Infant Monitors, Beckwith-Wiedemann Syndrome, CAR T-cell Therapy, Seed Grants, Mitochondrial Research

Super Bowl Sunday is right around the corner, and our biweekly installment of In the News is the perfect playing field for us to showcase the wide range of research projects that Children’s Hospital of Philadelphia experts tackle. Huddle up!

Consumer-Use Vital Signs Monitors May Cause Parents Undue Alarm

Parents should keep a watchful eye over the use of infant physiological monitors, according to an article appearing in the Journal of the American Medical Association (JAMA), in which CHOP experts question consumers’ use of the devices because they have little proven benefit for healthy infants.

“These devices are marketed aggressively to parents of healthy babies, promising peace of mind about their child’s cardiorespiratory health,” said CHOP pediatrician and safety expert Christopher P. Bonafide, MD. “But there is no evidence that these consumer infant physiological monitors are life-saving or even accurate, and these products may cause unnecessary fear, uncertainty, and self-doubt in parents.”

For example, he said, one reading of low oxygen saturation that would resolve on its own may trigger a visit to an emergency department, followed by unnecessary blood tests, X-rays and even hospital admission.

The researchers focused on five models of infant physiological monitors introduced over the past two years at costs ranging from $150 to $300. The manufacturers do not directly claim their products treat, diagnose, or prevent disease, and the monitors have not been regulated as medical devices by the U.S. Food and Drug Administration (FDA). David Jamison, executive director of Health Devices at ECRI Institute, a nonprofit research organization that evaluates medical devices, procedures, and processes, co-authored the JAMA article and shared his concerns regarding the safety and effectiveness of these products.

“There is no publicly available evidence that these baby monitors are accurate in measuring a baby’s vital signs,” Jamison said. “And since these baby monitors are not regulated by the FDA, we have to question what testing has been done to assure the safety and quality of these designs.”

Read the full press release for more details.

Beckwith-Wiedemann Syndrome Registry Helping to Find Cancer Mechanisms

CHOP cancer research scientist, attending physician, and geneticist Jennifer M. Kalish, MD, PhD, aims to answer key questions about cancer while helping children with Beckwith-Wiedemann Syndrome (BWS), an overgrowth disorder that can result when epigenetic regulation of growth-regulating regions of chromosome 11 goes awry. In an interview with Science magazine that appears in the January issue, Dr. Kalish describes the insights she’s gained about BWS from her patients with this rare disorder and why she opened an international registry to track patients with BWS and gather tissue samples.

Read more about Dr. Kalish’s work in Bench to Bedside and find out about five fascinating facets of BWS in a Cornerstone blog.

Exploring CAR T-cell Therapy

Stephan Grupp, MD, PhD, director of the Cancer Immunotherapy Frontier Program and director of Translational Research for the Center for Childhood Cancer Research at CHOP, explained to ASH Clinical News how chimeric antigen receptor (CAR) T-cell therapy, an investigational immunotherapy approach of re-engineering immune cells and then infusing them back into a patient’s body to become targeted “cancer killers,” is used to treat relapsed pediatric leukemia. Dr. Grupp also discussed the challenges of safely managing the side effects of the therapy. CAR T-cell therapy continues on its road to approval from the FDA.

“There is nothing like that out there,” Dr. Grupp told ASH Clinical News.

Read the full article on the ASH Clinical News website.

Three Developers of Medical Devices for Children Receive Grants

A powered orthotic arm brace that amplifies weak nerve signals, a hand-operated rapid blood delivery system for emergency situations, and a device that gradually corrects deformed ears in babies are three devices under development selected by the Philadelphia Pediatric Medical Device Consortium (PPDC) to receive seed grants of $50,000 each.

Funded by the FDA and based at CHOP, the PPDC provides know-how and seed funding to help innovators translate promising ideas into commercial medical devices for use in children. The PPDC is a collaboration among CHOP, Drexel University, and the University of Pennsylvania. New this year, the PPDC partnered with the Childress Institute for Pediatric Trauma to fund a device that can be used during the so-called “golden hour of care” immediately following a traumatic injury.

“We are once again delighted to support promising, innovative medical devices geared to the unmet clinical needs of children,” said CHOP engineer Matthew R. Maltese, PhD, the PPDC’s executive director and principal investigator. He added that extra support dedicated to pediatric trauma allowed the PPDC to offer a third award in this round of grants. Get more details about the medical devices in the full press release. And read about previous seed grant recipients chosen in 2015 and 2016. The PPDC is currently reviewing proposals for its fourth round of funding opportunities, and it will announce the award recipients later this year.

CHOP Expert Signs Mitochondrial Medicine Research Agreement

Marni J. Falk, MD, an attending physician and director of the Mitochondrial-Genetic Disease Clinic at CHOP, is helping to develop novel treatment opportunities for people with mitochondrial disorders by evaluating compounds from NeuroVive’s research program, NVP015, in certain experimental disease models. NeuroVive, a pharmaceutical company based in Sweden, is committed to the discovery and development of medicines that preserve mitochondrial integrity and function.

The research team will study energy metabolism and disease development in models of mitochondrial complex I dysfunction. One of the most common causes of mitochondrial diseases relates to complex I dysfunction, which occurs when energy conversion in the first of the five protein complexes in the mitochondrion that are involved in effective energy conversion does not function normally.

“We feel fortunate to be able to initiate this collaboration with a well-recognized institution like CHOP and Dr. Marni Falk, a well-esteemed leader in the mitochondrial disease field,” stated NeuroVive’s CMO Magnus Hansson. “This is an important step in further advancing and strengthening our NVP015 program, as well as in the development of novel treatment opportunities for people with mitochondrial disorders.”

Read the full press release. Find out more in Bench to Bedside about Dr. Falk’s efforts to move toward getting evidence-based and approved therapies to patients.

ICYMI

In case you missed it, this week on Cornerstone, we got to know Brian Estevez, PhD, a new diversity scholar who is working in the lab of Mortimer Poncz, MD, chief of the division of Hematology at CHOP. “I interviewed all over the United States, and what I found that was different about CHOP is that we’re good at research, but we’re also good at a lot of other things,” Dr. Estevez said. “What enticed me about this place is the substructure of the program was very rich.”

And here are our top headlines from our Jan. 13 segment of In the News:

• Study Suggests New Intervention Offers Adult Patients Relief From Plastic Bronchitis
• CHOP Neonatologist Reflects on Rate of NICU Improvement
• Teen Driver Research Steering in the Right Direction

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