Neurology & Neurosurgery

Children's National Hospital's 2023-2024 Academic Annual Report on a tablet

Children’s National delivers on the promise in 2024

Children's National Hospital's 2023-2024 Academic Annual Report on a tablet

The Children’s National 2023-2024 Academic Annual Report show on a tablet.

Children’s National Hospital has released its 2023-2024 Academic Annual Report, showcasing a year of transformative progress in pediatric medicine. The report highlights achievements across its research centers, institutes and Innovation Ventures, underscoring the hospital’s role as a leader in advancing child health through innovation and collaboration.

“This year’s report reflects the remarkable progress we have made in advancing the frontiers of pediatric medicine,” said Nathan Kuppermann, MD, MPH, Chief Academic Officer and Chair of Pediatrics. “It highlights groundbreaking work across our research centers, institutes, and Innovation Ventures, showcasing the collaborative spirit that drives our mission forward. These achievements underscore our shared commitment to delivering transformative research and the best possible outcomes for children and families.”

Delivering across centers

The report captures the contributions of each of Children’s National’s research centers, each pushing the boundaries of pediatric healthcare:

  • Center for Cancer & Immunology Research (CCIR): Delivering on the promise of cell and gene therapies, offering innovative treatments for pediatric cancers and immune disorders.
  • Center for Genetic Medicine Research (CGMR): Advancing pediatric genetic medicine through interdisciplinary efforts, addressing complex genetic conditions with cutting-edge science.
  • Center for Neuroscience Research (CNR): A year of growth in scientific excellence, advancing the understanding of brain development and neurological conditions.
  • Center for Prenatal, Neonatal & Maternal Health Research (CPHNMR): Revolutionizing neonatal care with its pioneering infant brain health neuromonitoring program.
  • Center for Translational Research (CTR): Facilitating groundbreaking work by new K awardees and driving translational research to bridge the gap between discovery and clinical care.
  • Sheikh Zayed Institute for Pediatric Surgical Innovation (SZI): Leading the way in advanced research projects in pediatric surgery, pushing technological boundaries to improve outcomes for children worldwide.

Taking the lead in innovation through collaboration

Innovation Ventures at Children’s National is advancing pediatric health security, addressing unique challenges with transformative solutions. Meanwhile, the Children’s National Research & Innovation Campus (CNRIC) continues to thrive as a hub for discovery and collaboration, hosting conferences on topics like artificial intelligence in healthcare, cell and gene therapy, and pediatric epilepsy research.

A vision for the future

The report also highlights Children’s National’s focus on integrating cutting-edge technologies like artificial intelligence into its research and clinical practices, as well as addressing global health challenges such as the effects of climate change on children’s health. These efforts reflect the hospital’s commitment to improving outcomes for children everywhere through innovation, teamwork, and forward-thinking leadership.

The 2023-2024 Academic Annual Report serves as a testament to the dedication and expertise of the Children’s National community, showcasing how collaboration and innovation are shaping the future of pediatric healthcare.

Dr. Gaillard, Dr. Schreiber and Dr. Cohen from Children’s National Hospital at the 2024 American Epilepsy Society meeting.

Advancing epilepsy care: Highlights from the 2024 American Epilepsy Society Conference

Dr. Gaillard, Dr. Schreiber and Dr. Cohen from Children’s National Hospital at the 2024 American Epilepsy Society meeting.

Dr. Gaillard, Dr. Schreiber and Dr. Cohen from Children’s National Hospital at the 2024 American Epilepsy Society meeting.

The 2024 American Epilepsy Society (AES) conference, held over five days, brought together healthcare professionals, scientists and industry leaders from around the globe. This annual event focuses on advancing outcomes for people with epilepsy through educational sessions, cutting-edge research and the latest technological innovations.

Recognized amongst his peers as an international leader in epilepsy research, William D. Gaillard, M.D., was honored with the 2024 Clinical Science Research Award at this year’s meeting. His pioneering work has significantly enhanced our understanding of epilepsy’s impact on brain structure and has led to improvements in surgical interventions and patient outcomes. Dr. Gaillard’s ongoing contributions to enhancing the lives of children with epilepsy earned him the Society’s most prestigious research award.

Dr. Gaillard serves as chief of the Divisions of Child Neurology and of Epilepsy and Neurophysiology, Director of the Comprehensive Pediatric Epilepsy Program and Associate Director of the Center for Neuroscience Research at Children’s National Hospital.

In addition to Dr. Gaillard, experts from the Comprehensive Pediatric Epilepsy Program at Children’s National were invited to present their research, insights and innovations during the conference. As a recognized Level 4 epilepsy center by the National Association of Epilepsy Centers, the program’s collaborative team is recognized worldwide for their leadership in improving the lives of children with epilepsy through personalized care, advanced technology and groundbreaking research.

Highlights from AES 2024 include:

Sanam Zarei, M.D., Children’s National Hospital Neurology fellow, presenting a poster at AES 2024.

Dr. Zarei, Children’s National Hospital Neurology fellow, presenting a poster at AES 2024.

Poster presentations:

  • Asymmetrical Hippocampal-cortical Connectivity Along Lateral-Medial Axis in Pediatric Focal Epilepsy Patients by Hua Xie, Ph.D., Chloe A. Hooker, William D. Gaillard, M.D.
  • Comorbidities and Concerns in Hypothalamic Hamartoma: Results of an International Caregiver Survey by Nathan Cohen, M.D.
  • Automated and Interpretable Detection of Hippocampal Sclerosis in temporal lobe epilepsy: AID-HS by Nathan Cohen, M.D.
  • Characteristics and Outcomes of Pediatric Patients with Prolonged Convulsive Status Epilepticus by William D. Gaillard, M.D.
  • High Accuracy for Detecting Tonic Clonic seizures (TCSs) Using an Apple Watch Seizure Detector by Dewi Depositario-Cabacar, M.D., William Gaillard, M.D.
  • Patient and Pre-surgical Testing Characteristics Contributing to Duration of Evaluation: Identifying Best Practices and Opportunities to Streamline Pediatric Epilepsy Surgery Evaluation by Dewi Depositario-Cabacar, M.D., William Gaillard, M.D.
  • Seizure Burden in Infants with Hemimegaloencephaly pre- and post Staged Transarterial Embolization by Ersida Buraniqi, M.D., Shani Israel, Lindsay Ruffini, CPNP, CPNP-AC, CPNP-PC, Tammy Tsuchida, M.D., Ph.D., Tayyba Anwar, M.D.
  • Acute Postoperative Seizures (APOS) in Pediatric Epilepsy Surgery Occurring Less Than 7 Days versus Greater Than 7 to 30 Days: Is There a Difference in Long Term Outcome? by Sanam Zarei, M.D., Kathryn Havens, PA-C, Nathan Cohen, M.D., John Schreiber, M.D., Thuy-Anh Vu, M.D., William Gaillard, M.D., Dewi Depositario-Cabacar, M.D.

Invited speakers and topics:

  • Speaker: John Schreiber, M.D., Moving Forward: Epilepsies with Concomitant Movement Disorders
  • Speaker: Laura Tochen, M.D., Moving Forward: Epilepsies with Concomitant Movement Disorders
  • Speaker: William Gaillard, M.D., US Health Care Inequities for People with Epilepsy
  • Session Moderator: Nathan Cohen, M.D., Neuroimaging

Learn more about the Comprehensive Pediatric Epilepsy Program at Children’s National Hospital.

illustration of brain with brainwaves

In the news: The role of genetic testing in pediatric epilepsy diagnosis and treatment

illustration of brain with brainwaves

Given the wide variety of potential causes and seizure types, diagnosing pediatric epilepsy can be complex.

