Neurology & Neurosurgery

Pediatric MOGAD: Brain lesions and optic neuritis outcomes

brain scans

In a new retrospective study, researchers analyzed pediatric patients with myelin oligodendrocyte glycoprotein antibody–associated disease (MOGAD) to determine whether the presence of brain lesions affected clinical outcomes in those presenting with optic neuritis (ON).

In a new retrospective study published in Multiple Sclerosis and Related Disorders, researchers analyzed pediatric patients with myelin oligodendrocyte glycoprotein antibody–associated disease (MOGAD) to determine whether the presence of brain lesions affected clinical outcomes in those presenting with optic neuritis (ON). By comparing patients with ON and brain lesions to those with ON without brain lesions, the investigators found that brain lesions were often asymptomatic and were not associated with additional clinical disability.

The big picture

The study retrospectively looked at 32 pediatric patients with MOGAD being followed by Children’s National Hospital and Johns Hopkins Hospital between August 2010 and July 2024. Of these, 18 presented with ON without brain lesions and 14 with ON and brain lesions. Overall, 69% of patients were female and the median age at symptom onset was 12 years old, with a younger onset among those experiencing acute disseminated encephalomyelitis. Across the cohort, a total of 35 ON episodes were analyzed.

Features of ON, including severity of visual impairment, were largely consistent between groups, although individuals with concurrent brain lesions more frequently exhibited additional neurologic symptoms. Outcomes were favorable overall, with substantial recovery noted by one month and minimal disability at three months and beyond. Follow-up imaging demonstrated improvement in most brain lesions over time, suggesting that neither their presence nor persistence reliably predicts long-term disability or a more severe ON presentation.

The hold up in the field

“MOGAD is a rare disorder that has only been commercially testable since 2017, so we are still learning about its clinical phenotypes and long-term outcomes,” said Vivien Xie, MD, pediatric neuroimmunology fellow at Children’s National and lead author of the study. “In pediatrics, it can be especially difficult to conduct research on rare diseases because large cohort numbers are needed to draw meaningful conclusions.”

Moving the field forward

MOGAD encompasses a range of clinical phenotypes, and it remains unclear whether these phenotypes carry distinct prognostic implications in children.

“This study advances our understanding of prognosis in MOGAD and supports a more individualized approach to monitoring and treatment, while also helping clinicians more confidently counsel patients and families about the implications of brain lesions identified on initial MRI,” said Dr. Xie.

Read the study, Significance of brain lesions associated with optic neuritis in pediatric myelin oligodendrocyte glycoprotein antibody-associated disease, in the journal Multiple Sclerosis and Related Disorders.

Additional authors from Children’s National include: Claire Har, Kyle Spagnolo, DO, Ilana Kahn, MD, Leigh Sepeta, PhD, Alexandra B. Kornbluh, MD.

Children’s National recertified as PPMD Certified Duchenne Care Center with Clinical Research Designation

The Children's National neuromuscular medicine team

Children National Hospital’s Neuromuscular Medicine Program team.

Children’s National Hospital has been recertified as a Parent Project Muscular Dystrophy (PPMD) Certified Duchenne Care Center and has received PPMD’s new Clinical Research Designation, recognizing both sustained excellence in multidisciplinary Duchenne care and a demonstrated commitment to clinical research.

The Neuromuscular Medicine Program at Children’s National offers care through a robust, multidisciplinary clinic that brings together specialists in neurology, cardiology, pulmonology, endocrinology and bone health, physical and occupational therapy, nutrition and other key disciplines. This integrated model ensures that patients and families receive coordinated care that addresses the complex and evolving needs associated with Duchenne.

PPMD requires centers to reapply for certification every five years to ensure ongoing adherence to strict criteria, including specialist expertise, coordinated care models and comprehensive services. Recertification provides assurance of consistent, evidence-based care for individuals with Duchenne muscular dystrophy.

Introduced in 2025, the Clinical Research Designation distinguishes Certified Duchenne Care Centers that actively conduct Duchenne clinical trials and research. This designation highlights programs that contribute to reducing variability in care, strengthening clinical trial outcome measures and accelerating the development of emerging therapies.

“Recertification as a Certified Duchenne Care Center affirms Children’s National’s commitment to coordinated, evidence-based Duchenne care,” said Sarah Wright, DO, program director of the Neuromuscular Medicine Program. “The Clinical Research Designation builds on that foundation, reflecting our focus on advancing research, expanding access to clinical trials, and improving outcomes for patients and families.”

Through recertification and this new research designation, Children’s National continues to serve as a clinical and research resource within the national Duchenne care network while also improving the care of all patients with neuromuscular diagnoses.

Recognizing cerebral clots in children with brain infections

Medical illustration of a human brain highlighting blood vessels and blood flow

New evidence shows that cerebral sinovenous thrombosis, or CSVT, is far more common than previously appreciated.

The big picture

Acute bacterial intracranial infections place children at risk for serious neurologic complications. In addition to inflammation and direct brain injury, these infections create conditions that promote clot formation through dehydration, venous stasis, endothelial injury and hypercoagulability. Cerebral sinovenous thrombosis, or CSVT, has historically been underrecognized in this population.

As highlighted in a recent Neurology editorial, new evidence shows that CSVT is far more common than previously appreciated. In a retrospective observational study of children hospitalized for acute bacterial intracranial infections, nearly one-third developed CSVT.  This finding reframes how clinicians should think about risk, screening and management in these high-risk patients.

The hold-up in the field

Despite clear risk factors for CSVT, there are no standardized guidelines for screening children with intracranial bacterial infections. Prior studies have often focused on CSVT as the starting point rather than asking how frequently it complicates infection.

“As clinicians, we see this as a common clinical presentation,” said Dana Harrar, MD, PhD, director of the Pediatric Stroke Program at Children’s National Hospital and one of the editorial authors. “This study confirms just how common CSVT is as a complication of intracranial bacterial infection.”

Diagnosis is further complicated by variability in neuroimaging. In the study discussed in the editorial, 63% of CSVT cases were not detected on initial imaging. Not all children underwent MRI, and no single imaging sequence consistently identified CSVT.

Uncertainty around treatment has also slowed progress. While the study was not designed to definitively assess safety or effectiveness, no patients experienced symptomatic bleeding related to anticoagulation. “This suggests that anticoagulation may be a safe treatment option in this population,” Dr. Harrar noted.

Children’s National Hospital leads the way

At Children’s National, this work serves as a foundation for advancing care. Multidisciplinary teams from Neurology, Neurosurgery, Hematology and Critical Care Medicine are collaborating to develop a standardized clinical pathway for diagnosing and managing infectious CSVT.

