Diacerein cream is a safe treatment for EBS
The results of a clinical study from the EB House
Generalized severe EBS is caused by mutations in the keratin 14 or keratin 5 gene, which lead to an accumulation of keratin proteins in the skin cells, triggering inflammatory reactions, that result in painful skin blistering.
Last year, we reported on the phase 2/3 trial, which showed that a diacerein containing cream that was developed by EB House researchers significantly reduced the number of blisters in EBS patients.
To confirm the safety of the treatment further investigations were carried out on a voluntary basis. For this purpose, the EB House researchers and cooperation partners investigated how diacerein is metabolised in the body. Diacerein is a small molecule that is converted to its active form rhein within the skin, which mediates the reduction of blister formation by suppressing skin inflammation. The researchers were especially interested in determining the level of rhein found in blood and urine after topical application of diacerein, since high rhein levels has been reported to cause major side effects affecting the gastro-intestinal tract when orally administered in other diseases.
Two EBS patients were willing to apply the diacerein cream over a period of four weeks onto 3 % of their blistering body surface. Systemic rhein levels were found in blood and urine after 4 weeks treatment at a 150-fold lower dose than reported levels after a single-dose oral intake. This means that even if the cream was applied to the entire body surface, the likelihood of the mentioned side effects would be low.
In order to study the ability of skin to uptake and transform diacerein into rhein, the researchers included laboratory experiments using pig skin. There, diacerein cream was applied onto the skin samples every 24 hours over a period of three days. Subsequent analysis of the treated skin confirmed the presence of rhein and thus the ability of the skin to convert diacerein into rhein.
According to these results recently published in the Orphanet Journal of Rare Diseases, local application of the diacerein cream is a potentially safe and well-tolerated therapy for the treatment of EBS.
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