Category: Health

Latest Findings Add Insight Into Targeted Cancer Immunotherapy

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Originally published on Cornerstone, the CHOP Research Blog.

I composed this original article based on the presentation abstracts for the highlighted scientific presentation, followed by email contact with the investigators.

Excerpt:

Researchers at The Children’s Hospital of Philadelphia reported their latest results from their studies of an investigational personalized cell therapy for a highly aggressive form of acute lymphoblastic leukemia (ALL). Developed by researchers at the University of Pennsylvania and CHOP, the therapy is made from patients’ own immune T cells, which are extracted and bioengineered into CTL019 cells that potentially seek and destroy leukemia cells.

Among the findings, the team reported that 93 percent of pediatric patients reached remission after receiving the therapy for relapsed/refractory ALL. ALL is the most common childhood cancer, with limited effective treatment options for the approximately 10 to15 percent of patients who relapse after standard therapies.

The research team presented these results and more at the American Society of Hematology (ASH) annual meeting in Orlando. They reflect the CHOP-Penn team’s continued process of discovery about the investigational therapy, which is now part of clinical trials active at 15 sites globally, including CHOP.

Drugs May Be What’s the Matter With White Matter in HIV

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Originally published in Bench to Bedside, the CHOP Research monthly publication

I composed this original article based on an interview with the investigators.

Excerpt:

Some of the neurological and psychiatric complications associated with HIV may be side effects of the medications that control the virus, and not caused by the virus itself, according to a new study from researchers at The Children’s Hospital of Philadelphia and the University of Pennsylvania. Their pre-clinical findings were published in the Journal of Neuropathology and Experimental Neurology.

Certain antiretroviral drugs were associated with problems in developing myelin proteins in cell models and animal models, and the drugs were associated with reductions in white matter in autopsy brain samples from a cohort of individuals with HIV, reported the research team led by co-senior authors Judith Grinspan, PhD, research professor of Neurology at CHOP, and Kelly Jordan-Sciutto, PhD, chair and professor of Pathology at Penn’s School of Dental Medicine.

Both senior researchers emphasized that individuals with HIV should continue taking lifesaving antiretroviral drugs as prescribed. They hope their current and future findings can help researchers refine drug designs to reduce side effects, and help clinicians pursue prescribing practices that are risk-informed and tailored to the patient’s age and stage of brain development. These future changes could be particularly important for children with HIV whose brains are still developing.

Educational Intervention Decreases Mean Girls’ Relational Aggression

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Originally published in Bench to Bedside, the CHOP Research monthly publication

I edited this article based in part on a CHOP press release and in part on a blog post published by the investigator.

Excerpt:

The “Mean Girls” phenomenon is not just the subject of fiction. Relational aggression, such as using gossip and social exclusion to harm others, is all too common among preadolescent and adolescent girls. A new study from The Children’s Hospital of Philadelphia suggests that educational interventions including problem-solving skills and leadership opportunities can help, with lasting effects.

“As a psychologist and researcher with a particular interest in bullying, I am always interested in digging deeper into the ‘why,’” wrote Stephen Leff, PhD, in a blog post about the study, which he led. “Why is relational aggression — which involves the manipulation of social standing or reputations through gossip and social exclusion — so predominant among girls? Why is it associated with detrimental long-term outcomes for victims such as high levels of anxiety and depression? And, beyond the ‘why,’ how can we develop and test interventions that can combat this pervasive type of school violence, before it has a chance to become entrenched?”

The study, published in the journal Psychology of Violence, provides a partial answer to that last question.

New CHOP Study to Understand Risk of Hospital Readmission

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Originally published in Bench to Bedside, the CHOP Research monthly publication

I composed this original article based on an interview with the investigator.

Excerpt:

One in 20 infants is admitted to the hospital during the first year of life. As frightening as it may be for families to have a child whose health condition requires hospitalization, in too many cases the experience gets worse when a relapse or problem managing the condition after discharge means their child must be re-admitted later. Babies born prematurely are among the groups of children are at highest risk of hospital readmission.

With a new grant from the U.S. Department of Health and Human Services’ Agency for Healthcare Research and Quality (AHRQ), researchers at The Children’s Hospital of Philadelphia are working to reduce the need for pediatric readmissions by taking a population-level look at clinical factors, and an up-close look at familial and social factors, that send high-risk groups of children back to the hospital.

