people could be tested in future clinical fecal transplantation studies, and could potentially replace fecal matter.”
Verdu said the implications of her study relate to the selection of healthy donors for fecal transplantation.
“In addition to screening for infections and disease, donors that harbour an abundance of the beneficial bacterial groups identified in our study could be selected to increase the chances of success of transplantation,” said Verdu.
Moayyedi and his team, including McMaster professors Michael Surette and Christine Lee, recruited 75 patients with a flare up of their UC and randomized them to fecal transplant therapy given as an enema derived from stool donated by an anonymous healthy donor once per week for six weeks, or a placebo consisting of a water enema. They found 24 per cent were in remission in the fecal transplant group compared to five per cent in the placebo group. There were two main healthy donors, donor A and donor B – one of which was the healthy donor from Verdu’s mouse study – and benefit seemed to be mostly related to those that received stool from donor B. The effect was also greater in those that had recently been diagnosed with UC.
“Many questions remain, but this provides interesting data suggesting that altering the gut microbial flora may be promising for treating ulcerative colitis,” the authors noted.
Moayyedi added that the data suggests more research is needed using the FMT approach.

FDA strengthens heart safety warnings on painkillers


People should think carefully about taking these drugs, both over-the-counter versions and prescription pills, the FDA says. It’s asking manufacturers to change the labels.”They used to say they might cause risk of heart attack or stroke. Now we are saying they do cause increased risk of heart attack and stroke,The warning covers drugs called nonsteroidal anti-inflammatory drugs or NSAIDS for short. They include ibuprofen, sold under brand names like Advil or Motrin; naproxen (Aleve), as well as prescription arthritis drugs known as COX-2 inhibitors, such as Celebrex. Tylenol, known generically as acetaminophen, is not an NSAID.Cough and cold remedies can also contain NSAIDs as an ingredient.”Because many prescription and OTC medicines contain NSAIDs, consumers should avoid taking multiple remedies with the same active ingredient,” the FDA said.
“FDA is strengthening an existing warning in prescription drug labels and over-the-counter (OTC) Drug Facts labels to indicate that nonsteroidal anti-inflammatory drugs (NSAIDs) can increase the chance of a heart attack or stroke, either of which can lead to death,”
“Those serious side effects can occur as early as the first few weeks of using an NSAID, and the risk might rise the longer people take NSAIDs. (Although aspirin is also an NSAID, this revised warning doesn’t apply to aspirin.)”
Last year, FDA said it was reviewing the safety of these drugs.
Some of the studies they looked at showed a clear pattern: people who took NSAIDS were more likely to have heart attacks or strokes.
“There is no period of use shown to be without risk,” said Dr. Judy Racoosin, deputy director of FDA’s Division of Anesthesia, Analgesia and Addiction Products.”In the coming months, the FDA will request that manufacturers update the existing cardiovascular risk information in Drug Facts labels for over-the-counter (OTC) non-aspirin NSAIDs. Consumers and health care professionals should remain alert for the development of heart- and stroke-related symptoms throughout the time a consumer takes any NSAID,” FDA said.This doesn’t mean people should just stop taking NSAIDS, FDA said.”Take the lowest effective dose for the shortest amount of time possible,” said FDA’s Dr. Karen Mahoney.The American Heart Association advises people to try acetaminophen (Tylenol) first.
“If you have heart disease or high blood pressure, consult a health care provider before using an NSAID,” FDA added.
“Balance the benefits of NSAIDs with the possible risks and weigh your options. If you take low-dose aspirin for protection against heart attack and stroke, you should know that some NSAIDs, including ibuprofen and naproxen, can interfere with that protective effect.”
And the agency gives advice on what symptoms to look for.
“Stop taking NSAIDs and seek medical help if you experience symptoms that might signal heart problems or stroke, such as chest pain, trouble breathing, sudden weakness in one part or side of the body, or sudden slurred speech.”
In 2013 Americans bought more than 275 million boxes of over-the-counter NSAIDs, racking up $1.7 billion in sales, according to retail tracker IRI.

FIRST EVER INHALABLE EBOLA VACCINE IS SUCCESSFUL IN SIMIAN TRIALS.



A collaborative team from The University of Texas and the National Institutes of Health have developed an inhalable vaccine that successfully protects primates against Ebola in simian trials. The team state that a needle-free, inhalable vaccine against Ebola presents certain advantages as it has been shown that this disease lines respiratory tract in infection, also, this will mean that immunization will not require trained medical personnel.  The opensource study is published in theJournal of Clinical Investigation.
Previous studies with primates suggest that aerosols of most biothreat agents, which are particles dispersed in the air, are infectious. Recent studies show that contact with the Ebola virus through the mucus membranes that line the respiratory tract results in infection, suggesting that airway linings may be important portals of entry for the virus. Aerosolized delivery has never before been tested for an Ebola vaccine or any other viral hemorrhagic fever vaccine.
The current study characterized the immune responses generated by vaccination against Ebola delivered to the respiratory tract as either an aerosol or liquid. Direct comparisons were made with an unrelated protective injectable Ebola vaccine. This included detailed comparisons between immune T cell responses in the lungs, spleen and blood. A single vaccination with the aerosol developed by the researchers protected non-human primates against the severe disease and death caused by lethal Ebola infection.
The team state that this study demonstrates successful aerosol vaccination against a viral hemorrhagic fever for the first time.  They go on to add that a single-dose aerosol vaccine would enable both prevention and containment of Ebola infections, in a natural outbreak setting where healthcare infrastructure is lacking or during bioterrorism and biological warfare scenarios.
The researchers surmise that the findings of this study provide the basis for advancing this experimental vaccine to an NIH phase I clinical study. They go on to conclude that in the future, pending approval through an Investigative New Drug Application, the aerosolized form of the vaccine will be evaluated for replication, safety and immunity development in a study in adults.

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.

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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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