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Pete Coffey: Driving stem cells to the clinic

Pete Coffey*

Pete Coffey (Image copyright: UCL)

Professor Pete Coffey, Professor of Cellular Therapies at the Institute of Ophthalmology, University College London, is an MRC-funded researcher who is developing a stem cell therapy for a degenerative eye condition that is the leading cause of blindness in UK adults. He spoke to Katherine Nightingale about the long road to the clinic.

Researchers seldom like to predict how long it might be before their discoveries are tested in patients. “At least five years” is a typical response, but one that should usually be taken with a pinch of salt. Research is complex, there are many obstacles to overcome, and some promising ideas never get anywhere near a clinic.

All the more surprising then that Pete Coffey gave himself and his team five years from 2007 to ready a stem cell therapy for a degenerative eye condition for clinical trials. And perhaps more surprising still — he’s done it.

The condition in question is age-related macular degeneration (AMD). Most people with AMD have the ‘dry’ form, which occurs when a carpet of cells behind the retina start to die. These retinal pigment epithelial (RPE) cells nourish the ‘seeing’ cells of the retina, as well as removing dead cells that would otherwise build up and cause damage. People with AMD gradually lose sight from a part of the retina called the macula, which is responsible for sharpness of vision in the centre of the visual field — the vision needed for reading, driving and recognising faces. There is no treatment for dry AMD.

But if everything goes to plan, Pete’s work will change this. His London Project to Cure Blindness, established in 2007 after a large donation from an anonymous individual, set itself the challenge of developing a stem cell therapy for dry AMD by the end of 2012. The ultimate aim is to replace the dying RPE cells before any damage is caused to the seeing cells, therefore keeping vision intact.

Through the hoops

Pete and his team have developed a way to make RPE cells from human embryonic stem cells (hESCs) in the lab. They use a particular hESC line called Sheff-1 which was created by University of Sheffield researcher Harry Moore in 2004 with MRC funds.

These RPE cells can stall loss of vision in rats. When implanted into the eyes of the rather grandly named Royal College of Surgeons rat (which goes blind after eight weeks because of a mutation in its RPE cells) the rats retained their vision.

But getting to a point where you’re ready to transplant stem cells into patients is much more of a challenge. The trial will be only the fourth in the world of cells derived from hESCs.

Given that this is relatively uncharted territory, “We’ve really had to come up with every step of the process ourselves,” says Pete. “That means things like testing the cells in animals to make sure that they don’t produce tumours, and producing the cells to the required, clinical-grade standard — for which we had to build our own facility.”

Actually getting the cells into the eye has also been a challenge. Pete and his team had to develop a membrane made from clingfilm-thin, super-strong material, onto which they seed the cells. This is then rolled up and delivered into the back of the eye by a specially designed surgical tool.

The team also had to perfect a new surgical technique, which has been tested on around 40 pigs. “The pig eye is the same size as a human eye, so we did the exact operation on pigs to ensure that it would work successfully. The operation will only take about 45 minutes and could be considered as an outpatient procedure,” says Pete.

And to tell whether the cells are working once they are in the eye, Pete and his team have also used MRC funds to develop imaging techniques that look at their survival and function after transplant.

Going to trial

So far, the team has developed and checked the manufacturing process for the cells, and the technique to deliver them into the eye. They have also received ethical and regulatory approval to conduct a study at Moorfields Eye Hospital in the United Kingdom.

The next step in the partnership is to start a Phase 1/2A study to evaluate the safety and feasibility of the technique, and whether it works in people with the ‘wet’ form of AMD who have experienced a recent rapid decline in their vision. Wet AMD progresses much faster than dry AMD, and is caused by new blood vessels growing in the wrong place and damaging the macula. The partners are in the process of producing the cells and expect patients to enter the clinic as soon as is feasible.

It won’t all be plain sailing. Even if the transplants work, no one really knows to what extent the patient’s immune systems will reject them. It might be that in the future, RPE cells made from a patient’s own cells — via so-called induced pluripotent stem cells — will be the best source for transplant.

Driving to the clinic

So does Pete have any regrets on setting himself the five-year limit? “Well, I’m not going to do it again — I’ve gone completely grey since starting the project!” he says.

