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FDA Approval, Mitochondrial Dysfunction, Pediatric Headaches

Published on August 18, 2023 in Cornerstone Blog · Last updated 2 days 12 hours ago


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In the News


In this week's news roundup, the U.S. Food and Drug Administration approved the first treatment for fibrodysplasia ossificans progressive, based on foundational research by the Pacifici Lab. Research findings indicate COVID-19 results in mitochondrial dysfunction in organs other than the lungs. And a radio discussion with a CHOP neurologist covers current studies and treatments for headaches in children.

FDA Approves First Drug to Treat Rare, Severe Bone Disease

The U.S. Food and Drug Administration approved the use of palovarotene, a retinoid agonist oral drug manufactured by the pharmaceutical company Ipsen based on foundational research by the Pacifici Laboratory at CHOP. Palovarotene is the first treatment for heterotopic ossification (HO) in adults and children with fibrodysplasia ossificans progressiva (FOP), a debilitating, ultra-rare and often fatal disease. It is approved for females who are 8 years old and older, and for males who are 10 years old and older.

FOP causes permanent and continuous bone growth in soft and connective tissues such as muscles, tendons, and ligaments. Once the bone forms, it is irreversible, leading to loss of mobility, severe health complications and shortened life expectancy.

Maurizio Pacifici
Maurizio Pacifici, PhD

Maurizio Pacifici, PhD, director of Research in the Center for Orthopedic Surgery, has been studying the retinoic pathway to regulate cartilage and bone formation for more than 20 years. The drug he and colleagues developed is designed to mediate the interactions between the receptors, growth factors, and proteins within the retinoid signaling pathway to reduce new abnormal bone formation.

Positive results from the MOVE trial, the first global multicenter Phase III trial in FOP, demonstrated a 62% reduction in mean annualized new HO volume in participants treated with palovarotene, and led the FDA Endocrinologic and Metabolic Drugs Advisory Committee to vote in favor of investigational palovarotene as an effective treatment.

See Ipsen's press release for more information.

Study Shows Mitochondrial Dysfunction in Heart, Other Organs Caused by COVID-19 Virus

Douglas C. Wallace
Douglas Wallace, PhD

A multi-institutional consortium of researchers led by a team from the Center for Mitochondrial and Epigenomic Medicine (CMEM) and the COVID-19 International Research Team (COV-IRT) found that the genes of mitochondria, the energy producers of our cells, can be negatively impacted by the SARS-CoV-2 virus, leading to dysfunction in multiple organs beyond the lungs.

"This study provides us with strong evidence that we need to stop looking at COVID-19 as strictly an upper respiratory disease and start viewing it as a systemic disorder that impacts multiple organs," said co-senior author Douglas Wallace, PhD, director of the CMEM. "The continued dysfunction we observed in organs other than the lungs suggests that mitochondrial dysfunction could be causing long-term damage to the internal organs of these patients."

Prior studies have shown that SARS-CoV-2 proteins can bind to mitochondrial proteins in host cells, so researchers analyzed mitochondrial gene expression in a combination of nasopharyngeal and autopsy tissues from affected patients and animal models to detect potential differences caused by the virus.

The study revealed mitochondrial gene expression was recovered in the lungs, but mitochondrial function remained suppressed in the heart, the kidneys, and liver, suggesting host cells respond to initial infection in a way that involves the lungs but restores mitochondrial function over time, while mitochondrial function in other organs, particularly the heart, remain impaired.

Learn more about the study in this CHOP news release.

CHOP Neurologist Shares Headache Expertise on Doctor Radio

Christina Szperka, MD, MSCE

Christina Szperka, MD, MSCE

Christina Szperka, MD, MSCE, pediatric neurologist and director of the Pediatric Headache Program, represented CHOP on NYU Langone Health's Doctor Radio broadcast on Sirius XM to discuss current studies, possible red flags of symptoms, and treatment options for children with headaches.

"I appreciate the opportunity to talk about this topic," Dr. Szperka said in the broadcast. "It is important to many patients because headaches are so common. About three quarters of children will have experienced a significant headache by the time they get to their teen years. It's a symptom, and the symptoms can represent many different things. So when should you worry that there's something else going on in your child who has a headache?"

Dr. Spzerka touched on best practices for prevention, dispelling the myth that caffeine is useful for treating migraines. She explained that patients are better off avoiding it, especially if they have frequent headaches and drink caffeine daily, because the body gets used to it, resulting in worsening headaches.

A new study that Dr. Spzerka is part of will analyze telehealth cognitive behavior therapy (CBT) to determine if the effects of CBT are substantial enough alone to improve migraine symptoms or when combined with migraine medicines. Cincinnati Children's Hospital is the primary site for the study.

Listen to the entire broadcast on Doctor Radio NYU.


Catch up on our headlines from our August 4 In the News:

  • Celebrate PolicyLab's 15th Anniversary with its 2023 Impact Report
  • CHOP and Penn Medicine Receive $26 Million Grant to Study Rare Genetic Diseases
  • CHOP Researchers Validate 'Allergic March'
  • Tool Developed to Help Predict Alzheimer's Risk in Various Ethnic Populations
  • CHI-RON Study Nears Recruitment Deadline and Meets a Major Milestone
  • CARRA-Arthritis Foundation Grant Aims to Improve Pediatric Rheumatology Care

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