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Research & Innovation Campus

A unified vision for children’s health

Research & Innovation Campus

The nation’s first research and innovation campus focused on children’s health is in the midst of a phased opening. Deemed a one-of-a-kind endeavor to transform pediatric research and health care, the Children’s National Research & Innovation Campus in Washington, D.C., has been years in the making.

How Children’s National Hospital, Virginia Tech, Johnson & Johnson Innovation – JLABS are creating a pediatric and healthcare research hub in Washington, D.C.

The nation’s first research and innovation campus focused on children’s health is in the midst of a phased opening. Deemed a one-of-a-kind endeavor to transform pediatric research and health care, the Children’s National Research & Innovation Campus in Washington, D.C., has been years in the making.

Leaders from Children’s National Hospital, Virginia Tech and Johnson & Johnson Innovation – JLABS detailed how unprecedented partnerships and a shared vision for the future were key to turning a decade-old dream into a reality during a panel discussion on February 24, 2021, at the Healthcare Project Delivery Conference. The virtual meeting brought together more than 150 senior hospital administrators from more than 60 hospitals and health systems, as well as healthcare facility management professionals, healthcare construction leaders, designers and architects.

“The idea for the campus started with an impressive, unique vision to create a pediatric research and innovation ecosystem where we could work alongside best-in-class research partners like Virginia Tech and Johnson & Johnson Innovation to advance discovery, while also allowing Children’s National to expand clinically on the main campus,” said Children’s National president and chief executive officer, Kurt Newman, M.D.. “It is our collective hope that the campus will accelerate the translation of breakthroughs into new treatments and technologies to benefit kids everywhere.”

The research partnership with Children’s National strategically triangulates the Virginia Tech’s billion-dollar investments in Southwest Virginia, the emerging Virginia Tech Innovation Campus in Alexandria and now the Children’s National Research & Innovation Campus in Washington, D.C.

“Fusing together strengths in cancer research, neuroscience, and computer engineering gives Virginia Tech a great opportunity to grow its physical presence in the D.C. area with a holistic purpose,” said Michael Friedlander, Ph.D., Virginia Tech’s vice president for health sciences and technology, and the Fralin Biomedical Research Institute at VTC’s executive director.

Friedlander has worked with Children’s National’s leadership for more than 25 years, and played a pivotal role in establishing the university’s footing on the Washington, D.C., campus.

Together Virginia Tech and Children’s National have launched an annual collaborative brain cancer pilot research program between the two institutions, as well as joint recruitment efforts for the first wave of Fralin Biomedical Research Institute faculty members to work in the campus’s state-of-the-art laboratories.

The 12-acre Children’s National Research & Innovation Campus, part of a 70-acre development that was formerly the Walter Reed Army Medical Center, will also become a hub for commercial innovation. In 2019, Johnson & Johnson Innovation and Children’s National collaboratively announced plans to launch JLABS @ Washington, DC, which aims to strengthen and expand the region’s network to attract the full breadth of science and technology innovators who are focused on developing transformative solutions to improve patients’ and consumers’ lives. The 32,000-square-foot life science incubator will house up to 50 start-up companies from across the pharmaceutical, medical device, consumer, and health technology sectors.

Researchers at Children’s National and Virginia Tech alike will benefit from the opportunity to collaborate with entrepreneurs working at the incubator to commercialize discoveries made in the lab.

“What an incredible opportunity for our researchers who are committed to bringing discoveries out of the lab to benefit the public,” Friedlander said. “These partnerships have established a special opportunity that aligns very well with the university’s strategic plan to grow in health sciences innovation and commercialization.”

Newman and Sally Allain, head of JLABS @ Washington, DC, both commented on how important it was to have an academic partner of Virginia Tech’s stature as one of the new enterprise’s anchoring tenants.

Recruitment for the first wave of Virginia Tech researchers to work on the new campus has just begun. Construction is anticipated to be completed by summer, 2021.