Pediatric epilepsy affects approximately 0.5-1% of children, with an increased prevalence in those with developmental disabilities. Epilepsy’s pathophysiology involves a range of factors including genetic mutations, structural brain abnormalities and metabolic disorders. Given the wide variety of potential causes and seizure types, diagnosing pediatric epilepsy can be complex.

Previously, finding the cause of pediatric epilepsy often involved laborious, piecemeal metabolic and chemical tests, making the process complicated for both patients and families. Technological advancements over the past decade, such as next-generation sequencing, have made genetic testing a vital tool in diagnosing and managing the disease through personalized treatment, leading to better outcomes.

In recent discussions, John Schreiber, M.D., explores the impact of genetic testing on pediatric epilepsy, highlighting its benefits, challenges and evolving role in treatment. Dr. Schreiber serves as Associate Chief of Epilepsy and Electroencephalography Operations, Medical Director of Electroencephalography and Medical Director of the Epilepsy Genetics Program at Children’s National Hospital.

“Genetic testing is becoming easier and less expensive to perform in patients with unexplained epilepsies, and thankfully now is being used much earlier in a patient’s epilepsy course,” Dr. Schreiber says. “It’s important to try to make that diagnosis early so we can ensure patients have the appropriate interventions early and avoid unnecessary tests and things that they don’t need.”

You can watch the full dialogue, “The Role of Genetic Testing on the Management Pediatric Epilepsy” on NeurologyLive.

James and family mountain biking

Genomics solves puzzles for patients with rare disease

Drs. Berger and WIlson

Dr. Seth Berger (right) and pediatric resident Dr. Kirkland Wilson.

Most patients with rare diseases still lack answers. Families may undergo years of searching in an often painful diagnostic odyssey.

Research by Seth Berger, M.D., Ph.D., a medical geneticist in our Center for Genetic Medicine Research and Rare Disease Institute, aims to harness technologies to shorten this journey and connect families with help sooner. Dr. Berger often publishes accounts of medical mysteries he has solved.

“It’s truly stunning what genetic sequencing can find. The outcomes can be life-changing. These cases with life-altering diagnoses don’t come along every day, but when they do, they make the hunt to find answers all the more worthwhile,” says Dr. Berger.

James finds a path to health

James, age 15, struggled a lot before a researcher at Children’s National found the needle in the haystack of his genome. Four years ago, he could not walk in a straight line down the sidewalk. Enjoying Halloween trick or treating in fall or a beach hike in summer? Out of the question. His gait had become increasingly unsteady.

Everything changed the day that Dr. Berger took a look at James’ exome — a subset of the genome that can reveal mutations — to help his family find answers. Dr. Berger used advanced biochemical testing, genomic sequencing and AI to sift through the patient’s data. He found the problem: dopa-responsive dystonia, a genetic condition seen in only one out of every 1 million children. In fact, James’ case was even rarer because he had an unusual recessive form.

James and family mountain biking

James (left) rides mountain bikes with his brother, Nicholas, and mom, Shannon.

This discovery led to a cascade of positive events that transformed James’ life for the better. Thankfully, his condition has a known treatment. Laura Tochen, M.D., who directs the hospital’s Movement Disorders Program, started James on carbidopa-levodopa, a drug combination used to treat Parkinson’s disease and other neurological disorders. Within two hours, he showed improvement and his gait was almost normal.

Today, James leads an active life. On vacation last summer, he went rock climbing on real rocks for the first time. He loves mountain biking and running along the Maine coast. “He is very proud of what he can do now,” says his father, Jeff. “We are so thankful for the team that helped get him here.”

Read more stories like this one in the latest issue of Believe magazine.

collage of news logos

Children’s National in the News: 2024

collage of news logosIn 2024, Children’s National Hospital continued to make remarkable strides across diverse areas of pediatric medicine, from groundbreaking technological innovations to critical health advocacy. The following compilation showcases ten significant stories that demonstrate the breadth and depth of the hospital’s impact, as featured in major national news outlets including NBC Nightly News, CNN, The Washington Post, The New York Times, NPR, The Today Show, Healio, and POLITICO. Delve into our 2024 news highlights for more.

1. World’s smallest pacemaker gives new hope to babies with heart defects

Charles Berul, M.D., and a patient family talk about the pill-sized pacemaker that saved the life of Abby, an infant born with deadly heart defects. (NBC Nightly News)

2. ‘A $10 death trip’: Fentanyl is killing teens. Meet one fighting for his life

Sivabalaji Kaliamurthy, M.D., addiction psychiatrist and director of the Addictions Program, spoke to CNN about the impact of drug addiction on teen health and the lack of resources available to treat opioid use disorder. (CNN)

3. Health panel urges interventions for children and teens with high BMI

Susma Vaidya, M.D., M.P.H., associate medical director of the IDEAL Clinic, shared her concerns about childhood obesity treatment recommendations issued today by a leading panel of independent U.S. health experts. (The Washington Post)

4. An Rx for food? Doctor’s offices offer groceries to those in need

Shideh Majidi, M.D., M.S.C.S., and Emily Frymark, clinical dietitian, spoke about how the food pharmacy, created in partnership with the Capital Area Food Bank, benefits patients with diabetes and other chronic conditions. (The Washington Post)

5. First patient begins newly approved sickle cell gene therapy

Kendric Cromer, a 12-year-old boy being treated at Children’s National Hospital, became the first person in the world with sickle cell disease to begin a commercially approved gene therapy that may cure the condition. “This is a big effort,” says David Jacobsohn, M.D., ScM, M.B.A. (The New York Times)

6. ‘We created this problem’: A pediatric surgeon on how gun violence affects children

Mikael Petrosyan, M.D., associate chief of General and Thoracic Surgery, discusses the stress medical staff face when treating young victims of gun violence. (NPR)

7. 7th grade boy rings bell after final round of chemotherapy

Landon, an 11-year-old patient, rang the bell at Children’s National Hospital with family, friends, doctors and nurses cheering after finishing his final round of chemotherapy. (The Today Show)

8. Study: One in three adolescents experience ‘period poverty’

Monika Goyal, M.D., M.S.C.E., pediatric emergency medicine specialist and co-director of the Center for Translational Research, emphasized the need for awareness in addressing period poverty in teenagers and young adults. (Healio)

9. The AI assurance labs are coming

Kolaleh Eskandanian, Ph.D., M.B.A., P.M.P., vice president and chief innovation officer, participates in a panel discussion covering AI data collection, associated risks, reliance and other topics related to artificial intelligence. (POLITICO)

10. First day of a ‘new life’ for a boy with sickle cell

Children’s National patient Kendric Cromer, 12, became one of the first children ever to be treated with a newly approved gene therapy that will free him from the sickle cell disease that has stolen his childhood. (The New York Times)

2024 with a lightbulb instead of a zero

The best of 2024 from Innovation District

2024 with a lightbulb instead of a zero2024 marked another groundbreaking year for Children’s National Hospital, showcasing remarkable advances across the spectrum of pediatric medicine, research and healthcare innovation. From pioneering surgical procedures to breakthrough artificial intelligence applications, the institution continued to push the boundaries of what’s possible in children’s healthcare. Read on for our list of the most popular articles we published on Innovation District in 2024.

