Allergy & Immunology

The international NexTGen team

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

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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.”

ARPA-H logo

Children’s National selected as member of ARPA-H Investor Catalyst Hub spoke network

ARPA-H logoThe hospital will advocate for the unique needs of children as part of nationwide network working to accelerate transformative health solutions.

Children’s National Hospital was selected as a spoke for the Investor Catalyst Hub, a regional hub of ARPANET-H, a nationwide health innovation network launched by the Advanced Research Projects Agency for Health (ARPA-H).

The Investor Catalyst Hub seeks to accelerate the commercialization of groundbreaking and accessible biomedical solutions. It uses an innovative hub-and-spoke model designed to reach a wide range of nonprofit organizations and Minority-Serving Institutions, with the aim of delivering scalable healthcare outcomes for all Americans.

“The needs of children often differ significantly from those of adults. This partnership reflects our commitment to advancing pediatric healthcare through innovation and making sure we’re addressing those needs effectively,” said Kolaleh Eskandanian, Ph.D., M.B.A., vice president and chief innovation officer at Children’s National. “Leveraging the strength of this hub-and-spoke model, we anticipate delivering transformative solutions to enhance the health and well-being of the patients and families we serve.”

Children’s National joins a dynamic nationwide network of organizations aligned to ARPA-H’s overarching mission to improve health outcomes through the following research focus areas: health science futures, proactive health, scalable solutions and resilient systems. Investor Catalyst Hub spokes represent a broad spectrum of expertise, geographic diversity and community perspectives.

“Our spoke network embodies a rich and representative range of perspectives and expertise,” said Mark Marino, vice president of Growth Strategy and Development for VentureWell and project director for the Investor Catalyst Hub. “Our spokes comprise a richly diverse network that will be instrumental in ensuring that equitable health solutions reach communities across every state and tribal nation.”

As an Investor Catalyst Hub spoke, Children’s National gains access to potential funding and flexible contracting for faster award execution compared to traditional government contracts. Spoke membership also offers opportunities to provide input on ARPA-H challenge areas and priorities, along with access to valuable networking opportunities and a robust resource library.

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.”

U.S. News Badges

Children’s National Hospital ranked #5 in the nation on U.S. News & World Report’s Best Children’s Hospitals Honor Roll

U.S. News BadgesChildren’s National Hospital in Washington, D.C., was ranked #5 in the nation on the U.S. News & World Report 2023-24 Best Children’s Hospitals annual rankings. This marks the seventh straight year Children’s National has made the Honor Roll list. The Honor Roll is a distinction awarded to only 10 children’s hospitals nationwide.

For the thirteenth straight year, Children’s National also ranked in all 10 specialty services, with eight specialties ranked in the top 10 nationally. In addition, the hospital was ranked best in the Mid-Atlantic for neonatology, cancer, neurology and neurosurgery.

“Even from a team that is now a fixture on the list of the very best children’s hospitals in the nation, these results are phenomenal,” said Kurt Newman, M.D., president and chief executive officer of Children’s National. “It takes a ton of dedication and sacrifice to provide the best care anywhere and I could not be prouder of the team. Their commitment to excellence is in their DNA and will continue long after I retire as CEO later this month.”

“Congratulations to the entire Children’s National team on these truly incredible results. They leave me further humbled by the opportunity to lead this exceptional organization and contribute to its continued success,” said Michelle Riley-Brown, MHA, FACHE, who becomes the new president and CEO of Children’s National on July 1. “I am deeply committed to fostering a culture of collaboration, empowering our talented teams and charting a bold path forward to provide best in class pediatric care. Our focus will always remain on the kids.”

“I am incredibly proud of Kurt and the entire team. These rankings help families know that when they come to Children’s National, they’re receiving the best care available in the country,” said Horacio Rozanski, chair of the board of directors of Children’s National. “I’m confident that the organization’s next leader, Michelle Riley-Brown, will continue to ensure Children’s National is always a destination for excellent care.”

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

“For 17 years, U.S. News has provided information to help parents of sick children and their doctors find the best children’s hospital to treat their illness or condition,” said Ben Harder, chief of health analysis and managing editor at U.S. News. “Children’s hospitals that are on the Honor Roll transcend in providing exceptional specialized care.”

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

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

The other two specialties ranked among the top 50 were cardiology and heart surgery, and urology.

Panel members at the NIAID symposium

CN-NIAID Symposium seeks ways to promote child health amid challenges

Panel members at the NIAID symposium

More than 30 million children seek emergency care each year, but 80 percent of these visits happen at hospitals that aren’t designed for pediatrics — a daunting figure during pandemics and other crises in healthcare. This considerable hurdle is one of many challenges that leaders in pediatric health came to discuss during a two-day symposium on promoting child health, hosted by Children’s National Hospital, the National Institute of Allergy and Infectious Diseases and the Pediatric Pandemic Network (PPN).

