Maria Susana Rueda Altez

Maria Susana Rueda Altez, M.D., to lead as Junior Section President-Elect

Maria Susana Rueda Altez

Maria Susana Rueda Altez, M.D., junior section president-elect for the Society for Pediatric Research (SPR).

Maria Susana Rueda Altez, M.D., was selected as junior section president-elect for the Society for Pediatric Research (SPR). During her tenure, Dr. Rueda Altez will ensure more trainees benefit from networking opportunities and leverage her online communications experience to increase awareness, membership and participation in SPR among students, residents and fellows.

The president of the junior section is a fellow who is elected by other junior member peers and is in-charge of managing and enhancing the junior section, by participating in SPR council meetings, promoting membership among trainees and reinforcing the pipeline from junior to active members.

“I am so honored, not only as a Peruvian physician, but as an international medical graduate (IMG), to have been elected for this position,” said Dr. Rueda Altez. “As an IMG, there are special challenges to conducting research, so I plan to raise awareness and provide support to my fellow IMG junior members.”

To Beth A. Tarini, M.D., M.S., SPR president and associate director for the Center for Translational Research at Children’s National Hospital, it is an honor for the hospital to have representatives in the roles of SPR president and SPR junior section president-elect simultaneously.

Dr. Rueda Altez added that there is an urgent need for increased funding in pediatric research, especially for minority and health disparities research. Through her participation in SPR, she will also have the opportunity to advocate for increases in child health research funding.

“I encourage all the trainees and junior faculty in our institution to join the SPR junior section,” said Dr. Rueda Altez. “It provides wonderful resources for career development and guidance, grant writing courses and invaluable mentorship.”

Her research interest is newborn infections, and her overall goal is to reduce the unnecessary use of antibiotics in this population.

“I am currently working on a quality improvement project to reduce the number of days NICU infants are exposed to antibiotics,” said Dr. Rueda Altez. “I have developed a project to ascertain the utility of microbial cell-free DNA next generation sequencing, a novel microbiologic diagnostic tool, for the diagnosis of neonatal infections.”

Dr. Rueda Altez’s work on neonatal sepsis will help scientists better distinguish between neonates who do and don’t have serious bacterial infections.

“Right now, when in doubt we tend to treat it as bacterial infections, which can lead to unnecessary medical treatment and worsen resistance to antibiotics,” said Tarini.

Dr. Rueda Altez also serves as an independent reviewer of investigational manuscripts for The Journal of Pediatrics and Pediatrics and guest editor for The Journal of Pediatrics. Her passion for the peer-review process also shows in her long list of published research.

Dr. Tarini also foresees multiple research trends in the next five years that might appear in peer-reviewed publications.

“We have so much to tackle in child health research, both ongoing and new challenges,” said Dr. Tarini.  “Some issues that come to mind are the mental health crisis in children and teens, continuing to make strides on treating and preventing childhood obesity, the effect of poverty on children’s health, and the pandemic’s effect on all of these issues and its direct effect on health outcomes.”

x-ray of human skull

Researchers awarded $3.5 million to study brain and cranium development in children

x-ray of human skull

Currently, studies on typical brain and cranium development are limited. One reason for this is that imaging techniques are optimized to best visualize either bone or soft tissue, but not both.

With prevalence of developmental disorders on the rise, the need to understand brain development has never been more critical. Development of the brain is strongly influenced by the cranium, but this relationship has not been adequately studied because of limitations in imaging technology. Now, researchers from Children’s Hospital Los Angeles and Children’s National Hospital are working together to develop techniques that will provide greater insight into this relationship. Their studies will be funded by The National Institute of Dental and Craniofacial Research, which has awarded them $3.5 million.

Natasha Leporé, Ph.D., of Children’s Hospital Los Angeles, studies methods to interpret brain imaging data. “There’s a lot of interaction between the skull and the brain,” she says, “and we want to better understand how they grow together.”

Currently, studies on typical brain and cranium development are limited. One reason for this is that imaging techniques are optimized to best visualize either bone or soft tissue, but not both.

The brain — mostly composed of water, protein and fat — doesn’t show up well on computerized tomography (CT) scans, which use X-ray images. In addition, radiation exposure limits the amount of CT scan data available in children. On the other hand, magnetic resonance imaging (MRI) scans are excellent for brain images but are not optimal for surrounding bone.

This presents researchers with a dilemma if they want to see the brain and the skull together in one image. Fortunately, research barriers like these are often overcome by collaboration.

Leporé will work with Marius George Linguraru, D.Phil, M.A., MS.c., principal investigator in the Sheikh Zayed Institute for Pediatric Surgical Innovation at Children’s National Hospital.

Linguraru works on a set of tools for cranial phenotyping, using existing CT images from typically developing children. In their collaboration, Leporé and Linguraru will extend the tools to MRI scans, allowing the team to analyze the brain and cranium simultaneously. The pair has received a $3.5 million award over 5 years.

“The tools we develop together will help us to better understand the healthy growth of children,” says Linguraru. “We will have the ability to analyze the joint cranial and brain development from large medical image datasets of pediatric patients.”

This, the team says, will be invaluable to the medical community.

“These tools will help clinicians to better assess, diagnose and plan treatment for infants with cranial deformities,” says Linguraru.

Collaborations like this allow expertise to be shared across specialties, ultimately benefiting children in need. Exceptional pediatric care is a result of teamwork; not only doctors, nurses and clinical staff, but also biomedical research, which arms clinicians with the information they depend on.

“We need to have a clear idea of what is expected in normal development,” says Leporé. “This allows doctors to detect and better understand differences in development.”

Other members of the research team include: Vidya Rajagopalan, Ph.D.; Marvin Nelson, M.D.; Alexis Johns, Ph.D.; Niharika Gajawelli, Ph.D. (from Children’s Hospital Los Angeles and University of Southern California); Robert Keating, M.D. (Children’s National Hospital); Yalin Wang, Ph.D. (Arizona State University); Antonio Porras, Ph.D. (University of Colorado); Sean Deoni, Ph.D. (Rhode Island Hospital and Brown University).

A version of this story appeared on the Children’s Hospital Los Angeles newsroom.

Bear Institute PACK logo

Winners of the first annual Bear Institute PACK Event

Bear Institute PACK logo

On August 26, 2021, the Bear Institute, along with Children’s National Hospital and Cerner Corporation, hosted the first annual Bear Institute PACK (Pediatric Accelerator Challenge for Kids). Bear Institute PACK is a start-up competition aimed to address the gap in digital health innovation funding dedicated to children.

“Children are a unique population that requires different health solutions than those designed for adults, which address their unique needs,” says Dr. Lu de Souza, Vice President and Chief Medical Officer, Cerner Corporation. “With Bear Institute PACK, we hope to increase focus and delivery of digital health innovations for kids. Bear Institute PACK brings together the pediatric health care community, including pediatric health care providers and hospital administrators from across the country to identify top start-up digital applications that best serve children.”