1. Prenatal COVID exposure associated with changes in newborn brain

A study led by researchers at Children’s National Hospital showed that babies born during the COVID-19 pandemic have differences in the size of certain structures in the brain, compared to infants born before the pandemic. The findings suggest that exposure to the coronavirus and being pregnant during the pandemic could play a role in shaping infant brain development.
(3 min. read)

2. Children’s National Hospital again ranked among the best in the nation by U.S. News & World Report

Children’s National Hospital was ranked as a top hospital in the nation by the U.S. News & World Report 2024-25 Best Children’s Hospitals annual rankings. This marks the eighth straight year Children’s National has made the Honor Roll list. The Honor Roll is a distinction awarded to only 10 children’s hospitals nationwide.
(2 min. read)

3. Children’s National performs first ever HIFU procedure on patient with cerebral palsy

In January 2023, a team of multidisciplinary doctors performed the first case in the world of using bilateral high intensity focused ultrasound (HIFU) pallidotomy on Jesus, a 22-year-old patient with dyskinetic cerebral palsy. The procedure is part of a clinical trial led by Chima Oluigbo, M.D., pediatric neurosurgeon at Children’s National Hospital.
(3 min. read)

4. Novel ultrasound device gets FDA breakthrough designation with Children’s National support

A novel ultrasound device developed by Bloom Standard received the Food and Drug Administration’s valued breakthrough device designation with the help of Children’s National Hospital. The device that enables autonomous, hands-free ultrasound scans to be performed anywhere, by any user.
(2 min. read)

5. First-of-its-kind pilot study on the impacts of Lyme disease in pregnancy and infant development

Understanding the effects of Lyme disease on the developing fetal brain is essential to ensure timely prenatal and postnatal treatments to protect the fetus and newborn. In response to this need, Children’s National Hospital is leading a pilot study to establish the groundwork needed for a larger study to determine the effect of in utero exposure to Lyme disease on pregnancy and early childhood neurodevelopmental outcomes.
(3 min. read)

6. Earliest hybrid HLHS heart surgery kids thrive 5 years later

Five years ago, Cayden was born 6 weeks early weighing less than four pounds and at risk of dying from her critical congenital heart disease. Today, she’s a happy five-year-old. Early diagnosis of her hypoplastic right ventricle, double inlet left ventricle and critical coarctation of the aorta allowed for the team at Children’s National Hospital to create a careful plan for safe delivery and to offer an innovative hybrid HLHS surgical approach at the hospital within 24 hours after she was born.
(1 min. read)

7. Wayne J. Franklin, M.D., F.A.C.C., named senior vice president of the Children’s National Heart Center

Children’s National Hospital appointed Wayne J. Franklin, M.D., F.A.C.C., as the new senior vice president (SVP) of the Children’s National Heart Center. In this role, Dr. Franklin oversees the full spectrum of heart care services including cardiac imaging and diagnostics, interventional cardiology, electrophysiology, cardiac anesthesia, cardiac surgery and cardiac intensive care.
(2 min. read)

8. Artificial – and accelerated – intelligence: endless applications to expand health equity

By pioneering artificial intelligence (AI) innovation programs at Children’s National Hospital, Marius George Linguraru, D.Phil., M.A., M.Sc., and the AI experts he leads are ensuring patients and families benefit from a coming wave of technological advances. The team is teaching AI to interpret complex data that could otherwise overwhelm clinicians.
(4 min. read)

9. Evidence review: Maternal mental conditions drive climbing death rate in U.S.

Painting a sobering picture, a research team led by Children’s National Hospital culled years of data demonstrating that maternal mental illness is an under-recognized contributor to the death of new mothers. They called for urgent action to address this public health crisis.
(3 min. read)

10. Nathan Kuppermann, M.D., M.P.H., named chief academic officer and chair of Pediatrics

Children’s National Hospital appointed Nathan Kuppermann, M.D., M.P.H., as its new executive vice president, chief academic officer and chair of Pediatrics. In this role, Dr. Kuppermann oversees research, education and innovation for the Children’s National Research Institute as well as academic and administrative leadership in the Department of Pediatrics at George Washington University School of Medicine & Health Services.
(2 min. read)

11. First global clinical trial achieves promising results for hypochondroplasia

Researchers from Children’s National Hospital presented findings from the first clinical trial of the medication vosoritide for children with hypochondroplasia – a rare genetic growth disorder. During the phase 2 trial, researchers found vosoritide increased the growth rate in children with hypochondroplasia, allowing them to grow on average an extra 1.8 cm per year.
(2 min. read)

12. Pioneering research center aims to revolutionize prenatal and neonatal health

Since its establishment in July 2023, the Center for Prenatal, Neonatal & Maternal Health Research at Children’s National Hospital has gained recognition through high-impact scientific publications, featuring noteworthy studies exploring the early phases of human development.
(3 min. read)

Attendees at Sidra Medicine's 4th annual Pediatric Neuroscience Conference (PNC 2024)

Insights and Innovations in Pediatric Neuroscience: Highlights from PNC 2024

Attendees at Sidra Medicine's 4th annual Pediatric Neuroscience Conference (PNC 2024)
Children’s National Hospital served as a strategic partner for Sidra Medicine’s 4th annual Pediatric Neuroscience Conference (PNC 2024) which took place in Doha, Qatar. This collaboration emphasizes a mutual commitment to advancing pediatric neurological care and research.

The conference offered a robust platform for presenting groundbreaking research and clinical advancements across diverse subfields such as pediatric neurology, neurosurgery, neuro-critical care, neurogenetics, neuroimmunology and neuroradiology. Esteemed medical professionals and researchers from around the world convened to share insights and innovations that are shaping the future of pediatric neurological health.

Various speakers from Children’s National led in-depth discussions on diagnostic and therapeutic innovations aimed at enhancing outcomes for children with chronic neurological and neurosurgical conditions. PNC 2024 provided an excellent platform for healthcare professionals, researchers and academics to update their knowledge and engage with leading specialists in the field.

Presenters and topics from Children’s National included:
Attendees at Sidra Medicine's 4th annual Pediatric Neuroscience Conference (PNC 2024)

The collaborative efforts with Sidra Medicine highlight a shared vision of pushing the boundaries of pediatric neurological research and treatment, ultimately aiming to improve outcomes for young patients worldwide.

Seven key takeaways from the early development and implementation of the NeuroCardiac Critical Care Program.

Sharing development of the NeuroCardiac Critical Care Program through Congenital Heart Academy

Seven key takeaways from the early development and implementation of the NeuroCardiac Critical Care Program.

Jones presented seven key takeaways from the early development and implementation of the NeuroCardiac Critical Care Program.

“Neurodevelopmental care is not a decision on a day-to-day basis. It is a series of micro-decisions embedded in our practice every single day,” says Melissa Jones, MSN, APRN, CPNP-AC, director of the NeuroCardiac Critical Care Program at Children’s National Hospital.

Several years ago, Jones and colleagues in the Cardiac Intensive Care Unit (CICU) at Children’s National launched the program, which involved team education, implementation of evidence-based best practices, research and quality improvement efforts with the goal of optimizing brain neurodevelopment for patients in cardiac intensive care.

More than 1,000 people around the world registered for a recent virtual educational webinar hosted by the Congenital Heart Academy focused on the topics of neuroprotection for children with congenital heart disease. During the webinar the team outlined the process and lessons learned from developing this important, novel program.

What it is

The NeuroCardiac Critical Care Program at Children’s National is an integrated, multidisciplinary group of clinicians focused on eliminating secondary brain injury, optimizing brain development and promoting healthy family bonding in the CICU.

