Cancer

robotic surgery apparatus

Charging ahead: Researchers develop robotic renal tumor surgery

robotic surgery apparatus

Researchers at Children’s National Hospital are developing supervised autonomous robotic surgery to make expert kidney tumor removal accessible in rural areas, combining robotics, AI and surgeon oversight for safer, more precise outcomes.

Imagine a robot capable of planning and executing the intricate removal of a cancerous kidney tumor — a concept that might sound like science fiction. Yet this groundbreaking work is underway at the Sheikh Zayed Institute (SZI) for Pediatric Surgical Innovation at Children’s National Hospital.

Called Supervised Autonomous Robotic Renal Tumor Surgery (SARRTS), the project aims to prove that a supervised autonomous kidney resection is feasible. Its goal is to enable general surgeons in rural hospitals to oversee robots performing complex resections, democratizing access to specialized surgical care. Backed by a $1 million contract from the Advanced Research Projects Agency for Health (ARPA-H), the initiative represents new opportunities in medical innovation.

“The hope is that, someday, patients will no longer have to travel to major oncology centers to get the best possible surgical outcome when faced with renal tumors,” said Kevin Cleary, PhD, associate director of engineering at SZI. “We hope to combine the precision of robotics with a surgeon’s clinical expertise to create consistently high outcomes.”

The patient benefit

Surgery is a cornerstone of cancer treatment, but access to skilled surgeons remains unevenly distributed nationwide. Autonomous robotic surgery could address this disparity by increasing access to expert-level care, enhancing the precision and consistency of procedures and unlocking new surgical possibilities beyond human surgeons’ capabilities.

Under the initial concept, the SARRTS system will use a combination of CT imaging and 3D mapping from a robot’s RGB-depth camera. While the robot independently plans and executes the incision and tumor resection, the supervising surgeon retains full control, with the ability to approve, modify or halt the procedure at any time — an interplay between human expertise and robotic precision to help ensure safety.

Testing will be conducted on realistic kidney models, called phantoms, which are designed to train and test surgical outcomes. The project aims to validate the feasibility of supervised autonomous tumor resection while advancing technologies that could pave the way for broader applications.

“Robotics and medicine have finally reached a point where we can consider projects requiring this level of complexity,” said Anthony Sandler, MD, senior vice president and surgeon-in-chief at Children’s National and executive director of SZI. By combining autonomous robotics, artificial intelligence and surgical expertise, we can profoundly impact the lives of patients facing life-altering cancer diagnoses.”

Children’s National leads the way

In addition to the kidney surgery initiative, the Children’s National team is pursuing other groundbreaking projects. These include a second ARPA-H contract focused on robotic gallbladder removal and a National Institutes of Health grant to explore robotic hip-pinning, a procedure used to repair fractured hips with pins, screws and plates.

Axel Krieger, PhD, an associate professor of mechanical engineering at Johns Hopkins University, is collaborating closely on the kidney resection and gallbladder projects. The interdisciplinary team believes this state-of-the-art care could be tested and developed within the next decade.

“This particular surgery is complex, and a robot may offer advantages to address difficulties created by patient anatomy and visibility within the surgical field,” said Dr. Sandler. “We can imagine a day – in the not too distant future – when a human and a robotic arm could team up to successfully advance this care.”

This project has been funded in whole with federal funds from ARPA-H under cooperative agreement AY1AX000023.

Children's National Hospital's 2023-2024 Academic Annual Report on a tablet

Children’s National delivers on the promise in 2024

Children's National Hospital's 2023-2024 Academic Annual Report on a tablet

The Children’s National 2023-2024 Academic Annual Report show on a tablet.

Children’s National Hospital has released its 2023-2024 Academic Annual Report, showcasing a year of transformative progress in pediatric medicine. The report highlights achievements across its research centers, institutes and Innovation Ventures, underscoring the hospital’s role as a leader in advancing child health through innovation and collaboration.

“This year’s report reflects the remarkable progress we have made in advancing the frontiers of pediatric medicine,” said Nathan Kuppermann, MD, MPH, Chief Academic Officer and Chair of Pediatrics. “It highlights groundbreaking work across our research centers, institutes, and Innovation Ventures, showcasing the collaborative spirit that drives our mission forward. These achievements underscore our shared commitment to delivering transformative research and the best possible outcomes for children and families.”

Delivering across centers

The report captures the contributions of each of Children’s National’s research centers, each pushing the boundaries of pediatric healthcare:

  • Center for Cancer & Immunology Research (CCIR): Delivering on the promise of cell and gene therapies, offering innovative treatments for pediatric cancers and immune disorders.
  • Center for Genetic Medicine Research (CGMR): Advancing pediatric genetic medicine through interdisciplinary efforts, addressing complex genetic conditions with cutting-edge science.
  • Center for Neuroscience Research (CNR): A year of growth in scientific excellence, advancing the understanding of brain development and neurological conditions.
  • Center for Prenatal, Neonatal & Maternal Health Research (CPHNMR): Revolutionizing neonatal care with its pioneering infant brain health neuromonitoring program.
  • Center for Translational Research (CTR): Facilitating groundbreaking work by new K awardees and driving translational research to bridge the gap between discovery and clinical care.
  • Sheikh Zayed Institute for Pediatric Surgical Innovation (SZI): Leading the way in advanced research projects in pediatric surgery, pushing technological boundaries to improve outcomes for children worldwide.

Taking the lead in innovation through collaboration

Innovation Ventures at Children’s National is advancing pediatric health security, addressing unique challenges with transformative solutions. Meanwhile, the Children’s National Research & Innovation Campus (CNRIC) continues to thrive as a hub for discovery and collaboration, hosting conferences on topics like artificial intelligence in healthcare, cell and gene therapy, and pediatric epilepsy research.

A vision for the future

The report also highlights Children’s National’s focus on integrating cutting-edge technologies like artificial intelligence into its research and clinical practices, as well as addressing global health challenges such as the effects of climate change on children’s health. These efforts reflect the hospital’s commitment to improving outcomes for children everywhere through innovation, teamwork, and forward-thinking leadership.

The 2023-2024 Academic Annual Report serves as a testament to the dedication and expertise of the Children’s National community, showcasing how collaboration and innovation are shaping the future of pediatric healthcare.

Children's National Hospital's winning team for the Brain Tumor Segmentation-Africa (BraTS-Africa) challenge

AI for good: Children’s National wins global competitions for measuring brain tumors

Children's National Hospital's winning team for the Brain Tumor Segmentation-Africa (BraTS-Africa) challenge

Meet the winners (left to right): Syed M. Anwar, Ph.D., M.S., principal investigator at Children’s National; Daniel Capellan Martin, M.Sc., Polytechnic University of Madrid; Abhijeet Parida, data scientist at Children’s National; and Austin Tapp, Ph.D., postdoctoral research fellow at Children’s National.

Using an award-winning artificial intelligence (AI) algorithm developed at Children’s National Hospital, researchers ranked first in the world in the Brain Tumor Segmentation-Africa (BraTS-Africa) challenge for their approach to identifying different parts of deadly gliomas. The details of their innovative method were recently published on arXiv, a curated research-sharing platform.

“Technology can bridge the gap in healthcare between high- and low-resource countries,” said Marius George Linguraru, D.Phil., M.A., M.Sc., the Connor Family Professor in Research and Innovation and principal investigator in the Sheikh Zayed Institute for Pediatric Surgical Innovation (SZI). “By tailoring methods we created at our hospital to fit the needs of specific regions, such as sub-Saharan Africa, our research helps improve medical imaging and diagnosis in challenging environments.”

