News Briefs

MR-guided right heart catheterization live streams at SCMR scientific sessions

 Interventional Cardiac Magnetic Resonance (ICMR) Program Team

The ICMR team who performed the livestream procedure during the member assembly session. The prestigious invitation came as a result of the innovative partnership between Children’s National and the NHLBI to form the Interventional Cardiac MR Program.

Cardiologists from the Interventional Cardiac Magnetic Resonance (ICMR) Program at Children’s successfully live streamed a right heart catheterization procedure guided by magnetic resonance (MR) imaging during the Member Assembly Session of the Society for Cardiac Magnetic Resonance Scientific Session in early February.

The ICMR program is a first-in-the-nation partnership between Children’s National and the National Heart, Lung, and Blood Institute that features a state-of-the-art dedicated cardiac specific MRI suite for diagnosis, evaluation and intervention for children with heart conditions. The program’s goal is to advance diagnostic and interventional cardiac magnetic resonance imaging techniques in pediatric cardiology and for adults with congenital heart disease. ICMR is cross-disciplinary, connecting researchers, clinicians, engineers and physicists to provide more precise and less invasive diagnostics and treatment options that also reduce radiation exposure for vulnerable patients.

 

Muscular Dystrophy Association awards grants to two Children’s National scientists

Marshall Hogarth, Ph.D

Marshall Hogarth, Ph.D

James Novak, Ph.D.

James Novak, Ph.D.

Two Children’s National Health System research scientists, Marshall Hogarth, Ph.D. and James Novak, Ph.D., have received Post-Doctoral Development Grants from the Muscular Dystrophy Association (MDA) as part of funding awarded to young, rising researchers who are poised to become independent investigators.

Over the next three years, Hogarth and Novak will be allotted $180,000 each to underwrite their individual research projects.

Hogarth’s research is focused on limb-girdle muscular dystrophy (LGMD), a disease which presents as muscle weakness when patients are in their late teens before rapidly progressing to severe debilitation. The MDA grant will allow Hogarth to continue his research investigating the replacement of muscle with fatty tissue and the role this plays in the late onset and subsequent progression of LGMD in patients.

Novak focuses mainly on researching Duchenne Muscular Dystrophy (DMD), a severely debilitating form of MD, that leads to progressive muscle weakness and respiratory and cardiac failure. Currently, the only Food and  Drug Administration (FDA)  approved treatment for DMD is exon-skipping. The MDA grant will support Novak’s study of the mechanisms that regulate the delivery of exon-skipping drugs in muscle, in order to identify new therapeutic targets and improve drug efficacy for patients with DMD.

While Hogarth and Novak focus on different aspects of neuromuscular disease, both look forward to making significant contributions that lead to overall improvements in the treatment of patients impacted by muscular dystrophy.

Javad Nazarian named scientific director of the Brain Tumor Institute

Javad Nazarian

Javad Nazarian, Ph.D., has been named scientific director of the Brain Tumor Institute of the Children’s National Health System. Since 2006, Dr. Nazarian has been an active member of the Brain Tumor Institute, contributing to the advancement in understanding pediatric brain tumors.

He has been instrumental in his role as a Principal Investigator in the Center for Cancer and Immunology Research where his laboratory actively investigates the molecular mechanisms of diffuse intrinsic pontine gilomas (DIPGs) and establishes preclinical models of pediatric brain tumors.

Dr. Nazarian has also contributed to the expansion of the comprehensive biorepository at Children’s National, growing from 12 samples six years ago to more than 3,000 specimens donated by more than 900 patients with all types of pediatric brain tumors, including DIPG. Recently he was appointed Scientific Co-chair of the Children’s Brain Tumor Tissue Consortium.

Doctor holding preemie's hand

Children’s National creates first-of-its-kind Rare Disease Institute

Children’s National Health System has announced the creation of the Children’s National Rare Disease Institute (CNRDI), a first-of-its-kind clinical research center focused exclusively on the care and treatment of children and adults with rare genetic disorders. Designated by the National Organization for Rare Disorders (NORD) as the first Center of Excellence for Clinical Care for Rare Diseases, the CNRDI will focus on developing care standards for patients, advancing research and developing new therapies to better treat rare diseases. The institute will be led by Marshall Summar, M.D., Chief of Genetics and Metabolism at Children’s National, and will collaborate with the NORD natural history/registry program, which was developed with input from the U.S. Food and Drug Administration and the National Institutes of Health to advance monitoring databases for patient outcomes and disease.

Bariatric Surgery Program achieves national accreditation

Children’s National Health System’s Bariatric Surgery Program has been accredited by the Metabolic and Bariatric Surgery Accreditation and Quality Improvement Program (MBSAQIP®), a joint program of the American College of Surgeons and the American Society for Metabolic and Bariatric Surgery . The designation makes Children’s National one of the first children’s hospitals to receive such accreditation, and the only hospital in the area to be accredited to perform bariatric surgery on adolescents.