“Future efforts should focus on establishing clearer approaches to identifying CSVT in children with intracranial bacterial infections, while also evaluating when and how anticoagulation should be used,” said Dr. Harrar. “Further study is also needed to understand how newer anticoagulants may fit into care.”

Read the full editorial, “Cerebral Sinovenous Thrombosis in Children With Bacterial Intracranial Infections”, in Neurology.

Shaping the future of epilepsy care: highlights from AES 2025

In early December, several doctors, fellows and faculty from the division of Neurology at Children’s National Hospital attended the American Epilepsy Society (AES) Annual Meeting in Atlanta, Georgia.

This meeting unites a global community of child and pediatric neurologists, neurosurgeons, psychiatrists, neuropsychologists, psychologists, basic translational and clinical scientists, advanced practice providers, advocates and industry professionals to share scientific breakthroughs, foster collaboration and improve outcomes for people living with epilepsy.

Children’s National experts were featured throughout the program, presenting on a wide range of topics in pediatric epilepsy.

Sessions and Workshops

  • Chima Oluigbo, MD: Epilepsy Surgery Skills Workshop | Overcoming Complications in Epilepsy Surgery
  • Dana Harrar, MD, PhD: Resident EEG Course
  • Hua Xie, PhD: Neuroimaging platform | Thalamo-lesional Connectivity Profile Predicting Focal to Bilateral Tonic-Clonic Seizures in Focal Cortical Dysplasia-related Epilepsy
  • John Schreiber, MD: Investigators Workshop | Mortality in DEEs Across the Lifespan: Timing, Causes and Prevention
  • Leigh Sepeta, PhD: Investigators Workshop | Hippocampal Long Axis Differentiation in Memory and Seizure Networks
  • Sara Preisendanz, MS, RD, CSP, LD: Professional Development | Career Pathways: Interprofessional Panel
  • Syed Anwar, PhD, MS: Pediatric State of the Art Symposium | Pediatric Epilepsy Surgery: It’s Not Just About Resection

Poster Presentations

To learn more about the meeting, visit the AES website for additional information.

The best of 2025 from Innovation District

2025 with a lightbulb instead of a zero

In 2025, Innovation District readers gravitated toward stories that explored how research and clinical innovation are reshaping pediatric care in real time. This year’s most popular articles highlighted advances in complex surgical care, evidence-based treatments for chronic and neuropsychiatric conditions and emerging technologies — from wearable data to artificial intelligence — that are changing how clinicians diagnose, treat and support children and families. Read on for our list of the most popular articles we published on Innovation District in 2025.

1. Life-changing care: How Children’s National tackles pediatric cervical spine injuries

The Cervical Spine program at Children’s National Hospital is responsible for treating a range of conditions, including trauma, congenital abnormalities and tumors. These conditions can lead to instability or misalignment of the cervical spine. “There are unique challenges in pediatric cases due to anatomical differences. The cervical spines of children are more at risk for injury because of their developmental stage and structural characteristics,” says Matthew Oetgen, MD, MBA, chief of Orthopaedic Surgery and Sports Medicine at Children’s National.
(2 min. read)

2. Pioneering evidence-based treatments for substance addictions

Increasing evidence-based treatment is a key component of the Addictions Program at Children’s National Hospital, created in 2022 and led by Sivabalaji Kaliamurthy, MD. “We really want to focus on intervening in an evidence-based manner in the primary care setting because that is where most of our patients are going to first access care outside of the emergency room,” explains Dr. Kaliamurthy.
(3 min. read)

3. Breaking barriers in growth disorder treatment for families

For many children with short stature and other rare genetic growth disorders, there have been no next steps after usual treatment options prove ineffective. Researchers at Children’s National Hospital are digging deeper to find the root genetic causes of short stature disorders and creating novel, nuanced treatment options that have the opportunity to change how the field approaches these cases.
(4 min. read)

4. The link between metabolic acidosis and cardiovascular disease in children with CKD

Denver D. Brown, MD, nephrologist at Children’s National, is looking at whether untreated metabolic acidosis could potentially contribute to cardiovascular outcomes in children with chronic kidney disease (CKD). Here, she explains her motivation, findings and future directions for this critical research.
(3 min. read)

5. Therapy approach shows promise for PANS/PANDAS

A multidisciplinary therapy model developed at Children’s National shows promise for children with PANS and PANDAS, significantly reducing symptoms through structured cognitive-behavioral therapy and family-centered care. The approach could offer a new standard for treating these rare, complex neuropsychiatric disorders.
(2 min. read)

6. Wearable tech data shows promise in ADHD detection

A study from Children’s National reveals that common wearable devices like Fitbits may hold the key to improving how we identify Attention-Deficit/Hyperactivity Disorder (ADHD) in adolescents. By analyzing patterns in heart rate, activity levels and energy expenditure, researchers were able to predict ADHD diagnoses with striking accuracy, offering a glimpse into a future where objective, real-time data supports earlier and more personalized mental healthcare.
(2 min. read)

7. Novel pediatric pacemaker shows safety, effectiveness for fragile infants in multi-center study

A novel implantable pacemaker designed specifically for infants has demonstrated safety and effectiveness in stabilizing heart rhythms for at least two years. The multi-center study of 29 infants showed stable pacing, normal electrical parameters and expected battery life, offering a viable alternative to standard-size devices for the smallest children.
(2 min. read)

8. Socioeconomic disadvantage associated with higher long-term mortality for children after heart surgery

Children who had heart surgery and come from less advantaged neighborhoods in the Washington, D.C., region are much more likely to die in the long term than those from neighborhoods with more wealth and opportunity. The finding was part of a presentation titled, Socioeconomic Disadvantage Is Associated with Higher Long-Term Mortality After Cardiac Surgery, by Jennifer Klein, MD, MPH, cardiologist at Children’s National Hospital, during the Society of Thoracic Surgeons Annual Meeting in Los Angeles.
(2 min. read)

9. Children’s National brings AI into the RHD early diagnosis equation

Experts from Children’s National traveled to Uganda to continue work on a pilot program applying artificial intelligence (AI) to the diagnosis of rheumatic heart disease (RHD). The team created a tool that uses AI to predict RHD by identifying leaky heart valves on handheld ultrasound devices, then prompts a referral for a full echocardiogram.
(2 min. read)

10. Fighting food insecurity with fresh produce and education

Food insecurity is rising in Washington, D.C. and it’s hitting families with children the hardest. That’s why Children’s National Hospital created the Family Lifestyle Program (FLiP) – a multi-layered intervention, which offers Patient Navigation (FLiP-PN) and a Produce Prescription Intervention (FLiPRx). FLiP is a Food Is Medicine, clinical-community initiative that helps families get access to fresh food, build healthy habits and lower their risk of diet-related diseases like diabetes and obesity.
(3 min. read)

Children’s National in the News: 2025

collage of news outlet logosIn 2025, Children’s National Hospital was featured in major national news outlets for pioneering advances in pediatric care, groundbreaking clinical research and powerful human stories of healing and hope. From gene therapy for sickle cell disease and innovative pacemakers for newborns to breakthrough transplants, cancer trials and emerging mental health concerns like AI psychosis, these stories highlight the hospital’s leadership across the full spectrum of pediatric medicine. The following ten highlights showcase the patients, families and experts behind this impact, as reported by outlets including NBC News, The Washington Post, Good Morning America, USA Today, Healio, ABC News and ESPN.

