The new partnership will advance pediatric health through innovative discoveries and therapies, with an initial focus on pediatric cancers, including brain tumors.
Children’s National Hospital and Virginia Tech are expanding their research partnership, building on a successful collaboration established in 2019. This partnership will advance pediatric health through innovative discoveries and therapies, with an initial focus on pediatric cancers, including brain tumors.
The partnership brings together Children’s National, ranked among the nation’s top pediatric hospitals by U.S. News & World Report, and Virginia Tech, a leading academic research institution. Together, they aim to deliver transformative advancements to enhance outcomes for children facing devastating diagnoses.
The goals of the research-focused partnership include:
Accelerating the understanding of the biology, improvements in prevention and treatment of pediatric cancers and other childhood diseases.
Developing new diagnostic and therapeutic tools to improve care for children.
Training the next generation of scientists and physician-scientists.
What they’re saying
“Over the years, our partnership with Virginia Tech has demonstrated the power of combining top-tier research expertise with a shared commitment to improving pediatric health,” said Catherine Bollard, MBChB, MD, senior vice president and chief research officer and director of the Center for Cancer and Immunology Research. “This expansion underscores our belief that by working together, we can accelerate discoveries and develop life-changing therapies for children with cancer and other rare diseases.”
“Children’s National Hospital has been an important partner for us in biomedical research and innovation,” said Michael Friedlander, PhD, Virginia Tech vice president for health sciences and technology. “Our collaboration deepened with the launch of Children’s National Research & Innovation Campus in Washington, D.C., and now, as our partnership grows even stronger, we’re poised together to take on some of the biggest challenges in cancer research to contribute to the health of children and adults.”
“Partnering with Children’s National connects us to a world-class clinical trial institute that has been a pioneer in treating brain tumors with focused ultrasound technology, and this presents a unique opportunity to help children and families struggling with cancer,” said Cheng-Chia “Fred” Wu, MD, PhD, a member of the Children’s National Brain Tumor Research Institute and a principal investigator in cancer research and faculty member at the Fralin Biomedical Research Institute in Roanoke and in the Virginia Tech Carilion School of Medicine.“I can’t wait to see where this takes us.”
Big picture
The initial focus of the collaboration is pediatric cancers, including brain tumors — among the most challenging childhood diagnoses. By combining Virginia Tech’s leading-edge technology and research infrastructure with Children’s National’s expertise in pediatric care, the organizations aim to make significant strides in understanding these diseases.
An interdisciplinary approach is at the heart of the ongoing strategy. The collaboration first began with the launch of a 12,000-square-foot Virginia Tech biomedical research facility within the Children’s National Research & Innovation Campus, which opened in 2020. Located on a 12-acre portion of the former Walter Reed Army Medical Center in Washington, D.C., the campus was the nation’s first innovation hub focused exclusively on pediatric research.
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The future of healthcare is unfolding before scientists and clinicians: Doctors are assisted by virtual scribes trained by artificial intelligence. Algorithms are reading MRIs. Smartphones are helping to detect strep throat. Machines diagnose children without access to care.
These and dozens of other artificial intelligence (AI) applications are being tested to enhance pediatric healthcare, and many were on display at the 2nd annual Children’s National Hospital-Virginia Tech Symposium on AI for Pediatric Health at the Children’s National Research & Innovation Campus.
Some highlights from the daylong conversation about the future of pediatric medicine, augmented by AI and generative AI models capable of producing new and critical content:
Marius George Linguraru, D.Phil., M.A., M.Sc., the Connor Family Professor in Research and Innovation and principal investigator in the Sheikh Zayed Institute for Pediatric Surgical Innovation: “Children are just not mini-adults. In pediatric care, we train pediatric specialists because kids die from different diseases than those that kill adults. Children also suffer from very impactful and rare conditions. If we train pediatric specialists well, we have to train AI algorithms in the same fashion.”