This year’s start-up participants competed across four innovation tracks, including rare disease, telemedicine, remote patient monitoring and patient education. Student teams competed in a separate student track. Bear Institute PACK consists of three rounds of judging: an initial review of applications from the Bear Institute PACK team, judging from participating pediatric healthcare providers and administrators and review from an expert panel of judges during finalist start-ups’ live pitches.

The start-ups competed for a rich prize pool, including cash prizes totaling over $100,000, on-site pilots and software development support. Winners were selected in each of the event’s four innovation tracks, as well as an additional two student team winners. This year winners are:

  • In the rare disease track, first place winner, Bloom Standard, Inc., with its solution Automated Ultrasound Wrap that screen infants and children for serious lung and cardiac conditions, and second place winner, Mira Medical LLC, with its solution Bear Growth: A Three-Dimensional Pediatric Growth Modeling App.
  • In the telehealth track, Keriton, Inc., with its solution Keriton Kare, a healthcare SaaS platform built to improve outcomes for neonatal and pediatric patients.
  • In the remote patient monitoring track, Sonavi Labs, with its solution Feelix, a platform that features proprietary hardware embedded with clinically validated diagnostic software capable of detecting respiratory diseases.
  • In the patient education track, Smileyscope, with its comprehensive virtual reality (VR) platform to help support patients with procedural pain management, drug-free anxiety care, education, and guided relaxation.
  • Student team first place winner, CASP Technologies, with its solution Operation Serenity, which allows pediatric patients to prepare for and understand their simulated surgery to reduce anxiety and second place winner, Ankle Rehab, with its solution Foot Joystick for Children with Cerebral Palsy meant to improve mobility.

More information on this year’s winners can be found on the Bear Institute PACK website.

“This year’s Bear Institute PACK had a lot of start-ups and student team participants with very impressive innovation solutions for kids. Selecting a single winner in each innovation track was a tough decision, and it was encouraging to see all the work being done to bring these solutions to market,” says Matt MacVey, Vice President and Chief Information Officer, Children’s National Hospital. “Thank you to everyone who participated and helped make the inaugural Bear Institute PACK a success! We hope to make next year even bigger as we continue to strive to close the gap in funding for children’s digital health innovation.”

More information on next year’s event will be forthcoming on the Bear Institute PACK website.

Could whole-exome sequencing become a standard part of state newborn screening?

smiling baby boy

There are concerns about implementing whole-exome sequencing since it takes away the child’s right to decide if they want to know — or not — about their specific inherited disease.

It is still premature to standardize an innovative methodology known as whole-exome sequencing (WES) as part of state newborn screening programs, argues Beth A. Tarini, M.D., M.S., associate director for the Center of Translational Research at Children’s National Hospital, in a new editorial published in JAMA Pediatrics.

About 4 million infants are born annually in the United States. Newborn screening is a mandatory state-run public health program that screens infants for inherited diseases in the first days of life so they can receive treatment before irreversible damage occurs. Several of these screening tests are done on blood drawn from an infant’s heel.

WES holds the potential to screen infants for thousands of disorders and traits, including those that appear in adulthood. But there are concerns about implementing WES since it takes away the child’s right to decide if they want to know — or not — about their specific inherited disease. There is also the unknown effect that it could have on their ability to obtain health insurance.

“As caretakers for their children, parents have the challenge of deciding what kind of information, including genetic, will be valuable for their child,” says Dr. Tarini. “As a society, we have the responsibility of deciding where the healthcare dollars get the best return – especially when it comes to children. We need to start that conversation for universal genomic sequencing of newborns sooner rather than later.”

The Pereira et al. study, appearing in the new edition of JAMA Pediatrics and referenced in Dr. Tarini’s editorial, is the first to demonstrate no significant harm in the initial 10 months of life after performing WES under the best conditions of access to resources and a controlled environment.

While the Pereira et al. study has limited data on the effects of WES on families from underrepresented backgrounds, Dr. Tarini notes that it does provide a critical first step in this area of pediatric genomic research and for policy decision-making about the widespread implementation of WES in newborns.

“Moving forward, the U.S. will have to make a collective decision about the value of WES for newborns,” says Dr. Tarini. That value calculus cannot be made without consideration of the general state of healthcare for infants. As she points out, “This is not an easy question to answer in a country whose infant mortality ranks 34th according to the Organization for Economic Co-operation and Development (OECD).”

Dr. Tarini’s research identifies ways to optimize the delivery of genetic services to families and children, particularly newborn screening. She has also chaired state newborn screening committees and served on several federal newborn screening committees.

morphine vial and needle

Replacing morphine with methadone in the NICU

morphine vial and needle

A synthetic analgesic drug, known as methadone, may serve as a better alternative for newborns in the neonatal intensive care unit (NICU) suffering from opioid withdrawal syndrome, according to a commentary published in Pediatric Research.

A synthetic analgesic drug, known as methadone, may serve as a better alternative for newborns in the neonatal intensive care unit (NICU) suffering from opioid withdrawal syndrome, according to a commentary published in Pediatric Research. Some existing literature suggests that methadone may also address painful stimuli that hinders neurodevelopment throughout adulthood, added Johannes van den Anker, M.D., Ph.D., division chief of Clinical Pharmacology at Children’s National Hospital.

The commentary was selected as the Editor’s Focus in Pediatric Research for the June editionsignaling the scientific community as noteworthy to further explore methadone’s potential as an alternative for pharmacologic treatments instead of morphine.

“It is important to define the pharmacokinetics and pharmacodynamics of methadone to treat pain in neonates in intensive care before replacing morphine with methadone. Pre-clinical research shows that the use of methadone might have fewer side effects than morphine,” said Dr. van den Anker. “If this is also the case in the human neonate, then a shift from morphine to methadone might be beneficial. However, first, we need to define what the safe and effective dose of methadone will be for this purpose.”

While there is a need to better understand how newborns and preemies metabolize methadone, there is existing knowledge that this drug minimizes pain. The commentary, too, raises the question for clinicians to possibly consider methadone as a better option to avoid long-term negative neurodevelopmental consequences — such as hypersensitivity to re-injury in later life — usually associated with pain.

The current but limited data out there still provides “exciting and stimulating” information about the possible use of methadone for the treatment of neonatal pain in the NICU, according to Dr. van den Anker. He believes that, in the future, methadone could also serve as mechanism-based analgesia in newborns experiencing pain.

“There needs to be a collaboration between neonatal medicine specialists, pharmacometricians and developmental pharmacologists to assure not only the generation of evidence-based data to determine these optimal dosing regimens, but also to facilitate the implementation of this new knowledge into daily clinical care in neonatal intensive care units across the globe,” added Dr. van den Anker.

