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Dr. Eric Vilain and researcher in a lab

Children’s National Hospital joins the Mendelian Genomics Research Consortium, receiving $12.8 million

Dr. Eric Vilain and researcher in a lab

Dr. Eric Vilain accompanied by a fellow researcher at the new Research & Innovation Campus.

Children’s National Hospital announces a $12.8 million award from the National Institutes of Health’s National Human Genome Research Institute (NHGRI) to establish the only Pediatric Mendelian Genomics Research Center (PMGRC) as part of a new Mendelian Genomics Research Consortium. Researchers at Children’s National and Invitae — a leading medical genetics company — will identify novel causes of rare inherited diseases, investigate the mechanisms of undiagnosed conditions, enhance data sharing, and generally interrogate Mendelian phenotypes, which are conditions that run in families.

“Our overall approach provides an efficient and direct path for pediatric patients affected with undiagnosed inherited conditions through a combination of innovative approaches, allowing individuals, families and health care providers to improve the management of the disease,” says Eric Vilain, M.D., Ph.D., director of the Center for Genetic Medicine Research at Children’s National.

To accelerate gene discovery for Mendelian phenotypes and the clinical implementation of diagnosis, the consortium will leverage the broad pediatric clinical and research expertise of the Children’s National Research Institute and laboratories in partnership with Invitae. The Molecular Diagnostics Laboratory at Children’s National will provide genetic testing for patients in the Washington, D.C., metropolitan area. Invitae will provide genetic testing for patients from elsewhere in the U.S., giving the project a national reach and allowing researchers to leverage more robust data. Integrative analyses will be performed jointly with scientists at Children’s National and Invitae.

“Some patients have genetic test results that are ‘negative,’ meaning the results do not explain their condition. When a patient receives a negative result, it is challenging for parents and doctors to know what to do next,” says Meghan Delaney, D.O., M.P.H., chief of the Division of Pathology and Laboratory Medicine and Molecular Diagnostics Laboratory at Children’s National. “The project will provide an avenue to possibly find an explanation of their child’s condition. Besides filling an important clinical gap, the results will add new knowledge for future patients and the scientific community.”

“Too often parents of children suffering from a rare condition find themselves in a protracted diagnostic odyssey when early intervention could mean better overall outcomes,” says Robert Nussbaum, M.D., chief medical officer of Invitae. “We are proud to partner with Children’s National Research Institute on this important effort to identify the genetic cause of these rare conditions earlier and improve the chances that children with such conditions can receive the appropriate treatments and live healthier lives.”

Deciphering Mendelian conditions will help diagnose more of the estimated 7,000 rare inherited diseases and predict the tremendous variability of clinical presentations in both rare and common conditions caused by the same gene.

There is also a need to establish a new standard of care to bridge the gap in the use of genomic information from diagnosis to improved outcomes. The consortium will establish best practices for obtaining a genetic diagnosis, offering an explanation for the condition to affected patients, and is likely to provide additional explanations for basic biological mechanisms, increasing the knowledge of physiopathology and possibly leading to better condition management.

The PMGRC will enroll an average of 2,600 participants per year with suspected Mendelian phenotypes and previously non-diagnostic tests and their family members. The integration of multiple genomic technologies, including short and long read genome sequencing, optical genome mapping and RNA-sequencing, will enable these discoveries. To disambiguate uncertain variants and candidate genes, the PMGRC will use whole transcriptome analysis, RNA-sequencing, CRE-sequencing and functional modeling.

Since many Mendelian conditions first appear prenatally or during infancy, Children’s National will have a unique bed-to-bench-to-bed symbiosis. Patients eligible for the study will come from across the multiple specialty divisions of Children’s National, including the Children’s National Rare Disease Institute, and nationally through the partnership with Invitae. From there, experts from the Children’s National Center for Genetic Medicine Research will enroll patients and integrate the initial clinical test results with broad-based genomic interrogation, leading to new diagnoses and novel discoveries. Finally, the results will be verified and returned to clinicians, which will help inform targeted therapies.

Typically, the patients eligible for this study jump from specialist to specialist without an answer, have a condition that appears in other family members or they have symptoms involving more than one affected organ, which suggests a complex developmental condition. The PMGRC at Children’s National will help find answers to the causes of many puzzling pediatric conditions, providing faster clinical diagnoses and opening up pathways to potentially better treatments.