1. Gene therapy treatment gives new hope to sickle cell patients

Children’s National patient Wedam, 19, begins the first steps for intensive gene therapy for sickle cell disease, discussing his skepticism while his mother expresses her joy and gratitude for the treatment. (NBC News)

2. Little pacemakers ‘reliable’ in stabilizing newborns needing pacing

Charles Berul, MD, pediatric electrophysiologist and emeritus chief of Cardiology, discusses his study highlighting the safety and efficacy of an innovative smaller pacemaker designed for newborns with critical congenital heart disease. (Healio)

3. Why this clinical trial is offering some young cancer patients hope

Catherine Bollard, MBChB, MD, senior vice president and chief research officer, and the NexTGen team are poised to recruit patients for a new clinical trial that will take on an old, implacable foe: children’s solid tumors. (The Washington Post)

4. 11-year-old receives living donor heart valve to replace artificial one in breakthrough surgery

Yves d’Udekem, MD, PhD, chief of Cardiac Surgery, talked to Good Morning America about how an 11-year-old’s groundbreaking partial heart transplant will change his life and the lives of other children in need of valve replacements. (Good Morning America)

5. 9-year-old fights brain cancer with magic in her heart

The Lilabean Foundation along with Brian Rood, MD, medical director of the Brain Tumor Institute, talked about how patients like Kasey Zachman are the motivation behind finding a cure for brain cancer. (ABC News)

6. How Little League helps father-son duo recover from kidney transplant

USA Today Sports spoke with Gavin Brown and his parents, as well as Yi Shi, MD, a pediatric nephrologist at Children’s National Hospital, about their kidney transplant journey. (USA Today)

7. Jayden Daniels surprises Children’s National patient

After Jayden Daniels visited Commanders fan Sarah Addison at Children’s National Hospital while she was being treated for myeloid leukemia, they quickly became friends. (ESPN)

8. At D.C. children’s hospital, opera singers offer light, hope and healing

International opera stars performed for children, their families and employees at Children’s National Hospital. (The Washington Post)

9. ‘Gift of life:’ Tiny heart transplant saves life of Maryland baby

A baby boy in Maryland is back home after being given a second chance at life, just before his first birthday. The boy’s mother and his surgeon, Manan Desai, MD, share the remarkable story of a moment that changed all of their lives. (NBC4)

10. AI psychosis: Kids left delusional and paranoid over conversations with chatbots

Ashley Maxie-Moreman, PhD, clinical psychologist, spoke to ABC7 about what AI psychosis is and what parents need to know. (ABC7)

Showcasing global expertise: Children’s National at the 2025 Pediatric Neuroscience Conference in Qatar

Dr. Elizabeth Wells presenting at the 2025 Pediatric Neuroscience Conference.

Dr. Elizabeth Wells presenting at the 2025 Pediatric Neuroscience Conference.

In early November, experts from the Neuroscience and Behavioral Medicine Center at Children’s National Hospital traveled to Doha, Qatar, to participate in the 5th Annual Pediatric Neuroscience Conference, presented by Sidra Medicine. This marks the second year of partnership between Children’s National and Sidra Medicine for this premier international event.

The annual conference brings together leading clinicians, researchers and innovators from across Neurology, Neurosurgery, Neuro-critical Care, Neurogenetics and Neuroradiology to share the latest insights driving the future of pediatric neuroscience. This year’s program explored the epidemiology and mechanisms of neurological diseases, cutting-edge clinical trials, AI innovations and new models of integrated, high-impact care.

Elizabeth Wells, MD, senior vice president of the Neuroscience and Behavioral Medicine Center at Children’s National and a member of the conference’s Scientific Committee, highlighted the continued impact of this international partnership. “By training and supporting physicians and allied health staff across the region, we’re expanding access to quality care close to home,” said Dr. Wells. “But when children require complex interventions or innovative treatments that aren’t available locally, Children’s National is ready to provide that next level of care. We’re proud to serve both as a partner in strengthening regional capacity and as a global leader in the most advanced pediatric neuroscience therapies.”

“It was a great pleasure to partner once again with Children’s National for this year’s Pediatric Neuroscience Conference,” said Ruba Benini, FRCPSC, MD, PhD, medical director of the Neurodiagnostic Laboratory at Sidra Medicine. “Their high-caliber speakers and the depth of experience they brought to the conference added tremendous value, and we look forward to continuing our collaboration in the future.”

Children’s National brought the largest contingent of external speakers to this year’s meeting, presenting across multiple high-visibility sessions:

Scientific Sessions & Presentations

SESSION II: Neuro-Immunology

Drs. William D. Gaillard, Adelaide Robb and Karen Smith at the 2025 Pediatric Neuroscience Conference.

Drs. William D. Gaillard, Adelaide Robb and Karen Smith at the 2025 Pediatric Neuroscience Conference.

  • Elizabeth Wells, MD — Autoimmune Encephalitis – Updates on Diagnosis & Treatment

SESSION III: Neuro-Oncology

  • Elizabeth Wells, MD — Session Moderator
  • Robert Keating, MDLatest Advances in Non-Invasive Focused Ultrasound Treatment for Brain Tumors

SESSION IV: Neurogenetics and Precision Medicine in Neurological Disorders

  • Sarah Wright, MD Progress and Innovations in Clinical Trials for Neuromuscular Diseases
  • William D. Gaillard, MD Developments in Epilepsy Clinical Trials: Emerging Therapies and Technologies

SESSION V: Epilepsy Symposium

  • William D. Gaillard, MD — Epilepsy Surgery: Patient Selection, Evaluation and Outcomes
  • Chima Oluigbo, MDAdvances in Epilepsy Surgery: Options and Procedures
  • Adelaide Robb, MD Neuropsychiatric Co-Morbidities in Pediatric Epilepsy

SESSION VI: Complex Neurosurgical Procedures

  • Robert Keating, MD — Two Decades of Experience With Chiari Malformations: Where Are We Today?

SESSION VII: Artificial Intelligence and Cutting-Edge Research in Neuroscience

  • Chima Oluigbo, MD — Artificial Intelligence in Neurosurgery: Transforming Diagnosis, Treatment, and Patient Outcomes

Following the conference, Dr. Adelaide Robb and Dr. William D. Gaillard presented Grand Rounds for the psychiatry and neurology departments at Sidra Medicine, further strengthening the educational and clinical collaboration between institutions.

