Research & Innovation Campus Archives

CRISPR gene editing identifies possible drug targets for HIV

October 19, 2022

Working with researchers at Johns Hopkins University, the Children’s National team used CRISPR gene technology to test drug targets that find and attack latent HIV, paving the way for drug treatments that may someday completely cure the virus.

Researchers at Children’s National Hospital have identified several new drug targets that may enhance the elimination of latent HIV in patients, a major bottleneck to the full treatment of the virus, according to new findings published in Science Translational Medicine.

Working with researchers at Johns Hopkins University, the Children’s National team used CRISPR gene technology to test drug targets that find and attack latent HIV, paving the way for drug treatments that may someday completely cure the virus. Currently, anti-retroviral therapies (ARTs) can only slow its progress.

Why we’re excited

“In less than one month, we were able to use CRISPR to test 20,000 gene candidates in one single experiment. It was an amazing application of the technology,” said Wei Li, Ph.D., a co-author of the study and assistant professor at the Center for Genetic Medicine Research at Children’s National. “The CRISPR technology provides a global, unbiased approach to understanding molecular aspects of HIV-1 infection, including the ways that HIV-1 enters cells and replicates. This research could someday revolutionize how we treat the virus pharmaceutically.”

The big picture

More than 30 million people worldwide live with HIV-1, the most common form of the virus that can eventually lead to AIDS. But no single agent can entirely eliminate HIV-1 in these patients.

Researchers have sought ways to attack this elusiveness and turned to the CRISPR gene-editing tool, which can locate specific bits of DNA inside a cell. They trained CRISPR screens on the HIV-1 genome to identify critical factors that allow or prevent the virus from lying latent. In the latter case, these pieces of DNA will be the ideal targets of a drug that will push the virus out of the latent stage so it can be targeted by therapies.

What’s ahead

The findings of the Children’s National and Johns Hopkins scientists point to novel drug therapies and validation systems that could someday eradicate HIV.

Bicna Song, a postdoctoral researcher in Li’s laboratory at the Center for Genetic Medicine, said that reversing HIV-1 latency will allow for the killing of infected cells and give researchers opportunities to actually cure patients with HIV.

“So far, no single latency-reversing agent – alone or in combination with another drug – has been shown to effectively reduce the latent reservoir size in persons living with HIV-1,” said Song, who contributed to the study. “With this work, we are meeting the urgent need to identify factors that can lead to new drug targets.”

Innovations for health equity: Food pharmacy app wins Hackathon

September 29, 2022

When families come into the endocrinology clinic, 66% of prediabetes and Type 2 diabetes patients screen positive for food insecurity. One remedy: a smartphone app envisioned by Children’s National Hospital researchers to communicate with families between visits and provide resources to help stock pantries with nutritious foods.

The Children’s National Food Pharmacy app is on its way from idea to reality, thanks to the inaugural Health Equity in Research Hackathon event at the Children’s National Research & Innovation Campus. This team-based, “Shark Tank”-like competition involved roughly 50 experts designing creative healthcare solutions that could be delivered through ubiquitous smartphones.

“It takes a village to raise a child, and we want to show that at Children’s National we are part of that community,” said food pharmacy founder and diabetes educator Alexis Richardson, M.S., R.D., L.D.N., C.D.C.E.S.

Why it matters

The rate of new-onset Type 2 diabetes increased by a staggering 182% during the first nine months of the COVID-19 pandemic. Now, the Children’s National food pharmacy provides families that screen positive for food insecurity during quarterly clinic appointments with a 50-pound bag of medically-tailored groceries.

The new app, as envisioned, would follow them home to connect them with food bank information and other nutritional resources, eliminating paper forms and other hurdles that get in the way of care.

What’s ahead

Children’s National leaders are committed to making the proposal a reality. “We are going to support today’s winner through the next steps to prepare them to enter the app development pipeline at the Sheikh Zayed Institute,” said Lisa Guay-Woodford, M.D., director for the Clinical and Translational Science Institute at Children’s National (CTSI-CN) and one of the main judges of the competition.

The app development will happen in the months ahead. Kevin Cleary, Ph.D., technical director of the Sheikh Zayed Institute of Pediatric Surgical Innovation, said the Hackathon planted the seeds. “It really depends on the drive of the individual to see the idea to fruition,” Cleary told competitors.

Other app entries were encouraged to continue their work:

The Surgical Checklist, led by Brian K. Reilly, M.D., co-director of the Cochlear Implant Program: this app would help patients and providers successfully navigate the often-confusing pre-operative checklist, including required physical exams, lab work, imaging and pre-procedure fasting. Reilly said the hospital handles about 15,000 cases a year, and about 10% are rescheduled, often for reasons that could be avoided with digital organization and reminders for families.
More than Determined, led by Pediatrician Jessica Lazerov, M.D., M.B.A.: this app aims to give time-strapped providers a platform to better understand and address social determinants of health – such as access to safe housing, education and jobs – that can promote better preventative care outcomes.

