Cancer Archives

Advancing early detection for kidney disease in sickle cell

February 4, 2026

Kidney disease is a leading cause of mortality in sickle cell disease, underscoring the urgent need for earlier and more precise diagnostics.

A new multi-institutional study, supported by a grant from the Commonwealth Health Research Board, brings together Children’s National Hospital, George Mason University and Old Dominion University to advance a novel, non-invasive approach to kidney disease detection in patients with sickle cell disease (SCD). Leveraging the Comprehensive Sickle Cell Disease Program at Children’s National, one of the largest in the nation and among the few offering gene therapy, the research integrates longitudinal patient data with advanced proteomic analysis.

Addressing a critical clinical need

Kidney disease is a leading cause of mortality in SCD, underscoring the urgent need for earlier and more precise diagnostics.

“We’ve watched as silent kidney damage progresses year after year, often going undetected until significant harm has occurred,” said Andrew Campbell, MD, director of the Comprehensive Sickle Cell Disease Program at Children’s National and co-investigator of the study. “By identifying kidney disease earlier, we can intervene sooner with more effective disease-modifying therapies and curative treatments, potentially preventing or slowing progression to kidney failure.”

From discovery to impact

“We measure damaged mitochondria shed by the kidney in a few milliliters of urine so we can develop an early warning system for the onset of kidney damage,” said Marissa Howard, PhD, systems biologist and research fellow at George Mason University and principal investigator of the study. “Our partners at Children’s National are known for identifying innovative approaches to treat SCD. This exciting collaboration is collectively motivated by our ultimate goal of reducing the high mortality rate caused by Sickle Cell kidney failure in children affected by this disease.”

By translating cutting-edge science into clinical impact, Children’s National continues to advance next-generation diagnostics that improve outcomes for children with SCD and may extend to other diseases linked to mitochondrial dysfunction.

U.S. News & World Report voting

February 3, 2026

Children’s National is ranked one of the top 10 pediatric hospitals in the nation by U.S. News & World Report. Our faculty and staff are proud of the impact made on the lives of children and families in our community. Your participation in the U.S. News & World Report annual reputational survey validates the quality of care we provide and reflects the mutual respect and trust we share as healthcare professionals.

How to determine your voting eligibility

Voting for the U.S. News & World Report Best Children’s Hospitals rankings can be done only through Doximity.

To participate, physicians must:

Be board-certified and meet the eligibility criteria for the voting categories.
For child and adolescent psychologists, your account must be up to date with your specialty and subspecialty correctly marked.
Be a credential-verified member of Doximity (you must have an active and claimed Doximity profile).
Have all certifications and board documents currently up-to-date in your Doximity profile.

View the full eligibility criteria

How to claim your Doximity profile to vote

You have to claim your profile on Doximity.com to participate in the online survey. If you have not yet claimed your Doximity profile, go to Doximity.com, and click “Find My Profile.”
Once your profile has been claimed, you must confirm your email address and board certifications.
Verified Doximity members will receive an email inviting them to participate in the U.S. News survey.
For more information on how to claim your profile, visit Doximity.com

How to update and verify existing Doximity account information

Your Doximity profile must have up-to-date licenses, certifications and board documents.

Once you are logged in, your profile will automatically be in “Edit Mode.” You are able to add new items or edit existing information.
Update your Doximity profile and ensure your information is current.

Once registered, users wishing to participate in the online survey should:

Watch for an email from Doximity about the annual member survey.
Even if you don’t see the email, if you are a registered Doximity user, you can still vote by logging in to Doximity.com with your username and password during the voting period.
Once logged in, look for a U.S. News graphic or button on the homepage and click on it.
The survey asks users to name the hospitals that provide the best care in your respective specialty, without consideration to location or cost. Pediatric specialists will list 10 hospitals. The order in which you list the hospitals does not matter.

Please note: Children’s National Hospital is listed as “Children’s National Hospital Washington, DC” on the survey.

Visit Doximity’s FAQs if you have issues or questions about registration or claiming your profile.

How to cast your vote

In February 2026 when voting opens, all survey-eligible physicians will receive a notification on the Doximity app for Android or iOS. If you do not use the Doximity app, you will receive an email when voting opens.