“Our work in the Gulf is rooted in a simple belief: every child deserves access to cutting-edge care and the hope it brings,” says Karen Smith, MD, executive medical director of Global Services at Children’s National. “By partnering with hospitals and government leaders across the region, we’re not only sharing expertise—we’re building sustainable systems that empower communities and transform outcomes for children for years to come.”

To learn more about the Pediatric Neuroscience Conference, visit the conference website.

Global leaders in pediatric neuroscience, including experts from Children’s National, gather at the 2025 Pediatric Neuroscience Conference in Qatar.

Global leaders in pediatric neuroscience, including experts from Children’s National, gather at the 2025 Pediatric Neuroscience Conference in Qatar.

CoQ10 linked to symptom resolution in children with New Daily Persistent Headache

Molecular structure of Coenzyme Q10

Among the 182 patients studied, researchers found that CoQ10 supplementation was significantly more common among children whose headaches ultimately resolved.

A new study from neurology experts at Children’s National Hospital suggests that Coenzyme Q10 (CoQ10) may play a meaningful role in the resolution of new daily persistent headache (NDPH), a rare, debilitating and historically treatment-resistant condition in children. This study analyzed 182 pediatric patients evaluated over six years, making it the largest single-center study of NDPH ever conducted.

The research was led by pediatric neurologist Mrinmayee Takle, MD, along with co-authors Jeffrey Strelzik, MD, Raquel Langdon, MD, Abigail Turner, MD, William McClintock, MD, John Barber, MS, Armelle Dejoie, MS, Emily McCracken, BS, Olivia Goucher, BS, and senior author Marc DiSabella, DO.

Why it matters

Progress in NDPH research has been slow, particularly in the pediatric population. The condition is both rare and highly heterogeneous, making research and controlled trials challenging. Many patients present only after experiencing long-standing symptoms and multiple unsuccessful treatments, complicating efforts to isolate which interventions are truly effective. Additionally, most pediatric headache studies and all industry-sponsored clinical trials specifically exclude children with continuous daily headaches like NDPH.

These obstacles have left clinicians with little evidence-based guidance and families with a trial-and-error approach that can feel discouraging. This new analysis offers hope, highlighting potential treatment avenues and underscoring the need for prospective, controlled studies.

What the study found

Among the 182 patients studied, researchers found that CoQ10 supplementation was significantly more common among children whose headaches ultimately resolved. While other interventions including magnesium, topiramate, onabotulinum toxin A and regular aerobic exercise showed encouraging trends, CoQ10 was the only treatment associated with statistically significant improvement.

“This is the first potential treatment signal we’ve seen in pediatric NDPH,” Dr. Takle explained. “Very few therapies have shown measurable impact in this population, so identifying an association with Coenzyme Q10 is particularly exciting.” The consistency of positive trends across other migraine-related treatments also suggests that NDPH may share more therapeutic overlap with migraine than previously assumed, an insight that could help reshape how clinicians think about the disorder.

Impact on patients and care

For families, the findings offer something the field has long lacked: a more informed starting point. NDPH is notoriously treatment-resistant, and many patients cycle through medications and lifestyle adjustments with little clarity on what may help. The study’s results provide clinicians with data-backed options to guide therapy, hopefully bringing relief to patients sooner.

Emphasizing lifestyle factors such as regular aerobic exercise also gives families accessible, proactive strategies that may support symptom improvement.

Children’s National leads the way

Children’s National houses one of the largest and most systematically characterized pediatric headache registries, enabling the type of robust analysis rarely possible in NDPH research. The hospital’s multidisciplinary Headache Program, led by Dr. Marc DiSabella, has developed standardized evaluations and long-term follow-up that support high-quality data collection and clinical insight.

This study is the first to identify CoQ10 as a potential contributor to symptom resolution in pediatric NDPH, highlighting the unique role Children’s National plays in advancing both scientific understanding and patient care for this challenging disorder.

Read the full study, Coenzyme Q10 Supplementation May Be Correlated With Resolution of New Daily Persistent Headache, in The Journal of Pediatric Neurology.

Patrick Cimino, MD, PhD, joins Children’s National as inaugural Professor of Molecular Neuropathology

Patrick Cimino

“It’s an honor to join Children’s National and become the first Professor of Molecular Neuropathology,” Dr. Cimino says.

Children’s National Hospital named Patrick “P.J.” Cimino, MD, PhD, as the inaugural Professor of Molecular Neuropathology.

Dr. Cimino began his role as a principal investigator in the Children’s National Research Institute in October 2025. He will lead new research and bring experts together across the Center for Cancer and Immunology Research, the Brain Tumor Institute and the Division of Pathology and Laboratory Medicine.

The big picture

Dr. Cimino joins a distinguished group of Children’s National physicians and scientists who hold an endowed chair. Children’s National is grateful to generous donors who have altogether funded 51 professorships.

Professorships support groundbreaking work on behalf of children and their families. They foster new discoveries and innovations in pediatric medicine. These appointments carry prestige and honor that reflects both the recipient’s achievements and the donor’s commitment to advancing and sustaining knowledge.

Why it matters

Dr. Cimino is a nationally recognized physician-scientist and board-certified neuropathologist. He specializes in the molecular genetics of gliomas. His work looks at brain and central nervous system tumors at the molecular level to understand how and why they develop, and guide more accurate diagnosis and treatment. At Children’s National, he will provide clinical pathology and neuropathology services and lead research that contributes to better therapies and patient outcomes.

Previously, Dr. Cimino served as head of the Neuropathology Unit in the Surgical Neurology Branch at the National Institute of Neurological Disorders and Stroke, part of the National Institutes of Health.

“It’s an honor to join Children’s National and become the first Professor of Molecular Neuropathology,” Dr. Cimino says. “I’m grateful to the generous donor who made this possible, and to the hospital for their trust and vision. Our shared commitment and collaboration to uncover what drives hard-to-treat tumors will lead to real breakthroughs for kids and families.”

Moving the field forward

This appointment was made possible by a $96 million strategic investment from an anonymous family in 2023 to advance pediatric brain tumor research and care. It is one of the largest donations in the hospital’s history, and is transforming the ability to give patients a better chance at healthy lifetimes. The Professorship in Molecular Neuropathology is the third created through this investment, which previously endowed the McCullough Distinguished Professorship in Neurosurgery and the Kurt D. Newman, MD, Professorship in Neuro-Oncology. The investment also elevated the Dr. Robert J. and Florence T. Bosworth Distinguished Professorship in Cancer and Transplantation Biology Research and fully funded the William Seamus Hughes Professorship in Neuro-Oncology and Immunology.