The team prioritizes several key areas, including:

  • Weekly neurodevelopmental rounds
  • Environmental changes
  • Pain and sedation management
  • Parent and caregiver engagement
  • Neuromonitoring guidelines

Jones presented the multidisciplinary team-oriented approach that led to the launch of the program, which continues to evolve and grow in the CICU today. She also offered a series of lessons learned, such as:

  • Even the patients who are the most fragile can receive developmentally supportive care.
  • Elevating and disseminating the expertise of the team is key.
  • Continuous staff educations and buy-in is essential.
  • Working with existing resources (people and material) is important.
  • Early and small wins can build traction for the team.
  • Taking care of parents is developmental care.
  • Culture change takes time, patience and persistence.

Children’s National leads the way

Children’s National is a national leader in the study of neurodevelopment across the lifespan of children born with congenital heart disease. This includes cutting edge work to understand the fetal brain, earlier diagnosis and intervention for heart disease and how congenital heart anomalies affect growth and development in utero, studies of neuroprotection strategies for use in the operating room, neurologically supportive approaches in cardiac critical care, and neuropsychological support systems as these children grow up and into adulthood.

Cardiac critical care and telehealth experts at Children’s National have been longstanding contributors to the knowledge sharing efforts of the Congenital Heart Academy from its beginning, including leading a precursor international, multi-disciplinary knowledge sharing telehealth series for critical care strategies started during the COVID-19 pandemic.

Watch the webinar: Development of the NeuroCardiac Critical Care Program

Illustration of low brain efficiency

Neighborhood disadvantage alters brain networks in unborn babies

Illustration of high and low brain efficiencyGrowing up in a disadvantaged neighborhood changes a child’s brain even before birth. These new findings, in the latest edition of The Journal of Pediatrics, underscore the need to support young families from a baby’s earliest days.

According to this new research from the Center for Prenatal, Neonatal & Maternal Health Research at Children’s National Hospital, exposure to neighborhood disadvantage lessens the functional integration of neural networks in the fetal brain, as seen on functional MRIs of healthy babies. The center compared the brains of 68 healthy babies on 79 scans. Researchers then mapped details about neural activity to a “social vulnerability index” from the Centers for Disease Control and Prevention, which indicates proximity to environmental stressors.

“We specifically looked at brain architecture to see how easily information flows between different regions,” said Kevin Cook, Ph.D., research faculty at the center and the manuscript’s first author. “To do this, we used graph theory, which borrows concepts from social network theory. It’s widely applied in computer science to understand how information flows within groups, and neuroscience has adapted it to study how information travels within the brain.”

What we found

Dr. Cook said researchers focused on the three metrics:

  • Path length, which measures how many stops information needs to make along its way through the brain.
  • Global efficiency, which measures the overall efficiency of the entire brain’s network.
  • Small-world propensity, which describes how the brain’s network is organized and indicates how well the brain is organized into smaller, efficient networks.

As social vulnerability increased, the research team found global efficiency decreased, meaning the brain’s neural network was less efficient. The path lengths were also longer in children with greater neighborhood disadvantage, reinforcing the global efficiency findings.

The fine print

The under-development and over-development of fetal brains may contribute to neurological disorders, such as autism, epilepsy and other conditions of interest to researchers. Yet science’s understanding of how the brain matures in utero is still limited.

In this study, researchers found a notable difference related to age. At the youngest gestational ages, path lengths are longer, and both global efficiency and small world propensity are lower. As the fetus gets closer to term, path length and global efficiency show rapid maturation and less advantaged fetuses catch up to their peers who have greater advantages.

Researchers saw the same findings for small-world propensity, but the maturation didn’t stop. These unborn babies overshot their peers and had greater small-world propensity, suggesting their brains are divided into a greater number of smaller networks than their advantaged peers.

“We believe that length and global efficiency are on a trend to overshoot,” Dr. Cook said. “These findings are notable because they agree with what we know about older children and adolescents. Greater disadvantage is associated with hyper- or over-maturation of the brain. Our findings suggest that this may be starting even before birth.”

What’s ahead

While still early, this research improves the understanding of how environmental complexities can impact an unborn baby. Catherine Limperopoulos, Ph.D., director of the research center, which opened in 2023, said this work will be foundational as they continue to study the impact of a child’s environment on development.

“These findings have important implications for understanding how status and disadvantage may have a cumulative effect on fetal brain development,” Dr. Limperopoulos said. “We must study and consider how to conceptualize the impact of socioenvironmental disadvantage in communities to better care for children and work to improve outcomes.”

Read the full study – “Greater Neighborhood Disadvantage Is Associated with Alterations in Fetal Functional Brain Network Structure” – in The Journal of Pediatrics.

conceptual illustration of brain and brainwaves

How targeted therapy can help overcome febrile infection-related epilepsy syndrome

conceptual illustration of brain and brainwaves

In their findings, experts achieved a promising functional and neurologic outcome.

Febrile infection-related epilepsy syndrome (FIRES) is a severe neurological condition characterized by the sudden onset of seizures following a febrile illness, often leading to significant morbidity. In a recent study led by Dana Harrar, M.D., Ph.D., director of Critical Care Neurology at Children’s National Hospital, a team of experts followed the case of a 5-year-old previously healthy male who presented with acute neurological decline. This culminated in super-refractory status epilepticus following a fever.

The study outlines a novel treatment regimen that involved early and aggressive cytokine-directed immunomodulatory therapy, alongside a comprehensive seizure management strategy that intentionally avoided barbiturate use. By tailoring treatment to the patient’s evolving cytokine profile and incorporating supportive measures such as the ketogenic diet and antiseizure medications, experts achieved a promising functional and neurologic outcome.

“Now, 2½ years later, he attends mainstream school, has attention-deficit hyperactivity disorder (ADHD), mild neurocognitive impairment and well-controlled epilepsy,” the experts write. “By using cytokine-directed immunotherapy and avoiding a barbiturate coma, we were able to successfully treat a pediatric patient with febrile infection-related epilepsy syndrome and achieve a good outcome.”

The study’s findings show that the understanding of FIRES is advancing as experts uncover the underlying mechanisms of this challenging neurological disorder. Physician-scientists in this field developed innovative treatment strategies targeting immune dysregulation and associated seizures. By following recent consensus recommendations and utilizing molecularly targeted immunotherapy along with serial cytokine profiling, Dr. Harrar and team successfully treated the 5-year-old patient, leading to a positive neurological outcome.

You can read the full study, Successful Management of Febrile Infection-Related Epilepsy Syndrome Using Cytokine-Directed Therapy, in the Journal of Child Neurology.

2024-25 US News Badges

Children’s National again ranked among the best in the nation by U.S. News & World Report

2024-25 US News BadgesChildren’s National Hospital in Washington, D.C., was ranked as a top hospital in the nation by the U.S. News & World Report 2024-25 Best Children’s Hospitals annual rankings. This marks the eighth straight year Children’s National has made the Honor Roll list. The Honor Roll is a distinction awarded to only 10 children’s hospitals nationwide.

This year, U.S. News ended ordinal rankings on its Honor Roll. Instead of assigning a numerical rank from 1 to 10, all hospitals on the Honor Roll will be recognized as having attained the highest standards of care in the nation.

In addition, Children’s National tied for #1 pediatric hospital in the Mid-Atlantic region, which includes New York, New Jersey, Delaware, Pennsylvania, the District of Columbia, West Virginia and Virginia. It’s also best in the Mid-Atlantic in Neonatology.

For the fourteenth straight year, Children’s National ranked in 10 specialty services. New this year, U.S. News included behavioral health as a service line in the rankings. Since it’s the first year, there are no ordinal rankings for behavioral health, but the Children’s National program was named one of the top 50 programs in the country.