US News badges

For fifth year in a row, Children’s National Hospital nationally ranked a top 10 children’s hospital

US News badges

Children’s National Hospital in Washington, D.C., was ranked in the top 10 nationally in the U.S. News & World Report 2021-22 Best Children’s Hospitals annual rankings. This marks the fifth straight year Children’s National has made the Honor Roll list, which ranks the top 10 children’s hospitals nationwide. In addition, its neonatology program, which provides newborn intensive care, ranked No.1 among all children’s hospitals for the fifth year in a row.

For the eleventh straight year, Children’s National also ranked in all 10 specialty services, with seven specialties ranked in the top 10.

“It is always spectacular to be named one of the nation’s best children’s hospitals, but this year more than ever,” says Kurt Newman, M.D., president and CEO of Children’s National. “Every member of our organization helped us achieve this level of excellence, and they did it while sacrificing so much in order to help our country respond to and recover from the COVID-19 pandemic.”

“When choosing a hospital for a sick child, many parents want specialized expertise, convenience and caring medical professionals,” said Ben Harder, chief of health analysis and managing editor at U.S. News. “The Best Children’s Hospitals rankings have always highlighted hospitals that excel in specialized care. As the pandemic continues to affect travel, finding high-quality care close to home has never been more important.”

The annual rankings are the most comprehensive source of quality-related information on U.S. pediatric hospitals. The rankings recognize the nation’s top 50 pediatric hospitals based on a scoring system developed by U.S. News. The top 10 scorers are awarded a distinction called the Honor Roll.

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.

Below are links to the seven Children’s National specialty services that U.S. News ranked in the top 10 nationally:

The other three specialties ranked among the top 50 were cardiology and heart surgerygastroenterology and gastro-intestinal surgery, and urology.

PAS Logo

Children’s National participants share their expertise at PAS meeting

PAS Logo

The 2021 Pediatric Academic Societies (PAS) Virtual meeting hosted live-streamed events, on-demand sessions with live Q+A, a virtual exhibit hall, poster presentations and networking events that attracted pediatricians and healthcare providers worldwide. Among the physician-scientists, there were over 20 Children’s National Hospital-affiliated participants at this year’s meeting, adding to the conversation of pediatric research in specialty and sub-specialty areas.

Children’s National experts covered a range of topics, including heart disease, neurology, abnormal glycemia in newborns and antibiotic use in hospitalized children.

The “Neurological Implications of Abnormal Glycemia in Neonatal Encephalopathy and Prematurity” was a hot topic symposium presented by a panel of experts, including Sudeepta Basu, M.B.B.S., M.S., neonatologist at Children’s National.

The experts addressed the importance of recognizing early blood glucose disturbances in newborns with encephalopathy following birth asphyxia and its likely impact on brain injury and long-term outcomes. Although whole body cooling for newborns with encephalopathy after birth asphyxia is now standard of care in most advanced centers like Children’s National, many newborns still die or have neurological impairments. Dr. Basu emphasized on the need of continued advances in newer therapies and optimizing intensive care support for these vulnerable newborns immediately after birth. Dr. Basu’s presentation focused on the association of not only low blood glucose (hypoglycemia) but also high blood glucose (hyperglycemia) with abnormal motor, visual and intellectual outcomes in surviving newborns.

“Recognizing the problem is the first step for further advancement,” Dr. Basu said. “The scientific community needs to recognize the importance of early glucose status as an early marker for disease severity and risk of brain injury.” To sum up, Dr. Basu drew attention to recent newborn resuscitation guidelines from the International Liaison Committee on Resuscitation (ILCOR), which recommends close monitoring of blood glucose levels and optimizing supportive care to maintain it within normal range. Dedicated clinical trials are the need of the hour to guide what are “normal” glucose levels in newborns with encephalopathy and what treatment options are most beneficial.

Rana F. Hamdy, M.D., M.P.H., M.S.C.E., director of the Children’s National Antimicrobial Stewardship Program, delved into the increased number of children receiving care for acute conditions – like acute respiratory tract infections – from urgent care centers and direct-to-consumer (DTC) telemedicine companies during her session “Implementing Antibiotic Stewardship in Telemedicine and Urgent Care Settings.”

Telemedicine, in this case, refers to DTC telemedicine companies—not to be confused with the telemedicine established with primary care providers, like the services provided by Children’s National.

There has been little research focused on promoting good antibiotic stewardship in urgent care settings that tend to overprescribe antibiotics compared to a primary care setting. In addition to her work focusing on improving antimicrobial use within Children’s National, Dr. Hamdy has led collaborative quality improvement work nationally in both the pediatric urgent care and DTC telemedicine settings.

“What we’ve learned from our work with the DTC telemedicine setting is that leadership commitment coming from the company is a necessary core element,” Dr. Hamdy said. “There may be unique opportunities in the telemedicine setting to employ the home-grown computer systems for antimicrobial stewardship interventions, for example, incorporating clinical decision support or feedback reports into the electronic health record systems or displaying a commitment letter in the virtual waiting room.”

In the urgent care setting, Dr. Hamdy’s team recruited approximately 150 pediatric urgent care providers to participate in the national quality improvement initiative. Communication training modules for pediatric urgent care providers with scripted language for target infectious conditions — acute otitis media, pharyngitis and otitis media with effusion — were among the successful intervention approaches that led to improved appropriate antibiotic prescribing practices, according to her team’s findings.

“Understanding the prescribing practices in the urgent care setting is important to knowing where and how to focus on target conditions and to be able to support with education and resources,” Dr. Hamdy said. “And understanding the perceived barriers to judicious antibiotic prescribing can help to identify the highest yield interventions.”

This also reflects the approach taken by the outpatient antibiotic stewardship team at the Children’s National Goldberg Center, led by Ariella Slovin, M.D., primary care pediatrics provider at Children’s National Hospital. Dr. Slovin’s oral abstract entitled “Antibiotic Prescribing Via Telemedicine in the Time of COVID-19,” examined the effect that a shift to telemedicine due to the COVID-19 pandemic had on antibiotic use for acute respiratory tract infections. Overall, her team found a decrease in the proportion of acute respiratory tract infections prescribed antibiotics and concluded that the shift to telemedicine did not adversely affect judicious antibiotic prescribing for acute respiratory tract infections.

Other participants from Children’s National included: Taeun Chang, M.D.; Yuan-Chiao Lu, Ph.D.; Chidiogo Anyigbo, M.D., M.P.H.; Panagiotis Kratimenos, M.D.; Sudeepta Basu, M.B.B.S., M.S.; Ashraf Harahsheh, M.D., F.A.C.C., F.A.A.P.; Rana F. Hamdy, M.D., M.P.H., M.S.C.E.; John Idso, M.D.; Michael Shoykhet, M.D., Ph.D.; Monika Goyal, M.D.; Ioannis Koutroulis, M.D., Ph.D., M.B.A.; Josepheen De Asis-Cruz, M.D., Ph.D.; Asad Bandealy, M.D., M.P.H.; Priti Bhansali, M.D.; Sabah Iqbal, M.D.; Kavita Parikh, M.D.; Shilpa Patel, M.D.; Cara Lichtenstein, M.D.