Through this incredible investment, Children’s National continues to partner with global experts to lead a powerful, collaborative effort to change what’s possible for children with brain tumors. It unites researchers and clinicians across disciplines to advance the science — developing new, often first-in-child therapies — while improving every aspect of care, from medical treatment to the psychosocial well-being of patients and their families.

“The vision of the anonymous family continues to create lasting impact,” says Roger J. Packer, MD, director of the Brain Tumor Institute and The Gilbert Distinguished Professor of Neurofibromatosis. “Dr. Cimino is an exceptional leader and scientist. We are thrilled to have him as the inaugural Professor of Molecular Neuropathology. His expertise brings exactly what we needed to move this work forward and deepen our understanding of how tumors grow and change.”

Researchers advance new drug development for fetal alcohol spectrum disorders

Pregnant woman saying no to a glass of wine.

Fetal alcohol spectrum disorders (FASD) are among the most common preventable causes of developmental disability, affecting an estimated 2% to 5% of children in the United States and Western Europe.

Fetal alcohol spectrum disorders (FASD) are among the most common preventable causes of developmental disability, affecting an estimated 2% to 5% of children in the United States and Western Europe. Yet despite their prevalence, treatment options remain limited, particularly for the cognitive and behavioral challenges that persist throughout life.

Researchers at Children’s National Hospital are working to change that. Through a new $2 million award from the National Institute on Alcohol Abuse and Alcoholism (NIAAA), investigators are advancing the development of a first-of-its-kind drug designed to improve learning and behavior in individuals with FASD.

Dive deeper

The study at Children’s National is led by site principal investigators Li Wang, PhD, and Anup Srivastava, PhD, in the Center for Neuroscience Research. The overall project is led by Masaaki Torii, PhD, in his role as principal investigator at a partnering start-up company co-founded by other Children’s National investigators, Kazue Hashimoto-Torii, PhD, and Hiroki Morizono, PhD, who contribute to the study in their company roles.

The research builds on discoveries made at Children’s National about how prenatal alcohol exposure disrupts brain development. The team identified a potassium channel called KCNN2, whose overactivity appears to play a key role in the neurobehavioral symptoms seen in FASD. To address this mechanism, the researchers developed FA-1, a small peptide compound that blocks KCNN2 activity. When delivered intranasally in preclinical models, FA-1 improved multiple behavioral outcomes, suggesting a potential path toward a targeted therapy.

The newly funded Phase II Small Business Technology Transfer (STTR) project will take FA-1 further along the translational pipeline. Researchers will optimize its intranasal formulation and evaluate its pharmacology, efficacy and safety in pre-clinical models. These studies will generate the data needed for an investigational new drug (IND) application with the U.S. Food and Drug Administration (FDA), paving the way for early clinical testing.

Why it matters

Fetal alcohol spectrum disorders currently have no FDA-approved medication that targets the root neurobiological causes of the disorder. Most available treatments only manage symptoms such as attention deficit or anxiety. If successful, FA-1 could become the first drug to directly improve the cognitive and behavioral functions affected by prenatal alcohol exposure.

“This project is an important step toward bringing a true, biology-based treatment for FASD to the children and families who need it,” said Drs. Wang and Srivastava. “The NIH’s support, combined with our partnership with the start-up, allows us to translate our discoveries at Children’s National into a therapy that we hope will make a meaningful difference in patients’ lives.”

Mapping the brain’s memory gradient: How the hippocampus helps children form lasting memories

illustration of the hippocampus

As children mature into adolescence, their hippocampus becomes increasingly specialized along its longitudinal axis, contributing to the development of memory and other cognitive functions.

In a study published in PNAS (Proceedings of the National Academy of Sciences), researchers found that the hippocampal gradient and its distinct connections with cortical systems are essential for memory development in children.

“This study is important because it helps us understand how the hippocampus develops and how it communicates with the rest of the brain to form episodic memory, or long-term memories of specific events,” said Hua (Oliver) Xie, PhD, research faculty at Children’s National Hospital and assistant professor at The George Washington University.

The big picture

Researchers looked at two resting-state functional magnetic resonance imaging (fMRI) datasets. The primary dataset included 598 participants between the ages of 8 and 21 and a secondary replication dataset of 196 participants from the Nathan Kline Institute (NKI)-Rockland Sample. Participants were scanned for a total of 26 minutes in resting-state where they stayed still, awake and looked at the fixation crosshair.

The results showed that the head and tail of the hippocampus form distinct communication patterns with other brain regions, which can help predict how well children form episodic memories.

As children mature into adolescence, their hippocampus becomes increasingly specialized along its longitudinal axis, contributing to the development of memory and other cognitive functions.

The hold up in the field

Previous studies have primarily focused on the hippocampus, with many researchers treating the anterior and posterior regions as distinct areas of study. By exploring how the hippocampus communicates with other parts of the brain, this study reveals a more comprehensive understanding than before.

“Our research shows that in children, it’s more of a gradual transition of function along the hippocampus’s long axis, like a color gradient rather than two distinct blocks,” Dr. Xie said.

You can read the full study, Longitudinal hippocampal axis in large-scale cortical systems underlying development and episodic memory, in PNAS.

Additional authors from Children’s National include Venkata Sita Priyanka Illapani, MS, Lauren T. Reppert, BS, Xiaozhen You, PhD, Manu Krishnamurthy, MS, Madison M. Berl, PhD, William D. Gaillard, MD, Leigh N. Sepeta, PhD

Children’s National Hospital designated Pediatric Center of Excellence in Rare Neuroimmune Disorders

Pediatric Center of Excellence in Rare Neuroimmune Disorders seal

Children’s National Hospital’s Neuroimmunology Program has been named a Pediatric Center of Excellence in Rare Neuroimmune Disorders by the Siegel Rare Neuroimmune Association.

The Neuroimmunology Program at Children’s National Hospital has been recognized by the Siegel Rare Neuroimmune Association (SRNA) as a Pediatric Center of Excellence in Rare Neuroimmune Disorders (CERND). This designation highlights the program’s expertise in caring for children with complex neuroimmune conditions and its commitment to advancing research and education in the field.

The program is led by Elizabeth Wells, MD, Senior vice president of the Neuroscience and Behavioral Medicine Center, Alexandra Kornbluh, MD, director of Neuroimmunology Investigational Therapeutics, and Ilana Kahn, MD, clinical director of Neuroimmunology. Together, they oversee a multidisciplinary team providing state-of-the-art diagnosis, treatment and long-term management for children with antibody-mediated autoimmune encephalitis (AE), acute disseminated encephalomyelitis (ADEM), febrile infection related epilepsy syndrome (FIRES), multiple sclerosis (MS), neuromyelitis optica spectrum disorders (NMOSD), myelin oligodendrocyte glycoprotein antibody-associated disease (MOGAD), opsoclonus myoclonus ataxia syndrome (OMAS), transverse myelitis (TM), optic neuritis (ON), primary CNS vasculitis and other central nervous system demyelinating and inflammatory conditions.

