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Participants at the annual “Make Your Medical Device Pitch for Kids!”™ competition

Two pediatric medical device companies awarded at pitch competition

Participants at the annual “Make Your Medical Device Pitch for Kids!”™ competition

Winners and finalists competed at UCLA during annual L.A. MedTech Week 2024. Left to right are: Nada Hanafi, MedTech Color Board treasurer; Vernessa Pollard, MedTech Color Board secretary; Dr. Sanna Gaspard, CEO and founder of Rubitection, (winner -$35,000 first prize); Dr. Kolaleh Eskandanian, Children’s National vice president and chief innovation officer; Kwame Ulmer, MedTech Color founder and board chair. Photo credit: MedTech Color

Alliance for Pediatric Device Innovation (APDI), a federally funded consortium led by Children’s National Hospital, and member MedTech Color announced the winners of the recent edition of the Make Your Medical Device Pitch for Kids!competition, which focused on recognizing and supporting African American and Hispanic pediatric medical device innovators. The awardees received a combined $50,000 in grant funding from APDI, made possible by the U.S. Food and Drug Administration (FDA) to support the advancement of pediatric medical technologies to the market.

The winners were selected from a field of five finalists who gave pitch presentations on their innovation’s attributes, benefits to patients and pathway to commercialization before a panel of five expert judges. The event was held on March 15 at the University of California, Los Angeles, as part of the annual MedTech Color Pitch Competition during L.A. MedTech Week 2024, powered by BioscienceLA.

The award-winning pediatric devices and companies are:

  • Rubitection, Pittsburgh, Pa.A low-cost skin assessment management tool for chronic wounds, Rubitection measures the properties of the skin. The system allows the user to monitor incremental changes in skin health to predict risk, monitor progression and customize care.
  • Kofimi Technology Inc., Danvers, Mass. This patent-pending pulse oximeter device is designed specifically for pediatric populations to provide superior accuracy for all levels of skin pigmentation. A pulse oximeter measures oxygen levels in the blood.

Why we’re excited

Funding innovators of African American and Hispanic backgrounds is crucial for advancing diversity, equity, and inclusion initiatives because it addresses systemic barriers and inequities that have historically hindered this group from accessing resources and opportunities in the life sciences sector,” said Kolaleh Eskandanian, Ph.D., M.B.A., P.M.P., vice president and chief innovation officer at Children’s National and APDI program director and principal investigator. “Supporting a diverse group of innovators enriches the research and development process by bringing a variety of perspectives and viewpoints representing all populations.”

Along with the grant award, all finalists receive access to a network of supportive resources and expertise as part of their connection to APDI and MedTech Color.

Along with the pitch presentations, the event program included a keynote talk from Tonya Kinlow, vice president of Community Engagement, Advocacy and Government Affairs at Children’s National, highlighting advocacy initiatives leading to an all-inclusive system of care for children.

Children’s National leads the way

Julia Finkel, M.D., pediatric anesthesiologist at Children’s National and principal investigator for APDI, participated in the panel discussion “Inequity within Inequity,” which highlighted how racial disparities in pediatric healthcare remain a critical challenge across the United States. APDI’s goal is to advance pediatric devices that meet patient needs while promoting a more inclusive approach to discovering and supporting talented innovators.

“We believe there is a community of companies focused on platforms that treat or diagnose pediatrics,” said Kwame Ulmer, founder of MedTech Color. “This partnership allows us to significantly increase the support we provide for entrepreneurs in this area. We are delighted to be a part of the Children’s National community to drive positive patient outcomes.”

The patient benefit

Founded in 2017, MedTech Color is a nonprofit organization built on the same ideal: diverse leadership in the medical technology ecosystem leads to greater innovation and better outcomes. The organization works to advance the representation of people of color in the medical device industry and to nurture the next generation of founders. For more information on MedTech Color, visit medtechcolor.org.

APDI is one of five nonprofit consortia in the FDA’s Pediatric Device Consortia program that receives funding to provide a platform of services, expertise and grants to support pediatric innovators in bringing medical devices to the market that address the needs of children. Along with Children’s National, APDI members include Johns Hopkins University, CIMIT at Mass General Brigham, Tufts Medical Center, Medstar Health Research Institute, OrthoPediatrics Corp. and MedTech Color.

Advancements in pediatric medical devices continue to lag significantly behind those of adults, which is why APDI is focused on helping more pediatric medical device innovations achieve commercialization. For more information on APDI, visit innovate4kids.org

Nathan Kuppermann, M.D., M.P.H.

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

Nathan Kuppermann, M.D., M.P.H.

Dr. Kuppermann will oversee research, education and innovation for the Children’s National Research Institute as well as academic and administrative leadership in the Department of Pediatrics at George Washington University School of Medicine & Health Services.

Children’s National Hospital has appointed Nathan Kuppermann, M.D., M.P.H., as its new executive vice president (EVP), chief academic officer (CAO) and chair of Pediatrics. In this role, Dr. Kuppermann will oversee research, education and innovation for the Children’s National Research Institute as well as academic and administrative leadership in the Department of Pediatrics at George Washington University School of Medicine & Health Services. He comes to Children’s National from UC Davis Health and UC Davis School of Medicine in Sacramento, CA, and will start in September.

After a national search, Dr. Kuppermann stood out for his exceptional contributions to clinical and academic research, focusing on pediatric emergency care, and his dedication to mentorship. For the past 18 years he has served as the Bo Tomas Brofeldt endowed chair of the Department of Emergency Medicine and is currently a distinguished professor of Emergency Medicine and Pediatrics, and the associate dean for Global Health at UC Davis Health.

“I was drawn to Children’s National by its nationally recognized work and dedication to innovation and team science,” says Dr. Kuppermann. “I’m eager to contribute to the remarkable work being done in both the research and education space to continue to improve the understanding, prevention and treatment of childhood diseases.”

Dr. Kuppermann is a pediatric emergency medicine physician and clinical epidemiologist, and a leader in emergency medical services for children, particularly in multicenter research. With more than 300 peer-reviewed research publications to his credit, Dr. Kuppermann has contributed extensively to high-impact journals including the New England Journal of Medicine, JAMA, BMJ and the Lancet.

“The Children’s National Research Institute is a key part of our health system’s ecosystem – it’s where we nurture innovation and pursue the most promising research,” says Michelle Riley-Brown, MHA, FACHE, president and CEO of Children’s National. “Dr. Kuppermann’s unwavering commitment to excellence in pediatric healthcare, research and innovation set him apart in a competitive field. I am confident he will advance our efforts in making breakthrough discoveries for kids everywhere.”

Dr. Kuppermann received his undergraduate degree from Stanford University, his medical degree from UC San Francisco School of Medicine and his Master of Public Health degree from the Harvard School of Public Health. He completed a pediatrics residency and chief residency at Harbor-UCLA Medical Center and a fellowship in Pediatric Emergency Medicine at Boston Children’s Hospital.

He has been recognized nationally and internationally for his research and mentorship. He was a Fulbright Distinguished Scholar in the U.K. and in 2010 was elected to the National Academy of Medicine. In 2022, he received the Maureen Andrew Mentor Award from the Society for Pediatric Research.

“Dr. Kuppermann’s leadership will undoubtedly propel the hospital’s efforts in advancing pediatric healthcare innovation, reinforcing Children’s National as a top-ranking institution,” says Horacio Rozanski, chair of the Children’s National Board of Directors. “We look forward to the positive impact he will make to the hospital’s overall mission, as well as its research and academic endeavors.”

Monika Goyal, M.D., MSCE

Q&A with Dr. Goyal: Trailblazing equity work leads to election to ASCI

Monika Goyal, M.D., MSCE, associate division chief of Emergency Medicine and Trauma Services

Monika Goyal, M.D., MSCE, associate division chief of Emergency Medicine and Trauma Services, is joining The American Society for Clinical Investigation (ASCI), one of a small cadre of pediatric emergency medicine physicians elected to the premier medical honor society for physician-scientists. Dr. Goyal considers her new honor a pivotal opportunity to represent pediatric emergency medicine, which is often underrepresented in research.

