If you’re looking for a spark of inspiration during January’s long and sometimes dreary days, don’t miss this week’s roundup of headlines from in and around Children’s Hospital of Philadelphia Research Institute. Our scientists’ passionate work in the lab found a spotlight in the mainstream media as “TODAY” featured how our stem cell research can help today’s cancer survivors become tomorrow’s parents. Meanwhile, eye-opening findings from the Lifespan Brain Institute (LiBi) sparked a wider conversation about how pediatricians and parents can stay alert for suicidal thoughts in teens. In more news, a recent study highlighted the need for more antibiotic stewardship in non-children’s hospitals, while a successful device consortium based at CHOP officially became a statewide affair.
Philadelphia Pediatric Device Consortium Expands to Span State of Pennsylvania
The Pennsylvania Pediatric Medical Device Consortium (PPDC), a network that provides support and seed funding to innovators with promising new technologies, is stretching its reach to become a state-wide collaborative effort by partnering with two programs at the University of Pittsburgh. The McGowan Institute for Regenerative Medicine and sciVelo, both based at the University of Pittsburgh, join a multi-institute organization that includes Drexel University, the University of Pennsylvania, and CHOP, where it is headquartered. Now re-named the Pennsylvania Pediatric Device Consortium, the PPDC expansion follows the receipt of a five-year, $5 million grant renewal from its sponsor, the U.S. Food and Drug Administration.
“Our new partnerships with these outstanding programs build on our existing success in collaborating with Drexel University and the University of Pennsylvania,” said Matthew Maltese, PhD, director of Biomechanics Research in the Department of Anesthesiology and Critical Care Medicine at CHOP, and the executive director and principal investigator of the PPDC. “This unification of Pennsylvania’s biomedical ecosystem expands the PPDC’s network of expertise in supporting the development of much-needed devices for children.”
Since 2015, the PPDC has provided seed funding for a variety of pediatric products, from an airway clearance system, to a powered arm brace, to a vision acuity test for preverbal children, and much more.
Read more about the expanded PPDC in the press release.
Evaluating Humanized CAR T-cell Therapy in Patients With Down Syndrome
A recent Cancer Connections story on features our cancer researchers’ impactful work investigating the use of chimeric antigen receptor (CAR) T-cell therapy for children with Down syndrome who also have acute lymphoblastic leukemia (ALL). Children with Down syndrome have an elevated risk of developing ALL and f experience higher frequency of toxicity with chemotherapy alongside generally poorer outcomes.
The story highlights the journey of Steven, a 21-year-old patient from Toronto, Canada, who has Down syndrome and was diagnosed with B-Cell ALL (B-ALL) at 4 years old. After his first relapse in 2015, Steven enrolled in a clinical trial within our Cancer Immunotherapy Program led by Shannon Maude, MD, PhD, attending physician in the Cancer Center at CHOP. Dr. Maude led the trial to evaluate an alternative, humanized CD19 CAR T-product called huCART19 or CTL119. More than a year and a half after receiving CAR T-cell therapy in 2016, Steven’s mother reports that he is thriving and living “an active and fulfilling life.” Steven’s story provides a hopeful background to our Cancer Immunotherapy Program’s current efforts to develop a clinical trial of CAR T-cell therapy for children with Down syndrome and B-ALL in first relapse.
Read the full inspirational story on chop.edu.
CHOP Study Makes Waves After Reporting Many Parents Unaware of Teen Suicidal Thoughts
A recent study of more than 5,000 adolescents from researchers at the Lifespan Brain Institute (LiBi) of Children’s Hospital of Philadelphia and the University of Pennsylvania made mainstream media headlines for sharing new insights into teen suicidal thoughts. In Pediatrics, the researchers reported that half of parents don’t know their adolescent has had thoughts about suicide, and more than 75 percent of parents are unaware their adolescent harbors thoughts of death and dying. Coverage of the study by philly.com highlighted the importance of parents listening to and conversing with their teens and speaking to local community advocates and other parents. Meanwhile, American Academy of Pediatrics (AAP) Gateway used the findings as a pivot to discuss ways in which pediatricians can repair the discrepancies between parent unawareness and teen denial. Reuters, the Huffington Post, Medscape, and a handful of other online and print news outlets also covered the study.
