Tag Archive for: Center for Translational Research

Monika K. Goyal, M.D., M.S.C.E., and Beth A. Tarini, M.D., M.S., M.B.A.

Drs. Goyal and Tarini to lead Center for Translational Research

Monika K. Goyal, M.D., M.S.C.E., and Beth A. Tarini, M.D., M.S., M.B.A.

As CTR co-directors, Drs. Goyal and Tarini will lead the hospital’s mission to advance translational science, clinical research and community health.

Children’s National Hospital has appointed two nationally regarded leaders in pediatric research – Monika K. Goyal, M.D., M.S.C.E., and Beth A. Tarini, M.D., M.S., M.B.A. – to head its Center for Translational Research (CTR), a hub of high-impact scientific investigation that touches nearly every pediatric specialty.

As CTR co-directors, Drs. Goyal and Tarini will lead the hospital’s mission to advance translational science, clinical research and community health. They will begin their new roles on July 1.

Moving the field forward

“It is truly an honor to lead the CTR at such a pivotal moment in pediatric health,” said Dr. Goyal, an emergency medicine specialist and health services researcher. “I look forward to helping Children’s National lead the science on advancing health equity for the patients, families and communities we serve, both locally and nationally.”

As the largest of the six centers within the Children’s National Research Institute, CTR is pivotal in finding groundbreaking ways to improve health across pediatric medicine. Using a “bench to bedside” approach, the CTR faculty strives to seamlessly translate science from the laboratory bench to the patient’s bedside, moving pediatric medicine forward as expeditiously as possible to bring advances into the community.

“CTR is uniquely positioned to solve the biggest healthcare issues facing our pediatric patients,” said Dr. Tarini, a pediatrician and national leader in newborn screening research and policy. “I look forward to leading our diverse faculty of physicians and researchers as they leverage their front-line experience and innovative research to improve child health.”

Why we’re excited

Dr. Tarini joined Children’s National in 2018 and is currently the associate director for CTR. She was recently promoted to tenured professor of Pediatrics at George Washington University and has extensively studied policies to optimize the delivery of genetic services to families of newborns. In January, Dr. Tarini was appointed to a National Academies of Sciences, Engineering and Medicine Committee to examine the current landscape of newborn screening systems, processes and research in the United States. Dr. Tarini has obtained $10 million in federal and foundation funding, and she has served as president of the Society for Pediatric Research.

Dr. Goyal joined Children’s National in 2012. She is the inaugural endowed chair for Women in Science and Health and has served as the associate division chief for Academic Affairs and Research within the Emergency Department since 2018. She was recently promoted to tenured professor of Pediatrics and Emergency Medicine at George Washington University. Dr. Goyal is a nationally renowned equity science scholar and has published over 130 peer-reviewed manuscripts. She has secured more than $25 million in federal and foundation funding to address disparities in adolescent sexual health, pain management and firearm violence.

Children’s National leads the way

Catherine Bollard, M.D., M.B.Ch.B., interim chief academic officer, said she looks forward to seeing the advances in pediatric health guided by these two outstanding researchers. “By harnessing the immense talent within Children’s National for our search, we found two exceptional leaders in Drs. Goyal and Tarini,” Dr. Bollard said. “Their work promoting research that accelerates discovery across the continuum of bench, bedside and community has already made a significant impact.”

Maureen Lyon, Ph.D.

Pilot program improves well-being of families during advanced care planning


Children with life-limiting rare diseases and their caregivers face tremendous stress and anxiety about the heart-breaking decisions before them. A new intervention – designed at Children’s National Hospital to support the palliative needs of these families – improved their spiritual and emotional well-being, according to new research published in the journal, Pediatrics.

Called FACE Rare (FAmily CEntered Pediatric Advance Care Planning Intervention for Rare Diseases), the counseling tools were found to be safe, effective and increased feelings of peace among families in this underserved population.