The symposium laid out a multitude of issues facing children and their doctors: growing mental health diagnoses, shrinking access to care in rural areas, asthma and eczema, winter respiratory surges and more.

Joelle Simpson, M.D., chief of emergency medicine at Children’s National and PPN principal investigator, said the network is drawing on expertise from 10 pediatric hospitals to ensure communities are better prepared for whatever challenges lie ahead, through training and support, collaboration among pediatric specialists, education on best practices and the promotion of equity and inclusion.

Built on a Health Resources and Services Administration grant, the network is focusing on four key areas: infectious disease and disease outbreaks, emergency and disaster management, mental and behavioral health, and health equity and community engagement. “This year, we know we are boiling the ocean as we come together,” Simpson said.

Miss the symposium? Check out the recordings available on YouTube, including the closing Q&A with many of the panelists and Sheryl Gay Stolberg, health policy reporter with the New York Times.

Day 1 of the 6th Annual Children’s National Hospital – NIAID Symposium

Day 2 of the 6th Annual Children’s National Hospital – NIAID Symposium

 

William Sheehan

Allergist taps into biologics to treat asthma

William SheehanWilliam Sheehan, M.D., joined Children’s National Hospital in 2017 as a board-certified allergist and immunologist with a passion for helping patients with asthma, allergies, eczema and immunodeficiency. During his fellowship at Children’s Hospital of Boston, he investigated the impact of inner-city and school environments on children’s asthma, and he found reward in helping children breathe easier. Dr. Sheehan will be discussing the impact of the environment on childhood asthma at the upcoming CNH-NIAID Symposium on Promoting Child Health: From Environmental Challenges to Pandemic. He shared with Innovation District an overview of his research and his hope for using biologics to treat asthma.

Q: Could you talk about the research you have been involved in?

A: Since my fellowship, my work has been specifically targeting the interaction between environmental exposures in school and asthma. By intervening in the school environment, we could help dozens of children with asthma. In recent years and here at Children’s National, I have become interested in research regarding using biological medicines to treat inner-city children with asthma. These antibody medications, given by injection, target cells that cause inflammation and are used in children who have severe asthma that is not responding to conventional therapies.

Q: What is new about biologics for treating childhood asthma? How are researchers at Children’s National contributing to the field?

A: The first biologic medicine for treating asthma, called Omalizumab, was approved in 2003. In the last five years, four more biologics have been approved for treating childhood asthma, with more in the pipeline. At Children’s National, we have been trying to identify which biologic therapy is most effective for a particular child with asthma by looking at specific biomarkers. We also are examining new biomarkers that may be beneficial. Specifically, we are looking at the levels of regulatory B cells, an immune cell that acts as a brake to control allergic inflammation.  We have shown that these cells are lacking in children with severe asthma and may make these children better candidates for biologic therapies.

Q: How prevalent are young patients in the asthma population in D.C., and do you believe environmental factors associated with living in a city contribute to the higher incidence of asthma in this population?

A: D.C. is a challenging city for pediatric asthma. Not only is asthma prevalence higher here, but we also see higher rates of severe asthma requiring emergency visits, hospitalizations and even admissions to the Pediatric Intensive Care Unit.

While environmental factors such as pollution, second-hand smoke and exposure to mice and cockroaches can contribute to childhood asthma, I don’t think it’s the sole factor in D.C. There might be multiple factors at play, including social structures that make it difficult for families to access and maintain asthma care and medications. Additionally, there seems to be a genetic component as Black and Hispanic children tend to suffer from worse asthma than white children, regardless of the environment.

Q: What specific research areas or topics interest you for future study?

A: We have made significant progress in controlling asthma with biologics, but prevention is the next frontier. Current studies are focusing on treating vulnerable children at a very young age with the goal of preventing the development of asthma later in childhood. If we can’t prevent asthma, we should strive to achieve asthma “remission” through the use current medications and biologic therapies. The goal would be for individuals to go through their year without any symptoms or exacerbations, essentially living as though they don’t have asthma at all.

Abstract Happy 2022 New Year greeting card with light bulb

The best of 2022 from Innovation District

Abstract Happy 2022 New Year greeting card with light bulbA clinical trial testing a new drug to increase growth in children with short stature. The first ever high-intensity focused ultrasound procedure on a pediatric patient with neurofibromatosis. A low dose gene therapy vector that restores the ability of injured muscle fibers to repair. These were among the most popular articles we published on Innovation District in 2022. Read on for our full top 10 list.

