OPTISTEM

Prof. Giulio Cossu

Scientific Coordinator of OptiStem, based at Stem Cell Research Institute at San Rafaelle, University of Milan, Italy

Research field

Stem Cells, Degenerative Disease Therapies

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Who?

Prof. Cossu: “My research focusses on cell and developmental biology of skeletal muscle as a basis for new cell therapy protocols for muscular dystrophies. Researchers and clinicians have joined forces in OptiStem to carry out a first in-man clinical trial for Duchenne muscular dystrophy."

Why?

Most genetic disorders still lack effective therapy. They create a life-altering experience for the person with the disorder and for their families, friends and employers and have an economic impact in Europe. "There is a need for novel regenerative therapies to treat these diseases", says Prof. Cossu.

What?

OptiStem is a large-scale integrated project funded by the European Commission. It aims to test new stem-cell-based approaches to treating degenerative diseases of the skeletal muscle (such as Duchenne muscular dystrophy) and diseased or damaged epithelial tissues, such as skin or the surface of the eye.

How?

OptiStem is a research consortium comprised of 18 partner institutions from six European countries. As such, we bring together leading scientists, companies and clinicians with expertise in basic stem cell biology, immunology, preclinical research and clinical trials. We aim to drive progress towards the clinic.

From fundamental stem cell biology to clinical trial

Mesoangioblasts, the cells used for transplantation in the Duchenne muscular dystrophy phase I/II clinical trial

Image credit: "Giulio Cossu"

A GMP facility is necessary to produce clinical grade cells suitable for transplantation

Good Manufacturing Practice Facility (Image credit: MRC Centre for Regenerative Medicine)

Satellite cells are the main stem cells responsible for muscle regeneration. However, OptiStem researchers have found other cells types that also contribute to muscle regeneration

A satellite cell on a muscle fibre (Image credit: "Pete Zammit")

OptiStem’s comic about stem cells, Hope Beyond Hype

OptiStem produced a comic to engage citizens in stem cell research and the process of turning a research idea into a therapy (View the whole comic at www.eurostemcell.org/hopebeyondhye)

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OptiStem project web page Eurostemcell web page Hope Beyond Hype In Focus 'Me, my stem cells and I'

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Background image: OPTISTEM

Portrait of the project coordinator: OPTISTEM

Timeline (in chronological order): 1961: Wikimedia Commons; 1974: Wikimedia Commons, Jacopo Werther; 1983: Wikimedia Commons,United States Navy; 1998: Wikimedia Commons, Ryddragyn; 2010: GNU Free Documentation License