October 2013

Offering Basic Scientists an Introduction to Translational Research

Translational_Research

In an example of CHOP Research’s commitment to educating the next generation of scientists, a recent workshop held at the Institute introduced young investigators to the principles of translational research. Led by a committee of Children’s Hospital faculty and organized by the CHOP Research Office of Postdoctoral Affairs, the 2013 Pediatric Translational Research Workshop for Basic Scientists brought together a group of graduate students, postdocs, and early-career researchers for a week of presentations, clinical visits, and discussions.

“The workshop was designed to provide participants with the fundamental tools and knowledge to bring their basic research discoveries to the clinic,” said Deputy Scientific Director of CHOP Research Tom Curran, PhD, FRS, who was the workshop’s facilitator and the lead faculty organizer. “In addition to cutting-edge scientific presentations from top CHOP and UPenn researchers, the rigorous agenda included lectures and discussions focused on basic/clinical research collaborations, navigating an Institutional Review Board, and evaluating the ethics of translational research,” he added.

Workshop attendees came from a number of Philadelphia-area universities. In addition to The Children’s Hospital of Philadelphia, institutions represented included the University of Pennsylvania, Temple University, Thomas Jefferson University, the University of Medicine and Dentistry of New Jersey (now Rutgers Biomedical and Health Sciences), and the Monell Chemical Senses Center.

In his welcome address on the workshop’s opening day, Dr. Curran acknowledged there was “no simple answer” to pursuing a career in translational research. However, in order to give the participants “a real feel for translational research,” the workshop would offer “specific, in-depth experiences,” he said. Dr. Curran also noted how important collaboration with other researchers — particularly cross-disciplinary collaboration — is to successful translational research.

“Working as part of a team can be one of the most rewarding experiences you’ll have,” Dr. Curran said.

The weeklong workshop featured speakers from a variety of disciplines from Children’s Hospital and the University of Pennsylvania. Among the leading CHOP researchers who presented were Philip R. Johnson, MD, director of CHOP Research, Louis Bell, MD, chief of the division of General Pediatrics, Peter Adamson, MD, chief of the division of Clinical Pharmacology and Therapeutics, and fetal surgeon Alan Flake, MD, director of the Center for Fetal Research. The sessions spanned the research spectrum, from those on cystic fibrosis therapies to mitochondrial disease to several talks on translational research in neurodevelopmental disorders.

Translational Research’s Impact

For his part, CHOP Research’s Dr. Johnson presented the workshop participants with three examples of how translational research has affected Children’s Hospital patients. Saying that his job was to make translational research “real” for the attendees, Dr. Johnson discussed three case studies: the work done by Stephan Grupp, MD, PhD, to treat acute lymphoblastic leukemia with T cell therapy; fetal surgery to correct myelomeningocele, a devastating form of spina bifida, which is led by CHOP’s N. Scott Adzick, MD; and the investigation by the University of Pennsylvania’s Albert M. Maguire, MD, and Jean Bennett, MD, PhD, into gene therapy to treat eye disease.

Translational research is not about “instant gratification,” Dr. Johnson said, noting that it can take decades for work to go from the bench to the bedside. But translational research offers investigators the opportunity to see their work pay off and make an impact on patients, he said.

To give attendees a sense of the effect translational research can have, the workshop also included visits from patients and patient families — including those with cystic fibrosis, the developmental disorder Cornelia de Lange Syndrome, the rare genetic disease Fibrodysplasia ossificans progressiva, and mitochondrial disease — and a visit to the Hospital’s pediatric intensive care unit (PICU). The PICU visit, and a discussion of the attendees’ impressions, was moderated by Athena Zuppa, MD, MSCE, an attending physician in Pediatric Critical Care medicine.

Several outside speakers gave talks during the week, including former FDA official Stephen Spielberg, MD, PhD, current editor-in-chief of Therapeutic Innovation and Regulatory Science, and Richard Woodward, PhD, chief executive officer of Vascular Magnetics, Inc. Dr. Spielberg discussed how industry, academics, and the FDA view translational medicine, while Dr. Woodward’s talk was focused on setting up a biotechnology business. Workshop participants were also given the chance to network with each other and CHOP and UPenn faculty at events both on and off campus.

And last but not least, Joseph W. St. Geme, III, MD, Children’s Hospital’s physician-in-chief and chair of the Department of Pediatrics at the University of Pennsylvania, delivered the workshop’s closing address. A noted microbiologist, Dr. St. Geme recently joined CHOP after spending a number of years at Duke University, where he served as chairman of the Department of Pediatrics and chief medical officer.

“The Pediatric Translational Research Workshop for Basic Scientists reflects the CHOP Research Institute’s commitment to the next generation of leaders in translational science,” said Dr. Curran.

To read more about the 2013 Pediatric Translational Research Workshop for Basic Scientists, click here. And to learn more about The Children’s Hospital of Philadelphia’s educational resources, see the CHOP Research site.

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