“In my first year here, I witnessed what makes Children’s National so special — our commitment to collaboration, empowering one another, and charting a bold path forward for pediatric care,” said Michelle Riley-Brown, MHA, FACHE, president and chief executive officer of Children’s National. “I’m proud U.S. News again recognized Children’s National as one of the top in the nation and the highest-ranked pediatric hospital in D.C., Maryland and Virginia. Together, we’ll continue to push the boundaries of care, research and innovation to make a difference for those who matter most — the kids.”

The annual rankings are the most comprehensive source of quality-related information on U.S. pediatric hospitals and recognizes the nation’s top 50 pediatric hospitals based on a scoring system developed by U.S. News.

“For nearly two decades, U.S. News has published Best Children’s Hospitals to empower the parents and caregivers of children with complex medical needs,” said Ben Harder, chief of health analysis and managing editor at U.S. News. “Children’s hospitals appearing on the U.S. News Honor Roll have a track record of delivering unparalleled specialized care.”

The bulk of the score for each specialty service is based on quality and outcomes data. The process includes a survey of relevant specialists across the country, who are asked to list hospitals they believe provide the best care for patients with the most complex conditions.

The Children’s National specialty services that U.S. News ranked in the top 10 nationally are:

The other four specialties ranked among the top 50 are Behavioral Health, Cardiology and Heart Surgery, Pulmonology and Lung Surgery, and Urology.

Magnetic resonance imaging of a brain with seizures

Making pediatric epilepsy surgery more accessible

Magnetic resonance imaging of a brain with seizures

Pediatricians play a vital role in boosting the use of epilepsy surgery for better patient outcomes.

Despite proven safety, efficacy and cost effectiveness, surgery for pharmacoresistant epilepsy remains one of the most underutilized, evidence-based treatments in modern medicine. Pediatricians are key stakeholders for increasing epilepsy surgical utilization.

A recent commentary from Children’s National Hospital experts — led by Nathan Cohen, M.D., neurologist; Chima Oluigbo, M.D., neurosurgeon; and William D. Gaillard, M.D., chief of Neurology — offers a new perspective that highlights the issue and offers practical solutions to increase primary care physician comfort in the referral of patients for epilepsy surgery.

“Pediatricians should feel empowered to open discussions with epilepsy patients and their families about the possibility of surgery,” the authors write. “Through partnership with managing neurologists and early referral to epilepsy surgery centers, together we can begin to dismantle the significant barriers to access. Only by overcoming our own fears of initiating these discussions can we offer hope to those patients and families who need it the most.”

You can read the full commentary, “Breaking Barriers to Pediatric Epilepsy Surgery Utilization,” in The Journal of Pediatrics.

Douglas Postels and other clinicians in Malawi

Think tank improves care for Malawian children with seizures, epilepsy

  • Scenes from Dr. Postels' trip to Malawi
  • Scenes from Dr. Postels' trip to Malawi
  • Scenes from Dr. Postels' trip to Malawi
  • Scenes from Dr. Postels' trip to Malawi
  • Scenes from Dr. Postels' trip to Malawi
  • Scenes from Dr. Postels' trip to Malawi
123456

Global health leaders from Children’s National Hospital joined Malawian clinical experts in a two-day think tank to improve the care of children with seizures in the southern African nation. Details were published recently in a perspective in the American Journal of Tropical Medicine and Hygiene.

“The meeting was eye-opening,” said Douglas Postels, M.D., a pediatric neurologist and faculty member with the Center for Translational Research at Children’s National. “Outcomes are changed when we collaborate directly and work through the best solutions for clinical care in low-resource settings. You must weigh what clinics need, what they can implement, and the impact on public health.”

The big picture

Dr. Postels does research and provides clinical care at Queen Elizabeth Central Hospital (QECH), the largest public hospital in Malawi. “Queen’s” is the only medical setting in a country of over 20 million people where an electroencephalogram (EEG) and specialty pediatric neurology services are available. QECH is an essential — and often overtaxed — component of neurological care in Malawi. Cerebral malaria, epilepsy and seizures from birth asphyxia drive the need for neurological evaluations.

EEG is available at QECH for all hospitalized children and is principally used to evaluate for subclinical seizures in those who are comatose. One of three trained technicians acquires the EEG, which is electronically sent to the United States for interpretation. Under best-case scenarios, the turnaround time between EEG collection and interpretation from a neurologist in the U.S. is about two hours. However, many obstacles can slow results, including misaligned schedules across time zones, internet outages and other complications.

With the support of the Center for Translational Research, Dr. Postels brought five U.S.-based colleagues to Africa to meet with Malawian clinicians to better understand local clinical care needs.  A think tank of U.S. and Malawian clinicians worked together to consider research directions and priorities for neurology care and EEG services.  Their conversation focused extensively on integrating technology in the places where it will be most clinically useful.

The fine print

Dr. Postels’ research group aims to aid in the clinical care of Malawian children with epilepsy and to make EEG accessible outside of Queen’s hospital. They hope to eventually create a point-of-care EEG system, linking a method of EEG acquisition that requires little training to apply with a software-based interpretation system.

Expanding the use of EEG in Malawi and throughout Africa has numerous clinical applications. Malawian clinicians hoped that EEG could be made available at their local hospitals and folded into a more comprehensive care algorithm for children at risk of seizures — “one aspect of a ‘packet of care’ that included better overall understanding of seizures, criteria for administration of antiseizure medications, and transfer to the central hospital,” according to the journal article.

Malawian clinicians were also interested in using EEG to detect increased intracranial pressure after head trauma, as neuroimaging services are nearly as rare as EEG in Malawi.  One pediatrician also wanted to use EEG to guide the treatment of neonatal seizures, which are common in children with birth asphyxia, and to use EEG as a prognostic biomarker.

“We hope to empower Malawian clinicians with clinical research skills and experiences so that they can lead and direct future discussions with patients, parents and stakeholders to advance neurologic care and research in their country,” Dr. Postels and his team wrote.

To dive deeper, read the complete perspective — “Establishing Priorities for Epilepsy Care and Electroencephalogram Use in Low-Resource Settings” — in the American Journal of Tropical Medicine and Hygiene.

Audience members at the NIAID Conference

Breaking free of illness: Experts explore solutions for chronic pediatric disorders

  • Scenes from Dr. Postels' trip to Malawi
  • Scenes from Dr. Postels' trip to Malawi
  • Scenes from Dr. Postels' trip to Malawi
  • Scenes from Dr. Postels' trip to Malawi
  • Scenes from Dr. Postels' trip to Malawi
  • Scenes from Dr. Postels' trip to Malawi
123456

When new patients with chronic illnesses come to Roberta DeBiasi, M.D., M.S., division chief of Infectious Diseases, they are often drained of energy, far behind in school and fatigued by the lack of coordinated care among multiple specialists. She envisions a better way to care for these children: a data-driven, multidisciplinary clinic that can help diagnose and treat disorders facing chronically ill children.

In a keynote address, Dr. DeBiasi laid out her vision for improving care during the 7th annual symposium hosted by Children’s National Hospital and the National Institute of Allergy and Infectious Diseases (NIAID): “A new paradigm: Infection-associated chronic illnesses (IACI) affecting children.” Experts from across the country came together to discuss these IACIs, the importance of finding biomarkers to diagnose and monitor them, data-driven therapeutics to treat them, and the urgent need for protocols to guide physicians.

The patient benefit

A range of IACIs – including long COVID, Lyme disease, postural orthostatic tachycardia syndrome (POTS), dysautonomia and more – are stealing normal, active lifestyles from children. Dr. DeBiasi said patients need researchers who understand disease pathogenesis and have standardized disease classification to diagnose and treat these disorders. Because of the complexity of these cases, care delivery and coordination also need to change. Patients, she said, would best be served by beginning with a three-hour appointment in a multidisciplinary clinic with experts from behavioral health, rehabilitative medicine and other specialties.