Dr. Linguraru was the program chair of the International Conference on Medical Image Computing and Computer Assisted Intervention (MICCAI) 2024 in Marrakesh, Morocco, the leading global meeting on AI in medical imaging.

Children’s National leads the way

Gliomas are a type of brain tumor with a high death rate and are especially difficult to diagnose in low- and middle-income countries. Given the increased need in Africa, researchers worldwide came together in Morocco to compete over the best way to accurately detect and measure tumors using MRI data and AI.

By applying advanced machine-learning techniques, the researchers adapted tools initially designed for well-resourced settings to work in countries with far fewer.

The study focused on transfer learning, a process in which an AI model is trained in advance on a large number of brain tumor images and then adjusted to work with smaller sets of new data. In this case, the models were adapted to work with local sub-Saharan African data using a strategy to combine different models’ strengths.

When tested, the approach achieved impressive accuracy scores. The Children’s National team, which included a colleague from the Polytechnic University of Madrid, ranked first in the BraTS-Africa 2024 challenge for identifying different parts of gliomas.

“To make the method widely available, the winning algorithm is shared online for others to use and improve upon,” Dr. Linguraru said. “My favorite part of these competitions is how they highlight the way innovation and collaboration can reduce global healthcare inequalities.”

The big picture

Children’s National researchers consistently lead global events using AI and advanced imaging to tackle complex healthcare challenges. In 2023, the team won a global contest to measure pediatric brain tumors at the MICCAI 2023 Conference. This year’s success in the BraTS-Africa challenge builds on this knowledge base and expands its use to adult gliomas.

At the Radiological Society of North America 2024 annual meeting, which drew 50,000 attendees, Zhifan Jiang, Ph.D., a staff scientist in the Precision Medical Imaging Lab at SZI, also won the Cum Laude Award for his scientific poster on applying AI to radiological images to predict severe outcomes for children with brain tumors caused by neurofibromatosis type 1.

“These achievements show how our science is leading the world in using AI for good,” Dr. Linguraru said. “Every day, we’re building on our knowledge of advanced imaging, brain tumors and AI to improve the diagnosis, measurement and treatment of deadly tumors — on a global scale.”

Attendees of the Brain Tumor Segmentation-Africa (BraTS-Africa) challenge

Kendric receives care at Children's National.

Rewriting the script for sickle cell disease

More than 100,000 Americans have sickle cell disease, an inherited blood disorder that can cause excruciating pain crises and shorter life expectancies.

Children’s National has one of the largest sickle cell programs in the United States. We are pioneering treatments and provide specialized care to about 1,500 patients each year. We participate in clinical trials to improve outcomes, shorten treatment time, reduce complications and minimize the need for opioids and chemotherapy.

Kendric receives care at Children's National.

Kendric receives care at Children’s National.

In recognition of our clinical and research excellence, Children’s National was one of a few U.S. pediatric hospitals selected to offer two promising new FDA-approved gene therapies.

Hematologist Robert Sheppard Nickel, M.D., leads a study to reduce toxicities in bone marrow transplants. “Years of development led to these curative therapies,” Dr. Nickel says. “I hope in the future we can safely cure more children with sickle cell disease.”

“The future looks promising to revolutionize the lives of our patients and make these therapies accessible worldwide,” says Andrew Campbell, M.D., director of our Comprehensive Sickle Cell Disease Program.

Kendric and Nasir find hope

In May 2024 at Children’s National, 12-year-old Kendric of Clinton, Maryland, became the world’s first patient with sickle cell disease to begin a commercially approved gene therapy that could dramatically reduce or even eliminate his pain. It involved extracting his bone marrow stem cells; genetically modifying them in a specialized lab to reduce the risk of sickling; and then, after chemotherapy, infusing them back into his bloodstream.

Expert, compassionate care empowered Kendric to understand the science behind his treatment and chart a path to recovery. “My care team taught me how to deal with my disease and everything that I need to know for the future,” he says. “They gave me hope that I could be cured.”

Nasir and his care team

Nasir and his care team.

Nasir, age 20, spent his childhood waiting to find a match for a stem cell transplant to address his sickle cell disease. Finally, in 2023, at Children’s National, he found an answer in gene therapy to alter his own cells.

Due to painful episodes and the need for frequent blood transfusions, both Kendric and Nasir missed out on a lot of school, important moments with friends and simply being kids. Now, they can explore a world in which patients like themselves can overcome this disease and reclaim their health.

“I have all of this oxygen and energy that came out of nowhere,” Nasir says. “It’s really a new life. I feel reborn.”

“The network of doctors at Children’s National gave us reassurance and lots of hope,” says Kendric’s mom, Deborah. “They made us feel like family. We are in awe of how quickly things moved and how much compassion they have shown us.”

Read more stories like this one in the latest issue of Believe magazine.

collage of news logos

Children’s National in the News: 2024

collage of news logosIn 2024, Children’s National Hospital continued to make remarkable strides across diverse areas of pediatric medicine, from groundbreaking technological innovations to critical health advocacy. The following compilation showcases ten significant stories that demonstrate the breadth and depth of the hospital’s impact, as featured in major national news outlets including NBC Nightly News, CNN, The Washington Post, The New York Times, NPR, The Today Show, Healio, and POLITICO. Delve into our 2024 news highlights for more.

1. World’s smallest pacemaker gives new hope to babies with heart defects

Charles Berul, M.D., and a patient family talk about the pill-sized pacemaker that saved the life of Abby, an infant born with deadly heart defects. (NBC Nightly News)

2. ‘A $10 death trip’: Fentanyl is killing teens. Meet one fighting for his life

Sivabalaji Kaliamurthy, M.D., addiction psychiatrist and director of the Addictions Program, spoke to CNN about the impact of drug addiction on teen health and the lack of resources available to treat opioid use disorder. (CNN)

3. Health panel urges interventions for children and teens with high BMI

Susma Vaidya, M.D., M.P.H., associate medical director of the IDEAL Clinic, shared her concerns about childhood obesity treatment recommendations issued today by a leading panel of independent U.S. health experts. (The Washington Post)

4. An Rx for food? Doctor’s offices offer groceries to those in need

Shideh Majidi, M.D., M.S.C.S., and Emily Frymark, clinical dietitian, spoke about how the food pharmacy, created in partnership with the Capital Area Food Bank, benefits patients with diabetes and other chronic conditions. (The Washington Post)

5. First patient begins newly approved sickle cell gene therapy

Kendric Cromer, a 12-year-old boy being treated at Children’s National Hospital, became the first person in the world with sickle cell disease to begin a commercially approved gene therapy that may cure the condition. “This is a big effort,” says David Jacobsohn, M.D., ScM, M.B.A. (The New York Times)

6. ‘We created this problem’: A pediatric surgeon on how gun violence affects children

Mikael Petrosyan, M.D., associate chief of General and Thoracic Surgery, discusses the stress medical staff face when treating young victims of gun violence. (NPR)

7. 7th grade boy rings bell after final round of chemotherapy

Landon, an 11-year-old patient, rang the bell at Children’s National Hospital with family, friends, doctors and nurses cheering after finishing his final round of chemotherapy. (The Today Show)