The Health Equity in Research Hackathon was created by the new Health Equity in Research Unit, a joint initiative between the CTSI-CN and the Center for Translational Research within the Children’s National Research Institute.

Dr. Lisa Guay-Woodford and the winners of the Health Equity in Research Hackathon

Dr. Lisa Guay-Woodford, director for the Clinical and Translational Science Institute, joins the winners of the inaugural Health Equity in Research Hackathon: the Children’s National Food Pharmacy. The team’s proposed app will connect families facing food insecurity with resources and guidance for nutritious eating.

How the circadian clock could help the brain recover after injury

September 16, 2022

A type of brain cell that can renew itself is regulated by circadian rhythms, providing significant insights into how the body’s internal clock may promote healing after traumatic brain injuries (TBI).

A type of brain cell that can renew itself is regulated by circadian rhythms, providing significant insights into how the body’s internal clock may promote healing after traumatic brain injuries (TBI), according to new research from Children’s National Hospital.

Released in the latest issue of eNeuro, the findings open new avenues of investigation for future TBI therapies. These injuries are currently managed only with supportive care and rehabilitation, rather than targeted drug treatment options. The findings also underscore the importance of addressing circadian disturbances to help injured brains heal.

Many of the body’s cells follow a 24-hour rhythm driven by their genes known as the circadian clock. The Children’s National research team found that a relatively newly discovered type of brain cell – known as NG2-glia, or oligodendrocyte precursor cells – also follow a circadian rhythm. This cell type is one of the few that continually self-renews throughout adulthood and is notably proliferative in the first week after brain injuries.

“We have found evidence for the role of this well-known molecular pathway – the molecular circadian clock – in regulating the ability for these NG2-glia to proliferate, both at rest and after injury,” said Terry Dean, M.D., Ph.D., critical care specialist at Children’s National and the lead author of the paper. “This will serve as a starting point to further investigate the pathways to controlling cellular regeneration and optimize recovery after injury.”

Sometimes called “the silent epidemic,” TBI afflicts an estimated 69 million people worldwide each year, with injuries ranging from mild concussions to severe injuries that cause mortality or lifelong disability. In the United States alone, approximately 2.8 million people sustain TBI annually, including 630,000 children. TBI is the leading cause of death in people under age 45, and those who survive are often left with persistent physical, cognitive and psychological disabilities.

Yet no targeted therapies exist for TBI, creating a critical need to uncover the mechanisms that could unlock the regeneration of these NG2-glia cells, which are the most common type of brain cell known to proliferate and self-renew in adult brains.

“It is essential for researchers to know that cell renewal is coordinated with the time of day,” said Vittorio Gallo, Ph.D., interim chief academic officer and interim director of the Children’s National Research Institute. “With this knowledge, we can dig deeper into the body’s genetic healing process to understand how cells regulate and regenerate themselves.”

Children’s National Innovation Day aims to advance pioneering pediatric life science projects

August 23, 2022

Children’s National Research & Innovation Campus’ Innovation Day will feature life science projects focused on improving pediatric care.

Pioneering life science projects focused on improving pediatric care will be on display at the Children’s National Research & Innovation Campus when the hospital hosts its 2022 Innovation Day on Friday, August 26. Hosted by Children’s National Innovation Ventures, the program’s goal is to showcase life sciences and healthcare projects that are mature enough to look for a co-developer, strategic partner, investor or licensing vehicle.

“For us, a successful Innovation Day means we are able to match these entrepreneurs with the strategic partner they need at this stage of their device development journey,” says Kolaleh Eskandanian, Ph.D., M.B.A., P.M.P., vice president and chief innovation officer at Children’s National Hospital and executive director of the Sheikh Zayed Institute for Pediatric Surgical Innovation.

“Data continues to show that the national Capital Region (NCR) remains one of the most robust life sciences and technology hubs in the country with no shortage of visionary leaders. There is no better place to conduct this important work as we seek more ways to advance pediatric medical technologies to improve health outcomes.”

A total of 17 projects will be showcased at the event, with each having up to 10-minute window to make their presentation. All attendees will be provided a survey, enabling valuable feedback for presenters as they look to take their next strategic step on the pathway to further development and commercialization.