Log in to your Doximity account at doximity.com or via the mobile app.
Click the Notifications icon or tap the “Submit your Nominations” button on the homepage. You can also search for “U.S. News Best Hospitals”
Select 10 hospitals in your respective specialty that you believe provide the best care in the United States.
Submit your vote

Having technical issues?

If you have difficulty registering with Doximity or completing the survey, please visit Doximity support for assistance.

Vote

The 2026 U.S. News & World Report Best Children’s Hospitals reputation voting will open in mid-February. Look for your Doximity notification to vote.

Cast your vote

In the News: Groundbreaking gene therapy for sickle cell disease

January 15, 2026

A recent NBC News story follows Wedam Minyila, a teenager living with sickle cell disease, as he undergoes a groundbreaking gene-editing treatment at Children’s National Hospital that could transform care for patients with the condition. A year ago, NBC News joined Wedam at the start of the intensive process, which included the removal of his stem cells for revolutionary gene editing and rounds of chemotherapy.

Now, NBC News’ Yamiche Alcindor checks in with Wedam and his family and to learn how he’s doing, what challenges remain and what the road ahead may hold – a journey that doctors at Children’s National hope could lead to a medical breakthrough for sickle cell patients around the world.

“He’s not needing transfusion support because his genetically modified bone marrow is now making his red blood cells,” said Henna Butt, MD, bone marrow and transplantation fellow at Children’s National. Watch the video on NBC News.

Integrating mental health into pediatric care

January 12, 2026

Explore how integrated pediatric mental health care is transforming neuro-oncology at Children’s national Hospital.

In the News: Why this clinical trial is offering some young cancer patients hope

January 9, 2026

For decades, children with solid tumors have had few new treatment options, even as cancer care has advanced dramatically for adults. Now, Children’s National Hospital is launching a first-of-its-kind clinical trial designed to tackle one of the biggest challenges in pediatric cancer: tumors that adapt quickly and evade single-target therapies. The effort was recently highlighted in The Washington Post, underscoring both the scientific challenge and the urgency facing families.

Led by Catherine Bollard, MD, MBChB, the trial uses each child’s own T cells, engineered to attack two tumor targets at the same time. “Cancers are really clever,” Dr. Bollard explained. “And one of the ways they get clever is they get rid of one of their targets.” If the cancer cells detect that a treatment like T cells is focusing on one target, she said, “then the cancer cell can just go, ‘Okay, I’m going to get rid of that target.’ But because our product is targeting two, it’s much harder for the tumor cell to catch up and delete them both.”

The study is part of the global Cancer Grand Challenges initiative and reflects years of work to move an idea from the lab to the clinic. While the trial is intentionally small, researchers say even early signs of success could help shift the field and offer new hope to children and families facing cancers where progress has been far too slow.

The best of 2025 from Innovation District

December 24, 2025

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

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

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

2. Pioneering evidence-based treatments for substance addictions

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

3. Breaking barriers in growth disorder treatment for families

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

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

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

5. Therapy approach shows promise for PANS/PANDAS

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

6. Wearable tech data shows promise in ADHD detection

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

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

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

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

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

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

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

10. Fighting food insecurity with fresh produce and education

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

Children’s National in the News: 2025

December 23, 2025

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

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

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

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

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

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

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

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

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

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

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

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

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

7. Jayden Daniels surprises Children’s National patient

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

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

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

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

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

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

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

Grant funds next-generation immunotherapy trial for DMG

December 15, 2025

Dr. Hwang’s research focuses on advancing novel immunotherapies for pediatric brain tumors.

Gene Hwang, MD, chief of Oncology at Children’s National Hospital, received a research grant from The Cure Starts Now Foundation to support a clinical trial targeting diffuse midline glioma (DMG), one of the most aggressive pediatric brain tumors.

The grant will fund collaborative work led by Dr. Hwang, in partnership with Elias Sayour, MD, PhD, at the University of Florida, to advance an innovative mRNA vaccine approach using “onion-wrapping” multilamellar nanoparticles. This technology, initially supported by a 2024 Cure Starts Now generational grant, has shown promising results, including curing cancer in preclinical models. The new award supports translating these findings into a clinical trial designed to evaluate safety, immune response and early therapeutic impact in children with DMG.