But he has learned two lessons: join forces with an industry partner, and work in parallel. The London Project formed a collaboration with Pfizer in 2009, giving Pete access to their expertise. “There’s no way an academic scientist like me could do this alone,” he says.

Similarly, the usual sequential style of academic research doesn’t suit a drive for the clinic.

“We could only do this so fast because we were developing each strand of work at the same time. If we’d done everything in separate steps, we’d be nowhere near where we are today,” he concludes.

28 Comments Post a comment
  1. Is there any possibility that my elderly father could be included in the imminent trials for age related macular degeneration?? It would change his life!

    November 18, 2014
  2. Sandy Moshier #

    I have wet AMD ib both eyes and still have some vision in my left eye. I am excited about this research!

    December 22, 2014
  3. From a UK/Australian citizen. UK trained RGN and Midwife.
    Congratulations to Prof Pete Coffee – amazing & exciting!
    At age 69 I have dry ARMD – Drusen +++ but reasonable vision.
    Is your project at the stage of human trial? When will it be? Can I volunteer? Keen to have intervention a.s.a.p.

    Thank you.

    January 10, 2015
    • Katherine Nightingale #

      Thanks for your comment Eleanor. We have this message from Pete Coffey’s team:

      The initial clinical trials will not be open to volunteers due to strict ethical guidelines and criteria at this point. There is also no list on which patients can record their interest in trials during this very early stage.

      Information on clinical trials will be made widely known through the international media when they have commenced and data begins to feed back.

      Kath Nightingale
      MRC Digital Content Editor

      January 12, 2015
  4. Sofia Lampropoulou #

    Dear Prof. Coffey,

    My mother suffers from a degeneration problem of retina (I am not sure about its name in English “Retinitis Pigmentosa?” it is like see looks from a very narrow tube….
    She is almost blind in 95% and she is now 75 years old.

    Do you know if there is any new treatment or clinical trial for these disorders of retina? I have heard and read your work with stem cells but are these appropriate therapies for “Retinitis Pigmentosa?”

    I would like to help my mother as she is so sad and her sciocology has affected a lot because of this chronic problem (since she was 18th).

    Thank you in advance for your time.

    I am looking forward for your response

    King regards

    Sofia Lampropoulou

    February 2, 2015
  5. Graham barton #

    My wife has wet AMD, she has had 23 injections in her left eye and is only some peripheral vision remaining. She is now getting distortion with her right eye and has had 3 injections so far.
    any news on wet AMD treatment would be gratefully received.
    Best of luck with the trials

    Graham

    May 29, 2015
  6. David Blane #

    What I did not get from journalistic reporting is why the absence of comparison to iPS cell work in Japan. Aren’t they doing the same but with iPS cells? and is the iPS cell way not easier and more effective and flexible?

    September 29, 2015
    • Katherine Nightingale #

      This MRC-funded work has been carried out with embryonic stem (ES) cells as there is much more pre-existing knowledge on ES cells than IPS cells. The UK Regenerative Medicine Platform, however – in which the MRC is a partner – has funded Professor Coffey’s research team to do preliminary work using IPS cells to ready them for testing in patients. You can find out more about that funding here: http://gtr.rcuk.ac.uk/projects?ref=MR/L022842/1

      Kath Nightingale
      MRC Digital Content Editor

      September 30, 2015
  7. Sonny #

    Hi There,

    Would it be a stretch to assume that this may also potentially help Stargardt’s disease?

    I’m 30 and this would definitely be life changing!

    Also, there was a press release in late September stating that the outcome of the trials would be better known at the end of the 2015, will there be a public update?

    Happy to join the queue of volunteers if the trial is extended to test Stargardts.

    January 6, 2016
    • Katherine Nightingale #

      Hi Sonny,

      Glad you enjoyed the article. The best people to ask are the research team at the UCL Institute of Ophthalmology or at the London Project to Cure Blindness.

      Best wishes,

      Kath Nightingale
      MRC Digital Content Editor

      January 7, 2016
  8. Mrs m sharpe #

    Frederick sharpe

    My husband and I also have been awaiting eagerly the results of the first stem cell implant as outlined in side view magazine. This article was so encouraging as he has wet Amd in both eyes. I read as much as possible about the subject as after all injections available to him he has been told there is nothing further that is available.God speed with your work.

