Target intelligence / Profile preview

Folic acid

Molecular classification
Other (Vitamin), Cofactor
01

Overview

Folic acid—commonly referred to as **folate** in its natural forms—is a water-soluble B-vitamin essential for the generation and maintenance of new cells. It is required for DNA and RNA synthesis through methylation reactions and plays a critical role in amino acid metabolism necessary for cell division and the maturation of blood cells. Naturally occurring folates exist in many chemical forms found in food; synthetic **folic acid** is used in fortified foods and supplements. Folic acid itself has no biological activity unless converted into active forms such as tetrahydrofolate by dihydrofolate reductase. Drugs that inhibit this enzyme—such as methotrexate—are used therapeutically to treat cancer by blocking DNA synthesis but can cause significant toxicity. Folinic acid (leucovorin) can be administered to mitigate these toxic effects without negating anticancer efficacy because it bypasses the blocked step. Deficiency of dietary folates leads to megaloblastic anemia; adequate intake is especially important during pregnancy due to its role in preventing neural tube defects.\n\n**Note:**\nFolate/folic acid is *not* itself a therapeutic target like an enzyme or receptor but rather an essential nutrient/cofactor targeted indirectly by certain drugs ("antifolates"). The term "Folate" does not refer specifically to any single protein or receptor but rather encompasses a family of related compounds with vitamin activity; thus, it does not fit standard drug-target conventions.

Other names
FolateVitamin B9FolacinPteroyl-L-glutamic acidVitamin MVitamin Bc
02

Mechanism of action

Antifolate drugs inhibit dihydrofolate reductase, blocking conversion of folic acid to tetrahydrofolate, thereby inhibiting DNA synthesis in rapidly dividing cells such as cancer cells.\nLeucovorin "rescue" provides reduced folates to normal cells during antifolate chemotherapy.

03

Biological functions

DNA synthesis and repairRNA synthesis and methylation reactionsAmino acid metabolism (especially for cell division and blood cell maturation)
04

Disease associations

Anemia (folate deficiency anemia)Neural tube defects in pregnancy (deficiency-related)Cancer (as a metabolic cofactor, not as a direct target)
05

Safety considerations

High-dose supplementation may mask vitamin B12 deficiency.Toxicity from antifolate drugs includes bone marrow suppression, gastrointestinal toxicity, liver and kidney failure.
06

Interacting drugs

Methotrexate

3 more in the full profile.

07

Biomarkers

Serum/plasma folate levels for deficiency screening or monitoring supplementation efficacy.Red blood cell folate concentration.

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