Dr. Vilain’s work will be based at the Children’s National Research & Innovation Campus on the grounds of the former Walter Reed Army Medical Center in Washington, D.C. The campus is also home to the Children’s National Rare Disease institute — one of the largest clinical genetics program in the United State that provides care to more than 8,500 rare disease patients.

facial recognition of noonan syndrome

Commercialization of novel facial analysis technology can improve diagnosis of rare disorders in pediatric patients

facial recognition of noonan syndrome

Children’s National Hospital has entered into a licensing agreement with MGeneRx Inc. for its patented pediatric medical device technology using objective digital biometric analysis software for the early and non-invasive screening of dysmorphic genetic diseases such as Noonan syndrome.

Children’s National Hospital has entered into a licensing agreement with life sciences technology company MGeneRx Inc. for its patented pediatric medical device technology using objective digital biometric analysis software for the early and non-invasive screening of dysmorphic genetic diseases. The technology, developed by a multidisciplinary Children’s National team led by Marius George Linguraru, D.Phil, M.A., M.Sc., of the Sheikh Zayed Institute for Pediatric Surgical Innovation and Marshall Summar, M.D., director of the Children’s National Rare Disease Institute (CNRDI), can provide a more advanced diagnostic tool for regions of the world with limited access to geneticists or genetic testing.

The application utilizes artificial intelligence (AI) and machine learning to analyze biometric data and identify facial markers that are indicative of genetic disorders. Physicians can capture biometric data points of a child’s face in real time within the platform, where it scans facial biometric features to determine the potential presence of a genetic disease, which can often be life-threatening without early intervention. Research studies conducted in conjunction with the National Human Genome Research Institute at the National Institutes of Health further enhanced the development of the application in recent years, showing the potential to detect, with a 90 percent accuracy, early diagnosis of 128 genetic diseases across pediatric subjects in 28 countries. These diseases include DiGeorge syndrome (22q11.2 deletion syndrome), Down syndrome, Noonan syndrome and Williams-Beuren syndrome.

“We are delighted to enter into this licensing agreement through Innovation Ventures, the commercialization arm of Children’s National Hospital, which seeks to move inventions and discoveries from Children’s National to the marketplace to benefit the health and well-being of children. Our mission is to add the ‘D’ in development to the ‘R’ in research to accelerate the commercialization of our intellectual property,” says Kolaleh Eskandanian, Ph.D., M.B.A., P.M.P., vice president and chief innovation officer at Children’s National and managing director of Innovation Ventures. “It is through partnerships with startups and the industry that we can achieve this goal and thus we highly value this new partnership with MGeneRx Inc. The acceleration and commercialization of this objective digital biometric analysis technology will not only help diagnose rare genetic disorders – it will also allow for earlier interventions that improve the quality of life for the children living with these conditions.”

Eskandanian adds that the social impact of this technology is especially profound in lower income nations around the world, where there is a high prevalence of rare genetic conditions but a severe lack in the specialty care required to diagnose and treat them. Additional data collected through the expanded use of the technology will help to further develop the application and expand its capabilities to identify and diagnose additional rare genetic conditions.

The licensing agreement was arranged by the Children’s National Office of Innovation Ventures, which is focused on the commercialization of impactful new pediatric medical device technologies and therapies to advance children’s health care. Created to catalyze the ongoing translational research of the Children’s National Research Institute (CNRI) as well as inventions by hospital’s clinicians, Innovation Ventures focuses on four core pillars to advance pediatric medical technologies including a Biodesign program, partnerships and alliances to augment internal capacity, seed funding to de-risk technologies and validate market and clinical relevance, and back-office operations to manage intellectual property and licensing activities. Since 2017, Children’s National intellectual property has served as the basis for over 15 licensing or option agreements with commercial partners.

Providing access to an array of experts and resources for pediatric innovators is one of the aims of the Children’s National Research & Innovation Campus, a first-of-its-kind focused on pediatric health care innovation, with the first phase currently open on the former Walter Reed Army Medical Center campus in Washington, D.C. With its proximity to federal research institutions and agencies, universities, academic research centers, as well as on-site incubator Johnson and Johnson Innovation – JLABS, the campus provides a rich ecosystem of public and private partners, which will help bolster pediatric innovation and commercialization.

pediatric cardiac and vascular MRI coil

Overcoming real and perceived barriers to pediatric device innovation

pediatric cardiac and vascular MRI coil

A working group of innovators, engineers and clinicians from Children’s National Hospital and other institutions came together to address the real and perceived barriers to the creation of pediatric devices, such as this pediatric cardiac and vascular MRI coil developed by Inkspace Imaging.

