Major External Grant Awards

Richard Finkel, MD, division chief of neurology and a specialist in neuromuscular disorders at Nemours Children’s Hospital (NCH) in Orlando, is one of a handful of pediatric researchers selected for the first targeted drug trial ever conducted for spinal muscular atrophy (SMA).

NCH, which opened in October 2012, is one of four participating hospitals worldwide and serves as the medical home base for two of the trial’s eight participants. The project is the hospital’s first inpatient clinical trial for a neuromuscular disease and is expected to last one year.

The COBRE grant is part of the NIH IDeA program, which has led to the development of the Center for Pediatric Research (CPR): Program Director; Thomas H. Shaffer, MS.E., PhD and Program Coordinator; Robert Mason, PhD). 

The CPR program (now up to almost $25 million; Phase I, ARRA and Phase II) was designed to enhance the infrastructure for research in Delaware. Phase I funding initially allowed Nemours  to upgrade our equipment, further develop our core facilities, establish mentoring programs, recruit research faculty and to become a key member of a local consortium of researchers in the Delaware Health Sciences Alliance (DHSA).

This expansion of infrastructure significantly increased the scientific output and national reputation of Nemours, and in the first phase led to new NIH R01 grants to two of the Center’s target investigators, Grace Hobson, PhD, and Julia Barthold, MD. These successes led to 3 ARRA Supplements and a COBRE Phase II, second phase award to bring in new investigators (Thierry Morlet, PhD; Kamin Johnson, PhD; Soonmoon Yoo, PhD; Anne-Marie Brescia, MD and Mark Ma, PhD), recruit established research faculty and to more firmly establish the center.

As the CPR has become established, we have begun to broaden its reach by providing junior investigators pilot funding for novel projects. It is particularly important that we develop a track record in sponsoring successful pilot projects because the third and final five-year phase of COBRE support is designed to consolidate core facilities and support only pilot programs as transitional and target investigators from COBRE Phase II graduate from the training program to lead their independent programs with institutional and/or alternative sponsored support. As for COBRE Phase I, graduating faculty continue to be members of the CPR, with access to core support and mentoring programs.

In our first and second round of pilot grant applications, our review panel identified the following pilot projects to support:

• Jobayer Hossain, PhD, who used our electronic medical records (EMR) to examine early childhood body mass index (BMI) trajectories as a potential predictor of obesity.
• Valerie Sampson, PhD, was funded for a pilot project to study the effect of the insulin-like growth factor-1 receptor (IGF-1R) inhibition on epidermal growth factor receptor (EGFR) expression in osteosarcoma (OS) and Ewing sarcoma (ES).
• Stephan Lindsey, PhD, developed a very interesting pilot project to design hydrogels to help delivery and stabilization of stem cells for therapy of spinal cord injury.
• Alfred Atanda, MD, is an orthopedic surgeon and has developed a project to study changes in the ulnar collateral ligament (UCL) in children who are baseball pitchers.

To help with research into applied behavior analysis therapy, the Martin and Gracia Andersen Foundation donated $70,000 to the Division of Behavioral Health at Nemours Children’s Hospital.

The gift will allow the hospital to establish a multidisciplinary autism early intervention program. Leslie Gavin, PhD, associate chief of the Division of Behavioral Health, will lead the project.

THE PLAYERS Center for Child Health at Wolfson Children’s Hospital in Jacksonville, Fla., awarded nearly $270,000 to Nemours researchers to study the feasibility and acceptability of a comprehensive measurement and intervention protocol that would be used for future trials of interventions targeting obese children. he pilot study, involving obese children ages 8–11 years old, lasted through Mar. 31, 2013. Participating Nemours investigators include J. Atilio Canas, MD (PI) and Amanda S. Lochrie, PhD, ABPP (PI), Babu Balagopal, PhD, and Robert Mason, PhD.

Preliminary work provided data for investigators to design and conduct a family-based lifestyle intervention program. The program sought to change the lifestyle behaviors of obese children and their families in order for them to adopt a healthier way of life and lose weight. It was highly focused on the physical aspects of the children, who were required to wear a Sensewear armband accelerometer that provided an assessment of total physical activity energy expenditure. The participants took part in different forms of therapy, life coaching and educational programs to assist in durable behavior change.

The interventions targeted participants before their conditions progress to serious medical comorbidities such as hypertension, dyslipidemia or type 2 diabetes, and featured active involvement of families. The study also investigated the relationship between carotenoid (beta carotene supplements) consumption and insulin resistance through fatty-acid metabolism to determine whether supplementation leads to significant changes that correlate with improved insulin sensitivity and lower abdominal fat.

A 3-year, $100,000 grant from the Nancy Taylor Foundation for Chronic Diseases will support research to identify prognostic biomarkers in the synovial fluid of patients with juvenile idiopathic arthritis (JIA). The grant went to AnneMarie Brescia, MD, principal investigator at the Translational Rheumatology Laboratory based at Nemours/Alfred I. duPont Hospital for Children in Wilmington, Del., where she’s also a pediatric rheumatologist.

The lab received additional gifts of $10,000 from the Open Net Foundation and $20,000 from an anonymous donor through the Delaware Community Foundation to support this work.

A $60,000 grant from the Pediatric Cancer Foundation, Moffitt Cancer Center Sunshine Project will support a research project titled, “Combination Testing of the Tubulin Binding Agent, Eribulin, with Conventional Chemotherapeutic Agents in In Vitro and In Vivo Models of Pediatric Osteosarcoma.” The project involves the preclinical testing of a promising chemotherapeutic drug (eribulin) in a pediatric model of osteosarcoma.