Rowland Illing, M.D., Ph.D., chief medical officer and director of global healthcare and nonprofits at Amazon Web Services: “In a short period of time, the complexity of the models available is astounding. Generative AI, just like AI, can impact outcomes at every step of the patient pathway, including the clinical workflow, care management and patient engagement. By creating a specific use case with generative AI, every step can be optimized to be smarter, which ultimately leads to improved patient care and outcomes.”
Children’s National Chief Academic Officer Nathan Kuppermann, M.D., M.P.H.: “AI in pediatric health is not just about identifying rare diseases. Its potential includes all aspects of clinical care, clinical operations, education and research. It has the potential to help educators enhance the novelty and impact of their methods and advance research with powerful tools to gather and analyze data.”
Alda Mizaku, vice president and chief data and artificial intelligence officer at Children’s National: “What excites me most about our future is the endless possibilities. We can use AI and data to uncover many things: rare diseases, operational efficiencies, time-saving and cost-saving solutions. This has to be done in a responsible way, and we must look at what some of the guardrails need to be.”
Throughout the day, expert panels offered insights into regulatory pathways to deploy AI in pediatric drugs and devices. The Food and Drug Administration’s Office of Science and Engineering Laboratories also provided guidance on collaborative tools for improving the representation of children and perinatal patients in AI-powered medical devices.
Moving the field forward
Early adopters of AI at Children’s National shared applications already under investigation, including efforts to segment and measure brain tumors on imaging, weigh the risk of strep throat with a smartphone camera and detect rheumatic heart disease with portable technology and an algorithm.
Dr. Linguraru, an expert in healthcare AI, said that artificial intelligence is no longer a hypothetical technology but is already remaking the healthcare system. “AI is here. What matters now is how we use it and how we train doctors to use it well,” he said.
The big picture
Through growing partnerships, Children’s National experts are teaming up with researchers at Virginia Tech on a series of AI-driven projects aimed at advancing pediatric health, including programs to rethink privacy in federated learning, forecast emergency department surges, extract clinical variables from documents to predict sepsis risks, identify rare genetic syndromes in children, and predict single-cell responses to genetic perturbations in pediatric developmental disorders.
Naren Ramakrishnan, Ph.D., director of the Sanghani Center at Virginia Tech and the Thomas L. Phillips Professor in the College of Engineering, said the partnership between the two academic centers is changing healthcare already and will continue to as the organizations offer future seed grants to support innovation in cardiology, neuroscience and oncology. “The roots have borne fruit,” he said.
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Jia-Ray Yu, Ph.D., will be an assistant professor at Virginia Tech’s Fralin Biomedical Research Institute at Virginia Tech Carilion and in the Department of Biomedical Sciences and Pathobiology in the Virginia-Maryland College of Veterinary Medicine, as well as an adjunct assistant professor at Children’s National Hospital starting Sept. 1.
Every year, 790 Americans are diagnosed with a rare and deadly form of brain cancer called a diffuse midline glioma, according to the National Cancer Institute. Tragically, only 2% of children with this disease will survive five years.
Jia-Ray Yu, Ph.D., a new assistant professor joining the Fralin Biomedical Research Institute at Virginia Tech Carilion and the Department of Biomedical Sciences and Pathobiology on Sept. 1, studies these fast-growing, treatment-resistant brain tumors, which commonly affect children, with hopes of identifying new therapeutic approaches. Yu will be the first of several cancer researchers to work in Virginia Tech’s brand-new research facility on the Children’s National Research & Innovation Campus in Washington, D.C.
Michael Friedlander, Virginia Tech’s vice president for health sciences and technology, and executive director of the Fralin Biomedical Research Institute, led Yu’s recruitment.
“Jia-Ray Yu is one of the rising leaders in understanding the molecular substrates of aggressive forms of pediatric brain cancer that can contribute to the identification of innovative therapeutic approaches. Moreover, his fundamental research into chromatin remodeling is at the very forefront of this area of emerging area importance in molecular biology,” Friedlander said. “We are very fortunate to have been able to attract Dr. Yu to Virginia Tech as we grow our greater cancer research community and our partnership with one of the nation’s pre-eminent children’s health care delivery and research systems, Children’s National Hospital.”