The Children's National Research & Innovation Campus

Children’s National Research & Innovation Campus welcomes Rare Disease Institute as first occupant

The Children's National Research & Innovation Campus

The Rare Disease 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.

The Children’s National Research & Innovation Campus (RIC), the first-of-its-kind pediatric research and innovation hub located in Washington, D.C., now has its first occupant – the Rare Disease Institute (RDI).

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.

With the advent of advanced DNA sequencing, databanks, informatics, new technology, pediatric consortiums and global partnerships, clinical researchers have never been in a better position to diagnose and treat rare diseases. As this field of medicine continues to rapidly evolve, the benefits provided to patients, families, clinicians and researchers through its new home at the RIC will further accelerate the trajectory of rare disease from an academic specialty into a mainstream medical field.

Marshall Summar, M.D., director of the RDI and chief of the Division of Genetics and Metabolism at Children’s National, is well-known for pioneering work in caring for children diagnosed with rare diseases. He developed and launched the world’s first RDI at Children’s National in 2017, and it became the first Clinical Center of Excellence designated by the National Organization for Rare Diseases (NORD). Dr. Summar discusses how this move will positively impact treatment, services and discovery on a national level.

Q: What are the patient benefits of the move to the RIC?

A: Patients with genetic conditions spend a lot of time visiting the hospital. By creating an easy access environment that is designed around their needs, we can provide world-class care to the families we work with. We designed extensive telemedicine capacity into the clinic so we can continue to expand our digital reach to wider areas. The parking facility is also a huge plus for our families with mobility impairments. The garage is only steps away from the clinic entrance. The architectural team worked closely with the clinical team to create a patient-centric facility for a safe and positive experience.

Q: What are the research benefits of being on the RIC?

A: One of our core goals at the RIC was to create research “neighborhoods.” A focus of the first phase of the RIC occupancy is genetics and the RDI is the clinical manifestation of that focus. Having the clinical service that sees patients with genetic disease, sharing space and campus with the Center for Genetic Medicine Research team and the molecular genetics laboratory creates that thematic neighborhood. Some of the best basic science ideas and projects come from the clinical world. Close interaction between the clinicians and the scientists will enhance those “spark” encounters. In addition, the physicians in the RDI who do bench research are also part of the genetic medicine program which furthers these interactions.

Marshall Summar

Marshall Summar, M.D., director of the RDI and chief of the Division of Genetics and Metabolism at Children’s National.

Q: What would you say has been the most significant change to your field in the past decade?

A: The ability to access next-generation genetic sequencing for more and more of our patients. The percentage of patients who can get a meaningful diagnosis with these technologies increases every year. With these techniques, we are finding new links between genes and disease at the rate of 5-10 per week.

Q: What excites you most about the future of medical genetics and rare diseases?

A: Two things are really exciting to me. The first is the ability to diagnose more patients than at any time in history. The second is the rate at which new genetic/rare disease therapies are being developed (around 50% of the FDA new drug approvals per year).

As the largest clinical program in North America and with our new location on this dedicated research and innovation campus in Washington, D.C., Children’s National and the RDI are uniquely poised to dramatically change the field of rare disease medicine. Our clinical models have started spreading to other centers across the country and will help shape the field for years to come. We are evolving rare disease into a true mainstream medical field, and the ability to make this type of change to a field is very unique to Children’s National.

Learn more about the Children’s National Research & Innovation Campus.

Research & Innovation Campus

Children’s National pain expert and innovator shares global summit spotlight

Research & Innovation Campus

As a Johnson & Johnson Innovation Quickfire Children’s Challenge awardee, Dr. Finkel and AlgometRx will be among the first group of startups taking up residence at the new JLABS @ Washington, DC, located on the Children’s National Research & Innovation Campus, when it opens in 2021 at the historic former Walter Reed Army Medical Center site.

