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DCRI researchers prepare antibody for prime time As a parent, few things are worse than the inability to stop a child’s pain. For children who suffer from lupus, juvenile arthritis, asthmas, and other inflammatory diseases, pain and emotional discomfort are something that mom, dad, nor a doctor can always take away. Enter patent holders and Darby Children’s Research Institute (DCRI) researchers Gary Gilkeson, M.D., Rheumatology and Immunology, and Steve Tomlinson, Ph.D., Microbiology and Immunology.   By holding patents for major discoveries that are currently poised to go to clinical trial phase testing, Gilkeson and Tomlinson hope to curb and possibly prevent several inflammatory diseases affecting children via the Childhood Rheumatic Diseases Center. There, researchers are readying the factor B antibody for human trials in asthma.   “In developing a patent for the factor B antibody, we set out to block complement proteins,” Gilkeson said. “As part of the immune system, the complement system helps fight infection, the growth of tumor cells and other immunity functions.”   There are three pathways for the complement cascade—classic, alternative and lectin. Factor B is a key component of the alternative pathway. “What we found, in looking at lupus, is that factor B in the alternative pathway is important in inflammatory diseases. By blocking this factor B, we can block inflammation,” Gilkeson explained.   Michael Holers, M.D., University of Colorado (with whom Gilkeson holds the factor B antibody patent), tested the antibody as an inhalation agent and found it effective in treating asthma attacks in mice. “The antibody has been humanized, and we’re now developing it to be used in trials for asthma. That’s the first disease in which we’re testing the antibody. We envision it being used for a variety of inflammatory diseases,” Gilkeson said.   Taligen, the company that’s developing the factor B antibody, will fund upcoming Phase I trials. “The hope is that this will stop acute asthma attacks, as well as prevent future attacks,” Gilkeson said. ‘The antibody will essentially act as an anti-inflammatory, and could decrease the need to use steroids.” Steroids are responsible for complications and side effects in children, including growth stunting, bone problems and weight gain.   Tomlinson, a professor of microbiology and immunology, holds the patent for and is developing targeted complement inhibitors for inflammatory conditions, which may also prove effective in treating asthma.   “The hope is that these complement inhibitors will be effective in treating lupus, childhood arthritis, heart and kidney transplants, stroke and spinal cord injury,” Tomlinson said.   In the spirit of blending basic science and clinical applications together to better the treatment of children’s diseases, both researchers emphasized the end goal of their work to be effective, less toxic treatments for pediatric patients.     Friday, June 8, 2007 Catalyst Online is published weekly, updated as needed and improved from time to time by the MUSC Office of Public Relations for the faculty, employees and students of the Medical University of South Carolina. Catalyst Online editor, Kim Draughn, can be reached at 792-4107 or by email, catalyst@musc.edu. Editorial copy can be submitted to Catalyst Online and to The Catalyst in print by fax, 792-6723, or by email to catalyst@musc.edu. To place an ad in The Catalyst hardcopy, call Island Publications at 849-1778, ext. 201.