1. Vosoritide shows promise for children with certain genetic growth disorders

Preliminary results from a phase II clinical trial at Children’s National Hospital showed that a new drug, vosoritide, can increase growth in children with certain growth disorders. This was the first clinical trial in the world testing vosoritide in children with certain genetic causes of short stature.
(2 min. read)

2. Children’s National uses HIFU to perform first ever non-invasive brain tumor procedure

Children’s National Hospital successfully performed the first ever high-intensity focused ultrasound (HIFU) non-invasive procedure on a pediatric patient with neurofibromatosis. This was the youngest patient to undergo HIFU treatment in the world.
(3 min. read)

3. Gene therapy offers potential long-term treatment for limb-girdle muscular dystrophy 2B

Using a single injection of a low dose gene therapy vector, researchers at Children’s National restored the ability of injured muscle fibers to repair in a way that reduced muscle degeneration and enhanced the functioning of the diseased muscle.
(3 min. read)

4. Catherine Bollard, M.D., M.B.Ch.B., selected to lead global Cancer Grand Challenges team

A world-class team of researchers co-led by Catherine Bollard, M.D., M.B.Ch.B., director of the Center for Cancer and Immunology Research at Children’s National, was selected to receive a $25m Cancer Grand Challenges award to tackle solid tumors in children.
(4 min. read)

5. New telehealth command center redefines hospital care

Children’s National opened a new telehealth command center that uses cutting-edge technology to keep continuous watch over children with critical heart disease. The center offers improved collaborative communication to better help predict and prevent major events, like cardiac arrest.
(2 min. read)

6. Monika Goyal, M.D., recognized as the first endowed chair of Women in Science and Health

Children’s National named Monika Goyal, M.D., M.S.C.E., associate chief of Emergency Medicine, as the first endowed chair of Women in Science and Health (WISH) for her outstanding contributions in biomedical research.
(2 min. read)

7. Brain tumor team performs first ever LIFU procedure on pediatric DIPG patient

A team at Children’s National performed the first treatment with sonodynamic therapy utilizing low intensity focused ultrasound (LIFU) and 5-aminolevulinic acid (5-ALA) medication on a pediatric patient. The treatment was done noninvasively through an intact skull.
(3 min. read)

8. COVID-19’s impact on pregnant women and their babies

In an editorial, Roberta L. DeBiasi, M.D., M.S., provided a comprehensive review of what is known about the harmful effects of SARS-CoV-2 infection in pregnant women themselves, the effects on their newborns, the negative impact on the placenta and what still is unknown amid the rapidly evolving field.
(2 min. read)

9. Staged surgical hybrid strategy changes outcome for baby born with HLHS

Doctors at Children’s National used a staged, hybrid cardiac surgical strategy to care for a patient who was born with hypoplastic left heart syndrome (HLHS) at 28-weeks-old. Hybrid heart procedures blend traditional surgery and a minimally invasive interventional, or catheter-based, procedure.
(4 min. read)

10. 2022: Pediatric colorectal and pelvic reconstructive surgery today

In a review article in Seminars in Pediatric Surgery, Marc Levitt, M.D., chief of the Division of Colorectal and Pelvic Reconstruction at Children’s National, discussed the history of pediatric colorectal and pelvic reconstructive surgery and described the key advances that have improved patients’ lives.
(11 min. read)

girl getting a vaccine

Second dose of Pfizer COVID-19 vaccine safe for children with allergic reaction to first dose

girl getting a vaccineA new study published in The Journal of Allergy and Clinical Immunology: In Practice found that pediatric patients who experienced an adverse reaction to the first dose of the Pfizer-BioNTech COVID-19 vaccine or with suspected polyethylene glycol or polysorbate allergies can safely receive the second dose in a supervised setting. Until now, previous studies demonstrating second dose safety after a reaction to the first dose have only included adult patients.

“These results reaffirm similar studies performed in adults and provide additional assurance specific to the pediatric population,” says Joel Brooks, D.O., M.P.H., allergist and immunologist at Children’s National Hospital and corresponding author of the study. “We found that most of these initial reactions are not supportive of an IgE-mediated mechanism.”

The researchers evaluated 13 children referred to a specialized vaccine clinic for suspected immediate allergic reactions to the first dose of the Pfizer-BioNTech COVID-19 vaccine from May 2021 to February 2022. Nine of the 13 children were evaluated after experiencing an allergic reaction following the first dose. All nine successfully received the second dose with no or minimal symptoms.