Interim Chief Academic Officer Catherine Bollard, M.D., M.B.Ch.B., nominated Dr. Goyal to ASCI because of her nationally renowned work as an equity science scholar, having published more than 130 peer-reviewed manuscripts and securing more than $25 million in funding from the National Institutes of Health (NIH). “From the very beginning of her career, Dr. Goyal has been a trailblazer as it relates to her research interests,” said Dr. Bollard, also an ASCI member.

Dr. Goyal serves as the inaugural chair for Women in Science and Medicine and associate division chief for Academic Affairs and Research at Children’s National Hospital. Her academic work has focused on disparities in the emergency setting, where she has studied the impacts of gun violence, pain management, sexual health and more. In 2023, the journal Pediatrics named a Children’s National manuscript on gun violence one of the 12 most significant papers in its 75-year history. Dr. Goyal was the first author and remains tremendously proud of the impact science can make on society.

Q: How did you find yourself investigating the science of gun laws?

A: A half-dozen years ago, three other pediatric emergency medicine physicians and I had been caring for countless numbers of children who had been victims of gun violence and felt motivated to act. I started SAFER at Children’s National – Safer through Advocacy, Firearm, Education and Research – which is now an institution-wide initiative to address gun violence within our community and beyond. More than 50 individuals at Children’s National are now active in our organization.

We’ve been able to publish a lot of research in this area, including our national study looking at the association between the strictness of gun laws with firearm-related deaths in children. We found that children are more likely to die from gun violence in states with less strict gun laws. It’s not surprising, yet the recognition by the American Academy of Pediatrics demonstrates the importance of using science to understand this, and it shows how far we’ve come in the medical community. Until recently, this issue has been under-recognized and under-supported, despite it being a massive public health crisis for our children and our country.

Q: Given the pace of emergency medicine, how did you find your way to research?

A: Early in my career, much of my work focused on adolescent sexual health. I kept finding that there was racial bias in terms of who we considered to be at risk for sexually transmitted infections. This finding then motivated me to investigate and understand whether racial bias impacted other aspects of care delivery across various clinical conditions in the Emergency Department.

I started by looking at whether differences existed in pain management based on a patient’s race. In evaluating data on children nationwide who are diagnosed with appendicitis, we found that Black children are less likely to receive appropriate pain management compared to white children, even after we adjusted for pain score and illness severity. We have found similar themes with respect to pain management among children diagnosed with fractures.

Q: What can be done?

A: We are grateful to have been awarded additional NIH funding to develop and test interventions to mitigate inequities. Currently, we are studying the impact of audit and feedback through the provision of what we are calling ‘equity report cards’ and clinical decision support embedded in the electronic health record to improve racial, ethnic and language equity in pain management.

Hundreds of studies have demonstrated that this is an issue at hospitals across the country. I am proud of Children’s National for having the humility for this self-reflection and the courage to do something about it. Our work here is helping to inform efforts across the country, and I am proud of our institution’s leadership in advancing health equity through community-informed, evidence-based interventions.

Newborn baby in a crib

Pioneering research center aims to revolutionize prenatal and neonatal health

Catherine Limperopoulos, Ph.D., was drawn to understanding the developing brain, examining how early adverse environments for a mother can impact the baby at birth and extend throughout its entire lifetime. She has widened her lens – and expanded her team – to create the new Center for Prenatal, Neonatal & Maternal Health Research at Children’s National Hospital.

“Despite the obvious connection between mothers and babies, we know that conventional medicine often addresses the two beings separately. We want to change that,” said Dr. Limperopoulos, who also directs the Developing Brain Institute. “Given the current trajectory of medicine toward precision care and advanced imaging, we thought this was the right moment to channel our talent and resources into understanding this delicate and highly dynamic relationship.”

Moving the field forward

Since its establishment in July 2023, the new research center has gained recognition through high-impact scientific publications, featuring noteworthy studies exploring the early phases of human development.

Dr. Limperopoulos has been at the forefront of groundbreaking research, directing attention to the consequences of maternal stress on the unborn baby and the placenta. In addition, under the guidance of Kevin Cook, Ph.D., investigators published a pivotal study on the correlation between pain experienced by premature infants in the Neonatal Intensive Care Unit and the associated risks of autism and developmental delays.

Another area of research has focused on understanding the impact of congenital heart disease (CHD) on prenatal brain development, given the altered blood flow to the brain caused by these conditions during this period of rapid development. Led by Josepheen De Asis-Cruz, M.D., Ph.D., a research team uncovered variations in the functional connectivity of the brains of infants with CHD. In parallel, Nickie Andescavage, M.D., and her team employed advanced imaging techniques to identify potential biomarkers in infants with CHD, holding promise for guiding improved diagnostics and postnatal care. Separately, she is investigating the impact of COVID-19 on fetal brain development.

In the months ahead, the team plans to concentrate its efforts on these areas and several others, including the impact of infectious disease, social determinants of health and protecting developing brains from the negative impacts of maternal stress, pre-eclampsia and other conditions prevalent among expectant mothers.

Assembling a team

Given its robust research plan and opportunities for collaboration, the center pulled together expertise from across the hospital’s faculty and has attracted new talent from across the country, including several prominent faculty members:

  • Daniel Licht, M.D., has joined Children’s National to build a noninvasive optical device research group to better care for children with CHD. Dr. Licht brings decades of experience in pediatric neurology, psychiatry and critical care and is recognized internationally for his expertise in neurodevelopmental outcomes in babies with CHD.
  • Katherine L. Wisner, M.S., M.D., has accumulated extensive knowledge on the impact of maternal stress on babies throughout her career, and her deep background in psychiatry made her a natural addition to the center. While Dr. Wisner conducts research into the urgent need to prioritize maternal mental health, she will also be treating mothers as part of the DC Mother-Baby Wellness Initiative — a novel program based at Children’s National that allows mothers to more seamlessly get care for themselves and participate in mother-infant play groups timed to align with their clinical appointments.
  • Catherine J. Stoodley, B.S., M.S., D.Phil., brings extensive research into the role of the cerebellum in cognitive development. Dr. Stoodley uses clinical studies, neuroimaging, neuromodulation and behavioral testing to investigate the functional anatomy of the part of the brain responsible for cognition.
  • Katherine M. Ottolini, M.D., attending neonatologist, is developing NICU THRIVE – a research program studying the effects of tailored nutrition on the developing newborn brain, including the impact of fortifying human milk with protein, fat and carbohydrates. With a grant from the Gerber Foundation, Dr. Ottolini is working to understand how personalized fortification for high-risk babies could help them grow.

Early accolades

The new center brings together award-winning talent. This includes Yao Wu, Ph.D., who recently earned the American Heart Association’s Outstanding Research in Pediatric Cardiology award for her groundbreaking work in CHD, particularly for her research on the role of altered placental function and neurodevelopmental outcomes in toddlers with CHD. Dr. Wu became the third Children’s National faculty member to earn the distinction, joining an honor roll that includes Dr. Limperopoulos and David Wessel, M.D., executive vice president and chief medical officer.

Interim Chief Academic Officer Catherine Bollard, M.D., M.B.Ch.B., said the cross-disciplinary collaboration now underway at the new center has the potential to make a dramatic impact on the field of neonatology and early child development. “This group epitomizes the Team Science approach that we work tirelessly to foster at Children’s National,” Dr. Bollard said. “Given their energetic start, we know these scientists and physicians are poised to tackle some of the toughest questions in maternal-fetal medicine and beyond, which will improve outcomes for our most fragile patients.”

