Clinical trial of mesenchymal stromal cells in Recessive Dystrophic Epidermolysis Bullosa
Lay summary
Mesenchymal stromal cells (MSCs) are adult stem cells isolated from umbilical cord blood, bone marrow and other sources. MSCs can transform into other types of cells including skin and corneal cells.
The EBSTEM trial was the first UK trial aiming to lead to a stem cell treatment for children with Recessive Dystrophic Epidermolysis Bullosa (RDEB). It sought to establish if MSCs, obtained from bone marrow from parents or unrelated donors without EB, can benefit children with RDEB in a safe efficacious way.
Ten children with RDEB took part in the trial. MSCs were injected into the blood stream of the participating children, on three occasions over the period of one month. The trial led to reduced skin inflammation, reduced blistering and better wound healing.
Find out more about Sohana’s experience of taking part in the EBSTEM trial.
Scientific Summary
Ten children, between the ages 1 to 11 years, with RDEB received three intravenous infusions of bone marrow MSCs on days 0, 7, and 28 at a dose of 1–3×106 cells / kg. Patients were followed up monthly for 6 months, followed by 2 further safety assessments (one in person, one by telephone) up to 12 months after the last infusion. Telephone interviews with the parents of trial participants were conducted at 9 months to explore perceptions of participation in the trial, the impact of the treatment on both the child and family life.
In terms of safety, 163 adverse effects were reported, but at least 78% of these were related to RDEB and not to the MSCs. Most were DMSO odour, which resolved within 48 hours. There were 2 cases of nausea, 1 of abdominal pain and 1 of bradycardia, all of which resolved without treatment within 15 minutes.
Laboratory assessments did not reveal any adverse impacts of treatment with BM-MSCs on renal, liver or bone marrow function. Enzyme-linked immunosorbent assay (ELISA) showed anti-collagen VII antibodies in 9/10 subjections at baseline – this was unchanged post-MSCs.
Qualitative responses revealed better wound healing and quality of life improvements for both the patient and family (e.g. better sleep, reduced caring needs time, etc). Although given the unblinded nature of the trial, there was potential for positive information bias.
Publications
Petrof G., et al. Potential of Systemic Allogeneic Mesenchymal Stromal Cell Therapy for Children with Recessive Dystrophic Epidermolysis Bullosa. J Invest Dermatol. 2015;135(9):2319-2321
Children on the EBSTEM trial with Professor John McGrath
