• Gene therapy to be tested in heart failure

    By Ben Hirschler, Reuters

    LONDON - British scientists are stepping up clinical tests of gene therapy in a bid to help people with advanced heart failure pump blood more efficiently.

    Researchers said on Tuesday they planned to enrol patients into two new clinical trials using Mydicar, a gene therapy treatment made by privately held U.S. biotech company Celladon.

    After more than 20 years of research, the ground-breaking method for fixing faulty genes is starting to deliver, with European authorities approving the first gene therapy for an rare metabolic disease last November.

    In the case of heart failure, the aim is to insert a gene called SERCA2a directly into heart cells using a modified virus, delivered via a catheter infusion. Lack of SERCA2a leads to ever weaker pumping in people with heart failure.

    Although drugs offer some relief, there is currently no way of restoring heart function and the prognosis for those with advanced disease is worse than for many cancers.

    One of the studies, led by scientists at Imperial College London, is part of a wider mid-stage Phase II project sponsored by Celladon that involves 200 patients worldwide, some of whom have already been treated in the United States and Denmark.

    The second trial, which is due to start in the summer, will test the same treatment in 24 British patients already fitted with mechanical heart pumps to see how the approach may help in this particular setting.

    It promises to be a long haul, with extensive Phase III studies still needed once results of the current mid-stage tests are received, which Celladon expects in the first half of 2015.

    Gene therapy has experienced a series of advances and setbacks over the decades. The most notable blow came in 1999 when an Arizona teenager died in a gene therapy experiment. More recent results, however, have been promising in fields ranging from immune system diseases to blindness.

    "It is a great example of the slow burn of good laboratory science translating into a potential clinical treatment," said Peter Weissberg, medical director of the British Heart Foundation, which is co-funding the second trial.

    Because gene therapy replaces or boosts the activity of a faulty gene, it offers the possibility of a one-time "fix" - and that creates an economic challenge.

    Any gene therapy is bound to be expensive, since a single dose could last a lifetime and the manufacturer will have just one shot at recouping its investment.

    But Alexander Lyon of Imperial College, lead investigator on both studies, said it could be a cost-effective solution in heart failure if it avoided the need for interventions such as heart transplants at 200,000 pounds ($300,000) each. 

    Comment

    Show more
    Explore related topics: , ,

  • Heart failure set to spike in US, Heart Association says

    By Maggie Fox, Senior Writer, NBC News

    Cases of heart failure – a chronic and expensive condition – are set to spike 46 percent by 2030, the American Heart Association projected on Wednesday.

    As Americans get older, and more of us develop various heart conditions, the number of those with heart failure will rise from 5 million now to 8 million in 2030, the group says.

    It’s a pricey condition to treat, and every American will end up paying $244 a year to care for people with the condition, through higher insurance rates and taxes, the Heart Association says.

    “If we don’t improve or reduce the incidence of heart failure by preventing and treating the underlying conditions, there will be a large monetary and health burden on the country,” said Dr. Paul Heidenreich of the Stanford University School of Medicine, who led the analysis published in the journal Circulation: Heart Failure.

    “Direct and indirect costs to treat heart failure could more than double from $31 billion in 2012 to $70 billion in 2030,” the Heart Association, which publishes the journal, said in a statement.

    “The costs will be paid for by every adult in this country, not just every adult with heart failure,” Heidenreich said.

    Heart failure developes when the heart has been weakened from heart disease, high blood pressure, diabetes or other conditions. As the heart struggles to pump, it can enlarge but it gets weaker. Fluid can build up around the heart, leaving patients tired and out of breath.

    Half of all Medicare patients hospitalized for heart failure die within three years.

    Treatments start out with cheap diuretics to get rid of the excess fluid, but it can quickly become expensive.  Heart failure is the leading cause of hospitalization for Americans over age 65. More effective dissemination and use of guideline-recommended therapy to prevent heart failure and improve survival.

    Patients and medical professionals alike need better information about how to treat the condition, the Heart Association said. Nurses, doctors, pharmacists and other professionals need specialized training, and certain ethnic groups, such as African-Americans, need targeted outreach.

    Related:

  • Breast cancer survivors may face second threat: heart failure

    By Maggie Fox, Senior Writer, NBC News

    Women who have survived breast cancer may have to fight another killer down the road -- heart failure, researchers report.

    They found a much higher rate of heart failure among breast cancer survivors than has previously been reported, and said their findings likely reflect the real-world risks that women have. The 12,000 women studied for the report had a 20 percent risk of developing heart failure over just five years if they got a common chemotherapy regimen, compared to just 3.5 percent of breast cancer patients who did not get chemo.

    "I think these drugs are critical to improving breast cancer survival," said Erin Aiello Bowles of the Seattle-based Group Health Research Institute, who led the study published in the Journal of the National Cancer Institute. "But these drugs are toxic. They are meant to target disease but they can often damage other parts of the body."