8. Study: One in three adolescents experience ‘period poverty’

Monika Goyal, M.D., M.S.C.E., pediatric emergency medicine specialist and co-director of the Center for Translational Research, emphasized the need for awareness in addressing period poverty in teenagers and young adults. (Healio)

9. The AI assurance labs are coming

Kolaleh Eskandanian, Ph.D., M.B.A., P.M.P., vice president and chief innovation officer, participates in a panel discussion covering AI data collection, associated risks, reliance and other topics related to artificial intelligence. (POLITICO)

10. First day of a ‘new life’ for a boy with sickle cell

Children’s National patient Kendric Cromer, 12, became one of the first children ever to be treated with a newly approved gene therapy that will free him from the sickle cell disease that has stolen his childhood. (The New York Times)

2024 with a lightbulb instead of a zero

The best of 2024 from Innovation District

2024 with a lightbulb instead of a zero2024 marked another groundbreaking year for Children’s National Hospital, showcasing remarkable advances across the spectrum of pediatric medicine, research and healthcare innovation. From pioneering surgical procedures to breakthrough artificial intelligence applications, the institution continued to push the boundaries of what’s possible in children’s healthcare. Read on for our list of the most popular articles we published on Innovation District in 2024.

1. Prenatal COVID exposure associated with changes in newborn brain

A study led by researchers at Children’s National Hospital showed that babies born during the COVID-19 pandemic have differences in the size of certain structures in the brain, compared to infants born before the pandemic. The findings suggest that exposure to the coronavirus and being pregnant during the pandemic could play a role in shaping infant brain development.
(3 min. read)

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

Children’s National Hospital was ranked as a top hospital in the nation by the U.S. News & World Report 2024-25 Best Children’s Hospitals annual rankings. This marks the eighth 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.
(2 min. read)

3. Children’s National performs first ever HIFU procedure on patient with cerebral palsy

In January 2023, a team of multidisciplinary doctors performed the first case in the world of using bilateral high intensity focused ultrasound (HIFU) pallidotomy on Jesus, a 22-year-old patient with dyskinetic cerebral palsy. The procedure is part of a clinical trial led by Chima Oluigbo, M.D., pediatric neurosurgeon at Children’s National Hospital.
(3 min. read)

4. Novel ultrasound device gets FDA breakthrough designation with Children’s National support

A novel ultrasound device developed by Bloom Standard received the Food and Drug Administration’s valued breakthrough device designation with the help of Children’s National Hospital. The device that enables autonomous, hands-free ultrasound scans to be performed anywhere, by any user.
(2 min. read)

5. First-of-its-kind pilot study on the impacts of Lyme disease in pregnancy and infant development

Understanding the effects of Lyme disease on the developing fetal brain is essential to ensure timely prenatal and postnatal treatments to protect the fetus and newborn. In response to this need, Children’s National Hospital is leading a pilot study to establish the groundwork needed for a larger study to determine the effect of in utero exposure to Lyme disease on pregnancy and early childhood neurodevelopmental outcomes.
(3 min. read)

6. Earliest hybrid HLHS heart surgery kids thrive 5 years later

Five years ago, Cayden was born 6 weeks early weighing less than four pounds and at risk of dying from her critical congenital heart disease. Today, she’s a happy five-year-old. Early diagnosis of her hypoplastic right ventricle, double inlet left ventricle and critical coarctation of the aorta allowed for the team at Children’s National Hospital to create a careful plan for safe delivery and to offer an innovative hybrid HLHS surgical approach at the hospital within 24 hours after she was born.
(1 min. read)

7. Wayne J. Franklin, M.D., F.A.C.C., named senior vice president of the Children’s National Heart Center

Children’s National Hospital appointed Wayne J. Franklin, M.D., F.A.C.C., as the new senior vice president (SVP) of the Children’s National Heart Center. In this role, Dr. Franklin oversees the full spectrum of heart care services including cardiac imaging and diagnostics, interventional cardiology, electrophysiology, cardiac anesthesia, cardiac surgery and cardiac intensive care.
(2 min. read)

8. Artificial – and accelerated – intelligence: endless applications to expand health equity

By pioneering artificial intelligence (AI) innovation programs at Children’s National Hospital, Marius George Linguraru, D.Phil., M.A., M.Sc., and the AI experts he leads are ensuring patients and families benefit from a coming wave of technological advances. The team is teaching AI to interpret complex data that could otherwise overwhelm clinicians.
(4 min. read)

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

Painting a sobering picture, a research team led by Children’s National Hospital culled years of data demonstrating that maternal mental illness is an under-recognized contributor to the death of new mothers. They called for urgent action to address this public health crisis.
(3 min. read)

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

Children’s National Hospital appointed Nathan Kuppermann, M.D., M.P.H., as its new executive vice president, chief academic officer and chair of Pediatrics. In this role, Dr. Kuppermann oversees research, education and innovation for the Children’s National Research Institute as well as academic and administrative leadership in the Department of Pediatrics at George Washington University School of Medicine & Health Services.
(2 min. read)

11. First global clinical trial achieves promising results for hypochondroplasia

Researchers from Children’s National Hospital presented findings from the first clinical trial of the medication vosoritide for children with hypochondroplasia – a rare genetic growth disorder. During the phase 2 trial, researchers found vosoritide increased the growth rate in children with hypochondroplasia, allowing them to grow on average an extra 1.8 cm per year.
(2 min. read)

12. Pioneering research center aims to revolutionize prenatal and neonatal health

Since its establishment in July 2023, the Center for Prenatal, Neonatal & Maternal Health Research at Children’s National Hospital has gained recognition through high-impact scientific publications, featuring noteworthy studies exploring the early phases of human development.
(3 min. read)

Kids sitting at desks in school

Radon in school: A hidden worry for eastern Pennsylvania students

Kids sitting at desks in school

Some students may be exposed to nearly twice the annual dose of natural background radiation, estimated by the U.S. Nuclear Regulatory Commission at 3.1 mSv (310 mrem).

A Children’s National Hospital researcher teamed up with a high school student from Bethlehem, Pa., to shed light on radon, a silent health risk that may be present in some schools.

They examined radon levels in five eastern Pennsylvania school districts. The neighborhoods surrounding all 37 public schools had average radon levels exceeding the federal action level, or 4.0 pCi/L. According to their findings, the same could be true of the school buildings.

In a new research letter published in JAMA Network Open, researchers found some students may be exposed to nearly twice the annual dose of natural background radiation, estimated by the U.S. Nuclear Regulatory Commission at 3.1 mSv (310 mrem).

An odorless and invisible gas, radon is the leading cause of lung cancer among nonsmokers and the second overall cause of lung cancer nationwide. Its greatest danger lies in prolonged exposure, a risk amplified in school settings where children and teachers spend extensive hours. The U.S. Environmental Protection Agency (EPA) estimates more than 70,000 classrooms have high short-term radon levels.

“This study highlights the urgent need for radon testing in schools,” said Beth Tarini, M.D., M.S., M.B.A., co-director of the Center for Translational Research at Children’s National and the manuscript’s senior author. “Unchecked exposure to radon in these settings could have significant short- and long-term health effects, particularly for children.”

The EPA has found that approximately 20% of schools nationwide have done some testing, and only . In the Washington, D.C., region, Dr. Tarini says testing is often done:

  • The District of Columbia requires public schools to test for radon and publicize the results. If the results are above the federal limit of >4pCi/L, the schools are required to mitigate the risk.
  • Maryland doesn’t require schools to test for radon, but some schools test. The state’s largest school district — Montgomery County Public Schools — has been testing for radon since the late 1980s and retests facilities every five years.
  • In Virginia, the commonwealth requires public schools to test for radon, make the results public and report the results to the state.

