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In the News: Clinical Research in the Spotlight

Published on
Jun 10, 2016

We like to highlight the whole range of research at The Children’s Hospital of Philadelphia, from basic discoveries at the lab bench to clinical findings applied at the patient’s bedside. But we have an abundance of updates to share today about the clinical side of research. So slide your chair away from the bench and over to the virtual patient bedside to check out these stories in our weekly news roundup.

Clinical Cancer Research Including T Cell Therapy Presented at ASCO

This week, the clinical cancer research community converged at the 2016 American Society of Clinical Oncology (ASCO) Annual Meeting. Among the highlights of presentations led by researchers at CHOP:

Shannon Maude, MD, PhD, a pediatric oncologist at CHOP and assistant professor of Pediatrics at the Perelman School of Medicine at Penn, led two studies with CHOP and Penn colleagues on targeted immunotherapy using bioengineered T cells to treat children with aggressive acute lymphoblastic leukemia (ALL). One study updated outcomes and longer follow-up results from a previously reported clinical trial of 59 children with relapsed or refractory ALL. The other described a clinical trial in children with ALL who relapsed after their first T cell treatment, and were subsequently treated with T cells engineered to carry a “humanized” protein more similar to human proteins than to the murine (mouse) protein used in the first treatment. Other CHOP investigators on the team include David Teachey, MD; Susan Rheingold, MD; Richard Aplenc, MD, PhD, MSCE; David Barrett, MD, PhD; Christine Barker; Colleen Callahan, RN, MSN, CNRP; and Stephan Grupp, MD, PhD.

In another presentation at ASCO, a team from the multicenter Children’s Oncology Group (COG) reported on combination therapies for relapsed or refractory neuroblastoma. Rochelle Bagatell, MD, was the senior author of this report on a Phase 2 randomized clinical trial, which led researchers to recommend investigating the combination of chemotherapy and immunotherapy they studied in a larger number of patients.

In addition, Frank M. Balis, MD, presented a poster from the COG Chemotherapy Standardization Task Force describing a new, uniform, table-based method of dosing anticancer drugs in infants that will be used in ongoing and future COG clinical trials. The advantages of this new infant dosing method include less complexity, improved safety, and elimination of the need for dose calculations. Read more about the promise of immunotherapies for childhood cancer in a blog post perspective by Dr. Barrett in’s cancer blog.

Read more about T Cell therapy presentations by Penn and CHOP researchers at ASCO in a Penn Medicine news release. Read more about all of the above highlights of CHOP’s presentations at ASCO in a CHOP news release.

No Cognitive Impact from Early Anesthesia in Healthy Children

A new study published in the Journal of the American Medical Association this week suggests that children who receive a single dose of anesthesia early in life don’t face an elevated risk of later cognitive problems. CHOP was one of four sites in the multi-site PANDA (Pediatric Anesthesia Neurodevelopment Assessment) study, the largest and most comprehensive of its kind. Researchers compared cognitive abilities of children who had been exposed to anesthesia early in life to unexposed siblings, later in childhood.

“A number of animal studies have suggested that exposure to commonly used anesthetic agents in early development could lead to deficits in learning, memory, attention, and other cognitive functions," said lead author and pediatric anesthesiologist Lena S. Sun, MD, of Columbia University and NewYork-Presbyterian/Morgan Stanley Children's Hospital, in a press release. "However, few clinical studies have adequately addressed whether this is also true in humans. Based on our findings, we can reassure parents that one exposure to anesthesia is safe for healthy young children.”

The study team noted that they need more research on the effects of anesthesia exposure that is prolonged or repeated or provided to children in more vulnerable subgroups, as well as on exposure to girls, who were underrepresented in the study sample’s exposure group.

Jerilynn Radcliffe, PhD, a psychologist at CHOP, and Lynne Maxwell, MD, associate director of the division of anesthesia and critical care medicine at CHOP and associate professor of Anesthesiology and Critical Care Medicine, co-authored the study.

Read more in the Columbia press release.

Golden Age for Clinical Trial Recruitment

In order for clinical research discoveries to happen, the voluntary participation of pediatric patients and their families is vital. This week, the Research in Action blog from CHOP’s Center for Injury Research and Prevention hosted a guest blog post from Chris Gantz, MBA, program manager of the Recruitment Enhancement Core in the Research Institute. Gantz highlighted both the opportunities of new technologies to improve the process of recruiting families to participate in trials, as well as some of the challenges and reasons that may prevent some families from participating.

Read more in Gantz’s post, and look for a possible follow-up post here on Cornerstone in the future. We’ll also bring you more stories behind the scenes of clinical trial participation in the coming months.


In case you missed it, earlier this week on Cornerstone we brought you a whole lot of good news among the highlights from the latest issue of our CHOP research newsmagazine, Bench to Bedside.

Last week’s In the News post covered a major study showing youth concussions may be undercounted in official statistics, a genetic finding linked to autism, new projects for child injury prevention research, and raising awareness about gun violence prevention.

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