Read more about the study in the press release.
‘Today’ Show Video Features CHOP/Penn Research Into Future Fertility for Boys With Cancer
A recent video on “NBC TODAY” featured the exciting research breakthrough of Jill Ginsberg, MD, a pediatric oncologist, and Thomas Kolon, MD, a pediatric urologist, as they work to improve the future fertility of childhood cancer survivors in collaboration with scientists at Penn Medicine.
“A recent breakthrough specific to boys who haven’t hit puberty yet could help today’s cancer survivors become tomorrow’s fathers,” said NBC reporter Kate Snow in the video.
Many boys who undergo cancer treatments before reaching puberty are left infertile and currently have no existing options for fertility preservation. But in a Nature Communications study published in November, Drs. Ginsberg, Kolon, and their colleagues discovered by using human sperm-producing stem cells (SSCs) in animal models, endothelial cells from the testes are a viable environment for human SSCs to expand. The researchers tested their findings and showed that infertile male mice were able to produce sperm.
In the “TODAY” video, Snow speaks with Dr. Ginsberg on why those findings were a game changer in their quest to preserve and expand the sperm-forming cells of boys with cancer.
“The roadblock to date has always been how to expand these sperm-forming cells in the lab, and that’s the step we think we’ve overcome now,” Dr. Ginsberg explained.
Watch the full “Today” video.
Children’s Hospitals More Likely to Give Recommended Antibiotics for Pneumonia
In a new study published online in JAMA Pediatrics, researchers in our Division of Infectious Diseases in collaboration with the University of Michigan reported that children with pneumonia may be more likely to receive recommended antibiotics when treated at a children’s hospital compared to anywhere else. The findings highlight the need for hospitals that don’t specialize in treating children to introduce more programs focused on antibiotic stewardship.
“This is one example of a scenario where, in general, children who are cared for in children’s hospitals appear to be receiving more guideline-recommended care than those who are not,” said Jeffrey Gerber, MD, PhD, attending physician in the Division of Infectious Diseases at CHOP and the study’s senior author.
To provide some background, narrow-spectrum antibiotics (penicillin, amoxicillin, and ampicillin) are recommended by U.S. guidelines for more serious cases of pneumonia, such as those that can lead to fatal lung infections. Compared to broad-spectrum antibiotics, narrow-spectrum antibiotics are recommended because they provide similar clinical cure rates but are less likely to cause antibiotic resistance and side effects. Narrow-spectrum antibiotics are also less expensive. In a JAMA paper published by Dr. Gerber and colleagues in 2017, researchers found that children who received narrow-spectrum antibiotics had a higher health-related quality of life and a reduced risk for side effects as compared to those receiving broad-spectrum antibiotics.
Mark Your Calendars: CAR Hosts SPARK for Autism Family Day in February
Our Center for Autism Research (CAR) at CHOP announced it will host another fun family research day Feb. 23 in partnership with We Rock the Spectrum in Audubon, NJ. The event promotes participation in SPARK for Autism research by inviting individuals with autism spectrum disorder (ASD) and their families to a day of fun, sensory-friendly activities where they can gather resources and participate in the SPARK research study. SPARK (Simons Powering Autism Research) is national research study that seeks to understand more about the influence genetics has in the development of autism.
Learn more about the event and register.
Catch up on our headlines from our Jan.11 In the News:
- FDA Approves Dasatinib Tablets in Combination with Chemotherapy
- Study Team Evaluates Video Game as Investigational Therapy for Autistic Children
- CHOP Researchers Call for Improved Pediatric Palliative Care Measures
- Study of Variation in Care Published in ‘Inflammatory Bowel Diseases’
- CHOP Adds Six New Frontier Programs
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