Before and after pictures of the patient's improved gait

Next-generation genomics testing holds key to undiagnosed rare disease

Before and after pictures of the patient's improved gaitSeth Berger, M.D., Ph.D., felt the pull to dig deeper when he started reading the chart. An 11-year-old boy had an abnormal gait and couldn’t even walk in a straight line down the sidewalk to go trick-or-treating. Yet workups with neurology, orthopedics and an exome analysis of the patient’s genetic code did not provide a diagnosis. He had been getting worse for roughly three years.

With one of the largest clinical genetics departments in the country, Children’s National Hospital receives more than 10,000 visits a year from patients like this middle schooler. Often, they are children and caregivers who are searching for answers and follow-up support for diagnoses of genetic disorders, which impact so few people that only highly trained geneticists and genetic counselors can get to the root of the disorder.

“In genetics, we are finding layers of understanding. A negative clinical test is not always the final answer because the significance of variants can often be missed or misunderstood,” said Dr. Berger, a medical geneticist and principal investigator in the Center for Genetics Medicine Research at Children’s National. “It can take extensive research and a deep knowledge of the limits of certain tests to reach a diagnosis.”

The fine print

On page 4 of the patient’s genetics report, Dr. Berger found a reference to a pair of variants with no known clinical impact. Dr. Berger recognized that the genes referenced could affect proteins that drive potentially treatable neurological outcomes.

Dr. Berger ordered further testing, including biochemical testing of the patient’s blood and a phenylalanine loading challenge, a test that measures how the body metabolizes certain amino acids. With the results, he confirmed a recessive GCH1 deficiency in the patient was causing a condition called DOPA-responsive dystonia, a disorder that causes involuntary muscle contractions, tremors and uncontrolled movements. Laura Schiffman Tochen, M.D., director of the Movement Disorders Program at Children’s National, started the patient on levodopa-carbidopa — a drug combination used to treat Parkinson’s disease and other neurological disorders — and within two hours the boy showed improvement. His gait was almost normal.

Why we’re excited

Dr. Berger presents at conferences on this case and several other medical mysteries that he’s recently solved in his clinical practice and his role at the Pediatric Mendelian Genomics Research Center, a Children’s National program immersed in a federally funded research study to better understand how differences in genetic material can affect human health. As part of his work, he’s joined the GREGoR project (Genomic Research to Elucidate the Genetics of Rare Disease), which hopes to increase the number of genetic disorders where a cause can be identified. The elite genetics consortium includes nationally recognized research centers – the University of California at Irvine, Broad Institute, University of Washington, Baylor University, Stanford University, Invitae and Children’s National – which are working together to harness cutting-edge genomics sequencing capabilities. They hope to enroll thousands in their research, funded by the National Institutes of Health.

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

pregnant woman talking to doctor

Prenatal COVID exposure associated with changes in newborn brain

pregnant woman talking to doctor

The team found differences in the brains of both infants whose mothers were infected with COVID while pregnant, as well as those born to mothers who did not test positive for the virus.

Babies born during the COVID-19 pandemic have differences in the size of certain structures in the brain, compared to infants born before the pandemic, according to a new study led by researchers at Children’s National Hospital.

The team found differences in the brains of both infants whose mothers were infected with COVID while pregnant, as well as those born to mothers who did not test positive for the virus, according to the study published in Cerebral Cortex.

The findings suggest that exposure to the coronavirus and being pregnant during the pandemic could play a role in shaping infant brain development, said Nickie Andescavage, M.D., the first author of the paper and associate chief for the Developing Brain Institute at Children’s National.

The fine print

The study’s authors looked at three groups of infants: 108 born before the pandemic; 47 exposed to COVID before birth; and 55 unexposed infants. In all cases, researchers performed magnetic resonance imaging (MRI) scans of the newborns’ brains during the first few weeks of life. The MRI scans, which are non-invasive and do not expose patients to radiation, provided 3D images of the brain, allowing doctors to calculate the volume of different areas.

Researchers found several differences in the brains of babies exposed to COVID. They had larger volumes of the gray matter that makes up the brain’s outermost layer, compared to the two other groups. In contrast, an inner area of the brain, known as deep gray matter, was smaller than in unexposed babies. These are areas that contain large numbers of neurons that generate and process signals throughout the brain. “Their brains formed differently if they were exposed to COVID,” said Dr. Andescavage, adding that “those exposed to COVID had unique signatures” in the brain.

Doctors also measured the depths of the folds in the babies’ brains – a way to determine how the brain is maturing during early development. Babies born to mothers who had COVID in pregnancy had deeper grooves in the frontal lobe, while babies born during the pandemic – even without being exposed to COVID – had increased folds and grooves throughout the brain, compared to babies born before the pandemic. “There was something about being born during the pandemic that changed how the brain developed,” Dr. Andescavage said.

What’s ahead

The study authors can’t fully explain what caused the differences in brain development in these babies, Dr. Andescavage said. But other studies have linked maternal stress and depression to changes in the newborn brain. In a future study, Dr. Andescavage and her colleagues will examine the relationship between infant brain development and how stress and anxiety during the pandemic may have played a role in early development.

Because the babies in the study were just a few weeks old, researchers don’t know if their altered brain development will affect how they learn or behave. Researchers plan to follow the children until age 6, allowing them to observe whether pandemic-era babies hit key developmental milestones on time, such as walking, talking, holding a crayon and learning the alphabet.

Researchers have been worried about the effect of COVID on the fetus since the beginning of the pandemic. Studies show that babies exposed to COVID in the womb may experience developmental impacts, and research is underway to better understand long-term outcomes.

Although the coronavirus rarely crosses the placenta to infect the fetus directly, there are other ways maternal infection can influence the developing baby. Dr. Andescavage said inflammation is one potential harm to a developing baby. In addition, if a pregnant woman becomes so sick that the levels of oxygen in her blood fall significantly, that can deprive the fetus of oxygen, she added.

In recent decades, studies of large populations have found that maternal infections with influenza and other viruses increased the risk of serious problems in children even years later, including autism, attention deficit hyperactivity disorder and schizophrenia, although the reasons behind the association are not well understood. Technology may allow doctors to answer a number of questions about COVID and the infant brain.

“With advanced imaging and MRI, we’re in a position now to be able to understand how the babies are developing in ways we never previously could,” Dr. Andescavage said. “That will better allow us to identify the exposures that may be harmful, and at what times babies may be especially vulnerable, to better position us to promote maternal wellness. This, in turn, helps infant wellness.”

mother kissing newborn baby

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

mother kissing newborn baby

More than 80% of maternal deaths in the United States are preventable, particularly the nearly 1 in 4 maternal fatalities that are attributable to mental health disorders.

Painting a sobering picture, a research team led by Children’s National Hospital culled years of data demonstrating that maternal mental illness is an under-recognized contributor to the death of new mothers. They are calling for urgent action to address this public health crisis in the latest edition of JAMA Psychiatry.

Backed by dozens of peer-reviewed studies and health policy sources, the journal’s special communication comes as maternal mortality soars in the United States to as much as three times the rate of other high-income countries.

“The contribution of mental health conditions to the maternal morbidity and mortality crisis that we have in America is not widely recognized,” said Katherine L. Wisner, M.D., associate chief of Perinatal Mental Health and member of the Center for Prenatal, Neonatal & Maternal Health Research at Children’s National. “We need to bring this to the attention of the public and policymakers to demand action to address the mental health crisis that is contributing to the demise of mothers in America.”

The evidence review laid out the risks facing new mothers: More than 80% of maternal deaths in the United States are preventable, particularly the nearly 1 in 4 maternal fatalities that are attributable to mental health disorders. Overdose and other maternal mental health conditions are taking the lives of more than twice as many women as postpartum hemorrhage, the second leading cause of maternal death. For non-Hispanic Black mothers, the mortality rate is a striking 2.6 times higher than non-Hispanic White mothers.

