Tag Archive for: NIH grant

DNA molecule

NIH awards $1m grant to study visual system

DNA molecule

The team will focus its work on FXS, a genetic condition that causes changes in a gene called Fragile X Messenger Ribonucleoprotein 1 (FMR1).

Researchers at Children’s National Hospital received a $1 million grant from the National Institutes of Health (NIH) to study the neural mechanisms behind visual deficits in fragile X syndrome (FXS). The work will provide new insights into how the visual system develops.

With the award from the National Eye Institute, the Children’s National team – led by Jason Triplett, Ph.D., principal investigator at the Center for Neuroscience Research – will work to unravel the poorly understood relationship between sensory deficits and neurodevelopmental disorders (NDDs). The findings are expected to provide clues into possible non-invasive therapeutics that could someday be used to resolve visual deficits in children with FXS and other disorders.

“Deficits in sensory processing, including vision, are common in many NDDs, but how these deficits arise is poorly understood, hampering the development of therapies,” Triplett said. “Using a powerful combination of molecular, anatomic and electrophysiologic techniques, we are hoping to get a comprehensive understanding of visual circuit development – and its disruption in fragile X syndrome.”

The big picture

The team will focus its work on FXS, a genetic condition that causes changes in a gene called Fragile X Messenger Ribonucleoprotein 1 (FMR1). The gene normally makes a protein needed for brain development, including the highly complex visual system. However, people with FXS do not properly make the protein, leading to a spectrum of developmental and cognitive delays.

Triplett’s team theorizes that ameliorating sensory deficits could improve other features of the disorder. Research has shown that sensory processing is critical for communication and learning, which are central components of the behavioral therapies aimed at treating intellectual delays and social anxiety.

Yet little is known regarding the neural basis of sensory deficits in FXS. Understanding how neuronal circuits are disorganized and dysfunctional in the context of the disorder will be a critical first step to developing therapeutics. In addition, given the prevalence of sensory dysfunction across NDDs, the work could have broader applications.

Children’s National Hospital leads the way

This NIH-supported work builds on prior research in the Triplett Laboratory. The collaborative nature among investigators in the Center for Neuroscience Research combined with the technical resources supported by the DC-Intellectual and Developmental Disabilities Research Center create an environment that maximizes the experimental capabilities of the Triplett Lab.

“We are so excited to continue this work,” Triplett said. “It highlights the importance of supporting fundamental research at the bench. We started with basic biological questions about how circuits wire up, and now we are embarking on research that could set the stage for potentially life-changing therapies.”

mitochondria

Grant funds study of two maternally inherited mitochondrial diseases

mitochondria

The National Institutes of Health awarded George Washington University and Children’s National Hospital a grant to study two maternally inherited mitochondrial diseases.

The National Institutes of Health awarded George Washington University and Children’s National Hospital a grant to study two maternally inherited mitochondrial diseases. Andrea Gropman, M.D., division chief of Neurodevelopmental Pediatrics and Neurogenetics at Children’s National, along with her co-investigator, Anne Chiaramello, M.D., from the George Washington University School of Medicine, will lead the study.

The proposed studies focus on two ultra-rare maternally inherited mitochondrial diseases:

  • Mitochondrial Encephalopathy, Lactic Acidosis and Stroke-like episodes (MELAS); and
  • Leber’s Hereditary Optic Neuropathy-Plus (LHON-Plus).

Both diseases are among those studied by the Rare Diseases Clinical Research Network.

“We are really pleased to be able to change the landscape for MELAS and LHON, two mitochondrial disorders with relentless progression and no treatment,” Dr. Gropman said. “This grant represents the fruition of an eight-year collaboration with my colleague Dr. Chiaramello and we are fortunate to be able to deliver this at Children’s National and serve our patients and community.”

Because patients currently do not have access to effective therapeutic intervention, this results in significant disability, morbidity and premature death. The UG3 phase of the study will focus on translational MELAS and LHON-Plus studies and submission of an IND protocol to the Food and Drug Administration. The UH3 phase will focus on a basket clinical trial with MELAS and LHON-Plus to:

  • Provide proof-of-concept that the basket design can be applied to divergent ultra-rare diseases.
  • Advance the dataset for safety and pharmacokinetics/pharmacodynamics of our lead compound for a larger number of patients than in a conventional clinical trial setting.
  • Gather outcomes and practical information for optimizing the design of future basket clinical trial.

“Dr. Gropman is dedicated to giving children with MELAS the very best care,” said Elizabeth Wells, M.D., vice president of Neuroscience and Behavioral Medicine Center at Children’s National. “This new research funding is exciting and means more patients can benefit from the expertise she has developed at Children’s National.”

illustration of Research & Innovation Campus

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

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

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

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

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

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

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

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

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

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

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

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

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

illustration of Research & Innovation Campus

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