A new stem cell-based therapy has shown some very promising results. This therapy was designed to treat a rare and debilitating skin condition that affects children, for which no cure currently exists. This cell-based therapy provided pain relief and reduced the severity of the skin condition for patients who participated in the clinical trial.
The clinical trial was led by scientists at King’s College London, who collaborated with researchers from the Great Ormond Street Hospital (GOSH). They recruited 10 children afflicted with a disease called recessive dystrophic epidermolysis bullosa (RDEB).
RDEB is a painful skin disease in which very minor skin injury leads to blisters and wounds that tend to heal very slowly or not at all. The skin of RDEB patients is quite fragile and it tends to scar, develops contractures, and is also prone to life-threatening skin cancers.
This clinical trial, known as the EBSTEM trial, is a The Phase I/II trial whose results were published early online in the Journal of Investigative Dermatology. This study was designed to test the safety of infusions of stem cells and to determine if this treatment could help diminish the severity of the disease and improve quality of life for these patients.
During the first six months of the trial, participants were given three infusions of bone marrow- derived mesenchymal stromal cells from unrelated donors. Mesenchymal stem cells (MSCs) have been shown to home to wounded tissue and mediate wound healing in several previous studies. Although these infused stem cells do not survive permanently, they may still deliver therapeutic benefits.
The treated children were then monitored for a year after these cell infusions. Several different clinical tests failed to reveal any serious adverse effects in patients as a result of the stem cell treatment. When the pain levels of patients were measured, patients consistently reported lower pain levels after the treatment than before the treatment. Also the severity of their disease was also reported to have lessened following the stem cell infusions. Parents of these children reported better wound healing in their children and they also showed less skin redness and fewer blisters.
Overall, the outcomes of the trial are promising. However, this is an unblinded study of participants and may, therefore, contain positive biases in the way the information is reported. In interviews with families, participants reported a range of benefits from sleeping better, to the parents being able to return to work part-time because their children required less intense care. In fact, one family was actually able to plan their first vacation together.
Thus, further work is required to better understand the mechanisms that helped patients improve. Did the stem cells trigger the production of a growth factors and immune system regulators? Did these secreted compounds stimulate wound healing and reduce inflammation in the skin? Or did the presence of the cells somehow improve skin quality? Further studies are also required to confirm the efficacy of the treatment and establish the optimal dose of cells to give RDEB patients.