Recently, a tracking experiment by a research team at the University of Minthes in Germany [1] confirmed that the combination of stem cells and genes in the treatment of "butterfly disease" has a long-term effect and can make the skin stable for a long time!
"Butterfly disease": epidermal release bullosa
I think everyone is impressed by the beautiful picture of the "butterfly princess" dancing among the butterflies with incense! But butterfly disease is not a beautiful disease, it has a sense of horror.
The professional name of "butterfly disease" is Epidermolysis bullosa (EB), and one of the most frightening things about the disease is that the skin dissolves at the slightest touch.
EB is a very rare inherited skin disease that often begins at birth or in early childhood, with an incidence of about 1 in 50,000 newborns. The disease is mainly manifested by an increase in the fragility of the skin, as long as the skin is slightly rubbed, it can lead to the separation of the epidermis and dermis of the skin, while producing blisters and blood blisters, gradually ulceration and conjugation, resulting in the loss of most of the hand function.
Figure 1, Figure 2, Figure 3 (Images are from references[2])
Not only is EB severely ill, but it is also difficult to treat, often fatal due to severe infections. But medical scientists are always working hard to bring new hope to such patients.
Stem cell transplanted skin grows healthy and 100% stable for 6 years
In 2015, a joint effort by Germany and Italy successfully saved a patient with skin surface damage of up to 80% through stem cell transplantation and genetic programming. This is also the first successful case of treating such a difficult "butterfly disease".
Stem cell therapy for EB is a very objective and attractive method. The most attractive thing about stem cells is that they have immunomodulating, stimulating tissue regeneration, reducing fibrosis (that is, preventing scarring), and migrating to damaged tissue. Therefore, there is a lot of potential in the treatment of EB.
Dr. Kückelhaus and Professor Tobias Hirsch of the University of Minters in Germany followed up a 7-year-old EB patient who was treated with stem cell transplantation and gene programming for a long time, and the results after 6 years confirmed that the treatment method was successful, and the transplanted skin of the boy grew well and was 100% stable.
At the same time, Professor Hirsch said that after achieving this positive and promising result, extensive clinical research will be further carried out, with the aim of making stem cell transplantation combined with gene programming therapy an option for more EB patients.
In 2016, Temcell, a bone marrow mesenchymal stem cell product for graft-versus-host disease, launched in Japan, is being extended to epidermolysis bullosa (EB), and its clinical research results are worth looking forward to.
Mesenchymal stem cell infusion therapy is effective in reducing the severity of EB
In addition, in 2021, a team of researchers at Yonsei University in Seoul, South Korea, who received intravenous infusion of mesenchymal stem cells in six EB patients (four adults and two children) and followed them up for 24 months, found that infusion of mesenchymal stem cells may be effective in reducing the severity of EB in patients, including reducing body surface area damage, the number of blisters, and pain and itching [3].
Figure 4 (Image reference[3])
Most patients achieve at least 50% healing in the 56 days after treatment, and it can be seen that intravenous infusion of mesenchymal stem cells also has a positive effect on the treatment of EB.
Stem cell therapy has brought new hope to this terrible disease, and also allowed more children to live freely in the sun like other children, play happily with their friends, and no longer fear. After years of hard work, we can find stem cells in various fields, just like the classic saying in this year's "Heartwarming Offer - Medical Student Season", "stem cells have unlimited possibilities".
bibliography
[1] Kueckelhaus, M., et al. Transgenic Epidermal Cultures for Junctional Epidermolysis Bullosa — 5-Year Outcomes. N Engl J Med 2021; 385:2264-2270. DOI: 10.1056/NEJMoa2108544.https://www.uni-muenster.de/news/view.php?cmdid=12229.
Meng Baoxi,Jian Yuluo,Wang Lu. Surgical methods for correcting hand scar contracture deformity in epidermolysis bullosa[J]. Chinese Journal of Aesthetic Plastic Surgery,2021,32(07):400-403.http://lib.cdutcm.edu.cn:7001/rwt/CNKI/https/NNYHGLUDN3WXTLUPMW4A.
[3] Lee Sang Eun,Lee SeungJu,Kim SongEe,etal. Intravenous allogeneic umbilical cord blood-derived mesenchymal stem cell therapy in recessive dystrophic epidermolysis bullosa patients. [J]. JCI insight,2021,6(2).https://kns.cnki.net/KNS8/manage/export.html?displaymode=GBTREFER.