Medical technology innovator Julia Finkel, M.D., principal investigator for the Pain Medicine Initiative of the Sheikh Zayed Institute for Pediatric Surgical Innovation at Children’s National Hospital, recently participated in Galen Growth’s 2020 Global Healthtech Summit on a virtual panel featuring resident companies from Johnson & Johnson Innovation – JLABS who are utilizing artificial intelligence (AI) with the aim to create advanced solutions for diagnostics, treatment and clinical trials. The summit, hosted in Singapore, brought the innovators together to discuss their views on their progress, the challenges and opportunities for bringing medtech innovations to market in the current climate, as well as the tools needed to succeed.

Dr. Finkel’s innovation, AlgometRx, is a real-time pain measurement technology that captures a digital image of a patient’s pupillary response to a non-invasive stimulus and applies proprietary algorithms to measure pain type and intensity. AlgometRx, a spin-off of Children’s National, recently received a JLABS @Washington DC Quickfire Children’s Challenge award.

Joining Dr. Finkel on the panel were JLABS resident company leaders Don Crawford, CEO, Analytics 4 Life; Jim Havelka, CEO, Inform AI; and Kim Walpole, CEO, Trials.ai, which leverages AI to help research teams design more effective clinical trials. The 50-minute program, moderated by Kara Bortone, senior director, Portfolio and Sourcing Management, Johnson & Johnson Innovation – JLABS, focused on topics such as how these startups approached the market and regulatory processes as well as the up-and-coming trends in health technology.

A pediatric anesthesiologist, Dr. Finkel explained the significance of achieving real-time, objective pain measurement. “Pain is one word that represents a myriad of conditions,” she says. “Pain from acute post-operative conditions is very different from peripheral neuropathic pain and different from the type of inflammatory pain seen in lupus and rheumatoid arthritis. Being able to discern the drivers of pain, the etiology, is essential to treating it well and to developing better therapeutics in the future.”

Dr. Finkel points out that AlgometRx measures nociception, which is pain fiber activation, and that is also what medications are addressing. “We’re not discounting a patient’s perception of pain, as we recognize that one’s experience of pain is very complex,” she says. “What we aim to measure is the activity being transmitted by the pain nerve and the type of nerve fiber that is doing the transmitting.”

Aiming to identify pain phenotypes is an important part of current AlgometRx development work, says Dr. Finkel, as it could significantly aid clinical decision-making in treating and monitoring patients’ pain. The company’s current regulatory focus is to seek FDA clearance related to its potential use for patients with peripheral neuropathy, which is pain and numbness resulting from damage to the nerves outside of the brain and spinal cord. The company has also identified fibromyalgia cases as a place where the technology could potentially benefit a large number of patients as it considers regulatory clearance targets.

As the COVID-19 pandemic presented many unique challenges to healthcare startups this year, panel participants were asked to discuss the hurdles they faced and how it impacted device development.

Dr. Finkel notes that the pandemic slowed patient enrollment in AlgometRx clinical studies, but also presented some upside. “At first, that had a negative impact, but it wound up being a good thing,” she says. “It gave us a moment to pause, regroup and examine the data we’d already generated. That break gave us improved information and a new, more powerful approach. It changed our trajectory by altering our regulatory path in terms of the order of things in our pipeline, so we’ve been enormously productive.”

As a Johnson & Johnson Innovation Quickfire Children’s Challenge awardee, Dr. Finkel and AlgometRx will be among the first group of startups taking up residence at the new JLABS @ Washington, DC, located on the Children’s National Research & Innovation Campus, when it opens in 2021 at the historic former Walter Reed Army Medical Center site. Along with a one-year residency at the new JLABS @ Washington DC facility,* AlgometRx will receive mentorship from experts at the Johnson & Johnson Family of Companies and grant funding to help support its continued advancement to commercialization.

*Residency at JLABS @ Washington subject to acceptance and execution of a License Agreement with Children’s National.

Annual Pediatric Device Innovation Symposium panelists

Accelerating pediatric device innovation through legislative processes and industry changes


Annual Pediatric Device Innovation Symposium panelists
While the way we deliver healthcare is changing rapidly, far too often the tools we use to treat children are stuck in the past.

