The Johns Hopkins Laboratory For Translational Research in Pemphigus

RESEARCH PROGRAM

The Department of Dermatology at Johns Hopkins has a long history of distinguished research in the field of autoimmunity and pemphigus. Dr. Anhalt and coworkers developed the first animal model for pemphigus and used this model to define the events that are critical to the development of blistering skin and mucous membranes in pemphigus. Their work has clarified that effective therapeutics for this disease must be directed at inhibition of autoantibody production, and their laboratory efforts are devoted to this goal.

Research performed in the immunodermatology laboratories at Johns Hopkins as well as in other centers has clarified the genetic basis of the disease, the role of autoantibodies in the patho-genesis of the blistering lesions, and the method by which this autoimmune disease might be defeated.

It is now clearly established that the disease affects individuals with a genetic predisposition; and, although the disease affects every race and population, the two genes that predispose to the development of pemphigus are found with higher prevalence in individuals of eastern European Jewish families and in patients from northern India and Asia.

It also is clear that the painful blisters of skin and mucous membranes are caused by antibodies produced in the bone marrow that reach the skin and cause disease. Presently, the only way to successfully treat the disease is by inhibiting production of antibodies by the bone marrow, which requires hazardous immunosuppressive treatment with medications such as prednisone and other similar drugs.

We now know enough about the molecular basis of the autoimmune disease to design treatments that will not require drugs like prednisone for successful treatment. Specific proteins or peptides that can be manufactured as a drug might specifically and efficiently inhibit antibody formation. These new targeted treatments will require development and testing through sustained research.