Cardiology Meeting, Superbugs, Ethics of MRT

Researchers at The Children’s Hospital of Philadelphia were on the road this week. Our weekly In The News update first takes us to sunny Orlando, Fla., where the 19^th Annual Update on Pediatric and Congenital Cardiovascular Disease began Feb. 24. The theme of the nation's largest pediatric cardiology conference is "Bringing Science into Clinical Practice" this year.

An international group of more than 800 medical experts gathered to discuss the latest research and clinical practices to provide optimal care for fetuses, neonates, children, and young adults with congenital heart disease (CHD). Affecting one in 120 children, CHD is the most common birth defect, and in its most severe forms is also the leading cause of death in infants and young children.

“The practice of pediatric and congenital cardiovascular care involves numerous conditions presenting at a variety of ages and complexities,” said Jack Rychik, MD, course director and medical director of the Fetal Heart Program and the Single Ventricle Survivorship Program at CHOP. “A myriad of various congenital malformations of the cardiovascular system exist in nature, each with its own unique pathophysiology. Previously healthy young hearts may also acquire disease. All of these factors contribute to the need for a more evidence-based research findings, which must be available to healthcare providers in order to offer the latest state-of-the-art care to our patients. Strong evidence for clinical practice can often be difficult to find due to all of the permutations of disease variety and complexity."

Eight oral presentations by researchers competing for the Outstanding Investigator Award will be featured during the conference — with the winner announced Feb. 27.

See the press release for more information.

Fighting Superbugs

The ability of bacteria or other microbes to resist the effects of antibiotics, known as antibiotic resistance, has been called one of the world's most pressing public health problems. As many types of bacteria become stronger, a super heroic response is needed. Enter Supermoms Against Superbugs, a movement of mothers, fathers, grandparents, and other caregivers who are fighting antibiotic resistance by working to end the overuse of these lifesaving drugs in food animal production.

At its annual lobby days held Feb. 24-25 in Washington, D.C., members advocated for increased funding to combat the growing public health threat of drug-resistant bacterial infections. Superdad Theoklis Zaoutis, MD, MSCE, is chief of the Division of Infectious Diseases at CHOP and the Thomas Frederick McNair Scott professor of Pediatrics and professor of Epidemiology at the Perelman School of Medicine at the University of Pennsylvania.

Read more about Dr. Zaoutis’ in a Q&A and find out about his current research focusing on addressing the problem of antibiotic resistance and improving antibiotic use.

Exploring Ethics of Mitochondrial Replacement Techniques

Mitochondrial replacement techniques (MRTs), aim to prevent mother-to-child transmission of mitochondrial DNA disease — a complex set of rare disorders caused by defective DNA within the mitochondria, the tiny energy-producing structures existing outside the nucleus of cells. Such disease may present at any age or level of severity, attacking a combination of organs and systems, often fatally.

But these emerging tools raise important ethical, social, and policy questions. Earlier this month, an expert panel of national experts formed by the Institute of Medicine at the request of the U.S. Food and Drug Administration recommended that research into MRTs should proceed under careful oversight. Three members of that panel, including Marni J. Falk, MD, director of the Mitochondrial-Genetics Clinic at The Children's Hospital of Philadelphia, wrote a Perspective article in the New England Journal of Medicine on MRTs’ implications for clinicians.

"Even though MRTs would not confer health benefits on patients who already have mitochondrial DNA diseases, many patients with a known risk for transmitting such a disease to their offspring are highly motivated to prevent that from occurring," Dr. Falk said. "That motivation is clear when we counsel patients and families affected by mtDNA disease, and recent patient surveys have reinforced that preventing disease transmission is a prevailing concern in this population."

Read more in the press release.