The promising results from this study suggest that the treatment not only enhances quality of life and activity levels but also offers hope for reducing long-term complications like skin cancer.

Following the trial, Gabrielius has been able to participate in activities like playing football and riding his bike, demonstrating the significant impact of the treatment on his daily life.

A 12-year-old boy from East London with the severe genetic skin condition RDEB has shown remarkable improvements after participating in a clinical trial involving stem cell infusions, leading to faster wound healing, less inflammation, and reduced itching.

His mother reports that these benefits have significantly enhanced his quality of life, enabling him to engage in activities like playing football and riding a bike, and his sleep has improved due to fewer dressing changes.

RDEB is a rare, severe genetic disorder caused by a defect in collagen VII, resulting in extremely fragile skin prone to blistering, wounds, scarring, and an increased risk of skin cancer, affecting about 150 children in the UK.

Currently, there is no cure for RDEB, and treatment mainly focuses on managing symptoms and preventing complications, with the risk of developing skin cancer being a major concern.

The clinical trial involved administering mesenchymal stromal cells (MSC) derived from umbilical cord tissue via intravenous infusion to 30 children at Great Ormond Street Hospital and Birmingham Children's Hospital, aiming to promote wound healing and reduce inflammation.

Participants received either the MSC treatment or a placebo in a controlled, two-phase study funded by NHS England, the NIHR, and Cure EB, with the goal of assessing safety and efficacy.

Researchers, including lead scientist Dr. Anna Martinez, confirmed that the treatment was safe and effective, particularly in children under 10 and those with intermediate RDEB, showing reductions in pain, itching, and disease activity.

Family and advocacy groups are hopeful that MSC infusions will become part of routine care for children with RDEB in the UK, alleviating suffering and improving outcomes.

The treatment, called CORDStrom, is currently being considered for regulatory approval, with ongoing studies to gather further data on long-term benefits and potential disease modification, aiming to become a standard care option.

Supported by NHS England, the NIHR, and Cure EB, INmuneBio has committed to providing CORDStrom free of charge for one year for trial participants, with the hope of wider clinical use in the future.

Experts emphasize that early and regular treatment could improve quality of life, reduce disease activity, and potentially lower the long-term risk of skin cancer, paving the way for broader clinical adoption.