Breakthrough trial opens door for rare skin disease

A 12-year-old with recessive dystrophic epidermolysis bullosa gains new freedom to play after a stem cell trial in the UK.

Rare skin disorder boy plays football after breakthrough trial

A 12-year-old boy with recessive dystrophic epidermolysis bullosa, a rare and painful skin disorder, has gained more freedom to play after a new stem cell trial. Gabrielius Misurenkovas took part in a study at Great Ormond Street Hospital in London and Birmingham Children’s Hospital, where 30 children received mesenchymal stromal cells through an intravenous drip. The trial used CORDStrom, a product from INmuneBio, and is funded by NHS England, the National Institute for Health and Care Research, and the charity Cure EB. Researchers are studying whether regular infusions can reduce inflammation and promote wound healing.

Family reports that the treatment led to faster wound healing, less redness and itching, quieter nights, and a chance for Gabrielius to join sports at school with a soft ball. He can now ride his bike and play with friends more easily, though care is still needed to avoid injuries. The company has agreed to provide the therapy free for a year, and regulators will decide if the treatment should be approved for broader use in the UK and beyond.

The story highlights how public health funding can support bold advances in rare diseases. It also underscores a shift toward cell-based therapies that blend public oversight with private development. If the benefits endure and scale, this could ease a liveset of burdens for families once defined by daily care and medical visits. Yet long-term safety, cost, and access remain open questions that will shape whether this progress reaches all children who need it.

Highlights

More work lies ahead to ensure safe, affordable access for all eligible children.