In addition to presenting projects, the 2022 Innovation Day will also feature startup companies whose mission is aligned with Children’s National’s quest to bring novel pediatric medical products to patients and families. Throughout the day, attendees will have the opportunity to meet with the hospital’s researchers, academic entrepreneurs and prominent stakeholders in the life science and healthcare innovation ecosystem in the region. Interested investors and strategics can also request one-on-one meetings with startups and research teams.

Eskandanian, who also serves as the executive director of the FDA-funded National Capital Consortium for Pediatric Device Innovation (NCC-PDI), notes that accelerating this pathway to commercialization for pediatric products will bring more viable technologies to market, an important step in addressing the ongoing development disparity in innovations developed for children versus adults.

“For too long, children have been left behind in the development and commercialization of medical products and we remain committed to altering the trend,” Eskandanian says. “Children’s National’s Innovation Ventures team is working closely with the presenting innovators to provide any and all support that we can to get these products to the next stage.”

Registration for Children’s National’s 2022 Innovation Day is currently open. Those interested in attending the day-long showcase can register at https://conta.cc/3CrAfTl.

Supporting the progress of pediatric innovators is a key focus of the new Children’s National Research & Innovation Campus, a one-of-its-kind ecosystem that drives discoveries that save and improve the lives of children. On a nearly 12-acre portion of the former, historic Walter Reed Army Medical Center in Northwest Washington, D.C., Children’s National has combined its strengths with those of public and private partners, including industry, universities, federal agencies, start-up companies and academic medical centers. The campus provides a rich environment of public and private partners which, like the NCC-PDI network, helps to bolster pediatric innovation and commercialization.

Five leading children’s hospitals secure funding to enhance future pandemic readiness

July 19, 2022

A group of children’s hospitals from across the country will coordinate on the response to future pandemics and other disasters through a new $29 million grant from the Health Resources and Services Administration (HRSA). The group, known as the Pediatric Pandemic Network (PPN), will focus on the unique needs and challenges to children during pandemics and disasters, ensuring that health equity is at the forefront of emergency planning.

The five hospitals are:

Children’s National Hospital in Washington, D.C. (lead institution)
Ann & Robert H. Lurie Children’s Hospital of Chicago
University of Alabama at Birmingham Department of Pediatrics at Children’s of Alabama
Children’s Mercy Kansas City
Seattle Children’s

“As the current pandemic has proven to the world, pandemics and public health crises magnify pre-existing environmental, health, social and economic inequities,” said Joelle Simpson, M.D., M.P.H., principal investigator of this grant and division chief of Emergency Medicine and medical director of Emergency Preparedness at Children’s National. “Communities of color not only feel the impact of pandemics and disasters far more severely than others, but also have more difficulty obtaining aid and assistance. If the needs of vulnerable populations are not addressed in emergency planning, the national disaster preparedness strategy could fail for all.”

The hospitals recognize that while the COVID-19 pandemic has been devastating for the entire world, the pediatric population in particular has been challenged by a lack of established coordination among pediatric care providers. In addition to addressing health equity, the funding facilitates the following:

Establishing pathways to gather and disseminate research-informed insights into how to care for children in a future pandemic to both medical providers and community organizations.
Developing a telehealth infrastructure to support the care of acutely ill children and expand mental health access.
Expanding pediatric-focused emergency preparedness and planning with a focus on behavioral health, social support, and educational services, all of which are typically provided by community organizations.

The hub for this grant will be located at the newly opened Children’s National Research & Innovation Campus – the site of the former Walter Reed Army Medical Center in Washington, D.C. Work will take place in the renovated Armed Forces Institute of Pathology, where past investigators made seminal discoveries in infectious diseases and pandemics. The former campus was named after Walter Reed because of his work around discovering the cause and prevention of outbreaks of yellow fever. This modern-day team of physician researchers intends to make equally important contributions to future pandemics and other disasters to help preserve the health of our nation’s children.

In September 2021, HRSA launched the Regional PPN by funding five pediatric hospitals to support the planning and preparation of children’s hospitals to respond to a global health threat. This new grant doubles the size and reach of the network in order to benefit all children in the nation.

Marshall Summar, M.D., receives Lifetime Achievement Award for rare disease work

June 27, 2022

For making strides to improve the lives of the rare disease community, the National Organization for Rare Disorders (NORD®) recognized Marshall Summar, M.D., with a Lifetime Achievement Award.

For making strides to improve the lives of the rare disease community, the National Organization for Rare Disorders (NORD®) recognized Marshall Summar, M.D., chief of the Division of Genetics and Metabolism and the director of the Rare Disease Institute at Children’s National Hospital, with a Lifetime Achievement Award.

This award honors individuals for outstanding career-long achievement on behalf of the rare disease community and commitment to improving the lives of those affected by rare diseases. It has been presented only a few times over NORD’s nearly 40-year history, most recently to former NIH Director Francis Collins, M.D., Ph.D., in 2015 and to clinician and researcher Robert Campbell, M.D., of Children’s Hospital of Philadelphia in 2018.