Dr. Hwang’s research focuses on advancing novel immunotherapies for pediatric brain tumors. The trial represents a major step toward expanding treatment options for children facing this devastating disease.

“This support accelerates our team’s ability to translate groundbreaking science into a potential treatment for children with DMG as fast as possible,” said Dr. Hwang. “Our collaboration with Dr. Sayour brings together unique expertise from multiple leading institutions, and together, we’re hopeful that this next-generation immunotherapy approach will open new doors for patients who currently have very few.”

Podcast: Whole Picture: Integrating Mental Health into Pediatric Care

December 10, 2025

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

December 2, 2025

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

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

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

The big picture

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

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

Why it matters

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

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

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

Moving the field forward

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

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

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

New insights into genetics of childhood brain and spinal tumors

November 25, 2025

New insights look into the genetics of childhood brain and spinal tumors.

Researchers at Children’s National Hospital in Washington, D.C. and Children’s Hospital of Philadelphia (CHOP), are advancing understanding of how rare inherited genetic variants contribute to the development of brain and spinal cord tumors in children. The new findings, published in Nature Communications, reveal how a child’s genetic makeup can shape cancer risk, tumor behavior, and potentially long-term outcomes.

“We are currently expanding our study to include parental sequencing and more than doubling the number of patients,” said Jo Lynne Rokita, PhD, co-senior study author and principal investigator at the Brain Tumor Institute and Center for Cancer and Immunology Research of Children’s National. “This will help us better understand how inherited or early arising genetic changes interact with those that develop in the tumor, ultimately improving how we diagnose, monitor and treat children with brain and spinal cord cancers.”

Cancers of the central nervous system (CNS) are the leading cause of cancer-related deaths in children, with more than 47,000 children and young adults diagnosed each year. Up to one in four children with cancer carry rare genetic variants that increase susceptibility, yet the mechanisms of inherited drivers of pediatric CNS tumors have remained poorly understood.

In this study, investigators from Children’s National, CHOP, and collaborating partners analyzed blood and tumor samples from 830 children with brain and spinal cord tumors using data from the Pediatric Brain Tumor Atlas. They looked for genetic changes present from birth — either inherited or newly occurring — that may predispose children to these cancers and compared those findings with each child’s medical history to identify known or previously unrecognized tumor predisposition syndromes.

“Our research provides a foundation for identifying patients whose genetic profiles influence both cancer susceptibility and how their disease behaves — in some cases signaling a higher risk for aggressive tumors, and in others, a better chance of survival,” said Sharon J. Diskin, PhD, senior author of the study and principal investigator at CHOP’s Center for Childhood Cancer Research. “By understanding these risk factors and patterns, we move closer to developing more personalized, effective care for children with CNS tumors.”

Researchers examined how pathogenic (P) and likely pathogenic (LP) germline variants influence tumor risk and clinical outcomes. Pathogenic variants are known to increase disease risk, while likely pathogenic variants are suspected but lack definitive evidence. Nearly one in four children (23.3%) carried a genetic change in a cancer-risk gene. Seven percent (57 children) had already been diagnosed with a genetic condition associated with tumor development, while another 6% (48 children) harbored cancer risk variants that had not yet been clinically recognized. These results underscore the need for more comprehensive genetic screening in pediatric CNS cancers, as many inherited or early-arising risks continue to go undetected.

The study also found that 35% of children with these variants had additional alterations in the same genes within their tumors, supporting the “two-hit” model of cancer development, where an inherited genetic change is followed by a second, tumor-specific event that drives cancer growth.

This work was supported in part by grants from the National Institutes of Health (NIH), the NIH Kids First Cloud Credits Program, the Children’s Brain Tumor Network, the Chad Tough Foundation and private donors to the Children’s National Hospital Brain Tumor Institute.

Learn more here.

Attendees at the 3rd Annual Cell and Gene Therapy Symposium

A regional engine for cell and gene therapy innovation

October 29, 2025

At the 3rd Annual Cell and Gene Therapy Symposium, leaders from research, biotech, and advocacy came together at the Children’s National Research & Innovation Campus to accelerate progress for children with rare and life-threatening diseases.

The 3rd Annual Cell and Gene Therapy Symposium gathered leaders in research, biotech, advocacy and policy at the Children’s National Research and Innovation Campus, reinforcing the Mid Atlantic as a rapidly rising hub for curing rare and life-threatening diseases. This event focused not only on scientific discovery, but also on how this region is transforming ideas into real impact for children and families.