A working group of innovators, engineers and clinicians from Children’s National Hospital, Children’s Hospital of Orange County and other institutions came together to address the real and perceived barriers to pediatric device innovation through opportunities that may change the return-on-investment in this market and improve health outcomes.

The new report, published in the Journal of Translational Engineering in Health and Medicine, mentions barriers that impede the advancement of pediatric devices, including excessive limitations for testing and validation, lack of incentives, inadequate research models and inconsistent pediatric-related knowledge among companies, regulatory experts, ethic review panels and government reviewers.

To remove the real and perceived barriers fostering excessive limitations, the researchers suggest reducing the perceived risk by establishing guidelines that standardize the review process and ethical research models, incentivizing small companies to participate in pediatric device innovation.

To increase incentives, the researchers call for the development of pediatric versions of adult devices with or without enforcement of regulations, develop incentives for small, medium and large-size companies, extend patent protection and more.

Since science corroborates the device’s effectiveness and safety, tailored pediatric research models are needed to help advance pediatric device innovation. While randomized trials in well-defined cohorts are commonly used in the scientific quest, they do not reflect the clinical practice in the pediatric devices field.

“Overcoming this barrier will require greater opportunity for creativity in the design of clinical trials, including delayed entry, intent-to-treat analysis, personalized outcome measures, and post-hoc subgroup analysis,” said Terence et al. “Effective research for pediatric device innovation will require greater ability to rely on ‘real-world’ data from post-market use of the proposed device or similar devices.”

On the education side, stakeholders, such as device sponsors and researchers, must receive an improved education on pediatric devices, according to the authors. Pediatricians should also be added to the regulatory review panels and advisory boards, so decision-makers can receive the pediatric perspective and fold it into their considerations.

“The consensus outcome of this meeting is that there are multiple opportunities, and a flexible combination of new programs and regulatory changes can be created to benefit the multiple stakeholders in pediatric device development,” said Terence et al. “An essential component will be building a cadre of experts with the development, regulatory, and clinical expertise to support all innovators.”

Kolaleh Eskandanian, Ph.D., M.B.A., P.M.P., vice president and chief innovation officer at Children’s National Hospital adds that supporting and expanding pediatric innovation is a key focus of the new Children’s National Research & Innovation Campus, the first-of-its-kind focused on pediatric health care innovation, with the first phase currently open on the former Walter Reed Army Medical Center campus in Washington, D.C. With its proximity to federal research institutions and agencies, universities, academic research centers, the campus provides a rich ecosystem of public and private partners which will help bolster pediatric innovation and commercialization.

Authors from Children’s Hospital Orange County include Terence Sanger, M.D., Nadine Afari, M.S., Anthony Chang, M.D., William Feaster, M.D., Sharief Taraman, M.D., Debra Beauregard, Brent Dethlefs, Tiffani Ghere, R.D., C.S.P., Mustafa Kabeer, M.D., and George Tolomiczenko.

girl with smart brain imagination doodle

Children’s National provides clinical validation, IP for health challenge designed to advance pediatric innovation

girl with smart brain imagination doodle

Reinforcing its commitment to expanding innovation in pediatric care, Children’s National Hospital has joined a strategic partnership with the Center for Advancing Innovation (CAI) , along with collaborators Resonance Philanthropies and Digital Infuzion, to launch the 2021-2022 Innovate Children’s Health Challenge. This year’s event, Innovate Children’s Health II, focuses on technologies that address pandemic resiliency and prevention in the pediatric population and seeks to advance diagnostics, therapeutics and digital health tools that address pediatric mental health.

The initiative matches entrepreneurial talent with breakthrough inventions to launch startups and connect them with capital. For this challenge, more than 15 startups will compete for the opportunity to commercialize promising mental health solutions from a variety of research partners, including Children’s National. Nationally recognized for its expertise and commitment to innovation in pediatric care, Children’s National will contribute to the clinical validation of selected technologies.

“In addition to our role in providing clinical validation, this initiative provides the opportunity for intellectual property (IP) developed by leading clinicians at Children’s National Hospital, as well as other great pediatric institutions, to be considered for partnership with entrepreneurs who can help bring these technologies to market,” says Kolaleh Eskandanian, PhD, MBA, PMP, vice president and chief innovation officer at Children’s National Hospital. “Our mission is to improve children’s healthcare and Innovate Children’s Health II is a great way to harness this trifecta model — innovation, talent and capital — in order to develop breakthrough solutions that address the unique needs of pediatric patients.”