    February 3, 2016
  9. Deborah king #

    I would gladly volunteer for trial using stem cells on macular degeneration. I hope my right eye hasn’t progressed too much by the time this begins

    March 1, 2016
  10. kathleen mckirkle #

    I am fortunate in that although I have wet amd in both eyes my sight is relatively good thanks to eylea injections and the care given by the doctors and staff who look after me. I am so worried all of the time I might lose my sight and thank goodness for Dr. Coffey. I am seventy two, and to be honest I would rather die than lose my sight. I couldn’t imagine life without being able to see. I know it sounds dramatic but is is a fact. I hope Dr. Coffeys wonderful work becomes available routinely for everybody affected by this disease. I wish him well and thank him on behalf of everyone who has amd for his fabulous work.

    March 19, 2016
  11. Sachin kumar kashyap #

    All sientest resercher your research be very succsfull and maney persons meet a new life.thanks all off you and god.

    April 10, 2016
    • Chiranjeev #

      Sachin sir , where r you from.

      October 19, 2016
  12. Sachin kumar kashyap #

    Sir ,My name is Sachin Kumar kashyap.sir my right eye ratina is dameged a long time and i can not see the right eye. Sir ,i prayer to god your stem cell experiment will be very succsfull and many person,s meet a new life.thank all of you sir.

    April 10, 2016
  13. Sunil Mehta #

    Can hardly wait for a successful outcome and for it to become available to a broader spectrum of patients. My mom suffers from this ailment and we are very hopeful that she will benefit from this breakthrough treatment. I have just relocated to London and this is without doubt the most significant attraction of the move.

    If at all there is anyway at any stage to participate in the trials please let me know.

    Sunil Mehta

    May 16, 2016
  14. Sonia Wappett #

    I am 68 and have dry eye macular degeneration. I would be happy to volunteer for any clinical trial that might impede the pace of further deterioration in my eyesight.

    July 6, 2016
    • Sylvie Kruiniger #

      Dear Sonia,
      Thanks for getting in touch. If you would like to find out more about current trials, the best person to speak to would be your GP.
      Best wishes,
      Sylvie

      July 11, 2016
  15. Chiranjeev #

    I am suffering from rt eye wet macular degeneration at the age of 27.
    Now I am 33 . Could you please test this therapy to my infected eye.
    Already OK injected lucentis-4 and avastine -9.

    Chiranjeev

    October 19, 2016
    • Sylvie Kruiniger #

      Hi Chiranjeev

      We would recommend speaking to your doctor about possible treatments and or becoming involved in a trial.

      Best wishes,

      Sylvie

      October 19, 2016
  16. Cheryl #

    My name is Cheryl. I got poke in my left eye with scissors when I was 3 years old and lost my vision. I have some peripheral vision with light and dark and can see shadows. I’ve been told there is no cure due to lazy eye and damage to my retina. I’ve had several cataract surgeries as a child. I am interested in participating in your clinical trials if there is any chance of me regaining some sight in my eye.

    October 23, 2016
    • Sylvie Kruiniger #

      Hi Cheryl
      We would recommend speaking to your doctor about possible treatments and or becoming involved in a trial.
      Best wishes,
      Sylvie

      October 24, 2016
  17. marshall firth #

    any more news and results from phase 1/2 trials when will it pass this phase and are you in need of teenage patients. my son has rpe and if there are any trials he want to participate to see if his eyes stop denigrating anymore.

    November 13, 2016
    • Kate Lin #

      Hi Marshall,

      The best people to ask about news and results from the trials are the research team at the UCL Institute of Ophthalmology or at the London Project to Cure Blindness.

      We would recommend speaking to your son’s GP about becoming involved in a trial.

      Best wishes,
      Kate Lin
      MRC Digital & Editorial Manager

      November 15, 2016
      • Marshall #

        Many thanks I just resent a email and it bought me back here again is there a email you have for me im in linked if you could pls give me a address pls

        July 28, 2017

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  1. Interesting Pete Coffey interview

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