Children’s National experts developed a novel visualization platform, called “HoloUS,” for ultrasound-guided procedures using commercially available see-through augmented reality (AR) glasses, according to a pre-clinical study published in the International Journal of Computer Assisted Radiology and Surgery. The finding suggests that the HoloUS application enhanced the user experience and simplified hand-eye coordination.
“By eliminating head-turning and back-and-forth viewing of the patient and the ultrasound screen, our solution could improve the safety, accuracy, intuitiveness, and length of ultrasound-guided procedures, while making them easier to learn and perform,” said Raj Shekhar, M.S., Ph.D., principal investigator for Sheikh Zayed Institute for Pediatric Surgical Innovation at Children’s National and senior author in the study.
Ultrasound-guided procedures demand a high degree of hand-eye coordination to advance a needle with one hand while holding the ultrasound probe with another as they visualize the live ultrasound image. With this application, the practitioner will wear the AR glasses that will allow them to visualize the ultrasound image rendered live in their field of view.
“We are excited about the possibility of improving the accuracy and efficiency from augmented reality visualization because these benefits may translate clinically in the upcoming years after conducting carefully designed clinical studies,” said Trong Nguyen, staff scientist for Sheikh Zayed Institute for Pediatric Surgical Innovation at Children’s National and lead author.
Microsoft’s HoloLense headset, often used for hands-free work and video gaming, set the stage to develop the custom application that would display information from a portable ultrasound machine connected through a wireless network in the form of a hologram. The voice controls on the HoloLense allowed scaling and movement of the ultrasound imaging while displaying in real-time.
Largely driven by video gaming and consumer applications, AR and virtual reality technologies have been growing and are expected to grow at an even faster clip in the coming decade.
“We are a handful of researchers exploring the innovative application of the technology for ultrasound visualization,” said Shekhar. “We are trying to establish ourselves as the leader in the use of AR for pediatric ultrasound procedures.”
To further advance this technology, Shekhar’s team has an IRB protocol pending that will continue to shed light on the benefits through more bench testing and an NIH grant proposal that is also pending. In the meantime, they will adopt second-generation glasses to continue to improve the technology.
The scientific community continues to debate AI’s possibility of outperforming humans in specific tasks. In the context of the machine’s performance versus the clinician, Linguraru et al. argue that the community must consider social, psychological and economic contexts in addition to the medical implications to answer this puzzling question.
In a special report published in Radiology: Artificial Intelligence, a Children’s National Hospital expert and other institutions discussed a shared multidisciplinary vision to develop radiologic and medical imaging techniques through advanced quantitative imaging biomarkers and artificial intelligence (AI).
“AI algorithms can construct, reconstruct and interpret radiologic images, but they also have the potential to guide the scanner and optimize its parameters,” said Marius George Linguraru, D.Phil., M.A., M.Sc., principal investigator in the Sheikh Zayed Institute for Pediatric Surgical Innovation at Children’s National. “The acquisition and analysis of radiologic images is personalized, and radiologists and technologists adapt their approach to every patient based on their experience. AI can simplify this process and make it faster.”
The scientific community continues to debate AI’s possibility of outperforming humans in specific tasks. In the context of the machine’s performance versus the clinician, Linguraru et al. argue that the community must consider social, psychological and economic contexts in addition to the medical implications to answer this puzzling question.
Still, they believe that developing a useful radiologic AI system designed with the participation of radiologists could complement and possibly surpass human’s interpretation of the visuals.
Given AI’s potential applications, the authors encouraged radiologists to access many freely available resources to learn about machine learning, and radiomics to familiarize with basic concepts. Coursera, for example, can teach radiologists about convolutional neural networks and other techniques used by AI researchers.
Conversely, AI experts must reach out to radiologists and participate in public speaking events about their work. According to the researchers, during those engagement opportunities, clinicians understood the labor-saving benefits of automatic complex measurements on millions of images—something that they have been doing manually for years.
There are also hurdles around this quest of automation, which Linguraru et al. hope both fields can sort out by working together. A critical challenge that the experts mentioned was earning the trust of clinicians that are skeptical about the “black box” functionality of AI models, which makes it hard to understand and explain the behavior of a model.
Some questions, too, need answers on how to best leverage both human intelligence and AI by using human-in-the-loop where people train, tune, and test a particular algorithm, or AI in-the-loop where this different framing generates AI input and reflection in human systems.
“The key is to have a good scientific premise to adequately train and validate the algorithms and make them clinically useful. At that point, we can trust the box,” said Linguraru. “In radiology, we should focus on AI systems with radiologists in-the-loop, but also on training radiologists with AI in-the-loop, particularly as AI systems are getting smarter and learning to work better with radiologists.”
The experts also provided possible solutions to sharing large datasets, how to build datasets that allows robust investigations and how to improve the quality of a model that might be compared against human’s gold standard.
This special report is the second in a series of panel discussions hosted by the Radiological Society of North America and the Medical Image Computing and Computer Assisted Intervention Society. The discussion builds upon the first in the series “Machine Learning for Radiology from Challenges to Clinical Applications” that touched on how to incentivize annotators to participate in projects, the promotion of “team science” to address research questions and challenges, among other topics.
https://innovationdistrict.childrensnational.org/wp-content/uploads/2021/12/illustration-of-AI-chip.png300400Innovation Districthttps://innovationdistrict.childrensnational.org/wp-content/uploads/2023/12/innovationdistrict_logo-1-1030x165.pngInnovation District2021-12-07 10:06:512021-12-07 10:06:51How radiologists and data scientists can collaborate to advance AI in clinical practice
The Society for Pediatric Research (SPR) announced five new members from Children’s National Hospital: Drs. Rana Hamdy, Panagiotis Kratimenos, Brynn Marks, Shayna Coburn and Katie Donnelly.
The Society for Pediatric Research (SPR) announced five new members from Children’s National Hospital. Established in 1929, SPR’s mission is to create a multi-disciplinary network of diverse researchers to improve child health.
Membership in SPR is a recognized honor in academic pediatrics. It requires nomination by academic peers and leaders as well as recognition of one’s role as an independent, productive child health researcher.
“I am so proud of our faculty and all that they have accomplished. I am thrilled that they have been recognized for their achievements,” said Beth A. Tarini, M.D., M.S., SPR president and associate director for the Center for Translational Research at Children’s National Hospital.
SPR 2021 active new members from Children’s National are:
Katie Donnelly, M.D., M.P.H., attending physician in the Emergency Department at Children’s National Hospital. She is the medical director for Safe Kids DC, an organization dedicated to preventing accidental injuries in children in Washington DC. Her personal research interest is in preventing firearm injuries in children and she is a member of Safer through Advocacy, Firearm Education and Research (SAFER), a multidisciplinary team dedicated to firearm injury prevention at Children’s National. She is also the medical director of the newly founded hospital-based violence intervention program at Children’s National and an associate professor of pediatrics and emergency medicine at The George Washington University.“To be recognized by my peers as a researcher with a significant contribution to our field is very validating. It also opens a world of potential collaborations with excellent scientists, which is very exciting!” said Dr. Donnelly. “I am grateful for the immense support offered to me by the Division of Emergency Medicine to complete the research I am passionate about, especially my mentor Monika Goyal.”
