Interview with Graziella Pellegrini – Using stem cells to cure blindness

Graziella PellegriniProfessor Graziella Pellegrini is one of the principal scientists on the ground breaking, corneal repair system Holoclar ®.  Working throughout Italy over the past 27 years she is now based at the Centre for Regenerative Medicine “Stefano Ferrari” at the University of Modena and Reggio Emilia. Her early work focused on cell differentiation and tissue engineering before moving to the Laboratory of the Epithelial Stem Cell Regional Research Center in Venice in 2002.  Since then she has been focusing on epithelial cells, with her current work based largely on the ocular surface of the eyes.  Working alongside Michele De Luca and others they have achieved Europe’s first approved advanced therapy medicinal product (ATMP).  This system is now in the process of being integrated into health services, to be available for patients across Europe in the future.

What does the approval of Holoclar®mean for regenerative medicine in Europe?

This approval is great news, following many years of work from myself and the other members of our team.  It’s particularly important because it means that this treatment will be available to patients across Europe.  The approval is particularly important with respect to delivery.  It allows the corneal epithelia, in culture, to be transported throughout Europe.  Previously, anyone requiring this kind of treatment would have to come to Italy.  This is, of course, difficult for people who have reduced visual capabilities, as they are often unable to travel alone.  It also excluded many people who were unable to afford the travel.  Therefore, this approval allows a higher number of people to be treated throughout Europe in the comfort of their own cities and countries and in their native languages.  Finally, it raises the profile of stem cell technology in the media and the public.  I feel it will be an important tool in future therapy and can be used safely if properly managed, which is important for the public to be aware of. 

How did your collaboration with Professor De Luca and the others involved in this project come about and how long have you been working together?

Centre for Regenerative Medicine “Stefano Ferrari”
Image: Stefano Ferrari, Centre for Regenerative Medicine

Professor De Luca and I originally worked together at the national cancer institute in Genova before teaming up to work on corneal epithelial cells.  We started on this research as a ‘proof of principle’ and produced a prototype, observing two initial patients for 2 years.  It was then obvious to us that this was something we could develop on a larger scale, but it was also obvious how delicate and fragile our materials were.  This work was in close collaboration with a very important ophthalmologist, who was critical in the clinical progression of this technology, Paolo Rama, who has continued to work alongside our team since.  Michele and I have worked separately on our own projects over the years, but our careers seem to have run in parallel since the 90s, while working on the research that went towards Holoclar®.  In 2007, new European rules came into force on ATMPs; this was when we moved to the Centre for Regenerative Medicine in Modena.  We were given the opportunity to work with the designers, to ensure the building and its facilities were exactly what we needed for our research.  It was during this move that we met Andrea Chiesi, a young, pharmaceutical entrepreneur.  He decided to get involved, beginning a new plan, which was very different to his usual work within the business.  We then all worked together towards the approval and certification of the therapy.  Andrea Chiesi didn’t just provide financial backing; he was also very involved in the decision-making process.  The team we had formed weren’t just working together, we were also suffering together through the harder times, suffering together definitely binds people strongly.  I think this was very important for us as a team and I think we worked better together because of it.  I think we have managed to be successful as a team because the 4 of us (Pellegrini, De Luca, Rama and Chiesi) were able to put aside our individual aspirations and work towards a common goal.  I would even say the close collaborations involved were a bigger success than the approval, as it’s difficult to work so closely for such a long time.  Professor Chris Holloway is another important collaborator involved in working towards approval and certification.  He was incredibly gracious and patient during such a tense time for us.  We cannot thank him enough for his patience.

What do you aspire to do next – following the success of Holoclar®?

The future doesn’t seem so far away anymore, as many of these projects have been going through the early ‘research’ stages recently and are on going.  We are currently focusing on gene therapy of epidermolysis bullosa (or ‘butterfly children disease’), a rare skin disorder where the patient has detachment of the skin from the entire body, somewhat similar to a burns patient.  We currently have approval for the phase one/two clinical trials, which is starting after the first proof of principle, published on Nature Medicine in 2006.  It is, however, a rare disease so we only have a handful of patients.  We are, so far, seeing positive results.  There is still a lot of work to go into this project before this can become a regular treatment for sufferers of this disorder; which is the end goal of this project.  I am also working on the very early stages of treatment of genetic diseases of the ocular surface, where there are no limbal stem cells available to create a culture using the Holoclar® method.  This causes bilateral blindness.   

What piece of advice would you give to a young scientist starting out in the field today?

Never give up.  You have to be sure that this is what you want to do and that you enjoy doing it.  You have to think in the terms that there is a solution and you just have to find it.  Constantly working towards the end goal, no matter how far you are from it.  You have to be willing to keep trying if something hasn’t worked and to try a different approach.  I know this is nothing new, but I think it’s very important for people to remember.  It’s what I tell the others working with me in the lab.  I also think that working together in a team, especially a mixed gender team is beneficial as you get different perspectives on a problem.  I would say that if you are not determined you can never reach the end of the story and building the entire path is usually the best part.

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