The other four children were evaluated after clinical histories of PEG/polysorbate allergy. Three of the four received both Pfizer vaccine doses with no symptoms. The fourth patient elected to receive the Janssen COVID-19 vaccine.

“It is important that children 6 months and older receive two doses of the COVID-19 vaccine for full protection from severe illness and hospitalization due to COVID-19 infection,” adds Dr. Brooks. “Children with potential anaphylaxis should undergo careful evaluation to weigh the benefits and risks of the second dose.”

You can read the full study, “Safety outcomes of SARS-CoV-2 vaccination in pediatric patients with a first dose reaction history or allergy to polyethylene glycol or polysorbate,” here.

emergency signs

Social risk interventions significantly reduce asthma-related ED visits

emergency signs

In a systematic review and meta-analysis published in JAMA Pediatrics, researchers at Children’s National Hospital found that interventions focused on health, environment and community were associated with the greatest reduction in asthma-related emergency department visits and hospitalizations among children.

In a systematic review and meta-analysis published in JAMA Pediatrics, researchers at Children’s National Hospital found that interventions focused on health, environment and community were associated with the greatest reduction in asthma-related emergency department visits and hospitalizations among children. The findings suggest that addressing social risks may be a crucial component of pediatric asthma care to improve health outcomes.

“There are persistent and striking disparities in asthma outcomes among children,” said Jordan Tyris, M.D., hospitalist fellow at Children’s National and lead author. “Understanding how to address these is of utmost importance.”

“Literature on the spectrum of social factors, including social needs, social risks and social determinants, has increased recently across many aspects of health care,” adds Dr. Tyris. “But much of this literature has focused on adults with chronic conditions, for example diabetes or high blood pressure.”

The study authors searched PubMed, Scopus, PsychINFO, SocINDEX and CINAHL from January 2008 to June 2021 for U.S.-based studies evaluating the associations of interventions addressing one or more social risks with asthma-related emergency department visits and hospitalizations among children. The systematic review included 38 of the original 641 identified articles (6%), and the meta-analysis included 19 articles (3%). Overall, participation in social risk–based interventions, particularly those that addressed health literacy, home environmental conditions and peer support were associated with significantly reduced risks for asthma-related emergency department visits and hospitalizations among children.

You can read the full study, “Social Risk Interventions and Health Care Utilization for Pediatric Asthma: A Systematic Review and Meta-analysis” in JAMA Pediatrics.

Epstein-Barr virus

Study with largest cohort in the Western world sheds light on Epstein-Barr virus

Epstein-Barr virus

Epstein-Barr virus is a member of the herpes family and it spreads primarily through saliva.

Children’s National Hospital experts provided a contemporary description of the epidemiology, clinical presentation and management of chronic active Epstein-Barr virus (CAEBV), shedding light on this very rare disease. The paper, published in Blood Advances, assessed 57 patients outside of Asia — the biggest international retrospective cohort study published in the Western world.

Epstein-Barr virus is a member of the herpes family and it spreads primarily through saliva. Once a person is infected with Epstein-Barr virus, the immune system will control the infections, but the virus lies in a dormant state in the patient’s B Cells. However, in some patients, there is a failure of the body to control the infection, and the virus is found inside the patient’s T and/or NK cells. These rare patients are diagnosed with CAEBV. The hallmark of the disease is proliferation of Epstein-Barr virus-infected T or NK cells that infiltrate tissues, leading to end-organ damage. Patients most often experience fevers, hepatosplenomegaly, liver inflammation, cytopenias and lymphoproliferation that may progress to lymphoma.

Given it is most prevalent in Asia, little is known about the disease in the Western world. There has only been one published paper regarding the outcomes of patients in the U.S., which included 19 patients amassed over 28 years, and was published a decade ago.

Multiple treatments have been attempted to control the disease, but none have resulted in consistent remission. Historically, the consensus is to use steroids and/or antiviral drug in combination with proteasome inhibitor agents. In some cases, clinicians also use cytotoxic chemotherapy to reduce disease burden and improve the patient’s condition before HSCT. Still, this approach is limited because most patients die due to the progression of their disease despite these interventions.

Ultimately, most of these patients are referred for allogeneic hematopoietic stem cell transplantation (HSCT), which is the only known curative therapy for CAEBV. However, the best approach to control disease prior to HSCT, as well as the optimal conditioning regimen, are unknown.

“For the first time in many years, we provide insight on contemporary treatment options to consider for patients with CAEBV, as well as identifying risk factors for worse outcomes,” said Blachy Dávila Saldaña, M.D., blood and marrow transplant specialist at Children’s National and lead author of the study. “HSCT is curative, but patients need to be considered prior to the evolution of more advanced disease, particularly lymphoma. We also provide a new platform that will inform research on new interventions and therapies for this population.”

