A 12-year-old boy with a rare, painful skin condition can now play football and ride his bike after taking part in a new clinical trial. Gabrielius Misurenkovas has recessive dystrophic epidermolysis bullosa (RDEB), a genetic disease that causes extremely fragile skin, leading to blistering, deep wounds and scarring.
Only around 150 children in the UK are affected by RDEB, which has no available treatment. Symptoms are managed by dressing wounds and applying eye ointments. Gabrielius needed his bandages changed three times a day, or more if injured.
He joined a trial at Great Ormond Street Hospital (GOSH) and Birmingham Children's Hospital, receiving infusions of mesenchymal stromal cells (MSC) via a drip. The treatment aims to promote wound healing, reduce inflammation and stimulate tissue regeneration.
Since the trial, Gabrielius's wounds heal quicker, his skin is less red and inflamed, and he is less itchy. He can now take part in some sports at school, ride his bike on grass, and play football with a soft ball. His mother, Jolita Cekaviciene, said the trial has given him more freedom, though he must still avoid falls or bumps.
Gabrielius was diagnosed with RDEB at two weeks old. The condition can lead to severe complications, including a type of skin cancer in young adults. The trial, funded by NHS England, the National Institute for Health and Care Research and charity Cure EB, involved 30 children. The manufacturer, INmuneBio, has agreed to provide the treatment free for a year for all participants.
