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This article was originally posted on the WCM Newsroom

In 2021, a group of scientists led by researchers at the University of North Carolina at Chapel Hill, Weill Cornell Medicine and NewYork-Presbyterian reported that the Moderna mRNA vaccine and a protein-based vaccine candidate containing an adjuvant, a substance that enhances immune responses, elicited durable neutralizing antibody responses to SARS-CoV-2 during infancy in pre-clinical research.

Now a follow-up study by the same group, published Dec. 1 in Science Translational Medicine, has found that the 2-dose vaccines still provide protection against lung disease in rhesus macaques one year after they had been vaccinated as infants.

An unusual type of antibody that even at miniscule levels neutralizes the Zika virus and renders the virus infection undetectable in preclinical models has been identified by a team led by Weill Cornell Medicine, NewYork-Presbyterian and National Institutes of Health (NIH) investigators. 

Because Zika can cause birth defects when passed from a pregnant person to their fetus, this discovery could lead to the development of therapies to protect babies from the potentially devastating effects of this disease. 

In research published Nov. 18 in Cell, the investigators isolated an ultrapotent immunoglobulin M (IgM) antibody — a five-armed immune protein that latches onto the virus— using blood cells taken from pregnant people infected with Zika. In experiments with mice, they determined that the antibody not only protected the animals from otherwise lethal infections, but also suppressed the virus to the point that it could not be detected in their blood. 

Eric J. Mallack, M.D., Director of the Leukodystrophy Center and Assistant Professor of Pediatrics and Neurology at Weill Cornell Medicine is the Co-Principal Investigator of a new phase 2/3 clinical trial, sponsored by Minoryx Therapeutics. The “NEXUS” study will assess the efficacy and safety of leriglitazone in pediatric patients with early-stage cerebral adrenoleukodystrophy (cALD).

 Alongside Co-PI, Patricia Musolino, MD, PhD from Massachusetts General Hospital, the team aims to confirm whether researchers can halt or stabilize early cerebral ALD, a progressive inflammatory neurodegenerative disease of childhood, with leriglitazone - a novel, orally bioavailable and selective PPAR gamma agonist.   If proven effective, this will be the first non-transplant-related therapy (e.g. stem cell transplant and gene therapy) that can halt this childhood neurodegenerative disorder.  And if delivered early enough, it may stop the disease in the pre-symptomatic period of disease, thus preserving full neurological function in these patients.  

 The imaging outcomes in the study were developed from Dr. Mallack’s quantitative MRI research in lesion volumetry (PMID: 34503945), and Dr. Musolino’s work in MR Perfusion (PMID: 22961546).

This article was originally posted on EurekAlert.

A multi-institutional team of scientists, led by researchers at Weill Cornell Medicine’s Gale and Ira Drukier Institute for Children's Health, have received a five-year $8.297 grant to continue funding a Center for Lupus Research. The grant, awarded by the National Institute of Arthritis and Musculoskeletal and Skin Diseases of the National Institutes of Health, will allow researchers to explore the underlying mechanisms of systemic lupus erythematosus (SLE) in children with the goal of better tailoring treatment.

“Pediatric lupus is often underrecognized, but up to a quarter of people with the illness have disease that starts in childhood,” said Dr. Virginia Pascual, program director of the Center for Lupus Research and the Drukier Director of the Drukier Institute for Children's Health at Weill Cornell Medicine.

We are pleased to announce Sheila J. Carroll, M.D. and Patrick A. Flynn, M.D. have been named Interim Co-Chiefs of Pediatric Cardiology, effective October 1, 2022. Dr. Carroll, who serves as the Director Fetal Cardiology, and Dr. Flynn, who serves as the Director of the Echocardiography Lab, will provide proven leadership and oversight for the Division’s activities, including liaising with Department administration and supporting the Department’s vision and missions in patient care, research, and education as well as quality, diversity, and inclusion.

We are grateful for the excellence and innovation of current chief Dr. Ralf Holzer. Under Dr. Holzer’s leadership from 2017 to 2022, the Division dramatically grew its pediatric cardiac catheterization program, expanded its clinical Pediatric Cardiology practice to Lower Manhattan, strengthened relationships with partner departments and institutions, and achieved the first premature infant transcatheter PDA closure in Manhattan. We wish Dr. Holzer well in his future endeavors.

Lisa Roth, M.D., Director of Pediatric Oncology and an associate professor in pediatrics, medicine and pathology and laboratory medicine at Weill Cornell Medicine has been awarded a grant from the NIH National Cancer Institute for her study, “Targeting Latency Switch in EBV+ Lymphomas”. 