Read the full press release.

Relationship between sedentary time and youth cardiovascular health

Heart disease is the leading cause of death in the United States, yet for many individuals it is preventable through modifications in diet, physical activity, and sedentary time – all major risk factors. Jacob Hartz, M.D., M.P.H., a cardiology fellow at Children’s National Health System, presented findings from his research on youth cardiovascular health during the American Heart Association (AHA) Scientific Sessions, held November 12-16 in New Orleans. The AHA Scientific Sessions featured the latest developments in science and cardiovascular clinical practice, including all aspects of basic, clinical, population, and translational science. Dr. Hartz spoke on “The Relationship Between Clustering of Cardiovascular Health Behaviors and Physical Fitness Among U.S. Adolescents – Data from the National Health and Nutrition Examination Survey,” and shared how grouping children with risk factors into clusters enabled researchers to pinpoint sedentary time as the leading predictor of heart health.

Read more about Children’s National AHA Scientific Sessions speakers.

Down syndrome indicators in diverse populations

According to a large international study published in the American Journal of Medical Genetics, physical features vary in patients with Down syndrome across diverse populations. The study, led by the National Human Genome Research Institute (NHGRI), part of the National Institutes of Health, used an objective digital facial analysis technology developed by the Sheikh Zayed Institute for Pediatric Surgical Innovation at Children’s National Health System to identify the most relevant facial features characteristic in Down syndrome in diverse populations from 12 countries. This study is the first to compare and contrast Down syndrome across diverse populations. It is the first in a series of studies to be used in the NIH’s Atlas of Human Malformation Syndromes in Diverse Populations, a free resource to help clinicians around the world diagnose birth defects and genetic diseases in people of diverse ancestry, and is the first in a series focused on different genetic syndromes.

Read more here.

Link between population health and heart disease

Gerard Martin

Although clinical advances have improved treatments and mortality among patients with cardiovascular disease, heart disease remains the leading cause of death worldwide. Gerard Martin, M.D., cardiologist and medical director of Global Health at Children’s National and Chair of the American College of Cardiology’s Population Health Policy and Promotion Committee shares how cardiologists can improve outcomes by focusing on the link between population health and heart disease in a just-published article in Cardiology.

Read more.

How mindfulness can improve adolescent heart health

Could yoga, meditation, group support, or online video chats improve outcomes for adolescents with cardiac diagnoses? Vicki Freedenberg, Ph.D., R.N., electrophysiology nurse scientist at Children’s National Health System, shared insights from her research using these interventions at the American Heart Association (AHA) Scientific Sessions, held November 12-16 in New Orleans. The AHA Scientific Sessions featured the latest developments in science and cardiovascular clinical practice, including all aspects of basic, clinical, population, and translational science. In her presentation titled “Mindfulness Based Stress Reduction and Group Support Decrease Stress, Anxiety, and Depression in Adolescents with Cardiac Diagnoses: A Randomized Two-Group Study,” Freedenberg presented findings from her study comparing outcomes between cardiac patients who participated in a Mindfulness Based Stress Reduction program, which used meditation, yoga, and group support, and a second group that participated in a clinician-led online video support group with peers. Early results suggest that stress significantly decreased in both groups, and higher baseline anxiety and depression scores predicted lower levels of post-intervention anxiety and depression.

Read more about Children’s National AHA Scientific Sessions speakers.

Advances in 3-D imaging and printing

Laura Olivieri

The emerging field of 3-D printing and 3-D digital displays has the potential to change the way surgeons and cardiologists make decisions. Laura Olivieri, M.D., a cardiologist at Children’s National Health System, shared insights on this topic during the American Heart Association (AHA) Scientific Sessions, held November 12-16 in New Orleans. The AHA Scientific Sessions featured the latest developments in science and cardiovascular clinical practice, including all aspects of basic, clinical, population, and translational science. In her presentation “Cardiovascular Imaging for 3-D,” Dr. Olivieri explained how cardiovascular imaging physicians can “image gently” to create 3-D models while reducing patients’ exposure to radiation and sedation. She also shared best practices for producing 3-D models from magnetic resonance imaging and echocardiograms, which are often underutilized for this purpose.

Read more about Children’s National AHA Scientific Sessions speakers.

Children’s National gastroenterologist honored with Excellence of Healthcare Award

Benny Kerzner, M.D., gastroenterologist and creator of the Division of Gastroenterology, Hepatology, and Nutrition at Children’s National Health System, received the Excellence of Healthcare award from the Crohn’s & Colitis Foundation of America (CFFA) – Greater Washington, D.C. and Virginia Chapters during the organization’s Evening of Hope Gala on November 17, 2016. The award is presented to a medical professional who has positively impacted patient lives and furthered the mission of the CCFA to cure Crohn’s disease and ulcerative colitis and to improve the quality of life of children and adults affected by these diseases. Learn more here.

