Smoothened agonist

Smoothened Agonist

A Smoothened Agonist is a type of pharmacological agent that acts on the Smoothened (SMO) receptor, a key component of the Hedgehog signaling pathway. This pathway plays a crucial role in the development and growth of cells and tissues in many organisms, including humans.

Mechanism of Action

Smoothened agonists work by binding to the Smoothened receptor, which is typically inhibited by another protein called Patched (PTCH). When a Smoothened agonist binds to the SMO receptor, it relieves this inhibition, allowing the Hedgehog signaling pathway to be activated. This can lead to increased cell proliferation and survival, which can be beneficial in certain therapeutic contexts.

Therapeutic Uses

Smoothened agonists have been investigated for their potential use in a variety of medical conditions. For example, they have been studied as potential treatments for basal cell carcinoma, a type of skin cancer that is often driven by mutations in the Hedgehog signaling pathway. They have also been explored as potential therapies for other types of cancer, as well as for diseases characterized by tissue degeneration or damage, such as multiple sclerosis and Parkinson's disease.

Side Effects and Risks

Like all pharmacological agents, Smoothened agonists can have side effects and risks. These can include nausea, vomiting, fatigue, and hair loss. In addition, because they can stimulate cell growth and proliferation, there is a risk that they could potentially contribute to the development of certain types of cancer.

Examples

Examples of Smoothened agonists include Vismodegib and Sonidegib, both of which have been approved by the Food and Drug Administration (FDA) for the treatment of advanced basal cell carcinoma.

See Also

• Hedgehog signaling pathway
• Basal cell carcinoma
• Pharmacology

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