Thalidomide: New Uses for an Old Drug
Thalidomide: New Uses for an Old Drug
The thalidomide story could well have ended with the tragedy of birth defects in the early 1960s but for a serendipitous finding: in a patient with mania and leprosy, thalidomide given as a sedative dramatically improved the leprotic skin lesions. Despite the major drawbacks of thalidomide - teratogenicity and axonal neuropathy - the drug has acquired a unique, albeit limited, role in the treatment of severe, disabling conditions where standard anti-inflammatory or immunosuppressive therapies have failed. Best studied amongst these are leprosy, Behçet's disease, graft-versus-host disease (GVHD) and some dermatoses.
However, success in patients with some conditions has been tempered by unacceptably high rates of neuropathy. To minimise risk of teratogenicity and neuropathy, guidelines for thalidomide use have been published and new analogues of the drug are being developed.
The thalidomide story could well have ended with the tragedy of birth defects in the early 1960s but for a serendipitous finding: in a patient with mania and leprosy, thalidomide given as a sedative dramatically improved the leprotic skin lesions. Despite the major drawbacks of thalidomide - teratogenicity and axonal neuropathy - the drug has acquired a unique, albeit limited, role in the treatment of severe, disabling conditions where standard anti-inflammatory or immunosuppressive therapies have failed. Best studied amongst these are leprosy, Behçet's disease, graft-versus-host disease (GVHD) and some dermatoses.
However, success in patients with some conditions has been tempered by unacceptably high rates of neuropathy. To minimise risk of teratogenicity and neuropathy, guidelines for thalidomide use have been published and new analogues of the drug are being developed.
Thalidomide is not available in Denmark, Germany and The Netherlands, and is only available under special conditions in France (where it is indicated for the conditions mentioned in this article as well as Jeener-Kanoff disease) and Spain. |
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