Brian Yang, the study’s first author, called for action in Pennsylvania and regions with known radon risks.

“This research underscores the need to test radon levels in schools and, if necessary, mitigate,” said Yang, a senior at Moravian Academy in Bethlehem, Pa. “Addressing this invisible and under-recognized threat should be a public health priority.”

For more details, read the full study — “Estimated Radon Exposure in Eastern Pennsylvania Schools” — in JAMA Network Open.

 

 

Attendees at the 2nd annual Cell & Gene Therapy Symposium

Regional powerhouse: Cell and Gene therapy leaders from mid-Atlantic forge connections

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Nearly 200 biomedical leaders from Washington, D.C., Maryland, and Virginia gathered at the Children’s National Research & Innovation Campus for the 2nd annual Cell & Gene Therapy Symposium. The event showcased groundbreaking developments in rare disease treatments and underscored the importance of regional collaboration.

“By targeting diseases at the cellular level, we are on the cusp of breakthroughs in cell and gene therapy that will transform medicine,” said Catherine Bollard, M.D., M.B.Ch.B., director of the Center for Cancer and Immunology Research (CCIR) at Children’s National Hospital and a host of the symposium. “Progress will accelerate if we build partnerships beyond our own organizations.”

The big picture

Scientists and clinicians have worked for more than two decades to develop cell and gene therapies aimed at treating diseases on a cellular level. The past few years have been particularly promising as investment in science has led to advancements. Children’s National is at the forefront, as one of the first pediatric hospitals in the world to offer commercial gene therapies for sickle cell disease.

Many more treatments for rare diseases are in development at Children’s National and beyond. Leaders at CCIR are actively building collaborations with companies, academic institutions and enterprises across the mid-Atlantic region to accelerate these efforts.

During the symposium, Eugene Hwang, M.D., chief of Oncology at Children’s National, addressed the urgent need for more effective and less toxic treatments for pediatric brain tumors. He highlighted the potential of combining immunotherapies with innovations like low-intensity focused ultrasound, which can open the blood-brain barrier temporarily to improve drug delivery to tumors.

“With collaboration between the lab and clinic, alongside industry partners and even between hospitals, we can finally make strides I haven’t seen in my entire career,” Dr. Hwang said. “It’s an incredibly inspiring time for all of us.”

Why it matters

Experts from organizations as diverse as MaxCyte, ScaleReady, RoosterBio, PSC Biotech, Qiagen, FujiFilm and the Frederick County Office of Economic Development came together for the daylong conversation.

Michael Friedlander, Ph.D., executive director of the Fralin Biomedical Research Institute at Virginia Tech, emphasized the critical role of regional partnerships in fulfilling the potential of these emerging therapies. He pointed to the collaborative research between Children’s National and Virginia Tech on brain tumors, where bioengineers and cancer researchers are working side-by-side to create new treatments.

“We are now able to begin delivering these leading-edge therapies to patients,” Dr. Friedlander said. “For example, those who live in rural settings often have much less access to such frontline medical innovations. By collaborating with Children’s National and gaining access to urban pediatric populations, as well as patients in our more rural area, we can start to bring these therapies to a much broader audience.”

What’s next

Patrick Hanley, Ph.D., chief and director of the Cellular Therapy Program at Children’s National, observed that other regions in the U.S. are uniting to advance scientific discoveries with the backing of government, academia and industry. He hopes to see similar collaboration across the D.C., Maryland, and Virginia area, known as the DMV. Children’s National is leading an initiative called CHARM – the Capital Health and Mid-Atlantic Regenerative Medicine – to bring regional experts together for webinars, networking events and partnership opportunities.

“There’s significant interest in cell and gene therapy worldwide,” said Dr. Hanley, a symposium host. “I see an even greater interest in creating cell and gene therapy hubs. The time is right for our mid-Atlantic region, and I’m excited to see what unfolds in the next five years.”

Drs. Wessel, Dome and Kim with Michelle Riley-Brown and the Speight’s

Honor bestowed on AeRang Kim, M.D., Ph.D.

Drs. Wessel, Dome and Kim with Michelle Riley-Brown and the Speight’s On November 1, Children’s National Hospital installed AeRang Kim, M.D., Ph.D., as the first holder of The Lexi Speight Chair in Pediatric Oncology.

Dr. Kim is clinical research director for the Oncology Division within the Cancer and Blood Disorders Center at Children’s National. She is a tenured professor of pediatrics at the George Washington University School of Medicine and Health Sciences.

The big picture

Dr. Kim 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 altogether have funded 49 professorships.

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

Why it matters

Since arriving at Children’s National in 2010, Dr. Kim has played a pivotal role in our solid tumor program. She leads the renowned multi-disciplinary Sarcoma Clinic, which includes a comprehensive team of oncologists, nurses, orthopedic surgeons, radiologists, a genetic counselor and a physical therapist. It is one of the few of its kind in the country. Dr. Kim’s dedication and leadership have been instrumental in earning Children’s National recognition as a Sarcoma Alliance Center of Excellence.

Dr. Kim leads clinical trials evaluating new pediatric solid tumor treatments across multiple institutions. She pioneers precision approaches using targeted drugs, advanced devices and cell therapies. Her innovative work attracts numerous grants, including a recent $1.3 million from the Department of Defense.

“The future of cancer treatment is in precision medicine,” says Dr. Kim. “As The Lexi Speight Chair of Pediatric Oncology, I will explore new ways to design clinical trials to accelerate development of new therapies for solid tumors. This could change the paradigm for pediatric cancer treatment.”

Moving the field forward

The Speight family, through their vision and generosity, are ensuring that Dr. Kim and future holders of this chair will launch bold, new initiatives to rapidly advance the field of pediatric oncology, elevate our leadership and improve the lifetimes of children with solid tumors.

“When Lexi died, we had a decision to make about how to channel our grief,” Cyndi Speight, Lexi’s mom, remembered. “And do what we could to help other children and families from experiencing what we did.” And so, Cyndi, her husband Petr, and their daughter Sam established The Lexi Speight Chair in Pediatric Oncology with support from community partners in loving memory of Lexi. The Speight family started Laps for Lexi, an annual 5K run/walk that raised money for both pediatric cancer at Children’s National and a scholarship at Sandy Spring Friends School. This successful event ran for 13 years, bringing hope to children and families. The Speight family’s dedication and passion for advancing solid tumor care ensures that Lexi’s legacy lives on in our pursuit of better treatments for children.

2024-25 US News Badges

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

2024-25 US News BadgesChildren’s National Hospital in Washington, D.C., was ranked as a top hospital in the nation by the U.S. News & World Report 2024-25 Best Children’s Hospitals annual rankings. This marks the eighth 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.

This year, U.S. News ended ordinal rankings on its Honor Roll. Instead of assigning a numerical rank from 1 to 10, all hospitals on the Honor Roll will be recognized as having attained the highest standards of care in the nation.

In addition, Children’s National tied for #1 pediatric hospital in the Mid-Atlantic region, which includes New York, New Jersey, Delaware, Pennsylvania, the District of Columbia, West Virginia and Virginia. It’s also best in the Mid-Atlantic in Neonatology.