To view the PAS phase I mini session list and the various areas of expertise at Children’s National, visit: https://innovationdistrict.childrensnational.org/childrens-national-hospital-at-the-2021-pediatric-academic-societies-meeting/

The PAS virtual conference phase II starts on Monday, May 10 and it goes through Friday, June 4. Those interested in attending may still register for phase II here: http://2021.pas-meeting.org/registration/

Purkinje cell

Premature birth disrupts Purkinje cell function, resulting in locomotor learning deficits

Purkinje cell

Children’s National Hospital researchers explored how preterm birth disrupts Purkinje cell function, resulting in locomotor learning deficits.

As the care of preterm babies continues to improve, neonatologists face new challenges to ensure babies are protected from injury during critical development of the cerebellum during birth and immediately after birth. How does this early injury affect locomotor function, and to what extent are clinicians able to protect the brain of preterm babies?

A new peer-reviewed study by Aaron Sathyanesan, Ph.D., Panagiotis Kratimenos, M.D., Ph.D., and Vittorio Gallo, Ph.D., published in the Proceedings of the National Academy of Sciences of the United States of America (PNAS), explores exactly what neural circuitry of the cerebellum is affected due to complications that occur around the time of birth causing these learning deficits, and finds a specific type of neurons — Purkinje cells — to play a central role.

Up until now, there has been a sparsity of techniques available to measure neuronal activity during locomotor learning tasks that engage the cerebellum. To surmount this challenge, Children’s National used a multidisciplinary approach, bringing together a team of neuroscientists with neonatologists who leveraged their joint expertise to devise a novel and unique way to measure real-time Purkinje cell activity in a pre-clinical model with clinical relevance to humans.

Researchers measured neural circuit function by pairing GCaMP6f fiber photometry, used to measure neuronal activity in the brain of a free moving subject, with an ErasmusLadder, in which it needs to travel from point A to point B on a horizontal ladder with touch-sensitive rungs that register the type and length of steps. By introducing a sudden obstacle to movement, researchers observed how the subject coped and learned accordingly to avoid this obstacle. By playing a high-pitch tone just before the obstacle was introduced, researchers were able to measure how quickly the subjects were able to anticipate the obstacle and adjust their steps accordingly. Subjects with neonatal brain injury and normal models were run through a series of learning trials while simultaneously monitoring brain activity. In this way, the team was able to quantify cerebellum-dependent locomotor learning and adaptive behavior, unlocking a functional and mechanistic understanding of behavioral pathology that was previously unseen in this field.

In addition to showing that normal Purkinje cells are highly active during movement on the ErasmusLadder, the team explored the question of whether Purkinje cells of injured pre-clinical models were generally non-responsive to any kind of stimuli. They found that while Purkinje cells in injured subjects responded to puffs of air, which generally cue the subject to start moving on the ErasmusLadder, dysfunction in these cells was specific to the period of adaptive learning. Lastly, through chemogenetic inhibition, which specifically silences neonatal Purkinje cell activity, the team was able to mimic the effects of perinatal cerebellar injury, further solidifying the role of these cells in learning deficits.

The study results have implications for clinical practice. As the care of premature babies continues to improve, neonatologists face new challenges to ensure that babies not only survive but thrive. They need to find ways to prevent against the lifelong impacts that preterm birth would otherwise have on the cerebellum and developing brain.

Read the full press release here.

Read the full journal article here.

coronavirus molecules with DNA

Novel SARS-CoV-2 spike variant found in a newborn in Washington, D.C.

coronavirus molecules with DNA

Researchers at Children’s National Hospital found a new SARS-CoV-2 spike variant in a neonatal patient, according to a study that genetically sequenced the virus in 27 pediatric patients. The newborn presented with a viral load of 50,000 times more particles than the average patient, which led to identifying the N679S spike protein variant — the earliest known sample of this coronavirus lineage in the U.S. mid-Atlantic region.

While the paper is posted to the preprint server medRxiv and has not been peer-reviewed, it represents an early step towards establishing better surveillance of the COVID-19 pandemic. The new variant helps understand the process of viral adaptation, potentially informing treatment development and vaccine design for any viral variants in the future.

All genomes change and evolve. Additional viral variants are expected to emerge as more patients are infected. The data analysis recognized eight other cases in Washington, D.C., with the N679S variant, pointing toward a European origin due to the genetic similarity between of SARS-CoV-2 strains in the U.S. and United Kingdom.

“We need to sequence more cases to identify variants and stay ahead of the virus,” said Drew Michael, Ph.D., molecular geneticist at Children’s National and senior author of the study. “The United States sequences a tiny fraction of all cases, and because we are not sequencing enough, we are not aware of the variants in SARS-CoV-2 that may be spreading in our community.”

“Novel SARS-CoV-2 spike variant identified through viral genome sequencing of the pediatric Washington D.C. COVID-19 outbreak,” was published on the preprint server medRxiv. Additional authors include Jonathan LoTempio, Erik Billings, Kyah Draper, Christal Ralph, Mahdi Moshgriz, Nhat Duong, Jennifer Dien Bard, Xiaowu Gai, David Wessel, M.D., Roberta L. DeBiasi, M.D., M.S., Joseph M. Campos, Ph.D., Eric Vilain, M.D., Ph.D. and Meghan Delaney, D.O., M.P.H.

You can read the full preprint on medRxiv.

illustration of brain with stem cells

Innovative phase 1 trial to protect brains of infants with CHD during and after surgery

A novel phase 1 trial looking at how best to optimize brain development of babies with congenital heart disease (CHD) is currently underway at Children’s National Hospital.

Children with CHD sometimes demonstrate delay in the development of cognitive and motor skills. This can be a result of multiple factors including altered prenatal oxygen delivery, brain blood flow and genetic factors associated with surgery including exposure to cardiopulmonary bypass, also known as the heart lung machine.

This phase 1 trial is the first to deliver mesenchymal stromal cells from bone marrow manufactured in a lab (BM-MSC) into infants already undergoing cardiac surgery via cardiopulmonary bypass. The hypothesis is that by directly infusing the MSCs into the blood flow to the brain, more MSCs quickly and efficiently reach the subventricular zone and other areas of the brain that are prone to inflammation. The trial is open to eligible patients ages newborn to six months of age.


Learn more in this overview video.

The trial is part of a $2.5 million, three-year grant from the National Institutes of Health (NIH) led by Richard Jonas, M.D.Catherine Bollard, M.B.Ch.B., M.D., and Nobuyuki Ishibashi, M.D.. The project involves collaboration between the Prenatal Cardiology program of Children’s National Heart Institute, the Center for Cancer and Immunology Research, the Center for Neuroscience Research and the Sheikh Zayed Institute for Pediatric Surgical Innovation.

“NIH supported studies in our laboratory have shown that MSC therapy may be extremely helpful in improving brain development in animal models after cardiac surgery,” says Dr. Ishibashi. “MSC infusion can help reduce inflammation including prolonged microglia activation that can occur during surgery that involves the heart lung machine.”

