June 1, 2015
Stem cell infusions help children with inherited skin blistering
Promising results from a trial of a new stem cell-based therapy for a rare and debilitating skin condition affecting children was found to provide pain relief and to reduce the severity of this skin condition for which no cure currently exists.
The clinical trial, led by King’s College London in collaboration with Great Ormond Street Hospital (GOSH), recruited 10 children with recessive dystrophic epidermolysis bullosa (RDEB).
RDEB is a painful skin disease in which very minor skin injury leads to blisters and poorly healing wounds. The fragile skin in RDEB also scars, develops contractures and is prone to life-shortening skin cancers.
The Phase I/II EBSTEM trial, whose results appear early online in the Journal of Investigative Dermatology, was designed to test the safety of infusions of stem cells and to establish whether the treatment could help to reduce 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 mesenchymal stromal cells (stem cells) grown from the bone marrow of unrelated donors. Mesenchymal stem cells (MSCs) have been shown to home to wounded tissue and mediate wound healing in previous studies. Although the cells do not survive permanently, they may still deliver clinical benefits.
The children were then monitored for a year after the cell infusions. A range of monitoring tests showed no serious adverse effects in patients who received the therapy. The pain score (level of pain) reported was on average lower than before treatment, and the severity of the condition was also reported to have lessened following the infusions. Parents also reported better wound healing, less skin redness and fewer blisters.
Overall, the outcomes of the trial are promising, according to the study’s authors, although this is an unblinded study of participants that may introduce a positive bias in the information reported. In interviews families reported a range of benefits from having better sleep, a parent being able to return to work part-time because of reduced caring needs and a family being able to plan their first vacation together.
However, further work is needed to better understand the mechanisms involved – for example, whether the stem cells trigger the production of a variety of growth factors and immune system regulators — to stimulate wound healing and reduce inflammation in the skin. Further studies are also needed to confirm the efficacy of the treatment and establish the optimal dose of cells to give RDEB patients.
Learn more:
http://www.kcl.ac.uk/newsevents/news/newsrecords/2015/May/Stem-cell-therapy-for-inherited-skin-blistering.aspx
http://www.nature.com/jid/journal/vaop/ncurrent/full/jid2015158a.html
- See more at: http://stemcellsportal.com/stem-cell-infusions-help-children-inherited-skin-blistering?sthash.OdFN0t2U.mjjo#sthash.OdFN0t2U.Fddon2py.dpuf
Stem cell infusions help children with inherited skin blistering
Promising results from a trial of a new stem cell-based therapy for a rare and debilitating skin condition affecting children was found to provide pain relief and to reduce the severity of this skin condition for which no cure currently exists.
The clinical trial, led by King’s College London in collaboration with Great Ormond Street Hospital (GOSH), recruited 10 children with recessive dystrophic epidermolysis bullosa (RDEB).
RDEB is a painful skin disease in which very minor skin injury leads to blisters and poorly healing wounds. The fragile skin in RDEB also scars, develops contractures and is prone to life-shortening skin cancers.
The Phase I/II EBSTEM trial, whose results appear early online in the Journal of Investigative Dermatology, was designed to test the safety of infusions of stem cells and to establish whether the treatment could help to reduce 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 mesenchymal stromal cells (stem cells) grown from the bone marrow of unrelated donors. Mesenchymal stem cells (MSCs) have been shown to home to wounded tissue and mediate wound healing in previous studies. Although the cells do not survive permanently, they may still deliver clinical benefits.
The children were then monitored for a year after the cell infusions. A range of monitoring tests showed no serious adverse effects in patients who received the therapy. The pain score (level of pain) reported was on average lower than before treatment, and the severity of the condition was also reported to have lessened following the infusions. Parents also reported better wound healing, less skin redness and fewer blisters.
Overall, the outcomes of the trial are promising, according to the study’s authors, although this is an unblinded study of participants that may introduce a positive bias in the information reported. In interviews families reported a range of benefits from having better sleep, a parent being able to return to work part-time because of reduced caring needs and a family being able to plan their first vacation together.
However, further work is needed to better understand the mechanisms involved – for example, whether the stem cells trigger the production of a variety of growth factors and immune system regulators — to stimulate wound healing and reduce inflammation in the skin. Further studies are also needed to confirm the efficacy of the treatment and establish the optimal dose of cells to give RDEB patients.
Learn more:
http://www.kcl.ac.uk/newsevents/news/newsrecords/2015/May/Stem-cell-therapy-for-inherited-skin-blistering.aspx
http://www.nature.com/jid/journal/vaop/ncurrent/full/jid2015158a.html
- See more at: http://stemcellsportal.com/stem-cell-infusions-help-children-inherited-skin-blistering?sthash.OdFN0t2U.mjjo#sthash.OdFN0t2U.Fddon2py.dpuf