Tag Archive for: Elizabeth Wells

illustration of antibodies attacking neurons

Therapy approach shows promise for PANS/PANDAS

illustration of antibodies attacking neurons

PANS and PANDAS are disorders characterized by an abrupt onset of neuropsychiatric symptoms in children that can be challenging to diagnose and treat.

Children diagnosed with pediatric acute-onset neuropsychiatric syndrome (PANS) and pediatric autoimmune neuropsychiatric disorders associated with streptococcal infection (PANDAS) experienced substantial symptom reduction following a cognitive-behavioral approach integrating the family system with the support of a multidisciplinary care team, according to a recent case study published in Clinical Case Studies.

The hold up in the field

PANS and PANDAS are disorders characterized by an abrupt onset of neuropsychiatric symptoms in children that can be challenging to diagnose and treat.

“More research is needed to explore PANS/PANDAS as many studies have included small samples or lacked a randomized controlled design,” says Lilia Mucka Andrew, PhD, clinical director, Psychology and Behavioral Health at Children’s National Hospital and corresponding author of the study. “Though therapeutic interventions like cognitive behavioral therapy with exposure response prevention (CBT/ERP) have good evidence for pediatric patients with obsessive-compulsive disorder (OCD), evidence is mixed on effectiveness in PANS/PANDAS patients.”

Moving the field forward

The researchers highlighted specific intervention techniques that worked well to improve behaviors and factors that warrant further exploration. This includes the benefit of early multidisciplinary evaluation for diagnosis and treatment of PANS/PANDAS and the integration of booster sessions to allow for ongoing maintenance and relapse prevention while providing openings for additional patients to be seen. This is critical given the shortage of evidence based behavioral health providers for youth.

The patient benefit

This study highlights effective strategies that support both patients and their families, offering a potential model of care to reduce suffering and improve long-term quality of life.

“We were excited to see that the application of key evidence-based therapy techniques including CBT/ERP and behavioral parent training (BPT) resulted in positive outcomes for patients and that the opportunity to transition to booster sessions was well received,” says Dr. Andrew.

Additional authors from Children’s National include: Julia Dorfman, MD, PhD, psychiatrist, and Elizabeth Wells, MD, senior vice president, Neuroscience and Behavioral Medicine Center.

You can read the full study, Assessment and Treatment of PANS/PANDAS: A Multidisciplinary Approach and Therapeutic Considerations, in Clinical Case Studies.

Code Stroke management algorithm

Stroke alert teams beneficial to timely diagnosis of pediatric stroke patients

Key driver diagram

1 – Interdisciplinary stroke simulations were canceled due to short staffing and insufficient nursing support during COVID-19 pandemic.

Diagnosis times improved for pediatric arterial ischemic stroke (AIS) patients in the emergency department after the implementation of a quality improvement initiative. According to a new study, published in Pediatrics, the median door-to-imaging time for children with suspected AIS improved from 128 minutes to 68 minutes post-intervention.

What it means

Pediatric AIS is an important cause of morbidity and mortality that requires early recognition to benefit from hyperacute therapies. A quality improvement study at Children’s National Hospital aimed to improve timely diagnosis of AIS through an interdisciplinary stroke response protocol. The researchers implemented a quality improvement initiative from November 2019 to June 2023 in the emergency department that included interventions under two categories – workflow efficiency improvement and staff education. A total of 71 patients who met the criteria for the study were analyzed. Median door-to-imaging time for all patients improved from 128 minutes during the baseline period to 68 minutes.

The researchers note that establishing a well-functioning stroke response protocol is a critical step in detecting pediatric stroke in the community and expediting care for this vulnerable patient population.

“This study highlights the value of quality improvement methodologies in coordinating multiple hospital divisions for the greater goal of improving patient care,” says Theodore Trigylidas, MD, corresponding author and emergency medicine physician at Children’s National. “We hope it serves as a template for other healthcare organizations in developing their own pediatric stroke protocols.”

Code Stroke management algorithm

Code Stroke management algorithm. CT/CTA, computed tomography with or without angiography. MRI/MRA, magnetic resonance imaging with or without angiography.