“I am honored to receive this award from NORD. It is so special to be recognized by the leading rare disease organization. This award comes from the work of so many people over the years, particularly our great team at Children’s National,” said Dr. Summar. “This acknowledgement of what we have done to date just gets me more excited about the future!”

Dr. Summar developed and launched the world’s first Rare Disease Institute at Children’s National in 2017, which is now located on the Children’s National Research & Innovation Campus, a first-of-its-kind pediatric research and innovation hub in Washington, D.C.

The institute, which includes the largest clinical group of pediatric geneticists in the nation, focuses on developing the clinical care field of the more than 8,000 rare diseases currently recognized and advancing the best possible treatments for children with these diseases.

Marshall and Karen Summar.

“Dr. Summar’s passion for serving patients is at the core of everything he does,” said Debra Regier, M.D., medical director of the Rare Disease Institute. “His mentorship for the next generation of medical and biochemical geneticists has become his legacy.”

The work Dr. Summar has done over the course of his career has resulted in new drugs in FDA trials for patients with congenital heart disease and premature birth. He also holds more than 60 patents and has published more than 160 peer-reviewed research studies.

“Beginning with his work as a clinician in the 1980s, Dr. Marshall Summar has spent a career forging partnerships, advocating at the highest level and developing new ways to treat rare disease patients,” said Peter L. Saltonstall, president and CEO of NORD.

“Dr. Summar served on the NORD Board of Directors for nine years, including six years as Chairman, and so we at NORD have been lucky enough to have years of firsthand experience with his leadership, community-building and innovation efforts in the rare disease field. This award is a recognition and appreciation for sustained excellence, including critical work with organizations such as the American College of Medical Genetics, the National Institutes of Health, NORD, and the Rare Disease Institute at Children’s National. For decades of commitment to families and organizations combating rare diseases, NORD is thrilled to present the Lifetime Achievement Award to Dr. Marshall Summar at the 2022 Rare Impact Awards,” Saltonstall added.

Learn more about the Rare Disease Institute at Children’s National.

Medical device pitch competition returns with $150K in FDA awards

April 12, 2022

“This pitch competition helps to recognize and support the advancement of innovations that can specifically address the needs of pediatric patients,” says Kolaleh Eskandanian, Ph.D., M.B.A., P.M.P., vice president and chief innovation officer at Children’s National Hospital and principal investigator of NCC-PDI.

Recognizing the continued gap in the development and commercialization of medical devices for children versus adults, the National Capital Consortium for Pediatric Device Innovation (NCC-PDI), in collaboration with MedTech Innovator, is accepting applications through April 22, 2022, for its annual “Make Your Medical Device Pitch for Kids!” competition. Recognizing the wide range of unmet needs for diagnostic and therapeutic devices designed especially for children, this year’s competition is open to any innovation in medical technology that addresses a significant unmet need in pediatric medical care.

“As one of the five FDA Pediatric Device Consortia, NCC-PDI is focused on seeking out and addressing significant unmet needs in pediatric medical technology,” says Kolaleh Eskandanian, Ph.D., M.B.A., P.M.P., vice president and chief innovation officer at Children’s National Hospital and principal investigator of NCC-PDI. “While great advances are made in adult medical devices, children are often left behind because the pediatric market is small and there are not incentives to develop for pediatrics. This pitch competition helps to recognize and support the advancement of innovations that can specifically address the needs of pediatric patients.”

Using a virtual format, semi-finalists chosen from all submissions will make their first pitch on May 20, 2022. Up to six finalists selected from this first round will earn participation in a special pediatric-focused track of the MedTech Innovator accelerator program, the largest medical device accelerator in the world, beginning in June 2022. These innovators will then participate in the competition finals in the fall 2022 where judges will award up to $150,000 in FDA-sponsored grants to the devices selected as most impactful and commercially viable.

Unlike devices for adults, the development and commercialization of pediatric medical devices lags behind by approximately five to 10 years. Programs like the NCC-PDI pitch competition and MedTech Innovator accelerator program offer innovators access to expert insight and consultation to help overcome regulatory hurdles and advance the product’s development path.

NCC-PDI is one of five members in the FDA’s Pediatric Device Consortia Grant Program created to support the development and commercialization of medical devices for children. NCC-PDI is led by the Sheikh Zayed Institute for Pediatric Surgical Innovation at Children’s National and the A. James Clark School of Engineering at the University of Maryland with support from partners MedTech Innovator, BioHealth Innovation and design firm Archimedic.