Panagiotis Kratimenos, M.D., Ph.D., newborn intensivist and neuroscientist at Children’s National. He studies mechanisms of brain injury in the neonate, intending to prevent its sequelae later in life. Dr. Kratimenos’ interest lies in identifying therapies to prevent or improve neurodevelopmental disabilities of sick newborns caused by prematurity and perinatal insults.“Being a member of SPR is a deep honor for me. SPR has always been a ‘mentorship home’ for me since I was a trainee and a member of the SPR junior section,” said Dr. Kratimenos. “A membership in the SPR allows us to access a very diverse, outstanding team of pediatric academicians and researchers who support the development of physician-scientists, honors excellence through prestigious grants and awards, and advocates for children at any level either locally, nationally, or internationally.”
Rana Hamdy, M.D., M.P.H., M.S.C.E., pediatric infectious diseases physician at Children’s National and Director of the Antimicrobial Stewardship Program. She is an assistant professor of pediatrics at George Washington University School of Medicine and Health Sciences. Her area of expertise focuses on the prevention and treatment of antimicrobial resistant infections and the promotion of good antimicrobial stewardship in inpatient and outpatient settings.“It’s an honor to be joining the Society for Pediatric Research and becoming part of this distinguished multidisciplinary network of pediatric researchers,” said Dr. Hamdy. “I look forward to the opportunity to meet and work with SPR members, make connections for future collaborations, as well as encourage trainees to pursue pediatric research through the opportunities that SPR offers.”
Shayna Coburn, Ph.D., director of Psychosocial Services in the Celiac Disease Program at Children’s National. She is a licensed psychologist specializing in coping and interpersonal relationships in chronic illness treatment, particularly for conditions involving specialized diets. She holds an appointment as assistant professor of psychiatry and behavioral sciences at The George Washington University School of Medicine and Health Sciences. Her work has focused on promoting effective doctor-patient communication, reducing healthcare disparities and supporting successful adherence across the developmental span of childhood and adolescence. She currently has a Career Development Award from National Institute of Diabetes and Digestive and Kidney Diseases to refine and test a group intervention designed to improve self-management and quality of life in teens with celiac disease.
“I hope that my background as a psychologist researcher will help diversify SPR. As an SPR member, I hope to encourage more opportunities for training, awards, and other programs that would be inclusive of clinician researchers who may not hold a traditional medical degree,” said Dr. Coburn.
Brynn Marks, M.D., M.S.-H.P.Ed., endocrinologist at Children’s National. As a clinical and translational scientist her goal is to use unique personal experiences and training to optimize both patient and provider knowledge of and behaviors surrounding diabetes technologies thereby realizing the potential of diabetes technologies improve the lives and clinical outcomes of all people living with diabetes. Her experiences as a person living with Type 1 diabetes have undoubtedly shaped her clinical and research interests in diabetes management and medical education.
“It is an honor to be accepted for membership in the Society for Pediatric Research,” said Dr. Marks. “Being nominated and recognized by peers in this interprofessional pediatric research community will allow me networking and growth opportunities as I continue to advance my research career.”
https://innovationdistrict.childrensnational.org/wp-content/uploads/2021/12/2021-SPR-members.png300400Innovation Districthttps://innovationdistrict.childrensnational.org/wp-content/uploads/2023/12/innovationdistrict_logo-1-1030x165.pngInnovation District2021-12-02 09:46:192022-07-15 09:32:51Five Children’s National Hospital faculty named to Society for Pediatric Research
Dr. Toner, who is an attending physician in the Center for Cancer and Blood Disorders and the Center for Cancer and Immunology Research at Children’s National, received a $300,000 Hyundai Scholar Hope Grant that she will use to develop and functionally evaluate a novel T cell therapy which can be translated to the clinic for treatment of pediatric patients with acute myeloid leukemia (AML).
Currently, patients with relapsed AML have very poor outcomes and the success that T cell therapy has had in treating B-cell malignancies has not yet been achieved for AML. Dr. Toner’s goal is to try to overcome some of these barriers with a novel T cell therapy which combines both native and chimeric T cell receptors to target AML.
“There are currently critical barriers to the success of T cell therapies for the treatment of AML,” Dr. Toner explains. “Successful completion of this research would allow for translation of a novel CAR-TAA-T therapy to the clinic for the treatment of relapsed/refractory AML, which has very poor prognosis.”
Meanwhile, Dr. Kinoshita, a pediatric hematology oncology fellow at Children’s National, received a $200,000 Hyundai Young Investigator Grant. She will use the funds to evaluate the immunobiology of multi-antigen specific T cell therapy infused to patients to reduce the two most common causes of morbidity and mortality following hematopoietic stem cell transplant (HSCT) for malignant disease: relapse and infection.
The administration of multiantigen specific T cells targeting tumor and viral-associated antigens following stem cell transplant may serve to prolong remission of malignant disease and prevent and treat viral infections that can cause devastating disease in children. Dr. Kinoshita’s study will evaluate the anti-viral and anti-leukemia immune response in vivo following targeted T cell therapy.
“There have been incredible advancements in the field of pediatric oncology and bone marrow transplant over the past 20-30 years but there are still many areas in which we need to continue to improve,” Dr. Kinoshita says. “Our patients and their families go through so much to get into remission and it is devastating if they relapse or develop severe infectious complications. Adoptive immunotherapy is a promising tool in aiding to treat and prevent these complications, particularly for patients with high-risk hematologic malignancies.”
The Hyundai Scholar Hope Grants and the Hyundai Young Investigator Grants are competitive research grants that are peer-reviewed by the Hyundai Hope on Wheels Medical Advisory Committee, which is comprised of leading pediatric oncologists from children’s hospitals and research institutions nationwide. The grants are open to U.S.-based Children’s Oncology Group member institutions.
The Meritorious Achievement Award recognizes a person whose achievements have made a significant impact in the field of congenital heart disease and heart health in the young and have helped to further the mission of the Young Hearts council. The council’s mission is to improve the health of children and adults with congenital heart disease and acquired heart disease during childhood through research, education, prevention and advocacy.
Dr. Berul received this award in recognition of his lifelong achievements in the field of pediatric electrophysiology. He is known for his development of innovative electrophysiologic studies for phenotypic evaluations of genetically manipulated pre-clinical models. Over the past two decades, his research focus and passion have been to develop novel minimally invasive approaches to the heart and improving methods for pediatric pacing and defibrillation.
He has also mentored dozens of trainees who have gone on to successful careers and particularly advocates for young investigators and clinician-scientists. He is known for his collaborative style and for supporting advancement of faculty physicians in academic medicine.