Yu studies how genes change when an ordinary brain cell develops malignant traits.
In particular, he examines changes in proteins called histones that spool strands of DNA molecules into a substance called chromatin, which forms chromosomes. In addition to packing genetic material into cells, these structures also play a key role in telling genes when to turn on or off.
Faulty histone proteins alter the chromatin’s structure, which in turn garbles the genetic instructions that regulate a cell’s behavior, growth rate, and identity. Furthermore, when this defective cell divides, its two daughter cells inherit the original cell’s chromatin, the malignant traits are passed on, and the cancer grows.
“These epigenetic features of chromatin are distinct from the DNA itself, yet they are inherited during cellular division,” said Yu.
Yu said 80% of tumors from diffuse midline gliomas begin with one cell that has a histone gene defect. He found when this tiny piece of a specific histone, called H3K27, stops working properly, it creates a series of domino-like reactions that cause normal cells to become cancerous.
Yu recently examined this molecular cascade as a postdoctoral fellow in the lab of Danny Reinberg, Terry and Mel Karmazin Professor in the NYU Grossman School of Medicine Department of Biochemistry and Molecular Pharmacology, and senior Howard Hughes Medical Institute Investigator.
The research team identified two genes, NSD1 and NSD2, appear to be the molecular fingers that tap the histone domino. When these genes are disabled, diffuse midline gliomas stop growing in a cultured lab dish, and in animal models. They also identified signaling pathways that could be targets for new drug therapies. Their findings are available in pre-print and will be published this summer in Science Advances.
Yu’s laboratory at the new Children’s National Research & Innovation Campus in Washington, D.C., will build on this fundamental question: How can chromatin-associated molecules be targeted to stop aggressive cancers?
Yu says that as he studies the molecular genesis of diffuse midline glioma, he may also identify therapeutic approaches for other diseases, such as leukemia and Sotos syndrome, that involve mutations in these chromatin-associated molecules.
His research team will combine biochemistry, single-molecule imaging, next-generation sequencing, biophysics, and preclinical research to develop and test new pharmaceutical alternatives to chemotherapy and radiation.
Yu was awarded a three-year American Cancer Society Postdoctoral Fellowship while working in Reinberg’s laboratory.
He completed a bachelor’s degree in biological science and technology at National Chiao Tung University in Taiwan, and his doctoral degree in genetics at Stony Brook University and Cold Spring Harbor Laboratory, where he studied signaling pathways in lung adenocarcinoma metastasis.
Recruitment for research positions in the Yu lab begins this summer.
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This pilot research program represents a growing academic research partnership between Children’s National and Virginia Tech. Last year, the two institutions announced that Virginia Tech will establish a biomedical research facility on the Children’s National Research & Innovation Campus.
Children’s National Hospital and Virginia Tech have awarded two $50,000 one-year pilot grants to multi-institutional teams of scientists for pediatric brain cancer research.
The inter-institutional program, which launched in December, promotes cross-disciplinary collaborations among researchers at both institutions. At Virginia Tech, the program is part of the Virginia Tech Cancer Research Alliance. Financial support for the program was provided by the Offices of the Physician-in-Chief and Chief Academic Officer at Children’s National, and by Virginia Tech’s Office of the Vice President for Health Sciences and Technology.
“We were delighted to see so many innovative and competitive research proposals for our first round of pilot grants in the area of brain cancer. By forging new research collaborations with our partners at Children’s National, we hope to make major strides in addressing one of the most common and devastating groups of cancers in children,” said Michael Friedlander, Virginia Tech’s vice president for health sciences and technology, and the executive director of the Fralin Biomedical Research Institute at VTC. “The pilot funding will bootstrap several programs to be able to acquire ongoing sustainable funding by providing the opportunity to test novel high impact ideas for new strategies for treating these disorders. There are simply too few good options for children in this space now and this partnership can change that for the better.”
