Vorasidenib

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Vorasidenib is an investigational drug that is being studied for its potential use in the treatment of certain types of cancer. It is a small molecule inhibitor that targets mutant forms of the isocitrate dehydrogenase (IDH) enzymes, specifically IDH1 and IDH2. These mutations are often found in various types of gliomas and other cancers.

Mechanism of Action

Vorasidenib works by inhibiting the mutant forms of the IDH1 and IDH2 enzymes. These mutations lead to the production of an oncometabolite called 2-hydroxyglutarate (2-HG), which can contribute to the development and progression of cancer. By inhibiting these mutant enzymes, vorasidenib reduces the levels of 2-HG, thereby potentially slowing down or stopping the growth of cancer cells.

Clinical Development

Vorasidenib is currently undergoing clinical trials to evaluate its safety and efficacy. These trials are focused on patients with gliomas that harbor IDH1 or IDH2 mutations. Preliminary results have shown promise, with some patients experiencing a reduction in tumor size and improved progression-free survival.

Potential Indications

The primary focus of vorasidenib's development is for the treatment of gliomas, particularly low-grade gliomas and anaplastic gliomas. However, its mechanism of action suggests that it may also be effective in other cancers with IDH1 or IDH2 mutations, such as certain types of acute myeloid leukemia (AML) and cholangiocarcinoma.

Side Effects

As with any investigational drug, vorasidenib may have side effects. Commonly reported side effects in clinical trials include nausea, fatigue, and diarrhea. More serious side effects may also occur, and patients in clinical trials are closely monitored for any adverse reactions.

See Also

• Isocitrate dehydrogenase
• Glioma
• Cancer
• Acute myeloid leukemia
• Cholangiocarcinoma

External Links

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