An Inserm team in Dijon directed by Fran
Researchers at Imperial College London have begun the first UK clinical trials of a gene therapy for heart failure.
Heart failure, when the heart is unable to pump blood adequately, affects more than 750,000 people in the UK, causing breathlessness and hindering day-to-day activities. The therapy is designed to increase the levels of SERCA2a protein in heart muscle cells by using a harmless virus to insert extra genes into the cells.
The two clinical trials announced mark the culmination of more than 20 years of research funded by the British Heart Foundation (BHF) at Imperial and the Royal Brompton Hospital, which have identified SERCA2a as an important factor affecting how well heart muscle cells can contract in people with heart failure.
The trials will be led in the UK by cardiologists and scientists at Brompton and Imperial, in collaboration with doctors at several UK hospitals including Harefield in London, Papworth in Cambridge and the Golden Jubilee National Hospital in Scotland. The announcement coincides with Fight For Every Heartbeat, a new hard-hitting campaign from the BHF that hails research as the weapon needed to win the battle against heart disease.
Dr Alexander Lyon, BHF Senior Lecturer at Imperial College London and Consultant Cardiologist at the Royal Brompton Hospital, who is the UK lead investigator for both studies, said: ‘Heart failure affects more than three quarters of a million people across the UK. Once heart failure starts, it progresses into a vicious cycle where the pumping becomes weaker and weaker, as each heart cell simply cannot respond to the increased demand.
‘Our goal is to fight back against heart failure by targeting and reversing some of the critical molecular changes arising in the heart when it fails.’
The trials are the next step in the research after laboratory studies found that the gene therapy can be used to effectively restore function to the failing heart, in collaboration with colleagues from the United States.
Doctors plan to study the gene therapy in two separate clinical trials. The first, called CUPID2, will begin treating people with heart failure in the next few weeks in the Cardiovascular Biomedical Research Unit at the Royal Brompton Hospital.
CUPID2 will assess whether cardiac gene therapy to increase SERCA2a is safe and can improve both quality and length of life, and reduce emergency hospital admissions, for heart failure patients. The trial will involve 200 patients with heart failure from the Royal Brompton Hospital and other centres across the world, and is funded by US biotech company Celladon.
The second trial, called SERCA-LVAD, is due to start recruitment in the summer of 2013. Co-funded by the BHF, this trial will test the SERCA2a gene therapy in 24 UK heart failure patients already fitted with mechanical heart pumps, known as left ventricular assist devices (LVADs). It will give vital information about the effectiveness of the therapy by measuring the amount of the SERCA2a gene and protein that has been introduced into heart muscle.
Professor Sian Harding, Professor of Cardiac Pharmacology and Head of the BHF Centre for Regenerative Medicine at Imperial College London, who developed the treatment, said: ‘It
The Centers for Disease Control and Prevention (CDC) and Maryland Department of Health and Mental Hygiene have confirmed that a patient who recently died of rabies in Maryland contracted the infection through organ transplantation done more than a year ago. The patient was one of four people who had received an organ from the same donor. This week, CDC laboratories tested tissue samples from the donor and from the recipient who died to confirm transmission of rabies through organ transplantation.
In early March, the Maryland Department of Health and Mental Hygiene initiated an investigation after the organ recipient died, which led to the rabies diagnosis. The investigation revealed that the organ recipient had no reported animal exposures, the usual source of rabies transmission to humans, and identified the possibility of transplant-related transmission of rabies, which is extremely rare.
The organ transplantation occurred more than a year before the recipient developed symptoms and died of rabies; this period is much longer than the typical rabies incubation period of 1 to 3 months, but is consistent with prior case reports of long incubation periods. CDC
When it comes to prescribing medications to their patients, physicians could use a dose of extra training, according to a new study led by a UCLA researcher.
In previous studies, Dr. Derjung Tarn and her colleagues found that when doctors prescribed medicines, the information they provided to patients was spotty at best, they rarely addressed the cost of medications and they didn’t adequately monitor their patients’ medication adherence.
The logical next step, Tarn said, was to devise an intervention aimed at improving how physicians communicate to their patients five basic facts about a prescribed medication: the medication’s name, its purpose, the directions for its use, the duration of use and the potential side effects. And it appears to have worked.
Tarn and her co-researchers found that physicians who completed the training demonstrated a significant improvement in how they communicated this crucial information. Compared to a control group that didn’t receive the training, these doctors discussed at least one additional topic out of the five
The father acts as an intermediary for dentist fear between both mother and children
Fear of visiting the dentist is a frequent problem in paediatric dentistry. A new study confirms the emotional transmission of dentist fear among family members and analyses the different roles that mothers and fathers might play.
A new study conducted by scientists at the Rey Juan Carlos University of Madrid highlights the important role that parents play in the transmission of dentist fear in their family.
Previous studies had already identified the association between the fear levels of parents and their children, but they never explored the different roles that the father and the mother play in this phenomenon.
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Johns Hopkins scientists have created a free, Web-based tool to help patients decide whether it’s best to accept an immediately available, but less-than-ideal deceased donor kidney for transplant, or wait for a healthier one in the future.