Staff from the Cellular Therapy Laboratory, led by director Patrick Hanley, Ph.D., manufactured the BM-MSC at the Center for Cancer and Immunology Research, led by Dr. Bollard.

The phase 1 safety study will set the stage for a phase 2 effectiveness trial of this highly innovative MSC treatment aimed at reducing brain damage, minimizing neurodevelopmental disabilities and improving the postoperative course in children with CHD. The resulting improvement in developmental outcome and lessened behavioral impairment will be of enormous benefit to individuals with CHD.

For more information about this new treatment, contact the clinical research team: Gil Wernovsky, M.D., Shriprasad Deshpande, M.D., Maria Fortiz.

MRI of the patient's head close-up

Early versus late MRI in newborn brain injury

MRI of the patient's head close-up

A single magnetic resonance imaging (MRI) performed in the first week after birth is adequate to assess brain injury and offer prognostic information in newborn infants with hypoxic ischemic encephalopathy (HIE) treated with therapeutic hypothermia, according to a new study published in The Journal of Pediatrics.

A collaborative team of neonatology, neurology and neuroradiology experts from Children’s National Hospital that included Gilbert Vezina, M.D., Taeun Chang, M.D., and An N. Massaro, M.D., came together to evaluate the agreement in brain injury findings between early and late MRI in newborn infants with hypoxic ischemic encephalopathy (HIE) treated with therapeutic hypothermia. The team then compared the ability of early versus late MRI to predict early neurodevelopmental outcomes.

This was a prospective longitudinal study of 49 patients with HIE who underwent therapeutic hypothermia and had MRI performed at both <7 and ≥7 days of age. MRIs were reviewed by an experienced neuroradiologist and assigned brain injury severity scores according to established systems. Scores for early and late MRIs were assessed for agreement using the kappa statistic. The ability of early and late MRI scores to predict death or developmental delay at 15-30 months of age was assessed by logistic regression analyses.

The results of the study found agreement between the early and late MRI was substantial to near perfect (k>0.75, p<0.001) across MRI scoring systems. In cases of discrepant scoring, early MRI was more likely to identify severe injury when compared with late MRI. Early MRI scores were more consistently predictive of adverse outcomes compared with late MRI.

Read the full study in The Journal of Pediatrics.

structure of EGFR

Study suggests EGFR inhibition reverses alterations induced by hypoxia

structure of EGFR

The study suggests that specific molecular responses modulated by EGFR (seen here) may be targeted as a therapeutic strategy for HX injury in the neonatal brain.

Hypoxic (HX) encephalopathy is a major cause of death and neurodevelopmental disability in newborns. While it is known that decreased oxygen and energy failure in the brain lead to neuronal cell death, the cellular and molecular mechanisms of HX-induced neuronal and glial cell damage are still largely undefined.

Panagiotis Kratimenos, M.D., and colleagues from the Center for Neuroscience Research at the Children’s National Research Institute, discovered increased expression of activated-epidermal growth factor receptor (EGFR) in affected cortical areas of neonates with HX and decided to further investigate the functional role of EGFR-related signaling pathways in the cellular and molecular changes induced by HX in the cerebral cortex.

The researchers found that HX-induced activation of EGFR and Ca2+/calmodulin kinase IV (CaMKIV) caused cell death and pathological alterations in neurons and glia. EGFR blockade inhibited CaMKIV activation, attenuated neuronal loss, increased oligodendrocyte proliferation and reversed HX-induced astrogliosis.

The researchers also performed, for the first time, high-throughput transcriptomic analysis of the cortex to define molecular responses to HX and to uncover genes specifically involved in EGFR signaling in brain injury. Their results indicate that specific molecular responses modulated by EGFR may be targeted as a therapeutic strategy for HX injury in the neonatal brain.

This study defines many new exciting avenues of scientific exploration to further elucidate the beneficial impact of EGFR blockade on perinatal brain injury at the cellular and molecular levels. This analysis could potentially result in the identification of new therapeutic targets associated with EGFR signaling in the developing mammalian brain that are linked with specific long-term abnormalities caused by perinatal brain injury.

Children’s National researchers who contributed to this study include Panagiotis Kratimenos, M.D., Ioannis Koutroulis, M.D., Ph.D., M.B.A., Susan Knoblach, Ph.D., Payal Banerjee, Surajit Bhattacharya, Ph.D., Maria Almira-Suarez, M.D., and Vittorio Gallo, Ph.D.

Read the full article in iScience.

newborn

Predicting risk for infantile spasms after acute symptomatic neonatal seizures

newborn

Infantile spasms (IS) is a severe epilepsy in early childhood. Early treatment of IS provides the best chance of seizure remission and favorable developmental outcome.

Taeun Chang, M.D., director of the Neonatal Neurology and Neurocritical Care Program at Children’s National Hospital, participated in a study with other national pediatric experts which aimed to develop a prediction rule to accurately predict which neonates with acute symptomatic seizures will develop IS.

The group of researchers found that multiple potential predictors were associated with IS, including Apgar scores, EEG features, seizure characteristics, MRI abnormalities and clinical status at hospital discharge. The final model born from this work included three risk factors: (a) severely abnormal EEG or ≥3 days with seizures recorded on EEG, (b) deep gray or brainstem injury on MRI and (c) abnormal tone on discharge exam.

The significance of these findings is that IS risk after acute symptomatic neonatal seizures can be stratified using commonly available clinical data. No child without risk factors, vs >50% of those with all three factors, developed IS. This risk prediction rule may be valuable for clinical counseling as well as for selecting participants for clinical trials to prevent post‐neonatal epilepsy. This tailored approach may lead to earlier diagnosis and treatment and improve outcomes for a devastating early life epilepsy.

Read the full study in Epilepsia.

Lee Beers

Lee Beers, M.D., F.A.A.P, begins term as AAP president

Lee Beers

“The past year has been a stark reminder about the importance of partnership and working together toward common goals,” says Dr. Beers. “I am humbled and honored to be taking on this role at such a pivotal moment for the future health and safety of not only children, but the community at large.”

Lee Savio Beers, M.D., F.A.A.P., medical director of Community Health and Advocacy at the Child Health Advocacy Institute (CHAI) at Children’s National Hospital, has begun her term as president of the American Academy of Pediatrics (AAP). The AAP is an organization of 67,000 pediatricians committed to the optimal physical, mental and social health and well-being for all children – from infancy to adulthood.

“The past year has been a stark reminder about the importance of partnership and working together toward common goals,” says Dr. Beers. “I am humbled and honored to be taking on this role at such a pivotal moment for the future health and safety of not only children, but the community at large.”

