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May 2007
Children's Research Institute News Brief
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Bernard L. Maria, MD, MBA
Executive Director
Darby Children's
Research Inst. |
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Inderjit Singh, PhD
Scientific Director
Darby Children's
Research Inst. |
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DCRI researchers prepare factor B antibody for prime time
Fellow DCRI researchers Gary Gilkeson and Steve Tomlinson hold patents on discoveries poised to go to trial in the near future, to hopefully curb and possibly prevent several inflammatory diseases affecting children, including asthma, lupus, juvenile arthritis and more.
Drs. Gilkeson and Tomlinson, both in the Childhood Rheumatic Diseases Center, 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," explains Dr. Gilkeson. "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," notes Dr. Gilkeson.
Dr. Michael Holers of the University of Colorado (with whom Dr. 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," explains Dr. Gilkeson. "That's the first disease in which we're testing the antibody. We envision it being used for a variety of inflammatory diseases."
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," says Dr. Gilkeson. "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.
Dr. 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," he explains.
Effective, less toxic treatments are the goal of both researchers.
As Drs. Gilkeson, Tomlinson and researchers across the DCRI continue to demonstrate, researchers are finding vital connections between the role of basic science and the treatment of children's diseases.
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