Over the last decade, pediatric medical device innovation, particularly for the youngest, most fragile children, has made dismal progress. Of the Class 3 (high risk/high benefit) medical devices approved by FDA for pediatrics in the last 10 years, less than 4% are for ages 0-2 years old; and even less for neo-natal patients. Simply put, as medical devices advance, children are not seeing the benefit of innovation.

The 8th Annual Pediatric Device Innovation Symposium presented by Children’s National Hospital in conjunction with the National Capital Consortium for Pediatric Medical Devices (NCC-PDI) featured a keynote panel, “Pediatric Device Innovation: What’s Next?”, to examine the legislative and industry changes needed to speed up device innovation for kids.

One of the keynote panelists, and leading voices on this issue, is Children’s National Hospital president and CEO Kurt Newman, M.D. Dr. Newman, a former pediatric surgeon, knows firsthand that every day in our nation’s pediatric hospitals, surgeons are manipulating adult medical devices to create creative solutions for children’s bodies because it’s the only available option.

“Children need and deserve devices that are conceived and designed with their biology and future in mind,” says Dr. Newman. “While children may only make up a small percentage of our population – maybe 20 or 25% – they are 100% of our future.”

Dr. Kurt Newman in front of the capitol building

“Children need and deserve devices that are conceived and designed with their biology and future in mind,” says Children’s National Hospital president and CEO Kurt Newman, M.D. “While children may only make up a small percentage of our population – maybe 20 or 25% – they are 100% of our future.”

Dr. Newman also addressed the current barriers to pediatric device innovation, which ranges from limited pediatric clinical trials to a market size that’s not financially appealing.

“The truth is, the frontiers of pediatric medicine are really in the innovative treatments, devices, therapies, and cures awaiting us on the other side of research and development.,” says Dr. Newman.

Former CNN correspondent, Jeanne Meserve, moderated the 45-minute keynote panel discussion, asking questions about the challenges to pediatric innovation, what policy changes need to take place to see improvement in the field of pediatric device innovation, and how federal funding can assist in creating change.

Michelle McMurry-Heath, new CEO of DC-based Biotechnology Innovation Organization (BIO), who joined Dr. Newman on this keynote panel, agreed that more needs to be done in the pediatric space. Dr. McMurry-Heath believes the Food and Drug Administration (FDA) is a public health advocate at heart and that the Pediatric Device Consortia (PDC), which Children’s National Hospital is part of, is starting to make new advancements in pediatric innovation by giving FDA clearance to more start-up companies than we’ve seen in the past.

“The FDA is interested in is what improves the health outcomes for the people and innovation is a huge piece. This is an important part of their mission and it is starting to yield benefits,” says Dr. Michelle McMurry-Heath. “Innovation is a team sport – it’s not easy. It takes a village of expertise and collaboration to progress and projects like the Pediatric Device Consortia is an important piece in this puzzle.”

NCC-PDI is one of five consortia in the FDA’s Pediatric Device Consortia (PDC) Grant Program created to support the development and commercialization of medical devices for children and 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.

To date, NCC-PDI has mentored over 100 medical device sponsors to help advance their pediatric innovations, with seven devices having received either their FDA market clearance or CE marking.

Dr. McMurry-Heath also addressed the challenge of diseases that don’t exist in adults and posed the question, “How do you create a device for kids if it doesn’t exist in adults?” She cited the lack of market in pediatrics and the difficulty in bringing a device to market as problems that hinder innovation, which is why advocating for these devices is crucial to children’s healthcare everywhere.

“So much of our innovation comes from our small, innovative companies,” say Dr. McMurry-Heath. “For example, my company is working on a COVID-19 tracker now and 70% of the innovation is coming from our smallest biotech companies. It’s a race against time for these companies to bring their innovation to market in order to keep the lights on and pay their scientists; this dog-eat-dog world isn’t immediately obvious to outsiders.

Beyond advocating, Dr. Newman and Children’s National are developing the first-of-its-kind pediatric research and innovation campus, which is currently under construction at DC’s former Walter Reed Army Medical Center site.