    Clinical trials of breast cancer patients -- designed to discover whether drugs fight disease and to show how safe they are -- have shown that the drugs can damage the heart and cause higher rates of heart failure. They generally demonstrate about a 4 percent increase in heart failure over three to five years for women getting chemo. But clinical trials usually involve a select group of patients who are healthy in other ways.

    Bowles said her team set out to look at real-world patients of all ages and with a range of health conditions on top of their breast cancer. They went through the medical records of women at eight health systems who were treated between 1999 and 2007 with two very common cancer drugs: a group of drugs called anthracyclines, such as adriamycin, and a targeted antibody drug called Herceptin or trastuzumab.

    Each drug raised the risk on its own, but the combination greatly raised heart failure rates.

    "It is important to note that these rates do vary by age," Bowles said in a telephone interview.  "They are much lower in the younger women." More than 40 percent of the women over the age of 75 who got a combination of an anthracycline and Herceptin also developed heart failure within five years. Just 13.7 percent of the breast cancer patients that age who did not get chemo developed heart failure.

    The study highlights a growing problem. The American Cancer Society estimates there are 12 million cancer survivors alive in the United States now. As many cancer patients survive their disease and lead ever-longer lives, they find they must fight second battles against the long-term effects of the treatments that saved their lives. Even so-called targeted therapies, which were designed to better target tumor cells while leaving healthy tissue alone, have been shown to cause long-lasting damage.

    And as they leave the care of a specialized oncologist and return to day-to-day care, they may not know they’re at special risk of other conditions – and their primary care doctors may not be aware, either. The American Society of Clinical Oncology has been warning about the problem for years, and released research at its annual meeting last June showing that 94 percent of primary care doctors didn't know about the potential long-term effects of drugs commonly used to treat breast and prostate cancer.

    Breast cancer is the leading cancer killer of U.S. women, after lung cancer. It is diagnosed in more than 220,000 women a year, according to the American Cancer Society, and will kill nearly 40,000 this year. About 20 percent of cases are a kind called HER-2 positive, and Herceptin was formulated to especially target this kind. It’s very effective and has saved thousands of lives, but it was known to also damage the heart, although doctors don’t understand just how.

    Heart failure is also very common. The National Heart, Lung and Blood Institute estimates 4.8 million Americans have congestive heart failure, which is a chronic condition in which the heart doesn’t pump blood effectively. Half of patients with heart failure die within five years, and 400,000 people get newly diagnosed every year.

    So what can women do if they’ve had chemo for breast cancer and want to watch their hearts?

    Cardiologist Dr. Larry Allen of the University of Colorado in Denver, who also worked on the study, said they first of all need to be educated about what drugs they have taken and what the side-effects are.

    “Second, patients should ask about what heart tests may be indicated before, during, and after treatment,” Allen said in a statement. These may include tests of how well the heart is pumping blood – tests that most women won’t get during a routine physical or well-woman visit.

    “Third, in addition to allowing doctors to monitor for heart problems, patients can monitor themselves for worsening heart function by understanding how heart problems may present -- including shortness of breath especially when lying flat, leg swelling, palpitations/heart fluttering, and exercise intolerance (these symptoms can represent non-heart disease too, but generally warrant additional evaluation),” Allen added.

    “Unfortunately, it is unknown if medications that are typically used to treat heart failure (such as beta-blockers and ACE inhibitors) might protect against heart damage from certain chemotherapy.”

    Related:

     

  • Men with heart failure more likely to die than women

    By MyHealthNewsDaily Staff

    Women with heart failure, a condition in which the heart fails to pump enough blood to meet the body's demands, may live longer than their male counterparts, a new study says.

    The results are based on an analysis of 31 studies involving 28,000 men and 14,000 women with long-term (chronic) heart failure who were followed for three years.

    After taking into account patients' age, men had a 31 percent higher risk of dying over the study compared with women, the researchers said.

    However, the absolute difference between men and women's mortality was only slight. Over the three year period, 25.3 percent of the women and 25.7 percent of the men died.

    The study is the largest to look at how gender affects risk of death for people with heart failure.

    A number of factors could explain the survival advantage in women, said study researcher Manuel Martinez-Selles, of the Gregorio Marañón University Hospital in Madrid. "The female heart appears to respond to injury differently from the male heart," Martinez-Selles said.

    For example, women appear to have less detrimental changes in heart function after an injury, and greater protection from irregular heartbeats, Martinez-Selles said.

    The study also found that overall women were prescribed fewer recommended treatments for heart failure than men, including angiotensin converting enzyme (ACE) inhibitors or angiotensin receptor blockers (ARBs) and beta blockers.