Patrick Hanley, PhD, Chief and Director of the Cellular Therapy Program at Children’s National Hospital, opened the program by acknowledging both the excitement and responsibility that come with treating children who urgently need new options.

The first keynote from Sadik Kassim, PhD, Chief Technology Officer at Danaher Corporation, responsible for the development and implementation of research and development strategies for genomic medicines, highlighted how quickly the world of cell and gene therapy is evolving and how essential it is that progress reaches patients in time.

Regional innovation at scale

Early presentations featured experts from institutions including the University of Virginia, Children’s National Hospital, Johns Hopkins Medicine and area startups. These talks demonstrated real momentum across regenerative medicine, pediatric immunotherapy and translational biotechnology, showing how research teams here focus relentlessly on advancing from concept to clinic. The message was simple. Innovation here does not stop at publication. It continues until those in need feel the impact.

Speakers including Camille Campbell, PharmD, RPh of Bella Peek LLC, Sneha Purvey, MD of VCU Health, and Brett Kopelan, MA of the Debra Foundation described the challenges families face in accessing advanced therapies. They focused on how outcomes for rare disease communities led to multiple drug approvals from the Food and Drug Administration (FDA) and stressed that equity must evolve alongside innovation. A breakthrough does not matter if a child cannot receive it.

The power of partnership

After lunch, moderator Mike Friedlander, PhD, Executive Director of the Fralin Biomedical Research Institute at Virginia Tech, led a panel featuring Catherine Bollard, MBChB, MD of Children’s National Hospital, Erica Cischke, MPH of the Alliance for Regenerative Medicine, Erin Kimbrel, PhD of Astellas Pharma, and Mark Stewart, PhD of Friends of Cancer Research. Their discussion underscored the essential roles of advocacy, regulation and industry alignment in shaping the future of care for children and adults.

A vision for a smarter biotech future

The innovation keynote from Murat Kalayoglu, MD, PhD, Managing Partner of SOAR Bio and co-founder of local biotech company Cartesian Therapeutics which has a CAR T product in a phase 3 trial for an autoimmune disease, offered a direct challenge to the field. He presented a vision where biotechnology companies are built more efficiently and with stronger operational roots to accelerate translation and sustainability. He noted that the Mid Atlantic region is well positioned to lead this approach because of its workforce, institutional partnerships, and growing investment footprint.

Science changing what is possible

The afternoon sessions explored advances that could fundamentally change the treatment landscape for children with complex and rare diseases by using precise treatment modalities.

Fred Wu, MD, PhD, Assistant Professor at the Fralin Biomedical Research Institute and Radiation Oncologist at Inova, presented focused ultrasound technology designed to temporarily open the blood brain barrier and enable therapeutics to reach tumors including diffuse midline glioma.
Kajal Chaudhry, PhD, of Children’s National Hospital, shared progress in multimodal T cell therapies that combine multiple immune mechanisms to overcome tumor defenses and strengthen responses in high-risk pediatric cancers. This work is a part of a large Cancer Grand Challenges award.
Sarah Nasr, PhD, from the Fischell Department of Bioengineering at the University of Maryland, discussed lung inspired RNA delivery strategies that could expand treatment potential for pulmonary disease.
Zachary Zamore, MD Candidate at Johns Hopkins School of Medicine, presented targeted gene therapy for nerve disorders to reduce chronic pain and spasticity without invasive neurosurgery.
Chelsea Stamm, PhD, of the Bacteriophage Medical Research Center at Catholic University of America, won the first-place prize for best presentation by an early stage investigator and shared new bacteriophage vector platforms capable of delivering large genetic cargo into stem cells.
Abby Lee, PhD Candidate at the George Washington University Cancer Center, showed research improving immune responses against ovarian cancer through novel T cell expansion strategies.
Emily Powsner, PhD Candidate in Biotherapeutic Development and Delivery at the University of Maryland, demonstrated scalable manufacturing of extracellular vesicles designed to support healing in chronic wounds where current options fall short.

Across each talk, the focus stayed consistent. These therapies are being advanced not for the future, but for children now.