“We secured 12 acres to create something that has never been done before and that’s a campus for innovation dedicated to children,” says Dr. Newman. “Our close proximity to federal research institutions and agencies enables the new Children’s National campus to leverage the rich ecosystem of public and private sectors to help bolster biohealth, medical device, and life science innovation.”

As Children’s National continues to champion ways to accelerate pediatric device development, one focus is the on-site incubator Johnson & Johnson Innovation – JLABS, which will help start-up companies strengthen their ideas by working with coaches, having access to mentors and learning how to interact with the FDA. This partnership also offers an audience for their device which could potentially lead to investments.

The Children’s National Research & Innovation Campus will create an ecosystem that can accelerate breakthroughs in pediatric healthcare discoveries and technologies: The new campus is currently under construction and expected to open in the first quarter of 2021.

Both panelists agreed they’d like to see more flexibility with regulators to work with innovators in order create more incentives for them to present their device, like the NCC-PDI “Make Your Medical Device Pitch for Kids!” Competition, which was recently held in September 2020. The six winners received up to $50,000 in FDA-funded grant awards in order to develop their device, eventually bring it to market in order to improve healthcare for kids.

NCC-PDI-COVID19-Edition-Competition

NCC-PDI launches special pediatric medical device competition focused on covid-19 innovations

Kolaleh-Eskandanian

“Innovation in children’s medical devices consistently lags behind that of adults and we need to change that if we are to confront the challenge to children’s health of COVID-19 and future pandemics,” said Kolaleh Eskandanian, Ph.D., MBA, PMP, vice president and chief innovation officer at Children’s National Hospital and principal investigator of NCC-PDI. 

As medical data increasingly highlights the serious impact of COVID-19 on children’s health, the National Capital Consortium for Pediatric Device Innovation (NCC-PDI) announces a special pitch competition focused on COVID-19-related pediatric medical devices that support home health monitoring and telehealth, and improve sustainability, resiliency and readiness in diagnosing and treating children during a pandemic.

The “Make Your Medical Device Pitch for Kids!” COVID19 edition is led by NCC-PDI co-founders 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 and powered by nonprofit accelerator and NCC-PDI member, MedTech Innovator. The finals in the virtual pitch event will be held on July 20, 2020. Winners will each receive a grant award of up to $50,000.

“Despite early reports that COVID-19 posed less of a threat to children, a recent study published by Children’s National shows that considerable numbers of pediatric patients are hospitalized and become critically ill from the disease,” said Kolaleh Eskandanian, Ph.D., MBA, PMP, vice president and chief innovation officer at Children’s National Hospital and principal investigator of NCC-PDI. “Innovation in children’s medical devices consistently lags behind that of adults and we need to change that if we are to confront the challenge to children’s health of COVID-19 and future pandemics.”

Funding for the competition is made possible by a grant from the Food and Drug Administration (FDA) and a philanthropic gift from Mei Xu, founder of e-commerce platform Yes She May, a site dedicated to women-owned brands.

Along with grant funding, one company from the competition will be selected by Johnson & Johnson Innovation – JLABS to receive a one-year residency at JLABS @ Washington, DC, which will be located on the new Children’s National Research & Innovation Campus currently under construction. In addition to the 2021 JLABS residency, the awardee will have access to the JLABS community and expert mentoring by the Johnson & Johnson family of companies.

Submissions for the competition are being accepted now through Monday, July 6, 2020z at the NCC-PDI website, Innovate4Kids.org, where complete details can be found.

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, which lags significantly behind the progress of adult medical devices. Along with Children’s National, University of Maryland and Medtech Innovator, NCC-PDI members include accelerator BioHealth Innovation and design firm Archimedic.

To date, NCC-PDI has mentored over 100 medical device sponsors to help advance their pediatric innovations, with seven devices having received either their FDA market clearance or CE marking. The consortium hosts a major pediatric pitch competition annually that showcases and awards promising pediatric innovations and provides a first-of-its-kind pediatric-focused accelerator program for finalists.