“CAEBV remains a challenging disorder to treat, especially once severe complications develop,” said Catherine Bollard, M.D., M.B.Ch.B., director of the Center for Cancer and Immunology Research at Children’s National. “However, our data suggests that T cell modulating therapies may enhance disease control, and future studies should address this question in a controlled setting.”

Future steps also include performing genetic studies to identify those at risk of developing the disease, and developing new platforms for treatment, including checkpoint inhibitors and cytotoxic lymphocyte therapies (CTL’s), which is a form of adoptive immunotherapy that employs virus-specific T cells.

The cohort includes patients treated in CNH and multiple institutions around the world, including Texas Children’s and the National Institutes of Health. “This work was only possible through our collaborative research in anti-EBV cellular therapies,” said Dr. Dávila.

child being bullied

Food allergy-related bullying assessment methods don’t fully capture hurdles

child being bullied

When asked a simple “yes” or “no” question about food allergy-related bullying, 17% of kids said they’d been bullied, teased or harassed about their food allergy. But when asked to reply to a multi-item list of victimization behaviors, that number jumped to 31%.

Living with a food allergy can greatly impact a child’s everyday life – from limiting participation in social activities to being treated differently by peers. While previous research indicates many kids experience food allergy-related bullying, a new study in the Journal of Pediatric Psychology found that offering kids with food allergies a multi-question assessment gives a more accurate picture of the size and scope of the problem.

When asked a simple “yes” or “no” question about food allergy-related bullying, 17% of kids said they’d been bullied, teased or harassed about their food allergy. But when asked to reply to a multi-item list of victimization behaviors, that number jumped to 31%. Furthermore, Children’s National Hospital researchers found that only 12% of parents reported being aware of it.

The reported bullying ranged from verbal teasing or criticism to more overt acts such as an allergen being waved in their face or intentionally put in their food. Researchers say identifying accurate assessment methods for this problem are critical so children can get the help they need.

“Food allergy-related bullying can have a negative impact on a child’s quality of life. By using a more comprehensive assessment, we found that children with food allergies were bullied more than originally reported and parents may be in the dark about it,” says Linda Herbert, Ph.D., director of the Psychosocial Clinical and Research Program in the Division of Allergy and Immunology at Children’s National and one of the study’s researcher.

“The results of this study demonstrate a need for greater food allergy education and awareness of food allergy-related bullying among communities and schools where food allergy-related bullying is most likely to occur,” Herbert adds.

The study looked at food allergy-related bullying among a diverse patient population and evaluated parent-child disagreement and bullying assessment methods. It included 121 children and 121 primary caregivers who completed questionnaires. The children ranged in age from 9 to 15-years-old and were diagnosed by an allergist with at least one of the top eight IgE-mediated food allergies – peanut, tree nut, cow’s milk, egg, wheat, soy, shellfish and fish.

Of the 41 youth who reported food allergy-related bullying:

  • 51% reported experiencing overt physical acts such as an allergen being waved in their face, thrown at them or intentionally put in their food.
  • 66% reported bullying experiences that are categorized as non-physical overt victimization acts including verbal teasing, remarks or criticisms about their allergy and verbal threats or intimidation.
  • Eight reported relational bullying, such as rumors being spread, people speaking behind their back and being intentionally ignored or excluded due to their food allergy.

The researchers also note that food allergy bullying perpetrators included, but were not limited to, classmates and other students, and bullying most commonly occurred at school.

The authors found that only 12% of parents reported that their child had been bullied because of their food allergy and of those, 93% said their child had reported the bullying to them. Some parents reported they had been made fun of or teased themselves because of concerns about their child’s food allergy.

“It’s important to find ways for children to open up about food allergy-related bullying,” Herbert says. “Asking additional specific questions about peer experiences during clinic appointments will hopefully get children and caregivers the help and support they need.”

Research reported in this publication was supported by the National Institute of Allergy and Infectious Diseases, part of the National Institutes of Health, under Award Number K23AI130184 and National Institute on Minority Health and Health Disparities, part of the National Institutes of Health, under Award Number P20MD000198. The content is solely the responsibility of the authors and does not necessarily represent the official views of the National Institutes of Health.

little boy at doctor

Demographic, clinical and biomarker features of MIS-C

little boy at doctor

In a new observational study, researchers provide insight into key features distinguishing MIS-C patients to provide a more realistic picture of the burden of disease in the pediatric population and aid with the early detection of disease and treatment for optimal outcomes.