For the fourteenth straight year, Children’s National ranked in 10 specialty services. New this year, U.S. News included behavioral health as a service line in the rankings. Since it’s the first year, there are no ordinal rankings for behavioral health, but the Children’s National program was named one of the top 50 programs in the country.

“In my first year here, I witnessed what makes Children’s National so special — our commitment to collaboration, empowering one another, and charting a bold path forward for pediatric care,” said Michelle Riley-Brown, MHA, FACHE, president and chief executive officer of Children’s National. “I’m proud U.S. News again recognized Children’s National as one of the top in the nation and the highest-ranked pediatric hospital in D.C., Maryland and Virginia. Together, we’ll continue to push the boundaries of care, research and innovation to make a difference for those who matter most — the kids.”

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 nearly two decades, U.S. News has published Best Children’s Hospitals to empower the parents and caregivers of children with complex medical needs,” said Ben Harder, chief of health analysis and managing editor at U.S. News. “Children’s hospitals appearing on the U.S. News Honor Roll have a track record of delivering unparalleled 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 Children’s National specialty services that U.S. News ranked in the top 10 nationally are:

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

Dr. Andrew Campbell

Meet Dr. Andrew Campbell: Trailblazing treatment for patients with sickle cell disease

Andrew Campbell, M.D., remembers the first time he met a patient with sickle cell disease in the early years of his medical training. It was a brief interaction that had a profound effect on the trajectory of his career.

Sickle cell disease is an inherited blood disorder that primarily affects African American and Hispanic American children. The disease can cause severe pain events as well as progressive organ damage in patients.

This life-altering disease has affected children for more than a century, yet only one FDA-approved drug for treatment of sickle cell disease was developed in the first 100 years of its existence. Recognizing the health inequities that have contributed to an overall lack of therapies and providers in the field, Dr. Campbell knew he had to act.

“It was an area that needed a lot of resources, but also a lot of research and understanding of what our patients are going through. So, that was my initial launch into the field of Hematology and Oncology,” he explained, and he’s been an instrumental leader ever since.

For the past seven years, Dr. Campbell has served as the director of the Comprehensive Sickle Cell Disease Program at Children’s National Hospital — one of the largest sickle cell programs in the country, treating nearly 1,400 patients a year. Locally, he is Principal Investigator for the American Society of Hematology Sickle Cell Research Collaborative’s DMV Sickle Cell Consortium that includes several area sickle cell clinics including Northern Virginia, Richmond, Washington DC and Maryland.

In December 2023, the FDA approved two new gene therapies (CASGEVY™ and LYFGENIA™) to treat patients with sickle cell disease. Children’s National became one of the few pediatric hospitals in the country to offer these therapies, going on to then treat the first patient in the U.S. using the gene therapy method.

Dr. Campbell, who is treating the 12-year-old patient, says his team is excited about the future of these non-chemotherapy treatments and the curative possibilities they will bring to the field.

Despite being part of this significant milestone in the sickle cell community, Dr. Campbell says the work doesn’t stop there. His passion for making a difference and his impact on patients extends far beyond just Washington, D.C., and even the U.S.

For example, Dr. Campbell directs a research group called the Consortium for the Advancement of Sickle Cell Disease Research (CASiRe), with other sickle cell providers across the world. He says one of their goals is to better understand the ways that sickle cell can present itself in patients based on the country in which they receive treatment. “It’s really showing that based on the geographic difference of patients, it has implication in how they receive care,” he explains, adding that the group hopes to take what they learn in these studies to design more inclusive clinical trials in the future.

When he’s not devoting his time to research, you can find Dr. Campbell taking the fight for patients with sickle cell disease directly to lawmakers.

“I have gone to Capitol Hill a number of times to advocate for access and improvement of treatments for sickle cell disease,” he says, hoping that by using his voice and presenting his research, he can help the current generation, as well as the future generation of patients, get the care they deserve.

Screen shot of educational video

What’s a TAA-T? Advocates create videos to translate science for patients

As researchers develop groundbreaking cellular therapies to treat pediatric solid tumors, physicians are preparing new ways to explain how these treatments work to patients and caregivers.

In a series of educational videos, scientists from Children’s National Hospital and institutions worldwide are offering tutorials on these novel treatments and how they target solid tumors at the cellular level.

“Let’s start by breaking down what a tumor-associated antigen-specific T (TAA-T) cell is,” Children’s National Research Technician Sammy Murphy says in one new video. “Our aim is to harness the power of T cells to identify and attack cancer cells.”

In less than six minutes, Murphy provides a short course on the details of these TAA-T cells and how her team combines their expertise in biology, medicine, bioinformatics, quality assurance and more to create the new therapies. “This collaborative team spirit has been a huge motivating factor and represents the best of what science can be,” she explains.

The big picture

Children’s National summer student Diana Kentell, a senior at Pratt Institute of Art studying digital art and 2D animation, created this video and a collection of others for the Cancer Grand Challenges (CGC), sponsored by the National Cancer Institute and Cancer Research UK. In 2022, the CGC awarded $25 million to Children’s National, the University College of London Cancer Institute and its partners on the NexTGen team to develop new therapies for pediatric solid tumors using CAR T cells.

The NexTGen team includes six patient advocates who have all been touched by pediatric tumors and support the scientists by providing the patient perspective on research and new treatments. These videos are a slice of the group’s efforts.

C. Russell Y. Cruz, M.D., Ph.D., a translational immunologist on the NexTGen team who oversaw Kentell’s video project, says bridging the gap between scientists and their patients who enroll in clinical trials will be essential to ensuring patients weigh the possibilities and the risks.

“Patient advocates help us understand our work from their perspective, making our science accessible to everyone,” Dr. Cruz said. “Engaging with such dedicated individuals often helps us refine our own ideas and provides invaluable insights. Most importantly, they remind us of our ultimate goal: to free future generations from the burden of pediatric cancer.”

Why we’re excited

In addition to the video on TAA-T cells, the team has assembled a collection of videos on killing assays, tumor slice assays and CAR T-cell manufacturing, which will help patients learn about the treatments when the clinical trials start.  Sara Wakeling, who leads the NexTGen team’s patient advocates, said this toolkit will be a vital resource.

“Each of us came to this advocacy work because we were deeply affected by pediatric cancer.  We aim to ensure that the child’s voice is central to the research and that the science is communicated in an informative and digestible way for patients’ families and the public,” Wakeling said. “With these videos, lay summaries of manuscripts and other explainers, we will have concrete information to share with families as soon as the new CAR T-cell therapies are ready for clinical trials.”

A clean room at CNRI.

CellBuilder: A ready-made solution for cell & gene therapy manufacturing

A clean room at CNRI.

With CellBuilder and our global partnerships, Children’s National hopes to expand access to groundbreaking cell and gene therapy treatments as they take off in the next five years.

With cell and gene therapies poised to reshape cancer and rare disease treatments, researchers at Children’s National Hospital are pioneering ready-to-use solutions that will bring these cutting-edge therapies directly to hospitals and other treatment centers, shrinking the distance between doctors and patients.

“The next five years are going to be a period of tremendous growth for cell and gene therapy,” said Patrick Hanley, Ph.D., chief and director of the Cellular Therapy Program at Children’s National. “Currently, there’s no shortage of interest from the medical community, but there’s a shortage of people who can manufacture and administer this care. We’re looking for ways to get these treatments to the patients by providing other institutions the tools they need to launch these programs cost-effectively, safely and efficiently.”