“Seventy-four percent of the families in that intervention group reported feelings of sadness, yet 100% reported our pilot intervention was a worthwhile experience,” said Maureen Lyon, Ph.D., a clinical psychologist and principal investigator at the Center for Translational Research at Children’s National. “If you’re talking about the possibility that the worst thing in the world would happen to you – that your child might die, and what you would want for them – the families found that our intervention helped. They had a place to process their feelings and consider what would be important to their child.”

The big picture

A rare disease is defined as any condition affecting fewer than 200,000 people in the United States. In pediatrics, these diseases often require constant caregiving and require families to face the cruel reality that the diagnosis may be life-limiting. In such cases, clinical teams often decide that conversations about advanced care planning (ACP) are needed.

The pilot-phase, randomized trial enrolled patients from Children’s National between 2021 and 2023. Research nurses underwent two days of training to be certified in the new intervention. Families assigned to the new approach received three, weekly 60-minute sessions and were evaluated using evidence-based assessment tools, including the Carer Support Needs Assessment Tool/Action Plan and the Next Steps: Respecting Choices Pediatric ACP Conversation. Measures of anxiety and spiritualization were tracked, and families returned after three months for follow-up.

Patients had a range of diagnoses that put tremendous strain on the caregivers, including complex digestive disorders, white-matter diseases and rare forms of epilepsy. Yet the caregivers expressed similar challenges: having time for themselves during the day, knowing what to expect in the future for their child’s care, and managing financial, legal and work issues.

What we found

Reinforcing the need to improve engagement and support for these families, the investigative team found that those who received the FACE Rare intervention reported higher levels of spiritual meaning and peace than those who received “treatment as usual.” All families living below the poverty line reported greater anxiety, and noteworthy to the team, black caregivers were less likely to report caregiver distress than non-black caregivers.

Dr. Lyon said future research needs to understand better how families respond to the challenges of rare diseases and unique social determinants of health that can change the approach to care.

“In some cultures, and families, having strong feelings is discouraged,” Dr. Lyon said. “We want to give families and caregivers space to have these emotions and think about what would be important for their child, if the worst were to happen. They appreciated that they participated in the program and had the space to consider these difficult questions.”

Read the full study, “Advance Care Planning for Children with Rare Diseases: A Pilot RCT,” in Pediatrics.

teen parents with baby

Community-wide program to support teen parents serves as a model for engagement

More than 500 adolescent mothers, caregivers and community members benefitted from a coordinated “collective impact” model to provide support aimed at addressing the litany of strains faced by teen parents, according to a case study published Wednesday in the journal Pediatrics.

Known as the District of Columbia Network for Parenting and Expectant Teens (DC NEXT), the model used well-tested pillars of community organization to provide services and care that bolstered the well-being of pregnant and expectant teens in the city. The case study found that the 3-year-old program, which included teen advisors to help guide the mission, could serve as a model in other areas with high rates of teen parents.

“There’s no system of care for teen parents in Washington, D.C., or really anywhere in our country,” said Yael Smiley, M.D., Children’s National Hospital pediatrician and the study’s lead author. “Our coalition came together to connect the resources and the people who care about young parents and their families to improve their health, their outcomes, their well-being, and set them up for success. We fuse representatives from healthcare, education and housing policy — people who care about creating the very best outcomes possible.”

The patient benefit

More than 140,000 teens became parents in 2021 in the United States. In Washington, D.C., more than 300 babies are born to teens each year, and the rate of births to young teens ages 15 to 17 is double the national average. These young parents often face barriers to childcare and education and poor birth outcomes, often stemming from a lack of access to prenatal care.

To address these obstacles, the network assembled a city-wide network in 2020 to follow the five pillars of an evidence-based “collective impact” model:

  • Create a common agenda
  • Design shared measurement systems
  • Host mutually reinforcing activities
  • Foster continuous communication
  • Rely on trusted backbone support

What’s unique

DC NEXT was housed at the District of Columbia Primary Care Association (DCPCA), which received the 3-year, $4.5 million federal grant and provided the leadership. At Children’s National, Dr. Smiley led a team that partnered with DCPCA and Howard University to direct a network of community clinics, nonprofits and other organizations whose mission was to support adolescent parents. Hundreds of client-facing staff members were trained to provide trauma-informed, human-centered care. DC NEXT also engaged directly with teen mothers through well-being surveys that led to improved access to essential programs, including resources for housing and food security. Over 550 young parents and caregivers have been impacted by the program.