The hold up in the field

The rarity of AIS in pediatric patients and stroke mimics, like hemiplegic migraine, may cause challenges for a timely diagnosis, and treatments for AIS – thrombolysis and thrombectomy – must be performed within specific time frames.

Moving the field forward

“By taking into consideration local staffing structures and institutional resource availability, the quality improvement approach described here can serve as a framework for how institutions might implement a pediatric acute stroke response protocol given their own unique systems of care and resource considerations,” says Dana Harrar, MD, PhD, study author and director, Pediatric Stroke Program at Children’s National.

The authors plan to expand this initiative to incorporate children at outside facilities with the goal of creating a stroke network with nearby hospitals.

Authors from Children’s National include: Theodore Trigylidas, MD, Nichole McCollum, MD, Kathleen Brown, MD, Paola Pergami, MD, Elizabeth Wells, MD, Jonathan Murnick, MD, PhD, Josh Heffren, PharmD, BCPPS and Deborah LaViolette, BSN, Dana Harrar, MD, PhD.

You can read the full study, Improving Timely Diagnosis of Arterial Ischemic Stroke at the Pediatric Emergency Department in Pediatrics.

illustration of the brain on black background

Children’s National Hospital to host 35th Annual Pediatric Neurology Update

illustration of the brain on black backgroundThe Divisions of Neurology and Neurosurgery at Children’s National Hospital are proud to host the 35th Annual Pediatric Neurology Update course.

Chair Elizabeth Wells, MD; Co-Chairs, Marc DiSabella, DO, John Schreiber, MD, William D. Gaillard, MD, Robert Keating, MD

The course attracts a national audience and brings together neuroscience clinicians and pediatricians in the Washington, D.C. and Mid-Atlantic region.

Guest speakers include Annapurna Poduri, MD, MPH, Deputy Director for NINDS, Emily Freilich, MD, from the FDA and Conor Mallucci, MBBS, Chief of Neurosurgery at Alder Hays, England.

This year’s course highlights 3 major areas:

  • Updates in Epilepsy
  • Innovations in Vascular Neurosurgery and Neurointerventional Radiology
  • Addressing Mental and Behavioral Health in Neurological Conditions

We invite you to join us for presentations from experts in the field during this full-day, CME accredited event on April 10, 2025. This is a hybrid event that will be held virtually or in-person at the Children’s National Research & Innovation Campus.

For more information and to register, visit ChildrensNational.org/NeurologyUpdate.

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.

Hiram receives the first commercial dose of Elevidys

Children’s National gives first commercial dose of new FDA-approved gene therapy for Duchenne muscular dystrophy

Hiram receives the first commercial dose of Elevidys

On the day before his 6th birthday, Hiram, 5, was the first patient ever with DMD to receive the drug after the U.S. Food and Drug Administration (FDA) approved its use last month.

Children’s National Hospital is the first pediatric hospital to administer a commercial dose of Elevidys (delandistrogene moxeparvovec-rokl), the first gene therapy for the treatment of pediatric patients with Duchenne muscular dystrophy (DMD).

On the day before his 6th birthday, Hiram, 5, was the first patient ever with DMD to receive the drug after the U.S. Food and Drug Administration (FDA) approved its use last month.

“The approval of Elevidys opens a new door for young patients with DMD and their families,” says Sarah Wright, D.O., neuromuscular neurologist at Children’s National. “This disease has had limited targeted treatments to date which can help alter the trajectory of disease.”

The background

On June 22, the FDA approved the use of Elevidys for patients 4 through 5 years of age with DMD with a confirmed mutation in the DMD gene who do not have a pre-existing medical reason preventing treatment with this therapy.

DMD is a rare and progressive genetic neuromuscular disease that predominantly affects males. It is caused by genetic changes in the DMD gene that affects the muscles, leading to muscle wasting that gets worse over time. Symptoms include progressive weakness and loss (atrophy) of both skeletal and heart muscle. Muscle weakness is usually noticeable in early childhood when signs like delayed ability to sit, stand or walk, and difficulties learning to speak manifest in a patient.