Dr. Berul has served on multiple society committees, task forces and writing groups, and is currently an associate editor for the Heart Rhythm Society’s journal. He is also actively involved in other key organizations such as Mended Little Hearts and the Pediatric and Congenital Electrophysiology Society (PACES). He has more than 300 publications and is an invited speaker nationally and internationally in the areas of pediatric cardiac electrophysiology and miniaturized device development.
Although diabetes technologies are associated with improvements in glycemic control and health-related quality of life among people with type 1 diabetes (T1D), the use and perceptions of continuous glucose monitors (CGM) and insulin pumps within the cystic fibrosis (CF) community have not been well documented.
In a recent study published in Diabetes Technology & Therapeutics, Brynn Marks, M.D., MS-HPEd, and co-authors, found that compared to T1D, rates of sustained diabetes technology use in the cystic fibrosis-related diabetes (CFRD) community are low, despite perceived benefits. The authors conclude that better insurance coverage to mitigate cost, better patient education and confirmation that these technologies improve health and patient-reported outcomes may increase uptake.
https://innovationdistrict.childrensnational.org/wp-content/uploads/2021/11/insulin-pump.png300400Innovation Districthttps://innovationdistrict.childrensnational.org/wp-content/uploads/2023/12/innovationdistrict_logo-1-1030x165.pngInnovation District2021-11-26 11:58:102022-07-15 09:32:57Diabetes technology use in the cystic fibrosis community
Epstein-Barr virus is a member of the herpes family and it spreads primarily through saliva.
Children’s National Hospital experts provided a contemporary description of the epidemiology, clinical presentation and management of chronic active Epstein-Barr virus (CAEBV), shedding light on this very rare disease. The paper, published in Blood Advances, assessed 57 patients outside of Asia — the biggest international retrospective cohort study published in the Western world.
Epstein-Barr virus is a member of the herpes family and it spreads primarily through saliva. Once a person is infected with Epstein-Barr virus, the immune system will control the infections, but the virus lies in a dormant state in the patient’s B Cells. However, in some patients, there is a failure of the body to control the infection, and the virus is found inside the patient’s T and/or NK cells. These rare patients are diagnosed with CAEBV. The hallmark of the disease is proliferation of Epstein-Barr virus-infected T or NK cells that infiltrate tissues, leading to end-organ damage. Patients most often experience fevers, hepatosplenomegaly, liver inflammation, cytopenias and lymphoproliferation that may progress to lymphoma.
Given it is most prevalent in Asia, little is known about the disease in the Western world. There has only been one published paper regarding the outcomes of patients in the U.S., which included 19 patients amassed over 28 years, and was published a decade ago.
Multiple treatments have been attempted to control the disease, but none have resulted in consistent remission. Historically, the consensus is to use steroids and/or antiviral drug in combination with proteasome inhibitor agents. In some cases, clinicians also use cytotoxic chemotherapy to reduce disease burden and improve the patient’s condition before HSCT. Still, this approach is limited because most patients die due to the progression of their disease despite these interventions.
Ultimately, most of these patients are referred for allogeneic hematopoietic stem cell transplantation (HSCT), which is the only known curative therapy for CAEBV. However, the best approach to control disease prior to HSCT, as well as the optimal conditioning regimen, are unknown.
“For the first time in many years, we provide insight on contemporary treatment options to consider for patients with CAEBV, as well as identifying risk factors for worse outcomes,” said Blachy Dávila Saldaña, M.D., blood and marrow transplant specialist at Children’s National and lead author of the study. “HSCT is curative, but patients need to be considered prior to the evolution of more advanced disease, particularly lymphoma. We also provide a new platform that will inform research on new interventions and therapies for this population.”
“CAEBV remains a challenging disorder to treat, especially once severe complications develop,” said Catherine Bollard, M.D., M.B.Ch.B., director of the Center for Cancer and Immunology Research at Children’s National. “However, our data suggests that T cell modulating therapies may enhance disease control, and future studies should address this question in a controlled setting.”
Future steps also include performing genetic studies to identify those at risk of developing the disease, and developing new platforms for treatment, including checkpoint inhibitors and cytotoxic lymphocyte therapies (CTL’s), which is a form of adoptive immunotherapy that employs virus-specific T cells.
The cohort includes patients treated in CNH and multiple institutions around the world, including Texas Children’s and the National Institutes of Health. “This work was only possible through our collaborative research in anti-EBV cellular therapies,” said Dr. Dávila.
https://innovationdistrict.childrensnational.org/wp-content/uploads/2021/11/Epstein-Barr-virus-illustration.png300400Innovation Districthttps://innovationdistrict.childrensnational.org/wp-content/uploads/2023/12/innovationdistrict_logo-1-1030x165.pngInnovation District2021-11-23 13:40:242024-11-27 09:40:12Study with largest cohort in the Western world sheds light on Epstein-Barr virus
Children’s National Hospital researchers Kristen Sgambat, Ph.D., and Asha Moudgil, M.D., were presented with the 2021 AJKD Editors’ Choice Award.
The American Journal of Kidney Disease (AJKD) announced the selection of the 2021 AJKD Editors’ Choice Award, recognizing outstanding articles published in their journal this year.
Children’s National Hospital researchers Kristen Sgambat, Ph.D., and Asha Moudgil, M.D., were presented with the 2021 AJKD Editors’ Choice Award for their July 2021 study, Social determinants of cardiovascular health in African American children with chronic kidney disease: An analysis of the chronic kidney disease in children (CKiD).
The study is the first to investigate the relationship between race, socioeconomic factors and cardiovascular health in children with chronic kidney disease. Dr. Sgambat, Dr. Moudgil and their collaborators found that African American children with chronic kidney disease had increased evidence of socioeconomic challenges, including food insecurity, reliance on public insurance, lower household incomes and lower levels of maternal education. These children had worse cardiovascular outcomes than Caucasian children with the same chronic kidney conditions. Notably, the cardiovascular outcomes of the two groups became more alike when statistical analysis was applied to equalize their socioeconomic factors. This suggests that these socioeconomic indicators do play a role in adverse cardiovascular health outcomes observed among African American children with chronic kidney disease.
“The findings of this study are important because they highlight the urgent need to shift the clinical research paradigm to investigate how social, rather than biological, factors contribute to racial differences in health outcomes,” said Dr. Sgambat. “Future studies should focus on the impact of systemic racism on cardiovascular health among children with chronic kidney disease, an area not well-studied so far.”
https://innovationdistrict.childrensnational.org/wp-content/uploads/2021/11/Drs.-Sgambat-and-Moudgil.png300400Innovation Districthttps://innovationdistrict.childrensnational.org/wp-content/uploads/2023/12/innovationdistrict_logo-1-1030x165.pngInnovation District2021-11-22 10:49:302024-11-08 13:32:30Kristen Sgambat, Ph.D., R.D. and Asha Moudgil, M.D. receive Editors’ Choice Award
A retrospective study found pediatric Type 1 diabetes cases rose 15.2% and Type 2 diabetes cases increased by 182% during the first year of the COVID-19 pandemic compared to the prior two years— affecting non-Hispanic Black youth the most.
