MRSA, EoE Clinical Trial, Rare Disease, Exploring the Hypothalamus

In the News

By Jillian Rose Lim

It’s all about discovery and driving pediatrics forward in this week’s roundup of research headlines. Learn how one Children’s Hospital of Philadelphia researcher and his lab are studying drug-resistant bacteria strains in Brazil, congratulate our rare disease and food allergy experts on new funding that will lead to novel breakthroughs, and get the latest on the hypothalamus – the part of your brain that influences sleep, stress, and body mass index – from our genomics scientists.

CDC Podcast Features New Research From Planet Lab

Paul Planet, MD, PhD, assistant professor of Pediatrics at CHOP, discussed multi-drug resistant and methicillin-resistant Staph aureus (MRSA) on the Center for Disease Control and Prevention’s Emerging Infectious Diseases (EID) podcast series. Speaking with podcast host Sara Gregory, Dr. Planet described his lab’s recent findings regarding a new strain of MRSA that has rendered the old circulating type of MRSA almost extinct.

A global problem, MRSA is a kind of Staph aureus germ that has acquired resistance to the best antibiotic to treat it. Dr. Planet worked with researchers in Rio de Janeiro, Brazil, to better understand the epidemiology of the MRSA emerging in hospitals in that region. In the EID journal, he describes his team’s discovery that the new strain of MRSA (or new group of MRSA strains) began to circulate and expand between the hospitals studied by his lab around the same time, in 2009. Compared to other closely related isolates, the strain, known as the Rio de Janeiro more effectively evaded immune function.

“There are two things that are happening here,” said Dr. Planet on the podcast. “One is that there’s the multidrug aspect of this new epidemic of MRSA. And then there’s also the immunization. So these are two things that are incredibly important for people’s health, but they also sort of exist in two different streams. The first one, the evasion of the immune system, is something that could lead to more invasive infection, worse infection, infections that are harder to control. From the multidrug standpoint, not only is this MRSA resistant to methicillin and oxacillin and other penicillin, but it’s also resistant to other drugs which we often use to treat MRSA.”

Listen to the podcast or read a transcript here.

Food Allergy Experts to Launch New Clinical Trial for Eosinophilic Esophagitis

CHOP researchers will launch a new clinical trial for patients with eosinophilic esophagitis (EoE), a chronic allergy inflammatory disease of the esophagus. EoE patients have previously been excluded from clinical trials for oral therapy because ingesting their treatment foods can cause abdominal pain, vomiting, and trouble swallowing. The new clinical trial will utilize a current FDA-medication called dupilumab to introduce foods that cause EoE into patients’ diets.

“One of the major treatments for EoE is food avoidance; patients with EoE have been told to avoid these foods for their entire life,” said Jonathan Spergel, MD, PhD, chief of the Allergy Program, in a press release. “Dupilumab is currently used as a medication for eczema and asthma. We surmise that leveraging use of the existing drug for this new purpose will allow our patients to expand their diet and add these foods into their meals.”

The Food Allergy Center at CHOP has one of the largest teams of pediatric food allergy experts in the world and is nationally recognized for its expertise in diagnosing and treating all four types of food allergies. A generous gift from the Mondre and Lane families, who have firsthand experience living with severe food allergies, will support the clinical trial that could potentially bring an oral therapy to children with EoE.

“We are incredibly proud to partner with the Lane family to support Dr. Spergel and his team at CHOP to advance their research on food allergies and EoE,” said Alexandra and Greg Mondre in a press release. “This study has the potential to change the lives of thousands of children suffering from dangerous food allergies, and we are honored to support it.”

Learn more.

NORD Names CHOP and Penn a Rare Disease Center of Excellence

Congratulations to our rare disease researchers at CHOP and Penn Medicine, as the two institutes have been jointly named an inaugural Rare Disease Center of Excellence by the National Organization for Rare Diseases (NORD) after a competitive application process. There are 31 Rare Disease Centers of Excellence across the U.S. whose goal is to bring together leaders in the study and treatment of rare diseases to reduce the time required to properly diagnose rare diseases and to coordinate multi-specialty clinical care. 

“The program is being led by NORD to promote outstanding treatment for rare disease patients regardless of disease or geography, elevate collaboration, improve standards of care, advance research, and increase awareness about rare diseases in the broader medical and patient communities,” stated NORD in a press release. “These Centers of Excellence will strive to push the rare disease field forward by collaborating to develop new care guidelines, improve medical and family education, create safe and effective referral pathways, and innovate around new treatments, therapies, and research.”

The hefty application process required evidence of staffing with experts across multiple specialties to meet the needs of rare disease patients, as well as significant contributions to rare disease patient education, physician training, and research.

Learn more.

Hypothalamus Model Helps Implicate Genes Associated with Sleep, Stress, and More

Findings from a new study led by CHOP researchers could help clinicians identify potential causes of dysfunction for important traits regulated by the hypothalamus – a brain region notoriously difficult to study – such as sleep, stress, and reproduction. Describing their work in Nature Communications, the researchers implicated several genes associated with those traits by using an embryonic stem cell (ESC) model of the hypothalamus.

The ESC model allowed them to study gene expression during the hypothalamus’ development – first in hypothalamic progenitor cells (cells prior to their full development into a hypothalamus) and then in arcuate nucleus-like hypothalamic neurons. The researchers integrated results from a number of genome-wide association studies (GWAS) to identify both known and novel genes associated with puberty, body mass index, height, bipolar disorder, sleep, major depressive disorder, and more. Their data confirmed, for example, the role of the BDNF gene in influencing body mass index and obesity risk. Meanwhile, the PER2 gene, implicated in sleep regulation, was also identified.

“By studying the three-dimensional genomic architecture of these cell models, we can see the dynamic process of how the hypothalamus is formed over different stages of development,” said senior study author Struan F.A. Grant, PhD, director of the Center for Spatial and Functional Genomics. “The information we yielded in this study provides us with more concrete information about diseases that are relevant to hypothalamic function.”

Learn more.

ICYMI

Catch up on our headlines from our November 12 In the News:

• COVID-Lab Forecasting Model Highlights Variation in Regional COVID-19 Transmission
• CHOP and Penn Collaborators Launch Open Access Resource for Academic Entrepreneurship
• CHOP Researchers Present at Kidney Week
• Daniel Rader Honored with AHA Research Achievement Award

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