Innovative Gene-Therapy Gel Promising for Blistering Skin Disease (Cure for ‘Butterfly Children’?)

A team of researchers from Stanford has been developing gene therapy for epidermolysis bullosa or the butterfly disease for more than a decade.

The newly-developed gel is said to help their wounds heal and remain healed. This was acquired from results in their clinical trial.

During the trial, the gene therapy gel was applied to the wounds of nine people, three of them were kids, with this condition. 

The gel helped heal their wounds and these healed skin areas remained like that for several months. 

Innovative Gel for Treatment of Blistering Skin Caused by Butterfly Disease

The trial is the first of its type that shows the effectiveness of gene therapy vectors for skin illness when topically applied. What’s more, it’s also the first trial of gene therapy in kids with the condition.

It’s amazing that a big, 10-year-old wound covering the side of a patient’s body was healed 70 percent whereas all of the other wounds closed entirely thanks to the treatment. 

Another patient with a chronic wound that has been going on for five years experienced entire closure after two rounds of therapy and stayed closed throughout the eight-month period of monitoring. 

According to Peter Marinkovich, MD and director of Stanford Medicine’s Blistering Disease Clinic, the wounds healed fast, but more importantly, they remained closed. 

This therapy, he explained, makes the skin stronger and ends the continuous and destructive wound opening and closing cycle that is common in patients with this disease.

What Kind of Gel Is This?

This gel is described as stable at room temperature and it can be applied to the skin without any expertise during the routine changes of bandages. This is an amazing advantage for patients globally who can’t get specialized care. 

Marinkovich who is an associate professor of dermatology and senior author of the study published in Nature Medicine journal explains that the nine people in the trial had a form of this disease known as recessive dystrophic epidermolysis bullosa or RDEB. 

These people have a gene mutation that encodes a protein known as collagen VII. 

This protein binds the middle and outer layers of the skin together. Without it, the layers will slide across each other, resulting in blisters that develop into painful wounds that stay open.

This condition is also known as butterfly disease because the skin is so fragile that even the slightest touch can trigger blisters. These people also have an increased risk of infections and skin cancer.

Unfortunately, many of them die in early adulthood. 

For more than ten years, the researchers at Stanford Medicine have been working on therapies that would deliver a copy of the gene known as COL7A1 to patients with this debilitating condition.