Yet the research team found that recent national efforts to combat maternal mortality have failed to address maternal mental health as “the public health crisis that it represents.” Even methodologies to measure maternal health statistics are inconsistent, which challenges efforts to shape health policy.

In examining 30 recent studies and another 15 historical references, the team – which included Caitlin Murphy, MPA, PNP, research scientist at the Milken School of Public Health at George Washington University, and Megan Thomas, M.D., FACOG, obstetrician at the University of Kansas School of Medicine – found ample data to support the need to elevate maternal mental health as a priority. Some examples:

  • Multiple studies show that the perinatal period puts women at higher risk for new and recurrent psychiatric disorders, with 14.5% of pregnant mothers having a new episode of depression and another 14.5% developing an episode three months after birth.
  • Nationwide, more than 400 maternity healthcare centers closed between 2006 and 2020, creating “maternity care deserts” that left nearly 6 million women with limited or no access to maternity care.
  • Mental health conditions such as suicide or opioid overdose are to blame for nearly 23% of maternal deaths in America, according to reports from three dozen Maternal Morbidity and Mortality Review Committees, which are state-based organizations that review each maternal death within a year of pregnancy. That’s followed by hemorrhage (13.7%), cardiac conditions (12.8%) and infection (9.2%).

Even with these sobering statistics, Dr. Wisner says that only 20 percent of women are screened for depression postpartum. “Given that this is a time that many mothers have contact with healthcare professionals, it’s critically important that all mothers are screened and offered treatment,” she said. “Mental health is fundamental to health — of the mother, the child and the entire family.”

Dr. Wisner is board-certified in general and child psychiatry. Throughout her research career, she has conducted research on maternal-infant interactions and family health. She recently joined the new Center for Prenatal, Neonatal & Maternal Health Research because of its vision to improve outcomes for the entire family by understanding the relationship between mothers and their babies.

“Throughout my career, I have fought hard against these silos that try to lock psychiatry into certain age categories,” Dr. Wisner said. “At Children’s National, we have a huge interest in reunifying the family. We want to ensure that we’re caring for unborn babies, infants and toddlers, while focusing on maternal health and the family in its broader context.”

Daniel J. Licht, M.D.

Q&A with Daniel J. Licht, M.D.: The future of medicine is in light

Daniel J. Licht, M.D.

A pediatric neurologist who specializes in children with congenital heart disease, Dr. Licht initially came to this area of research as he considered ways to ensure children’s brains have adequate oxygen delivery during heart care, preserving neurological health and improving long-term outcomes.

Daniel J. Licht, M.D., joins Children’s National Hospital with a vision: He believes non-invasive devices built using biomedical optics – or instruments using light – can give clinicians invaluable information about how the brain and other organs are functioning.

A pediatric neurologist who specializes in children with congenital heart disease, Dr. Licht initially came to this area of research as he considered ways to ensure children’s brains have adequate oxygen delivery during heart care, preserving neurological health and improving long-term outcomes. He sees countless applications for using the properties of light in pediatric medicine.

Dr. Licht, whose name coincidentally also means “light” in German, is planning to establish a program for biomedical optics at Children’s National, built on the pillars of education, innovation and commercialization. He wants to tap into the resources of the Sheik Zayed Institute of Pediatric Surgical Innovation and expertise across the hospital. He is launching this effort as part of the new Center for Prenatal, Neonatal & Maternal Health Research.

Q: How can light be used diagnostically?

A: I believe that light is truly the future of biomedical devices, especially in children. Light can penetrate human tissues deeply, whether it’s muscle, liver or kidney. For example, you can put a light at the end of an endoscope and someday do virtual biopsies. It’s all a matter of understanding the properties of light, and how to manipulate light to give you the answers that you need. The applications are truly infinite.

Q: What has your initial work in neurology shown?

A: One of the instruments that we have developed can measure cerebral blood flow and quantitatively show the oxygen use of the brain. That’s important because it’s easy to measure oxygen delivery, but it’s hard to balance supply-and-demand without knowing the patient’s unique demand. We now have preclinical data and information from about 500 patients.

In terms of what’s ahead, many therapies today aren’t targeted to the individual, so Johnny’s brain-oxygen demand may not be the same as Sarah’s brain-oxygen demand, even if they both have congenital heart disease. As a patient waits for surgery, we also have found that the brain-oxygen demand increases, but if the demand is not met, this can lead to pre-operative brain injury. This technology could change the whole conversation about the timing of surgery. In addition, we can measure the brain-oxygen demand intraoperatively. We are finding that we can actually define the right perfusion strategy for each patient, rather than making uniform decisions for all patients with a shared diagnosis.

Lastly, beyond the operating room, we can use this technology for countless conditions. It would help with the treatment of almost any disease in the critical care unit, when we are using tools like ECMO (extracorporeal membrane oxygenation, a salvage technique), and we need to monitor a patient’s status. We can also use it to measure intracranial pressure. In very simple terms, if a child with a shunt comes into the emergency room with a headache, we can noninvasively measure the pressure and see how it’s changed without a head CT. We can decide who needs to go to the operating room – and who doesn’t – without radiation.

Q: How did your career bring you to this point?

A: My interest has always been in brain injury and kids with congenital heart disease. Years ago, I started out using MRI because it was the technology that was bright and shiny at the time. I was part of a team that developed an MRI sequence for measuring cerebral blood flow. We made some discoveries that indicated the culprit for brain injury was not the surgeries. Instead, there was something with the babies.

Unfortunately, with MRI, it’s a big, expensive instrument, and you have to take the baby to the machine for a single point-in-time measurement. So I started working with a physicist at the University of Pennsylvania to develop a way to measure the motion of particles, specifically red blood cells, to study cerebral blood flow. We found ways to use light, and this is what I hope to build and commercialize at Children’s National. By the end of my career, I hope to be able to say that we got this into clinical care.

Boy lying in a hospital bed, surrounded by medical equipment

Black, Hispanic children at greater risk for complications during hospitalization

Boy lying in a hospital bed, surrounded by medical equipment

The research team found that patients who are Black and Medicaid-insured patients experienced the greatest disparities in postoperative sepsis, a rare complication in which patients suffer from infection that can cause multi-organ failure.

Evaluating more than 5 million pediatric hospital stays nationwide, researchers found children who are Black, Hispanic or insured with Medicaid face a greater risk of health events after surgeries than white patients, according to a new study published in the journal Pediatrics.

“We looked at the data, and we calculated the risks,” said Kavita Parikh, M.D., MSHS, medical director of Quality & Safety Research, research director of the Division of Hospital Medicine and first author on the multi-institute study. “Despite decades of focus on eliminating medical errors, we know that children continue to suffer substantial harms in hospital settings, and our study highlights where children who are Black, Hispanic or insured with Medicaid are at the greatest risk.”

The big picture

The study analyzed data from more than 5.2 million hospitalizations collected by the 2019 Kids’ Inpatient Database, a national repository of data on hospital stays. It includes a 10% sample of newborns and an 80% sample of other pediatric discharges from 4,000 U.S. hospitals. More than 80% of patients were younger than 1 year of age.

The research team found that patients who are Black and Medicaid-insured patients experienced the greatest disparities in postoperative sepsis, a rare complication in which patients suffer from infection that can cause multi-organ failure. Patients who are Hispanic experienced the greatest disparity in postoperative respiratory failure, a complication that can limit breathing and ventilation.

Plausible factors cited include structural racism in the U.S. healthcare system, clinician bias, insufficient cultural responsiveness, communication barriers and limited access to high-quality healthcare.

