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When the FDA approved the drug mepolizumab on November 4th to help treat severe asthma, it was a blip on the radar screen of health industry news. The medication targets a subset of asthma patients (ages 12 and up) whose current drug regimens are insufficient to control their condition.
Absent from official press announcements about mepolizumab’s approval on Nov. 4 are the many years of research and testing by countless physicians and scientists at institutions around the world. Cincinnati Children’s and its Division of Allergy and Immunology were key parts to this effort.
Doctors may soon have a new tool to help them time births in the case of delicate, pre-mature babies. A study led by researchers at Cincinnati Children’s Perinatal Institute have identified a way to test RNA and specific genetic signatures in amniotic fluid to see whether fetal lungs – and potentially other organs – are mature enough for a safe and viable delivery. “This will allow pediatricians and neonatologists to prepare for the various neonatal morbidities these preterm infants may face, and allow obstetricians to better weigh risks to the baby when making decisions about preterm delivery,” says Beena Kamath-Rayne, MD, MPH, one of the study’s lead authors. The findings are published in BMC Medical Genomics.
A new computer program developed at Cincinnati Children’s analyzes functional brain MRIs to predict whether hearing impaired children will develop effective language skills within two years of cochlear implantation surgery. “This study identifies two features from our computer analysis that are potential biomarkers for predicting cochlear implant outcomes,” says collaborator Long (Jason) Lu, PhD from the Division of Biomedical Informatics. The findings are published in the Oct. 12 online edition of Brain and Behavior.
A new study presented at a national meeting on October 4, 2015 reveals that patients with congenital heart disease and ADHD can safely benefit from stimulant medications. “Children with congenital heart disease are at high risk for ADHD, but fears about cardiovascular side effects, including sudden death, limit the use of stimulant medications,” says Julia Anixt, MD, senior author of the study. “This study indicates that stimulants are both effective and safe when prescribed with appropriate monitoring and in collaboration with the patient’s cardiologist.”
A study led by researchers in the Division of Allergy and Immunology. reports findings that offer insight into new therapeutic strategies and diagnostics for severe food allergies triggered by immunoglobin E (IgE). The study, published in the journal Immunity, reveals the discovery of IL-9-producing mucosal mast (MM9) cells that produce large amounts of an inflammatory protein, interlukin 9 (IL-9), known to amplify anaphylactic shock in response to certain foods. “Our study suggests that although you need to have some level of IgE to trigger a food allergy response, you also have to produce MMC9 cells to get a severe response and anaphylaxis,” says Yui-Hsi Wang, PhD, the study’s co-first author. “Without these cells you will not get severe food allergies.”
We are excited to announce the launch of a new, innovative, Master’s Program at Cincinnati Children’s Hospital Medical Center, in conjunction with the University of Cincinnati. The Biomedical Research Master’s Program is born from the recognition that there is a growing job market, in both academia and industry, for scientists who have a strong foundation in the basic methods of biomedical laboratory research, and are trained in emerging techniques and technologies. We have designed this new program to provide students with extensive hands-on training in basic molecular biology, cell biology and biochemistry as well as rotations in our state-of-the art core facilities where they will be provided one-on-one training in the use of stem cell techniques, the generation of transgenic animals, flow-sorting, histology, high-end imaging techniques, animal handling, and single-cell techniques. Our first class of five students started classes on August 24 and we are expecting that they will graduate in May of 2017. If you would like more information this new and exciting program, please contact us at BMRMSProg@cchmc.org.
Cincinnati Children’s Hospital Medical Center is among a group of medical research institutions awarded $52.4 million by the National Institutes of Health (NIH) over the next four years to identify the potential medical effects of rare genomic variants in about 100 clinically relevant genes.
Led by principal investigator John Harley, MD, PhD, director of the Center for Autoimmune Genomics & Etiology at Cincinnati Children’s, the medical center will receive $3.4 million over four years to evaluate the role of 100 genes in the genomes of 2,500 patients who agree to receive their test results.
A study published in the journal Magnetic Resonance in Medicine and led by scientists at Cincinnati Children’s has demonstrated the feasibility of using ultrasound as a non-invasive therapy to destroy fat. This development may lead to new treatments for metabolic syndrome. The lead author, Charles Dumoulin, PhD, is the director of the Imaging Research Center at Cincinnati Children’s.