How it works

Elevidys is a one-time intravenous gene therapy that aims to delay or halt the progression of DMD by delivering a modified, functional version of dystrophin to muscle cells. The dystrophin gene is the largest known human gene.

“Elevidys is a viral vector (the ‘envelope to deliver the gene of interest’) mediated gene therapy that allows for the introduction of a gene that codes for a shortened form of dystrophin protein, or microdystrophin,” Dr. Wright explains. While not a cure for DMD, trials of Elevidys have demonstrated increases in dystrophin expression and improved functional results in young children with the disease.

“We have years of dedicated work on the part of researchers, clinician leaders and advocacy organizations in the field of muscular dystrophy to thank for this ground-breaking moment,” says Dr. Wright. “The approval of Elevidys offers families of patients ages 4-5 with DMD the option to receive this gene therapy that is designed to target the underlying cause of the disease.”

“The time-sensitivity of this medication illustrates the importance of going to a top academic pediatric hospital early on in neurologic care,” adds Elizabeth Wells, M.D., senior vice president of the Center for Neuroscience and Behavioral Medicine at Children’s National.

What’s next

The neuromuscular team at Children’s National is looking forward to offering this therapy to young patients with DMD and to the completion of additional trials/results for therapies in the DMD drug development pipeline.

“The research and approval of novel therapies provides more options for our DMD patients and their families, which is a critical step toward improving the lives of patients with DMD,” Dr. Wright says.

Drs. Wells and Kenworthy

Center for Neuroscience and Behavioral Medicine announces new leaders

Drs. Wells and Kenworthy

Elizabeth M. Wells, M.D., M. H. S., was named Senior Vice President of the Center for Neuroscience and Behavioral Medicine, and Lauren Kenworthy, Ph.D., was named division chief of Neuropsychology.

The Center for Neuroscience and Behavioral Medicine at Children’s National Hospital recently announced the appointment of two new leaders. Elizabeth M. Wells, M.D., M. H. S., was named Senior Vice President of the Center for Neuroscience and Behavioral Medicine, and Lauren Kenworthy, Ph.D., was named division chief of Neuropsychology.

Dr. Wells obtained her undergraduate degree in psychology and biology at Harvard University and her medical degree at the George Washington University School of Medicine and Health Sciences. She was an Intramural Research Training Award fellow at the National Institute of Mental Health and holds a master’s in Health Science from the NIH/Duke Clinical Research Training Program. She completed pediatrics and neurology training at Children’s National and joined the faculty in the Brain Tumor Institute in 2011.

Dr. Wells has led the Children’s National Inpatient Neurology program and developed the hospital’s multidisciplinary Neuro-immunology program into a destination program for unsolved neuroinflammatory diseases. She serves on numerous national and international associations and working groups and is a member of the scientific selection committee for the Child Neurology Society.

Dr. Wells has served in leadership roles for the Clinical and Translational Science Institute at Children’s National and the District of Columbia Intellectual and Developmental Disabilities Research Center. She is principal investigator for a 10-year translational research study within the Children’s National partnership with the National Institute of Allergy and Infectious Diseases and was the Children’s National Hospital Medical Staff President from 2020-2022.

Dr. Kenworthy received a B.A. from Yale University and Ph.D. from the University of Maryland. She completed her internship and residency training in clinical psychology/pediatric neuropsychology at Harvard Medical School, Children’s Hospital Boston, Johns Hopkins Medical School and Mount Washington Pediatric Hospital. She has been on the faculty at Children’s National and GW since 1995. She is a national leader in autism research, as well as a distinguished author and speaker.

neurons

Recommendations for the treatment of pediatric NMDAR antibody encephalitis

neurons

NMDA receptor antibody encephalitis (NMDARE) is one of the most common autoimmune encephalitides characterized by a recognizable constellation of neurologic and psychiatric features alongside positive NMDAR antibodies.

