IMPORTANT UPDATES

Arthur M. Blank Hospital is Built for Breakthroughs

As Children’s grows, so, too, does its research capabilities.

Arthur M. Blank Hospital, now under construction on Children’s 78-acre North Druid Hills campus, is the largest healthcare project ever in the state of Georgia. Designated to be the only dedicated Level 1 pediatric trauma center in Georgia, the hospital is on schedule to open in 2025. In addition to overall patient care, the 1.5-million-square-foot facility will help accelerate research by creating a dedicated space for breakthroughs, including clinical research and trials, that will transform the future of pediatric healthcare for generations to come.

To that end, the hospital will house a technologically advanced GMP (good manufacturing practice) facility to produce cell therapies for The Marcus Center for Pediatric Cellular Therapy which will be used in clinical trials for many pediatric diseases, including asthma, cancer, osteogenesis imperfecta, and others, with hopes of finding cures and treatments.

“Arthur M. Blank Hospital will provide the best care to the kids who need us most for many generations to come,” says Donna Hyland, Chief Executive Officer of Children’s. “We are proud of the pediatric future we are building for our city, state and country.”

Emory University Builds Innovative Biomedical Research Facility

The new Health Sciences Research Building II (HSRB-II) at Emory University will be an eight-story facility with research space for imaging sciences, biomedical engineering, cardiovascular medicine, cancer, inflammation, immunity and immunotherapeutics, emerging infections and other innovative interdisciplinary research programs. The new facility is funded in part by the Robert W. Woodruff Foundation and Children's with approximately two floors of laboratory space reserved for pediatric research. HSRB-II is scheduled to open in 2022.

"This expansion of laboratory space will allow us to align our research across specific disease groups to optimize collaboration between investigators,” says Doug Graham, MD, PhD, Chief of Aflac Cancer and Blood Disorders Center of Children’s. “We will also be able to recruit top national researchers into this state-of-the-art environment, which will help us in our goal to be one of the premier research centers in the country. The research performed in this space will directly advance the care we can provide to children."

An out-of-this-world experiment may lead to new ways to treat heart disease in children and adults.

Researchers at the Emory-Children’s Center, after more than a year of analysis, determined that cardiac stem cells grew into numerous, mature, beating heart cells while aboard NASA’s SpaceX-20 mission in March 2020. Chunhui Xu, PhD, Associate Professor in the Department of Pediatrics at Emory, Kevin Maher, MD, Pediatric Cardiologist for Children’s, and their colleagues dived into the data from the space experiment, seeking to determine the effects of real zero gravity conditions on stem cells and to optimize the generation of clinically relevant cardiac muscle cells on earth.

Their research is critical in finding ways to apply cell therapy so that kids, such as Brody Parker, pictured above, will have enough robust heart cells for surgery, or children with damaged heart valves may one day receive a replacement valve grown from their own cells. Before having astronauts conduct the experiment on the International Space Station (ISS), the team used space-simulation machines to enhance the ability of pluripotent, or immature, stem cells to turn into cardiac muscle cells.

“These cells have the potential to treat heart disease in kids and adults, but repairing a damaged heart requires a large number of mature heart cells,” says Dr. Xu. “This space experiment may teach us to identify a more effective way to generate these cells.”

While the world is understandably focused on COVID-19, research continues on another deadly disease: HIV.

Despite major advances in prevention and treatment, millions of children and adolescents continue to live with HIV each day. Ann Chahroudi, MD, PhD, Infectious Disease Specialist at Children’s and Associate Professor of Pediatrics at Emory, was awarded $27.6 million from the NIH to accelerate the search for a cure for HIV in children and adolescents, as co-principal investigator with the Yerkes National Primate Research Center and Johns Hopkins University School of Medicine.

The grant is part of the Martin Delaney Collaboratories for HIV Cure Research program called the Pediatric Adolescent Virus Elimination (PAVE) Collaboratory, which uses a multidisciplinary, multicultural and iterative approach to study pediatric HIV.

“With this funding and a truly outstanding group of investigators and industry partners, we are thrilled to synergize the pediatric cure research efforts globally. The award will also allow us to apply state-of-the-art scientific tools to understand HIV persistence in children and adolescents with the ultimate goal of achieving HIV remission or eradication,” says Dr. Chahroudi.

The PAVE Collaboratory aims to identify and harness the unique immunovirological features of HIV infection in children and adolescents. PAVE team members will conduct preclinical safety and effectiveness research studies of novel treatments, focusing on developing procedures, tools and techniques, including imaging, specifically for infants, children and adolescents.

Emergency medicine research has seen tremendous gains since the emergency department at Egleston Hospital was selected a Pediatric Emergency Care Applied Research Network (PECARN) site in 2019.