Kolaleh-Eskandanian

“In addition to our role in providing clinical validation, this initiative provides the opportunity for intellectual property (IP) developed by leading clinicians at Children’s National Hospital, as well as other great pediatric institutions, to be considered for partnership with entrepreneurs who can help bring these technologies to market,” says Kolaleh Eskandanian, PhD, MBA, PMP, vice president and chief innovation officer at Children’s National Hospital.

There are three ways to participate in Innovate Children’s Health II:

  • Entrepreneurial-minded people, alone or as members of multidisciplinary teams, may compete to commercialize vetted inventions;
  • Existing startups may enter the challenge with other public health-related inventions, including their own and/or others to which they have access;
  • Participants may submit ideas that they believe will improve emergency preparedness and pandemic response.

Inventors and technology licensing officers may submit inventions to be evaluated and made available for licensing to challenge winners. Innovate Children’s Health II will accept invention submissions until September 1, 2021. Anyone with an entrepreneurial spirit and interest in stopping current and future pandemics is invited to sign up to learn more about the challenge. Teams may also enroll in the challenge to choose a featured invention, bring in a third-party invention or get matched with an invention based on area of interest.

“The COVID-19 pandemic has made our children anxious, depressed and pessimistic about their futures. Through Innovate Children’s Health II, CAI and our strategic partner Children’s National will strive to give our children hope,” says Rosemarie Truman, founder and CEO of CAI. “We are grateful to Digital Infuzion and Resonance Philanthropies for their support, which makes this challenge possible.”

Eskandanian adds that supporting and expanding pediatric innovation is a key focus of the new Children’s National Research & Innovation Campus, the first-of-its-kind focused on pediatric health care innovation, with the first phase currently open on the former Walter Reed Army Medical Center campus in Washington, D.C. With its proximity to federal research institutions and agencies, universities, academic research centers, as well as on-site incubator Johnson and Johnson Innovation – JLABS, the campus provides a rich ecosystem of public and private partners which will help bolster pediatric innovation and commercialization.

Research & Innovation Campus building entrance

Children’s National Research & Innovation Campus welcomes new resident company, AlgometRx

Research & Innovation Campus building entrance

Located on a nearly 12-acre portion of the former Walter Reed Army Medical Center campus, the Children’s National Research & Innovation Campus is the nation’s first campus dedicated to pediatrics, which formally opens in September 2021.

On April 26, 2021, AlgometRx Inc., a Children’s National spinout company developing a handheld device to objectively measure pain by pupillary response, will relocate to Johnson & Johnson Innovation – JLABS @ Washington, DC on the Children’s National Research & Innovation Campus. The AlgometRx move comes following the company being awarded the JLABS @ Washington, DC Children’s QuickFire Challenge, which includes a one-year residency at the newly opened JLABS @ Washington, DC – a 32,000-square-foot incubator located at the new Children’s National Research & Innovation Campus in northwest Washington, D.C. As an awardee, AlgometRx also receives access to research and development space, capital equipment, mentorship, resources and programming.

Located on a nearly 12-acre portion of the former Walter Reed Army Medical Center campus, the Children’s National Research & Innovation Campus is the nation’s first campus dedicated to pediatrics, which formally opens in September 2021. This campus aims to help address a significant problem: the development of medical and surgical devices for children has long lagged behind that for adults. Over the past decade, only one in four medical devices approved by the Food and Drug Administration (FDA) were indicated for use in children, and the majority were for those ages 12 and up.

By bringing together public and private partners, the campus is a one-of-a-kind innovation ecosystem that aims to accelerate breakthrough discoveries into new treatments and technologies.

AlgometRx was founded by pediatric anesthesiologist Julia C. Finkel, M.D., and originated at the Sheikh Zayed Institute for Pediatric Surgical Innovation at Children’s National.

“Pain is the only vital sign that is not objectively measured,” Finkel said. “The current standard of measuring pain is the 0-10 scale, which is based on observations and subjective assessment. This technique increases the likelihood for inaccuracies, especially for infants and children who cannot clearly communicate their pain.”

Finkel’s research was inspired by a desire to find an objective measurement of pain in nonverbal pediatric patients so physicians can better determine the appropriate pain treatment or the effectiveness of a treatment.

“The Children’s National Research & Innovation Campus enables AlgometRx to focus almost exclusively on collecting data, which is the most crucial step at this time,” Finkel said.

AlgometRx aims to submit a formal application to the FDA in the next 12-18 months, with the next six months dedicated to validating the device through a clinical trial.