“The long-term goal is to take this information and develop real-time predictions,” said study leader Scott Lorch, MD, MSCE, director of the Center for Perinatal and Pediatric Health Disparities Research at CHOP and associate professor of pediatrics at the Perelman School of Medicine at the University of Pennsylvania. “We are looking for things that can be fed back to the clinical team to say, based on these factors, this patient has a higher than usual risk of readmission.”

Stroke Rehabilitation Research Connects Brain to Gait

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Originally published in Bench to Bedside, the CHOP Research monthly publication

I composed this original article based on an interview with the investigator.

Excerpt:

If you have ever hit your stride on a moving walkway, the type commonly found in airports, consider how it felt when you stepped back onto solid ground. You may have felt a sudden but brief discombobulation while your brain worked to correct its temporary mismatch with your body’s sense of movement. Laura Prosser, PhD, PT, is trying to induce a similar reaction to rehabilitate children after stroke.

As a research scientist in the Division of Rehabilitation Medicine at The Children’s Hospital of Philadelphia, Dr. Prosser’s work is focused on how the brain and its connection to the body change after damage and during rehabilitation. Her focus on children addresses an under-researched area in rehabilitation.

“Understanding how rehabilitation can impact neuroplasticity is the most exciting aspect of this research to me,” Dr. Prosser said. “Not much of this work has been done in children. At CHOP we are in a unique position to understand how the brains of children respond differently to rehabilitation than the brains of adults who have had an injury.”

Dr. Prosser is now conducting a small pilot study testing physical therapy outcomes after pediatric stroke using high-tech tools including a split-belt treadmill and brain-stimulating technology called transcranial magnetic stimulation (TMS). She aims to learn which approaches seem most promising to pursue in future larger trials.

Why Kids Need Bug Squashers: An Infectious Disease Q&A

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Originally published on Cornerstone, the CHOP Research Blog

I conducted this interview, transcribed and edited the Q&A, and wrote the introduction.

Excerpt:

Pediatric infectious disease specialists, the doctors who specialize in wiping out microbial and other infections in children, are a dedicated bunch. These physicians work at the front lines of diseases new and old, as well as protecting against complications from other medical interventions.

Last month, members of this profession honored Theoklis E. Zaoutis, MD, MSCE, chief of the Division of Infectious Diseases at The Children’s Hospital of Philadelphia, as an exemplary leader in their midst with the Distinguished Service Award from the Pediatric Infectious Diseases Society (PIDS). This award, presented at the Society’s annual IDWeek meeting, recognizes a member who has made an outstanding contribution to the specialty of pediatric infectious diseases.

On the occasion of this recognition, Cornerstone sat down with Dr. Zaoutis, who is also director of the Center for Pediatric Clinical Effectiveness Research at CHOP and Thomas Frederick McNair Scott Professor of pediatrics and professor of epidemiology at the Perelman School of Medicine at the University of Pennsylvania. We wanted to know what is hot in the field of pediatric infectious disease research and what lies ahead. Read on for the edited conversation.

Teens Receive Inconsistent Emergency Care After Sexual Assault

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Originally published on Cornerstone, the CHOP Research Blog

I composed this original article based on an interview with the investigator.

Excerpt:

About 10 percent of high school girls and half as many high school boys report that they have been sexually assaulted in Centers for Disease Control and Prevention (CDC) surveys. But when these young victims come to pediatric emergency rooms,, they are not consistently getting recommended tests and treatment, according to new research from The Children’s Hospital of Philadelphia.

“Our study was remarkable in that there was so much variation in the care adolescents received after sexual assault,” said Samantha Schilling, MD, MSHP, an assistant clinical professor at the University of North Carolina School of Medicine who is first author of the study, conducted when she was a fellow at CHOP and at the Leonard Davis Institute of Health Economics at the University of Pennsylvania (LDI). “While overall performance wasn’t ideal, there were also wide ranges in testing and treatment for infections and pregnancy.”

On the Trail of a Cancer Predisposition Syndrome

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Originally published in Bench to Bedside, the CHOP Research monthly publication

I composed this original article and complementary blog post based on interviews with the investigator and two parents of children with this syndrome.

Excerpt:

Inside the cells of a developing human embryo is a little piece of “Alice in Wonderland.” While most of our bodies’ genes are expressed from both our mothers’ and fathers’ chromosomes, there is a particular growth-regulating region of chromosome 11 where Dad’s genes make you grow bigger, and Mom’s genes make you small. As in Alice’s adventure, there is potential for some difficult situations to occur when that growth process is not handled with exacting care.

With new grants awarded by the St. Baldrick’s Foundation and the National Cancer Institute, attending physician and geneticist Jennifer M. Kalish, MD, PhD, at The Children’s Hospital of Philadelphia, is going down the rabbit hole to try to set things right. She 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.