Eskandanian adds that supporting the progress of pediatric innovators is a key focus of the new Children’s National Research & Innovation Campus, a one-of-its-kind ecosystem that drives discoveries that save and improve the lives of children. On a nearly 12-acre portion of the former, historic Walter Reed Army Medical Center in Northwest Washington, D.C., Children’s National has combined its strengths with those of public and private partners, including industry, universities, federal agencies, start-up companies and academic medical centers. The campus provides a rich environment of public and private partners which, like the NCC-PDI network, will help bolster pediatric innovation and commercialization.

Applications for the “Make Your Medical Device Pitch for Kids!” competition are open now through April 22 for innovations that address unmet pediatric needs.

Pediatric medical device competition takes aim at congenital heart disease

December 13, 2021

Consistent with its mission of addressing the most pressing pediatric device needs, this year’s competition focused on innovations in electrophysiology devices that monitor and treat congenital heart disease (CHD) and arrhythmias in pediatric patients.

The National Capital Consortium for Pediatric Device Innovation (NCC-PDI) announces five awardees chosen in its prestigious annual “Make Your Medical Device Pitch for Kids!” competition to share $150,000 in grant funding from the U.S. Food and Drug Administration (FDA) to support the advancement of pediatric medical devices. In an unprecedented decision, the competition judges determined that all five finalists were deserving of a grant award and recognition for the potential patient benefit and commercial viability of their innovations.

Consistent with its mission of addressing the most pressing pediatric device needs, this year’s competition, conducted by NCC-PDI partner MedTech Innovator, focused on innovations in electrophysiology devices that monitor and treat congenital heart disease (CHD) and arrhythmias in pediatric patients. The virtual pediatric pitch event was part of the 9^th Annual Symposium on Pediatric Device Innovation.

This year’s pediatric device innovation awardees are:

PeriCor – The Children’s Hospital at Montefiore – New York, NY, and Children’s National Hospital – PeriTorq, a catheter grip tool for use during pediatric cardiac interventional procedures;
Inkspace Imaging – Pleasanton, CA – a pediatric cardiac and vascular MRI coil;
Karios Technologies – Charlottesville, VA – Tissue Shield, a technology to prevent scar tissue formation (adhesions) on the heart after surgery;
Sibel – Niles, IL – ANNE One, ICU-grade wireless sensors for cardiopulmonary monitoring in neonates with congenital heart defects;
Starlight Cardiovascular – San Diego, CA – Project Lifeline, a less-invasive way to maintain sufficient circulation in newborns with ductal-dependent circulation that increases safety, procedural success and ease of use.

Congenital heart disease (CHD) affects six out of 1,000 babies born in the U.S. each year and is often complicated by arrhythmias, a condition where the heart beats too rapidly, too slowly or irregularly due to a misfiring of the body’s electrical impulses. While the last decade brought great advances in technologies that improve the care of adult arrhythmias, pediatric patients have been left behind, with only five devices approved for use in children in the same period. As a result, pediatric specialists are often using off-label or improvised devices to treat pediatric arrhythmias, including in the smallest newborns.

“Recognizing this unmet need, NCC-PDI opened the challenge earlier this year to select companies to enter MedTech Innovator’s pediatric accelerator program, made possible by NCC-PDI. The five companies have immensely benefited from the accelerator program and are well-positioned to compete for funding. They have the potential to advance pediatric health and provide a greater standard of care for children living with CHD,” says Kolaleh Eskandanian, Ph.D., M.B.A, P.M.P, vice president and chief innovation officer at Children’s National Hospital and principal investigator of NCC-PDI. “For too long, the unique needs of children have been overlooked in pediatric device development. Thanks to the support of the FDA, we are able to build our challenge competitions around the direst unmet needs, which are determined through a thorough needs assessment and market analysis conducted to inform each request for proposal. The funding incentivizes pediatric innovation and helps more companies navigate the path to commercialization.”

NCC-PDI is one of five consortia in the FDA’s Pediatric Device Consortia Grant Program created to support the development and commercialization of medical devices for children, which lags significantly behind the progress of adult medical devices. NCC-PDI is led by the Sheikh Zayed Institute for Pediatric Surgical Innovation at Children’s National Hospital and the A. James Clark School of Engineering at the University of Maryland, with support from partners MedTech Innovator, BioHealth Innovation and design firm Archimedic.

A pediatric accelerator program, powered by MedTech Innovator, is the consortium’s latest addition to a network of resources and experts that NCC-PDI provides in support of pediatric innovators. All five of this year’s competition finalists had an opportunity to participate in the year-long accelerator program.

