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They looked at two disorders in particular. About one-third of patients with glycogen storage disease 1B -- which causes abnormal glucose metabolism -- also had Crohn's disease symptoms. They found the same thing in patients with the genetic disorder chronic granulomatous disease.

Patients with these and other genetic immune system disorders often are treated with drugs that stimulate the body's immune response. One such drug is a recombinant version of a protein produced by the body to enhance the immune response by increasing the number and function of white blood cells. Granulocyte-macrophage colony stimulating factor (GM-CSF), also known by the trade name Leukine (sagramostim), not only assists with the primary symptoms of these immune disorders, but also helps eliminate Crohn's disease symptoms.

Another distinction between this treatment and traditional Crohn's therapies is that GM-CSF targets the innate immune response -- the body's built-in defense against infectious organisms. Most current Crohn's disease treatments interfere with what's called acquired immunity, which develops over time as the body encounters new infections. Generally, the innate immune system attacks infectious agents first, holding down the fort until the acquired immune system can take over.

Because of the link between immune system disorders and Crohn's disease, and because of the connection between treatment with GM-CSF and recovery from Crohn's-like symptoms, Dieckgraefe and Korzenik tested the drug in 15 patients with moderate to severe Crohn's disease.

"Conventional thinking would have predicted that these drugs could worsen the disease," Dieckgraefe says. "But we thought that these immune deficiencies provided a good model for how our Crohn's patients would respond. Furthermore, we knew that GM-CSF was a natural protein that already was prese
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Contact: Jim Dryden
drydenj@msnotes.wustl.edu
314-286-0110
Washington University School of Medicine
7-Nov-2002