Darmacare.us

New therapeutic avenue to treat a rare disease of the skin

Posted on October 20th, 2009 in Uncategorized skin disease

Spanish scientists have successfully eliminated in mice a rare skin disease characterized by the appearance of erosions in the mouth and blisters on the dermis. How? With two drugs that are currently listed in other disorders, such as some cancers.

This is the pemphigus vulgaris and currently the only existing treatment are the cortisone. “In high doses have significant side effects. While curbing the disease, the risk of complications is high: infections, broken bones, diabetes …”, says Agustin Spain, head of the Department of Dermatology, University Clinic of Navarre and one of the authors of the study.

Vultar Pemphigus is an autoimmune disease that manifests in the skin anywhere on the body and mucous membranes in the form of erosions and blisters. Experts say not only causes pain, can also lead to some disease. Depending on the affected areas, the patient will have different difficulties. For example, in the nasal mucosa, bleeding occurs frequently and in the mouth, can prevent swallowing.

Although its prevalence is low (20 new patients each year), could cause death if “is widespread in different parts of the body due to infection and dehydration resulting from the rupture of the blisters,” the authors explain in their article.

The severity of the disease itself and the limitations of medication are the reasons why in their day started this research, published in ‘Experimental Dermatology’. To carry out those responsible for this work have worked on newborn mice.

“It’s about playing the disease in mice by injecting antibodies obtained from patients,” says Maider Pretel Irazábal, Department of Dermatology and Venereologica Medical Surgical Clinic University of Navarra. The next step was to treat them with drugs in test: erlotinib (suitable for some types of cancer) or rapamycin (used in transplants). “In both cases, pemphigus vulgaris, and ceases to be visually displayed either at the molecular level. Completely block the disorder with no side effects,” says Dr. Spain.
An autoimmune disease

To understand how these drugs act in the mice with this rare skin disease, before it is important to know more about the mechanism of it. Being an autoimmune disease, patients who suffer from abnormally produce antibodies that act against some proteins of the skin, whose function is to keep the cells attached to the dermis. As a consequence, these cells separate and form blisters.

According to experts, yet little is known about how it is developed pemphigus vulgaris in man. “In this investigation, we discovered that the molecular pathways that are activated by this metabolic disorder which could have a key role in other diseases such as certain types of cancer.”

On the one hand, rapamycin inhibited mTOR kinase (a type of enzyme) to block the immune system of patients and get so they do not reject the transplanted organs, especially kidneys.

The second drug, Erlotinib, blocks other kinases (HER) that are activated in some cancers and also in pemphigus vulgaris.

“Precisely because the HER pathway and mTOR are activated in the cells of the skin of diseased mice when administered earlier drugs, the skin disorder is completely eliminated,” concluded Dr. Spain.

For the authors of this research, “this new application opens new possibilities in the treatment of pemphigus vulgaris. To have found in mice is a preliminary step to later remove people.”

Given these results, the scientific journal that echoes the research also published an editorial written by a group of experts from several countries, praising the work and highlight its implications: “create new treatment options and new channels of research “.