Nursing Research Recognition, CAR T-Cell Therapy, Stand Up to Cancer Grant, Neuropsychiatric Disorders in Lupus, Newly Named Genetic Disorder

“Bold” is perhaps the best word to describe this week’s roundup of Research Institute news, as Children’s Hospital of Philadelphia nurses and investigators alike made headlines for their experimental work in pediatric CAR T-cell therapy: a cancer-fighting immunotherapy treatment that aims to re-engineer a patient’s own T cells to detect and destroy tumor cells. Read on to learn more about this, and other recent stories, in our quest to improve children’s health.

Clinical Journal of Oncology (CJON) Features CAR T-Cell Contributions From CHOP Nurses

Five nurse practitioners in CHOP’s oncology department contributed their knowledge in pediatric care coordination for CAR T-cell therapy to the scientific journal, Clinical Journal of Oncology this month. The journal’s April 2017 supplement edition focuses specifically on immunotherapy in cancer care and contains five papers with the writing and contributions of Colleen Callahan, MSN, CRNP; Diane Baniewicz, MSN, CRNP; Laura T. Smith, MSN, CRNP; Kimberly Venella, MSN, CRNP; and Beth Ely, RN, PhD. The CHOP nurses were asked to participate in the pediatric section of the supplement by Sloan Kettering and MD Anderson Hospitals because of our institution’s reputation as a leading investigator into CAR T-cell therapy in children.

Some of their topics included inpatient nursing care for patients who develop Cytokine Release Syndrome (a frequent complication of the therapy), and the care coordination of CAR T-cell therapy for children with relapsed or refractory acute lymphoblastic leukemia (ALL). This is the first time that pediatric nurses have discussed the clinical care of children who receive CAR T-cell treatment in a scientific paper. You can find the CJON supplement available online.

David Barrett Receives Grant to Further Explore CAR T-Cell Therapy

Nurses weren’t the only ones to make headlines about the novel cancer treatment this week: David M. Barrett, MD, PhD, a physician and immuno-oncology researcher at CHOP, received an Innovation Research Grant of $750,000 from Stand Up to Cancer (SU2C) — a  charitable organization that raises funds for accelerating cancer research — to further explore CAR T-cell therapy.

The Innovative Research Grant is given to research projects that are “high-risk” but “high-impact,” with the potential to save lives, according to SU2C. Dr. Barrett will investigate ways to help more children with ALL successfully respond to the treatment. Currently, ALL is one of the most common childhood cancers, and it occurs when the body produces too many immature white blood cells that lack the power to fight bacteria and viruses in the body. SU2C announced the exciting award at the 2017 Annual Meeting of the American Association for Cancer Research. Read more on the SU2C website.

Novartis Announces Pediatric CAR T-Cell Therapy, CTL019, is Under FDA Priority Review

In a press release last week, Basel-based pharmaceutical company Novartis announced that the Food and Drug Administration (FDA) has placed their Biologics License Application for CTL019 — a CAR T-cell therapy for pediatric and young adult patients with B-cell acute lymphoblastic leukemia — under priority review. This designation means that the FDA will expedite their standard process for reviewing and approving the drug, which is targeted to pediatric patients whose ALL is relapsed and refractory (a condition known as r/r ALL). Novartis’ application for CTL019 includes data from two CHOP-led studies that explored the safety and efficacy of the therapy in pediatric and young adult ALL patients, with Stephan Grupp, MD, director of the Cancer Immunotherapy program at CHOP, as lead investigator.

In a Novartis press release, Dr. Grupp made a statement: "Even if a patient has difficult-to-treat relapsed/refractory leukemia, we have seen treatment with CTL019 in clinical trials put cancer into remission. This could be a first-of-its-kind treatment with exciting potential to help pediatric and young adult r/r B-cell ALL patients."

New Grant Helps Researchers Study Neuropsychiatric Symptoms of Lupus

Besides its more obvious external symptoms like rashes and swelling, the autoimmune disease, lupus, also affects the brain. Up to 50 percent of children with lupus experience neuropsychiatric problems like depression or anxiety in conjunction with their diagnosis. Now, a new Novel Research Grant from the Lupus Research Alliance will allow Andrea Knight MD, a physician in the division of Rheumatology at CHOP, to develop a new biomarker to predict neuropsychiatric conditions in children with lupus. These biomarkers will help physicians to intervene and treat brain health in a timely and effective way. The Lupus Research Alliance offers Novel Research Grants to 10 projects that they believe “represent innovative approaches in lupus-treatment development.” We previously reported on Dr. Knight’s research on the mental health aspect of lupus when she received a Clinical Futures Pilot Grant Award in 2014. Read more about Dr. Knight’s new grant in the press release.

Barbara Medoff-Cooper Receives ENRS Nursing Research Award

Barbara Medoff-Cooper, RN, PhD, a professor of Nursing at the University of Pennsylvania School of Nursing and Ruth M. Colket Professor in Pediatric Nursing at CHOP, has studied how babies eat, babble, and behave for decades. A resident expert on infant development and care, Dr. Medoff-Cooper was recently announced as the Eastern Nursing Research Society (ENRS)’s selection for their Distinguished Contributions to Nursing Research Award for 2017. It is a title bestowed on a senior investigator each year for their contributions to the field, and the ENRS will officially award it at their annual conference April 7. Dr. Medoff-Cooper’s work is recognized globally, most especially two of her innovations: the Infant Temperament Questionnaire, an assessment that helps to treat difficult infants under 4 months old, and the NeoNur, a feeding device that allows physicians to detect developmental problems based on how an infant feeds. Read more in the press release.

New Genetic Disorder Named After CHOP Scientists

Finally, here’s one story to tell the grandkids: Three CHOP scientists have gone down in medical history after a new genetic disorder was named in their honor, thanks to their dedicated research in children with unexplained growth failure. Mulchandani-Bhoj-Conlin syndrome (MBCS) is named after Surabhi Mulchandani, MS, manager of the Genomics Diagnostics Laboratory in the division of Genomics Diagnostics; Elizabeth Bhoj, MD, a clinician-researcher in the division of Human Genetics; and Laura Conlin, PhD, director of the Genomics Diagnostics Laboratory.

MBCS is an imprinting disorder likely caused by an abnormality of chromosome 20 (in which a child doesn’t inherit a copy of the chromosome from their father), and it is characterized by early growth failure that is treatable with growth hormones. In the years leading up to the disorder’s definition, Mulchandani, Bhoj, and Conlin studied a group of children who shared mostly similar symptoms, including their extremely small size as infants and severe feeding problems. The researchers’ work — in conjunction with clinicians from several other institutions — was published in Genetics in Medicine before being recognized by the Online Mendelian Inheritance in Man (OMIM), an online authority for genetic diseases. Read more in the press release.

ICYMI

On Cornerstone, we debuted our first Snapshot Science post, Do Teen Mothers In Vulnerable Populations Need Better Health Services?, as part of a series that will summarize exciting new research going on at the Research Institute. We also learned how the Center for Childhood Cancer Research Biorepository is a unique and potent resource for pediatric oncology researchers at CHOP.

And check out our top headlines from our March 24 segment of In the News:

• PolicyLab Speaks Out on Vaccine Hesitancy
• Eight-year-old Neuroblastoma Survivor Reflects on Cancer Journey
• Medicaid Reforms Should Address Children With Complex Conditions
• CHOP Fellow Receives 2017 Richard King Trainee Award
• CHOP Ranked Best Pediatric Program in Natio

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