Called CellBuilder, the starter kits for cell and gene therapy programs could transform the landscape for pediatric patients.

The big picture

Dr. Hanley and many members of the Children’s National team have been working in cell and gene therapy for more than a decade, gaining extensive experience in the technical and regulatory hurdles inherent in creating treatments that target diseases at their source.

In cell therapy, a specific cell type is modified and transferred to a patient with a payload that can target a disease or disorder. For example, T cells may be modified and delivered to patients to teach their immune systems to fight cancer.  In gene therapy, a patient’s genetic code is modified to treat or prevent diseases, such as sickle cell disease, cancers and other genetic disorders. This can be done by introducing a healthy copy of a gene, repairing a faulty gene or altering a gene’s function.

Children’s National has become a leader in manufacturing virus-specific T cells, one method of delivering cell therapies, and the Cellular Therapy Program has conducted consortium-led, multi-center trials. Many other healthcare sites across the country want to start programs offering this care at their facilities.

The holdup in the field

Starting a cell and gene therapy program from scratch can take years of effort, training and money. That’s why Jay Tanna, M.S., R.A.C., quality assurance manager of the Cellular Therapy Program at Children’s National, said the team is creating CellBuilder starter kits, which include the manufacturing protocol and the resources necessary to launch a cell therapy program almost instantly.

“With a suitable knowledge base, institutions can start their own cellular therapy program at the point of care, using our manufacturing protocols, vetted reagents and other key elements of the process,” Tanna said. “Of course, interested institutions would have to meet regulatory requirements and establish a clean room to manufacture these therapies. If they want to use CellBuilder to run a clinical trial, they can do that. If they want to take it to become a licensed product, they can do that, too.”

Children’s National has worked with more than five institutions to build their virus-specific T-cell program and is now using the kits to accelerate and commercialize the process to increase patient access. The lab has also entered into a memorandum of understanding with the Tokyo-based Hitachi Global Life Solutions, Inc., an innovative modular clean room manufacturer, with the goal of offering a bundled solution.

Why we’re excited

Dr. Hanley and his colleagues say that the partnerships Children’s National is forging as they consult with other experts in this field will expand access to cell and gene therapy across the country—and hopefully around the world.

“It used to be that, to get a CAR T cell, you would collect the cells at Children’s National, ship them to a company like Novartis, have the therapy manufactured there and then shipped back,” said Michael Keller, M.D., who co-led a first-of-its kind immunotherapy trial as the Translational Research Laboratory director at the Children’s National Cell Enhancement and Technologies for Immunotherapy Program. “It was expensive, time-consuming and limited patient access. Now, there’s growing interest in manufacturing at each site, just like you would with a bone marrow transplant.”

With CellBuilder and our global partnerships, Children’s National hopes to expand access to groundbreaking cell and gene therapy treatments as they take off in the next five years, extending lifetimes and improving the quality of life for children suffering from rare disorders.

“We’re trying to capture the momentum underway in the field by providing this kit so that institutions don’t have to know how to do everything,” Dr. Hanley said. “We provide all the knowledge, a reagent list and everything else they need—and they provide the care.”

Leaders from pediatric healthcare, federal organizations, academia, industry and patient advocacy groups gathered at the Children’s National Research & Innovation Campus.

Access4Kids: A new model to pay for pediatric cell and gene therapies

Science is pioneering cures for pediatric rare diseases in a coming wave of new cell and gene therapies. However, the biopharmaceutical industry’s insistence on large patient populations and high profit margins may prevent these life-saving treatments from reaching the children who desperately need them. When successful therapeutics fail to see commercialization, experts say they have fallen into the “Valley of Death.”

To address this, leaders from pediatric healthcare, federal organizations, academia, industry and patient advocacy groups gathered at the Children’s National Research & Innovation Campus. Their objective: build a new framework to deliver these transformative drugs to clinics worldwide.

Meet the team forming Access4Kids, a nonprofit whose mission is to build new pathways to pay for cures and provide hope to children with life-limiting diseases. This group is working to change medicine and how we pay for it, under the leadership of Catherine Bollard, M.D., M.B.Ch.B., director of the Children’s National Center for Cancer and Immunology Research, Crystal Mackall, M.D., director of the Stanford Center for Cancer Cell Therapy, Julie Park, M.D., Oncology Department chair at St. Jude Children’s Research Hospital, and Alan Wayne, M.D., pediatrician-in-chief at Children’s Hospital Los Angeles.

Cheng-Chia “Fred” Wu, M.D., Ph.D.

Expanding team innovates to take on pediatric brain tumors

Cheng-Chia “Fred” Wu, M.D., Ph.D.

Cheng-Chia “Fred” Wu, M.D., Ph.D., joins the team at the campus as an assistant professor at Virginia Tech’s Fralin Biomedical Research Institute. Image credit: Fralin Biomedical Research Institute.

Experience, talent and technology are coming together at the Children’s National Research & Innovation Campus to solve the complex challenges of treating pediatric brain tumors through a growing partnership between Children’s National Hospital and Virginia Tech.

Cheng-Chia “Fred” Wu, M.D., Ph.D., joins the team at the campus as an assistant professor at Virginia Tech’s Fralin Biomedical Research Institute. He hopes to improve treatment for pediatric brain tumors and other cancers by leveraging technological advances in focused ultrasound and studying how this modality can be combined with other novel therapies. Children’s National physicians and scientists are collaborating with Virginia Tech scientists to develop these new approaches.

Why we’re excited

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, said collaborations — including the partnership between Children’s National and Virginia Tech — are essential to solving formidable scientific challenges.

“We are thrilled to be working with Dr. Wu to develop cutting-edge therapeutics for pediatric brain tumors,” Dr. Bollard said. “With Dr. Wu’s experience and the talents of our team at Children’s National, I have great hope that we will be able to combine our novel technologies and therapies to provide tremendous breakthroughs for treating pediatric patients with brain tumors.”

Before joining Virginia Tech, Dr. Wu was an assistant professor of radiation oncology at Columbia University Irving Medical Center in New York, where he treated pediatric cancers and central nervous system malignancies.

“Partnering with Children’s National connects us to a world-class clinical trial institute that has been a pioneer in treating brain tumors with focused ultrasound technology, and this presents a unique opportunity to help children and families struggling with cancer,” Dr. Wu said. “I can’t wait to see where this takes us.”

Dr. Wu played a key role in the Initiative for Drug Delivery Innovation for Childhood Brain Tumors at Columbia, developing a bench-to-bedside platform to facilitate the translation of promising technologies for targeted drug delivery in children with brain tumors.

Within three years, the team demonstrated the safety and feasibility of using focused ultrasound in the brainstem and adding radiation in preclinical models. They then opened two clinical trials for kids with relapsed diffuse midline glioma, an aggressive and difficult-to-treat brain tumor that occurs in the brain stem, thalamus and spinal cord.

What’s ahead

Dr. Wu will be involved in a wide range of research touching both organizations, including veterinary medicine and biomedical engineering at Virginia Tech.

Michael Friedlander, Ph.D., executive director of the Fralin Biomedical Research Institute at Virginia Tech Carilion and vice president for health sciences and technology at Virginia Tech, welcomed Dr. Wu’s experience as a pediatric radiation oncologist, translational physician-scientist and clinical trialist who has led an innovative drug delivery program.