What’s ahead

The study – “Disparities in Racial, Ethnic, and Payor Groups for Pediatric Safety Events in U.S. Hospitals” – is foundational in understanding what is happening among pediatric patients. Dr. Parikh said that researchers now must conduct further studies into these alarming disparities and qualitative work to understand drivers, with the action-oriented goal of developing interventions to improve patient safety in the hospital for all children.

“We brought together leaders in pediatric medicine, health policy and public health to analyze this data, and we are committed to taking the next steps to improve outcomes for pediatric patients,” Dr. Parikh said. “It will take more patient-centered work and research, resources and multifaceted strategies to resolve these worrying disparities for our pediatric patients nationwide.”

AI system that can detect RHD

Novel AI platform matches cardiologists in detecting rheumatic heart disease

Artificial intelligence (AI) has the potential to detect rheumatic heart disease (RHD) with the same accuracy as a cardiologist, according to new research demonstrating how sophisticated deep learning technology can be applied to this disease of inequity. The work could prevent hundreds of thousands of unnecessary deaths around the world annually.

Developed at Children’s National Hospital and detailed in the latest edition of the Journal of the American Heart Association, the new AI system combines the power of novel ultrasound probes with portable electronic devices installed with algorithms capable of diagnosing RHD on echocardiogram. Distributing these devices could allow healthcare workers, without specialized medical degrees, to carry technology that could detect RHD in regions where it remains endemic.

RHD is caused by the body’s reaction to repeated Strep A bacterial infections and can cause permanent heart damage. If detected early, the condition is treatable with penicillin, a widely available antibiotic. In the United States and other high-income nations, RHD has been almost entirely eradicated. However, in low- and middle-income countries, it impacts the lives of 40 million people, causing nearly 400,000 deaths a year.

“This technology has the potential to extend the reach of a cardiologist to anywhere in the world,” said Kelsey Brown, M.D., a cardiology fellow at Children’s National and co-lead author on the manuscript with Staff Scientist Pooneh Roshanitabrizi, Ph.D. “In one minute, anyone trained to use our system can screen a child to find out if their heart is demonstrating signs of RHD. This will lead them to more specialized care and a simple antibiotic to prevent this degenerative disease from critically damaging their hearts.”

The big picture

AI system that can detect RHD

The new AI system combines the power of novel ultrasound probes with portable electronic devices installed with algorithms capable of diagnosing RHD on echocardiogram.

Millions of citizens in impoverished countries have limited access to specialized care. Yet the gold standard for diagnosing RHD requires a highly trained cardiologist to read an echocardiogram — a non-invasive and widely distributed ultrasound imaging technology. Without access to a cardiologist, the condition may remain undetected and lead to complications, including advanced cardiac disease and even death.

According to the new research, the AI algorithm developed at Children’s National identified mitral regurgitation in up to 90% of children with RHD. This tell-tale sign of the disease causes the mitral valve flaps to close improperly, leading to backward blood flow in the heart.

Beginning in March, Craig Sable, M.D., interim division chief of Cardiology, and his partners on the project will implement a pilot program in Uganda incorporating AI into the echo screening process of children being checked for RHD. The team believes that a handheld ultrasound probe, a tablet and a laptop — installed with the sophisticated, new algorithm — could make all the difference in diagnosing these children early enough to change outcomes.

“One of the most effective ways to prevent rheumatic heart disease is to find the patients that are affected in the very early stages, give them monthly penicillin for pennies a day and prevent them from becoming one of the 400,000 people a year who die from this disease,” Dr. Sable said. “Once this technology is built and distributed at a scale to address the need, we are optimistic that it holds great promise to bring highly accurate care to economically disadvantaged countries and help eradicate RHD around the world.”

Children’s National Hospital leads the way

To devise the best approach, two Children’s National experts in AI — Dr. Roshanitabrizi and Marius George Linguraru, D.Phil., M.A., M.Sc., the Connor Family Professor in Research and Innovation and principal investigator in the Sheikh Zayed Institute for Pediatric Surgical Innovation — tested a variety of modalities in machine learning, which mimics human intelligence, and deep learning, which goes beyond the human capacity to learn. They combined the power of both approaches to optimize the novel algorithm, which is trained to interpret ultrasound images of the heart to detect RHD.

Already, the AI algorithm has analyzed 39 features of hearts with RHD that cardiologists cannot detect or measure with the naked eye. For example, cardiologists know that the heart’s size matters when diagnosing RHD. Current guidelines lay out diagnostic criteria using two weight categories — above or below 66 pounds — as a surrogate measure for the heart’s size. Yet the size of a child’s heart can vary widely in those two groupings.

“Our algorithm can see and make adjustments for the heart’s size as a continuously fluid variable,” Dr. Roshanitabrizi said. “In the hands of healthcare workers, we expect the technology to amplify human capabilities to make calculations far more quickly and precisely than the human eye and brain, saving countless lives.”

Among other challenges, the team had to design new ways to teach the AI to handle the inherent clinical differences found in ultrasound images, along with the complexities of evaluating color Doppler echocardiograms, which historically have required specialized human skill to evaluate.

“There is a true art to interpreting this kind of information, but we now know how to teach a machine to learn faster and possibly better than the human eye and brain,” Dr. Linguraru said. “Although we have been using this diagnostic and treatment approach since World War II, we haven’t been able to share this competency globally with low- and middle-income countries, where there are far fewer cardiologists. With the power of AI, we expect that we can, which will improve equity in medicine around the world.”

Drs. Catherine Limperopoulos, Yao Wu and David Wessel

AHA’s Outstanding Research Award: Three generations of pediatric cardiac excellence

Drs. Catherine Limperopoulos, Yao Wu and David Wessel

Catherine Limperopoulos, Ph.D., Yao Wu, Ph.D., and David Wessel, M.D.

Children’s National Hospital is celebrating a remarkable milestone as three of its faculty members have been honored over 15 years with the American Heart Association’s Outstanding Research in Pediatric Cardiology Award. Yao Wu, Ph.D., became the latest researcher to earn the accolade for her groundbreaking work into congenital heart disease (CHD).

A research faculty member with the newly established Center for Prenatal, Neonatal & Maternal Health Research, Dr. Wu received the award specifically for her studies on the role of altered placental function, measured by advanced in utero imaging, and neurodevelopmental outcomes in toddlers with CHD.

Honored at the association’s annual meeting in Philadelphia, Dr. Wu returned to Children’s National to warm congratulations from her colleagues who had previously won the award: David Wessel, M.D., executive vice president and chief medical officer, and Catherine Limperopoulos, Ph.D., director of the new center.

“I am thrilled to pass the baton to one of our own,” Dr. Limperopoulos said. “Dr. Wu’s recognition speaks to the outstanding and innovative research happening at Children’s National among junior faculty who are focusing on advancing our understanding of congenital heart disease and its long-term neurodevelopmental outcomes.”

Why we’re excited

The prestigious award represents more than individual accomplishments; it symbolizes three generations of mentorship and collaboration at the hospital. In 2007, Dr. Wessel joined Children’s National to enhance the care of newborns across specialty services by expanding programs and research, with a focus on critically ill newborns with heart disease. He recruited and mentored Dr. Limperopoulos in 2010, who became one of his research partners and creator of the hospital’s Center for Prenatal, Neonatal & Maternal Health Research. Dr. Limperopoulos, in turn, recruited and mentored Dr. Wu, providing her with the tools to conduct advanced imaging on in-utero brains and placentas, as well as the development of children with CHD.

“Each one of us is in different phases of our careers, yet we are connected by our deep interest in advancing cardiac care for critically ill newborns,” Dr. Wessel said. “In this collaborative environment, we learn from each other to improve entire lifetimes for our patients.”

Dr. Wu said she believes in sharing scientific developments for the advancement of the entire medical community. “It was an honor to be chosen to join this esteemed club, which has a relentless focus on improving health outcomes,” she said.

