Institute of Child Health

Mr Paulo de Coppi, and colleagues today announce an innovative strategy for regenerating skeletal muscle tissue using cells from the recipient’s own body.  Mr de Coppi is a surgeon at Great Ormond Street Hospital and an academic at the UCL Institute of Child Health.

Damaged muscle tissues treated with satellite cells in a special degradable hydrogel showed satisfactory regeneration and muscle activity.  Muscle activity in repaired muscle in a mouse model was comparable with untreated muscles.  This is the first time muscle function has been proved by physiological tests.

This is published (online first) in the Journal of the Federation of American Societies for Experimental Biology (FASEB) and represents another impressive development in the growing field of regenerative medicine.

Satellite cells (SCs), freshly isolated or transplanted within their niche, are presently considered the best source for muscle regeneration.  They are located around existing muscles.  Hence, a patient’s own cells can be used, from a muscle biopsy.

A key issue for regeneration is how cells grow as a structure, they usually require some form of framework.  A hard framework would impede muscle growth and muscle cell penetration. The hydrogel, by contrast, provides a supportive structural skeleton but degrades quickly as muscle tissue returns and the support becomes unnecessary.   The gel is initially liquid, hardens in place under UV light, and is easily penetrated by muscle cells.

“We focused on a simple, robust, and reproducible technique that could be easily adapted to clinical requirements” said Mr De Coppi, who receives funding from Great Ormond Street Hospital Children’s Charity (www.gosh.org).

“This is using the patients own cells, without any lengthy culturing process, which means we could take a biopsy, produce the cells in a couple of hours, and implant them where needed - it can be done in theatre as one process.  Using the patient’s own cells eliminates any tissue rejection.”

“Nor are there ethical debates as for embryonic stem cells, or culturing and availability issues, such as amniotic stem cells.”  

The lab model has the potential to be translated into significant clinical benefit for babies and children born with defective organs, or caused by injury or pathological conditions, that currently require complicated and potentially devastating reconstructive surgery.   

The focus for initial clinical research in humans will be relatively small muscles at first, like deformities in the face and palate, or in the hand.  It will be technically more demanding to grow larger muscles with more structure, which would require their own nerves and blood supply.   

“We want to move to safe and effective human trials” said Mr de Coppi, “but of course we are not there yet.  Furthermore scaling up from small muscles to larger structures will undoubtedly be challenging.”

Professor Andrew Copp, Director, ICH said: ""This is a very exciting research finding that may significantly advance our ability to repair muscle damage or defects in future.  It is a great example of translational research in action, from the laboratory to a near-clinical application."

Dr Jane Collins, chief executive, Great Ormond Street Hospital said: "This is an exciting development, in a new area of medicine, which has the potential to help many children cared for by Great Ormond Street Hospital."

Contact information:

For further information please contact Stephen Cox, ICH press office, 0207 239 3119 or email coxs@gosh.nhs.uk