Postdoctoral researcher receives Urology Care Foundation Research Scholar Award

Evaristus Mbanefo, Ph.D., M.S., M.Sc., a postdoctoral researcher at Children’s National Health System, was awarded the 2017 Urology Care Foundation Research Scholar Award in recognition of his potential for a successful career in urology research. Mbanefo’s project, “Therapeutic Exploitation of Interleukin-4-Inducing Principle from Schistosoma Mansoni Eggs: A Urogenital Parasite-Derived Host Immunomodulatory Protein for Hemorrhagic Cystitis and Bladder Hypersensitivity,” was among 70 applications for the highly competitive annual award. He works in the lab run by Michael H. Hsieh, M.D., Ph.D., Director of the Clinic for Adolescent and Adult Pediatric Onset Urology (CAPITUL) at Children’s National.

Thrasher Research Fund supports Zika virus neurologic outcomes study

The Thrasher Research Fund will fund a Children’s National project, “Neurologic Outcomes of Apparently Normal Newborns From Zika Virus-Positive Pregnancies,” as part of its Early Career Award Program, an initiative designed to support the successful training and mentoring of the next generation of pediatric researchers.

The project was submitted by Sarah B. Mulkey, M.D., Ph.D., a fetal-neonatal neurologist who is a member of the Congenital Zika Virus Program at Children’s National. During the award period, Dr. Mulkey will be mentored by Adre du Plessis, M.B.Ch.B., director of the Fetal Medicine Institute, and Roberta L. DeBiasi, M.D., M.S., chief of the Division of Pediatric Infectious Diseases. Drs. du Plessis and DeBiasi co-direct the multidisciplinary Zika program, one of the nation’s first.

In the award letter, the fund mentioned Children’s institutional support for Dr. Mulkey, as demonstrated by the mentors’ letter of support, as “an important consideration throughout the funding process.”

Doctors working together to find treatments for autoimmune encephalitis

Children’s and Regeneron partner in exome sequencing study

Children’s National, in partnership with the Regeneron Genetics Center (RGC, a subsidiary of Regeneron Pharmaceuticals, Inc.), has announced the launch of a major three-year research study that will examine the links between undiagnosed disease and an individual’s genetic profile.

The program, directed by Children’s National Geneticist Carlos Ferreira Lopez, M.D., and coordinated by Genetic Counselor Lindsay Kehoe, hopes to include as many as 3,000 patients in its initial year and even greater numbers in the following two years.

During the course of the study, RGC will conduct whole exome sequencing (WES) to examine the entire protein-coding DNA in a patient’s genome. Children’s National geneticists will analyze and screen for certain findings that are known to be potentially causative or diagnostic of disease. Children’s National scientists and providers will confirm preliminary research findings in a lab certified for Clinical Laboratory Improvement Amendments (CLIA), per federal standards for clinical testing, and refer any confirmatory CLIA-certified cases to appropriate clinicians at Children’s National for further care.

According to Marshall Summar, M.D., Chief of Genetics and Metabolism at Children’s National, the WES study could finally provide patients and their families with the medical answers they have been looking for, allowing for treatment appropriate to their specific genetic condition.

Because pediatric diseases can often elude diagnosis, they can pose a number of detrimental effects to patients and their families, including treatment delays, multiple time- and cost-intensive tests, and stress from lingering uncertainty regarding the illness. With this genomic data, Regeneron will be able to utilize findings to continue its efforts to improve drug development.

Since its inception in 2014, the RGC has strategically partnered with leading medical institutions to utilize human genetics data to speed the development and discovery of new and improved therapies for patients in need.

Children’s National spin-off ReveraGen announces agreement with actelion for Duchenne Muscular Dystrophy treatment

Earlier this month, ReveraGen BioPharma announced an exclusive option agreement with Actelion Ltd for lead compound vamorolone, a non-hormonal steroid modulator that is primarily used for the treatment of Duchenne Muscular Dystrophy (DMD). ReveraGen, the first Children’s National private spin-off company, is engaged in the discovery and development of proprietary therapeutic products for neuromuscular and inflammatory diseases.

Under the terms of the license agreement, Actelion and ReveraGen will partner to research and co-develop the novel compound vamorolone, which holds the potential to preserve muscle function and prolong ambulation in DMD patients, without some of the side effects that are commonly associated with glucocorticoid therapy. Those commonly associated include growth stunting and immune suppression, which can pose significant challenges for very young patients.