Momentum moving forward

The event closed with remarks from Dr. Hanley, followed by a reception where new partnerships formed and plans for the next studies and trials began taking shape in real time. The optimism was grounded in data. The urgency was grounded in the needs of families.

This region is proving that when researchers, clinicians and advocates align their priorities, the pace of progress can accelerate. The Mid Atlantic is not simply part of the conversation. It is leading the way. Children’s National Hospital and its partners across Virginia, Maryland, Delaware and Washington, DC, are demonstrating how to build systems where breakthroughs move faster, reach farther and change more lives.

OpenPedCan expands access to pediatric cancer data for researchers worldwide

October 23, 2025

The OpenPedCan Project harmonizes genomic and clinical data from over 6,000 pediatric tumors across 100 cancer types, creating an open-access resource to advance collaboration and accelerate childhood cancer research.

Modern cancer breakthroughs depend on collaboration and open data. The Open Pediatric Cancer (OpenPedCan) Project, published last month in GigaScience, is opening new doors for discovery by harmonizing genomic and molecular data from more than 6,000 tumors across 100 types of cancer.

Led by teams at Children’s National Hospital and Children’s Hospital of Philadelphia, OpenPedCan creates one of the largest harmonized open-source pediatric cancer datasets ever assembled.

Why it matters

Pediatric cancers are rare and complex, which makes collaboration essential. No single institution can collect enough samples to reveal the full picture of how these diseases develop or respond to treatment.

OpenPedCan changes that. The project combines whole-genome or exome sequencing, RNA sequencing, methylation profiling, proteomics and clinical data from multiple international cohorts into a unified, open platform. Researchers can now explore harmonized data covering nearly 48,000 total biospecimens — including tumor and normal tissues — to identify patterns, compare findings and accelerate the search for new treatments.

What’s new

OpenPedCan builds on the success of The Open Pediatric Brain Tumor Atlas (OpenPBTA), which characterized more than 1,000 pediatric brain tumors. This new release expands beyond brain cancers to include a broad range of childhood solid tumors and leukemias.

It also introduces molecular subtyping that aligns with the World Health Organization’s (WHO) 2021 classification of central nervous system tumors, along with new layers of data such as proteomics, phospho-proteomics and microRNA sequencing. Together, these additions give scientists a deeper, multiomic view of pediatric cancers than ever before.

The big picture

“OpenPedCan puts the power of harmonized data into the hands of every lab,” said Jo Lynne Rokita, PhD, of Children’s National, senior author of the study.

“OpenPedCan puts the power of harmonized data into the hands of every lab,” said Jo Lynne Rokita, PhD, of Children’s National, senior author of the study. “By aligning previously un-subtyped tumors with WHO 2021 classifications and integrating new layers like proteomics and methylation, we are helping researchers worldwide find answers faster and bring new hope to children and families.”

Making these datasets and tools openly available to anyone reduces barriers to entry and encourages collaboration across institutions and disciplines. The data has already been used to update initial diagnoses, identify potential therapeutic targets and strengthen clinical trial design.

OpenPedCan helps redefine what collaboration looks like in pediatric cancer research. By connecting genomic data, analytical tools and global expertise, the project gives scientists a faster path to discovery — and gives children with cancer more reasons for hope.

You can read the full journal article “The Open Pediatric Cancer Project” in GigaScience here. This project was funded in part with federal funds from the National Cancer Institute, National Institutes of Health (NIH), under Contract No. 75N91019D00024, Task Order No. 75N91020F00003 and the U24OD038422 from the Office of the Director at NIH.

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

October 7, 2025

Children’s National Hospital in Washington, D.C., was ranked as a top hospital in the nation by the U.S. News & World Report 2025-26 Best Children’s Hospitals annual rankings. This marks the ninth straight year Children’s National has made the Honor Roll list. The Honor Roll is a distinction awarded to only 10 children’s hospitals nationwide.

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

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

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

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

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

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

Neurology and Neurosurgery (No. 4), led by Division Chiefs William D. Gaillard, MD, and Robert F. Keating, MD
Diabetes and Endocrinology (No. 6), led by Division Chief Andrew Dauber, MD, MMSc
Cancer (No. 8), led by Division Chief Eugene Hwang, MD
Nephrology (No. 10), led by Division Chief Marva Moxey-Mims, MD
Orthopedics (No. 10), led by Division Chief Matthew Oetgen, MD, MBA

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

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September 24, 2025

Advancements in treating rare pediatric kidney cancers

September 11, 2025

Clear Cell Sarcoma of the Kidney overall survival by stage.

