Research Yields Innovations in the Treatment of Pediatric Patients

In July 2019, Karen Murray, MD, joined Cleveland Clinic as Chair of the Pediatric Institute, Physician-in-Chief of Cleveland Clinic Children’s and President of Cleveland Clinic Children’s Hospital for Rehabilitation. In her new role, Dr. Murray leads a group of more than 300 pediatric specialists who are leaders in research for cardiac care, neurological conditions, digestive diseases and other conditions.

In this Q&A, Dr. Murray shares her research interests and how she balances academic pursuits with her clinical practice.

Q: You were instrumental in revolutionizing the treatment of hepatitis C virus (HCV) in children and teens. Can you tell us more about that research?

Dr. Murray: There are a number of highly effective treatment regimens available to adults with HCV; however, the treatment options for children and adolescents do not reflect these medical advances. We conducted several trials of the direct acting antivirals ledipasvir and sofosbuvir for the treatment of chronic HCV infection in children. These two new medications were approved for use in adults about six years ago. They have no significant adverse effects and virtually eradicate the virus, with sustained virologic response after 12 weeks of therapy (SVR12) reached in 97%-100% of patients, depending on genotype.1-3 In 2017, the medications were approved by the FDA for use in pediatric patients with genotype 1, 4, 5 or 6 chronic HCV infections, who are 12 years of age or older and weigh more than 35kg. Very recently, we published trials that extended the use of these medications to children aged 3 to 12 years old, which found similar SVR12 rates.4,5 Before these medications were available, we relied on interferon-based therapies, which were less effective and poorly tolerated. These drugs dramatically change treatment outcomes for our patients.

Q: What do you consider to be the most exciting areas of opportunity in pediatrics in the next 5-10 years?

Dr. Murray: Innovations in therapeutics — I think we are on the precipice of changes that can really benefit children, such as immunotherapies, gene therapy, and the creation of medications to reverse physiological challenges. Take muscular dystrophy, for example — children suffer and ultimately succumb to many of these illnesses. With the new therapies, we can replace missing proteins, prevent further muscle degeneration, and in turn reduce suffering and perhaps even reverse the course of the disease.

Q: How can physicians optimize their research time?

Dr. Murray: As physicians, we face a variety of demands on our time and energy, compounded by the needs for high-utilization of our clinics, patient access, and of course quality and safety, while sometimes practicing with suboptimal support. In the academic world, the biggest driver of burnout is when a provider is unable to spend time in activities they value the most due to these time inefficiencies or administrative tasks that erode time. I want to assure that, culturally and operationally, physicians have the ability to optimize the time spent in the various aspects of their professional lives, and can protect their non-clinical time as intentionally as they should be focusing their clinical work. At Cleveland Clinic Children’s, I hope to support academic productivity, safeguarding administrative, research and teaching time, and encourage a culture in which these activities are valued and celebrated as much as excellence in clinical care.

References

1Balistreri WF, Murray KF, Rosenthal P, et al. The safety and effectiveness of ledipasvir-sofosbuvir in adolescents 12-17 years old with hepatitis c virus genotype 1 infection. Hepatology. 2017:66(2):371-378.

2Wirth S, Rosenthal P, Gonzalez-Peralata RP. Sofosbuvir and ribavirin in adolescents 12-17 years old with hepatitis c virus genotype 2 or 3 infection. Hepatology. 2017;66(4):1102-10.

3Murray KF, Balistreri WF, Bansal S, et al. Safety and efficacy of ledipasvir-sofosbuvir with or without ribavirin for chronic hepatitis c in children ages 6-11. 2018;68(6):2158-66.

4Rosenthal P, Schwarz KB, Gonzalez-Peralta RP, et al. Sofosbuvir and ribavirin therapy for children 3 to <12 years old with hepatitis c virus genotype 2 or 3 infection. Hepatology. 2019 Jun 20. Doi: 10.1002/hep.30821. [Epub ahead of print]

5Schwarz KB, Rosenthal P, Murray KF, et al. Ledipasvir-sofosbuvir for 12 weeks in children 2 to <6 years old with chronic hepatitis c. Hepatology. 2019 June 20. Doi: 10.1002/hep.30830. {Epub ahead of print]

About Karen Murray, MD

Dr. Murray joined Cleveland Clinic from the University of Washington School of Medicine in Seattle, where she served as Vice Chair of Clinical Affairs for the Department of Pediatrics and Professor and Chief of the Division of Gastroenterology and Hepatology. Dr. Murray held several leadership positions at Seattle Children’s Research Institute, including serving on the Steering Committee of the Center for Clinical and Translational Research, and as co-leader for research recruitment for Seattle Children’s Strategic Development. Most recently, she was Interim Chair of the Department of Pediatrics and Pediatrician-in-Chief of Seattle Children’s Hospital.