“When one of these patients comes to us, we feel helpless as physicians,” Dr. DeBiasi said. “There’s no diagnostic, and we don’t know how to treat them…. It is, to me, very energizing that we’re going to be able to have a coordinated way to help these families. But to do that, we have to have standardized disease classification.”

Moving the field forward

In 2017, NIAID and Children’s National launched a clinical research partnership devoted to advancing the health of children with allergic, immunologic, auto-inflammatory and infectious diseases through collaborative research and education. The partnership — co-led by H. Clifford Lane, M.D., NIAID’s deputy director for Clinical Research and Special Projects, and Catherine Bollard, M.D., M.B.Ch.B., director of the Center for Cancer and Immunology Research at Children’s National — promotes the prevention, diagnosis, treatment and cure of these childhood diseases. Every year, the partnership organizes and funds a symposium to explore cutting-edge research and pressing issues in pediatric care.

At this year’s gathering, experts examined the specifics of various disorders, including Alexandra Yonts, M.D. She provided new data from the Children’s National Post-Covid Program and explained that patients “are clamoring” for help with symptom management.

“There have been virtually no randomized, controlled trials and very little evidence on any sort of aggressive treatments, or pharmaceutical management options, especially in the pediatric population,” Dr. Yonts said. “Upon recent attendance at some long COVID meetings, there is an expressed concern, especially among pediatric providers, about trying medications off-label in these long COVID patients.”

Miss the symposium? You can learn more about the science they explored in the symposium’s recording on topics including:

  • Potential overarching mechanisms of disease, including pathogen- and host-mediated factors.
  • Identifying potential biomarkers for chronic illnesses
  • Treatments and multidisciplinary approaches for patients with IACIs
  • Patient advocacy
A clean room at CNRI.

CellBuilder: A ready-made solution for cell & gene therapy manufacturing

A clean room at CNRI.

With CellBuilder and our global partnerships, Children’s National hopes to expand access to groundbreaking cell and gene therapy treatments as they take off in the next five years.

With cell and gene therapies poised to reshape cancer and rare disease treatments, researchers at Children’s National Hospital are pioneering ready-to-use solutions that will bring these cutting-edge therapies directly to hospitals and other treatment centers, shrinking the distance between doctors and patients.

“The next five years are going to be a period of tremendous growth for cell and gene therapy,” said Patrick Hanley, Ph.D., chief and director of the Cellular Therapy Program at Children’s National. “Currently, there’s no shortage of interest from the medical community, but there’s a shortage of people who can manufacture and administer this care. We’re looking for ways to get these treatments to the patients by providing other institutions the tools they need to launch these programs cost-effectively, safely and efficiently.”

Called CellBuilder, the starter kits for cell and gene therapy programs could transform the landscape for pediatric patients.

The big picture

Dr. Hanley and many members of the Children’s National team have been working in cell and gene therapy for more than a decade, gaining extensive experience in the technical and regulatory hurdles inherent in creating treatments that target diseases at their source.

In cell therapy, a specific cell type is modified and transferred to a patient with a payload that can target a disease or disorder. For example, T cells may be modified and delivered to patients to teach their immune systems to fight cancer.  In gene therapy, a patient’s genetic code is modified to treat or prevent diseases, such as sickle cell disease, cancers and other genetic disorders. This can be done by introducing a healthy copy of a gene, repairing a faulty gene or altering a gene’s function.

Children’s National has become a leader in manufacturing virus-specific T cells, one method of delivering cell therapies, and the Cellular Therapy Program has conducted consortium-led, multi-center trials. Many other healthcare sites across the country want to start programs offering this care at their facilities.

The holdup in the field

Starting a cell and gene therapy program from scratch can take years of effort, training and money. That’s why Jay Tanna, M.S., R.A.C., quality assurance manager of the Cellular Therapy Program at Children’s National, said the team is creating CellBuilder starter kits, which include the manufacturing protocol and the resources necessary to launch a cell therapy program almost instantly.

“With a suitable knowledge base, institutions can start their own cellular therapy program at the point of care, using our manufacturing protocols, vetted reagents and other key elements of the process,” Tanna said. “Of course, interested institutions would have to meet regulatory requirements and establish a clean room to manufacture these therapies. If they want to use CellBuilder to run a clinical trial, they can do that. If they want to take it to become a licensed product, they can do that, too.”

Children’s National has worked with more than five institutions to build their virus-specific T-cell program and is now using the kits to accelerate and commercialize the process to increase patient access. The lab has also entered into a memorandum of understanding with the Tokyo-based Hitachi Global Life Solutions, Inc., an innovative modular clean room manufacturer, with the goal of offering a bundled solution.

Why we’re excited

Dr. Hanley and his colleagues say that the partnerships Children’s National is forging as they consult with other experts in this field will expand access to cell and gene therapy across the country—and hopefully around the world.

“It used to be that, to get a CAR T cell, you would collect the cells at Children’s National, ship them to a company like Novartis, have the therapy manufactured there and then shipped back,” said Michael Keller, M.D., who co-led a first-of-its kind immunotherapy trial as the Translational Research Laboratory director at the Children’s National Cell Enhancement and Technologies for Immunotherapy Program. “It was expensive, time-consuming and limited patient access. Now, there’s growing interest in manufacturing at each site, just like you would with a bone marrow transplant.”

With CellBuilder and our global partnerships, Children’s National hopes to expand access to groundbreaking cell and gene therapy treatments as they take off in the next five years, extending lifetimes and improving the quality of life for children suffering from rare disorders.

“We’re trying to capture the momentum underway in the field by providing this kit so that institutions don’t have to know how to do everything,” Dr. Hanley said. “We provide all the knowledge, a reagent list and everything else they need—and they provide the care.”

Cheng-Chia “Fred” Wu, M.D., Ph.D.

Expanding team innovates to take on pediatric brain tumors

Cheng-Chia “Fred” Wu, M.D., Ph.D.

Cheng-Chia “Fred” Wu, M.D., Ph.D., joins the team at the campus as an assistant professor at Virginia Tech’s Fralin Biomedical Research Institute. Image credit: Fralin Biomedical Research Institute.

Experience, talent and technology are coming together at the Children’s National Research & Innovation Campus to solve the complex challenges of treating pediatric brain tumors through a growing partnership between Children’s National Hospital and Virginia Tech.

Cheng-Chia “Fred” Wu, M.D., Ph.D., joins the team at the campus as an assistant professor at Virginia Tech’s Fralin Biomedical Research Institute. He hopes to improve treatment for pediatric brain tumors and other cancers by leveraging technological advances in focused ultrasound and studying how this modality can be combined with other novel therapies. Children’s National physicians and scientists are collaborating with Virginia Tech scientists to develop these new approaches.

Why we’re excited

Catherine Bollard, M.D., M.B.Ch.B., director of the Center for Cancer and Immunology Research and interim chief academic officer at Children’s National, said collaborations — including the partnership between Children’s National and Virginia Tech — are essential to solving formidable scientific challenges.

“We are thrilled to be working with Dr. Wu to develop cutting-edge therapeutics for pediatric brain tumors,” Dr. Bollard said. “With Dr. Wu’s experience and the talents of our team at Children’s National, I have great hope that we will be able to combine our novel technologies and therapies to provide tremendous breakthroughs for treating pediatric patients with brain tumors.”

Before joining Virginia Tech, Dr. Wu was an assistant professor of radiation oncology at Columbia University Irving Medical Center in New York, where he treated pediatric cancers and central nervous system malignancies.

