Thalidomide - Possible Indications

Possible Indications

Serious infections including sepsis and tuberculosis cause the level of tumor necrosis factor-alpha (TNFα) to rise. TNFα is a chemical mediator in the body, and may enhance the wasting process in cancer patients, as well. Thalidomide may reduce the levels of TNFα, and it is possible that the drug's effect on ENL is caused by this mechanism.

Thalidomide also has potent anti-inflammatory effects that may help ENL patients. In the United States, Celgene distributes thalidomide under the brand name Thalomid for treatment of ENL. Pharmion Corporation, who licensed the rights to market thalidomide in Europe, Australia, and various other territories from Celgene, received approval for its use against multiple myeloma in Australia and New Zealand in 2003. Thalomid, in conjunction with dexamethasone, is now standard therapy for multiple myeloma.

Thalidomide has also been found to be effective in treating actinic prurigo, an autoimmune skin disease. Thalidomide has been used in chronic bullous dermatosis of childhood (CBDC) with encouraging results. Peripheral neuritis may be a limiting factor for long term use of thalidomide.

Thalidomide also inhibits the growth of new blood vessels (angiogenesis); this inhibition, which is suspected as a prime cause of the inhibition of normal development and growth of limbs, may be useful in treating macular degeneration and other diseases. This effect helps AIDS patients with Kaposi's sarcoma, although there are better and cheaper drugs to treat the condition. Thalidomide may be able to fight painful, debilitating aphthous lesions in the mouth and esophagus of AIDS patients that prevent them from eating. The FDA formed a Thalidomide Working Group in 1994 to provide consistency between its divisions, with particular emphasis on safety monitoring. The agency also imposed severe restrictions on the distribution of thalomide through the STEPS program.

Thalidomide is also being investigated for treating prostate cancer, glioblastoma, lymphoma, arachnoiditis, Behçet's disease, and Crohn's disease. In a small trial, Australian researchers found thalidomide caused a doubling of the number of T cells in patients, allowing the patients' own immune system to attack cancer cells.

Studies carried out in animal models have suggested the use of combined therapy with thalidomide and glucantime could have a therapeutic benefit in the treatment of visceral leshmaniasis.

A study published in April 2010 discussed the ability of thalidomide to induce vessel maturation, which may be useful as a therapeutic strategy for the treatment of vascular malformations. The research was conducted in an experimental model of the genetic disease hereditary hemorrhagic telangiectasia.