A new gene therapy gel is the first approved treatment for the most serious condition of “butterfly disease,” a rare and painful skin blister condition. announced by the US Food and Drug Administration (FDA). Friday (May 19th).
Dystrophic epidermolysis bullosa (DEB) is a genetic disorder that predisposes the skin to blistering and chronic painful bruising. This scar increases the risk of life-threatening infections, deformities of the extremities, and a skin cancer called squamous cell carcinoma.
A genetic mutation prevents the patient from making certain types of collagen in the skin. This collagen usually holds different layers of skin together and prevents them from rubbing against each other and causing blisters.
The newly approved gene therapy, called Vižvek (generic name Beremagene Gepelpavek), works by delivering a working copy of the defective collagen gene directly into the patient’s cells. The treatment itself is a gel containing herpes simplex virus type 1, a type of herpes virus that has been modified so that it cannot replicate in human cells and has two copies of the collagen gene, COL7A1.
Related: Gene therapy: what is it and how does it work?
Gel effect was tested with 2 clinical trials The study included approximately 40 adults and children with either a dominant or recessive form of DEB, the latter causing much more severe symptoms than the former. In both trials, the gel significantly improved the rate of wound closure in patients compared to the placebo gel. The treatment also helped prevent the skin from blistering again quickly, as the working copy of COL7A1 remained inside the skin cells for some time.
According to the FDA, the most common side effects included itching, chills, redness, rash, cough and runny nose.
The gene therapy gel is now approved for use in DEB patients over six months of age, and medical professionals can apply the treatment in the clinic or at the patient’s home, according to the company. crystal biotechthe sponsor of the trial.
“Until now, doctors and nurses have had no way to stop blisters and scars forming on the skin of patients with dystrophic EB, but have been forced to bandage and helplessly watch as new blisters form.” said the investigator. Dr. M. Peter MarinkovicThe director of the Bullous Disease Clinic at Stanford Healthcare and an associate professor of dermatology at Stanford University School of Medicine, said in a statement from Crystal Biotech.
“because [the drug is] It is safe, easy to apply directly to the wound, and does not require much support skills or expertise, making VYJUVEK easily accessible to patients who live far from specialty centers.” he said. Marinkovic previously told Live Science: