A pioneering treatment with stem cells improves the symptoms of a rare disease

Dystrophic epidermolysis bullosa causes erosions, blisters, inflammation of the skin and mucous membranes, among other conditions

This innovation is the result of scientific collaboration between the hospital Arrixaca and Dr. Robert Sackstein

The Virgen de la Arrixaca hospital carries out a pioneering treatment of cell therapy with modified mesenchymal stem cells in a rare and incurable disease such as dystrophic epidermolysis bullosa.

Dystrophic epidermolysis bullosa is a very rare congenital disease, produced by mutations in the gene COL7A1, which causes the lack of collagen 7, the protein that serves as anchor between the dermis and the epidermis, so these patients have erosions, blisters, Persistent inflammation of the skin and mucous membranes and suffer infections, malnutrition, pain and increased risk of skin cancer.

For this pathology there is no curative treatment and the therapy is based on avoiding infections, and performing extensive, frequent and costly cures both for families and for the health system.

A possible alternative therapy includes treatment with mesenchymal stem cells, which are capable of synthesizing collagen 7, have anti-inflammatory properties and stimulate the regeneration of wounds.

Advances

For years, the Hematology Service of the Virgen de la Arrixaca Hospital, led by Dr. José María Moraleda, has been working to make available to patients access to research drugs based on cells, including mesenchymal stem cells.

In recent years, this line of research has received an important boost thanks to the scientific collaboration established with Dr. Robert Sackstein of Harvard University, recently appointed Doctor Honoris Causa by the University of Murcia, which is committed to the modification of cells by fucosylation to direct them towards damaged tissues.

Fucosylated mesenchymal stem cells can get better to the skin and are more potent.

In this specific case, the infusion of the cells donated by the patient's mother was carried out in the pediatric ICU of the Virgen de la Arrixaca hospital.

Subsequently, he remained under observation with the pediatric oncohematology team and was discharged without complications.

After the initial observation that the treatment is safe, in the coming weeks two additional infusions will be made according to the protocol, and a continuous clinical and biological follow-up of the patient will be carried out;

in order to detect improvements in the natural evolution of the disease that open a door to hope in the treatment of epidermolysis bullosa.

Next month the infusion will be done in a second patient.

Investigation

The Murcian Institute of Biosanitary Research (IMIB), has among its platforms a white cell production room, coordinated by Dr. Ana María García-Hernández, in which the technology of mesenchymal stem cell fucosylation is exclusively available. that allows this experimental treatment, for the first time, in a human being with this disease.

This milestone is the result of the collaboration of a multidisciplinary and multi-center team made up of health professionals from the Carlos III University of Madrid, the Dermatology Service of the La Paz Hospital in Madrid, and the Clinical Genetics, Dermatology, Oncohematology and Pediatric ICU Units of the Virgen de la Arrixaca hospital.

Source: CARM

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