“He is armed with a unique set of skills to identify promising new technology and implement it in areas of great need for treatment of pediatric cancers,” Dr. Friedlander said. “We are absolutely delighted to have Dr. Wu as part of the team.  He represents a powerful new part of the strong partnership between Virginia Tech and Children’s National Hospital for addressing pediatric brain cancer.”

Assessing psychosocial risk, patient readiness for sickle cell gene therapy

The CureSCi Patient Readiness and Resilience Working Group brought together behavioral health clinicians and scientists from across the U.S. with expertise in sickle cell disease to develop recommendations for assessing and promoting patient readiness for gene therapy.

Two gene therapies for sickle cell disease were recently approved by the U.S. Food and Drug Administration (FDA) and are now commercially available in the U.S. This marks a historic shift in the treatment of sickle cell disease (SCD) and represents a leap forward more broadly for the medical community, opening a range of exciting possibilities for the development of novel therapeutics for other diseases. However, these new therapies are not without medical and psychological risks; therefore, the Cure Sickle Cell Initiative (CureSCi) of the National Heart, Lung and Blood Institute (NHLBI) convened a Patient Readiness and Resilience Working Group to develop recommendations for the assessment of psychosocial readiness for gene therapy.

What’s been the hold-up in the field?

Clinicians have long recognized that psychological and social issues have the potential to affect treatment outcomes following disease-modifying or transformative treatments, such as hematopoietic stem cell transplants. The same concerns exist for gene therapies, but there has not been clear guidance about the best ways to evaluate patient readiness and psychosocial risk and resilience factors in these contexts.

How does this work move the field forward?

The CureSCi Patient Readiness and Resilience Working Group brought together behavioral health clinicians and scientists from across the U.S. with expertise in SCD, as well as caregivers and patients with the lived experience of having SCD, to develop recommendations for assessing and promoting patient readiness for gene therapy. The resulting consensus statement outlines clear and practical guidance for conducting pre-gene therapy patient readiness assessments.

“This is an exciting time for the sickle cell and medical communities,” says Steven Hardy, Ph.D., director of Behavioral Health Services in the Divisions of Hematology, Oncology and Blood and Marrow Transplantation at Children’s National Hospital and lead author on the consensus statement. “But it is also a time to exercise caution to ensure that, in the cloud of such enthusiasm, we do not lose sight of the complex ways that human psychology, relationships and biology interact to influence health.”

How will this work benefit patients?

This new guidance for evaluating psychosocial readiness will ensure that important issues — such as the degree to which patients have been informed of and understand key treatment details, are interested in and motivated to pursue treatment, and have considered how undergoing gene therapy will affect their activities, relationships and mental health — are considered and patients are provided the necessary supports.

“These recommendations offer a blueprint and a charge to institutions, payors and policymakers around the world to prioritize the psychosocial well-being of patients with SCD undergoing gene therapy,” says Dr. Hardy.

How is Children’s National leading in this space?

Children’s National has participated in gene therapy clinical trials and is the first institution globally to treat a patient with SCD with one of the new commercially available gene therapies. Dr. Hardy chaired the CureSCi Patient Readiness and Resilience Working Group that developed the consensus recommendations. Psychologists in the Divisions of Hematology, Oncology and Blood and Marrow Transplantation have adopted a standard protocol, informed by the consensus recommendations, for conducting pre-gene therapy assessments of patient readiness.

You can read the full consensus statement, Assessing Psychosocial Risk and Resilience to Support Readiness for Gene Therapy in Sickle Cell Disease: A Consensus Statement, in JAMA Network Open.

Catherine Bollard, M.D., M.B.Ch.B., director of the Children’s National Center for Cancer and Immunology Research.

Closing the ‘Valley of Death’: Pioneering – and paying for – new therapies

Catherine Bollard, M.D., M.B.Ch.B., director of the Children’s National Center for Cancer and Immunology Research

“To cure brain tumors, sickle cell disease and scores of other illnesses that have an outsized impact on pediatric patients, we will need an innovative mechanism to ensure that money is not a barrier to scientific advancement and world-class care,” says Dr. Bollard.

With the advent of life-changing cell and gene therapies (CGTs) to treat pediatric diseases, price is becoming a significant obstacle to care and cures.

Successful therapies can wind up shelved or in hard-to-reach clinical trials for a litany of reasons: high manufacturing costs, significant regulatory burdens, a lack of enthusiasm from the pharmaceutical industry in the small pediatric market and the simple fact that insurance companies resist paying the price of $1 million or more for a therapeutic. When successful treatments are set aside and become victims of this market failure, leaders in pediatric medicine say the drug has been relegated to the “Valley of Death.”

Experts at Children’s National Hospital and other leading U.S. research institutions are working to ferry drugs across it.

A new way forward

The Access4Kids think tank is bringing together healthcare leaders to chart another way – with its hosts Catherine Bollard, M.D., M.B.Ch.B., director of the Children’s National Center for Cancer and Immunology Research, Crystal Mackall, M.D., director of the Stanford Center for Cancer Cell Therapy, Julie Park, M.D., Oncology Department chair at St. Jude Children’s Research Hospital, and Alan Wayne, M.D., pediatrician-in-chief at Children’s Hospital Los Angeles. Organized at the Children’s National Research & Innovation Campus, the think tank will include voices from Seattle Children’s Hospital in Washington state and the Moonshot Presidential Office in Washington, D.C., along with experts from academic institutions, the federal government and patient advocacy groups.

They explained their vision in their recent Nature Medicine perspective,  “Enhancing pediatric access to cell and gene therapies.” One idea under consideration is to create a biotech enterprise – backed by public monies, foundations and philanthropies – to support the late-stage development and commercialization of pediatric CGTs. Called a Pediatric Advanced Medicines Biotech (PAMB), this new organization would follow a novel pathway outside the traditional biopharma development model.

“To cure brain tumors, sickle cell disease and scores of other illnesses that have an outsized impact on pediatric patients, we will need an innovative mechanism to ensure that money is not a barrier to scientific advancement and world-class care,” Dr. Bollard said. “My colleagues and I aren’t exaggerating when we say this is a ‘save the world’ effort. It’s an initiative to disrupt the pharmaceutical industry and impact medicine, ultimately saving lives and entire lifetimes.”

The think tank is the second in a series aimed at solving this formidable challenge. Led by scientists, the work goes to the heart of healthcare economics.

“We need to find a way to support the academic ecosystem by reducing costs, creating efficiencies in manufacturing and working with regulatory bodies to bolster business models that lead to safe and effective therapeutics,” Dr. Mackall said. “We went to school to study science and medicine. Now, our mission includes revamping business models, as we find ways to increase access to lifesaving treatments for children in need.”

Why it matters

Creating novel pathways to pay for CGTs presents a challenge and an opportunity for researchers in pediatrics and rare diseases. Drs. Mackall, Bollard and their colleagues are exasperated when scientists discover successful treatments for life-threatening illnesses, but market forces lock the drug away in hard-to-reach clinical trials or – even worse – land it on a shelf.

Consider just one example, laid out in the Nature Medicine perspective: Children with adenosine deaminase-deficient severe combined immunodeficiency (ADA-SCID) lack nearly all protection from viruses, bacteria and fungi, leaving them vulnerable to a world of opportunistic organisms. Researchers at San Raffaele Telethon Institute for Gene Therapy in Italy won U.S. and European regulatory approvals for a gene replacement therapy to treat the life-threatening disorder. Yet currently the therapy is only available in clinical trials, with support from research grants, philanthropic donations and institutional resources.

