New Topical Gel: Will This Cheaper, Easier Approach Totally Heal 'Butterfly Children' Disease in Patients?

Researchers at Stanford University have recently come up with a cheaper and easier treatment in the form of a topical gel that could bring longed-for relief to many people affected by a skin disease known as epidermolysis bullosa or EB.

A ScienceAlert report specified that this incredible new gel could help "close blistering wounds" associated with the unusual skin disease called "butterfly children disease."

As described in Mayo Clinic, EB is an inherited disease that causes the skin to be fragile. It can tear even at the slightest touch, like a butterfly's wings. Whereas some mild forms of this disease can improve with age, there is no cure for severe occurrences, which can eventually be deadly.

Gene Replacement for a Specific Collagen

Stem cell treatments have proven efficacy for some butterfly children cases. However, this invasive technique necessitates skin grafting, which is costly and requires anesthesia and hospitalization.

This newly formulated topical gel can be applied during the regular dressing of the wound. It also contains a therapeutic gene that is directly spread onto the skin.

This said treatment is effective by replacing a gene that encodes for collagen VII or C7, a protein that's missing in those with recessive dystrophic epidermolysis bullosa or RDEB.

This specific collagen anchors the foundational human skin structure, which holds the epidermis and dermis together. Minus C7 patients who have RDEB are spending their lives suffering from painful blisters, scarring, and even skin cancers.

Easy-to-Administer Gene Treatment

In this new study published in Nature Medicine, the authors specified that taken together, they demonstrate a "novel, easy-to-administer and highly accessible" gene treatment that can reverse genetic disease through repeated application directly to the patient's skin wounds.

If applied early enough in the wounding process, the gel has the potential to prevent further skin-tearing and scarring, thereby lessening the risk of skin cancer development and prolonging patients' lives. Other study investigators have attempted to make similar gels, although this is the first to pass rigorous clinical trials "with flying colors."

When applied to the wounds of nine patients with RDEB above six years of age, the gel exhibited substantial results in randomized, placebo-controlled trials.

All Wounds Healed

Three months and three doses later, all wounds applied with the topical gel had closed and healed. A few weeks after, they remained closed.

Meanwhile, wounds treated with placebo continued to heal, and then re-blister in a painful and vicious cycle. Only two wounds during the trial did not completely heal after three months of gel application.

Meanwhile, the placebo-treated wounds continued to heal and then re-blister in a vicious and painful cycle. Furthermore, one wound had been bothering a patient for roughly five years, although after a second three-month treatment cycle, the wound closed and stayed healed for eight months. Another wound that had persisted for four years and covered most of the side of a patient was 70 percent recovered with the help of the gel.

No Severe Negative Effect Observed

All patients' biopsies showed that the gel triggered collagen production as early as nine days into the trial. Even better, there were no severe negative side effects triggered by the gel, and it worked the same way for all age groups and genders.

According to Peter Marinkovich, a dermatologist from Stanford University, the wounds heal rapidly, but even more essentially, they remain closed.

He added, the treatment has strengthened the skin and broken the painful and destructive cycle of would opening and closing that patients with butterfly children disease experience.

A related report about the Butterfly Children Disease is shown on CNA Insider's YouTube video below:

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