OPTISTEM

Optimization of stem cell therapy for clinical trials of degenerative skin and muscle diseases

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 Obiettivo del progetto (Objective)

'This proposal aims to develop and implement efficacy of clinical trials with adult, tissue stem cells for degenerative diseases of epithelia and skeletal muscle. Extraordinary progress in the understanding of several key features of stem cells has been rapidly translated into novel cell therapy protocols that have yielded positive results in pre-clinical models of genetic and acquired diseases and in the first clinical trials. Much remains to be done on the basic biology of stem cells to provide new tools for cell therapy trials. Also transplantation related issues, such as engraftment, angiogenesis, tissue remodeling and immune response remain to be addressed. In general, cell therapy necessitates a continuous cross-talk among biologists and clinicians as well as crossing boundaries among different tissues/diseases. Despite many structural and functional differences, epithelia and skeletal muscle share some key features such as spatially ordered cell architecture, centered on a supporting basal lamina to which stem cells are anchored. Indeed mutations in structural proteins linking the cell membrane to the basal lamina are responsible for similarly devastating diseases such as muscular dystrophies and epidermolysis bullosa. A multi-tissue approach allows jointly addressing and solving not only scientific and clinical problems but also regulatory and ethical issues related to these novel procedures. Finally, sharing high valued dedicated platforms such as large animal facilities and GMP cell culture facilities increases performance and reduces costs. Based on these considerations, a network of internationally recognized experts in muscle and epithelial cell biology, immunology, angiogenesis, and molecular pharmacology with two stem cell-based Companies has been assembled, creating a synergistic approach that will increase the chance of success for ongoing and future clinical trials with stem cells, that are integral part of this proposal.'

Introduzione (Teaser)

The objective of the OPTISTEM project is to develop new applications of stem cells for degenerative diseases. The primary focus of the project is on clinical trials (CTs) with epithelial and muscle stem cells.

Descrizione progetto (Article)

The organism makes new cells all the time. Some specialised cells, such as blood and muscle cells, are unable to make copies of themselves through cell division. Instead, they are replenished from populations of stem cells. Stem cells may also hold the key to replacing cells lost in many devastating diseases for which there are currently no cures.

'Optimisation of stem cell therapy for clinical trials of degenerative skin and muscle diseases' (OPTISTEM) is an EU-funded project investigating how stem cells could be used to treat degenerative diseases of skeletal muscle or epithelia such as skin or the surface of the eye. The project includes 18 partners from six European countries. OPTISTEM combines basic research about stem cells with pre-clinical work and CTs. The project began in March 2009 and will run for five years.

Primarily, project members aim to develop cell therapy trials for patients with muscular, skin or ocular disorders. To support and optimise CTs, large animal models are being used for in-depth investigation of the strategy. Small animal models and cell models are being employed to characterise pathways that control stem cell activity.

A preliminary validation study for children affected by Duchenne muscular dystrophy (DMD) has been completed. The CT based on transplanting mesoangioblasts (muscle stem cells) in DMD patients started in March 2011. In total, five patients have undergone the transplant and only one serious adverse event has been observed.

The first effective autologous cell therapy CT of stem cell-derived cornea transplantation has been completed, demonstrating the potential of stem cell therapeutics in treating a common disorder. Three patients with massive unilateral destruction of the corneal surface have been treated with eye stem cells from their healthy eye.

During the course of OPTISTEM a total of 10 patients with one or two blind eyes have been treated. So far, all patients treated had a substantial improvement of corneal transparency, an amelioration of the clinical signs and an improvement of visual acuity in the treated eye.

Researchers also identified a new type of muscle stem cell, novel methods to generate muscle stem cells from reprogrammed cells and new factors important for muscle formation. Results of the project were presented in more than 70 publications and are available on the http://www.OPTISTEM.org (project website) .

Stem cells have great potential for treating disease. OPTISTEM aims to find the way for new stem cell therapies through a combination of basic stem cell biology, pre-clinical work and CTs.