NCC-PDI-COVID19-Edition-Competition

Vittorio Gallo and Mark Batshaw

Children’s National Research Institute releases annual report

Vittorio Gallo and Marc Batshaw

Children’s National Research Institute directors Vittorio Gallo, Ph.D., and Mark Batshaw, M.D.

The Children’s National Research Institute recently released its 2019-2020 academic annual report, titled 150 Years Stronger Through Discovery and Care to mark the hospital’s 150th birthday. Not only does the annual report give an overview of the institute’s research and education efforts, but it also gives a peek in to how the institute has mobilized to address the coronavirus pandemic.

“Our inaugural research program in 1947 began with a budget of less than $10,000 for the study of polio — a pressing health problem for Washington’s children at the time and a pandemic that many of us remember from our own childhoods,” says Vittorio Gallo, Ph.D., chief research officer at Children’s National Hospital and scientific director at Children’s National Research Institute. “Today, our research portfolio has grown to more than $75 million, and our 314 research faculty and their staff are dedicated to finding answers to many of the health challenges in childhood.”

Highlights from the Children’s National Research Institute annual report

  • In 2018, Children’s National began construction of its new Research & Innovation Campus (CNRIC) on 12 acres of land transferred by the U.S. Army as part of the decommissioning of the former Walter Reed Army Medical Center campus. In 2020, construction on the CNRIC will be complete, and in 2012, the Children’s National Research Institute will begin to transition to the campus.
  • In late 2019, a team of scientists led by Eric Vilain, M.D., Ph.D., director of the Center for Genetic Medicine Research, traveled to the Democratic Republic of Congo to collect samples from 60 individuals that will form the basis of a new reference genome data set. The researchers hope their project will generate better reference genome data for diverse populations, starting with those of Central African descent.
  • A gift of $5.7 million received by the Center for Translational Research’s director, Lisa Guay-Woodford, M.D., will reinforce close collaboration between research and clinical care to improve the care and treatment of children with polycystic kidney disease and other inherited renal disorders.
  • The Center for Neuroscience Research’s integration into the infrastructure of Children’s National Hospital has created a unique set of opportunities for scientists and clinicians to work together on pressing problems in children’s health.
  • Children’s National and the National Institute of Allergy and Infectious Diseases are tackling pediatric research across three main areas of mutual interest: primary immune deficiencies, food allergies and post-Lyme disease syndrome. Their shared goal is to conduct clinical and translational research that improves what we know about those conditions and how we care for children who have them.
  • An immunotherapy trial has allowed a little boy to be a kid again. In the two years since he received cellular immunotherapy, Matthew has shown no signs of a returning tumor — the longest span of time he’s been tumor-free since age 3.
  • In the past 6 years, the 104 device projects that came through the National Capital Consortium for Pediatric Device Innovation accelerator program raised $148,680,256 in follow-on funding.
  • Even though he’s watched more than 500 aspiring physicians pass through the Children’s National pediatric residency program, program director Dewesh Agrawal, M.D., still gets teary at every graduation.

Understanding and treating the novel coronavirus (COVID-19)

In a short period of time, Children’s National Research Institute has mobilized its scientists to address COVID-19, focusing on understanding the virus and advancing solutions to ameliorate the impact today and for future generations. Children’s National Research Institute Director Mark Batshaw, M.D., highlighted some of these efforts in the annual report:

  • Eric Vilain, M.D., Ph.D., director of the Center for Genetic Medicine Research, is looking at whether or not the microbiome of bacteria in the human nasal tract acts as a defensive shield against COVID-19.
  • Catherine Bollard, M.D., MBChB, director of the Center for Cancer and Immunology Research, and her team are seeing if they can “train” T cells to attack the invading coronavirus.
  • Sarah Mulkey, M.D., Ph.D., an investigator in the Center for Neuroscience Research and the Fetal Medicine Institute, is studying the effects of, and possible interventions for, coronavirus on the developing brain.

You can view the entire Children’s National Research Institute academic annual report online.