Overall survival by stage in Clear Cell Sarcoma of the Kidney (CCSK). Stage I/II patients demonstrate excellent outcomes, while stage IV shows meaningful improvement compared with historical cohorts, underscoring the need for continued therapeutic advances in high-risk disease.

Rare kidney cancers account for a quarter of pediatric kidney tumors and pose unique challenges for oncologists. In the past year, Jeffrey Dome, MD, PhD, and colleagues in the Children’s Oncology Group published three landmark studies that are reshaping treatment for focal anaplastic Wilms tumor (FAWT), clear cell sarcoma of the kidney (CCSK), and malignant rhabdoid tumor (RT).

Focal anaplastic Wilms tumor

Recent findings show that children with stage I–II FAWT achieve 100% 4-year overall survival with vincristine/dactinomycin/doxorubicin chemotherapy (DD4A) plus flank radiation, while children with stage IV disease benefited from intensified regimens. These results confirm that therapy can be adjusted to disease stage, balancing survival with long-term side effects.

Clear cell sarcoma of the kidney

For CCSK, new evidence supports omitting radiation in stage I patients when lymph nodes are sampled, reducing treatment burden without sacrificing outcomes. Standard therapy consisting of vincristine/doxorubicin/cyclophosphamide/etoposide (Regimen I) plus flank radiation remains effective for stage II–III disease. For stage IV disease, addition of carboplatin to the standard regimen improves survival, although outcomes remain unsatisfactory, with the brain as the most common site of recurrence.

Malignant rhabdoid tumor

Rhabdoid tumors remain the most aggressive pediatric kidney cancer. Intensified chemotherapy has modestly improved survival, a result driven by patients with Stage I/II disease. Despite this improvement, outcomes for patients with rhabdoid tumor remain unsatisfactory and there is a need for novel therapeutic strategies, such as targeting SMARCB1 gene alterations, and immunotherapy approaches.

Why it matters

Families: Some children can avoid radiation or prolonged chemotherapy, reducing long-term side effects.
Clinicians: Clear guidance on when therapy can be reduced – or intensified.
Researchers: New targets like SMARCB1 highlight the need for precision medicine in rhabdoid tumor, where intensification of chemotherapy has had only modest efficacy

Looking ahead

“These studies show real progress in tailoring treatment,” said Dr. Dome. “But the next leap forward will come from biological therapies that directly target the drivers of these cancers.”

With no active frontline trials currently open, the Children’s Oncology Group is designing the next generation of studies to deliver more effective and less toxic therapies for these rare cancers.

Conclusion

From reducing radiation in CCSK to improving survival in FAWT and setting the stage for precision medicine in rhabdoid tumors, these advances are reshaping the outlook for children with rare kidney cancers. At Children’s National, leaders like Dr. Dome are ensuring that even the rarest diagnoses are met with innovation and hope.

Read the full studies here: focal anaplastic Wilms tumor (FAWT), clear cell sarcoma of the kidney (CCSK), and malignant rhabdoid tumor (RT).

Successful leiomyoma removal with STER shows promise for future cases

August 19, 2025

The procedure was successfully performed by team in the Esophageal and Thoracic Surgery Program at Children’s National, including Timothy Kane, MD, and Mikael Petrosyan, MD, MBA, who co-authored a case study.

Pediatric surgeons at Children’s National Hospital successfully removed a pediatric esophageal leiomyoma, a rare, benign tumor of the esophagus, through minimally invasive submucosal tunneling and endoscopic resection (STER) rather than the standard of care endoscopic submucosal dissection (ESD) technique that has traditionally had higher complication rates.

The procedure was successfully performed by team in the Esophageal and Thoracic Surgery Program at Children’s National, including Mikael Petrosyan, MD, MBA, and Timothy Kane, MD, who co-authored a case study, Submucosal Tunneling and Endoscopic Resection of an Esophageal Leiomyoma in a 13‑Year‑Old Male, in the Annals of Surgical Oncology about the case.