“Partnering with Children’s National connects us to a world-class clinical trial institute that has been a pioneer in treating brain tumors with focused ultrasound technology, and this presents a unique opportunity to help children and families struggling with cancer,” Dr. Wu said. “I can’t wait to see where this takes us.”

Dr. Wu played a key role in the Initiative for Drug Delivery Innovation for Childhood Brain Tumors at Columbia, developing a bench-to-bedside platform to facilitate the translation of promising technologies for targeted drug delivery in children with brain tumors.

Within three years, the team demonstrated the safety and feasibility of using focused ultrasound in the brainstem and adding radiation in preclinical models. They then opened two clinical trials for kids with relapsed diffuse midline glioma, an aggressive and difficult-to-treat brain tumor that occurs in the brain stem, thalamus and spinal cord.

What’s ahead

Dr. Wu will be involved in a wide range of research touching both organizations, including veterinary medicine and biomedical engineering at Virginia Tech.

Michael Friedlander, Ph.D., executive director of the Fralin Biomedical Research Institute at Virginia Tech Carilion and vice president for health sciences and technology at Virginia Tech, welcomed Dr. Wu’s experience as a pediatric radiation oncologist, translational physician-scientist and clinical trialist who has led an innovative drug delivery program.

“He is armed with a unique set of skills to identify promising new technology and implement it in areas of great need for treatment of pediatric cancers,” Dr. Friedlander said. “We are absolutely delighted to have Dr. Wu as part of the team.  He represents a powerful new part of the strong partnership between Virginia Tech and Children’s National Hospital for addressing pediatric brain cancer.”

Dr. Craig Sable in Uganda

Around the world

Our Global Health Initiative launched in 2016 with the goal of eliminating pediatric health disparities around the world. We aim to address the most pressing pediatric health issues through better care for medically underserved populations. This leadership helps us achieve our mission of caring for all children. A broad range of education and research projects improves health outcomes. They also offer enriching opportunities for experienced faculty and emerging leaders to advance clinical excellence.

Healing hearts in Uganda

Dr. Craig Sable in Uganda

Dr. Craig Sable and team train partners in Uganda.

Craig Sable, M.D., interim chief of Cardiology, improves care for young people with rheumatic heart disease (RHD) in Uganda. Donors, including the Karp Family Foundation, Huron Philanthropies, Zachary Blumenfeld Fund and the Wood family, make this possible. RHD affects 50 million people, mostly children, worldwide. It claims 400,000 lives each year.

Dr. Sable and Ugandan partners completed important research showing that early RHD detection, coupled with monthly penicillin treatment, can protect the heart. They are working on practical solutions, such as a new portable device with artificial intelligence (AI) that can easily screen for RHD.

In 2023, Dr. Sable led two missions in Uganda where he and his team did surgeries and special tests for 18 children with RHD. They also taught local doctors new skills to help more kids on their own.

Plastic surgery and reconstructive care in Kenya and Nepal

Each year our Craniofacial & Pediatric Plastic Surgery team, under the leadership of Johnston Family Professor of Pediatric Plastic Surgery and Chief of Pediatric Plastic Surgery Gary Rogers, M.D., J.D., LL.M., M.B.A., M.P.H., provides opportunities for fellows to participate in surgical missions.

In 2024, Perry Bradford, M.D., traveled to the Moi Teaching Hospital in Eldoret, Kenya where she provided patients with burn, pressure wound and cleft reconstruction. She built community connections with the local plastic surgeons and educated registrars and medical students. “This gave me firsthand experience working in a community with limited resources and forced me to be more creative,” Dr. Bradford says. “The experience inspired me to examine what it means to have consistent access to advanced tools and equipment.”

In 2022, a group traveled to Nepal to provide care. Some patients arrived after days of travel by yak or buffalo. One child with a burn injury recovered use of her hand. The team educated local providers to deliver life-changing treatments unavailable in Nepal.

Dr. Tesfaye Zelleke in Ethiopia

Dr. Tesfaye Zelleke, left, and team in Ethopia.

Elevating epilepsy care in Ethiopia

Neurologist Tesfaye Zelleke, M.D., and partners in Ethiopia are seeking to improve the lives of children with epilepsy. The BAND Foundation provides support. Ethiopia has a population of about 120 million yet only a handful of pediatric neurologists.

Dr. Zelleke’s team trained nonspecialist providers to diagnose and treat children in the primary care setting. They also launched a mobile epilepsy clinic to provide community care and build the capacity of local clinicians. In collaboration with advocacy groups, the team educates the public about epilepsy with a goal of reducing stigma.

New hope in Norway

In 2023, our Division of Colorectal & Pelvic Reconstruction shared its expertise with clinicians at Oslo University Hospital, Rikshospitalet, in Norway. This effort was a key first step in Oslo becoming the first dedicated colorectal center in Scandinavia.

Marc Levitt, M.D., and team members performed complex surgeries otherwise unavailable for waiting patients. They led an academic conference. They held clinics to educate nurses, reviewed patient records and made care recommendations. Specialized care enabled a young patient with significant bowel difficulties to recover function and lead a normal life.

The team will travel to South Africa, the Czech Republic and Spain in 2024. Donors, including The Dune Road Foundation and Deanna and Howard Bayless, make this work possible.

Improving outcomes for babies in the Congo

AI can be a valuable tool for diagnosing genetic conditions. It detects unique facial patterns that clinicians without genetics training can miss. However, existing facial analysis software struggles in nonwhite populations.

A team led by Marius George Linguraru, D.Phil., M.A., M.Sc., the Connor Family Professor of Research and Innovation and principal investigator in the Sheikh Zayed Institute for Pediatric Surgical Innovation, is working to improve the newborn diagnosis rate worldwide. They are testing smartphone software in the Democratic Republic of Congo. Diverse newborn data improves AI’s ability to detect a variety of genetic conditions in more children. Early detection, diagnosis and informed care lead to better health outcomes.

Nephrology care for kids in Jamaica

Dr. Moxey-Mims and team in Jamaica

Jennifer Carver and Dr. Marva Moxey-Mims, center, with staff at Bustamante Children’s Hospital.

Marva Moxey-Mims, M.D., chief of Nephrology, is bringing care to children with kidney disease in Jamaica, with a goal of improving health equity. An International Pediatric Nephrology Association grant helped make it possible.

On a recent trip, Dr. Moxey-Mims and a small team — including Jennifer Carver, RN, CNN, lead peritoneal dialysis nurse at Children’s National, and three pediatric nephrologists from Jamaica — trained nearly 30 nurses from Jamaican hospitals. Nurses received hands-on dialysis education to improve their clinical skills. The team also worked to educate the community in disease awareness and prevention.

Read more stories like this one in the latest issue of Believe magazine.

stressed pregnant woman

Pandemic stress in pregnant mothers may affect anxiety regions of babies’ brains

stressed pregnant woman

The research from Children’s National Hospital provides mounting evidence that children of the pandemic, even those far too young to understand it, need ongoing assessments of developmental or mental health support later in life.

A critical part of the brain linked to risks for anxiety later in life – the left amygdala – was significantly smaller by volume in babies of mothers who reported stress during the COVID-19 pandemic, according to a new manuscript published in JAMA Network Open.

The right hippocampus, which governs spatial, visual and verbal memories, and the white matter were also reduced in children whose mothers reported stress.

The research from Children’s National Hospital provides mounting evidence that children of the pandemic, even those far too young to understand it, need ongoing assessments of developmental or mental health support later in life.

