Such a long journey – Europe approves retinal cell therapy based on decades of good science

Author: David Kent, 03/24/15

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At one of my very first Canadian Stem Cell Network Annual General Meetings, when I was just a couple of years into my stem cell biology training, I remember sitting down just after lunch when a jovial Italian man sauntered up to the stage and apologized to the audience for the timing of his talk. We were then treated to an incredible video display of an eye surgery that replaced damaged tissue in one eye with cells extracted from the other eye and grown outside the body – perhaps unsettling for some, but the exciting part for most was that the procedure showed that long term stable grafting of retinal cells resulted in improved vision. This was at a time in the early 2000s when stem cell therapies were only really starting to be talked about and here was a guy trying it in actual people. This scientist’s name was Michele de Luca and he is one of the big reasons why I believe that stem cell therapies can be developed in a highly regulated and successful manner.

I must admit, all sorts of alarm bells rang in my head at the time – too soon! Do they really know what the long term effects are? Are they showing us results from all the patients? Etc., etc… It turns out, though, that the principles were sound and the project announced some exciting news last month. The foundation of excellent science and long term support for many related projects over the years from both the public and private sectors made this decades long journey possible and this team of researchers has been granted the first European Commission “conditional marketing authorization” for a stem cell therapy.

A really nice description of the therapy and the process can be found on the EuroStemCell website which, by the way, is an excellent resource for those interested in stem cell and regenerative medicine developments across the pond. Briefly, the new therapeutic system, called Holoclar®, uses stem cells from an undamaged area of the patient’s eye to repair the damaged area. First, a tiny fraction of cells (~1-2 mm2) are removed from the undamaged eye in a small biopsy that is not classed as a surgery. These cells are then grown into a sheet of cells that look pretty much like a contact lens and that sheet is then transplanted onto the patient’s damaged eye. The cells then repair the damaged portion of the eye and prevent further damage to neighbouring cells.

This technique is particularly useful when a single eye is damaged as a result of an accident (e.g., chemical or physical burns) since the other eye offers the perfect source of donor tissue that will not be rejected because it is from the same patient. The therapy will become available to European patients soon, although a timeline has not been specified. It is quite exciting to see this come to the fore of medical applications and I hope to see many more based on the incredible amount of investment in stem cells and regenerative medicine over the last ten years.

On a final note, I feel it is important to highlight the role that Professor de Luca has played as a pioneer for evidence-based decision-making in the stem cell therapy world. The International Society for Stem Cell Research has recognized his contributions with their 2014 public service award with the fight against unproven stem cell therapies being one of his key areas of public service. Paul Knoepfler has written a nice piece related to the latter and awarded Elena Catteneo his 2013 stem cell person of the year award for her role in the same fight against the privately owned Stamina Foundation in Italy. Stories like this give you quite a lot of hope for stem cell therapies – I just hope that readers can appreciate the length of time and number of people required to advance things in a properly controlled and clinically validated manner. The journey from discovery to treatment requires care and patience.

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David Kent

David Kent

Principal Investigator at University of Cambridge
Dr. David Kent is a Principal Investigator at the University of Cambridge in the Cambridge Stem Cell Institute (http://www.stemcells.cam.ac.uk/researchers/principal-investigators/dr-david-kent). His laboratory's research focuses on fate choice in single blood stem cells and how changes in their regulation lead to cancers. David is currently the Stem Cell Institute’s Public Engagement Champion and has a long history of public engagement and outreach including the creation of The Black Hole in 2009. He has been writing for Signals since 2010.
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