Children’s National leads the way

The award winners shared five collaborations published in leading journals to contribute to the ongoing dialogue in the field and the innovative work happening at Children’s National:

mother with newborn baby

Perinatal Mood and Anxiety Lab to launch at Children’s National

mother with newborn baby

The hospital has been working for years on improving screenings and support for perinatal mood and anxiety disorders.

Physician researchers at Children’s National Hospital secured a $1.8 million grant from the Agency for Healthcare Research and Quality (AHRQ) that will fund a Perinatal Mood and Anxiety Patient Safety Lab. Neonatologists, pediatric emergency medicine physicians, psychologists, computer scientists and the Perinatal Mood and Anxiety Disorder team from Children’s National will partner with systems engineers at Virginia Tech and Human Factors experts at MedStar Patient Safety Institute to set up a learning lab. The lab will improve mental health screening, referral and treatment of parents and caregivers at the hospital.

The need

“After multi-month admission to our NICU, 45% of parents screen positive for depression. I can’t think of any other disorder or disease that screens positive at 45%. This can’t be ignored,” says Lamia Soghier, M.D. M.Ed., M.B.A., neonatologist and medical director of the Neonatal Intensive Care Unit (NICU) at Children’s National. “Our goal is to provide safe, comprehensive, point-of-care access to mental health services for caregivers of infants treated at our hospital. I can’t think of a better team on the cutting edge that’s qualified to tackle this issue.”

The big picture

The new grant will tackle three major aims:

  • Optimize screening, referral and treatment for postpartum depression in the NICU and the Pediatric Emergency Department (ED).
  • Design and develop a novel software dashboard for real-time tracking of the screening, referral and treatment stages for eligible mothers.
  • Implement new solutions and evaluate latent safety threats related to missed screening, referral or treatment in current and future systems.

Researchers from the Center for Prenatal, Neonatal & Maternal Health Research and population health experts from the Child Health Advocacy Institute at Children’s National will also support this work.

Leading the way

“Children’s National is truly an innovator in this space,” says Dr. Soghier. “There are very few pediatric hospitals working with families to screen for mental health in the NICU, and fewer tackling the problem in the ED. Our team is dedicated to paving this path.”

The hospital has been working for years on improving screenings and support for perinatal mood and anxiety disorders, which was originally made possible by an investment from the A. James & Alice B. Clark Foundation to Children’s National aimed at providing families with greater access to mental health care and community resources. This new AHRQ grant will support the trajectory and goals of this work.

illustration of laser damaging the plasma membrane

The microscopic world of cell healing: A window into future therapies

illustration of laser damaging the plasma membrane from Advanced Science coverUnraveling how cells mend after injury serves as a key to unlocking potential therapies. Recent findings from the Center for Genetic Medicine Research at Children’s National Hospital offered surprising insights into the cell’s healing mechanisms by illuminating the intricate cellular responses to various types of injuries.

The study, featured on the back cover of the latest issue of Advanced Science, found that cells respond in distinct ways depending on the type of injury, such as a traumatic muscle tear that creates a large injury or tiny holes in the cell membrane caused by pathogenic proteins. Daniel Bittel, DPT, Ph.D., a research postdoctoral fellow at the Center for Genetic Medicine Research, said that cells are routinely injured from even everyday activities, such as walking up a flight of stairs.

“Injuries often involve damage to the plasma membrane,” Bittel said. “We wanted to investigate how healing happens at the subcellular level to better understand diseases and develop targeted therapies. We were especially curious about muscle cells because, interestingly, healthy ones get stronger the more that they are injured.”

The fine print

Using the center’s unique, custom-built microscope, the research team zoomed in on the process of cellular healing to watch how cells activate repair after injuries. Using a laser to damage the plasma membrane, they mimicked mechanically induced trauma. They also used a pathogen-derived protein to create nanoscale pinprick injuries in a cell’s plasma membrane that resemble those that are seen after strenuous muscle exertion.

Then, they watched as cells went to work within seconds, engaging healing mechanisms tailored to the type of injury. In the case of a cell facing numerous pinpricks along the cell membrane, it immediately deployed the endocytic pathway used by the cells to eat and drink. This process helped remove the injurious agents and the tiny holes they made. However, with a larger mechanical injury, the cells demonstrated patience, allowing the plasma membrane to seal before clearing up the damage by the same endocytic pathway.

 The big picture

The paper is part of an ongoing body of research on cell injury that will inform future investigations into a wide range of pediatric health issues including muscular dystrophies, injuries to neurons, orthopedic injuries from sports and other mechanical damage to tissues.

Jyoti Jaiswal, M.Sc., Ph.D., senior investigator at the Center for Genetic Medicine Research, said this work is foundational in the development of new therapies. “Knowing where the problem lies will help us figure out what therapy will work best and target the therapy to address the specific deficit,” he said. “This work will pave the way to help tailor therapies and tackle diseases more effectively.”

The international NexTGen team

NexTGen team assembles to delve into progress on CAR T-cell therapies

The international NexTGen team assembled at the Children’s National Research & Innovation Campus for their annual meeting to share progress made in their first full year of work on the $25-million Cancer Grand Challenge, focused on creating a CAR T-cell therapy for pediatric solid tumors.

“It was invigorating to bring the whole team together from our eight institutions in the U.S., U.K. and France, as we uncover opportunities in our research and share the headway that we have made,” said Catherine Bollard, M.D., M.B.Ch.B., director of the Center for Cancer and Immunology Research and interim chief academic officer at Children’s National Hospital. “Breakthroughs happen when Team Science collaborates, and that is exactly what is happening here with the NexTGen team.”

Why we’re excited

Over the course of two days, more than 85 team members met to discuss the six work packages that are coming together, with the ambitious goal of making CAR T-cell therapies the standard of care for solid tumors within the next decade:

  • Discovery of new targets
  • The tumor microenvironment
  • Component engineering
  • Integration and modeling
  • Clinical studies
  • Data integration

Each work package includes a patient advocate – individuals with a personal connection to cancer as a family member or survivor – who offers their invaluable perspectives on the research and treatment process. Many attended the meeting, sitting alongside the oncologists, immunologists, mathematicians, molecular biologists and other leading experts.

The big picture

The Cancer Grand Challenges are funded by grants from the National Cancer Institute, Cancer Research U.K. and the Mark Foundation for Cancer Research. Their goal is to drive progress against cancer by empowering global leaders in the research community to take on tough challenges and think differently.

“They call it a ‘grand challenge’ for a reason,” Dr. Bollard said. “It’s going to take the effort and expertise of all these individuals to make a new therapy a reality. I have confidence that we can do it.”

pregnant woman looking at sonogram

Babies with congenital heart disease display disrupted brain function before birth

pregnant woman looking at sonogram

In their study, the team at Children’s National Hospital found that specific brain regions become especially vulnerable to injury around 24 weeks of pregnancy when developing babies begin to have high energy demands and rapid neurovascular changes.

For the first time, researchers have found that babies born with congenital heart disease (CHD) have alterations to the emerging functional connectivity of their brains in utero. The changes are related to the subtype of their CHD and their oxygen status before they have lifesaving surgery to treat their cardiac malformation, according to new findings published in the American Heart Association’s Circulation Research.

In their study, the team at Children’s National Hospital found that specific brain regions become especially vulnerable to injury around 24 weeks of pregnancy when developing babies begin to have high energy demands and rapid neurovascular changes. That leaves certain parts of the brain, including the brainstem, more susceptible to injury from cardiac complications and poor circulation.

“We used a special type of magnetic resonance imaging to safely study the brains of these unborn babies, and we found that they have weakened connectivity in the deep grey structures, which are responsible for sensation, movement, alertness and other core functions,” said Josepheen De Asis-Cruz, M.D., Ph.D., assistant professor at the Developing Brain Institute at Children’s National and an author of the study. “This offers an important clue in utero to the type of care the babies will eventually need when they are born.”