“Looking ahead, we want to use this information – and studies with similar findings – to empower pregnant mothers to request support to mitigate their stress, especially in the event of another global health crisis,” said Nickie Andescavage, M.D., a neonatologist and principal investigator at the Center for Prenatal, Neonatal & Maternal Health Research. “We also want to make sure babies born during COVID-19 get the services that they need in life if they develop anxiety or other mental health disorders.”

The fine print

Researchers at the center used magnetic resonance imaging (MRI) to compare the brains of 103 babies born between 2014 and 2019 prior to the pandemic to 59 born between 2020 and 2022. Mothers who had COVID-19 or other complications in their pregnancies were excluded. The babies underwent MRI imaging while in utero and again soon after delivery.

The mothers were evaluated for stress and anxiety, using the Spielberger State-Train Anxiety Inventory and other evidence-based scoring measures. Pre-pandemic, 21% of mothers reported elevated symptoms of anxiety; in the pandemic cohort, that number jumped to nearly 62%.

Their babies’ brains were also changed, as regions widely understood to control emotion and anxiety displayed smaller volumes on MRI imaging. Given the global impact of the pandemic and universal reports of mental distress worldwide, the potential impact of these findings may impact an entire generation of children born during the pandemic. The team is just beginning to unravel the medical significance.

What’s next

Catherine Limperopoulos, Ph.D., director of the Center for Prenatal, Neonatal & Maternal Health Research, said understanding the impact of stress is vital in supporting the healthy development of young children. Current studies are underway at her center to tease apart the role of stress in prenatal development and examine its long-term impact on development, including cognition, behavior and mental health.

“We all know that being pregnant can be quite stressful, and there are certain times of collective stress that can provide us windows to understand how the body and mind manage it,” Dr. Limperopoulos said. “At our center, we care deeply about the health of mothers and babies, and our researchers plan to continue investigating the role of stress in development to continue building data to show that mental health must be a greater priority.”

This study – “Prenatal maternal psychological distress during the COVID-19 pandemic and newborn brain development” – was supported by the National Institutes of Health, the Intellectual and Developmental Disabilities Research Center, and the A. James & Alice B. Clark Foundation. You can read the full study in JAMA Network Open.

illustration of neural network

Tailored care for children with autism improves EEG experience

illustration of neural network

Most children with a developmental disability can complete EEG with sufficient support.

Electroencephalographic (EEG) monitoring is an important tool for diagnosing seizures, epilepsy and other neurologic conditions. However, many children with developmental disabilities – including autism – have difficulty undergoing hospital tests like EEG. Furthermore, sometimes these tests are not prescribed or turn out to be unsuccessful.

In a new study published in Journal of Child Neurology, findings from a Children’s National Hospital team indicate that most children with a developmental disability can complete EEG with sufficient support. Special planning can identify children who would benefit from accommodations to help them complete the study successfully.

What this means

Children with developmental disabilities have an increased risk of epilepsy and need for overnight video EEG monitoring. However, video EEGs have historically been considered difficult to complete for this population. Experts at Children’s National implemented a coordinated team approach to help children with developmental disability tolerate overnight video EEGs. The project involved a caregiver preprocedure questionnaire shared with the team to create personalized care plans.

These findings suggested that most children with developmental disability can complete video EEG with sufficient support. Preprocedure planning can identify children who would benefit from additional accommodations.

Moving the field forward

The findings show new knowledge through vigorous research about the patient experience and the value of a team dedicated to helping children with development disabilities undergo hospital tests.

“We were excited to find that with special planning more children with developmental disabilities could complete an EEG,” said Elizabeth Wells, M.D., senior vice president at the Center for Neuroscience and Behavioral Medicine at Children’s National, and one of the study’s authors. “We thought it was important to study and discover that children from minority or underrepresented groups were less likely to access support services, which means more work and research is necessary to ensure that services are accessible to these children.”

The authors also found that parent-reported difficulties with communication and cooperation were associated with video EEG success, which means doctors and hospital teams should always ask about and listen to parent input in planning the tests for children.

How Children’s National is leading the way

Healthcare workers often express lack of confidence in their ability to provide care for patients with autism and associated disabilities and express an interest in gaining access to resources and training to better care.

By developing a program to improve care and then studying it through rigorous research, the authors aspire to spread a message of hope. Programs like these can ensure all kids get the tests and care they need. Children’s National is also a leader in showing the importance of parent input in medical care planning, which too often is not a routine part of care at hospitals.

Further research is necessary to clarify which supports are most helpful.

Three prior students who trained at Children’s National are lead authors, Kalyn Nix, Jessica Smith and Atara Siegel, Ph.D. Additional authors from Children’s National include Kathleen Atmore, Psy.D., and Elizabeth Wells, M.D.

You can read the full study, Individualized Care Delivery for Children With Autism and Related Disabilities Undergoing Overnight Video Electroencephalography (EEG): One Hospital’s Experience With a Coordinated Team Approach, in the Journal of Child Neurology.

illustration of a neural network

Adjusting key protein could improve brain function in children with fetal alcohol syndrome

illustration of a neural network

Researchers at Children’s National are testing whether controlling the level of apolipoprotein E could serve as an effective treatment for the poor neurobehavioral outcomes tied to fetal alcohol spectrum disorders.

Reduced levels of a protein – called apolipoprotein E – are responsible for the lifelong cognitive and neurological symptoms in fetal alcohol spectrum disorders (FASD), according to a new study published in the journal Molecular Psychiatry. For the first time, researchers at Children’s National Hospital are testing whether controlling the level of this protein could serve as an effective treatment for the poor neurobehavioral outcomes tied to FASD, which is believed to affect roughly 5% of school-aged children.

Apolipoprotein E is controlled by a gene called APOE, a well-known risk gene for Alzheimer’s disease. It contains the instructions that guide how the connections between neurons in the brain strengthen or weaken over time. This process, known as synaptic plasticity, is crucial for learning and memory formation. In this study, plasma was collected from participants at two sites in Western Ukraine, along with information about drinking behavior reported by their mothers.

What this means 

“In addition to Alzheimer’s risk, we found this gene is also a crucial contributor to cognitive problems in children with FASD,” said Kazue Hashimoto-Torii, Ph.D., a principal investigator at the Center for Neuroscience Research at Children’s National. “The new data shows that we understand the mechanism by which prenatal alcohol exposure causes a decrease of the APOE level in the brain. We will continue this work to help improve our understanding of FASD, hoping to replenish this important protein and improve outcomes for children with these disorders.”

FASD is an umbrella diagnosis for the physical and developmental challenges that face children who are exposed to alcohol in utero, including intellectual disability, delay in motor and language development and other neurological diagnoses. While alcohol alone is problematic to many aspects of development, evidence also suggests that genetic factors play a role. Only 4.3% of children with heavy alcohol exposure develop FASD, and twin studies have revealed that fraternal twins have different FASD outcomes compared to identical twins.

What’s next 

The research team at Children’s National wanted to pinpoint places where genes could play a role and consider therapies. For the first time, researchers found that plasma levels of APOE were reduced in children with FASD, which strongly supports a potential target for therapy. In addition, their genomics study found a variant of APOE increases the risk of cognitive problems in subjects who were prenatally exposed to alcohol, especially those of African ancestry.

“We found that providing a drug that activates the APOE receptor rescued learning deficits and anxiety in pre-clinical models,” said Masaaki Torii, Ph.D., principal investigator at the Center for Neuroscience Research. “The implications offer an exciting glimpse into possible therapies for some of the neurological harms associated with prenatal alcohol exposure and FASD.”

Read the full manuscript – Reduction of APOE accounts for neurobehavioral deficits in fetal alcohol spectrum disorders – in Molecular Psychiatry.