The Center for Clinical and Translation Science and Training (CCTST) has been awarded $16.7 million from the National Institutes of Health (NIH) to further enhance its efforts in translating basic scientific discoveries from the laboratory bench to patients’ bedsides. "This new funding will enable us to build on the transformation accomplishments we’ve been able to instigate or serve as a catalyst for at the CCTST,” says James Heubi, MD, co-director of the CCTST. The Center is a collaborative research resource among Cincinnati Children’s Hospital Medical Center, the University of Cincinnati and the Cincinnati Department of Veterans Affairs Medical Center.
A study published online Aug. 24 in the journal Pediatrics finds a significant decrease in the use of computed tomography (CT) scans at children’s hospitals for 10 common childhood diagnoses. Study authors hypothesize the decline in CT usage may be attributable to a growing body of evidence linking ionizing radiation from CT scans to an increased risk of cancer in patients. “This study reinforces the pediatric community’s commitment to think about both immediate and long term risks and benefits of our treatment,” said Michelle Parker, MD, the study’s lead investigator and a physician in the Division of Hospital Medicine at Cincinnati Children’s.
Data reported Aug. 6 in Molecular Cell highlight a technology developed at Cincinnati Children’s that might offer scientists new ways of approaching previously unanswerable questions in the field of tissue development and disease research. Senior author Raphael Kopan, PhD, director of Developmental Biology, calls the SpDamID method “transformative for our research.”
“Providers must be more systematic in the screening, diagnosis and management of mental health conditions in children and teens with Down syndrome,” concludes Julia S. Anixt, MD, lead author of a study recently completed at Cincinnati Children’s. Researchers studied trends related to the prescription of stimulants, selective serotonin reuptake inhibitors (SSRIs) and atypical antipsychotics (AAPs).
A study led by Andreas Loepke, MD, PhD and published in the journal Pediatrics found that children under age 4 who received general anesthesia for surgery risk diminished language comprehension, lower IQ and decreased gray matter density in posterior regions of the brain.
Cincinnati Children’s researchers recently identified that parent-reported responses to the Pediatric Eosinophilic Esophagitis Symptom Score (PEESS® v2.0) questionnaire correspond to clinical and biologic features of eosinophilic esophagitis (EoE). The study’s first author, Lisa Martin, PhD, reports, “Because eosinophilic esophagitis is a disease with multiple symptoms, the ability to capture patient and parent perceptions of these symptoms is a major unmet need.”
Published in Environmental Health, a recent study found a link between exposure to pyrethroid pesticides and ADHD in children and young teens. Corresponding author Tanya Froehlich, MD states, “Given the growing use of pyrethroid pesticides and the perception that they may represent a safe alternative, our findings may be of considerable public health importance.”
A study published in Science Signaling by Kakajan Komurov, PhD suggests that maxing out the inherently stressed nature of treatment-resistant breast cancer cells thwarts their adaptive ability to evolve genetic workarounds to treatment.
A study recently published in Pediatrics finds thatpharmacies in neighborhoods with high rates of asthma-related emergency-room use and hospitalization filled fewer asthma controller medications compared to asthma rescue medications. Lead author, Andrew Beck, MD, MPH states, “Tracking medication fills could highlight ways in which pharmacies could deliver proactive, as opposed to reactive, asthma care.”
A Cincinnati Children’s-led study concludes that breastfeeding Arab mother and infants have a very high prevalence of Vitamin D deficiency. The study’s lead author, Adekunle Dawodu, MBBS, points to Arab women’s traditional style of dress as a major factor, as it largely prevents exposure of the skin to sunlight.
Why do some asthma patients respond well to corticosteroids while others do not? A new study reveals that VNN-1 gene expression is required for corticosteroids to be effective during an asthma attack. Gurjit Khurana Hershey, MD, PhD and her research team discovered the revealing bio-marker and are using their findings to develop new treatments for hard-to-treat asthma cases.