The leadership quickly realized that the teen parents needed a voice and created a “context team” of paid teen advisors who provided insights into their unique experiences and needs as parents. They help set the agenda, choose program offerings and tailor communications to their peers.

“If the network can continue to grow and support young parents to achieve their health and well-being goals, the impact will be felt across generations,” said Dr. Smiley.

 

cystic kidney disease

NIH $4 million grant funds new core center for childhood cystic kidney disease

cystic kidney disease

The University of Alabama at Birmingham (UAB), in collaboration with Children’s National Hospital has received a five-year, $4 million grant from the National Institute of Diabetes and Digestive and Kidney Diseases, part of the National Institutes of Health (NIH) to create a core center for childhood cystic kidney disease (CCKDCC). The UAB-CCKDCC will conduct and facilitate research into the causes of and possible treatments for cystic kidney diseases, particularly those that present in childhood.

The UAB/Children’s National grant is a U54 center grant, an NIH funding mechanism to develop a multidisciplinary attack on a specific disease entity or biomedical problem area. With this grant, UAB joins with investigators at the University of Kansas and the University of Maryland-Baltimore as part of the NIH Polycystic Kidney Disease Research Resource Consortium. The NIH describes the consortium as a framework for effective collaboration to develop and share research resources, core services and expertise to support innovation in research related to polycystic kidney disease.

“Infants with childhood cystic kidney disease may develop kidney failure within a few years after birth and some need dialysis and kidney transplantation before they reach adulthood,” said Lisa Guay-Woodford, M.D., director of the Clinical and Translational Science Institute at Children’s National and co-director of the UAB-CCKDCC. “In many cases, the earlier the onset of symptoms, the more severe the outcome.”

“The intent is to accelerate the science and advance research into new therapies for cystic kidney disease through enhanced sharing of resources and the establishment of a robust research community,” said Bradley K. Yoder, Ph.D., professor and chair of the UAB Department of Cell, Developmental and Integrative Biology and co-director of the UAB-CCKDCC. “Childhood polycystic disease can be a devastating condition for children and their families.”

The UAB-CCKDCC will focus primarily on childhood polycystic kidney disease, a condition that affects about one in 20,000 infants in the United States. The center’s primary goals are:

  • Provide the Polycystic Kidney Disease Research Resource Consortium members with access to phenotypic, genetic and clinical information and biomaterials from CCKD patients
  • Analyze pathways involved in cyst pathogenesis through the generation of verified genetic model systems and biosensor/reporter systems
  • Assess the impact of patient variants on cystic disease proteins through generation and validation of innovative models
  • Provide ready access to biological materials from genetic CCKD models
  • Develop efficient pipelines for in vitro and in vivo preclinical testing of therapeutic compounds

Dr. Guay-Woodford is an internationally recognized pediatric nephrologist with a research program focused on identifying clinical and genetic factors involved in the pathogenesis of inherited renal disorders, most notably autosomal recessive polycystic kidney disease (ARPKD). Her laboratory has identified the disease-causing genes in several experimental models of recessive polycystic kidney disease and her group participated in the identification of the human ARPKD gene as part of an international consortium. In addition, her laboratory was the first to identify a candidate modifier gene for recessive polycystic kidney disease. For her contributions to the field, she was awarded the Lillian Jean Kaplan International Prize for Advancement in the Understanding of Polycystic Kidney Disease, given by the Polycystic Kidney Disease Foundation and the International Society of Nephrology.

Beth Tarini

Getting to know SPR’s future President, Beth Tarini, M.D., MS

Beth Tarini

Quick. Name four pillar pediatric organizations on the vanguard of advancing pediatric research.

Most researchers and clinicians can rattle off the names of the Academic Pediatric Association, the American Academy of Pediatrics and the American Pediatric Society. But that fourth one, the Society for Pediatric Research (SPR), is a little trickier. While many know SPR, a lot of research-clinicians simply do not.

