Alicaforsen

An antisense oligonucleotide drug

Alicaforsen
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Alicaforsen is an antisense oligonucleotide drug designed to target and inhibit the production of intercellular adhesion molecule 1 (ICAM-1). It is primarily investigated for its potential therapeutic effects in treating inflammatory bowel diseases such as ulcerative colitis and Crohn's disease.

Mechanism of Action

Alicaforsen works by binding to the messenger RNA (mRNA) of ICAM-1, a protein that plays a crucial role in the inflammatory response. By binding to the mRNA, alicaforsen prevents the translation of ICAM-1, thereby reducing its expression on the surface of endothelial cells and other cell types involved in inflammation. This reduction in ICAM-1 levels can lead to decreased leukocyte adhesion and transmigration, which are key processes in the inflammatory cascade.

Clinical Applications

Alicaforsen has been studied in various clinical trials for its efficacy in treating inflammatory bowel diseases. It has shown promise in reducing inflammation and promoting mucosal healing in patients with ulcerative colitis. Additionally, it has been explored as a treatment for pouchitis, a condition that can occur after ileal pouch-anal anastomosis surgery in patients with ulcerative colitis.

Ulcerative Colitis

In patients with ulcerative colitis, alicaforsen has been administered as an enema formulation to directly target the inflamed colonic tissue. Clinical trials have demonstrated that this localized delivery can lead to improvements in disease symptoms and endoscopic findings.

Pouchitis

Alicaforsen has also been investigated for the treatment of pouchitis, a complication that can arise after surgical treatment for ulcerative colitis. The drug's ability to reduce ICAM-1 expression and subsequent inflammation makes it a potential therapeutic option for this condition.

Development and Research

Alicaforsen was developed by Isis Pharmaceuticals, now known as Ionis Pharmaceuticals. The drug has undergone several phases of clinical trials to evaluate its safety and efficacy. While it has shown potential in certain studies, further research is needed to fully establish its therapeutic role and to gain regulatory approval for widespread clinical use.

Related Pages

• Antisense therapy
• Inflammatory bowel disease
• Ulcerative colitis
• Crohn's disease
• Intercellular adhesion molecule 1