“This happens to successful therapeutics, and it’s heartbreaking. The current business model in medicine can lead to situations where successful therapies have extremely restricted availability, and there are no built-in guarantees of sustained access,” Dr. Bollard said. “We can and must do better – and we believe we have a way forward.”

graphic explaining LIFU and CAR T

Mission critical: Pentagon awards grant to combine two novel brain tumor therapies

graphic explaining LIFU and CAR TA Children’s National Hospital researcher is embarking on a two-year project that could revolutionize the care of rare pediatric brain tumors, through a Department of Defense (DOD) award to study the efficacy of combining the powers of acoustic therapy and cellular immunotherapy to treat the deadliest forms of medulloblastoma.

Designed by DOD to advance novel innovations that could revolutionize medicine, the prestigious Idea Award supports independent investigators with high-risk, but potentially high-reward, concepts to fill gaps in cancer prevention and treatment. Dalia Haydar, Pharm.D., Ph.D., principal investigator at the Cell Enhancement and Technologies for Immunotherapy Program, believes her first-of-its-kind approach could do just that.

A one-two punch

Dr. Haydar is building on promising research into a novel type of immunotherapy – chimeric antigen receptor (CAR) T-cell therapy – which uses the body’s own immune system to target and destroy pediatric brain tumors. She hopes to combine the potential of this immunotherapy with the early success seen in an acoustic therapy, called low-intensity focused ultrasound (LIFU). Akin to the imaging technology used during pregnancy, the sound waves can be directed toward any organ of the body for therapeutic reasons. With LIFU, Dr. Haydar hopes to tune the right amount of energy toward a brain tumor to temporarily open the blood-brain barrier and improve the delivery of therapeutics.

“We’re using two treatments that are tested in the clinic. We know how they work individually, but we’re investigating methods to combine the best aspects of both,” Dr. Haydar said. “I hope to use it, not just to open the channels in the blood-brain barrier, but also to cause a certain kind of immune response in the brain, which will help the CAR T-cells fight cancer.”

This collaborative grant includes experts in the field, including Yanxin Pei, Ph.D., principal investigator at Children’s National and an expert in medulloblastoma modeling, and Natasha Sheybani, Ph.D., assistant professor at the University of Virginia and a leading figure in the application of focused ultrasound.

Why we’re excited

Dr. Haydar is initially focused on high-risk medulloblastoma, specifically sonic hedgehog and group 3 subtypes, both of which are often fatal with existing chemotherapy and radiation treatments. Even when successful, the standard treatments can lead to life-long developmental side effects. Dr. Haydar hopes to show that the combined treatment improves survivorship, and she anticipates expanding her work to other types of pediatric brain tumors.

The $600,000 award shows the importance of well-resourced research institutions uniquely focused on pediatrics. Dr. Haydar noted that Children’s National is one of few research hospitals in the country with the necessary laboratory and ultrasound equipment on-site to develop a customizable treatment protocol. “We’re not just proposing to test this as a science project,” she said. “We’re proposing something that we can very well translate into the clinic and into a new approach for pediatric cancer patients.”

What we hope to discover

Through congressional funding, the Defense Department – one of the largest providers of pediatric healthcare for U.S. children – looks for opportunities to support the needs of military personnel and their dependents. Much of Dr. Haydar’s work will focus on determining safety and optimizing the delivery of the two treatments.

“You have to find the ideal timeline,” Dr. Haydar said. “Which therapy do you do first? How much spacing is best between them, and when do you repeat the therapy? What is the best dosing? It’s a puzzle, and I am hoping to solve it to give children with these often-fatal diagnoses a longer life – and a better one than existing treatments provide.”

researchers in a lab at Children's National

Pioneering gene therapy as a treatment for sickle cell disease

Gene therapy is a new and exciting treatment option available for patients with sickle cell disease (SCD). Children’s National Hospital is one of the few pediatric hospitals in the country that offers both FDA-approved sickle cell disease gene therapies: CASGEVY™ (exagamglogene autotemcel) and LYFGENIA™ (lovotibeglogene autotemcel).

What this means

Gene therapy involves an autologous transplant, taking the patient’s own stem cells, genetically changing those stem cells and then, after chemotherapy, infusing those stem cells back into the patient to make healthy blood.

“I’m excited about gene therapy for sickle cell disease. I think it has the potential to be a curative option for every single child with sickle cell disease,” said Robert Nickel, M.D., hematologist at Children’s National.

Currently both treatments are only approved for patients 12 years and older with severe disease. Children’s National was the first hospital in the world to collect stem cells for the LYFGENIA™ treatment.

Moving the field forward

Clinical trials hold incredible promise to advance the care of SCD. Children’s National continues to pioneer transplant therapies to cure SCD and is one of the leading centers participating in clinical trials of new treatments for this condition.

Experts at Children’s National are leading a multi-site clinical trial of a chemotherapy-free transplant approach for SCD using a matched sibling donor. This chemotherapy-free approach has less toxicity and side effects for children undergoing transplant.

In addition, Children’s National has been leading the way with innovative approaches to support sickle cell patients. “We’re providing alternative approaches to pain such as healing touch, acupuncture, massage VR technology, physical therapy and exploring other ways of treating pain in an integrated manner,” said Andrew Campbell, M.D., director of the Comprehensive Sickle Cell Disease Program at Children’s National.

The team is also exploring non-opioid treatments, such as intravenous citrulline, a naturally occurring amino acid that has been proven to enhance blood flow and potentially alleviate pain in treated patients in preliminary studies under the direction of Suvankar Majumdar, M.D., chief of Hematology at Children’s National.

illustration of sickled blood cells

Children’s National experts showcase sickle cell disease research

illustration of sickled blood cellsAndrew Campbell, M.D., director of the Comprehensive Sickle Cell Disease program, assessed the lifetime value of cell and gene therapy (CGTS) through a case study at The American Society of Gene and Cell Therapy’s (ASGCT) Annual Meeting.

Dr. Campbell and other Children’s National researchers will be presenting again at the Foundation for Sickle Cell Disease Research’s (FSCDR) Annual Sickle Cell Disease Research and Educational Symposium, June 7-9, 2024. The symposium includes more than 500 leading researchers, physicians, clinicians and social workers from all over the world.

Here’s a look at the presentations from Children’s National:

Day Time Presenter(s) Title
Sunday, June 9, 2024 8:00 AM – 9:15 AM Andrew Campbell, M.D. Update on Sickle Cell Legislation in the US

 
Sunday, June 9, 2024 3:00 PM – 3:15 PM Andrew Campbell, M.D., Deepika Darbari, M.D., and Regine Hyppolite, MSA Diagnostic Potential of Platelet-Neutrophil Ratio (PNR) for Stroke Risk in SCD Children

 
Sunday, June 9, 2024 3:15 PM – 3:30 PM

 

 Andrew Campbell, M.D. A Pilot Study to Increase Naloxone Education and Prescriptions in Sickle Cell Clinics

 
Sunday, June 9, 2024 4:00 PM – 4:15 PM Steven Hardy, Ph.D. Correlation Between VOC and Cognitive Function Using The NIH ToolBox in SCD
Sunday, June 9, 2024 4:15 PM – 4:30 PM

 

 

 Andrew Campbell, M.D., Deepika Darbari, M.D., and Regine Hyppolite, MSA Platelet to neutrophil ratio as a novel marker for monitoring SCD patients on hydroxyurea