cells. They went on to show that the mutation of the gene encoding Jak3 was responsible for a form of severe combined immunodeficiency (SCID). Because Jak3 is essential for immune cell function, and because its expression is limited to blood cells, the team proposed that inhibiting Jak3 might be the basis for a new class of immunosuppressant drugs. The group then entered into a collaborative research and development agreement with Pfizer -- a partnership that has enabled Pfizer to develop this new drug.

CP-690,550 is the first Jak3 inhibitor to show positive results in primates. Further animal studies are being conducted to determine if this drug could be used successfully and safely in humans.

Immunosuppressant drugs, which inhibit the body's immune response, are given to prevent the body from rejecting transplanted organs, and are also used to treat autoimmune diseases such as lupus, rheumatoid arthritis, eczema and psoriasis. Autoimmune diseases cause the immune system to attack healthy, normal tissue as if it were a foreign substance. The finding that CP-690,550 selectively suppresses the immune response in transplant rejection with minimal toxicity also suggests that a Jak3 inhibitor might be useful in the treatment of autoimmune diseases.

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Contact: Ray Fleming
flemingr@mail.nih.gov
301-496-8190
NIH/National Institute of Arthritis and Musculoskeletal and Skin Diseases
30-Oct-2003

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