What it means

A 13-year-old male presented with dysphagia (difficulty swallowing) and frequent abdominal pain and was diagnosed with a 2-cm distal esophageal mass. The team completed full resection of the mass with no complications. At 1-year post-surgery, the patient continued to show no signs of dysphagia, gastroesophageal reflux, or chest pain.

In the case study, the authors write that the successful application of STER “represents a valuable advancement in the endoscopic management of submucosal tumors (SMTs), offering a less invasive and potentially safer approach for tumor removal.”

They assert that while ESD, the current most common approach, also involves removing lesions endoscopically, “ESD involves direct dissection of the lesion, potentially leading to larger mucosal defects and increased risk of complications. STER, with its tunnel approach, maintains mucosal integrity, potentially leading to better outcomes.”

Children’s National leads the way

The Esophageal and Thoracic Surgery Program at Children’s National is a national leader in pioneering more precise and less invasive procedures for children who have diseases and disorders of the lungs, mediastinum and esophagus.

For example, Children’s National performs more peroral endoscopic myotomy (POEM) procedures as a primary intervention for children with esophageal achalasia and pediatric gastroparesis than any other hospital in the United States.

Minimally invasive procedures for children with these conditions are particularly challenging due to a child’s small anatomy and a lack of right-sized surgical tools. The application of endoscopic procedures such as STER and POEM offer fewer risks of complications and faster recovery times than the current standard approaches for children. Though many of these procedures are readily available for adults, applying them in pediatrics requires technical skill and expertise not available everywhere.

Read the case study, “Submucosal Tunneling and Endoscopic Resection of an Esophageal Leiomyoma in a 13‑Year‑Old Male, in the Annals of Surgical Oncology.

Dr. Jeffrey Dome poses with books he authored

Textbook contributions that shape pediatric cancer care

August 8, 2025

Dr. Dome has authored more than 200 articles, books, letters and chapters on pediatric cancer.

Jeffrey Dome, MD, PhD, senior vice president of the Cancer and Blood Disorders Center at Children’s National Hospital, recently authored chapters for Pizzo and Poplack’s Pediatric Oncology and Abeloff’s Clinical Oncology. These are just two of the internationally recognized oncology textbooks for which Dr. Dome has been invited to serve as senior author, with a focus on Wilms tumor and other pediatric solid tumors.

Contributions to medical textbooks are one of the important avenues for leading oncologists to share their expertise and knowledge with other specialists and medical students.

“Being invited to contribute to these textbooks is both an honor and a responsibility, it’s one way I can give back to the field and help prepare the next generation of physicians,” says Dr. Dome. “I like to tell people that at Children’s National, we don’t just follow the recipes for treatment. We write the cookbooks, or in this case, the textbooks.”

About Dr. Dome

Jeffrey Dome, MD, PhD, is senior vice president of the Cancer and Blood Disorders Center at Children’s National and professor of pediatrics at the George Washington University School of Medicine and Health Sciences. He holds the Thomas Willson and Lenore Williams McKnew professorship of pediatric oncology.

Dr. Dome is an internationally recognized expert on pediatric solid tumors, with an emphasis on kidney tumors and sarcomas. He chaired the Children’s Oncology Group (COG) Renal Tumor Committee for more than a decade. He is presently on the Scientific Council of the COG and is past chair of the American Board of Pediatrics Subboard of Hematology/Oncology. Dr. Dome serves on numerous medical advisory boards for cancer foundations and currently is the North American Continental President of the International Society of Pediatric Oncology (SIOP). He has authored more than 200 articles, books, letters and chapters on pediatric cancer.

Other textbook contributions authored by Dr. Dome include

Lanzkowsky’s Manual of Pediatric Hematology and Oncology
Gunderson & Tepper Clinical Radiation Oncology
Cassidy and Allanson’s Management of Genetic Syndromes
Nathan and Oski’s Hematology and Oncology of Infancy and Childhood
Rudolph’s Pediatrics
Raghavan’s Textbook of Uncommon Cancer, 5^th Edition
Malkin’s Hereditary Basis of Childhood Cancer

Insights into paclitaxel-containing therapy for recurrent Wilms tumor

July 28, 2025

Paclitaxel-containing regimens for patients with recurrent Wilms tumor show promising results in a new multi-institutional retrospective study published in Pediatric Blood and Cancer.