As a designated CERND, Children’s National offers:

  • Expert-led care: Specialists at the forefront of clinical practice and research.
  • Comprehensive services: Coordinated care across neurology, neurosurgery, neuropsychology, psychiatry, psychology, neuroradiology, physical medicine and rehabilitation, critical care, social work, pharmacy and speech therapy.
  • Research opportunities: Access to clinical trials and studies that drive progress in therapies and patient outcomes.

In addition to delivering high-quality clinical care, the program partners with SRNA to expand educational resources for patients, families and clinicians. Its work aligns with SRNA’s broader objectives to strengthen the CERND network, support innovative research and improve both acute therapies and long-term management strategies for rare neuroimmune disorders.

Advancing innovative treatments for neurofibromatosis type 1

Gilbert Family Foundation logoA new $8 million investment from the Gilbert Family Foundation will support groundbreaking research on neurofibromatosis type 1 (NF1) at Children’s National Hospital. The generous five-year grant continues the Foundation’s longstanding research partnership with the hospital. It will accelerate investigations aimed at improving the lives of children and young adults affected by this genetic condition.

“This grant will enable us to expand basic science, translational and clinical research. It will help us perform two to three innovative clinical trials in the next five years — trials we will share with the NF community as a whole,” says Roger J. Packer, MD, the Gilbert Family Distinguished Professor of Neurofibromatosis at Children’s National.

Dr. Packer will lead the research team, which also includes Javad Nazarian, PhD, Steven Stasheff, MD, Karin S. Walsh, PsyD and Sridevi Yadavilli, MD, PhD.

Why this matters

NF1 is one of the most common genetic conditions in young people. It affects approximately 1 in 2,500 children born each year. It causes potentially deadly nervous system tumors and can pose lifelong health complications. These include vision and learning disabilities, skeletal abnormalities and increased cancer risks.

Dive deeper

The new funding will:

  • Fuel clinical trials testing new therapies that combine MEK inhibitors, which block tumor growth, and checkpoint inhibitors, which help the immune system attack cancer, to treat aggressive NF1 brain tumors. Trials will target high-grade astrocytomas with piloid features (HGAPs) and recalcitrant gliomas. The goal is to improve outcomes for these aggressive tumors by enhancing the body’s immune response to cancer.
  • Drive cognitive research to understand learning, behavior and social deficits in children with NF1.
  • Better detect and understand vision loss in children with optic pathway tumors. Discoveries will help doctors predict, treat and prevent disease progression as well as assess the potential benefits of treatment.
  • Build novel lab models to supercharge drug discovery. The grant will support the expansion and validation of investigational models, including the development of NF1 zebrafish models and patient-derived organoids. NF1 tumor organoid tissues, grown in the lab, mimic the functioning of an actual organ.

Children’s National Leads the Way

In 2007, Dan and Jennifer Gilbert established The Gilbert Family Neurofibromatosis Institute at Children’s National. The Institute advances basic and clinical research, providing a global destination for care. The Gilbert family endowed three professorships that help the hospital advance the field.

Previous funding from the Gilbert Family Foundation has improved our understanding of NF1-related gliomas and how best to treat them. It has supported pioneering work in evaluating the safety and cognitive benefits of novel treatments, primarily MEK inhibitors. The Foundation’s new award will build upon this work. It will yield new insights into one of the most lethal manifestations of NF1: transformation of low-grade gliomas into malignant lesions. Children’s National will explore novel therapies, including immunotherapy, with the potential to save lives.

Gilbert Family Neurofibromatosis Institute accomplishments include:

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

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 2025-26 Best Children’s Hospitals annual rankings. This marks the ninth 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.

For the fifteenth straight year, Children’s National ranked in 10 specialty services and is the highest U.S. News ranked children’s hospital in Washington, D.C., Maryland and Virginia. Last year, U.S. News introduced pediatric & adolescent behavioral health as a service line in its rankings. While there are no ordinal rankings for behavioral health, the Children’s National program was named one of the top 50 programs in the country for the second year in a row.

“To be named among the nation’s top children’s hospitals for nine years in a row is a reflection of the extraordinary expertise, innovation and heart that our teams bring to every child and family we serve,” said Michelle Riley-Brown, MHA, FACHE, president and chief executive officer of Children’s National. “Our leadership in specialties like neurology, cancer, and diabetes and endocrinology underscores the national impact of our work, and we remain focused on setting new standards in pediatric care.”

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.

“Being a top-ranked pediatric hospital means more than just excelling in a single specialty — it means being a pillar of outstanding care for your entire region,” said Ben Harder, chief of health analysis and managing editor at U.S. News. “Our rankings acknowledge these hospitals for their comprehensive excellence, helping families find the very best care conveniently located within their state and community.”

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 six specialties ranked among the top 50 are Behavioral Health, Cardiology and Heart Surgery, Gastroenterology and GI Surgery, Neonatology, Pulmonology and Lung Surgery, and Urology.

Children’s National experts at the Child Neurology Society 2025 Annual Meeting

Child Neurology Society (CNS) Annual Meeting logoPhysicians, fellows and faculty from the Neuroscience and Behavioral Medicine Center at Children’s National Hospital will participate in this year’s Child Neurology Society (CNS) Annual Meeting, held October 8–11 in Charlotte, North Carolina. This premier gathering offers child neurologists the opportunity to exchange knowledge, foster collaboration, share research and advance patient care in the field.

Experts from Children’s National will be featured throughout the program, showcasing their leadership and expertise across a range of sessions and presentations.

Speakers and topics

Wei-Liang Chen MD
Seminar 9: Games Neurologists Play: AI for Enhanced Diagnosis in Neurology – Brain2Gene – Next Generation Phenotyping Using Artificial Intelligence to Predict the Genetic Diagnosis by Brain MRI Analysis
Friday, October 10, 2025 – 9:30-10:45 AM EDT/EST

Marc DiSabella, DO
Seminar 8: The Evolution of Migraine Disease from Childhood to Young Adult
Friday, October 10, 2025 – 9:30-10:45 AM EDT/EST

Hannah Grindling, MMSc, GC
CNS Genetics Summit: Demystifying Next-generation Sequencing: A Hands-On Workshop for Pediatric Neurologists
Saturday, October 11, 2025 – 12:30-4:30 PM EDT/EST

Dana Harrar MD, PhD
Seminar 7: Optimizing NeuroICU Care: Treating Patients Rather Than Seizures – Novel Therapies and Thresholds
Friday, October 10, 2025 – 9:30-10:45 AM EDT/EST