“The campus also allows us to take advantage of a vast network of the nation’s most innovative pediatric researchers who can provide mentorship on subjects like clinical trial design, prototyping and grant applications,” Finkel said. “Just outside the campus, our team has proximity to relevant federal agencies, such as the FDA, meaning that to date, we’ve only met with FDA officials in person. This advantageous environment will accelerate our progress and allow us to use this technology to more quickly benefit children in pain.”

After its September grand opening, the Children’s National Research & Innovation Campus aims to expand its role as a biomedical incubator to include about 50 start-up companies, working to translate potential breakthrough discoveries into new treatments and technologies.

Learn more at www.childrensnational.org/innovationinstitute.

Children’s National Research & Innovation Campus garage solar panel

D.C. leaders unveil city’s largest solar canopy at Children’s National Research & Innovation Campus

Children’s National Research & Innovation Campus garage solar panel

The clean energy generated by this solar array, which is on the RIC parking garage, will be distributed through the Solar for All program, Mayor Bowser’s initiative to provide 100,000 low-to-moderate income families with the benefits of locally generated clean energy.

Washington, D.C.’s largest solar canopy was recently unveiled on the grounds of the Children’s National Research & Innovation Campus (RIC), located on the former Walter Reed Army Medical Center campus. The installation is part of the District’s Solar for All program and will provide more than 325 income-qualified households with clean, renewable energy and electricity bill savings over the next 15 years.

“When we began to plan the Children’s National Research & Innovation Campus, we wanted to do more than discover new and better ways to care for children. We also wanted to support the local community,” said Children’s National Hospital President and CEO Kurt Newman, M.D. “I’m proud that we could incorporate the solar design into our campus thereby returning clean energy to the residents of D.C.’s Ward 4 and doing our part to support the environment.”

The District of Columbia Department of Energy & Environment (DOEE) awarded Children’s National and partner New Columbia Solar (NCS) with the 2021 District Sustainability Award for the execution of this project.

“I am pleased to celebrate this innovative, award-winning project, accomplished with our partners for the benefit of our residents and community,” said DOEE Director Tommy Wells. “The completion of this project by New Columbia Solar is a tremendous achievement that will not only help to meet Mayor Muriel Bowser’s climate and clean energy goals for the District, but will also help to reduce energy costs for low-income households. The District is proud to be a national leader in sustainability, and this project further demonstrates our commitment to deploying solar and developing scalable solutions in a way that prioritizes equitable access for all.”

L-R: Children’s National Hospital Vice President of Community Engagement, Advocacy & Government Affairs Tonya Kinlow; New Columbia Solar CEO Mike Healy; Councilmember Mary Cheh; DOEE Director Tommy Wells; PSC Commissioner Emile Thompson; Children’s National Hospital Chief Operating Officer Kathy Gorman; and DCSEU Director Ted Trabue.

The clean energy generated by this solar array, which is on the RIC parking garage, will be distributed through the Solar for All program, Mayor Bowser’s initiative to provide 100,000 low-to-moderate income families with the benefits of locally generated clean energy. This installation will serve more than 325 income-qualified D.C. families, saving each household up to $500 annually and saving these families up to $2.4 million over 15 years.

“This project is the perfect example of why operating a solar company in the District is so rewarding,” said NCS CEO Mike Healy. “I look at this project and see major decision-makers in D.C. coming together, in the middle of a global pandemic, to prioritize powering our city through clean energy and to offset utility expenses for the families in our community who are most in need.”

The installation began when Children’s National acquired a large five-story above-grade parking garage, which provided the perfect location for a cutting-edge solar array. The original goal for the parking garage was always to incorporate a solar array. However, the installation of the 1,148 kW system was an engineering feat, representing one of the District’s most complex solar systems.

Over the past two years operating DOEE’s Solar for All program, the D.C. Solar Energy Utility (DCSEU) has worked with local solar developers to install 130 community solar facilities across the District. These installations are expected to serve more than 4,000 income-qualified D.C. families, with more community solar projects slated to be developed in 2021 to serve an additional 2,000 households.

“It’s an honor to deliver the Solar for All program in partnership with the District government and the D.C. business community,” said DCSEU Director Ted Trabue. “These projects bring opportunities to District businesses, jobs to D.C. residents and critical electricity bill savings to families who need it, all while helping work toward a carbon-free D.C.”

As part of the larger commitment of Children’s National to positively impact the environment and the community, the organization has formed a Sustainability Council with the overall purpose to build a long-term commitment to sustainable practices; integrate sustainability in the areas of education, research, operations and community service; and incorporate sustainable designs in future construction plans.

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.