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Aminopterin

Generic Name
Aminopterin
Brand Names
-
Drug Type
Small Molecule
Chemical Formula
C19H20N8O5
CAS Number
54-62-6
Unique Ingredient Identifier
JYB41CTM2Q
Background

Aminopterin is an amino derivative of folic acid, which was once used as an antineoplastic agent in the treatment of pediatric leukemia. In the 1950's its production was discontinued in favor of methotrexate, which is less potent but less toxic. Off label, aminopterin has also been used in the treatment of psoriasis. Clinicians need to be aware of the characteristic teratologic effects of aminopterin and methotrexate.

Indication

Prior to its withdrawal, aminopterin was initially used in the treatment of childhood leukemia; specifically to induce remissions. Later, aminopterin was used off-label in the United States to treat psoriasis, yielding dramatic lesion clearing. Aminopterin was later supplanted by methotrexate for treating cancer because of its better therapeutic index. Aminopterin (as well as methotrexate) has also been explored for use as an abortifacient. However, their association with severe congenital malformations and teratogenic effects have become known as fetal aminopterin syndrome.

Associated Conditions
-
Associated Therapies
-

Biology-driven cancer drug development: back to the future

Cancer drug development evolved from chemical warfare agents to targeted therapies. Key milestones include nitrogen mustards, folate analogs, and purine analogs for chemotherapy, combination drug therapy, anti-hormone therapy like tamoxifen, and molecularly targeted therapies such as imatinib for CML. Advances in biology and molecular genetics have enabled the design of drugs targeting specific molecular lesions, improving tumor selectivity and treatment outcomes.
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