Five Fascinating Facets of Beckwith-Wiedemann Syndrome

Originally published on Cornerstone, the CHOP Research Blog

I composed this blog post as a complement to the above article about the new investigation into BWS.

Excerpt:

1. BWS is a mosaic condition. Some cells and organs or limbs grow unusually large in children with BWS, and some do not. Patterns of this overgrowth vary from child to child. This mosaicism happens because the changes in gene expression that cause BWS arise early when the developing human embryo has relatively few cells — and the changes occur in only some of them. Cells and organs descended from those dysregulated cells have unusual growth patterns, while cells and organs descended from normally developing cells continue to grow at normal rates. This results in a widely varying presentation of the syndrome in different children.

The mosaic nature of the condition makes it challenging to manage the elevated cancer risk that goes along with BWS. As Dr. Kalish noted, “I can see if a child’s arms are bigger. I cannot see what is going on in the liver or kidney.” Each child’s areas of overgrowth are variable, so all young children with BWS must undergo regular cancer screenings.

Cornering a Cancer-Connected Autoimmune Disease

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Originally published in Bench to Bedside, the CHOP Research monthly publication

I composed this original article and related behind-the-science human interest blog post based on interviews with the investigators.

Excerpt:

It is certainly not good news for children to get a double whammy of both cancer and autoimmune disease. Unfortunately, for a small subset of children with neuroblastoma, a common childhood cancer of the peripheral nervous system, an extremely rare autoimmune disorder called OpsoclonusMyoclonus Ataxia Syndrome (OMAS) comes along for the ride. The overactive immune response is believed to be triggered by the cancer.

But there is a twist.

“Patients with neuroblastoma who have OMAS have better outcomes, in terms of their tumor, than patients with neuroblastoma who don’t have OMAS,” said Jessica Panzer, MD, PhD, a pediatric neurologist and attending physician at The Children’s Hospital of Philadelphia who is studying this disease.

That pattern leads Dr. Panzer and other researchers to wonder: Is it possible that OMAS is a case of the body’s immune system finding a successful defense against cancer (but taking it a little too far against healthy cells)? And could we learn safe ways to harness its ability to help more children with neuroblastoma, or even other cancers?

These are among many long-term questions on the distant horizon for researchers who study this little-understood autoimmune disease. First, they need to understand the basics.

Dancing Eyes Brought a Research Team Together

Originally published on Cornerstone, the CHOP Research Blog

I composed this story as a complement to the above article highlighting the science of this team’s collaboration.

Excerpt:

It started at the end of a long day. Jessica Panzer, MD, PhD, then just a few weeks into her pediatric neurology residency at The Children’s Hospital of Philadelphia, was about to go home. Instead, she was called to the emergency room to consult on a 3-year-old girl who could barely walk. What happened then opened up new questions in her budding research career.

Not long after that, Miriam Rosenberg, PhD, started on a convergent path when her own 19-month-old daughter got sick. The toddler first developed problems with excessive drooling and stumbling while she walked. Within a few months, she had a sudden onset of more severe symptoms — unable to walk, severe tremor, unable to feed herself. Dr. Rosenberg and her husband brought their child to the nearest hospital.

CHOP-led Consortium Focuses on Patient-Reported Outcomes

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Originally published in Bench to Bedside, the CHOP Research monthly publication

I composed this original article based on an interview with the investigators.

Excerpt:

“How tired do you feel?” a doctor asks a child with a chronic disease. Or, “How well are you managing stress?”

The answers to questions like these are even more important, from many patients’ and families’ perspectives, than the particular numerical result of their lab test results.

But the answers are less useful to doctors than they could be. Doctors do not have validated tools to use such patient-reported outcomes to track progress managing a condition over time in the same way they can compare results of blood tests over time. In clinical research, they are unable to compare the answers across patients to ultimately show an experimental drug meaningfully improves fatigue or other patient-reported measures.

“Our vision is that patient-reported outcomes become like lab tests,” said Christopher Forrest, MD, PhD, a pediatrician and researcher at The Children’s Hospital of Philadelphia and professor of pediatrics at the Perelman School of Medicine at the University of Pennsylvania. “Soon doctors will use patient-reported outcomes to monitor patients’ clinical care in the same way they use lab tests or X-rays.”

Dr. Forrest and colleagues at CHOP and partner institutions received a new grant from the National Institutes of Health to advance the science of patient-reported outcome measures to one day achieve that vision.