Promising research focused on redirecting an expectant mother’s immune cells away from the fetus suggests new therapeutic strategies for preventing pregnancy complications, including prematurity and stillbirth. Senior author Sing Sing Way, MD, PhD hopes these findings – published in the Journal of Clinical Investigation – will spark a renewed interest in the biomedical community to establish the underlying causes of certain pregnancy complications and the development of new therapeutic approaches.
Cardiovascular risks for severely overweight teens are much higher than previously realized, according to an article published online in JAMA Pediatrics. A part of the ongoing Teen Longitudinal Assessment of Bariatric Surgery (Teen-LABS), the findings include increased risk of elevated blood pressure, unhealthy cholesterol and insulin resistance in severely overweight teenagers. Thomas Inge, MD, PhD, acts as Chair for the Teen-LABS study.
Findings published online in the December 15th issue of JAMA Pediatrics report on a multi-dimensional study, co-led by Cincinnati Children’s. The study found that osteomyelitis treatment can be equally as effective through IV and oral antibiotics. According to study co-author Samir Shah, MD, MSCE, serious complications can be avoided by opting for oral antibiotics over IV treatment.
New research links ADHD and conduct disorder in young adolescents with increased alcohol and tobacco use. William Brinkman, MD, was lead author in the study published in the journal Drug and Alcohol Dependence.
A sickle Cell Anemia Clinical Trial ended early as conclusive data shows that hydroxyurea therapy offers safe and effective disease management of sickle cell anemia (SCA) and reduces the risk of stroke. Russell E. Ware, MD, PhD, was principal investigator of the study.
A new type of cell transplantation developed to treat mice mimicking a rare lung disease could one day be used to treat this and other human lung diseases caused by dysfunctional immune cells. The study was published online Oct. 1 by Nature.Bruce Trapnell, MD is senior author.
Treating infants with high doses of steroids fails to improve medical outcomes in the end-stage pediatric liver disease, according to a study published in the May 7, Journal of the American Medical Association. Jorge Bezerra, MD was principal investigator.
By studying the genomes of twin 3-year-old sisters, researchers have uncovered a molecular pathway involving the gene SETD2 that could lead to better treatments for aggressive leukemia. The study was published online Feb. 9 in Nature Genetics. Gang Huang, PhD, a researcher in the divisions of Pathology and Experimental Hematology and Cancer Biology was a co-corresponding author.
In a mouse study, researchers have successfully used blood platelets and bone marrow cells to deliver potentially curative gene therapy to treat Hurler syndrome. The study appeared online Feb. 3 in Proceedings of the National Academy of Sciences (PNAS). Dao Pan, PhD, a researcher in the Division of Experimental Hematology and Cancer Biology, was corresponding author.
A new study published online Feb. 2 in Pediatrics finds no evidence that the human papillomavirus (HPV) vaccine leads to unsafe sexual behaviors among teenage girls and young women. Jessica Kahn, MD, a physician in the Division of Adolescent and Transition Medicine was senior author.
A new test that measures five key biomarkers can quickly and accurately predict the risk of death in children with septic shock, according to a study published online Jan. 29 in PLOS ONE. The multi-institutional study was led by Hector Wong, MD, Director, Division of Critical Care Medicine at Cincinnati Children’s and Christopher Lindsell, PhD, UC College of Medicine.
A basic signaling pathway known to play important roles in normal cell and cancer cell formation also plays an unexpected role as a molecular switch that controls the aging process of hematopoietic stem cells (HSCs), according to a study published online Oct. 20, 2013, in Nature. The study was led by Hartmut Geiger, PhD, Experimental Hematology and Cancer Biology.
Severely obese teens are four times more likely to have swollen legs with skin ulcers as adults and three times more likely to develop severe walking limitations and abnormal kidney function, according to a study published online Nov. 18, 2013, in Pediatrics by Thomas Inge, MD, PhD, and colleagues.
Treatment with antioxidants may help reduce behavioral issues linked to neurofibromatosis 1 (NF1) and an associated condition, Costello syndrome. Findings were posted Sept. 12 in Cell Reports. The study was led by Nancy Ratner, PhD, Division of Experimental Hematology and Cancer Biology.