The collaborative research initiative began through an agreement between the Fralin Biomedical Research Institute and the Children’s National Research Institute. The collaborative teams formed through a series of interactive discussions among Virginia Tech’s Cancer Research Alliance faculty members from the university’s Blacksburg and Roanoke campuses, and Children’s National’s neuro-oncology researchers.
“I am extremely excited by this collaboration between VT and CNH that is focused on pediatric brain tumors which is such an area of unmet need,” said Catherine Bollard, M.D., M.B.Ch.B.,, director of Children’s National’s Center for Cancer and Immunology Research. “I am confident that the funded proposals will soon advance our understanding of pediatric brain tumors and, more importantly, facilitate more joint efforts between two world-class institutions which is especially timely with the development of the Children’s National Research & Innovation Campus.”
Yanxin Pei, Ph.D., an assistant professor in the Center for Cancer Immunology Research at Children’s National, and Liwu Li, Ph.D., a professor of biological sciences in Virginia Tech’s College of Science, were awarded one of the pilot research grants to study how white blood cells called neutrophils are involved in metastatic MYC-driven medulloblastoma, an aggressive type of brain tumor in children that often resists conventional radiation and chemotherapies.
Yuan Zhu, Ph.D., the Gilbert Family Professor of Neurofibromatosis Research at Children’s National, and Susan Campbell, Ph.D., an assistant professor of animal and poultry sciences in Virginia Tech’s College of Agriculture and Life Sciences, were awarded funds to study glioma-induced seizures in mice with a genetic mutation that inhibits the production of P53, a key protein involved in suppressing cancer cell growth and division.
The successful applicants will receive funding starting this month and are expected to deliver preliminary data to support an extramural research application by 2024.
This pilot research program represents a growing academic research partnership between Children’s National and Virginia Tech. Last year, the two institutions announced that Virginia Tech will establish a biomedical research facility on the Children’s National Research & Innovation Campus. It will be the first research and innovation campus in the nation focused on pediatrics when it opens later this year and will house newly recruited teams of pediatric brain cancer researchers.
Liwu Li, Ph.D., Yanxin Pei, Ph.D., Susan Campbell, Ph.D., and Yuan Zhu, Ph.D., were awarded funding through the new pilot research program.
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Children’s National Hospital and Virginia Tech create formal partnership that includes the launch of a Virginia Tech biomedical research facility within the new Children’s National Research & Innovation Campus.
Children’s National Hospital and Virginia Tech recently announced a formal partnership that will include the launch of a 12,000-square-foot Virginia Tech biomedical research facility within the new Children’s National Research & Innovation Campus. The campus is an expansion of Children’s National that is located on a nearly 12-acre portion of the former Walter Reed Army Medical Center in Washington, D.C. and is set to open its first phase in December 2020. This new collaboration brings together Virginia Tech, a top tier academic research institution, with Children’s National, a U.S. News and World Report top 10 children’s hospital, on what will be the nation’s first innovation campus focused on pediatric research.
“Virginia Tech is an ideal partner to help us deliver on what we promised for the Children’s National Research & Innovation Campus – an ecosystem that enables us to accelerate the translation of potential breakthrough discoveries into new treatments and technologies,” says Kurt Newman, M.D., president and CEO, Children’s National. “Our clinical expertise combined with Virginia Tech’s leadership in engineering and technology, and its growing emphasis on biomedical research, will be a significant advance in developing much needed treatment and cures to save children’s lives.”
Earlier this year, Children’s National announced a collaboration with Johnson & Johnson Innovation LLC to launch JLABS @ Washington, DC at the Research & Innovation Campus. The JLABS @ Washington, DC site will be open to pharmaceutical, medical device, consumer and health technology companies that are aiming to advance the development of new drugs, medical devices, precision diagnostics and health technologies, including applications in pediatrics.