Multisystem Inflammatory Syndrome in Children (MIS-C) significantly affected more Black and Latino children than white children, with Black children at the highest risk, according to a new observational study of 124 pediatric patients treated at Children’s National Hospital in Washington, D.C. Researchers also found cardiac complications, including systolic myocardial dysfunction and valvular regurgitation, were more common in MIS-C patients who were critically ill. Of the 124 patients, 63 were ultimately diagnosed with MIS-C and were compared with 61 patients deemed controls who presented with similar symptoms but ultimately had an alternative diagnosis.

In the study, published in The Journal of Pediatrics, researchers provide insight into key features distinguishing MIS-C patients to provide a more realistic picture of the burden of disease in the pediatric population and aid with the early detection of disease and treatment for optimal outcomes. The COVID-linked syndrome has affected nearly 4,000 children in the United States in the past year. Early reports showed severe illness, substantial variation in treatment and mortality associated with MIS-C. However, this study demonstrated that with early recognition and standardized treatment, short-term mortality can be nearly eliminated.

“Data like this will be critical for the development of clinical trials around the long-term implications of MIS-C,” says Dr. Roberta DeBiasi, M.D., lead author and chief of the Division of Pediatric Infectious Diseases at Children’s National. “Our study sheds light on the demographic, clinical and biomarker features of this disease, as well as viral load and viral sequencing.”

Of the 63 children with MIS-C, 52% were critically ill, and additional subtypes of MIS-C were identified including those with and without still detectable virus, those with and without features meeting criteria for Kawasaki Disease, and those with and without detectable cardiac abnormalities. While median age (7.25 years) and sex were similar between the MIS-C cohort and control group, Black (46%) and Latino (35%) children were overrepresented in the MIS-C group, especially those who required critical care. Heart complications were also more frequent in children who became critically ill with MIS-C (55% vs. 28%). Findings also showed MIS-C patients demonstrated a distinct cytokine signature, with significantly higher levels of certain cytokines than those of controls. This may help in the understanding of what drives the disease and which potential treatments may be most effective.

In reviewing viral load and antibody biomarkers, researchers found MIS-C cases with detectable virus had a lower viral load than in primary SARS-CoV-2 infection cases, but similar to MIS-C controls who had alternative diagnoses, but who also had detectable virus. A larger proportion of patients with MIS-C had detectable SARS-CoV-2 antibodies than controls. This is consistent with current thinking that MIS-C occurs a few weeks after a primary COVID-19 infection as part of an overzealous immune response.

Viral sequencing was also performed in the MIS-C cohort and compared to cases of primary COVID-19 infection in the Children’s National geographic population. 88% of the samples analyzed fell into the GH clade consistent with the high frequency of the GH clade circulating earlier in the pandemic in the U.S. and Canada, and first observed in France.

“The fact that there were no notable sequencing differences between our MIS-C and primary COVID cohorts suggests that variations in host genetics and/or immune response are more likely primary determinants of how MIS-C presents itself, rather than virus-specific factors,” says Dr. DeBiasi. “As we’ve seen new variants continue to emerge, it will be important to study their effect on the frequency and severity of MIS-C.”

Researchers are still looking for consensus on the most efficacious treatments for MIS-C. In a recent editorial in the New England Journal of Medicine, Dr. DeBiasi calls for well-characterized large prospective cohort studies at single centers, and systematic and long-term follow-up for cardiac and non-cardiac outcomes in children with MIS-C. Data from these studies will be a crucial determinant of the best set of treatment guidelines for immunotherapies to treat MIS-C.

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 surgery, gastroenterology and gastro-intestinal surgery, and urology.

Epinephrine auto-injector for allergy

Assessing daily food allergy self-management among adolescents

Epinephrine auto-injector for allergy

Adolescents reported that epinephrine auto-injectors were frequently available, but least likely to be present outside of the home or school.

Severe food allergic reactions can be life-threatening or fatal and are experienced by up to 40% of children with food allergies, with adolescents at greatest risk. To assess early adolescents’ food allergy self-management, Linda Herbert, Ph.D., and her colleagues at Children’s National Hospital, had 101 adolescents ages 10-14 years complete the Food Allergy Management 24-Hour Recall as an interview.

Adolescents reported that epinephrine auto-injectors were frequently available, but least likely to be present outside of the home or school. Adolescents also relied on past experience with food to determine safety, which is not a recommended strategy. Appropriate assessment of food safety and problem-solving involving how to keep epinephrine auto-injectors with adolescents outside the home should be primary intervention targets.

Study authors from Children’s National include: Linda Herbert, Ph.D., Ashley Ramos, Ph.D., Frances Cooke, Kaushalendra Amatya, Ph.D., and Hemant Sharma, M.D., M.H.S.