ReveraGen completed Phase I clinical trials for vamorolone in late 2015, and a Phase IIa program is currently underway to investigate the safety and efficiency of vamorolone in male DMD patients between four and seven years of age who have not taken deflazacort or prednisone. A Phase IIb program is also in early planning stages.

ReveraGen Co-Founder and CEO Eric Hoffman, PhD, has worked on DMD since the late 1980’s and has led his own research group for nearly 20 years at Children’s National. He co-founded ReveraGen back in 2008 with John McCall, PhD and Kanneboyina Nagaraju, PhD, DVM, before being named CEO in 2014. Children’s National maintains a 38 percent stake in ReveraGen.

Brain tumor expert from Children’s National speaks at Society for Neuro-Oncology’s scientific meeting and Education Day

Roger Packer

Roger Packer, M.D., Senior Vice President for the Center of Neuroscience and Behavioral Medicine and Director of the Brain Tumor Institute at Children’s National Health System, will be speaking at the 21st Annual Meeting and Education Day of the Society for Neuro-Oncology. From November 17-20, 2016, the conference will gather neuro-oncologists, medical oncologists, adult and pediatric neurosurgeons, pediatric neuro-oncologists, neuroradiologists, neuropathologists, radiation oncologists, neuropsychologists, and epidemiologists from across the country to discuss the future of neuro-oncology. Dr. Packer will be sharing his expertise in treating neurofibromatosis and pediatric brain tumors. He also will be part of a working group to discuss guidelines for response assessment in PDCT-13 medulloblastoma and other leptomeningeal seeding tumors.

2016 Neurobiology of Disease in Children Symposium: neurofibromatosis features Children’s National expert

Roger Packer, M.D., Senior Vice President for the Center of Neuroscience and Behavioral Medicine and Director of the Gilbert Neurofibromatosis Institute at Children’s National Health System, was an invited speaker at the 2016 Neurobiology of Disease in Children Symposium: Neurofibromatosis (NF). This conference brought together experts from around the world to discuss the newest developments in the understanding and treatment of children with NF. While at the conference, which was held on October 26, 2016, Dr. Packer gave an update of the Department of Defense-sponsored Neurofibromatosis Clinical Trial Consortium. The Neurofibromatosis Clinical Trials Consortium, of which Dr. Packer is the group chair, was established by the Department of Defense Neurofibromatosis Research Program to develop and perform clinical trials for the treatment of NF complications in children and adults.

Read more about the symposium.

Brain Tumor Institute Director Speaks at Coalition against Childhood Cancer Meeting

Roger Packer, MD, Senior Vice President for the Center of Neuroscience and Behavioral Medicine and Director of the Brain Tumor Institute at Children’s National Health System, was an invited speaker at the Coalition Against Childhood Cancer meeting at Cold Springs Harbor Laboratory on October 31 and November 1, 2016. This international conference was a unique collaborative effort between multiple foundations, the National Cancer Institute, and industry experts to develop a new path forward for the treatment of childhood cancer. Dr. Packer spoke on “Pediatric Brain Tumors: Where Are We Now” and shared his expertise in treating pediatric brain tumors and what he hopes the future of pediatric brain tumor research will look like. Pediatric brain tumors recently surpassed leukemia as the most deadly form of childhood cancer.

Read more about the conference here.

Efficacy of family-centered advanced care planning for adolescents with HIV and their families

Led by experts at Children’s National Health System and the Adolescent Palliative Care Consortium, a new study published in Pediatrics reports that pediatric advanced care planning (pACP) can provide a positive environment for adolescents with Human Immunodeficiency Virus (HIV) and their families to discuss end of life care. Being born with HIV increases an adolescent’s risk of dying from an opportunistic infection or chronic illness, underscoring the need for pACP and the significance of this research.

Read more here.

eKare wins Washington Business Journal 2106 Innovation Award

eKare, Inc., a spin-off company out of the Sheikh Zayed Institute for Pediatric Surgical Innovation at Children’s National, was selected as a Washington Business Journal 2016 Innovation Award honoree for their 3D wound assessment technology.

eKare’s inSight is a cost efficient solution that enables healthcare providers to take accurate and reliable measurements of wounds using a technology that is built on top of an iPad. The co-founder, Kyle Wu, M.D., a former Joseph E. Robert Jr. Fellow, worked with engineers and clinicians from the Sheikh Zayed Institute to develop the technology because he was frustrated with the primitive and subjective nature of standard wound measurement practices, which involve using a disposable ruler to measure the wound length/width and a Q-tip inserted in the wound to determine its depth.

In 2014, eKare spun off from the Sheikh Zayed Institute and, by November 2015, launched its product.

The Washington Business Journal Innovation Awards honor the most innovative people, places, and products in Greater Washington. eKare was one of the 15 honorees in the inaugural Innovation Awards.