Under the leadership of Children’s Pediatric Emergency Medicine Physicians Claudia R. Morris, MD, Professor of Pediatrics at Emory, and Harold Simon, MD, MBA, Vice Chair of Pediatrics at Emory, the program now receives millions of dollars annually in federal grants and is a high-enrolling site for several NIH-funded multi-center studies – with one of the largest being an $8.7 million NIH grant for the Sickle cell disease Treatment with Arginine Therapy (STArT) trial.

Several other studies made significant strides in 2021, as follows:

  • Rob Grell, MD, Pediatric Emergency Medicine Fellow of Children’s and Emory, received the Warshaw Fellow’s Research Grant to study the use of a virtual reality device to detect concussion in children presenting to the emergency department with head injury. He is collaborating with Michelle C. LaPlaca, PhD, Professor at the Wallace H. Coulter Department of Biomedical Engineering at Georgia Tech and Emory.
  • During the pandemic, the emergency department was an active contributor to enrollment for evaluating COVID-19 diagnostics for RADx Atlanta, as many children with COVID-19 and MIS-C presented there for acute care.
  • A longitudinal surveillance study of SARS-CoV-2 antibodies in pediatric healthcare workers, led by Dr. Morris and Miriam Vos, MD, Pediatric Hepatologist with Children’s and Professor of Pediatrics at Emory, identified novel mechanisms of disease with potential therapeutic opportunities. The study, funded in part by the Emory Woodruff Health Science Center Synergy Award, enrolled more than 640 participants, was published in many multidisciplinary publications, and will now look into SARS-CoV-2 antibodies in response to vaccination.
  • The work of Chris A. Rees, MD, MPH, Pediatric Emergency Medicine Physician at Children’s and Assistant Professor of Pediatrics and Emergency Medicine at Emory, examined race and ethnicity of children enrolled in clinical trials in the U.S., and observed they were not representative of the nation as a whole. He presented these findings at the 2021 American Academy of Pediatrics National Conference & Exhibition. “Our study identifies key areas in which we, as a pediatric research community, can improve enrollment in clinical trials to be more equitable for all groups,” said Dr. Rees. “Results from clinical trials that lack American Indian, Alaska Native, Asian and Native American-Pacific Islander participants may not be generalizable to all populations that may benefit from trial results.”

Race plays a role in genetic risk factors for inflammatory bowel disease (IBD), according to breakthrough research from Children’s and Emory.

Results of the first whole-genome study of IBD in African Americans show the genetic risk landscape is very different in African Americans than people with European ancestry. Lead author Subra Kugathasan, MD, Pediatric Gastroenterologist at Children’s, says future clinical research on IBD needs to take ancestry into account.

Findings of the multi-center study, which analyzed the whole genomes of more than 1,700 affected individuals with Crohn’s disease and ulcerative colitis and more than 1,600 healthy controls, were published in the American Journal of Human Genetics. As part of the analysis, the researchers developed an algorithm that corrects for ancestry when calculating IBD polygenic risk scores. These scores are tools for calculating gene-based risk for a disease and are used for IBD as well as other complex conditions such as coronary artery disease. The study showed that a gene, PTGER4, that plays an important role in determining the risk for IBD in African Americans, plays a relatively minor role in European populations. In contrast, two gene loci that are important in Europeans – NOD2 and IL23R – play smaller roles in African Americans.

“Even though the disease destination looks the same, the populations look very different, in terms of what specific genes contribute to risk for IBD,” says Dr. Kugathasan, Marcus Professor of Pediatrics and Human Genetics at Emory. “It shows that you can’t develop a polygenic risk score based on one population and apply it to another.”

The National Organization for Rare Disorders (NORD) has named the Division of Medical Genetics a NORD Rare Disease Center of Excellence. A partnership of Children’s and Emory, the newly designated center is one of 31 medical centers nationwide that are part of an innovative network aimed at expanding access, advancing care and enhancing research for rare disease patients in the United States.

Children and adults living with rare diseases frequently face many challenges in finding a diagnosis and quality clinical care. The average rare disease diagnosis can take several years and require many tests and visits to specialists. There are over 7,000 rare diseases – most of them with a genetic origin -- and 25-30 million Americans are estimated to be currently living with rare diseases.

Within the Division of Medical Genetics, researchers focus on a number of specialty areas, such as fragile X syndrome and lysosomal storage disorders. But clinicians have broad experience with many rare diseases. The center is also responsible for follow-up after newborn screening for metabolic diseases within the state of Georgia, and hosts a genetic counseling training program. In addition, genetics researchers counsel families who learn that a child has a previously unrecognized genetic disorder. Several clinical trials are testing the first therapies available for a specific condition, such as an ongoing study of the medication vosoritide, which enhances bone growth in children with achondroplasia; and another study of trofinetide for girls and adolescent females with Rett syndrome.

“We are delighted that NORD has recognized our team’s commitment to patients and their families with rare disease,” says Michael J. Gambello, MD, PhD, Clinical Geneticist at Children’s and Vice Chair of the Department of Human Genetics at Emory. “We have an exceptionally dedicated group of geneticists, genetic counselors, metabolic dietitians and support staff.”