General Clinical Research Center

Overview

A major priority for investigators at Childrens Hospital Los Angeles is understanding how disease and treatments may interfere with the normal growth and development of children otherwise cured of serious, life-threatening illnesses.  

The General Clinical Research Center (GCRC) at Childrens Hospital Los Angeles - a satellite of the GCRC at the Keck School of Medicine of the University of Southern California (USC) - provides critical infrastructure and support for clinical research with a pediatric focus. The Childrens Hospital GCRC was established in 1995, along with a satellite at City of Hope National Medical Center.

Edward M. Gomperts, MD, serves as program director of the Childrens Hospital GCRC. John Wood, MD, PhD, is associate director.

The National Center for Research Resources of the National Institutes of Health (NIH) funds 82 GCRCs nationwide.  In supporting the GCRC, the NIH has attempted to address the problem that the most novel diagnostic and treatment modalities are unlikely to be available through the health insurance system.  Support for GCRC studies comes through payment of patient care costs, such as the cost of hospitalization that ordinarily might not be reimbursable by insurers.

In the future, the GCRC will be replaced by a new research support grant called the Clinical and Translational Science Award, which will be the new face of clinical research.

Program Focus

The GCRC at Childrens Hospital Los Angeles seeks to advance multidisciplinary studies into new diagnostic tools and treatments for infants and children. It currently works with about 20 investigators and 10 divisions, and supports a wide range of clinical research in the areas of:

1. gene therapy
2. bone marrow transplantation
3. diabetes
4. neurology
5. oncology 
6. HIV/AIDS

Other studies focus on the treatment of autoimmune diseases, muscle diseases and emergency trauma, as well as the use of innovative imaging techniques to detect subtle, but important changes, in brain function and development that result from certain pediatric illnesses.

Support

The Childrens Hospital GCRC provides clinical investigators with the support of a neuropsychologist and a dedicated, three-member nursing staff.  In addition, researchers receive support in biostatistical analysis and data management. 

Facilities

In November 2006, Childrens Hospital Los Angeles established a new, 1,625-square foot clinical research outpatient facility - the Childrens Clinical Investigation Center - which is shared with the Childrens Imaging Research Program.  The space dedicated for use by GCRC investigators includes:

• four cubicles for subject evaluation and sample collection
• three research nurse work stations
• offices for a nurse manager, neuropsychologist and testing requirements

A nearby 8,000-square foot administrative space with a lecture hall and videoconferencing capabilities is also available to the GCRC for use 

Accomplishments

Childrens Hospital was the first GCRC to provide neuropsychological evaluations to measure the effects of disease and treatment in children, a modality that other GCRCs nationwide have since adopted.   Some of the accomplishments of the GCRC since its inception have been:

• The first gene therapy trials using umbilical cord blood stem cells
• he first use of gene therapy to treat HIV in children
• The first use of a designer peptide hormone antagonist to treat infantile spasms
• The evaluation of strategies to prevent Type I diabetes
• The first evaluation of a novel drug (BSO) to increase the effectiveness of chemotherapy for neuroblastoma
• The first demonstration that transplanted human bone marrow cells could produce new muscle cells in muscular dystrophy

Currently underway:  New protocols to evaluate the effects of childhood illness and treatments on bone development.

Membership