Ilana L Kahn, MD
Seminar 2: Updates in Pediatric Multiple Sclerosis
Thursday, October 9, 2025 – 2:00-3:15 PM EDT/EST

Alexandra B. Kornbluh, MD
Seminar 2: Updates in Pediatric Multiple Sclerosis – Updates in MS Diagnostic Criteria and Implications for Pediatric MS Treatment
Thursday, October 9, 2025 – 2:00-3:15 PM EDT/EST

Juma Shee Mbwana, MD
Symposium III: Year in Review Symposium – Global Pediatric Neurology
Saturday, October 11, 2025 – 9:00 AM-12:00 PM EDT/EST

John Schreiber, MD
CNS Genetics Summit: Demystifying Next-generation Sequencing: A Hands-On Workshop for Pediatric Neurologists
Saturday, October 11, 2025 – 12:30-4:30 PM EDT/EST

Kuntal Sen, MD
Workshop 7: Discovering Mentors: Early Career Leadership Guidance
Wednesday, October 8, 2025 – 3:00-4:00 PM EDT/EST
CNS Genetics Summit: Demystifying Next-generation Sequencing: A Hands-On Workshop for Pediatric Neurologists
Saturday, October 11, 2025 – 12:30-4:30 PM EDT/EST

Jeffrey Strelzik, MD
Seminar 8: The Evolution of Migraine Disease from Childhood to Young Adult – Migraine Disease in Early Childhood
Friday, October 10, 2025 – 9:30-10:45 AM EDT/EST
CNS Genetics Summit: Demystifying Next-generation Sequencing: A Hands-On Workshop for Pediatric Neurologists
Saturday, October 11, 2025 – 12:30-4:30 PM EDT/EST

Sinan Turnacioglu, MD
Seminar 9: Games Neurologists Play: AI for Enhanced Diagnosis in Neurology
Friday, October 10, 2025 – 9:30-10:45 AM EDT/EST

Poster and abstract presentations

  • From Bedside to Smartphone: Standardizing Pediatric Seizure Discharge Education Through Digital Access, Ilyse Genser, MD, Anne Vasiliadis, NP, CPNP-AC, Lauren Vilardo, MD
  • Spectrum of Neurofascin-155 Antibody Mediated Paranodopathy in a Pediatric Cohort: A Single Center Experience, Allison Saunders, MD

Additionally, Dr. DiSabella will be honored with the prestigious Program Director Award, recognizing his outstanding leadership in education, mentorship and curriculum development within child neurology and neurodevelopmental pediatrics.

For more information and to view the full schedule, please visit the CNS conference website.

A new electroencephalogram (EEG) measure may help track brain growth in Angelman syndrome

A pair of hands holding a cutout of a head

A joint study from Children’s National and UCLA findound that peak alpha frequency (PAF), a simple EEG signal, may help track brain growth in Angelman syndrome.

Angelman syndrome (AS) affects how a child’s brain develops and communicates. Families often face limited or absent speech, seizures, sleep problems and global delays. As several treatments move through late-stage trials, teams need clear ways to see if a therapy is changing the brain. A joint study from Children’s National Hospital and the University of California, Los Angeles (UCLA) points to a simple signal from an electroencephalogram (EEG), a test that records brain activity through small sensors on the scalp.

The signal is called peak alpha frequency (PAF). Alpha is a natural brain rhythm. In most children, the speed of this rhythm rises through childhood as networks mature. The study shows that this pattern is different in Angelman syndrome. Many children with AS have no clear alpha peak. When a peak is present, it is slower than in peers and does not speed up with age. That pattern suggests stalled or altered maturation of brain circuits.

Researchers looked at hundreds of EEGs from children with AS and from age-matched peers. They measured alpha activity in two independent ways to make sure the results were not due to a single analysis choice. Both approaches told the same story. In children who are typically developing, more than nine out of ten EEGs showed a distinct alpha peak, and that peak tended to get faster with age. In children with AS, fewer than half of EEGs showed a peak. When a peak appeared, it sat at a lower frequency and stayed flat across ages.

The team also confirmed a second feature of Angelman EEGs that others have reported: more slow-wave (delta) activity and less overall alpha power than peers. Taken together, these signals point to broad differences in how circuits work in Angelman syndrome.

What could this mean for care and for trials? EEG is familiar, safe, and widely available. If a therapy begins to change how circuits function, that shift could show up in PAF before day-to-day behavior changes are obvious. Adding PAF to trial measures could give clinicians and families an earlier read on brain effects. It can also sit alongside delta power to give a fuller picture – delta reflects broad disruption, while PAF tracks the speed of a key rhythm tied to growth.

There are limits to keep in mind. The EEGs came from several sites and systems, and some recordings were longer than others. The team accounted for these factors in their models, but future work with unified setups will help. A major next step is to follow the same children over time to see whether PAF changes within a child and how those changes relate to speech, movement, seizures, or sleep. The study also hints that genetic subtypes may differ, which could sharpen how trials are designed.

This project was a true collaboration between Children’s National Hospital (PI and corresponding author: Michael S. Sidorov, PhD, co-first author: Sapphire Bowen-Kauth) and UCLA (co-first author: Abigail H. Dickinson, PhD), with contributions from colleagues across both centers. Funding came from the Foundation for Angelman Syndrome Therapeutics (FAST).

For families, the takeaway is hopeful. A routine test can capture a signal linked to brain growth. In Angelman syndrome, that signal is often missing or slow, but it can be measured and tracked. As trials continue, PAF may help teams spot early signs that a therapy is reaching the brain’s core rhythms, information that matters for decisions in clinics and for progress in research.

Read the full study, “Atypical alpha oscillatory EEG dynamics in children with Angelman syndrome,”  in Neuroimage: Clinical.

Podcast: Breaking Barriers: Emerging Therapies in Pediatric Brain Tumors

Dr. Matthew Bramble named Pew Scholar in Biomedical Sciences

Matthew Bramble

“This award is a tremendous honor,” said Dr. Bramble, principal investigator in the Center for Precision Medicine and Genomics Research at Children’s National. “It will allow us to push the boundaries of our work and explore new ways to understand and treat devastating childhood diseases.”

Children’s National Hospital is proud to congratulate Matthew Bramble, PhD, on being named a 2025 Pew Scholar in the Biomedical Sciences. This highly competitive honor recognizes outstanding early-career researchers who are driving innovation and discovery in human health. Dr. Bramble is one of just 22 scientists nationwide selected this year to join the Pew Scholars Program, which provides four years of funding and access to a network of more than 1,000 leading biomedical researchers.

“This award is a tremendous honor,” said Dr. Bramble, principal investigator in the Center for Precision Medicine and Genomics Research at Children’s National. “It will allow us to push the boundaries of our work and explore new ways to understand and treat devastating childhood diseases.”

