Tag Archive for: LIFU

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Children’s National in the News: 2023

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Explore some of the notable medical advancements and stories of bravery that defined 2023, showcasing the steadfast commitment of healthcare professionals at Children’s National Hospital and the resilient spirit of the children they support. Delve into our 2023 news highlights for more.

1. COVID during pregnancy dramatically increases the risk of complications and maternal death, large new study finds

According to a study published in British Medical Journal Global Health, women who get COVID during pregnancy are nearly eight times more likely to die and face a significantly elevated risk of ICU admission and pneumonia. Sarah Mulkey, M.D., prenatal-neonatologist neurologist, discussed findings based on her work with pregnant women and their babies.
(Fortune)

2. Rest isn’t necessarily best for concussion recovery in children, study says

A study led by Christopher Vaughan, Psy.D., pediatric neuropsychologist, suggests that — despite what many people may presume — getting kids back to school quickly is the best way to boost their chance for a rapid recovery after a concussion.
(CNN)

3. Pediatric hospital beds are in high demand for ailing children. Here’s why

David Wessel, M.D., executive vice president, chief medical officer and physician-in-chief, explained that one reason parents were still having trouble getting their children beds in a pediatric hospital or a pediatric unit after the fall 2022 respiratory surge is that pediatric hospitals are paid less by insurance.
(CNN)

4. Anisha Abraham details impact of social media use on children: ‘True mental health crisis’

Anisha Abraham, M.D., M.P.H., chief of the Division of Adolescent and Young Adult Medicine, joined America’s Newsroom to discuss the impact social media access has had on children’s mental health.
(FOX News)

5. Saving Antonio: Can a renowned hospital keep a boy from being shot again?

After 13-year-old Antonio was nearly killed outside his mom’s apartment, Children’s National Hospital went beyond treating his bullet wounds. Read how our Youth Violence Intervention Program team supported him and his family during his recovery.
(The Washington Post)

6. Formerly conjoined twins reunite with doctors who separated them

Erin and Jade Buckles underwent a successful separation at Children’s National Hospital. Nearly 20 years later they returned to meet with some of the medical staff who helped make it happen.
(Good Morning America)

7. Asthma mortality rates differ by location, race/ethnicity, age

Shilpa Patel, M.D., M.P.H., medical director of the Children’s National IMPACT DC Asthma Clinic, weighed in on a letter published in Annals of Allergy, Asthma & Immunology, asserting that the disparities in mortality due to asthma in the United States vary based on whether they occurred in a hospital, ethnicity or race and age of the patient.
(Healio)

8. How one Afghan family made the perilous journey across the U.S.-Mexico border

After one family embarked on a perilous journey from Afghanistan through Mexico to the U.S.-Mexico border, they eventually secured entry to the U.S. where Karen Smith, M.D., medical director of Global Services, aided the family’s transition and provided their daughter with necessary immediate medical treatment.
(NPR)

9. When a child is shot, doctors must heal more than just bullet holes

With the number of young people shot by guns on the rise in the U.S., providers and staff at Children’s National Hospital are trying to break the cycle of violence. But it’s not just the physical wounds though that need treating: young victims may also need help getting back on the right track — whether that means enrolling in school, finding a new group of friends or getting a job.
(BBC News)

10. This 6-year-old is a pioneer in the quest to treat a deadly brain tumor

Callie, a 6-year-old diagnosed with diffuse intrinsic pontine glioma, was treated with low-intensity focused ultrasound (LIFU) at Children’s National Hospital and is the second child in the world to receive this treatment for a brain tumor. LIFU is an emerging technology that experts like Hasan Syed, M.D., and Adrianna Fonseca, M.D., are trialing to treat this fatal childhood brain tumor.
(The Washington Post)

11. F.D.A. approves sickle cell treatments, including one that uses CRISPR

The FDA approved a new genetic therapy, giving people with sickle cell disease new opportunities to eliminate their symptoms. David Jacobsohn, M.B.A., M.D., confirmed that Children’s National Hospital is one of the authorized treatment centers and talked about giving priority to the sickest patients if they are on Vertex’s list.
(The New York Times)

12. 6-year-old fulfils wish to dance in the Nutcracker

After the potential need for open-heart surgery threatened Caroline’s Nutcracker performance, Manan Desai, M.D., a cardiac surgeon, figured out a less invasive procedure to help reduce her recovery time so she could perform in time for the holidays.
(Good Morning America)

child in hospital bed

$96 million philanthropic investment will transform rare pediatric brain tumor research and care

child in hospital bedChildren’s National Hospital announced a $96 million investment from an anonymous donor family to transform rare childhood brain tumor research and care. The donation, which strengthens our globally recognized leadership in the field, is one of the largest in the hospital’s history.

Children’s National will harness the investment to recruit more top talent and advance the most promising research. This will produce safer, more effective treatments. It also will elevate standards of care to help children with rare brain tumors thrive for a lifetime.

The big picture

Brain tumors are the most common solid tumors affecting children. They are especially challenging in kids because their brains are still developing. The disease and current treatments can put them at risk for lifelong complications.

The anonymous family’s investment provides new hope for patients who face rare and often challenging brain tumor diagnoses — in the Washington, D.C., community and around the world.

“This incredible partnership will lift up one of the nation’s top pediatric brain tumor programs into the stratosphere,” said Kurt Newman, M.D., president and CEO of Children’s National. “It will immediately propel our best-in-class research and care, allowing us to bring new therapies to children with brain tumors. This fundamentally changes the healthcare journey and long-term outcomes for children and their families.”

Why it’s important

This transformational investment will have a far-reaching impact on our ability to save and improve the lives of children with brain tumors. Funds will fuel collaborative breakthroughs across a range of scientific and psychosocial approaches.

The partnership will supercharge highly individualized and promising treatments for children with brain tumors. We will radically transform the research landscape with a focus on:

  • Low intensity focused ultrasound (LIFU) – Advancing laboratory research and a clinical program designed to treat childhood brain tumors with LIFU therapy
  • Cellular immunotherapy – Testing new gene-engineered immune cell products and accelerating their integration into standards of care
  • Rare Brain Tumor Program – Propelling new clinical trials through the hospital’s national and global leadership in pediatric brain tumor consortia. Already, Children’s National is leading a new collaborative with hospitals in North America, South America and Europe to better understand and find novel treatments for these rare diseases
  • Neurosurgery innovation – Exploring multiple ways to perform safer, more effective neurosurgery and developing new methods to enhance drug/agent delivery
  • Precision medicine – Recruiting leading scientists to advance biology-informed therapies that can be targeted for children across a spectrum of brain tumors
  • Good Manufacturing Practices (GMP) facility – Expanding our GMP, one of the first standalone facilities at a children’s hospital in the country, to translate new discoveries into clinical trials more rapidly
  • Additional priorities including expansion of clinical research infrastructure and growth of bioinformatics, brain tumor repository and molecular diagnostics initiatives

The partnership also transforms how we approach care. It will power our pursuit of psychosocial, behavioral health and neuroscientific initiatives to help kids live well and cope with the unique circumstances of their diagnosis. We will focus on:

  • Lifetime health and wellness – Building a world-class research and clinical care program to shape a new paradigm for supporting a child’s physical and emotional health during and long after cancer treatment
  • Child Mental Health & Behavioral Brain Tumor Lab – Establishing a robust neuro-oncology mental health program that delivers timely interventions and specialized psychiatric care for patient well-being
  • Additional priorities including a new Neuroscience Nursing Excellence Program and growth of psychosocial support activities that bring comfort and encouragement to children during their treatment journey

Children’s National is proud to lead the way to a better future for pediatric rare brain tumor patients and expand our internationally recognized capabilities for neuro-oncology care.

illustration of a brain's neural activity

Debuting sonodynamic therapy with ALA to treat rare brain tumors

illustration of a brain's neural activity

Preclinical studies show that guided focused ultrasound and ALA can slow growth of gliomas and extend survival.

Children’s National Hospital is conducting a first-in-human study of aminolevulinic acid (ALA) sonodynamic therapy (SDT) for diffuse intrinsic pontine glioma (DIPG).

Preclinical studies led by experts at Children’s National have shown that SDT through MR guided focused ultrasound (MRgFUS) to activate protoporphyrin IX (PpIX), an ALA, can slow growth of gliomas and extend survival in animal models.

In a recently published technical communication in the Journal of Neuro-Oncology, the authors briefly detail the rationale and mechanism behind the use of SDT using ALA for DIPG, review criteria for patient inclusion, and describe the first patient selected for this clinical trial.

“Diffuse intrinsic pontine glioma (DIPG) is a devastating pediatric brain tumor that occurs in children between 2 and 9 years of age,” writes Hasan Syed, M.D., co-director of the Focused Ultrasound Program at Children’s National and lead author of the findings. “Despite standard therapy, prognosis remains poor with an average survival of 9–12 months after diagnosis.”

Future procedures will involve ascending drug and low-intensity focused ultrasound (LIFU) energy dose combinations with evaluations of pharmacokinetics and radiographic evidence of tumor physiological changes.

illustration of how LIFU works

Understanding the use of focused ultrasound in pediatrics

The fundamental principle of focused ultrasound (FUS) is almost analogous to using a magnifying glass to focus beams of sunlight on a single point. Experts at Children’s National Hospital are using FUS as an acoustic lens that uses multiple intersecting beams and targets — specifically deep within the brain — to treat brain tumors in pediatric patients.

Hasan Syed, M.D., co-director of the Focused Ultrasound Program at Children’s National, explains how two FUS methods are currently being used in two different trials — sonodynamic therapy and blood-brain barrier disruption — for the first time in pediatrics.

What is focused ultrasound?

FUS has diverse biological effects that can be categorized as thermal or mechanical: high-intensity focused ultrasound (HIFU) and low-intensity focused ultrasound (LIFU).

The treatments: 5-ALA with sonodynamic therapy and microbubbles for blood-brain barrier disruption

The difference between 5-aminolevulinic acid (5-ALA) medication and microbubbles has to do with the mechanism of treatment.

Dr. Syed explains that 5-ALA is activated by the focused ultrasound. Once activated, the goal is that the drug leads to tumor cell death.

Microbubbles, however, are used specifically to open the blood-brain barrier with focused ultrasound. When that happens, medications — or in our case the chemotherapy agents we’re using in our clinical trial — will hopefully have a better effect on treating the patient and taking care of the tumor.

Children’s National has now treated a series of patients with sonodynamic therapy — or LIFU and 5-ALA. There haven’t been any adverse events — the first time in the world that something like this has happened.

“I think it’s very exciting, and it brings us hope for new treatment options,” Dr. Syed said. Children’s National continues to recruit patients for this trial.

Abstract Happy 2022 New Year greeting card with light bulb

The best of 2022 from Innovation District

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

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

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

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

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

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

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

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

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

5. New telehealth command center redefines hospital care

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

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

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

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

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

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

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

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

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

10. 2022: Pediatric colorectal and pelvic reconstructive surgery today

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

Jeffrey Dome

Jeffrey Dome, M.D.: Making strides in the fight against pediatric cancer

Jeffrey DomeJeffrey Dome, M.D., Ph.D., senior vice president of the Center for Cancer and Blood Disorders and chief of the Division of Oncology (ranked number 6 in the nation by U.S. News & World Report 2022-23 Best Children’s Hospitals annual rankings) at Children’s National Hospital in Washington, D.C., 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, which oversees clinical research on kidney tumors at more than 200 children’s hospitals around the world for more than 10 years. Dr. Dome is currently the Continental President of North America for the International Society of Paediatric Oncology (SIOP) and serves on several medical advisory boards for cancer centers and foundations.

“This is a remarkably exciting time to be in the field of pediatric oncology, with an explosion of knowledge on cancer biology and genetics and the availability of new treatment modalities including molecularly targeted therapy, immunotherapy and devices to improve drug delivery and local control,” says Dome. “I am proud of the multidisciplinary and cross-center collaborations at Children’s National to deliver the latest innovative therapies.”

The team at Children’s National is making strides across all programs to benefit patients with pediatric cancer. A few highlights include:

  • The Brain Tumor lnstitute is one of the most active clinical and translational research programs in the country. Collaborating with other leading institutions, the Brain Tumor Institute is supported by a robust brain tumor bench research program with focused laboratories in medulloblastoma, high-grade glioma, midline diffuse glioma, diffuse intrinsic pontine glioma, low-grade glioma and immunotherapy. The Brain Tumor Institute is leading two national studies, both funded through the Moon Shot lnitiative. In addition, it works closely with the Virginia Tech brain tumor laboratories on the new Children’s National Research & Innovation Campus.
  • Children’s National is the first children’s hospital in the United States with a Focused Ultrasound Program. This pediatric dedicated program includes high-intensity (HIFU) and low-intensity focused ultrasound (LIFU), offering minimally invasive surgical options for children with extra-cranial solid tumors, low-grade brain tumors and novel, potentially life-saving therapy with LIFU-mediated blood-brain barrier disruptions for diffuse intrinsic pontine gliomas.
  • Children’s National has developed multi-antigen specific T cells that have shown success in early phase clinical trials for leukemias, solid tumors and brain tumors. This promising area of research earned a major boost in the form of a $25 million dollar grant from Cancer Grand Challenges, founded in 2020 by Cancer Research UK and the National Cancer Institute in the U.S. This award supported the foundation of NexTGen, a team of scientists and clinicians with expertise in immunology, proteomics, mathematics and more, across eight institutions in the U.S., U.K. and France. The Center for Cancer and Immunology Research at Children’s National is one of the leaders of this effort.
  • The Blood and Marrow Transplantation team, one of the only dedicated pediatric bone marrow transplant programs in the greater Washington, D.C., region, is celebrating its 35th anniversary, with a history of clinical and research accomplishments for both malignant and non-malignant disorders. This program has seen tremendous success in their day 100 transplant-related mortality (TRM). Recently, for the first time, the day 100 TRM average was 0%, meaning that the program did not lose a patient due to transplant complications in the first 100 days – a remarkable achievement in the world of transplantation.
  • The Cancer Genetics Program has grown tremendously in the past few years, reflecting recognition that approximately 10% of childhood cancers have an underlying cancer predisposition. Despite COVID-19, during the past fiscal year, there were 282 patient visits which is a 40% increase from the prior year. The team has developed a collaboration with researchers in the Rare Disease Institute and now can offer studies for patients with Beckwith-Wiedemann syndrome, children with previously undiagnosed developmental delay and children with undiagnosed syndromes. Further, the team was awarded a grant from the Children’s Cancer Foundation to allow testing for those without insurance coverage.
illustration of the brain

LIFU successfully delivers targeted therapies past the blood-brain barrier

illustration of the brain

LIFU offers doctors the first opportunity to open the blood-brain barrier and treat the entire malignant brain tumor.

Children’s National Hospital will leverage low-intensity focused ultrasound (LIFU) to deliver therapy directly to a child’s high-grade glioma. The approach offers doctors the first opportunity to open the blood-brain barrier and treat the entire malignant brain tumor.

Children’s National will be the first hospital in the U.S. to treat high-grade pediatric brain tumors with LIFU to disrupt the blood-brain barrier. Crossing it has been a major hurdle for effective therapy. The barrier, a network of blood vessels and tissue, prevents harmful substances from reaching the brain but also stops molecular targeted therapy and immunotherapy from getting into the tumor site and staying there.

“LIFU gives us a way to potentially transiently open up the barrier, so we can deliver novel therapy directly to the tumor and improve the likelihood of survival,” said Roger Packer, M.D., senior vice president of the Center for Neurosciences and Behavioral Medicine at Children’s National. “It is the greatest breakthrough we’ve potentially had in the past 50 years or more for the management of these tumors. We made great strides in our understanding of molecular genetics and the molecular drivers of tumors, but we have not yet translated that knowledge into better therapies; this may be our most effective mechanism to overcome the barrier.”

In 2020, Children’s National was recognized as the first worldwide Center of Excellence by the Focused Ultrasound Foundation.

Focused ultrasound (FUS) is a non-invasive therapeutic technology with the potential to transform the treatment of many medical disorders by using ultrasonic thermal energy to specifically target tissue deep in the body. The technology can treat without incisions or the need of radiation.

How it works

Doctors at Children’s National will be using LIFU in two different types of procedures:

  • 5-ALA: Doctors will give the patient 5-aminolevulinic acid (5-ALA) with the LIFU treatment. 5-ALA enters rapidly dividing cells and is activated by the ultrasound to a state where it kills the dividing cells of the tumor. The surrounding normal brain cells around the tumor are not dividing, so they do not take up the 5-ALA and are left unharmed after ultrasound therapy.
  • Microbubbles: While receiving different doses of LIFU over a one- to two-hour period, the patient is given “microbubbles,” which are widely used in medical imaging and as carriers for targeted drug delivery. These microbubbles bounce around against the walls like seltzer, opening the blood vessels and transiently opening that space.

Both studies are the first in the world for pediatric gliomas of the brain stem, allowing experts to treat patients 4-6 weeks after radiotherapy. The patient then receives medication orally or intravenously as it passes through the bloodstream. It does not go at high levels anywhere within the brain except where the blood-brain-barrier was opened, allowing oral medication or immune therapies to rush into the tumor.

The launch of this program comes a few months after the hospital successfully performed the first-ever high-intensity focused ultrasound surgery on a pediatric patient with neurofibromatosis.

Watch this video to learn more.

Drs. Packer and van den Acker at the Pediatric Device Innovators Forum

Pediatric Device Innovators Forum explores state of focused ultrasound

For children living with pediatric tumors, less invasive and less painful treatment with no radiation exposure was not always possible. In recent years, the development of technologies like Magnetic resonance guided high intensity focused ultrasound (MR-HIFU) and Low intensity transcranial focused ultrasound (LIFU) is helping to reverse that trend.

This topic was the focus of the recent Pediatric Device Innovators Forum (PDIF) hosted by the National Capital Consortium for Pediatric Device Innovation (NCC-PDI) in partnership with the U.S. Food and Drug Administration’s (FDA) Pediatric Device Consortia (PDC) grant program. A collaboration between Children’s National Hospital and University of Maryland Fischell Institute for Biomedical Devices, NCC-PDI is one of five PDCs funded by the FDA to support pediatric device innovators in bringing more medical devices to market for children.

The discussion, moderated by Kolaleh Eskandanian, Ph.D., MBA, PMP, vice president and chief innovation officer at Children’s National and principal investigator of NCC-PDI, explored the use of focused ultrasound’s noninvasive therapeutic technology for two pediatric indications, Osteoid Osteoma (OO) and Diffuse Intrinsic Pontine Glioma (DIPG), and the ways it can increase the quality of life for pediatric patients while also decreasing the cost of care.

The discussion also examined the most common barriers preventing more widespread implementation of focused ultrasound technology, specifically small sample size for evidence generation, lack of funding opportunities and reimbursement issues that can make or break a technology’s chances at reaching the patients that need it.

Karun Sharma, M.D., director of Interventional Radiology at Children’s National, emphasized the potential for focused ultrasound to treat localized pain relief and treat other diseases that, like OO, do not have any other therapeutic alternative

“At Children’s National, we use MR-HIFU to focus an ultrasound beam into lesions, usually tumors of the bone and soft tissues, to heat and destroy the harmful tissue in that region, eliminating the need for incisions,” says Sharma. “In 2015, Children’s National doctors became the first in the U.S. to use MR-HIFU to treat pediatric osteoid osteoma (OO), a painful, but benign, bone tumor that commonly occurs in children and young adults. The trial demonstrated early success in establishing the safety and feasibility of noninvasive MR-HIFU in children as an alternative to current, more invasive approaches to treat these tumors.”

In November 2020, the FDA approved this MR-HIFU system to treat OO in pediatric patients.

Roger Packer, M.D., senior vice president of the Center for Neuroscience and Behavioral Medicine at Children’s National, also discussed how focused ultrasound, specifically LIFU, has also proven to be an attractive modality for its ability to non-invasively, focally and temporarily disrupt the blood brain barrier (BBB) to allow therapies to reach tumors that, until recently, would have been considered unreachable without severe intervention.

“This presents an opportunity in pediatric care to treat conditions like Diffuse Intrinsic Pontine Glioma (DIPG), a highly aggressive brain tumor that typically causes death and morbidity,” says Packer.

Packer is planning a clinical trial protocol to investigate the safety and efficacy of LIFU for this pediatric indication.

The forum also featured insight from Jessica Foley, M.D., chief scientific officer, Focused Ultrasound Foundation; Arjun Desai, M.D., chief strategic innovation officer, Insighttec; Arun Menawat, M.D., chairman and CEO, Profound Medical; Francesca Joseph, M.D., Children’s National; Johannes N. van den Anker, M.D., Ph.D., vice chair of Experimental Therapeutics, Children’s National; Gordon Schatz, president, Schatz Reimbursement Strategies; Mary Daymont, vice president of Revenue Cycle and Care Management, Children’s National; and Michael Anderson, MD, MBA, FAAP, FCCM, FAARC, senior advisor to US Department of Health and Human Services (HHS/ASPR) and Children’s National.

Anthony Sandler, M.D., senior vice president and surgeon-in-chief of the Joseph E. Robert Jr. Center for Surgical Care and director of the Sheikh Zayed Institute for Pediatric Surgical Innovation at Children’s National Hospital, and Sally Allain, regional head of Johnson & Johnson Innovation, JLABS @ Washington, DC, opened the forum by reinforcing both organizations’ commitment to improving pediatric health.

In September 2020, the Focused Ultrasound Foundation designated Children’s National Hospital as the first global pediatric Center of Excellence for using this technology to help patients with specific types of childhood tumors. As a designated COE, Children’s National has the necessary infrastructure to support the ongoing use of this technology, especially for carrying out future pediatric clinical trials. This infrastructure includes an ethics committee familiar with focused ultrasound, a robust clinical trials research support team, a data review committee for ongoing safety monitoring and annual safety reviews, and a scientific review committee for protocol evaluation.

The Pediatric Device Innovators Forum is a recurring collaborative educational experience designed by the FDA-supported pediatric device consortia to connect and foster synergy among innovators across the technology development ecosystem interested in pediatric medical device development. Each forum is hosted by one of the five consortia. This hybrid event took place at the new Children’s National Research and Innovation Campus, the first-of-its-kind focused on pediatric health care innovation, on the former Walter Reed Army Medical Center campus in Washington, D.C.

To view the latest edition of the forum, visit the NCC-PDI website.

Panelists at the Pediatric Device Innovators Forum

The recent Pediatric Device Innovators Forum (PDIF) exploring the state of focused ultrasound was held at the new Children’s National Research and Innovation Campus, a first-of-its-kind focused on pediatric health care innovation.

Roger Packer with patient

A lifetime of achievements: Roger Packer, M.D.

Roger Packer with patient

Over the years, Dr. Packer and his team in Washington, D.C., have made meaningful contributions to children all around the world diagnosed with childhood brain tumors, including medulloblastoma and gliomas.

Earlier in December, Roger Packer, M.D., senior vice president of the Center for Neurosciences and Behavioral Medicine at Children’s National Hospital, received the 2020 Lifetime Achievement Award from the International Symposium on Pediatric Neuro-Oncology at the meeting organized in Karuizawa, Japan. The prestigious recognition is a testament to the years of commitment and dedication Dr. Packer has devoted to the care of children with brain tumors and as such, have placed him as a top leader.

This award is a recognition of how the field has grown since the first International Symposium on Pediatric Neuro-Oncology Dr. Packer organized in Seattle in 1989. “It grew from a small gathering of investigators to now a multidisciplinary group of over 2,000 investigators,” Dr. Packer says.

Over the years, Dr. Packer and his team in Washington, D.C., have made meaningful contributions to children all around the world diagnosed with childhood brain tumors, including medulloblastoma and gliomas. These findings have contributed to an increase of the survival rate from 50% to over 80% for children with medulloblastoma. In addition, his contributions have led to newer molecular targeted therapies and improved the quality of life of children who are long-term survivors.

“The field, especially in the last decade, rapidly transitioned to a more biologically informed field,” Dr. Packer explains. “We are now utilizing new, exciting discoveries in biology and genetics to inform new approaches to treatment. This kind of transition gives us great hope for the future.”

In his early career, Dr. Packer worked with two neuro-oncology patients who died and would impact his decision to further study this field. At that time, there was minimal understanding of the nature of neuro-oncology diseases or how to best treat them. As a neurologist, he was frustrated by the lack of understanding and as a pediatrician, he was frustrated at the lack of ability to do success management.

“I saw this as a gap in my personal knowledge and found that the field was struggling to come up with new answers and new approaches,” he says. “But at the same time other, advances were being made in child cancer care, such as with leukemia. However, there was no wide focus on pediatric brain tumors.”

Combining his knowledge of neurology with his curiosity and relying on other leaders that surrounded him in the same field, Dr. Packer worked on driving this new work forward. Today, he is still heavily involved in the development of treatment protocols that are increasingly transitional for a variety of brain tumors, including low-grade and high-grade gliomas.

“With the help of our great colleagues at Children’s National, we continue to try to develop new means to treat these tumors, including immunological approaches and the incorporation in the use of novel means, such as low-intensity and high-intensity focused ultrasound,” he says. “We also have an excellent multidisciplinary team at Children’s National that has grown over the last decade some of whom are acknowledged national leaders in the fields of brain tumors, clinical research and clinical care. We also have a robust program focusing on the neurocognitive outcome of children and ways to intervene to ameliorate intellectual compromise and improve quality of life.”