Over the next few years, Beth A. Tarini, M.D., MS, will make it her personal mission to ensure that more pediatric researchers get to know SPR and are so excited about the organization that they become active members. In May 2019 Dr. Tarini becomes Vice President of the society that aims to stitch together an international network of interdisciplinary researchers to improve kids’ health. Four-year SPR leadership terms begin with Vice President before transitioning to President-Elect, President and Past-President, each for one year.

Dr. Tarini says she looks forward to working with other SPR leaders to find ways to build more productive, collaborative professional networks among faculty, especially emerging junior faculty. “Facilitating ways to network for research and professional reasons across pediatric research is vital – albeit easier said than done. I have been told I’m a connector, so I hope to leverage that skill in this new role,” says Dr. Tarini, associate director for Children’s Center for Translational Research.

“I’m delighted that Dr. Tarini was elected to this leadership position, and I am impressed by her vision of improving SPR’s outreach efforts,” says Mark Batshaw, M.D., Executive Vice President, Chief Academic Officer and Physician-in-Chief at Children’s National. “Her goal of engaging potential members in networking through a variety of ways – face-to-face as well as leveraging digital platforms like Twitter, Facebook and LinkedIn – and her focus on engaging junior faculty will help strengthen SPR membership in the near term and long term.”

Dr. Tarini adds: “Success to me would be leaving after four years with more faculty – especially junior faculty – approaching membership in SPR with the knowledge and enthusiasm that they bring to membership in other pediatric societies.”

SPR requires that its members not simply conduct research, but move the needle in their chosen discipline. In her research, Dr. Tarini has focused on ensuring that population-based newborn screening programs function efficiently and effectively with fewer hiccups at any place along the process.

Thanks to a heel stick to draw blood, an oxygen measurement, and a hearing test, U.S. babies are screened for select inherited health conditions, expediting treatment for infants and reducing the chances they’ll experience long-term health consequences.

“The complexity of this program that is able to test nearly all 4 million babies in the U.S. each year is nothing short of astounding. You have to know the child is born – anywhere in the state – and then between 24 and 48 hours of birth you have to do testing onsite, obtain a specific type of blood sample, send the blood sample to an off-site lab quickly, test the sample, find the child if the test is out of range, get the child evaluated and tested for the condition, then send them for treatment. Given the time pressures as well as the coordination of numerous people and organizations, the fact that this happens routinely is amazing. And like any complex process, there is always room for improvement,” she says.

Dr. Tarini’s research efforts have focused on those process improvements.

As just one example, the Advisory Committee on Heritable Disorders in Newborns and Children, a federal advisory committee on which she serves, was discussing how to eliminate delays in specimen processing to provide speedier results to families. One possible solution floated was to open labs all seven days, rather than just five days a week. Dr. Tarini advocated for partnering with health care engineers who could help model ways to make the specimen transport process more efficient, just like airlines and mail delivery services. A more efficient and effective solution was to match the specimen pick-up and delivery times more closely with the lab’s operational times – which maximizes lab resources and shortens wait times for parents.

Conceptual modeling comes so easily for her that she often leaps out of her seat mid-sentence, underscoring a point by jotting thoughts on a white board, doing it so often that her pens have run dry.

“It’s like a bus schedule: You want to find a bus that not only takes you to your destination but gets you there on time,” she says.

Dr. Tarini’s current observational study looks for opportunities to improve how parents in Minnesota and Iowa are given out-of-range newborn screening test results – especially false positives – and how that experience might shake their confidence in their child’s health as well as heighten their own stress level.

“After a false positive test result, are there parents who walk away from newborn screening with lingering stress about their child’s health? Can we predict who those parents might be and help them?” she asks.

Among the challenges is the newborn screening occurs so quickly after delivery that some emotionally and physically exhausted parents may not remember it was done. Then they get a call from the state with ominous results. Another challenge is standardizing communication approaches across dozens of birthing centers and hospitals.

“We know parents are concerned after receiving a false positive result, and some worry their infant remains vulnerable,” she says. “Can we change how we communicate – not just what we say, but how we say it – to alleviate those concerns?”