Dive deeper

Dr. Bramble’s research focuses on konzo, a little-known but serious neurological condition that causes sudden and irreversible paralysis in children. Konzo primarily affects families in rural regions of sub-Saharan Africa who rely on improperly processed cassava as a staple food. The disease is linked to compounds in cassava that become toxic when combined with poor nutrition.

“Konzo is heartbreaking because it strikes vulnerable children and is entirely preventable,” said Dr. Bramble. “If we can better understand the molecular pathways that lead to paralysis, we can develop strategies to protect children and potentially inform treatments for other neurological disorders.”

His lab uses advanced OMIC’s technologies to study how toxins from cassava interact with a child’s biology, aiming to uncover how the disease damages the nervous system. By examining the molecular biology of konzo, Dr. Bramble hopes to reveal insights not only for affected communities abroad but also for understanding broader principles of brain health and development.

Why it matters

The Pew Scholars Program in the Biomedical Sciences, run by The Pew Charitable Trusts, has supported early-career scientists for 40 years. Scholars receive multiyear funding to pursue bold, creative approaches to complex scientific problems. Many alumni have gone on to win major scientific awards and make discoveries that have shaped modern medicine.

For Dr. Bramble, joining the program provides vital support for high-risk, high-reward science and connects him to a collaborative network of researchers across disciplines.

“This award validates the importance of studying conditions like konzo that don’t often receive attention but have a profound impact on children’s lives,” he said.

Dr. Bramble’s recognition underscores not only Children’s National Hospital’s commitment to advancing pediatric research and fostering discoveries that improve children’s health worldwide but also the institution’s commitment to providing resources and a setting for this type of unique, cutting-edge work. By supporting investigators who explore fundamental biology and rare diseases, the hospital helps build the foundation for tomorrow’s treatments.

With the support of the Pew award, Dr. Bramble and his team are poised to deepen their understanding of konzo and its impact on the nervous system—and, in the process, generate insights with far-reaching implications for pediatric neurology.

Marc DiSabella, DO, honored with Child Neurology Society 2025 Training Director Award

Marc DiSabella, DO

Marc DiSabella, DO, of Children’s National Hospital has been named the 2025 recipient of the Child Neurology Society (CNS) Training Director Award.

Marc DiSabella, DO, of Children’s National Hospital has been named the 2025 recipient of the Child Neurology Society (CNS) Training Director Award. This national recognition honors program directors who demonstrate outstanding leadership in education, mentorship and curriculum development within child neurology and neurodevelopmental pediatrics.

Dr. DiSabella joined Children’s National and 2008 and currently serves as associate chief of Neurology Operations, director of the Headache Program.  He previously led the Child Neurology Residency Program and served as director of Neurology Education.

A dedicated educator, Dr. DiSabella completed the Master Teacher Leadership Development Program at the George Washington University. His innovative teaching style and commitment to excellence have helped raise the bar for neurology education at Children’s National and beyond.

“Dr. DiSabella is an exceptional, enthusiastic and lucid teacher who has introduced several successful innovations over the past 15 years,” said William D. Gaillard, MD, Chief of Neurology at Children’s National. “He has trained more than 50 child neurologists, many now leaders in the field, and built one of the country’s foremost multidisciplinary headache programs. His administrative talents and positive, can-do attitude continue to benefit the entire department.”

Dr. DiSabella will be honored during the CNS Annual Meeting in Charlotte, North Carolina, in October.

The CNS Training Director Award reflects the organization’s mission to recognize the achievements of child neurologists  who are model educators, and to recognize those who shape the future of child neurology through education, mentorship and leadership.

New advances in AI help doctors understand pediatric brain tumors

person studying brain scans

One of the first and most important steps in fighting brain tumors is to understand exactly where they are and how big they are – using magnetic resonance imaging (MRI) scans.

Brain tumors are the most common cause of cancer-related death in children. These tumors can be very difficult to treat because they often grow in very delicate areas of the brain. One of the first and most important steps in fighting these tumors is to understand exactly where they are and how big they are – using magnetic resonance imaging (MRI) scans.

Traditionally, doctors have had to look at these scans and manually draw outlines around the tumors. This takes a lot of time and can vary from one doctor to another. But what if artificial intelligence (AI) could help make this easier and more efficient?

A recent international research effort called BraTS-PEDs 2023 (short for Brain Tumor Segmentation in Pediatrics) explored exactly that. This challenge co-led by Children’s National Hospital and Children’s Hospital of Philadelphia invited research teams from around the world – including a team led by Marius George Linguraru, DPhil, MA, MSc – to develop the best AI tools for automatically finding and measuring brain tumors in children’s MRIs.

A global AI competition for pediatric care

The BraTS-PEDs 2023 challenge was the first of its kind focused specifically on children. Teams were given MRI scans from 167 pediatric patients with brain tumors collected by leading consortia – the Children’s Brain Tumor Network, DMG/DIPG Registry – and Collaborative Network for Neuro-oncology Clinical Trials and reviewed by volunteer neuroradiologists from the American Society of Neuroradiology. They used these scans to train their AI programs and then tested them on new cases to see how well they performed. Our team’s algorithm was the best at measuring these tumors.

Most teams used a special type of AI network called a “U-Net,” which is great at looking at images and figuring out shapes and boundaries. The challenge showed that AI can do a very good job at finding the tumor and measuring its volume. However, it was more difficult for AI to accurately mark the small, active parts inside the tumor, known as “enhancing tumor” regions.

These results are a huge step forward. Using AI can help doctors make faster, more precise decisions, and reduce differences between hospitals or between individual doctors.

Why this matters

When doctors can accurately map out a brain tumor, they can plan surgery better, target radiation therapy more precisely, and track how well treatments are working over time. This can lead to fewer side effects and better outcomes for children.

In some cases, timely and accurate measurements can even be the difference between life and death. Having advanced AI tools could mean that children get the right treatments faster and with more confidence.

Looking ahead

The research community is now working on including even more data from different hospitals around the world and making the data and algorithm public, like Children’s National has done here. They also plan to study more types of brain tumors and scans taken after surgery and treatments. In the future, AI could become a regular part of how doctors look at brain tumors — like an extra set of smart eyes that never get tired.

At Children’s National, leaders like Dr. Linguraru are helping turn this vision into reality, giving children with brain tumors a better chance at a healthy future. You can read the full study – BraTS-PEDs: Results of the Multi-Consortium International Pediatric Brain Tumor Segmentation Challenge 2023 – in the MELBA Journal.

Other authors from Children’s National include Roger J. Packer, MD, Brian Rood, MD, Miriam Bornhorst, MD, Xinyang Liu, PhD, Zhifan Jiang, PhD, and Syed Muhammad Anwar, PhD, MS.