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The challenge in treating autoimmune disorders has been suppressing the immune system’s inflammatory attacks on body tissues without generally suppressing immune function. Now, researchers Mark Sundrud, PhD, and Anjana Rao, PhD, of PCMM/IDI, find promise in halofuginone, a drug from the hydrangea root that’s used as an antimalarial in traditional Chinese medicine.
In the June 5 Science, Sundrud and Rao show that halofuginone prevents the development of Th17 cells, a recently recognized kind of immune cell that’s been implicated in a variety of autoimmune disorders, including inflammatory bowel disease, rheumatoid arthritis, multiple sclerosis (MS), type 1 diabetes, eczema and psoriasis. Th17 cells inflict their harm mainly by secreting IL-17, a cytokine (chemical messenger) that triggers an inflammatory response. But in the presence of halofuginone, IL-17 levels were suppressed, and disease severity was significantly reduced in mice with an autoimmune disease resembling MS, marked by infiltration of Th17 cells into the central nervous system.
Halofuginone’s effect was highly selective: Other kinds of T cells involved in normal immune function weren’t altered. The drug appears to work by turning on signals telling cells that amino acids are in short supply. Sundrud speculates that the body tries to conserve amino acids by discouraging protein-intensive activities, including making cells like Th17 and cytokines like IL-17 that are involved in inflammatory responses.
Because halofuginone is a small-molecule drug, it has the potential to be taken orally rather than injected. It is in the public domain, so is of little interest commercially, but that could change if effective analogues are developed.
An ambitious project that ultimately seeks to partner with every Children’s patient to do genetic studies launches this summer in the Developmental Medicine Center, through a collaboration between the Program in Genomics, Division of Genetics, Informatics Program and Information Services Department. After a one-time consenting process, patients provide a DNA sample and enroll in a registry, giving researchers permission to include them in studies. Unlike other DNA banks, patients can opt to receive relevant study results.
The project, not yet formally named, recently merged with a similar effort, led by David Williams, MD, PhD, to establish a central tissue repository. Plans are for it to expand to the Emergency Department in September. For more, see the spring issue of Vector.
childrenshospital.org/vector.
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