Read the full study in the Annals of Allergy, Asthma, and Immunology.

piece of bread with question mark cut out

The Food Allergy Parent Mentoring Program: A pilot intervention

piece of bread with question mark cut out

Parents of young children with newly diagnosed food allergy are at risk for poor psychosocial outcomes due to food allergy’s life-threatening nature and demanding management routines. Presently, there are no interventions to support food allergy parents during this adjustment phase.

Ashley Ramos, Ph.D., and colleagues at Children’s National Hospital conducted a pilot study to explore the feasibility, acceptability and preliminary efficacy of a novel intervention using peer mentorship to improve psychosocial functioning in parents of young children with newly diagnosed food allergy. Parent mentors were trained in mentorship and matched with a mentee, a parent of a child under the age of 5 years with newly diagnosed food allergy, for a 6-month intervention period.

Their findings indicate the use of a peer mentorship program to support parents of children with newly diagnosed food allergy is feasible and helpful. It may be appropriate to develop and implement such programs in allergy clinics.

Study authors from Children’s National include: Ashley Ramos, Ph.D., Frances Cooke, Emily Miller and Linda Herbert, Ph.D.

Read the full study in the Journal of Pediatric Psychology.

girl with asthma inhaler

Children’s National becomes part of CAUSE Network

girl with asthma inhaler

Seven clinical sites in six different cities will join forces to perform mechanistic and translational studies examining the basic immunology of pediatric asthma among urban, under-resourced and largely minority children and adolescents.

The National Institute of Allergy and Infectious Diseases (NIAID) allocated $10 million in funding to establish the Childhood Asthma in the Urban Setting (CAUSE) network. The NIAID plans to increase this number by $70 million over seven years to support the network. Children’s National Hospital will be part of the new research network, which is a 7-year consortium comprising of seven clinical sites in six different cities that will join forces to perform mechanistic and translational studies examining the basic immunology of pediatric asthma among urban, under-resourced and largely minority children and adolescents.

Children’s National is the home of Improving Pediatric Asthma Care in the District of Columbia (IMPACT DC). The program focuses on research, care and advocacy to decrease asthma morbidity experienced by at-risk youth in the region while serving as a model program for the nation. NIAID gave an initial $3 million to IMPACT DC to conduct its own pilot study of anti-IgE therapy to prevent asthma exacerbations. Additional support for this and other studies will come from subcontracts from the CAUSE Coordinating Center at the University of Wisconsin in Madison.

“This new award allows IMPACT DC to remain part of one of the nation’s most prestigious pediatric asthma research consortia,” said Stephen Teach, M.D, M.P.H., chair for the Department of Pediatrics at George Washington University School of Medicine and Health Sciences. “It will allow us to both pursue an independent research agenda while collaborating with similar academic centers nationwide.”

Pediatric asthma is the most common chronic disease in children, and it is estimated that about 6.1 million children under 18 years suffer from this condition. It disproportionately affects urban, minority and under-resourced children and adolescents.

“It is essential to develop an understanding of the basic immunology of the disease and therapeutic options to ameliorating these disparities,” said Dr. Teach.

CAUSE researchers will explore the mechanisms of immune tolerance to allergens, the role of early environmental exposures in the pathogenesis of asthma, the pathogenesis and mechanisms of non-atopic asthma, the role of the respiratory epithelium in asthma and more.

The CAUSE network comprises of seven clinical research centers, including Children’s National led by principal investigator, Dr. Teach, and the following research centers:

  • Boston Children’s Hospital. Principal investigators: Wanda Phipatanakul, M.D., and Talal Chatila, M.D.
  • Cincinnati Children’s Hospital Medical Center. Principal investigator: Gurjit Khurana Hershey, M.D., Ph.D.
  • Columbia University Health Sciences, New York. Principal investigator: Meyer Kattan, M.D.
  • Icahn School of Medicine at Mount Sinai, New York. Principal investigators: Paula Busse, M.D., Supinda Bunyavanich, M.D., and Juan Wisnivesky, M.D.
  • Lurie Children’s Hospital of Chicago. Principal investigators: Rajesh Kumar, M.D., and Jacqueline Pongracic, M.D.
  • University of Colorado Denver. Principal investigator: Andrew Liu, M.D.
t-cells attacking cancer cell

Children’s National spin-out cell therapy company receives funding

t-cells attacking cancer cell

Ongoing efforts by researchers at Children’s National Hospital to improve T-cell therapies have led to a spin-out company MANA Therapeutics which has announced a $35 million Series A financing. MANA is a clinical stage company creating nonengineered, allogeneic and off-the-shelf cell therapies that target multiple cancer antigens. Its EDIFY™ platform aims to educate T-cells that target multiple target multiple cell surface and intracellular tumor-associated antigens across a broad range of liquid and solid tumors, with an initial focus on relapsed acute myeloid leukemia (AML).

