Boy, 12, with rare genetic skin disorder can now play football after breakthrough medical trial
A rare, painful skin condition was given the freedom to play football and cycling through a new clinical work.
Gabrielius Misurenkovas has suffered from recessive dystrophic epidermolysis Bullosa (RDEB) since her infancy.
The inflammatory disease is seriously weakening and people with it have an extremely fragile skin with easily damaged, swelling, deep wounds and scarring.
In the UK, only 150 children are affected by genetic disease, but should be managed without treatment, by dressing the wounds of symptoms and applying eye ointments.
Gabrielius jumped to chance when he offered the chance to participate in a clinical research on Great Ormond Street (Gosh).
“I wanted to participate in the hearing because I was excited to see if he could help improve my situation, and so I can do more than things I enjoy playing football and spending time with my friends,” he said.
Young, who can speak three languages and a big fan of football player Lionel Messi, needed more if their bandages changed or injured three times a day.
However, since the hearing, he has managed to do a few things he could not do before treatment, including participating in some sports at school, riding a bike in the park on the grass and playing football with a soft ball with his friends.
“Gabrielius was really successful at the hearing. Wounds healed faster and his skin was less red and inflamed.
“His skin was less itchy, which reduced his scratch. He was less uncomfortable because he did not need to change his special wound sauces frequently, so this was a great benefit.”
Ms. Cekaviciene said that the hearing gave her son more freedom than before, but she should be careful in any activity to avoid falling or hitting.
Gabrielius was referred to Gosh as a baby after seeing a wound when medical officials were born and RDEB was diagnosed at two weeks.
Over time, the situation can lead to serious complications, and when many patients with conditions are young adults, it develops a kind of skin cancer called squamous cell carcinoma – this is the most common cause of death for RDEB patients.
Cekaviciene said: “The first two years it was really hard to watch him all the time – even rubbing his eyes could cause painful swelling.
“He couldn’t even tell us how painful or pain he suffered.”
Gabrielius is one of the 30 children who participated in the new clinical study at Gosh and Birmingham Children’s Hospital, where he took certain stem cells with an intravenous drop.
In the study, medical officials evaluated whether it improves symptoms for children with regular infusions with mesenchymal stromal cells (MSC), and then the studies suggest that this treatment could promote wound healing, reduce inflammation and stimulate tissue regeneration.
Patients go to the hospital and have a drip that gives cells more than 10 to 15 minutes.
NHS England, National Health and Care Research Institute and charity therapy financed by EB, children were given a treatment called Cordstrom produced by Inmunbio.
Inmunebio has agreed to offer cordstrom free of charge for all children who participated in the experiment, so that the researchers may continue to examine them and patients are expected to receive two infusion of treatment every four months.
The company is looking for regulatory approval for treatment to be approved for use in the UK and other countries.