Dr. Beers has pledged to continue AAP’s advocacy and public policy efforts and to further enhance membership diversity and inclusion. Among her signature issues:

  • Partnering with patients, families, communities, mental health providers and pediatricians to co-design systems to bolster children’s resiliency and to alleviate growing pediatric mental health concerns.
  • Continuing to support pediatricians during the COVID-19 pandemic with a focus on education, pediatric practice support, vaccine delivery systems and physician wellness.
  • Implementation of the AAP’s Equity Agenda and Year 1 Equity Workplan.

Dr. Beers is looking forward to continuing her work bringing together the diverse voices of pediatricians, children and families as well as other organizations to support improving the health of all children.

“Dr. Beers has devoted her career to helping children,” says Kurt Newman, M.D., president and chief executive officer of Children’s National. “She has developed a national advocacy platform for children and will be of tremendous service to children within AAP national leadership.”

Read more about Dr. Beer’s career and appointment as president of the AAP.

Associations Between Resting State Functional Connectivity and Behavior in the Fetal Brain

Maternal anxiety affects the fetal brain

Associations Between Resting State Functional Connectivity and Behavior in the Fetal Brain

Anxiety in gestating mothers appears to affect the course of brain development in their fetuses, changing neural connectivity in the womb, a new study suggests.

Anxiety in gestating mothers appears to affect the course of brain development in their fetuses, changing neural connectivity in the womb, a new study by Children’s National Hospital researchers suggests. The findings, published Dec. 7, 2020, in JAMA Network Open, could help explain longstanding links between maternal anxiety and neurodevelopmental disorders in their children and suggests an urgent need for interventions to diagnose and decrease maternal stress.

Researchers have shown that stress, anxiety or depression in pregnant mothers is associated not only with poor obstetric outcomes but also social, emotional and behavioral problems in their children. Although the care environment after birth complicates the search for causes, postnatal imaging showing significant differences in brain anatomy has suggested that these problems may originate during gestation. However, direct evidence for this phenomenon has been lacking, says Catherine Limperopoulos, Ph.D., director of the Developing Brain Institute at Children’s National.

To help determine where these neurological changes might get their start, Dr. Limperopoulos, along with staff scientist Josepheen De Asis-Cruz, M.D., Ph.D., and their Children’s National colleagues used a technique called resting-state functional magnetic resonance imaging (rs-fMRI) to probe developing neural circuitry in fetuses at different stages of development in the late second and third trimester.

The researchers recruited 50 healthy pregnant volunteers from low-risk prenatal clinics in the Washington, D.C. area who were serving as healthy “control” volunteers in a larger study on fetal brain development in complex congenital heart disease. These study participants, spanning between 24 and 39 weeks in their pregnancies, each filled out widely used and validated questionnaires to screen for stress, anxiety and depression. Then, each underwent brain scans of their fetuses that showed connections between discrete areas that form circuits.

After analyzing rs-fMRI results for their fetuses, the researchers found that those with higher scores for either form of anxiety were more likely to carry fetuses with stronger connections between the brainstem and sensorimotor areas, areas important for arousal and sensorimotor skills, than with lower anxiety scores. At the same time, fetuses of pregnant women with higher anxiety were more likely to have weaker connections between the parieto-frontal and occipital association cortices, areas involved in executive and higher cognitive functions.

“These findings are pretty much in keeping with previous studies that show disturbances in connections reported in the years and decades after birth of children born to women with anxiety,” says Dr. De Asis-Cruz. “That suggests a form of altered fetal programming, where brain networks are changed by this elevated anxiety even before babies are born.”

Whether these effects during gestation themselves linger or are influenced by postnatal care is still unclear, adds Dr. Limperopoulos. Further studies will be necessary to follow children with these fetal differences in neural connectivity to determine whether these variations in neural circuitry development can predict future problems. In addition, it’s unknown whether easing maternal stress and anxiety can avoid or reverse these brain differences. Dr. Limperopoulos and her colleagues are currently studying whether interventions that reduce stress could alter the trajectory of fetal neural development.

In the meantime, she says, these findings emphasize the importance of making sure pregnant women have support for mental health issues, which helps ensure current and future health for both mothers and babies.

“Mental health problems remain taboo, especially in the peripartum period where the expectation is that this is a wonderful time in a woman’s life. Many pregnant mothers aren’t getting the support they need,” Dr. Limperopoulos says. “Changes at the systems level will be necessary to chip away at this critical public health problem and make sure that both mothers and babies thrive in the short and long term.”

Other Children’s National researchers who contributed to this study include Dhineshvikram Krishnamurthy, M.S., software engineer; Li Zhao, Ph.D., research faculty; Kushal Kapse, M.S., staff engineer; Gilbert Vezina, M.D., neuroradiologist; Nickie Andescavage, M.D., neonatologist; Jessica Quistorff, M.P.H., clinical research program lead; and Catherine Lopez, M.S., clinical research program coordinator.

This study was funded by R01 HL116585-01 from the National Heart, Lung, and Blood Institute and U54HD090257 from the Intellectual and Developmental Disabilities Research Center.

depressed mom holding baby

New grant to help establish maternal mental health telehealth program

depressed mom holding baby

Children’s National has received a $76,000 grant from the Health Resources & Services Administration (HRSA) which will allow a cross-functional team of neonatologists and psychologists to establish a parental mental telehealth program.

Worldwide about 10% of pregnant women and 13% of women who have just given birth experience a mental health disorder, primarily depression, according to the World Health Organization.

“This is a topic that is quickly garnering attention but remains extremely underfunded,” says Lamia Soghier, M.D., F.A.A.P., C.H.S.E., medical director of the Neonatal Intensive Care Unit (NICU) at Children’s National Hospital. “We tend to focus on the babies but don’t pay enough attention to the parents.”

Dr. Soghier’s focus has been on NICU parents who experience postpartum mood and anxiety disorders (PMADs), often due to their uniquely stressful experiences.

“We have been screening on a small scale for many years and have noticed a 33-45% rate of postpartum depression symptoms in our NICU families,” she says.

Maternal mental disorders are treatable with effective screening and interventions. Children’s National has received a $76,000 grant from the Health Resources & Services Administration (HRSA) which will allow a cross-functional team of neonatologists and psychologists to establish a parental mental telehealth program to expand screening and provide diagnosis, therapy and counseling to NICU parents who experience postpartum mood and anxiety disorders.

Dr. Soghier, along with Ololade ‘Lola’ Okito, M.D., neonatologist at Children’s National, and Erin Sadler, Psy.D., psychologist in the Division of Psychology and Behavioral Health at Children’s National, discuss the importance of this work.

Q: Tell us more about the program you’re establishing.

A: Dr. Soghier: This program will allow us to hire a licensed psychologist who will see families both in the NICU and through follow-up telehealth visits. It provides a one-stop shop for our families, which is particularly important during the COVID-19 pandemic. The grant will also allow us to develop an iPad loaner program to give loaner iPads to low income families who do not have access to a device or to reliable internet services so that they can receive therapy at home.