While the effects of COVID-19 on diabetes-related outcomes are extensively studied in adults, data about the incidence and severity of presentation of pediatric new-onset Type 1 diabetes (T1D) and Type 2 diabetes (T2D) is limited. A new retrospective study of 737 youth diagnosed with diabetes at Children’s National Hospital between March 11, 2018 and March 10, 2021 found pediatric T1D cases rose 15.2% and T2D cases increased by 182% during the first year of the COVID-19 pandemic compared to the prior two years — affecting non-Hispanic Black youth the most.
The study, published in Hormone Research in Paediatrics, compared T1D and T2D cases during the first 12 months of the pandemic, between March 11, 2020 and March 10, 2021, to the same time in the previous two years. This increase in cases was accompanied by a nearly six-fold rise in diabetic ketoacidosis (DKA) and a 9.2% incidence of hyperosmolar DKA during the pandemic as compared to no cases in the two years prior.
“A better understanding of the impact of the COVID-19 pandemic is crucial for raising public awareness, shaping policy and guiding appropriate health screenings,” said Brynn Marks, M.D., M.S.H.P.Ed., endocrinologist at Children’s National and lead author of the study.
Children’s National provides clinical care to approximately 1,800 youth with T1D and 600 youth with T2D annually. In the two years before the pandemic, cases of T2D accounted for 25.1% of all newly diagnosed diabetes at Children’s National compared to 43.7% during the pandemic. Before the pandemic, females accounted for 59.6% of youth with new-onset T2D but 58.9% of new-onset T2D cases were among males during the pandemic.
The researchers noted that the rise in cases of T2D and severity of presentation of both T1D and T2D during the pandemic disproportionately impacted non-Hispanic Black youth (NHB). NHB youth accounted for 58% of cases of T2D pre-pandemic, which further increased to 77% during the pandemic. The findings also showed that cases of DKA among NHB youth newly diagnosed with T1D increased during the pandemic compared to the two years before (62.7% vs. 45.8%, p=0.02). Before the pandemic, there was no significant difference in A1c at T1D diagnosis between racial and ethnic groups. However, during the pandemic, hemoglobin A1c levels were higher among NHB youth.
“Future studies are needed to understand the root cause of the disproportionate impact of the COVID-19 pandemic on non-Hispanic Black youth with newly diagnosed diabetes,” said Dr. Marks. “These outcomes during the pandemic will likely worsen pre-existing health care disparities among youth with diabetes. In understanding the indirect effects of our response to the pandemic, we can better inform future emergency responses and develop strategies to improve outcomes for all youth living with diabetes.”
https://innovationdistrict.childrensnational.org/wp-content/uploads/2021/11/doctor-taking-blood-sample-from-child.png300400Innovation Districthttps://innovationdistrict.childrensnational.org/wp-content/uploads/2023/12/innovationdistrict_logo-1-1030x165.pngInnovation District2021-11-19 11:18:482022-07-15 09:33:03Study shows increase in diabetes cases during COVID-19 pandemic
Study suggests permanent injury to the cholinergic system after neonatal hypoxia-ischemia is responsible for the poor executive functions and difficulties in learning and memory.
Newborn babies who go through periods of low oxygen — also known as hypoxic-ischemic encephalopathy — during their first hours of life often experience difficulties in learning, memory and executive functions later on. Even when treated with therapeutic hypothermia, memory deficits and executive functions remain severely affected. These functions are linked to a neurotransmitter network called the cholinergic system.
“Complications from hypoxic-ischemic brain injury contribute to one-quarter of neonatal deaths worldwide and cause significant long-term neurological morbidity,” explains Panagiotis Kratimenos, M.D., Ph.D., neonatologist at Children’s National Hospital and Assistant Professor of Pediatrics at the George Washington University School of Medicine and Health Sciences.
In a study published in the Journal of Comparative Neurology led by Frances Northington, M.D., co-director of Neurosciences Intensive Care Nursery at Johns Hopkins and Professor of Pediatrics at Johns Hopkins University School of Medicine, with contributions from Dr. Kratimenos, the authors found significant injury to the neurons of the cholinergic systems in specific parts of the brain after exposure to low oxygen and restricted blood flow. These areas included the ipsilateral medial septal nucleus (MSN), the ipsilateral nucleus basalis of Meynert (nbM) and striatum. Within the injured part of the cortex at the site of injury, acetylcholine — the neurotransmitter found in cholinergic systems — was abnormally overactivated.
The authors hypothesize that permanent injury to the cholinergic system after neonatal hypoxia-ischemia is responsible for the poor executive functions and difficulties in learning and memory.
“Because cholinergic systems can easily be manipulated pharmacologically with already established treatments that have been used in other areas of medicine, they could be a good a target for therapeutic interventions for neonates with hypoxic-ischemic encephalopathy,” says Dr. Kratimenos.
https://innovationdistrict.childrensnational.org/wp-content/uploads/2021/04/neurons.png300400Innovation Districthttps://innovationdistrict.childrensnational.org/wp-content/uploads/2023/12/innovationdistrict_logo-1-1030x165.pngInnovation District2021-11-18 11:36:502021-11-18 12:06:57Neonatal hypoxia-ischemia causes damage to the cholinergic system
Children’s National Hospital experts developed a new approach that discovered unique proteins in an individual tumor’s cells, which then helped scientists generate personalized T cells to target and kill tumors.
Children’s National Hospital experts developed a new approach that discovered unique proteins in an individual tumor’s cells, which then helped scientists generate personalized T cells to target and kill tumors, according to a pre-clinical study published in Nature Communications.
This effort is the first to create a new workflow for neoantigen identification that incorporates both genetic sequencing and protein identification to create a personalized treatment for medulloblastoma in children, a common malignant brain tumor. Given these promising findings, the researchers are now designing a phase I clinical trial slated to open in 12-18 months.
“This work is an incredibly exciting advancement in personalized medicine. It will allow us to treat patients with a novel T cell therapy that is developed for each individual patient to specifically attack and kill their tumor,” said Catherine Bollard, M.D., M.B.Ch.B., director of the Center for Cancer and Immunology Research at Children’s National and co-author on the paper. “This treatment will offer a potential option for children with hard-to-treat brain tumors for which all other therapeutic options have been exhausted.”
Catherine Bollard, M.D., M.B.Ch.B., director of the Center for Cancer and Immunology Research at Children’s National and co-senior author on the paper.
First, the researchers sequenced the DNA of small tissue samples while studying its complete set of proteins that influence cancer biology — also named a “low-input proteogenomic approach” by the authors. After analyzing the empirical data, which shies away from the commonly used predictive models, the researchers developed a T cell immunotherapy that targets the tumor’s unique proteins and allows the T cells to distinguish between healthy cells and tumor cells. This means that Rivero-Hinojosa et al. managed to merge two research fields, proteogenomics and immunotherapy, and lay the groundwork for personalized, targeted T cell therapies to treat children with brain tumors.