MANA was founded in 2017, and was based on the research and human proof-of-concept clinical trials conducted by Catherine Bollard, M.D., M.B.Ch.B., Conrad Russell Y. Cruz, M.D., Ph.D., Patrick Hanley, Ph.D. and other investigators at Children’s National along with their colleagues at Johns Hopkins Medical Center. The trials demonstrated safety and anti-tumor activity of MANA’s approach, and Children’s National provided an exclusive license to MANA to further develop this promising technology into commercial products in the field of immuno-oncology.

MANA Therapeutics recruited an experienced leadership team from industry including Martin B. Silverstein, M.D., president and CEO, who is a former senior executive at Gilead Sciences when they acquired Kite Pharma, one of the leading cell therapy companies, as well as Madhusudan V. Peshwa, Ph.D., chief technology officer, who joined from GE Health Care where he had been Chief Technology Officer and Global Head of R&D for Cell and Gene Therapies.

“MANA is building upon the strong foundational science established at Children’s National with a unique approach that promises to produce off-the-shelf allogeneic therapies that do not compromise on safety or efficacy,” said Marc Cohen, co-founder and executive chairman of MANA Therapeutics. “I look forward to continuing to support the MANA team as they advance their internal pipeline for the treatment of AML and select solid tumors, and expand the potential of EDIFY through strategic partnerships focused on new target antigens and cancer types.”

An international leader in the immunotherapy field, Dr. Bollard was an early believer in the potential of immune cell therapies to dramatically improve the treatment of patients with cancer and patients with life-threatening viral infections. Recently, she and her team at the Children’s National Center for Cancer and Immunology Research published findings in Blood showing T-cells taken from the blood of people who recovered from a COVID-19 infection can be successfully multiplied in the lab and maintain the ability to effectively target proteins that are key to the virus’s function.

“Over the past decade we have seen tremendous progress in cancer research and treatment and are beginning to unlock the potential of cell therapy for a variety of tumor types,” said Dr. Bollard. “The human proof-of-concept trials conducted by my team and colleagues showed potential for a nonengineered approach to educating T-cells to attack multiple tumor antigens, which MANA is expanding even further through refinement of the manufacturing process for an allogeneic product and application to a broader set of antigens in a variety of clinical indications and settings.”

common food allergens

Psychotherapeutic treatment for psychosocial concerns related to food allergy

common food allergens

Pediatric food allergy is a growing public health concern, with 8 percent of children in the United States affected. Although new treatments for food allergies are being developed, the vast majority of cases are currently managed by daily evaluation of food safety and vigilance for accidental allergen exposure and allergic reactions. This often impacts patients’ and caregivers’ quality of life and overall psychosocial functioning.

In a recent article published in the Journal of Allergy and Clinical Immunology: In Practice, Linda Herbert, Ph.D., and Audrey DunnGalvin, Ph.D., provide a review of mental health concerns related to food allergy. The authors present two cases in which patients received psychological services for food allergy-related anxiety. For both cases, treatment resulted in decreased anxiety and improved food allergy management/oral immunotherapy treatment engagement.

The authors also discuss unmet food allergy-related psychosocial needs, including the lack of food allergy-specific anxiety measures, psychosocial domains that warrant investigation, development of supportive interventions for patients engaging in allergen immunotherapy and the lack of adequate mental health providers with food allergy expertise.

Read the full article in the Journal of Allergy and Clinical Immunology: In Practice.

foods that cause allergies

Patients’ perspectives and needs on novel food allergy treatments

foods that cause allergies

Food allergy is a major public health concern in many countries around the world. In the United States, studies suggest that it affects up to 8% of children and 10% of adults and is responsible for an emergency room admission every three minutes. Historically, the only treatment for food allergy has been complete allergen avoidance combined with rescue medications when accidental exposures occur. Fortunately, advances in food allergy research over the past decade have yielded new treatments, but with these new treatments come new stressors.

In a recent study published in the journal Current Treatment Options in Allergy, Linda Herbert, Ph.D., and colleagues provide an overview of the current state of the literature regarding patients’ and caregivers’ food allergy experiences and needs within the United States. The authors also put forth a set of recommendations regarding how best to proceed with patient-centered development and evaluation of new food allergy treatments.

Read the full study in Current Treatment Options in Allergy.

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.