Pelabresib

A BET inhibitor used in cancer treatment

Pelabresib
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Pelabresib is a small molecule inhibitor of the bromodomain and extra-terminal domain (BET) family of proteins. It is being investigated for its potential use in the treatment of various types of cancer, including myelofibrosis and other hematological malignancies.

Mechanism of Action

Pelabresib functions by inhibiting the activity of BET proteins, which are epigenetic regulators that play a crucial role in the transcription of genes involved in cell cycle progression and apoptosis. BET proteins, such as BRD2, BRD3, and BRD4, recognize acetylated lysine residues on histone tails, facilitating the recruitment of transcriptional machinery to chromatin. By inhibiting these interactions, pelabresib disrupts the expression of oncogenes and other genes critical for cancer cell survival and proliferation.

Clinical Development

Pelabresib is currently undergoing clinical trials to evaluate its efficacy and safety in patients with various cancers. It has shown promise in early-phase trials, particularly in combination with other therapeutic agents. The drug is being studied in both monotherapy and combination therapy settings, with a focus on hematological malignancies such as myelofibrosis.

Pharmacokinetics

The pharmacokinetic profile of pelabresib includes its absorption, distribution, metabolism, and excretion characteristics. It is administered orally, and its bioavailability and half-life are subjects of ongoing research to optimize dosing regimens. The metabolism of pelabresib involves hepatic pathways, and its excretion is primarily through the renal and fecal routes.

Side Effects

As with many cancer therapies, pelabresib is associated with a range of potential side effects. Common adverse effects observed in clinical trials include fatigue, nausea, diarrhea, and hematological abnormalities such as thrombocytopenia and anemia. The safety profile of pelabresib continues to be evaluated in ongoing studies.

Research and Future Directions

Research on pelabresib is focused on understanding its full therapeutic potential and identifying biomarkers that predict response to treatment. Ongoing studies aim to explore its use in combination with other targeted therapies and immunotherapies to enhance its efficacy and overcome resistance mechanisms.

Related Pages

• Bromodomain and extra-terminal domain (BET) proteins
• Myelofibrosis
• Cancer treatment
• Epigenetics