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Download Entire Issue (PDF): 1MB Summer 2007  •  Vol. XXXI, No. 3



  • Cover Story

Forging a Path From Laboratory to Clinic


Resource Brief

Funding Matters

Science Advances

News from NCRR

Critical Resources

Forging a Path From Laboratory to Clinic

CTSA consortium accelerates the process of bringing research discoveries to patients.
by Laura Bonetta

Physician-scientist Stephan Grupp at the Children’s Hospital of Philadelphia is testing a new therapy for neuroblastoma, a common and deadly cancer among children. Using resources provided through the Clinical and Translational Science Award at the University of Pennsylvania, Grupp was able to enlist the help of a collaborating institution to complete his study more rapidly. (Photo courtesy of Children’s Hospital of Philadelphia)

After administering two rounds of very intensive chemotherapy and radiation, followed each time by infusions of stem cells to help replace blood cells damaged during the course of treatment, Stephan Grupp and colleagues at Children’s Hospital of Philadelphia can cure about half of their patients with neuroblastoma, one of the most common and deadly solid tumors among children. The success rate, much higher than that achieved by standard therapy without the stem cell infusions, “is better, but not nearly good enough yet,” says Grupp.

To improve the outcome even further, he wanted to add another step to the therapy regimen by immunizing his patients against their cancer. Using a “cancer vaccine,” Grupp could teach the patients’ immune systems to seek out and destroy any remaining neuroblastoma cells, thus minimizing the chances of a relapse. But before embarking on this pioneering work, Grupp had to overcome one major obstacle: The immune systems of children who have undergone chemotherapy and radiation are severely weakened.

A Consortium for Transforming Clinical and Translational Research

The Clinical and Translational Science Awards (CTSAs) established a new consortium to transform how clinical and translational research is conducted, ultimately enabling researchers to provide new treatments to patients more efficiently and quickly. Launched on October 3, 2006, the consortium includes 12 academic health centers located throughout the nation. When fully implemented in 2012, about 60 institutions will be linked together. “Different CTSAs have different strengths,” says Robert Califf, vice chancellor for clinical and translational research at Duke University Medical Center. “Our job is to create some common goals by sharing ideas and different expertise.”

Representatives from all CTSAs meet several times a year to share experiences and establish best practices in a range of areas, including the following:

  • Biostatistics and epidemiology
  • Research design
  • Communications
  • Clinical research ethics and resources for research participants
  • Participant and clinical interaction resources
  • Regulatory knowledge
  • Pediatrics
  • Informatics
  • Community engagement in clinical research
  • Education and career development
  • Institutional and national evaluation
  • Translational research in traditional academic settings and in public-private partnerships
  • Translational research

Information about the committees responsible for these areas and their activities is available at the Consortium’s Web site.

Grupp and colleagues, including Carl June at the University of Pennsylvania, tried “rescuing” the immune system by collecting blood cells from patients at the time of diagnosis and then coaxing the immune cells into dividing and multiplying outside the body. In a pilot clinical trial funded by the National Cancer Institute, the researchers transplanted the multiplied immune cells back into patients after they had received therapy. Preliminary data suggest that these transplanted cells are fully functional and allow a patient with a devastated immune system to mount an immune response to a vaccine.

To finish the current trial as quickly as possible and move on to testing the cancer vaccine, Grupp decided to enlist the help of a collaborating institution. That’s when he came up against a mountain of administrative paperwork.

Reconfiguring the Research Enterprise

Clinical trials, such as the one Grupp is conducting, are a critical step in translating scientific discoveries arising from laboratory, clinical, or population studies into practical applications that can improve human health. But researchers engaged in this “translational research” encounter numerous challenges on the path from bench-to-bedside testing. Unlike scientific research focused on a particular approach or discipline, translational research crosses boundaries between basic science and clinical applications, requiring intense interactions among investigators with diverse backgrounds and types of expertise and among members of both academic and industrial communities.

To strengthen and accelerate the process of bringing scientific discoveries to the community, the Clinical and Translational Science Award (CTSA) consortium, established by NCRR as part of the NIH Roadmap for Medical Research, strives to remove roadblocks and ease challenges in clinical and translational research. (See sidebar, “A Consortium for Transforming Clinical and Translational Research.)