“We are proud to welcome Virginia Tech to our historic Walter Reed campus – a campus that is shaping up to host some of the top minds, talent and innovation incubators in the world,” says Washington, D.C. Mayor Muriel Bowser. “The new Children’s National Research & Innovation Campus will exemplify why D.C. is the capital of inclusive innovation – because we are a city committed to building the public and private partnerships necessary to drive discoveries, create jobs, promote economic growth and keep D.C. at the forefront of innovation and change.”
Faculty from the Children’s National Research Institute and the Fralin Biomedical Research Institute at Virginia Tech Carilion (VTC) have worked together for more than a decade, already resulting in shared research grants, collaborative publications and shared intellectual property. Together, the two institutions will now expand their collaborations to develop new drugs, medical devices, software applications and other novel treatments for cancer, rare diseases and other disorders.
“Joining with Children’s National in the nation’s capital positions Virginia Tech to improve the health and well-being of infants and children around the world,” says Virginia Tech President Tim Sands, Ph.D. “This partnership resonates with our land-grant mission to solve big problems and create new opportunities in Virginia and D.C. through education, technology and research.”
The partnership with Children’s National adds to Virginia Tech’s growing footprint in the Washington D.C. region, which includes plans for a new graduate campus in Alexandria, Va. with a human-centered approach to technological innovation. Sands said the proximity of the two locations – just across the Potomac – will enable researchers to leverage resources, and will also create opportunities with the Virginia Tech campus in Blacksburg, Va. and the Virginia Tech Carilion Health Science and Technology campus in Roanoke, Va.
Carilion Clinic and Children’s National have an existing collaboration for provision of certain specialized pediatric clinical services. The more formalized partnership between Virginia Tech and Children’s National will drive the already strong Virginia Tech-Carilion Clinic partnership, particularly for children’s health initiatives and facilitate collaborations between all three institutions in the pediatric research and clinical service domains.
Children’s National and Virginia Tech will engage in joint faculty recruiting, joint intellectual property, joint training of students and fellows, and collaborative research projects and programs according to Michael Friedlander, Ph.D., Virginia Tech’s vice president for health sciences and technology, and executive director of the Fralin Biomedical Research Institute at VTC.
“The expansion and formalization of our partnership with Children’s National is extremely timely and vital for pediatric research innovation and for translating these innovations into practice to prevent, treat and ultimately cure nervous system cancer in children,” says Friedlander, who has collaborated with Children’s National leaders and researchers for more than 20 years. “Both Virginia Tech and Children’s National have similar values and cultures with a firm commitment to discovery and innovation in the service of society.”
“Brain and other nervous system cancers are among the most common cancers in children (alongside leukemia),” says Friedlander. “With our strength in neurobiology including adult brain cancer research in both humans and companion animals at Virginia Tech and the strength of Children’s National research in pediatric cancer, developmental neuroscience and intellectual disabilities, this is a perfect match.”
The design of the Children’s National Research & Innovation Campus not only makes it conducive for the hospital to strengthen its prestigious partnerships with Virginia Tech and Johnson & Johnson, it also fosters synergies with federal agencies like the Biomedical Advanced Research and Development Authority, which will collaborate with JLABS @ Washington, DC to establish a specialized innovation zone to develop responses to health security threats. As more partners sign on, this convergence of key public and private institutions will accelerate discoveries and bring them to market faster for the benefit of children and adults.
“The Children’s National Research & Innovation Campus pairs an inspirational mission to find new treatments for childhood illness and disease with the ideal environment for early stage companies. I am confident the campus will be a magnet for big ideas and will be an economic boost for Washington DC and the region,” says Jeff Zients, who was appointed chair of the Children’s National Board of Directors effective October 1, 2019. As a CEO and the former director of President Obama’s National Economic Council, Zients says that “When you bring together business, academia, health care and government in the right setting, you create a hotbed for innovation.”
Ranked 7th in National Institutes of Health research funding among pediatric hospitals, Children’s National continues to foster collaborations as it prepares to open its first 158,000-square-foot phase of its Research & Innovation Campus. These key partnerships will enable the hospital to fulfill its mission of keeping children top of mind for healthcare innovation and research while also contributing to Washington D.C.’s thriving innovation economy.
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