The study will investigate the effectiveness of eribulin in combination with irinotecan and ternozolomide (both are existing chemotherapeutic agents) and compare results against single-agent therapies in osteosarcoma cell lines and xenografts. The objective of the project is to provide strong biological rationale to inform decisions for the evaluation of eribulin in early-phase clinical trials in pediatric patients with osteosarcoma.

The project is headed by E. Anders (Andy) Kolb, MD, director of the Nemours Center for Cancer and Blood Disorders and head of the Cancer Therapeutics Laboratory, and Valerie Sampson, PhD, research associate in the Cancer Therapeutics Laboratory.

The Center for Medicare & Medicaid Innovation recently awarded a 3-year, $3.6 million Health Care Innovation Challenge grant to Nemours Health & Prevention Services (NHPS) to support its “Optimizing Health Outcomes for Children with Asthma in Delaware” project.

Led by NHPS Executive Director Mary Kate Mouser, the project aims to reduce asthma-related pediatric emergency department visits and hospitalizations in Delaware by 50 percent. The work will focus on improving asthma outcomes and health for Medicaid beneficiaries in selected areas.

The project trains Nemours Associates in both community and clinic settings to create close relationships between existing primary care practices and the surrounding communities.

Shunji Tomatsu, MD, PhD, director of the Skeletal Dysplasia Laboratory in the Nemours Center for Orthopedic Research and Development (CORD), received a 2-year, $80,000 grant from the National MPS (Mucopolysaccharidoses) Society.

The grant supports Dr. Tomatsu’s project titled, “Development of Long Circulating Enzyme Replacement Therapy for MPS IVA.” Mucopolysaccharidosis type IVA (or MPS IVA, also known as Morquio syndrome) is a lysosomal storage disorder in which patients develop skeletal abnormalities such as short stature, bone deformities and spinal cord compression.

The abnormalities occur because patients lack an enzyme that breaks down deposits of sugar carbohydrate chains called glucosaminoglycans (GAGs). The enzyme replacement therapy could prove useful in correcting or improving bone pathology associated with the condition.

Hyundai Hope On Wheels and Wilmington, Del.,-area Hyundai dealers have awarded Nemours/Alfred I. duPont Hospital for Children a $75,000 Hyundai Scholar Grant.

The grant was presented to Emi H. Caywood, MD, a pediatric hematologist/oncologist at the hospital, to fund her work in developing a research infrastructure to enhance the accuracy and completeness of data used in clinical trials and improve data management and best practices across four Nemours clinical sites (all part of the Nemours Center for Cancer and Blood Disorders).

Hyundai Scholars pursue research and clinical programs aimed at improving the lives of children battling pediatric cancer.

Tim Wysocki, PhD, ABPP, co-director of the Nemours Center for Healthcare Delivery Science and a pediatric psychologist at Nemours Children's Specialty Care, Jacksonville, Fla., was awarded a 3-year, $1.5-million grant from the Patient-Centered Outcomes Research Institute (PCORI) for his project titled, “Shared Medical Decision Making in Pediatric Diabetes.”

The grant was among the 25 funded applications (out of 688 submitted) for PCORI’s first-ever funding cycle. The project will determine if a shared medical decision-making intervention can promote beneficial metabolic and behavioral outcomes among adolescents with type 1 diabetes who are candidates for incorporating certain technological advances into their care.

Diane J. Abatemarco, PhD, MSW, a researcher at Nemours/Alfred I. duPont Hospital for Children, has received a 4-year, $1.7-million grant entitled “Comprehensive Support Services for Families Affected by Substance Abuse and/or HIV/AIDS” from the Department of Health and Human Services Administration for Children and Families.

The project combines enhanced safety practices among pediatricians and enhanced family-centered programs that promote parenting skills, child development and family unity.

The Leukemia Research Foundation of Delaware, Inc. made a $1-million donation to the Nemours Center for Cancer and Blood Disorders (NCCBD) at Nemours/Alfred I. duPont Hospital for Children. The foundation funds blood cancer research while advocating for advanced therapies and care; its donation will support pediatric cancer research at the center.

The foundation’s founder, Denni Ferrara, was inspired to support pediatric cancer research after her daughter Natalia’s successful battle with leukemia and the experiences of close friend Christine Meyer, whose son Bradley had cancer.

Patient-Centered Outcomes Research Institute: January 2014 through June 2015

Nemours is one of eight major pediatric care organizations comprising PEDSNet that together were selected by the Patient-Centered Outcomes Research Institute (PCORI) as one of its 11 Clinical Data Research Networks. Nemours’ partners in PedsNet include Children’s Hospital of Philadelphia (the lead institution), Cincinnati Children’s Hospital and Medical Center, Boston Children’s Hospital, St. Louis Children’s Hospital, Nationwide Children’s Hospital, Children’s Hospital of Colorado and Seattle Children’s Hospital. PedsNet has combined de-identified electronic medical record data comprising 4.7 million clinical encounters and 2.3 million children from all eight organizations to create an exceptionally valuable resource for comparative effectiveness research on children’s health conditions.

During the 18-month Phase I of this grant support, which is complete, PEDSNet achieved a variety of technical and administrative milestones. In October 2015, PEDSNet was awarded Phase II funding that will enable completion of several research studies to demonstrate the network’s capabilities, to illustrate how other researchers can make use of this unique resource and to implement a sustainability plan over the next 3 years.

Nemours Principal Investigators: Tim Bunnell, PhD and Tim Wysocki, PhD

PedsNet Principal Investigator: Christopher B. Forrest, MD, PhD, Children’s Hospital of Philadelphia