Clinical trial helping butterfly children

Epidermyosis bullosa patients in SA experience first relief from pain

SAN ANTONIO – A new FDA trial of a cream called Zorblisa is giving children who suffer from epidermyosis bullosa a chance to not only heal their wounds, but have hope surviving their childhood.

The children who suffer from epidermyosis bullosa, or EB, live lives of desperate suffering, with itching, stinging blisters all over their bodies. It’s a rare condition that makes their tissue so fragile that in some cases, they can’t wear shoes, eat solid food or even sweat. The term "butterfly children" is often used to describe children with EB because of the fragile nature of their skin.

Esmeralda Delagarza said when her son Tristan was born, they noticed the blisters under his diaper that would not heal, but no one would diagnose it as EB for years. In fact, it wasn’t until her daughter was born that a doctor made the connection. By then, Tristan, who suffers from a more severe form of the disease, had sores 6 or 7 inches in diameter on his back that would not heal.

"We went through so much. Before all this, there were nights when he couldn't sleep. He would get stuck to his bed at night. It was horrible," Delagarza said.

But this year, he and his sister volunteered to be a part of a fast-tracked clinical trial for Zorblisa. Both say it makes a big difference.

"To be a little kid and growing up with it and soaking in the tub just to take a bath, or just pull your skin off just sleeping, there's a big difference," Tristan Delagarza said.

He can now do things outside the house and wear jeans for the first time. His sister is wearing shoes comfortably for the first time.

"I'd like to see it progress and go worldwide and help other kids," Elizabeth Delagarza said.

Dr. John Browning with Texas Dermatology and Laser Specialists is one of the few doctors in the state who has ever treated the disease. He said the No. 1 effect of Zorblisa is to stop the constant itching, but it also prompts the wounds to heal faster and not come back. It’s hoped that continued use will help the EB children not fall victim to a host of deadly conditions that usually end their lives far too early.

"In its worst form, there is death from bacterial infection of the blood, airway compromise, metastatic skin cancer by the time you're an adult," Browning said.

For mothers like Esmeralda Delagarza, whose life used to consist of constant bandaging, bathing and care, she has hope for the first time.

"It's heart-breaking. But with this, there may be a difference. He may be able to be someone normal as an adult. You don't want to see your child suffer. There may be a chance for him to grow up and be an adult in the real world," she said.

Zorblisa is the first EB drug to reach stage 3 of clinical trials in the U.S. It is hoped that the FDA will clear it for use sometime this year.

To learn more about the disease, click here.