Eskandanian adds that supporting the progress of pediatric innovators is a key focus of the new Children’s National Research & Innovation Campus, a one-of-its-kind ecosystem that drives discoveries that save and improve the lives of children. On a nearly 12-acre portion of the former, historic Walter Reed Army Medical Center in Northwest Washington, D.C., Children’s National has combined its strengths with those of public and private partners, including industry, universities, federal agencies, start-up companies and academic medical centers, the campus provides a rich environment of public and private partners which, like the NCC-PDI network, will help bolster pediatric innovation and commercialization.

Children’s National Rare Disease Institute named a Center of Excellence

November 4, 2021

RDI, which includes the largest clinical group of pediatric geneticists in the nation, focuses on developing the clinical care field of more than 8,000 rare diseases currently recognized and advancing the best possible treatments for children with these diseases.

The Rare Disease Institute (RDI) at Children’s National Hospital announced its designation as a NORD Rare Disease Center of Excellence, joining a highly select group of 31 medical centers nationwide. This new, innovative network seeks to expand access and advance care and research for rare disease patients in the United States. The program is being led by the National Organization for Rare Disorders (NORD), with a goal to foster knowledge sharing between experts across the country, connect patients to appropriate specialists regardless of disease or geography, and to improve the pace of progress in rare disease diagnosis, treatment and research.

“Children’s National has worked closely with NORD to move this program forward and is very proud to be amongst the first group of recognized centers,” said Marshall Summar, M.D., chief of the Division of Genetics and Metabolism and the director of RDI at Children’s National. “This is a recognition of the institutional efforts, as we take care of patients with the rare disease and help set the standard for the field.”

RDI, which includes the largest clinical group of pediatric geneticists in the nation, focuses on developing the clinical care field of more than 8,000 rare diseases currently recognized and advancing the best possible treatments for children with these diseases.

In February 2021, RDI became the first occupant of the new Children’s National Research & Innovation Campus, a first-of-its-kind pediatric research and innovation hub. The campus now also houses the Center for Genetic Medicine Research, and together researchers are constantly pursuing high-impact opportunities in pediatric genomic and precision medicine. Both centers combine its strengths with public and private partners, including industry, universities, federal agencies, start-up companies and academic medical centers. They also serve as an international referral site for rare disorders.

People living with rare diseases frequently face many challenges in finding a diagnosis and quality clinical care. In establishing the Centers of Excellence program, NORD has designated clinical centers across the U.S. that provide exceptional rare disease care and have demonstrated a deep commitment to serving rare disease patients and their families using a holistic, state of the art approach.

“Right now, far too many rare diseases are without an established standard of care. The Centers of Excellence program will help set that standard – for patients, clinicians, and medical centers alike,” said Ed Neilan, chief scientific and medical officer of NORD. “We are proud to announce Children’s National as a NORD Rare Disease Center of Excellence and look forward to their many further contributions as we collectively seek to improve health equity, care and research to support all individuals with rare diseases.”

Each center was selected by NORD in a competitive application process requiring evidence of staffing with experts across multiple specialties to meet the needs of rare disease patients and significant contributions to rare disease patient education, physician training and research.

Q&A with Dr. Javad Nazarian on his upcoming work on low-grade gliomas

October 28, 2021

Supported by the Gilbert Family Foundation, Dr. Nazarian’s return is part of a special research program within the Gilbert Family Neurofibromatosis Institute that focuses on NF1 research.

Javad Nazarian, Ph.D., M.Sc., associate professor of Pediatrics at George Washington University and professor at the University of Zurich, has expanded his research group at Children’s National to focus on Neurofibromatosis type 1 (NF1) transformed low-grade gliomas (LGGs). Dr. Nazarian will apply his expertise from establishing a successful DIPG (diffuse intrinsic pontine glioma) and DMG (diffuse midline glioma) program in Zurich Switzerland and previously at Children’s National.

In addition to his continued research in Zurich, as a principal investigator at the Department of Genomics and Precision Medicine at Children’s National Dr. Nazarian plans on aggregating his knowledge to the new research and work spearheaded at Children’s National. As one of the first research teams to move to the Children’s National Research & Innovation Campus, Dr. Nazarian’s group is excited to use the opportunity to establish cutting-edge and clinically translational platforms.

Supported by the Gilbert Family Foundation, Dr. Nazarian’s return is part of a special research program within the Gilbert Family Neurofibromatosis Institute that focuses on NF1 research. This research includes associated gliomas with a special emphasis on NF1-associated transformed anaplastic LGGs. His team will develop new avenues of research into childhood and young adult NF-associated LGGs with a special emphasis on transformed high-grade gliomas.

Dr. Nazarian is excited for what’s to come and his goals are clear and set. Here, Dr. Nazarian tells us more about his main objectives and what it means for the future of pediatric neuro-oncology care at Children’s National.