Dr. Sadler: We’ll be examining how the implementation of these services can increase accessibility and reduce barriers that prevent assessment and initiation of crucial mental health services for at-risk mothers. Our partnerships will be key. Mothers experiencing barriers to participating in care services in the NICU will also have access to an in-house, licensed psychologist through telehealth services within the comfort of their homes. Families experiencing problems accessing telehealth technology due to economic limits would get the loaner iPad. We’re meeting our families where they are in order to provide these critical services.

Q: Why is grant funding to important in this space?

A: Dr. Okito: Access to perinatal mental health services is limited at the local and national levels, particularly for vulnerable parents of infants admitted to the NICU. Little is known about the effect of interventions to address depression and anxiety among NICU parents, and this grant will allow us to contribute to this very important area of research.

Dr. Sadler: It is not enough to recognize the health disparities that exist amongst communities in our nation. It is imperative that we’re able to explore and examine solutions that can aid in enhancing the equity of care for children and adults alike. As Dr. Okito mentions, there is little to no research available that looks at the feasibility of the support programs we intended to put in place. We hope to create a viable model that could be used to help NICU families across the country.

Q: How is Children’s National uniquely positioned to do this work?

A: Dr. Soghier: Healthy moms and healthy dads equal happy babies. That’s why we will be taking care of the family as a whole. This is truly family-centered care and at the heart of what Children’s National is all about.

Dr. Sadler: The Children’s National NICU team has an established postpartum depression screening program. Through the piloted work, staff have identified notable barriers to universal screening, access to perinatal mental health support and the impact of PMADs on parent engagement in newborn care.  As a result, Children’s National is uniquely positioned to directly address such barriers and provide specialized care.

Q: What excites you about this work?

A: Dr. Sadler: As a specialist in perinatal and infant mental health, I look forward to being able to demonstrate the lasting impact maternal mental health services can provide for not only newborns and their families, but for care providers as well. I am excited to have additional opportunities to advocate for the integration of perinatal and infant mental health in non-traditional spaces.

Dr. Okito: I am most excited about the potential to expand universal depression screening among NICU parents. Having done this work for the past three years, I know there are limitations in screening because we’ve only been able to screen parents that are at the patient’s bedside. More screening will lead to more parents getting the referrals and services that they need.

Research & Innovation Campus

Boeing gives $5 million to support Research & Innovation Campus

Research & Innovation Campus

Children’s National Hospital announced a $5 million gift from The Boeing Company that will help drive lifesaving pediatric discoveries at the new Children’s National Research & Innovation Campus.

Children’s National Hospital announced a $5 million gift from The Boeing Company that will help drive lifesaving pediatric discoveries at the new Children’s National Research & Innovation Campus. The campus, now under construction, is being developed on nearly 12 acres of the former Walter Reed Army Medical Center. Children’s National will name the main auditorium in recognition of Boeing’s generosity.

“We are deeply grateful to Boeing for their support and commitment to improving the health and well-being of children in our community and around the globe,” said Kurt Newman, M.D., president and CEO of Children’s National “The Boeing Auditorium will help the Children’s National Research & Innovation campus become the destination for discussion about how to best address the next big healthcare challenges facing children and families.”

The one-of-a-kind pediatric hub will bring together public and private partners for unprecedented collaborations. It will accelerate the translation of breakthroughs into new treatments and technologies to benefit kids everywhere.

“Children’s National Hospital’s enduring mission of positively impacting the lives of our youngest community members is especially important today,” said Boeing President and CEO David Calhoun. “We’re honored to join other national and community partners to advance this work through the establishment of their Research & Innovation Campus.”

Children’s National Research & Innovation Campus partners currently include Johnson & Johnson Innovation – JLABS, Virginia Tech, the National Institutes of Health (NIH), Food & Drug Administration (FDA), U.S. Biomedical Advanced Research and Development Authority (BARDA), Cerner, Amazon Web Services, Microsoft, National Organization of Rare Diseases (NORD) and local government.

The 3,200 square-foot Boeing Auditorium will be the focal point of the state-of-the-art conference center on campus. Nationally renowned experts will convene with scientists, medical leaders and diplomats from around the world to foster collaborations that spur progress and disseminate findings.

Boeing’s $5 million commitment deepens its longstanding partnership with Children’s National. The company has donated nearly $2 million to support pediatric care and research at Children’s National through Chance for Life and the hospital’s annual Children’s Ball. During the coronavirus pandemic, Boeing fabricated and donated 2,000 face shields to help keep patients and frontline care providers at Children’s National safe.

Baby in the NICU

Quality improvement initiative reduces vancomycin use in NICU

Baby in the NICU

A quality improvement initiative in the Neonatal Intensive Care Unit (NICU) at Children’s National Hospital led to a significant reduction in treatment with intravenous vancomycin, an antibiotic used for resistant gram positive infections, which is often associated with acute kidney injury.

A quality improvement initiative in the Neonatal Intensive Care Unit (NICU) at Children’s National Hospital led to a significant reduction in treatment with intravenous vancomycin, an antibiotic used for resistant gram positive infections, which is often associated with acute kidney injury. The findings, published in the journal Pediatrics, show the initiative reduced vancomycin use in patients by 66%, and the NICU has sustained the reduction for more than a year.

Vancomycin is a broad-spectrum antibiotic often used to treat methicillin-resistant Staphylococcus aureus (MRSA) infection. It’s one of the most commonly prescribed antibiotics in NICUs, but its overuse poses an increased risk of morbidity. Benchmarking data showed that in 2017, vancomycin use at Children’s National Hospital was significantly higher than use at peer institutions, suggesting there was likely an opportunity to optimize use of this drug.

The intervention program was led by Rana Hamdy, M.D., M.S.C.E., M.P.H., an infectious diseases specialist at Children’s National, Lamia Soghier, M.D., medical unit director of the Children’s National NICU, and other team members from neonatologyinfectious diseases, pharmacy, nursing and quality improvement. The team accomplished the prescribing reduction by sequentially implementing a four-step approach involving interdisciplinary team building and provider education, pharmacist-initiated 48-hour time-outs, clinical pathway development and prospective audit with feedback.

“Our interdisciplinary quality improvement team was devoted to this project and implemented interventions that, early on, led not only to reduction in vancomycin use, but to better outcomes in our patients with fewer episodes of vancomycin-associated acute kidney injury,” said Dr. Hamdy. “This led to early buy-in from the prescribers, ultimately changing the culture of antibiotic prescribing in the NICU.”

Following the NICU’s intervention program to improve patient safety, vancomycin use in patients decreased from 112 days of therapy per 1,000 patient-days to 38 days of therapy per 1,000 patient-days. During the intervention program, the researchers noted that this was “the first work to show a significant change in vancomycin-associated acute kidney injury in neonates.”