Controlling the protein RhoA could help fight a variety of blood and immune system disorders, according to a study published online Oct. 7 in the Journal of Experimental Medicine. Yi Zheng, PhD, director of Experimental Hematology and Cancer Biology, was principal investigator.
RNA sequencing reveals in detail how insulin resistance can lead to insufficient breast milk production. The study, led by Laurie Nommsen-Rivers, PhD, at Cincinnati Children’s and scientists at the University of California Davis, was published in July 5, 2013, in PLOS ONE. The findings suggest a potential biomarker to predict which women may have difficulty breastfeeding.
Everolimus can dramatically reduce seizures in patients with tuberous sclerosis complex (TSC), according to a study posted online June 24, 2013, in Annals of Neurology. The study was led by Darcy Krueger, MD, PhD, at Cincinnati Children’s in collaboration with a team at Texas Children’s Hospital.
Scientists at Cincinnati Children’s have successfully targeted a malfunctioning immune system enzyme to kill diseased cells from patients with myelodysplastic syndrome (MDS). The study, led by Daniel Starczynowski, PhD was published July 8, 2013, in Cancer Cell.
In a study published May 21, 2013 in Environmental Health Perspectives, a team led by Nicholas Newman, DO, MS, director of the Pediatric Environmental Health and Lead Clinic at Cincinnati Children’s, reported that children exposed to high levels of traffic-related air pollution (TRAP) during their first year of life were more likely to have “at risk” scores for hyperactivity by age 7.
The same modern medical devices that have helped extend and enhance life for so many children also can cause complications that have not been well-understood, according to a study led by Patrick Brady, MD, MSc, a physician in the Division of Hospital Medicine. Findings were posted online June 7, 2013, in the Journal of Hospital Medicine.
Beyond the known effects that surgical anesthesia can have on the developing brains of young children, new findings posted June 5 the Annals of Neurology suggest the threat may also apply to adult brains. The study was led by Andreas Loepke, MD, PhD, a physician and researcher in the Department of Anesthesiology.
An international team of researchers led by Cincinnati Children’s has confirmed 14 more genes linked to juvenile idiopathic arthritis (JIA), bringing the total to 17. The study involved patient DNA samples from across the United States, Germany and United Kingdom, says Susan Thompson, PhD, a researcher in the Division of Rheumatology who was a leader in the project. Findings were published April 21, 2013, in Nature Genetics.
Two distinct microbial imbalances in the digestive tract may serve as biomarkers to predict the onset of necrotizing enterocolitis (NEC), according to a study led by Ardythe Morrow, PhD. Findings were published April 16, 2013, in the journal Microbiome.
Manipulating bile acid levels may be enough to recreate the key effects of bariatric surgery without the need for an invasive procedure, according to a study led by Rohit Kohli, MBBS, MS, a member of the Division of Gastroenterology. Findings were published online April 16, 2013, in Endocrinology.
For children with X-SCID, a new version of gene therapy shows renewed promise. A boy treated when he was 8 months old is doing well several months after receiving experimental therapy through a clinical trial at Cincinnati Children’s.
Scientists at UC and Cincinnati Children’s report that a new molecular pathway may offer a new way to kill leukemia cancer stem cells that survive traditional forms of treatment. Findings were published in June 2012 in the journal Blood. Our researchers also are using new lines of “humanized” mice to explore the potential of microRNA inhibitors and small-molecule inhibitors as anti-cancer weapons.
The 10-center Hybrid Immunotherapy for HLH (HIT-HLH) trial, led by Michael Jordan, MD, is the first US-based clinical trial to focus on this condition. Researchers at Cincinnati Children’s also are studying a promising antibody, a possible gene replacement therapy and targeted drug therapies to improve HLH treatment.
Even in the womb, the eye needs light to develop normally. This and other unexpected findings could change our understanding of how the retina develops, according to a study published online Jan. 16, 2013, in Nature. The paper was co-authored by Richard Lang, PhD, a researcher in the Division of Pediatric Ophthalmology at Cincinnati Children’s and David Copenhagen, PhD, a scientist at UCSF.