“Neoantigen discovery techniques have either been dependent upon in silico prediction algorithms or have required a significant amount of tumor tissue, making them inappropriate for most brain tumors,” said Brian Rood, M.D., medical director of Neuro-oncology and the Brain Tumor Institute at Children’s National. “This neoantigen identification pipeline creates a new opportunity to expand the repertoire of T cell-based immunotherapies.”
Tumor cells have damaged DNA that create mutations during the repair process because they do not do a good job at maintaining their DNA fidelity. The repairs therefore create aberrant DNA that codes for proteins that were never intended by the genetic code and, consequently, they are unique to the individual’s tumor cells.
Brian Rood, M.D., medical director of Neuro-oncology and the Brain Tumor Institute at Children’s National and co-senior author on the paper.
“We developed a new filtering pipeline to remove non-annotated normal peptides. Targeting antigens that are completely specific to the tumor, and expressed nowhere else in the body, will potentially increase the strength of tumor antigen-specific T cell products while decreasing the toxicity,” said Samuel Rivero-Hinojosa, Ph.D., staff scientist at Children’s National and first author of the study.
Once the experts identified these unique peptides, they used them to select and expand T cells, which showed specificity for the tumor specific neoantigens and the ability to kill tumor cells. The next step is to conduct a clinical trial in which a patient’s own T cells are trained to recognize their tumor’s unique neoantigens and then reinfused back into the patient.
From an immunotherapy standpoint, tumor specificity is important because when clinicians treat patients with T cell therapies, they want to make sure that the T cells directly target and kill the tumor and will not cause devastating harm to healthy cells. This paper demonstrated that it may be possible to create a better efficacy and safety margin with this new approach.
In the past five years, under the leadership of Dr. Bollard, the Center for Cancer and Immunology Research at Children’s National has advanced the scientific knowledge in preclinical and clinical settings. The center discovered a signaling pathway that can be hijacked to prevent brain tumor development, and further advanced translational research with several key first-in-human studies that utilized novel cell therapies to treat cancer and life-threatening viral infections.
https://innovationdistrict.childrensnational.org/wp-content/uploads/2021/11/t-cells-fighting-cancer-cell.png300400Innovation Districthttps://innovationdistrict.childrensnational.org/wp-content/uploads/2023/12/innovationdistrict_logo-1-1030x165.pngInnovation District2021-11-18 05:00:232021-12-22 15:01:27Personalized T cell immunotherapy for brain tumors closer to becoming reality
Anti-Mullerian hormone (AMH), also known as Mullerian inhibiting substance (MIS), is a hormone produced exclusively in the gonads. It was originally described in the context of sexual differentiation in the male fetus but has gained prominence now as a marker of ovarian reserve and fertility in females.
In a mini-review published in The Journal of Clinical Endocrinology and Metabolism, Roopa Kanakatti Shankar, M.D., pediatric endocrinologist at Children’s National Hospital and an associate professor of pediatrics at the George Washington University School of Medicine and Health Sciences, and co-authors offer an updated synopsis on AMH and its clinical utility in pediatric patients.
The authors performed a systematic search for studies related to the physiology of AMH, normative data and clinical role in pediatrics. After reviewing 70 clinical studies and systematic reviews, they conclude that, “AMH has widespread clinical diagnostic utility in pediatrics but interpretation is often challenging and should be undertaken in the context of not only age and sex but also developmental and pubertal stage of the child.”
https://innovationdistrict.childrensnational.org/wp-content/uploads/2021/11/Blood-sample-tube-for-anti-Mullerian-hormone.png300400Innovation Districthttps://innovationdistrict.childrensnational.org/wp-content/uploads/2023/12/innovationdistrict_logo-1-1030x165.pngInnovation District2021-11-16 11:42:412021-11-16 12:39:59A look at the clinical utility of anti-Mullerian hormone
“We know from previous studies that though it is not always well-documented, sub-Saharan Africa continues to have some of the highest numbers of people with rheumatic heart disease and the highest numbers of people dying from it,” said Craig Sable, M.D., associate chief of Cardiology at Children’s National Hospital and co-senior author of the study. “This study is the first large-scale clinical trial to show that early detection coupled with prophylactic treatment of penicillin is feasible and can prevent rheumatic heart disease from progressing and causing further damage to a child’s heart.”
Penicillin, a widely available and affordable antibiotic, may be one key to turning the tide on the deadly impacts of rheumatic heart disease (RHD) for children in developing nations. This according to the new findings of a large-scale, randomized controlled trial completed in Uganda and published in the New England Journal of Medicine.
The most devastating feature of RHD is severe heart valve damage that is caused by rheumatic fever — a condition that results from the body’s immune system trying to fight poorly treated, repeat infections from streptococcus bacteria, also known as strep throat. Though widely eradicated in nations such as the United States due to the swift detection and treatment of strep throat, rheumatic fever remains prevalent in developing countries including those in sub-Saharan Africa. Current estimates are that 40.5 million people worldwide live with rheumatic heart disease, and that it kills 306,000 people every year. Most of those affected are children, adolescents and young adults under age 25.
“We know from previous studies that though it is not always well-documented, sub-Saharan Africa continues to have some of the highest numbers of people with rheumatic heart disease and the highest numbers of people dying from it,” said Craig Sable, M.D., associate chief of Cardiology at Children’s National Hospital and co-senior author of the study. “This study is the first large-scale clinical trial to show that early detection coupled with prophylactic treatment of penicillin is feasible and can prevent rheumatic heart disease from progressing and causing further damage to a child’s heart.”
The study was led by an international panel of pediatric cardiac experts from institutions including Children’s National, Cincinnati Children’s Medical Center, the Uganda Heart Institute and Murdoch Children’s Research Institute in Melbourne, Australia.
“Our study found a cheap and easily available penicillin can prevent progression of latent rheumatic heart disease into more severe, irreversible valve damage that is commonly seen in our hospitals with little or no access to valve surgery,” said co-lead author Emmy Okello, M.D., chief of Cardiology at the Uganda Heart Institute.
To Andrea Beaton, M.D., associate professor of Cardiology at Cincinnati Children’s and co-lead author, this is the first contemporary randomized controlled trial in rheumatic heart disease. “The results are incredibly important on their own, but also demonstrate that high-quality clinical trials are feasible to address this neglected cardiovascular disease,” she said.
Beaton et al. named the trial Gwoko Adunu pa Lutino (GOAL), which means “protect the heart of a child.” The study enrolled 818 Ugandan children and adolescents ages 5 to 17 years old who were diagnosed with latent rheumatic heart disease to see if an injection of penicillin was effective at preventing their heart condition from worsening.
“There are many challenges with recruitment and retention of trial participants in areas like our study region in Uganda,” said Dr. Sable. “But it is critical to work together and overcome barriers, because we must study these treatments in the people most affected by the condition to understand how they, and others like them, may benefit from the findings.”
Of the 799 participants who completed the trial, the group receiving a prophylactic injection of penicillin (399 volunteers) had three participants show evidence of worsened rheumatic heart disease on repeat echocardiogram after two years. In contrast, 33 of the 400 volunteers in the control group, who received no treatment, showed similar progression on echocardiogram results.