What excites you most about being back at Children’s National?

I have received most of my training at Children’s National, so this is home for me. Being one of the nation’s top children’s hospitals gives a unique advantage and ability to advocate for childhood diseases and cancers. It is always exciting to play a part in the vision of Children’s National.

What are some of the lessons learned during your time working in Zurich? And how do you think these will compliment your work at Children’s National?

We developed a focused group with basic research activities intertwined with clinical needs. The result was the launch of two clinical trials. I also helped in developing the Diffuse Midline Glioma-Adaptive Combinatory Trial (DMG-ACT) working group that spans across the world with over 18-member institutions that will help to design the next generation clinical trials. I will continue leading the research component of these efforts, which will have a positive impact on our research activities at Children’s National.

How does your work focusing on low-grade gliomas formulating into high-grade gliomas expand and place Children’s National as a leader in the field?

Scientifically speaking, transformed LLGs are very intriguing. I became interested in the field because these tumors share molecular signatures similar to high-grade gliomas (HGGs). Our team has done a great job at Children’s National to develop tools – including biorepositories, avatar models, drug screening platforms, focused working groups, etc. – for HGGs. We will apply the same model to transformed LGGs with the goal of developing biology-derived clinical therapeutics for this patient population.

How will this work support families and patients seeking specific neuro-oncology care?

We will develop new and high thruput tools so that we can better study cancer formation or transformation. These tools and platforms will allow us to screen candidate drugs that will be clinically effective. The main focus is to accelerate discovery, push drugs to the clinic, feed information back to the lab from clinical and subsequently design better therapies.

You are one of the first scientists to move to the Children’s National Research & Innovation Campus. What are some of the valuable changes or advancements you hope to see as a result of the move?

The campus will provide high-end facilities, including cutting-edge preclinical space, and allow for team expansion. The close proximity to Virginia Tech will also provide an environment for cross-discipline interactions.

Anything else you think peers in your field should know about you, the field or our program?

The team at Children’s National includes Drs. Roger Packer and Miriam Bornhorst. Both have provided constant clinical support, innovation and clinical translation of our findings. I look forward to working with them.

illustration of Research & Innovation Campus

NIH awards $6.7M to build additional lab space at Children’s National Research & Innovation Campus

September 22, 2021

Children’s National Hospital today announced a $6.7 million award from the National Institute of Health (NIH) for the new Children’s National Research & Innovation Campus (RIC). The funds will help transform a historic building on the former site of Walter Reed Army Medical Center into new research labs. The NIH construction grant marks the first secured grant funding for Phase II of the campus project, signaling continued momentum for the first-of-its-kind pediatric research and innovation hub.

The funding was announced as D.C. Mayor Muriel Bowser, D.C. Deputy Mayor for Planning and Economic Development John Falcicchio and D.C. Council Chair Phil Mendelson took their first tour of the already-renovated Phase I of the RIC. The campus began opening in early 2021 and brings together Children’s National with top-tier research and innovation partners: Johnson & Johnson Innovation – JLABS @ Washington, DC and Virginia Tech. They come together with a focus on driving discoveries and innovation that will save and improve the lives of children.

“This NIH award is the latest confirmation that we are creating something very special at the Children’s National Research & Innovation Campus,” said Kurt Newman, M.D., president and CEO of Children’s National. “Only the D.C. region can offer this proximity to federal science agencies and policy makers. When you pair our location with these incredible campus partners, I know the RIC will be a truly transformational space where we develop new and better ways to care for kids everywhere.”

The campus is an enormous addition to the BioHealth Capital Region, the fourth largest research and biotech cluster in the U.S., with the goal of becoming a top-three hub by 2023. The RIC exemplifies the city’s commitment to building the partnerships necessary to drive discoveries, create jobs, promote economic growth, treat underserved populations, improve health outcomes, and keep D.C. at the forefront of innovation and change.

“We are proud to officially welcome the Children’s National Research & Innovation Campus to the District and to the Ward 4 community,” said Mayor Bowser, after touring the campus. “This partnership pairs a world-class hospital with a top university and a premier business incubator – right here in the capital of inclusive innovation. Not only will our community benefit from the jobs and opportunities on this campus, but the ideas and innovation that are born here will benefit children and families right here in D.C. and all around the world.”

The NIH grant funding announced today will go toward the expansion and relocation of the DC Intellectual and Developmental Disabilities Research Center (DC-IDDRC). This research center will increase the efforts to improve the understanding and treatment of children with developmental disabilities, including autism, cerebral palsy, epilepsy, inherited metabolic disorders and intellectual disability.

