Unique Collaboration Leads to “Dream Teams” to Advance Pediatric Research

Jul 30 2014

Unique Collaboration Leads to “Dream Teams” to Advance Pediatric Research

dream teamTwo dedicated “dream teams” of investigators will shape innovative solutions to address unmet pediatric medical needs, as part of a research partnership with The Children’s Hospital of Philadelphia, Drexel University, and The Hebrew University of Jerusalem.

The joint projects each will receive $250,000 over two years in institutional funding, as administrators seek external investors interested in advancing exciting pediatric translational research with commercial viability. The dream teams rose to the top of 20 proposals submitted as a result of a research symposium in January that gave researchers from all three institutions the opportunity to connect and share ideas.

“We are thrilled by the potential for discovery that this portfolio of projects holds, and much of this important work should appeal to future donors,” said The Children’s Hospital of Philadelphia CEO Steven M. Altschuler, MD.

Funding these initial grants internally demonstrates the institutions’ commitment to be a hub of collaboration and exemplifies the inventive, diverse, and entrepreneurial spirit that formed their research consortium initiative. Dr. Altschuler, Drexel President John A. Fry, and The Hebrew University of Jerusalem President Menahem Ben-Sasson signed the research agreement in November as part of a trade mission that Philadelphia Mayor Michael A. Nutter took to Israel.

“These inaugural awards are just the beginning of what the ongoing collaboration between The Children’s Hospital of Philadelphia, Hebrew University and Drexel can accomplish,” said Drexel President John A. Fry. “Together we can create new and unique opportunities that will address unmet needs in pediatrics through innovative commercial pediatric therapeutics and diagnostics.”

One dream team will be based at Drexel with Amy Throckmorton, PhD, as the principal investigator of the “Giving Kids a Chance” project that will investigate a new intravascular blood pump for pediatric patients with congenital heart disease (CHD).

The treatment of single ventricle (SV) anomalies is a formidable and costly challenge for clinical teams caring for patients with CHD, which is the most common major birth defect affecting nearly 1 percent of all newborns. Palliative repair of a SV is generally performed in a series of open-heart procedures over several years. The end result is a man-made physiology in which a SV drives blood flow through the entire circulatory system without the presence of a “right-sided” pulmonary ventricle to pump blood to the lungs. A heart transplant is a treatment option in difficult cases, if the child can survive the waiting period.

As interest is growing about the use of mechanical assistance as a bridge-to-transplant or treatment strategy, the dream team aims to develop a uniquely designed, new therapeutic device for patients with dysfunctional SV physiology. Their goal is to advance the state-of-the-art in blood pump technology, reversing the deleterious characteristics of current approaches and helping to prevent premature congestive heart failure.

dream team

The interdisciplinary team will combine its broad expertise in engineering, pediatric cardiology, congenital heart and cardiothoracic surgery, medical device development, and manufacturing for artificial organs. Members from Drexel will include Dr. Throckmorton, J. Yasha Kresh, PhD, and Randy Stevens, MD. They will be joined by four investigators from CHOP: David J. Goldberg, MD; Matthew Gillespie, MD; Kevin K. Whitehead, MD, PhD; and Joseph Rossano, MD. Amnon Hoffman of The Hebrew University of Jerusalem and Amiram Nir of Hadassah Medical Center will round out the team.

They will work with four industry partners: Rotor Bearing Solutions International of Charlottesville, Va.; Cardiac Assist Inc. of Pittsburgh, Pa.; Applied Rapid Technologies of Fredericksburg, Va.; and Laserage Technology Corp. of Waukegan, Ill.

A second dream team will be based at CHOP with Robert J. Levy, MD, as the principal investigator of a research project “Pediatric Transcatheter Valve Replacements: Preventing Device Failure due to Structural Degeneration.”

These investigations also will focus on CHD with a concentration on Tetralogy of Fallot (TOF). These “blue babies” have insufficient oxygen and need cardiac surgery early in life. Postoperatively patients with TOF are left with chronic malfunction of their pulmonary valve.

Ongoing research at CHOP has demonstrated that transcatheter pulmonary valve (TPV) therapy — the current best option — is susceptible to oxidative damage and structural failure. This interdisciplinary team seeks to gain a better understanding of the inflammatory and oxidative events responsible and to inhibit the early inflammatory response to TPV. They will attempt to modify the TPV material with an antioxidant as a way to prevent oxidative damage to the TPV leaflets.

Dr. Levy will collaborate with Matthew Gillespie, MD from CHOP and Joseph H. Gorman, MD and Robert C. Gorman, MD, from Penn, along with Kenneth Barbee, PhD, and Kara Spiller, PhD, from Drexel, and Gershon Golomb from The Hebrew University of Jerusalem.