Professor Andrew Steer, who is theme director of Infection and Immunity at Murdoch Children’s Research Institute in Melbourne and who served as senior author of the study, said screening for latent rheumatic heart disease was critical to stop progression because heart valve damage was largely untreatable. “Most patients are diagnosed when the disease is advanced and complications have already developed. If patients can be identified early, there is an opportunity for intervention and improved health outcomes.”
The results were shared in a special presentation at the American Heart Association’s Scientific Sessions on the same day that the findings were published in the New England Journal of Medicine.
The trial was supported by the Thrasher Pediatric Research Fund, Gift of Life International, Children’s National Hospital Foundation: Zachary Blumenfeld Fund, Children’s National Hospital Race for Every Child: Team Jocelyn, the Elias/Ginsburg Family, Wiley-Rein LLP, Phillips Foundation, AT&T Foundation, Heart Healers International, the Karp Family Foundation, Huron Philanthropies and the Cincinnati Children’s Hospital Heart Institute Research Core.
Learn more about the challenges of rheumatic heart disease in sub-Saharan Africa and other developing parts of the world through the Rheumatic Heart Disease microdocumentary series:
https://innovationdistrict.childrensnational.org/wp-content/uploads/2021/11/Craig-Echoing-in-village.png300400Innovation Districthttps://innovationdistrict.childrensnational.org/wp-content/uploads/2023/12/innovationdistrict_logo-1-1030x165.pngInnovation District2021-11-15 09:48:352025-05-06 13:50:49Penicillin slows impacts of rheumatic heart disease in Ugandan children
When comparing the first and second wave of patients diagnosed with multi-system inflammatory syndrome in children (MIS-C), the second wave patients had more severe illness, according to a new prospective cohort study at Children’s National Hospital in Washington, D.C.
When comparing the first and second wave of patients diagnosed with multi-system inflammatory syndrome in children (MIS-C), the second wave patients had more severe illness, according to a new prospective cohort study of 106 patients at Children’s National Hospital in Washington, D.C. The results, published in The Pediatric Infectious Disease Journal, show that despite increased severity in the second wave cohort, both cohorts had similarities in cardiac outcomes and length of stay. Researchers are still working to better understand the exact immunologic mechanisms that trigger MIS-C and the specific factors accounting for its rare occurrence.
“We’ve now seen three distinct waves of MIS-C since the beginning of the pandemic, each wave following national spikes in cases,” said Roberta DeBiasi, M.D., chief of the Division of Pediatric Infectious Diseases at Children’s National and co-author of the study. “Kids in the second wave cohort had potentially experienced intermittent and/or repeated exposures to the virus circulating in their communities. In turn, this may have served as repeated triggers for their immune system which created the more severe inflammatory response.”
In this new study, key demographic features Children’s National researchers previously identified held true across both waves – including the fact that Black and Latino children are significantly more affected than white children. Of the 106 patients, 54% were Black and 39% were Hispanic. The authors also noted that 75% of the patients were otherwise healthy children with no underlying medical conditions.
“While we believe the most recent third wave associated with the delta variant surge is tapering off, the findings from the first two waves provide important baseline information and are highly relevant for clinicians across the country that are evaluating and treating kids with MIS-C,” said Dr. DeBiasi.
Children’s National has cared for more than 4,200 symptomatic patients with SAR-CoV-2 infection and more than 185 MIS-C patients since the pandemic began. The first wave of MIS-C patients were hospitalized between March 2020 and October 2020. Second wave patients were hospitalized between November 2020 and April 2021. Each wave came 4-6 weeks following periods of COVID-19 surges in the community.
In the study, researchers compared patient demographics, clinical features, laboratory results, radiographic images, therapies and outcomes. The second wave cohort had a higher proportion of children 15 years of age or older. Patients also presented more frequently with shortness of breath and required more advanced respiratory and inotropic support. Researchers also found that patients in the second wave were less likely to test positive for SARS-CoV-2 on a PCR test.
Dr. DeBiasi and her team hope to unlock even more insights as they now analyze data from the third wave associated with the delta variant, which currently appears to have affected less children than the previous two. Children’s National is also working in collaboration with the National Institute of Allergy and Infectious Diseases (NIAID) to study the long-term effects of MIS-C and COVID-19 on the pediatric population after recovery. This is among the largest and longest studies being conducted, and researchers are hopeful the findings will help improve treatment of COVID-19 and MIS-C in the pediatric population both nationally and around the world.
“Our timely established multidisciplinary MIS-C task force here at Children’s National allowed us to reduce the learning curve,” said Ashraf S. Harahsheh, M.D., F.A.A.P., F.A.C.C., director of Quality Outcomes in Cardiology and co-first author of the study. “Experience from other centers showed that immunotherapy was utilized more frequently in recent MIS-C cohorts leading to reduction in percentage of cardiac complications. On the other hand, and despite having increased illness severity in the second cohort, our approach with prompt immunotherapy helped stabilize the rate of cardiac complications.”
https://innovationdistrict.childrensnational.org/wp-content/uploads/2020/10/sick-child-in-palliative-care-hospital-bed.png300400Innovation Districthttps://innovationdistrict.childrensnational.org/wp-content/uploads/2023/12/innovationdistrict_logo-1-1030x165.pngInnovation District2021-11-12 13:30:132021-11-12 13:30:13New study compares first and second wave of MIS-C
RDI, which includes the largest clinical group of pediatric geneticists in the nation, focuses on developing the clinical care field of more than 8,000 rare diseases currently recognized and advancing the best possible treatments for children with these diseases.
The Rare Disease Institute (RDI) at Children’s National Hospital announced its designation as a NORD Rare Disease Center of Excellence, joining a highly select group of 31 medical centers nationwide. This new, innovative network seeks to expand access and advance care and research for rare disease patients in the United States. The program is being led by the National Organization for Rare Disorders (NORD), with a goal to foster knowledge sharing between experts across the country, connect patients to appropriate specialists regardless of disease or geography, and to improve the pace of progress in rare disease diagnosis, treatment and research.
“Children’s National has worked closely with NORD to move this program forward and is very proud to be amongst the first group of recognized centers,” said Marshall Summar, M.D., chief of the Division of Genetics and Metabolism and the director of RDI at Children’s National. “This is a recognition of the institutional efforts, as we take care of patients with the rare disease and help set the standard for the field.”
RDI, which includes the largest clinical group of pediatric geneticists in the nation, focuses on developing the clinical care field of more than 8,000 rare diseases currently recognized and advancing the best possible treatments for children with these diseases.
In February 2021, RDI became the first occupant of the new Children’s National Research & Innovation Campus, a first-of-its-kind pediatric research and innovation hub. The campus now also houses the Center for Genetic Medicine Research, and together researchers are constantly pursuing high-impact opportunities in pediatric genomic and precision medicine. Both centers combine its strengths with public and private partners, including industry, universities, federal agencies, start-up companies and academic medical centers. They also serve as an international referral site for rare disorders.
People living with rare diseases frequently face many challenges in finding a diagnosis and quality clinical care. In establishing the Centers of Excellence program, NORD has designated clinical centers across the U.S. that provide exceptional rare disease care and have demonstrated a deep commitment to serving rare disease patients and their families using a holistic, state of the art approach.
“Right now, far too many rare diseases are without an established standard of care. The Centers of Excellence program will help set that standard – for patients, clinicians, and medical centers alike,” said Ed Neilan, chief scientific and medical officer of NORD. “We are proud to announce Children’s National as a NORD Rare Disease Center of Excellence and look forward to their many further contributions as we collectively seek to improve health equity, care and research to support all individuals with rare diseases.”
Each center was selected by NORD in a competitive application process requiring evidence of staffing with experts across multiple specialties to meet the needs of rare disease patients and significant contributions to rare disease patient education, physician training and research.
https://innovationdistrict.childrensnational.org/wp-content/uploads/2021/11/RDI-sign.png300400Innovation Districthttps://innovationdistrict.childrensnational.org/wp-content/uploads/2023/12/innovationdistrict_logo-1-1030x165.pngInnovation District2021-11-04 09:00:262024-09-06 15:14:45Children’s National Rare Disease Institute named a Center of Excellence
Supported by the Gilbert Family Foundation, Dr. Nazarian’s return is part of a special research program within the Gilbert Family Neurofibromatosis Institute that focuses on NF1 research.
Javad Nazarian, Ph.D., M.Sc., associate professor of Pediatrics at George Washington University and professor at the University of Zurich, has expanded his research group at Children’s National to focus on Neurofibromatosis type 1 (NF1) transformed low-grade gliomas (LGGs). Dr. Nazarian will apply his expertise from establishing a successful DIPG (diffuse intrinsic pontine glioma) and DMG (diffuse midline glioma) program in Zurich Switzerland and previously at Children’s National.
In addition to his continued research in Zurich, as a principal investigator at the Department of Genomics and Precision Medicine at Children’s National Dr. Nazarian plans on aggregating his knowledge to the new research and work spearheaded at Children’s National. As one of the first research teams to move to the Children’s National Research & Innovation Campus, Dr. Nazarian’s group is excited to use the opportunity to establish cutting-edge and clinically translational platforms.
Supported by the Gilbert Family Foundation, Dr. Nazarian’s return is part of a special research program within the Gilbert Family Neurofibromatosis Institute that focuses on NF1 research. This research includes associated gliomas with a special emphasis on NF1-associated transformed anaplastic LGGs. His team will develop new avenues of research into childhood and young adult NF-associated LGGs with a special emphasis on transformed high-grade gliomas.
Dr. Nazarian is excited for what’s to come and his goals are clear and set. Here, Dr. Nazarian tells us more about his main objectives and what it means for the future of pediatric neuro-oncology care at Children’s National.
What excites you most about being back at Children’s National?
I have received most of my training at Children’s National, so this is home for me. Being one of the nation’s top children’s hospitals gives a unique advantage and ability to advocate for childhood diseases and cancers. It is always exciting to play a part in the vision of Children’s National.
What are some of the lessons learned during your time working in Zurich? And how do you think these will compliment your work at Children’s National?
We developed a focused group with basic research activities intertwined with clinical needs. The result was the launch of two clinical trials. I also helped in developing the Diffuse Midline Glioma-Adaptive Combinatory Trial (DMG-ACT) working group that spans across the world with over 18-member institutions that will help to design the next generation clinical trials. I will continue leading the research component of these efforts, which will have a positive impact on our research activities at Children’s National.
How does your work focusing on low-grade gliomas formulating into high-grade gliomas expand and place Children’s National as a leader in the field?
Scientifically speaking, transformed LLGs are very intriguing. I became interested in the field because these tumors share molecular signatures similar to high-grade gliomas (HGGs). Our team has done a great job at Children’s National to develop tools – including biorepositories, avatar models, drug screening platforms, focused working groups, etc. – for HGGs. We will apply the same model to transformed LGGs with the goal of developing biology-derived clinical therapeutics for this patient population.
How will this work support families and patients seeking specific neuro-oncology care?
We will develop new and high thruput tools so that we can better study cancer formation or transformation. These tools and platforms will allow us to screen candidate drugs that will be clinically effective. The main focus is to accelerate discovery, push drugs to the clinic, feed information back to the lab from clinical and subsequently design better therapies.
You are one of the first scientists to move to the Children’s National Research & Innovation Campus. What are some of the valuable changes or advancements you hope to see as a result of the move?
The campus will provide high-end facilities, including cutting-edge preclinical space, and allow for team expansion. The close proximity to Virginia Tech will also provide an environment for cross-discipline interactions.
Anything else you think peers in your field should know about you, the field or our program?
The team at Children’s National includes Drs. Roger Packer and Miriam Bornhorst. Both have provided constant clinical support, innovation and clinical translation of our findings. I look forward to working with them.
https://innovationdistrict.childrensnational.org/wp-content/uploads/2021/10/Dr.-Nazarian.gif300400Innovation Districthttps://innovationdistrict.childrensnational.org/wp-content/uploads/2023/12/innovationdistrict_logo-1-1030x165.pngInnovation District2021-10-28 10:19:102024-09-06 15:12:09Q&A with Dr. Javad Nazarian on his upcoming work on low-grade gliomas
Understanding factors that foster resilience and buffer against the negative psychological impact of COVID-19 is critical to inform efforts to promote adjustment, reduce risk and improve care, particularly for adolescents with neurodevelopmental disorders.
In a new prospective longitudinal study, Melissa Dvorsky, Ph.D., director of the ADHD & Learning Differences Program at Children’s National Hospital and lead author, and others address this gap by investigating the impact of the COVID-19 pandemic on adolescents’ mental health and substance use, and by assessing specific positive coping strategies among adolescents with and without attention-deficit/hyperactivity disorder (ADHD).
https://innovationdistrict.childrensnational.org/wp-content/uploads/2017/10/Group-of-teens.jpg300400Innovation Districthttps://innovationdistrict.childrensnational.org/wp-content/uploads/2023/12/innovationdistrict_logo-1-1030x165.pngInnovation District2021-10-27 08:59:422021-10-27 09:02:30Assessing the pandemic’s impact on adolescents with and without ADHD
Cardiopulmonary bypass, more commonly known as heart-and-lung bypass, has some unique impacts on the creation and growth of brain cells in the area of a child’s brain called the subventricular zone (SVZ), according to a study in the Annals of Neurology. The SVZ is a critical area for the growth and migration of neurons and nerve cells called neuroblasts, both of which ultimately contribute to the proper development of key brain structures and functions during the early years of life.
The findings, from a study conducted in the Cardiac Surgery Research Laboratory at Children’s National Hospital, provide new insight into the cellular impacts of the cardiopulmonary bypass machine on brain growth and development for newborn infants with congenital heart disease. They will have an important role in the refinement of strategies to help protect the fragile brains of children who require lifesaving cardiac surgery with cardiopulmonary bypass immediately after birth.
