Clinical applications

Fibrin substratum prepared from a surgical glue. From Ronfard et al., TRANSPLANTATION 2000Stem cells have great potential for treating disease. In fact, skin stem cells have already been used for over 25 years to grow skin grafts for patients with extensive burns. However, problems still exist with these skin grafts, and we have a lot to learn before other types of stem cells can be used routinely in medicine.

OptiStem aims to pave the way for new stem cell therapies through a combination of basic stem cell biology, pre-clinical work and clinical trials.

Our clinical work focuses on potential treatment strategies for:

• Duchenne muscular dystrophy
• Blindness caused by destruction of the entire ocular surface (surface of the eye)
• Severe burns and degenerative skin diseases
• Severe damage to oral mucosa (thin layers lining the mouth)

Information for patients

Due to the experimental nature of these studies we cannot at the moment offer treatments; opportunities to participate in clinical trials will become available on the website at appropriate times. This website provides scientific information about our clinical research. If you are looking for general information on stem cells and their potential use in treating disease, please visit the Frequently Asked Questions section of EuroStemCell.org.

Pre-clinical work

Our pre-clinical work bridges the gap between fundamental stem cell biology and clinical trials. This part of our work includes:

• Detailed analysis of the long-term fates and in vivo efficacies of transplanted or engrafted cells in animal model systems
• Investigation of approaches to increase angiogenesis in damaged tissue to ensure sufficient nutrient and oxygen supply to regenerating tissue (a pivotal factor in efficient cell therapy)
• In-depth immunological studies to determine whether the stem cells we use elicit any adverse immune response
• Further studies to modulate the immune response and/or induce tolerance
• Development of reliable micro-surgical techniques

These investigations will allow us to optimize our protocols while progressing to clinical trials.

Clinical trials

We will carry out a number of early-stage (Phase I/IIa) clinical trials:

• Duchenne muscular dystrophy: Transplantation of pediatric patients with mesoangioblasts from healthy donors
• Duchenne muscular dystrophy: Transplantation of patients with CD133+ cells (genetically corrected stem cells from their own bodies)
• Ocular, urethral or oral epithelial reconstruction: Treatment of patients with grafts prepared using stem cells extracted from biopsies taken from their own bodies

Clinical trials will begin in 2010. More information will be posted here as the trials progress.