Forodesine hydrochloride

Forodesine Hydrochloride is a pharmaceutical drug used in the treatment of certain types of cancer, specifically cutaneous T-cell lymphoma (CTCL). It is a purine nucleoside phosphorylase (PNP) inhibitor, which means it works by blocking the action of an enzyme called purine nucleoside phosphorylase, which is involved in the growth of cancer cells.

Mechanism of Action

Forodesine hydrochloride works by inhibiting the enzyme purine nucleoside phosphorylase (PNP). This enzyme is crucial for the survival of cells, as it is involved in the breakdown of purine nucleosides, which are necessary for the synthesis of DNA and RNA. By inhibiting PNP, forodesine hydrochloride prevents the breakdown of these nucleosides, leading to an accumulation of deoxyguanosine triphosphate (dGTP) in lymphocytes. This accumulation leads to the death of the lymphocytes, thereby slowing or stopping the growth of the cancer.

Clinical Uses

Forodesine hydrochloride is used in the treatment of cutaneous T-cell lymphoma (CTCL), a type of non-Hodgkin lymphoma that affects the skin. It is typically used in cases where other treatments have been unsuccessful. The drug is administered orally.

Side Effects

Like all chemotherapy drugs, forodesine hydrochloride can cause side effects. These can include nausea, vomiting, diarrhea, and fatigue. More serious side effects can include infections, due to the drug's effect on the immune system, and changes in blood counts.

Research and Development

Forodesine hydrochloride was developed by the pharmaceutical company BioCryst Pharmaceuticals. It was granted orphan drug status by the Food and Drug Administration (FDA) in 2004 for the treatment of CTCL.

See Also

• Cutaneous T-cell lymphoma
• Purine nucleoside phosphorylase
• Chemotherapy
• BioCryst Pharmaceuticals