The big picture

In the past decade, the survival rate for fetuses with CHD has greatly improved. About 80% of cases – even some of the most high-risk heart defects – can be successfully treated or palliated with surgery and survive. Yet Dr. Cruz said researchers are finding that the rates of poor neurodevelopmental outcomes are about the same. That’s why she and her colleagues are looking at what precisely may be injuring the brains of these newborns prior to surgery, offering a possible roadmap to interventions.

The fine print

The research team studied 107 healthy, low-risk pregnancies and 75 pregnancies known to be complicated by CHD. They used functional connectivity magnetic resonance imaging (fcMRI) to examine the emerging connections of the brains of unborn babies, given fcMRI’s unique ability to query the brain in a resting state when a patient is unable to respond to tasks. They also studied the oxygen saturation levels of the babies after they were born and then mapped all of this information to the type of CHD that they were diagnosed with.

“Our findings indicate that the compromised connectivity in the brains of CHD patients before delivery is linked to hypoxia after birth,” Cruz said. “There were important differences in the low- and high-risk CHD groups. Babies born with transposition of the great arteries or hypoplastic left heart syndrome – two of the most high-risk diagnoses – have notable changes in their brain function, which could someday be used as biomarkers to guide their care.”

What’s ahead

Researchers at Children’s National are working together, using a variety of modalities, to move toward precision imaging in utero to help predict a child’s neurodevelopmental outcomes. The ultimate goal: better interventions sooner.

“This work is foundational. As we fine-tune more techniques to identify babies at risk, we can understand how environmental, genetic and epigenetic factors impact brain development and guide care decisions,” said Catherine Limperopoulos, Ph.D., director of the Center for Prenatal, Neonatal & Maternal Health Research and a senior author of the paper. “We can imagine a day where we can offer pregnant mothers highly detailed and personalized information about their unborn baby, and individualized interventions that lead to healthier lifetimes.”

Motor neuron connecting to muscle fiber

FDA approves muscular dystrophy drug built on Children’s National research



Motor neuron connecting to muscle fiber

Duchenne muscular dystrophy (DMD) is the most common hereditary neuromuscular disease.



Boys with Duchenne muscular dystrophy (DMD) have a clinically proven, new treatment option with the Food and Drug Administration’s approval of vamorolone, a steroidal-type, anti-inflammatory drug developed based on research performed at Children’s National Hospital.

Created by ReveraGen BioPharma Inc., vamorolone has a molecular structure similar to traditional corticosteroids, which are currently used to treat DMD. Yet its structure was found to be chemically different enough to reduce unwanted side effects, including brittle bones and reduced stature. Nearly two decades ago, ReveraGen leaders – President and CEO Eric Hoffman, Ph.D., and Vice President for Research Kanneboyina Nagaraju, D.V.M., Ph.D. – launched research efforts into the drug when they led the Center for Genetic Medicine Research at Children’s National. They worked with then-Chief Academic Officer Mark Batshaw, M.D., on the new clinical option.

“Throughout my career, I have treated children with DMD, and I have seen over time how their shorter heights and brittle bones impact them physically and emotionally – in terms of their self-esteem and ability to participate in activities,” Dr. Batshaw said. “This drug should help these boys function more effectively and prevent certain long-term complications.”

The patient benefit

Muscular dystrophy includes a group of degenerative genetically inherited neuro-muscular diseases that strike only boys. DMD is the most common, severe and life-threatening form of muscular dystrophy. ReveraGen studied vamorolone for patients ages two years and up in the hopes of providing a new, FDA-approved treatment option for these children. In clinical trials, daily treatment with vamorolone improved muscle strength and stature with results comparable to prednisolone, but without some of the most impactful side effects of steroids, particularly the stunted growth and weakened bones.

Children’s National Hospital leads the way

Kolaleh Eskandanian, Ph.D., M.B.A., P.M.P., vice president and chief innovation officer for Children’s National, said Drs. Hoffman and Nagaraju’s work on the drug paved the way for entrepreneurship at the hospital, as they were the first faculty members to launch a spin-off company. Since then, more than 130 faculty members have been named as inventors on 132 patents. Children’s National is now home to Innovation Ventures, the hospital’s intellectual property development and commercialization arm, which provides guidance and resources to academic entrepreneurs who introduce a concept for pediatric medical products.

“We cannot wait to see the tremendous effort behind vamorolone in the hands of patients and clinicians treating Duchenne muscular dystrophy,” Eskandanian said. “Today’s FDA approval for ReveraGen shows the importance of supporting clinicians and researchers who are developing solutions to advance healthcare for children.”

Why we’re excited

Hoffman said the drug has been through a series of clinical trials showing advantages over the current treatment options. In 2024, Catalyst Pharma will market vamorolone under the trade name Agamree in the United States.

“Vamorolone was developed using a different business model and drug development approach, including partnerships with the National Institutes of Health, Department of Defense, the European Commission and more than a dozen international nonprofit foundations,” Dr. Hoffman said. “The collaborative, community-engaged approach—including 32 academic clinical sites in 11 countries — and the participation of hundreds of DMD families led to this approval today.”

collage of hyperspectral imaging (sHSI) camera and brain surgery

Novel camera + machine learning = hope for more precise neurosurgery

collage of hyperspectral imaging (sHSI) camera and brain surgery

Researchers at Children’s National Hospital developed a compact imaging camera capable of seeing beyond the human visual spectrum to help segment healthy brain tissue from tumors during surgery. The groundbreaking technology will allow neurosurgeons to make more precise, real-time decisions in the operating room, rather than sending samples to pathology labs for biopsies.

In a manuscript published in Bioengineering, the team of engineers and neurosurgeons details how its snapshot hyperspectral imaging (sHSI) camera can be used to capture and process images of brain tissue, using the wide spectrum of light between visible and infrared wavelengths. That additional information — beyond the human eye — has the potential to allow for more accurate and complete tumor removal.

“In the hands of a neurosurgeon, this camera, when combined with machine learning, could dramatically improve outcomes for some of our most vulnerable brain tumor patients,” said Richard Jaepyeong Cha, Ph.D., an optical engineer and principal investigator at the Sheikh Zayed Institute of Pediatric Surgical Innovation. “We are able to attach the camera to a surgical microscope and process a significant amount of information from the patient while in the operating room. Not only could this lead to more complete tumor resection, it will also allow the surgeon to save as much healthy brain tissue as possible and reduce lifelong neurological complications.”

Why we’re excited

Brain tumors are the most common solid tumors in children, accounting for the highest number of pediatric cancer deaths globally each year. To develop a treatment plan, neurosurgeons need to understand the tumor’s features, including its type, grade of malignancy, location and its categorization as a primary or metastatic cancer. This information leads to decisions about how to remove or biopsy a tumor.

Under the current protocols, surgeons evaluate tumor margins in the operating room by examining the appearance of the brain tissue and sending out small samples to the pathology department for biopsies. This can lead to longer surgeries and difficult real-time surgical decisions. For instance, some low-grade tumors are visually indistinguishable from healthy brain tissue.

In four investigational cases approved by the hospital’s institutional research board, the sHSI camera was used in the operating room to help segment healthy pediatric brain tissue from tumors. Unlike the conventional red-green-blue (RGB) imaging cameras, which use only those three colors, HSI captures spectral data at each pixel of the image — a task too complex for the human eye — and sends it instantly for processing by an algorithm designed to assist in tumor segmentation.

What’s ahead

Despite the small dataset, the researchers were able to successfully segment healthy brain tissue from lesions with a high specificity during pediatric brain tumor resection procedures. Significant work remains to refine the technology and the machine learning behind it. Researchers also plan to integrate the sHSI camera into a laparoscope to visualize tumors that are not on the brain’s surface and collect data from more angles.

