A 12-year-old boy from East London, suffering from a rare skin condition that causes damage even with the slightest friction, has been given a glimmer of hope after participating in a trial for the first potential treatment for the genetic disorder.

Gabrielius Misurenkovas was diagnosed with an inflammatory disease known as recessive dystrophic epidermolysis bullosa (RDEB) when he was just a baby.

Thanks to a groundbreaking trial where he received specific stem cells via an intravenous drip, many symptoms of this painful condition have been alleviated. This comes as parents paid tribute to their daughter, who died after being left alone by her carer who fled the country.

This rare genetic disease is severely debilitating, causing extremely fragile skin which is easily damaged, leading to blistering, deep wounds and scarring. Approximately 150 children in the UK are affected by RDEB, a condition that not only causes pain but also severe itchiness.

With no disease-modifying treatment currently available, patients and their families manage symptoms by dressing wounds and applying eye ointments.

Over time, the condition can lead to severe complications and many patients develop a type of skin cancer called squamous cell carcinoma when they are young adults – this is the most common cause of death for RDEB patients.

Gabrielius has been under the care of medics at the world-renowned children's hospital Great Ormond Street (GOSH) since he was an infant.

When the opportunity to join the clinical trial was suggested by his doctors, he said he jumped at the chance.

"I wanted to take part in the trial as I was excited to see if it could help improve my condition and so I could do more of the things I enjoy, like playing football and spending time with my friends," he shared.

The multilingual youngster, who is a big fan of football legend Lionel Messi, required his bandages to be changed thrice daily, or even more frequently if he sustained an injury.

His mother, Jolita Cekaviciene, proudly stated: "Gabrielius did really well on the trial. His wounds healed quicker, and his skin was less red and inflamed.

"His skin was also less itchy which also reduced him scratching.

"His sleep was also less disturbed as he didn't need to have his special wound dressings changed as often, so this was a great benefit."

She revealed that Gabrielius has managed to engage in a few activities that were previously impossible for him before the treatment, including participating in some school sports, cycling his bike on the grass in the park, and playing football with his mates, using a soft ball.

Ms Cekaviciene acknowledged that the trial has granted her son more freedom than before, but he still needs to exercise caution during any activity to avoid falls or bumps.

Gabrielius was referred to GOSH as an infant after medical professionals noticed a wound at his birth, and he was diagnosed with RDEB when he was just two weeks old.

Ms Cekaviciene recalled: "The first two years were really hard as you had to watch him constantly – even rubbing his eyes could cause painful blistering.

"He couldn't even tell us how much pain he was in or where he was hurting."

RDEB is a genetic condition caused by a fault in the gene that produces collagen VII, a protein that binds skin layers together.

Doctors wanted to evaluate if regular infusions with mesenchymal stromal cells (MSC) could improve symptoms for children with this condition, following studies indicating that MSC treatments might promote wound healing, reduce inflammation and stimulate tissue regeneration.

In the trial, funded by NHS England, the National Institute for Health and Care Research and the charity Cure EB, children were administered a treatment called CORDStrom, produced by INmuneBio.

Gabrielius is one of 30 children who participated in the new clinical trial at GOSH and Birmingham Children's Hospital. Patients would enter the hospital and receive a drip delivering the cells over a period of 10 to 15 minutes.

In the study, patients were divided into two groups, with about half receiving the treatment, derived from umbilical cord tissue, as an infusion, twice over two weeks. The other half were given a placebo.

They then had a nine-month gap, referred to as a wash-out period, during which the group initially given the dummy drug received the treatment, while the group that first received therapy were given a dummy drug.

The study, published in the journal eClinicalMedicine, reported a positive outcome of the treatment. The most significant improvements were observed in children under 10 years old, and in those children diagnosed with the intermediate form of RDEB.

Families participating in the research reported beneficial effects on everyday life and wellbeing, with decreases in both discomfort and itching.

The therapy was described as being well accepted by the youngsters.

INmuneBio have committed to supplying CORDStrom at no cost for 12 months to all children who participated in the trial, enabling researchers to continue monitoring them, with patients anticipated to receive two infusions of the therapy every four months.

The firm is pursuing regulatory clearance for the treatment to enable its approval for use in the UK and other nations.

Lead investigator of the research, Dr Anna Martinez, paediatric dermatologist at GOSH, commented: "Our study has demonstrated that CORDStrom was safe and can help reduce disease activity in some patients with RDEB.

"Administering the treatment early and at regular intervals appears to reduce itch and improve wound healing, which over time we hope will reduce inflammation, modify the condition and may reduce over time the future risk of squamous cell carcinoma.

"It's fantastic to see how the patients, like Gabrielius, had improvements in their symptoms and their quality of life."

She further stated: "The 12-month open label study will allow us to collect more data to hopefully show an increased benefit from treatments administered in children and young people with RDEB, to continue research in support of the treatment being rolled out more widely."

Sharmila Collins, founder trustee of Cure EB, expressed: "Since funding the first trial and by contributing to Mission EB we have been working towards bringing MSC infusions forward for all children in the UK with RDEB.

"We hope these cells will become part of routine clinical care to alleviate the significant suffering of children with this condition."