Introducing Basic Scientists to Translational Research

In an example of CHOP Research’s commitment to educating the next generation of scientists, a recent workshop introduced a group of young investigators to the principles of translational research. The 2013 Pediatric Translational Research Workshop for Basic Scientists brought together 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.

The weeklong workshop featured speakers from a variety of disciplines, with sessions spanning the research spectrum, from those on spina bifida and cystic fibrosis therapies to mitochondrial disease, to several talks on translational research in neurodevelopmental disorders. Workshop participants were also given the chance to network with each other and CHOP and UPenn faculty at events both on and off campus.

For his part, Philip R. Johnson, MD, director of CHOP Research presented the participants with 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.

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. 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 read more about the 2013 Pediatric Translational Research Workshop for Basic Scientists, see this month’s Bench to Bedside. And see below for a video about Dr. Adzick’s study of fetal surgery to correct spina bifida.