“As we develop these groundbreaking tools, we plan to continue to expand the dataset and refine the algorithm to make pediatric neurosurgery continually more precise,” said Naomi Kifle, M.S., research and development engineer at Children’s National and first author on the paper. “As our dataset grows, we hope to create a model that can distinguish healthy brain tissue, tumor and skull. This groundbreaking surgical tool shows significant promise.”

data science illustration

Federated learning: A solution to AI’s data-sharing challenges

data science illustration

Federated learning can solve data-sharing challenges, allowing nimble collaboration across institutions to drive medical advances using artificial intelligence (AI).

Federated learning can solve data-sharing challenges, allowing nimble collaboration across institutions to drive medical advances using artificial intelligence (AI), according to a new manuscript from 10 thought leaders in AI and machine learning in medicine.

In Health Informatics Journal, these leading experts on how technology is shaping medicine shared a conversation that they had at the Radiology Society of North America’s conference. They weighed challenges facing AI, including barriers to data sharing because of privacy rules that prevent the distribution of information to different institutions. With federated learning, models are shared – rather than data – allowing institutions to aggregate information and collaborate with a master model.

“Federated learning offers tremendous promise,” said Marius George Linguraru, D.Phil., M.A., M.Sc., the Connor Family Professor of Research and Innovation, principal investigator at the Sheikh Zayed Institute of Pediatric Surgical Innovation and senior author on the manuscript. “As a community of experts, we have found that federated learning allows us to move away from the challenges of sharing data in central repositories. Instead, we share the models, which can be designed to protect privacy by limiting what’s shared outside of any given institution.”

A champion of pediatric health, Linguraru wants to ensure that children are represented in the development of models that advance science and medicine. “Sharing data is even more crucial when there are few patients, such as in rare diseases or pediatric populations,” he said. “In general, healthcare data suffers from inequitable representation in our public health systems and services.”

Learn more here about the challenges and potential solutions from experts at Rhino Health, Johns Hopkins University School of Medicine, NVIDIA, University of Cambridge, Ben-Gurion University Israel, MD Anderson Cancer Center, Dana-Farber Cancer Institute and Children’s National Hospital.

Winners of the International Conference on Medical Image Computing and Computer Assisted Intervention

AI team wins international competition to measure pediatric brain tumors

Winners of the International Conference on Medical Image Computing and Computer Assisted Intervention
Children’s National Hospital scientists won first place in a global competition to use artificial intelligence (AI) to analyze pediatric brain tumor volumes, demonstrating the team’s ground-breaking advances in imaging and machine learning.

During the International Conference on Medical Image Computing and Computer Assisted Intervention (MICCAI), the Children’s National team demonstrated the most accurate algorithm to study the volume of brain tumors – the most common solid tumors affecting children and adolescents and a leading cause of disease-related death at this young age. The technology could someday help oncologists understand the extent of a patient’s disease, quantify the efficacy of treatments and predict patient outcomes.

“The Brain Tumor Segmentation Challenge inspires leaders in medical imaging and deep learning to try to solve some of the most vexing problems facing radiologists, oncologists, computer engineers and data scientists,” said Marius George Linguraru, D.Phil., M.A., M.Sc., the Connor Family Professor in Research and Innovation and principal investigator in the Sheikh Zayed Institute for Pediatric Surgical Innovation. “I am honored that our team won, and I’m even more thrilled for our clinicians and their patients, who need us to keep moving forward to find new ways to treat pediatric brain tumors.”

Why we’re excited

With roughly 4,000 children diagnosed yearly, pediatric brain tumors are consistently the most common type of pediatric solid tumor, second only to leukemia in pediatric malignancies. At the urging of Linguraru and one of his peers at the Children’s Hospital of Philadelphia, pediatric data was included in the international competition for the first time, helping to ensure that children are represented in medical and technological advances.

The contest required participants to use data from multiple institutions and consortia to test competing methods fairly. The Children’s National team created a method to tap into the power of two types of imaging and machine learning: 3D convolutional neural network and 3D Vision Transformer-based deep learning models. They identified regions of the brain affected by tumors, made shrewd data-processing decisions driven by the team’s experience in AI for pediatric healthcare and achieved state-of-the-art results.

The competition drew 18 teams who are leaders from across the AI and machine learning community. The runner-up teams were from NVIDIA and the University of Electronic Science and Technology of China.

The big picture

“Children’s National has an all-star lineup, and I am thrilled to see our scientists recognized on an international stage,” said interim Executive Vice President and Chief Academic Officer Catherine Bollard, M.D., M.B.Ch.B., director of the Center for Cancer for Immunology Research. “As we work to attack brain tumors from multiple angles, we continue to show our exceptional ability to create new and better tools for diagnosing, imaging and treating these devastating tumors.”

Patient and doctor demoing Rare-CAP technology

M.D. in your pocket: New platform allows rare disease patients to carry medical advice everywhere

When someone has a rare disease, a trip to the emergency room can be a daunting experience: Patients and their caregivers must share the particulars of their illness or injury, with the added burden of downloading a non-specialist on the details of a rare diagnosis that may change treatment decisions.

Innovators at Children’s National Hospital and Vanderbilt University Medical Center, supported by Takeda, are trying to simplify that experience using a new web-based platform called the Rare Disease Clinical Activity Protocols, or Rare-CAP. This revolutionary collection of medical information allows patients to carry the latest research-based guidance about their rare disorders in their phones, providing a simple QR code that can open a trove of considerations for any medical provider to evaluate as they work through treatment options for someone with an underlying rare disease.

“No one should worry about what happens when they need medical help, especially patients with rare diseases,” said Debra Regier, M.D., division chief of Genetics and Metabolism at Children’s National and Rare-CAP’s lead medical advisor. “We built this new tool because I have watched as my patient-families have wound up in an emergency room — after all, kids get sprains or fractures — but they don’t have the expertise of a rare disease specialist with them. My hope is that they’re going to pull out their phones and access Rare-CAP, which will explain their rare disease to a new provider who can provide more thoughtful and meaningful care.”

The big picture

A rare disease is defined as any disorder that affects less than 200,000 people in the United States. Some 30 million Americans are believed to be living with one of the 7,000 known rare disorders tracked by the National Organization of Rare Diseases (NORD). Led by Dr. Regier, the Rare Disease Institute at Children’s National is one of 40 NORD centers for excellence in the country that provide care, guidance and leadership for the wide array of disorders that make up the rare disease community.

While a key goal of Rare-CAP is to bolster patient self-advocacy, the platform will also allow medical providers to proactively search for protocols on rare diseases when they know they need specialized advice from experts at Children’s National, a network of tertiary care centers and patient organizations.

As a leading values-based, R&D-driven biopharmaceutical company, Takeda has committed $3.85 million to the project to help activate meaningful change and empower a brighter future for rare disease communities, providing a unique understanding of the struggle that patients and caregivers face when they need care.

“Our team, alongside the medical and rare disease community, saw the need for a single portal to collect standardized care protocols, and we are thrilled to see this innovative tool come to life,” said Tom Koutsavlis, M.D., head of U.S. Medical Affairs at Takeda. “People with rare diseases and their caregivers need faster access to authoritative medical information that providers anywhere can act on, this will lead to improving the standard of care, accelerating time to diagnosis and breaking down barriers to increase equitable access.”

The patient benefit

The creators of Rare-CAP imagined its use in a wide range of settings, including emergency rooms, surgical suites, dental offices, urgent care offices and school clinics. The platform will eventually profile thousands of rare diseases and lay out the implications for care, while also creating a dynamic conversation among users who can offer updates based on real-world experience and changes in medical guidance.

“Our patients are unique, and so is this tool,” Dr. Regier said. “As we roll out Rare-CAP, we believe it is just the beginning of the conversation to expand the platform and see its power for the patient and provider grow, with each entry and each new rare disease that’s added to the conversation.”