The space where the new lab will be built used to be the Armed Forces Institute of Pathology Building, a portion of the Walter Reed Army Medical Center. The site closed and Children’s National secured 12 acres in 2016, breaking ground on Phase I construction in 2018.

The new space will offer highly cost-effective services and unique state-of-the-art research cores that are not available at other institutions, boosting the interdisciplinary and inter-institutional collaboration between Children’s National, George Washington University, Georgetown University and Howard University. Investigators from the four institutions will access the center, which includes hoteling laboratory space for investigators whose laboratories are not on-site but are utilizing the core facilities — Cell and Tissue Microscopy, Genomics and Bioinformatics, and Inducible Pluripotent Stem Cells.

“While we have explored outsourcing some of these cores, especially genomics, we found that expertise, management, training and technical support needed for pediatric research requires on-site cores,” said Vittorio Gallo, Ph.D., interim chief academic officer, interim director of the Children’s National Research Institute, and principal investigator for the DC-IDDRC. “The facility is designed to support pediatric studies that are intimately connected with our community. We operate in a highly diverse environment, addressing issues of health equity through research.”

The RIC provides graduate students, postdocs and trainees with unique training opportunities, expanding the workforce and talent of new investigators in the D.C. area. Young investigators will have job opportunities as research assistants and facility managers as well. The new labs will support these researchers so they can tackle pressing questions in pediatric research by integrating pre-clinical and clinical models.

Phase II will place genetic and neuroscience research initiatives of the DC-IDDRC at the forefront to treat a variety of pediatric developmental disorders. Other Children’s National research centers will also benefit from this additional space. The clinical and research campuses will be physically and electronically integrated with new informatics and video-communication systems.

The total projected cost of Phase II is $180 million, with design and construction to take up to three years to complete once started.

Phase II will place genetic and neuroscience research initiatives of the DC-IDDRC at the forefront to treat a variety of pediatric developmental disorders. Other Children’s National research centers will also benefit from this additional space. The clinical and research campuses will be physically and electronically integrated with new informatics and video-communication systems.

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

Pediatric Device Innovators Forum explores state of focused ultrasound

August 18, 2021

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.

Children’s National and Johnson & Johnson launch JLABS @ Washington, DC

June 6, 2019

Children’s National President and CEO Kurt Newman, M.D.

On April 9, 2019, Children’s National Health System and Johnson & Johnson Innovation LLC announced a collaboration to launch JLABS @ Washington, DC, a 32,000-square foot facility that will be located at the new Children’s National Research & Innovation Campus. The new site will serve as an incubator for pharmaceutical, medical device, consumer and health technology companies. The JLABS @ Washington, DC will be the first and only JLABS embedded in an academic environment with a strong pediatric focus. This new endeavor creates additional opportunities for Children’s National and Johnson & Johnson, together with partners, to shape the landscape of policy and funding to improve research and innovation in pediatric health care.

“The vision we pursued for this campus required a global innovation partner with a strong commitment to pediatric health and a clear understanding of the next big areas of opportunity for improving human health. We believe the JLABS model is exactly what is needed to help us drive discoveries that are then rapidly translated into new treatments and technologies,” said Kurt Newman, M.D., president and chief executive officer of Children’s National.

In addition to fast-tracking scientific innovation, JLABS will serve as a significant economic engine by creating new high-paying jobs in Washington, ultimately attracting venture investment in the region. An economic impact report suggests that the completion of the project will produce up to 110 permanent jobs and $150 million in revenue for the city by 2020. By 2030 the project will produce $6.2 billion in cumulative economic activity, 2,100 permanent jobs and $290 million in cumulative tax revenue for the district.

JLABS provides a continuum of innovators from first-time entrepreneurs to serial scientific founders representing diverse experiences across academic, startup, corporate, government regulators, funders and venture worlds.

“The best part of our collaboration with JLABS is facilitating speed to market for breakthrough therapies and technologies that are conceived in our region, including here in our own institution,” said Kolaleh Eskandanian, Ph.D., M.B.A., P.M.P., vice president and chief innovation officer at Children’s National. “I am excited that our partnership will unlock the untapped talent and great science in our region and bring better innovation to market faster with a strong focus on pediatric health.”

The facility will house state-of-the-art research labs and space for pediatric device development. This will expand Children’s National’s molecular genetic testing and biochemical analysis capabilities and enhance device development and computing infrastructure through collaborations with industry, universities, federal agencies and academic medical centers. “The selection of resident companies for the JLABS @ Washington, DC space will be done on a very competitive basis,” says Dr. Eskandanian. “Startup companies that qualify and are selected by JLABS will be offered laboratory and office space as well as mentorship.”

Read more about the partnership in the Washington Business Journal and watch an interview on WJLA with Dr. Newman.