Specifically, the research team found that during cardiopulmonary bypass:
Creation of neurons (neurogenesis) in the neonatal and infant subventricular zone is altered.
Migration of nerve cells, called neuroblasts, to the frontal lobe is potentially disrupted.
Changes to the growth and movement of neurons in the SVZ are prolonged.
Cortical development and expansion is impaired.
Specific types of neurons found only in the brain and spinal cord, called interneurons, are also affected.
The study uses an innovative pre-clinical model of the developing brain that is more anatomically and physiologically similar to human neonates and infants than those used in prior studies and in most neurological laboratory-based research.
Cardiopulmonary bypass is one of several key factors thought to cause children with congenital heart disease to sometimes demonstrate delays in the development of cognitive and motor skills. These disabilities often persist into adolescence and adulthood and can ultimately represent long-term neurocognitive disabilities. It is also believed that genetic factors, abnormal blood flow to the brain while in utero or low cardiac output after surgical procedures on the heart may contribute to these challenges.
“Unraveling cellular and molecular events during surgery using this preclinical model will allow us to design therapeutic approaches that can be restorative or reparative to the neurogenic potential of the neuronal stem precursor cells found in the subventricular zone of the neonatal or infant brain,” says Nobuyuki Ishibashi. M.D., Foglia-Hills Professor of Pediatric Cardiac Research, director of the Cardiac Surgery Research Laboratory at Children’s National and senior author on the study. “In particular, previous studies in our laboratory have shown improvement in the neurogenic activities of these precursor cells when they are treated with mesenchymal stromal cells (MSCs).”
The findings from this study further support the work already underway in the NIH-funded MeDCaP clinical trial for neonates and infants undergoing cardiac surgery using the cardiopulmonary bypass machine. That trial uses the heart and lung machine itself to deliver MSCs directly into the main arteries that carry blood to the brain.
https://innovationdistrict.childrensnational.org/wp-content/uploads/2019/12/brain-network-illustration.png300400Innovation Districthttps://innovationdistrict.childrensnational.org/wp-content/uploads/2023/12/innovationdistrict_logo-1-1030x165.pngInnovation District2021-10-26 09:45:532021-10-29 10:30:44Cardiopulmonary bypass may cause significant changes to developing brain and nerve cells
Denver D. Brown, M.D., recipient of the Child Health Research Career Development Award.
Linear growth (i.e., height) impairment is commonly observed in children with chronic kidney disease (CKD). Several studies have suggested metabolic acidosis, a frequent consequence of mild to moderate CKD in children, as a contributing factor to linear growth failure in these patients. Grant awardee Denver D. Brown, M.D., aims to conduct a pilot trial in children with mild metabolic acidosis and CKD, comparing differences in linear growth between an observation period versus a period of supplementation with alkali therapy (i.e., treatment for metabolic acidosis).
“This grant is so important because there has never been a clinical trial of alkali therapy in children with CKD despite its frequent use in this population” says Dr. Brown. “This research has the potential to better inform treatment practices with the aim of improving the care of our young, vulnerable patients.”
The Child Health Research Career Development Award (CHRCDA) of $125,000 will support Dr. Brown in her efforts to carry out this pilot trial.
“Funding for this pilot study could lay the groundwork for a large, randomized controlled clinical trial, which would help fill a major gap in knowledge as to the precise benefits of alkali therapy, especially regarding growth in children with impaired kidney function.”
https://innovationdistrict.childrensnational.org/wp-content/uploads/2021/10/Denver-Brown.png300400Innovation Districthttps://innovationdistrict.childrensnational.org/wp-content/uploads/2023/12/innovationdistrict_logo-1-1030x165.pngInnovation District2021-10-25 15:42:112021-11-01 11:53:15New grant to conduct single center pilot trial of alkali therapy in children with CKD
Maria Susana Rueda Altez, M.D., junior section president-elect for the Society for Pediatric Research (SPR).
Maria Susana Rueda Altez, M.D., was selected as junior section president-elect for the Society for Pediatric Research (SPR). During her tenure, Dr. Rueda Altez will ensure more trainees benefit from networking opportunities and leverage her online communications experience to increase awareness, membership and participation in SPR among students, residents and fellows.
The president of the junior section is a fellow who is elected by other junior member peers and is in-charge of managing and enhancing the junior section, by participating in SPR council meetings, promoting membership among trainees and reinforcing the pipeline from junior to active members.
“I am so honored, not only as a Peruvian physician, but as an international medical graduate (IMG), to have been elected for this position,” said Dr. Rueda Altez. “As an IMG, there are special challenges to conducting research, so I plan to raise awareness and provide support to my fellow IMG junior members.”
To Beth A. Tarini, M.D., M.S., SPR president and associate director for the Center for Translational Research at Children’s National Hospital, it is an honor for the hospital to have representatives in the roles of SPR president and SPR junior section president-elect simultaneously.
Dr. Rueda Altez added that there is an urgent need for increased funding in pediatric research, especially for minority and health disparities research. Through her participation in SPR, she will also have the opportunity to advocate for increases in child health research funding.
“I encourage all the trainees and junior faculty in our institution to join the SPR junior section,” said Dr. Rueda Altez. “It provides wonderful resources for career development and guidance, grant writing courses and invaluable mentorship.”
Her research interest is newborn infections, and her overall goal is to reduce the unnecessary use of antibiotics in this population.
“I am currently working on a quality improvement project to reduce the number of days NICU infants are exposed to antibiotics,” said Dr. Rueda Altez. “I have developed a project to ascertain the utility of microbial cell-free DNA next generation sequencing, a novel microbiologic diagnostic tool, for the diagnosis of neonatal infections.”
Dr. Rueda Altez’s work on neonatal sepsis will help scientists better distinguish between neonates who do and don’t have serious bacterial infections.
“Right now, when in doubt we tend to treat it as bacterial infections, which can lead to unnecessary medical treatment and worsen resistance to antibiotics,” said Tarini.
Dr. Rueda Altez also serves as an independent reviewer of investigational manuscripts for The Journal of Pediatrics and Pediatrics and guest editor for The Journal of Pediatrics. Her passion for the peer-review process also shows in her long list of published research.
Dr. Tarini also foresees multiple research trends in the next five years that might appear in peer-reviewed publications.
“We have so much to tackle in child health research, both ongoing and new challenges,” said Dr. Tarini. “Some issues that come to mind are the mental health crisis in children and teens, continuing to make strides on treating and preventing childhood obesity, the effect of poverty on children’s health, and the pandemic’s effect on all of these issues and its direct effect on health outcomes.”
https://innovationdistrict.childrensnational.org/wp-content/uploads/2021/10/Maria-Susana-Rueda-Altez.png300400Innovation Districthttps://innovationdistrict.childrensnational.org/wp-content/uploads/2023/12/innovationdistrict_logo-1-1030x165.pngInnovation District2021-10-20 10:47:482021-10-20 10:47:48Maria Susana Rueda Altez, M.D., to lead as Junior Section President-Elect