In a new study published in Neuroimmunology and Neuroinflammation, authors, including Elizabeth Wells, M.D., vice president of Neuroscience and Behavioral Medicine Center at Children’s National Hospital, created a consensus recommendation for the treatment of pediatric NMDARE, which was pragmatic and relevant to a global community and could serve as a practical decision support tool for the clinician confronted with this rare and challenging condition.

The authors conclude that their recommendations for the management of pediatric NMDARE aim to standardize the treatment and provide practical guidance for clinicians, rather than absolute rules. A similar recommendation could be applicable to adult patients.

Read the full article in Neuroimmunology and Neuroinflammation.

Dr. Wells with patient

Elizabeth Wells, M.D., named Vice President of the Neuroscience and Behavioral Medicine Center

Dr. Wells with patient

Elizabeth Wells, M.D., Vice President of the Neuroscience and Behavioral Medicine Center, interacting with patient.

Elizabeth Ann Molloy Wells, M.D., MHS, has been appointed to the role of Vice President of the Neuroscience and Behavioral Medicine Center at Children’s National Hospital. This new role has been created to further the growth of the Center, broaden and deepen the leadership structure and allow Children’s National to continue to deliver the highest level of care, education, safety and scholarship for our patients and families. “I joined Children’s National 15 years ago as a pediatric neurology resident because I thought it was the best place to train and develop in academic neurology, and I am so honored to serve as the Neuroscience Center Vice-President” said Dr. Wells.

Dr. Wells is a graduate of Harvard University and the George Washington University School of Medicine and Health Sciences. She holds a master’s in Health Science from the NIH-Duke Clinical Research Training Program. Dr. Wells completed her pediatrics and neurology training at Children’s National and has been on staff as a pediatric neurologist within the Brain Tumor Institute and the Division of Neurology for the past 10 years. In addition to caring for children with neurologic effects from cancer, Dr. Wells developed the multidisciplinary program in pediatric neuro-immunology. She serves on numerous national committees and receives national and international referrals for children with neuro-inflammatory disorders. She is a principal investigator for translational research studies and serves in a leadership role for the Clinical and Translational Science Institute and the District of Columbia Intellectual and Developmental Disabilities Research Center.  Dr. Wells has been director of Inpatient Neurology and the Neuroscience Medical Unit since 2015 and was elected president of the medical staff in July 2020.

During her time at Children’s National, Dr. Wells has become known for her communication skills, team building and tireless commitment to excellence. She will expand the Neuroscience Center’s work on quality and safety, medical informatics, diversity and inclusion and patient experience.  “I am especially excited to promote growth and visibility for developing and expanding Neuroscience programs. Doing so will enable us to serve more kids and spread knowledge and expertise for children affected by brain disorders and injuries. I also look forward to fostering our culture of teamwork” said Dr. Wells. “There is a sense of urgency in the Neuroscience and Behavioral Medicine Center to rapidly translate discoveries into answers for children and families, better treatments and tools to support strong and healthy lives.”

Acute flaccid myelitis concept illustration

Causes, diagnosis and management of acute flaccid myelitis

Acute flaccid myelitis concept illustration

Acute flaccid myelitis (AFM) is a disabling, polio-like illness mainly affecting children. Outbreaks of AFM have occurred across multiple global regions since 2012, and the disease appears to be caused by non-polio enterovirus infection, posing a major public health challenge. Children’s National Hospital was part of a multi-center study focused on AFM and published in The Lancet.

Children’s National authors include Elizabeth Wells, M.D., director of Inpatient Neurology; Jessica Carpenter, M.D., director of the Neonatal and Childhood Stroke Program and co-director of the Neurocritical Care Program; and Roberta DeBiasi, M.D., M.S., chief of the Division of Infectious Diseases.

This review describes the epidemiology, clinical features, course and outcomes of AFM to help to guide diagnosis, management and rehabilitation. Future research directions include further studies evaluating host and pathogen factors, including investigations into genetic, viral and immunological features of affected patients, host-virus interactions and investigations of targeted therapeutic approaches to improve the long-term outcomes in this population.