“These findings provide an additional treatment option for pediatric oncologists caring for patients with recurrent Wilms tumor and merit prospective evaluations of paclitaxel-based regimens,” says Jeffrey Dome, MD, PhD, senior vice president of the Cancer and Blood Disorders Center at Children’s National Hospital and corresponding author of the study.

The big picture

The study analyzed 28 patients with recurrent or treatment refractory Wilms tumor from 12 institutions who were treated with paclitaxel or nab-paclitaxel. Out of 26 patients with measurable disease, 42.3% of patients demonstrated a partial response and 15.3% demonstrated stable disease for a clinical benefit rate of 57.6%. Two patients treated adjuvantly without measurable disease were without progression for 12 and 16 months.

Moving the field forward

Over the last two decades, heavily pre-treated patients with Wilms tumor have seen few advances in available therapies. A review of 257 patients with recurrent Wilms tumor who enrolled in 79 phase 1 and 2 studies showed objective responses in only 8.5% of patients. Therefore, the response rate observed in this study is noteworthy. The authors note that the strength of this study is the large case series of a rare patient population, which provides a robust dataset of paclitaxel treatment in patients with refractory or recurrent Wilms tumor.

“Our initial observation that paclitaxel-containing regimens showed meaningful activity in several of our patients here at Children’s National prompted us to reach out to colleagues at other leading institutions,” said Dr. Dome. “That collaboration ultimately enabled this multi-institutional effort.”

Additional authors from Children’s National include Amy Frantz, NP, CPNP-AC, and Alissa Groisser, MD.

You can read the full study, Anti-tumor activity of paclitaxel-containing regimens in recurrent/refractory Wilms tumor, in Pediatric Blood and Cancer.

Michael D. Keller, MD, named as Inaugural Professor of Precision Medicine

July 21, 2025

Michael Keller, MD Children’s National Hospital named Michael D. Keller, MD, as the inaugural Professor of Precision Medicine.

Dr. Keller, a pediatric immunologist, directs the Translational Research Laboratory within the Center for Cancer and Immunology Research (CCIR). He also leads the Jeffrey Modell Diagnostic and Research Center for Primary Immunodeficiency Disorders.

A generous gift from the Indian Paintbrush Foundation endowed this new professorship.

The big picture

Dr. Keller joins a distinguished group of Children’s National physicians and scientists who hold an endowed chair. Altogether, generous donors have funded 51 professorships at the hospital.

Professorships support groundbreaking work on behalf of children and their families. They foster discoveries and innovations. They honor the recipient’s achievements and the donor’s commitment to advancing knowledge.

Why it matters

Dr. Keller’s research focuses on adoptive T-cell therapies for treating infections in immunocompromised patients. He seeks to improve outcomes for children with primary immunodeficiency disorders and those undergoing bone marrow transplants for cancer. He leads several Phase I-II studies of virus-specific T-cell therapies.

Dr. Keller has authored many peer-reviewed articles and helped shape guidelines on the treatment and diagnosis of primary immunodeficiency disorders. He is a member of the American Academy of Allergy, Asthma and Immunology; the Clinical Immunology Society; the European Society of Immunodeficiency and the Primary Immunodeficiency Treatment Consortium.

“This professorship will amplify Dr. Keller’s pioneering work, which promises to transform care for children with immunodeficiencies,” says Catherine Bollard, MBChB, MD, Senior Vice President and Chief Research Officer. Dr. Bollard leads the Cell Enhancement and Technologies for Immunotherapy (CETI) Program and holds the Dr. Robert J. and Florence T. Bosworth Distinguished Professorship in Cancer and Transplantation Biology Research.

Moving the field forward

The Indian Paintbrush Foundation’s support enabled Children’s National to establish the professorship. Endowment funds will help Dr. Keller and future chairholders launch bold precision medicine initiatives. Targeted diagnostics and therapies will deliver maximum health benefits with minimal side effects.

“The Indian Paintbrush Foundation’s deep commitment to the well-being of children and families will drive our work to personalize treatment and expand access to care,” says Nathan Kuppermann, MD, MPH, Executive Vice President and Chief Academic Officer. Dr. Kuppermann directs the Children’s National Research Institute and holds the Fight For Children Distinguished Chair of Academic Medicine.