Four key interventions were sequentially implemented to successfully achieve and sustain the reduction in vancomycin use. Intervention 1 was the development of an interdisciplinary and provider education team that addressed institutional antibiotic prescribing practices. Intervention 2, a pharmacist-initiated 48-hour time-out, involved clinical pharmacists identifying patients who have been on antibiotics for ≥ 48 hours and encouraged their providers to either discontinue vancomycin or to switch to a narrow-spectrum antibiotic. Intervention 3 consisted of the development of new clinical pathways including discontinuing vancomycin in infants at low risk for MRSA. Lastly, intervention 4, antimicrobial stewardship program (ASP) prospective audit and feedback, involved an ASP member reviewing all NICU vancomycin orders and issuing appropriate recommendations for NICU providers and pharmacists to be carried out within 24 hours.

This project was taken on as part of Children’s National Quality Improvement and Leadership Training (QuILT) course sponsored by the Quality & Safety Department. This notable work was highlighted in the 2019 annual Quality and Safety report and by the Magnet® program as an exemplary example of nursing-physician partnership working to improve patient care.

The associated article, “Reducing Vancomycin Use in a Level IV Neonatal Intensive Care Unit,” will be published July 1 in Pediatrics. The lead author is Dr. Rana Hamdy, an infectious diseases specialist and director of the Antimicrobial Stewardship Program. Twenty notable co-authors are also from Children’s National.

Annual Pediatric Device Innovation Symposium panelists

Accelerating pediatric device innovation through legislative processes and industry changes


Annual Pediatric Device Innovation Symposium panelists
While the way we deliver healthcare is changing rapidly, far too often the tools we use to treat children are stuck in the past.

Over the last decade, pediatric medical device innovation, particularly for the youngest, most fragile children, has made dismal progress. Of the Class 3 (high risk/high benefit) medical devices approved by FDA for pediatrics in the last 10 years, less than 4% are for ages 0-2 years old; and even less for neo-natal patients. Simply put, as medical devices advance, children are not seeing the benefit of innovation.

The 8th Annual Pediatric Device Innovation Symposium presented by Children’s National Hospital in conjunction with the National Capital Consortium for Pediatric Medical Devices (NCC-PDI) featured a keynote panel, “Pediatric Device Innovation: What’s Next?”, to examine the legislative and industry changes needed to speed up device innovation for kids.

One of the keynote panelists, and leading voices on this issue, is Children’s National Hospital president and CEO Kurt Newman, M.D. Dr. Newman, a former pediatric surgeon, knows firsthand that every day in our nation’s pediatric hospitals, surgeons are manipulating adult medical devices to create creative solutions for children’s bodies because it’s the only available option.

“Children need and deserve devices that are conceived and designed with their biology and future in mind,” says Dr. Newman. “While children may only make up a small percentage of our population – maybe 20 or 25% – they are 100% of our future.”

Dr. Kurt Newman in front of the capitol building

“Children need and deserve devices that are conceived and designed with their biology and future in mind,” says Children’s National Hospital president and CEO Kurt Newman, M.D. “While children may only make up a small percentage of our population – maybe 20 or 25% – they are 100% of our future.”

Dr. Newman also addressed the current barriers to pediatric device innovation, which ranges from limited pediatric clinical trials to a market size that’s not financially appealing.

“The truth is, the frontiers of pediatric medicine are really in the innovative treatments, devices, therapies, and cures awaiting us on the other side of research and development.,” says Dr. Newman.

Former CNN correspondent, Jeanne Meserve, moderated the 45-minute keynote panel discussion, asking questions about the challenges to pediatric innovation, what policy changes need to take place to see improvement in the field of pediatric device innovation, and how federal funding can assist in creating change.

Michelle McMurry-Heath, new CEO of DC-based Biotechnology Innovation Organization (BIO), who joined Dr. Newman on this keynote panel, agreed that more needs to be done in the pediatric space. Dr. McMurry-Heath believes the Food and Drug Administration (FDA) is a public health advocate at heart and that the Pediatric Device Consortia (PDC), which Children’s National Hospital is part of, is starting to make new advancements in pediatric innovation by giving FDA clearance to more start-up companies than we’ve seen in the past.

“The FDA is interested in is what improves the health outcomes for the people and innovation is a huge piece. This is an important part of their mission and it is starting to yield benefits,” says Dr. Michelle McMurry-Heath. “Innovation is a team sport – it’s not easy. It takes a village of expertise and collaboration to progress and projects like the Pediatric Device Consortia is an important piece in this puzzle.”

NCC-PDI is one of five consortia in the FDA’s Pediatric Device Consortia (PDC) Grant Program created to support the development and commercialization of medical devices for children and is led by the Sheikh Zayed Institute for Pediatric Surgical Innovation at Children’s National Hospital and the A. James Clark School of Engineering at the University of Maryland, with support from partners MedTech Innovator, BioHealth Innovation and design firm Archimedic.

To date, NCC-PDI has mentored over 100 medical device sponsors to help advance their pediatric innovations, with seven devices having received either their FDA market clearance or CE marking.

Dr. McMurry-Heath also addressed the challenge of diseases that don’t exist in adults and posed the question, “How do you create a device for kids if it doesn’t exist in adults?” She cited the lack of market in pediatrics and the difficulty in bringing a device to market as problems that hinder innovation, which is why advocating for these devices is crucial to children’s healthcare everywhere.

“So much of our innovation comes from our small, innovative companies,” say Dr. McMurry-Heath. “For example, my company is working on a COVID-19 tracker now and 70% of the innovation is coming from our smallest biotech companies. It’s a race against time for these companies to bring their innovation to market in order to keep the lights on and pay their scientists; this dog-eat-dog world isn’t immediately obvious to outsiders.

Beyond advocating, Dr. Newman and Children’s National are developing the first-of-its-kind pediatric research and innovation campus, which is currently under construction at DC’s former Walter Reed Army Medical Center site.

“We secured 12 acres to create something that has never been done before and that’s a campus for innovation dedicated to children,” says Dr. Newman. “Our close proximity to federal research institutions and agencies enables the new Children’s National campus to leverage the rich ecosystem of public and private sectors to help bolster biohealth, medical device, and life science innovation.”

As Children’s National continues to champion ways to accelerate pediatric device development, one focus is the on-site incubator Johnson & Johnson Innovation – JLABS, which will help start-up companies strengthen their ideas by working with coaches, having access to mentors and learning how to interact with the FDA. This partnership also offers an audience for their device which could potentially lead to investments.

The Children’s National Research & Innovation Campus will create an ecosystem that can accelerate breakthroughs in pediatric healthcare discoveries and technologies: The new campus is currently under construction and expected to open in the first quarter of 2021.

Both panelists agreed they’d like to see more flexibility with regulators to work with innovators in order create more incentives for them to present their device, like the NCC-PDI “Make Your Medical Device Pitch for Kids!” Competition, which was recently held in September 2020. The six winners received up to $50,000 in FDA-funded grant awards in order to develop their device, eventually bring it to market in order to improve healthcare for kids.

NCC-PDI device competition

Medical device competition announces six winners to share in $250K

Judges award grants for pediatric medical devices that address cardiovascular, NICU, and orthopaedic and spine device innovations.