Andrew Dauber

Andrew Dauber, M.D., MMSc, caps off research success with award and reception

Andrew Dauber

Unfortunately, we’ve been notified that the ENDO2020 conference has been canceled due to concerns of COVID-19. Because of this, we will not be hosting our reception in honor of Andrew Dauber, M.D., on Sunday, March 29.

We hope to see you at a future Endocrinology or Pediatric Endocrinology event.

Children’s National Hospital is incredibly proud of the work Dr. Dauber has done in the endocrinology community.

Andrew Dauber, M.D., MMSc, division chief of Endocrinology at Children’s National Hospital, will be awarded the 2020 Richard E. Weitzman Outstanding Early Career Investigator Award at ENDO 2020. The prestigious award will be presented at the annual meeting of the Endocrine Society in recognition of Dauber’s work in understanding the regulation of growth and puberty, and applying innovative genetic technologies to studying pediatric endocrinology. Dauber credits many collaborators throughout the world, as well as the team at Children’s National for the award.

With a five-year grant from the National Institutes of Health (NIH), Dauber and colleagues from the Cincinnati Children’s Hospital Medical Center, Boston Children’s Hospital and the Children’s Hospital of Philadelphia are using electronic health records to identify children who likely have rare genetic growth disorders. Using cutting-edge DNA sequencing technologies, including whole exome sequencing, the researchers are aiming to identify novel genetic causes of severe growth disorders. The first paper describing genetic findings in patients with high IGF-1 levels was published in Hormone Research in Paediatrics in December 2019.

Dauber and researchers at Cincinnati Children’s Hospital Medical Center are exploring how to treat patients with mutations in the PAPPA2 gene. In 2016, the group described the first patients with mutations in this gene who had decreased the bioavailability of IGF-1, stunting their growth and development. In their current phase of research, findings are emphasizing the importance of this gene in regulating IGF-1 bioavailability throughout childhood. The ultimate aim is to create therapies to increase IGF-1 bioavailability, thereby supporting healthy growth and development in children. Their first study to track PAPPA2 and intact IBGBP-3 concentrations throughout childhood was published in the European Journal of Endocrinology in January 2020.

Dauber is particularly interested in studying children with dominantly inherited forms of short stature. Along with collaborators in Cincinnati, he currently has an ongoing treatment trial using growth hormone in patients with Aggrecan gene mutations.  Dauber hopes to announce soon a new clinical trial for children with all forms of dominantly inherited short stature.

Study upon study has shown us that there are many factors that affect an individual’s height and growth. As these studies and the conversation around how to identify and address genomic anomalies become more prevalent, the team at Children’s National is increasingly interested in engaging with other centers around the country. In the coming months, the Children’s National Research & Innovation Campus will open on the grounds of the former Walter Reed Army Medical Center, which will serve as a one-of-a-kind pediatric research and innovation hub. A critical component to this campus is the co-location of Children’s National research with key partners and incubator space.

Dr. Kurt Newman in front of the capitol building

Making healthcare innovation for children a priority

Dr. Kurt Newman in front of the capitol building

Recently, Kurt Newman, M.D., president and CEO of Children’s National Hospital, authored an opinion piece for the popular political website, The Hill. In the article, he called upon stakeholders from across the landscape to address the significant innovation gap in children’s healthcare versus adults.

As Chair of the Board of Trustees of the Children’s Hospital Association,  Dr. Newman knows the importance of raising awareness among policy makers at the federal and state level about the healthcare needs of children. Dr. Newman believes that children’s health should be a national priority that is addressed comprehensively. With years of experience as a pediatric surgeon, he is concerned by the major inequities in the advancements of children’s medical devices and technologies versus those for adults. That’s why Children’s National is working to create collaborations, influence policies and facilitate changes that will accelerate the pace of pediatric healthcare innovation for the benefit of children everywhere. One way that the hospital is tackling this challenge is by developing the Children’s National Research & Innovation Campus, which will be the nation’s first innovation campus focused on pediatric research.