Epstein-Barr virus (EBV) is a herpesvirus that infects B cells, the immune cells that make antibodies and that cause lymphoma when the cells grow uncontrollably. Many EBV-infected lymphoma cells are able to evade an immune response against EBV because the virus establishes a latent, or largely dormant, infection, restricting expression of its genes so that only one protein, which elicits a weak immune response, is expressed. In this study, Dr. Roth will develop a novel approach to the treatment of EBV-infected lymphomas by using drugs that affect gene expression to convert dormant virus in tumors to a more active state, thereby sensitizing resistant tumors to an anti-tumor immune response directed at EBV.

This article was originally posted in the Yale Medicine Newsroom. 

In a first of its kind pilot study, Oliver Karam, MD, PhD, chief of pediatric critical care at Yale School of Medicine, and Marianne Nellis, MD, MS, associate professor in pediatric critical care at Weill Cornell Medicine, have been funded by the National Institutes of Health (NIH) to study different platelet transfusion thresholds for children supported by ECMO machines (extracorporeal membrane oxygenation). The ECmo hemoSTAtic Transfusions In Children (ECSTATIC) pilot trial will test two different platelet transfusion strategies, based on two different platelet count thresholds in critically ill infants and children.

Patients have received blood transfusions since as far back as 1820 but they became more widely used on the battlefield during World War I. Ever since, people have been giving and receiving blood, but there are relatively few studies to help doctors determine when and how blood products may be used most effectively.

This article was originally posted in the WCM Newsroom. 

Unlike COVID-19 or the flu, polio is entirely and permanently preventable. In fact, until this year, there had not been one case of polio that originated in this country since 1979. A case in 1993 was brought to these shores by someone who had traveled to a country where the disease was endemic.

As long as you and your children are vaccinated, you have nothing to worry about. But if you are unvaccinated or under-vaccinated, doctors and public health officials urge you to waste no time in getting the polio vaccine, which confers lifelong protection against a potentially disabling disease.

The public health picture

A handful of polio-caused paralysis cases in New York, London and Jerusalem may seem minor compared to the massive global impact of the COVID-19 pandemic. But in public health terms, the recent polio outbreak is anything but minor. That’s because the poliovirus causes paralysis in fewer than 1 in 200 of the people it infects, according to the World Health Organization (WHO). Think of the one case of paralysis in New York’s Rockland County as the tip of a very large iceberg.

Ms. Claire Otero, a graduate assistant in Pediatrics, has been awarded a grant from the National CMV Foundation for her study, “Pre-clinical assessment of HCMV viral Fc receptors as vaccine targets.” The study aims to improve vaccine immunogenicity by targeting proteins expressed by cytomegalovirus (CMV) that interfere with key antibody responses.

 CMV is the most common congenital infection, leading to a high burden of hearing loss and neurologic impairment in many affected infants. However, a vaccine against CMV has yet to be licensed, in part due to unclear immune correlates of protection. Recent work has implicated Fc mediated antibody effector functions (aka non-neutralizing antibodies) in prevention of placental CMV transmission.

This is a proof-of-concept study to define the immunogenicity of CMV viral Fc receptors (vFcRs), which function to inhibit Fc-mediated antibody responses. The overall goal is to improve Fc mediated antibody responses against standard vaccine antigens by targeting one or more vFcRs via active vaccination.

Oleh Akchurin, M.D., Ph.D., Assistant Professor of Pediatrics and Associate Director of the Physician Scientist Training Program in Pediatrics has received funding for his study, “Role of myeloid cells in the pathophysiology of anemia in children with chronic kidney disease” from the Sy Syms Foundation and Van Pelt/Bass Foundation.

Macrophages are known to play a major role in iron metabolism, as evident from non-kidney related pre-clinical and patient-oriented studies. At the same time, disruptions in iron metabolism play a critical role in the development of anemia in children with chronic kidney disease (CKD).  However, the role of macrophages, and their precursors, monocytes, in the development of anemia in children with CKD remains poorly understood. In this study, stemming from his previous work, Dr. Akchurin will test how the changes in monocyte/macrophage behavior in CKD affect the production and survival of red blood cells. His research team will specifically focus on the function of ferritin in macrophages in monocytes.

Pediatrics Weill Cornell Medicine Appointments & Referrals: (646) 962-KIDS (646) 962-5437 Chair's Office: Weill Cornell Medicine 525 E 68th St.
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