A Widely Used Antifungal Drug Could be Effective For Treating Congenital Erthropoietic Porphyria

According to a story from Medical Xpress, an international group of scientists from the US, France, and Spain may have discovered that an antifungal agent commonly used to treat skin disorders could be a viable treatment for congenital erythropoietic porphyria (CEP). This data was first published in the the academic journal Science Translational Medicine

About Congenital Erthropoietic Porphyria (CEP)

CEP is a type of chronic porphyria, which is a genetic disorder in which substances known as porphyrins accumulate in the body, causing abnormalities of the nervous system and skin. CEP is caused by a deficiency of the enzyme uroporphyrinogen synthase (UROS). In CEP, the symptoms of the disease can cause extensive damage to the skin, to the point that the ears, lips, and nose begin to erode away. The skin is very sensitive to light, which causes open sores, blistering, lesions, darkened skin, abnormal hair growth, shortened digits, loss of hair and nails, eye abnormalities, and damaged and discolored teeth and gums. The best way to manage this disease is to avoid sunlight as much as possible. Patients may require special clothing and tinted windows to avoid damage. A bone marrow transplant has been curative in a limited number of cases. To learn more about CEP, click here.

New Treatment Needed

There are serious limitations to the approaches and medications used to manage CEP, and there is a desperate need for more effective treatments. Thankfully, this discovery could be an effective way to reduce symptoms. The antifungal agent, which is known as ciclopirox, was discovered after an extensive search through a list of compounds; the researchers hoped to find a compound that would bind to UROS, and this was the one that they found. 

Promising Results

In a mouse model of CEP, the researchers found that the administration of ciclopirox helped restore UROS back to levels seen in health mice, and they concentration of porphyrins in the liver, blood, and urine was reduced. The treatment also allowed for a more normal process of skin healing.

The results of the mouse model tests are certainly encouraging. The next step will be research to investigate whether long term use of ciclopirox is safe.

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