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Projects. Life and Matter Sciences

The transplant of bioengineered skin for cutaneous regeneration in patients with Dystrophic Epidermolysis Bullosa

Lead Researcher:
María José Escámez Toledano

Research Centre:
Universidad Carlos III de Madrid.


María José Escámez ToledanoThe goal of this project consists of the implementation of rational therapeutic tools to combat and/or relieve the symptoms of severe generalised Recessive Dystrophic Epidermolysis Bullosa (EBDRsg), a rare genetic disease which causes cutaneous fragility and has no cure. The functional deficiency of the Col VII protein plays a key role in cutaneous anchorage and is the main pathological cause underlying this disease.

Patients with EBDRsg develop multiple erosions distributed all over their body and which often become chronic due to the presence of sustained inflammation. One of the three proposed cell therapy strategies for the treatment of the chronic lesions was initiated during the first six months of the project. Originally the use of transplant of bioengineered skin containing allogenic fibroblasts (and autotransplanted epidermal stem cells) was considered, as these cells are able to supply a stimulus for the regeneration of the skin and an alternative and transitory source of collagen VII. Nevertheless, evidence has been accumulated indicating that the immunomodulating effect of mesenchymal 1 stem cells may be fundamental for the control of the persistent inflammation associated with chronic lesions. Therefore, in collaboration with the Cell and Gene Therapy Unit at the Niño Jesús Hospital in Madrid, accredited by the Ministry of Health for the production of health care products, the manufacture of sheets has begun based on human plasma containing allogenic mesenchymal human cells obtained through liposuction. The production protocol is currently being drawn up prior to being submitted for evaluation by the Spanish Medicines and Health Care Products Agency (AEMPS).

Scientific production
4 articles published in Journals
2 papers at  national conferences
6 papers at international conferences

María José Escámez Toledano

Degree in Biology from the Autonomous University, Madrid (specialising in Biochemistry and Molecular Biology). She studied for her doctorate in the Biomedical Research Institute (UAM-CSIC, Madrid). In 2001 she joined the CIEMAT, where she was awarded grants and contracts financed by private bodies (Marcelino Botín Foundation, Pfizer) as well as public ones (the European Commission. VI Framework Programme: life sciences, genomic and biotechnology for health), until she gained her current position as tenured scientist in the Regenerative Medicine Unit (Epithelial Biomedicine, Basic Research Dept.) and a research member of the CIBER for Rare Diseases (CIBERER). Her interests centre on the design of innovative intervention strategies in the field of skin regeneration, with a clear vocation for this to find clinical expression. Her achievements include the development of a preclinical model of human scarring which is highly useful for the design and validation of pharmacological strategies, as well as cellular and gene therapy. Once translated into a diabetic context (impeded scarring and chronic ulcers), this humanised model gave rise to a doctoral thesis (directed by Dr. Escámez). In 2007, she was involved in the implementation of a diagnostic platform and molecular study of a rare disease, Epidermolysis Bullosa (EB). This project, under the leadership of Dr. Del Río, is taking place within the context of CIBERER. Her participation in a pilot clinical trial for the treatment of patients with EB should also be underlined, within the new context of Advanced Therapies and financed by the Ministry of Health. In 2009 she received an award from the association of EB patients, DEBRA Spain, for her contribution to progress in the knowledge of EB and the improvement in patients' quality of life. In 2010 she made a decisive contribution to the launch of the new degree in Bioengineering of the UC3M, with which she remains linked as associate professor.

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