Thousands of people have had kidneys removed unnecessarily because doctors misdiagnose patients who actually have tuberous sclerosis complex (TSC), according to a study published online Jan. 11, 2013, in Lancet. Proper diagnosis could have led to a medication that would have made surgery or kidney removal unnecessary, according to John Bissler, MD, a nephrologist at Cincinnati Children’s and lead author of the study.
Thirty percent of teen girls report having offline meetings with strangers and semi-strangers they meet on the Internet, according to a study published online in Pediatrics. The study, led by says Jennie Noll, PhD, a psychologist in Behavioral Medicine and Clinical Psychology at Cincinnati Children’s, shows the risk is further heightened for teen girls who have been victims of abuse or neglect.
A preclinical study in mice successfully used targeted molecular therapy to block mostly untreatable nerve tumors caused by the genetic disorder Neurofibromatosis 1 (NF1). The study was led by Nancy Ratner, PhD, program leader for the Cancer Biology and Neural Tumors Program in the Cancer and Blood Disorders Institute. Findings were published online Dec. 10, 2012, in the Journal of Clinical Investigation.
Teenage girls who smoke accumulate less bone during a critical growth period and carry a higher risk of developing osteoporosis later in life, according to research published Dec. 4, 2012, in the Journal of Adolescent Health. The study was led by Lorah Dorn, PhD, director of research in the Division of Adolescent Medicine.
A research team led by Cincinnati Children’s has discovered a genetic mutation responsible for deafness associated with Usher syndrome type 1. These findings offer a potential target for new therapies, says Zubair Ahmed, PhD, senior investigator. The study results were published online Sept. 30 in Nature Genetics.
Marijuana consumption may increase risks of preeclampsia and other pregnancy complications, according to SK Dey,PhD, director, Division of Reproductive Sciences. Analysis of mouse models indicates that THC can affect placenta development. Findings appeared Sept. 14 in The Journal of Biological Chemistry.
Warning labels are not working. Tougher rider training and helmet laws are needed to reduce deaths among underaged All-Terrain Vehicle (ATV) riders, according to Rebeccah Brown, MD. She presented new findings about ATV crashes Oct. 22 at American Academy of Pediatrics annual meeting in New Orleans.
Molecular disruptions in granule cells – located in the dentate gyrus region of the brain – caused brain seizures in mice similar to those seen in human temporal lobe epilepsy, according to a study led by Steven Danzer, PhD, a neuroscientist in the Department of Anesthesia. The findings appeared Sept. 19 in Neuron.
A small-molecule-inhibiting drug dubbed “Rhosin” stopped breast cancer cells from metastasizing and promoted nerve cell growth in early laboratory cell tests. Although years away from market, Rhosin eventually could treat a variety of cancers and could promote spinal cord regeneration, says lead investigator Yi Zheng, PhD, director of Experimental Hematology and Cancer Biology. Findings appeared June 21 in Chemistry & Biology.
A geographic social risk index based on income, home values and the parents’ education levels can help hospitals predict which children with asthma are most likely to need readmission, according to research led by Andrew Beck, MD. Study results were posted online Oct. 18 in the American Journal of Public Health.
The mechanisms involved in preventing a pregnant woman’s body from rejecting her baby as a foreign object eventually may lead to a new class of vaccines that could prevent preterm births and possibly other autoimmune diseases, according to Sing Sing Way, MD, PhD, a physician researcher in Infectious Diseases. Findings were published online Sept. 26 in the journal Nature.
Persistent and loud snoring in young children can make preschool behavior problems worse, according to Dean Beebe, PhD, director of the neuropsychology program at Cincinnati Children’s. Behaviors affected by snoring include hyperactivity, depression and inattention. Findings were published online Aug. 13 in Pediatrics.
The HPV vaccine does more than protect the vaccinated. A study led by Jessica Kahn, MD, MPH, a physician in the division of Adolescent Medicine at Cincinnati Children’s, is the first to document that the vaccine also provides herd immunity. Findings were published online July 9 in Pediatrics.
A 9-minute modified echocardiogram is more effective than EKG at detecting athletes at risk of sudden cardiac death, according to a study led by Michelle Grenier, MD, a physician at the Cincinnati Children’s Heart Institute Findings were presented in July at the annual meeting of the American Society of Echocardiography.
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