Historically, the researchers say, it has been difficult, if not impossible, to accurately quantify the risk of accepting a deceased-donor kidney that may have been infected by hepatitis C, as compared to waiting what could be months or years for a better organ. There is a 5 to 15 percent chance of dying every year on the waiting list. Often, organs that may have been at risk of infection are thrown away and never transplanted.
In a new study the Johns Hopkins researchers showed there are some types of patients for whom survival benefit outweighs the risks of accepting a possibly infected organ. They then developed a Web-based mathematical model to help predict which patients they would be. The easy-to-use website can be found at www.transplantmodels.com/ird.
‘Because the supply of the healthiest donor organs is too small, patients need to consider all organ offers or risk dying while waiting for an organ. But this is a very hard decision, and many people turn down transplant offers that, in reality, would provide them significant benefit. Often they would have done much better taking the organ at hand than waiting for the next available one,’ says study leader Dorry L. Segev, M.D., Ph.D., an associate professor of surgery at the Johns Hopkins University School of Medicine. ‘This is the most important decision of a transplant candidate’s life, and we have developed a novel tool we believe can help patients make the best choice.’
EurekAlert
Clinical trial results published this week offer additional evidence that a new device may help relieve chronic heartburn symptoms that standard treatment cannot. The Keck Medical Center of USC was one of 14 U.S. and European medical centres to test the device prior to its March 2012 approval by the U.S. Food and Drug Administration.
John Lipham, M.D., associate professor of surgery at the Keck School of Medicine of USC, led clinical investigation of the device at USC as part of his ongoing work to find alternative ways to treat gastroesophageal reflux disease (GERD), or chronic heartburn.
‘These results show that there is another option for the millions of people suffering from chronic reflux,’ Lipham said. ‘Currently, the Keck Medical Center of USC is one of only 30 sites in the country certified to implant the device.’
The LINX Reflux Management System, manufactured by Minnesota-based Torax Medical, Inc., is like a bracelet made up of magnetic, titanium beads implanted around the end of the oesophagus, where the lower oesophageal sphincter is located. The lower oesophageal sphincter is the valve that prevents reflux, and GERD develops when this valve is weakened.
Implantation of the device is potentially an outpatient procedure that can be completed in 15 to 20 minutes, Lipham said.
Lipham and his colleagues assessed 100 patients with GERD before and after surgery, finding that acid reflux decreased, reflux symptoms improved and the use of medication to manage those symptoms decreased for most patients. Severe regurgitation was eliminated in all patients. More than 9 in 10 patients reported satisfaction with their overall condition after having the procedure, compared to 13 percent before treatment while taking medication.
Follow-up studies are still required to assess long-term safety.
Lipham says the LINX device is best for patients with mild to moderate reflux that cannot be adequately controlled by medication or for patients who do not want to take medication to manage the disease. More than 60 million Americans experience heartburn at least once a month and some studies have suggested that more than 15 million experience heartburn symptoms every day, according to the American College of Gastroenterology.
Surgical treatment of reflux disease had been limited to a procedure called a Nissen fundoplication, which involves recreating the oesophageal sphincter. While fundoplication is recommended for those with severe reflux, it is a complicated procedure that prevents the ability to belch or vomit and often leads to bloating or gas problems.
The most common adverse events experienced with the LINX included difficulty swallowing, pain when swallowing food, chest pain, vomiting, and nausea. It is important to note that patients with LINX will no longer be able to undergo magnetic resonance imaging (MRI) procedures. The magnetic beads interfere with the machine and can cause the device to be damaged and the patient to be injured. Keck Medical Center
Telestroke networks that enable the remote and rapid diagnosis and treatment of stroke can improve the bottom line of patients and hospitals, researchers report.
A central hub hospital delivering rapid stroke diagnosis and treatment partnering with typically smaller spoke hospitals in need of those services means more patients recover better and the network
White blood cells, or leukocytes, are the immune system’s warriors. So when an infection or disease attacks the body, the system typically responds by sending more white blood cells into the fray. This means that checking the number of these cells is a relatively easy way to detect and monitor such conditions.
Currently, most white blood cell counts are performed with large-scale equipment in central clinical laboratories. If a physician collects blood samples from a patient in the office
Lung cancer is likely to overtake breast cancer as the main cause of cancer death among European women by the middle of this decade, according to new research published. In the UK and Poland it has already overtaken breast cancer as the main cause of cancer deaths in women.
The study by researchers in Italy and Switzerland predicts that just over 1.3 million people will die from cancer (737,747 men and 576,489 women) in the 27 countries of the European Union in 2013. Although the actual numbers have increased when compared with 2009 (the year for which there are World Health Organisation mortality data for most EU countries), the rate (age-standardised per 100,000 population) of people who die from the disease has declined. Since 2009 there has been 6% fall among men and 4% fall among women.
However, despite the decline in cancer deaths overall, lung cancer death rates continue to rise among women in all countries, while breast cancer rates fall. In 2013 there will be an estimated 88,886 deaths (14.6 per 100,000 women) from breast cancer and 82,640 deaths (14 per 100,000 women) from lung cancer. Lung cancer deaths have risen by 7% among women since 2009.
One of the study
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