Published on 11 Nov 2014

Eureka moment offers hope of cystic fibrosis cure

Twenty-three years ago, my daughter went to University College Hospital, London, for a small operation to insert grommets in her ear. She’d been suffering repeated infections – and that was what they did in those days.

Shortly before the anaesthetic was being administered, a young paediatrician asked me if he could extract some of her nasal cells (amongst the most active regenerating cells in the human body) for some research he was doing. I signed the consent form and forgot all about it – until two weeks ago.

My five-year-old is now 28. And in those long 23 years, Dr Anil Mehta has been researching a new approach to reversing cystic fibrosis. It’s a disease which afflicts 10,000 Britons today and continues to kill young adults in mid life, typically in their 40’s.

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Stuart Gordon died 18 years ago, at the age of 23. He is pictured above with Katy Plater who also died of cystic fibrosis and South African professional golfer Ernie Els. His parents have steadfastly fundraised for Dr Mehta and his research ever since. Two weeks ago, the good doctor – now leading the research at the amazing microbiology centre in Dundee University Medical School – emailed me, telling me that the nasal cells had hit their mark and that he and a consortium of Italian and French researchers had had a eureka moment.

Until very recently, cystic fibrosis research for most patients had endlessly drawn a blank. Anil Mehta met his Italian opposite number who was working with a leading French scientist at a conference some four years ago. The Italian expert had found a drug that is used in a completely unrelated disease that cleared the debris that destroys the lung in a patient with cystic fibrosis. His approach amazed Dr Mehta because it was totally new to the field.

But this wasn’t enough. Dr Mehta’s team already had a totally different drug, based on nasal cells, which had reversed the environment in the cystic fibrosis cell present as they regenerated that tissue. They next approached a doctor in Naples who applied the two drugs together to 10 humans, nine of whom no longer display the hallmarks of the disease after only two months of therapy.

The cystic fibrosis mice that they have trialled it upon have provided the same results when measured against dummy drugs. Now all that these teams require is £1.3m to carry out a fully comprehensive trial on 120 sufferers. There is now real hope of a new effective approach for babies and children with this disease.

The work shows how the altruism of ordinary folk in donating their cells coupled to a long struggle can crack seemingly untreatable diseases.

Update: 12 Nov 2014

Many of you have written to us asking if it is possible to donate to Dr Anil Mehta’s research. Stuart Gordon’s parents have volunteered to donate all proceeds given to the Stuart Gordon Fund before the end of the year to Dr Anil’s research who they have steadfastly fundraised for in search for a cure.

The Stuart Gordon Fund, Scottish Charity SC028229, and the Charity Choice link is www.charitychoice.co.uk/the-stuart-gordon-fund

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57 reader comments

  1. Dave Soper says:

    Please note that the person in the middle of the first picture is Katy Plater, who also died a number of years ago with cystic fibrosis. Katy was in her thirties.

  2. Dave Soper says:

    Word of caution.

    In 1989 the gene that causes cystic fibrosis was isolated. Soon afterwards, at a public meeting in London, eminent scientists told the public that this would lead to a cure within ten years. Twenty-five years on we no have new hope, but in the ensuing years there have been a number of “breakthroughs” that have lead to nothing.

    Let’s hope this does work, but people with CF must keep taking their medicine, because any valuable outcome might be years away or may never come.

  3. Graham Russell says:

    This article only makes reference to babies and children. So what is the position for CF adults?

    1. Jon Snow says:

      Sadly by the time of adolescence and beyond the lining of lung has consolidated to a kind of lino like surface which as yet is irreversable and therefore not responsive to Dr Mehta’s ‘breakthrough”

      1. Graham Russell says:

        Thank you for your reply. My son is 25 years old and has CF. When we heard this news he was initially very excited, but then alas, we are used to eureka moments as we’ve had so many let downs in the past. We wish good luck to all the parents of younger CFs.

  4. Kim Fisher says:

    My one year old grandaughter has cystic fybrosis this is a light at the end of a dark tunnel.I hope and pray it will help her.

  5. Kayleigh says:

    my son, leo is 22 months old with cystic fibrosis. This gives us and so many others hope of being one step closer to finding a cure!

  6. sheena wightman says:

    My grandson who is 5 years old has cystic fibrosis x if this is true its the best news i could ever imagine x

  7. Sarah Jane Esslemont says:

    Brilliant news. Let’s hope the funds are available to continue to make this miracle happen. Keep the fight CF

  8. Big Tick says:

    Is there a way to crowd source medical research? How can we contribute to this study?

    1. Ruth Gordon says:

      If you wish to contribute to this research, a donation may be made at http://www.charitychoice.co.uk/the-stuart-gordon-fund

      All donations made between yesterday and 31 December this year will be passed to Dr Mehta for his research project.

      Thank you so much for your interest.

  9. stacie wallace says:

    As a parent of a child that has cystic fibrosis I would like to know how we can contribute towards the 1.3 million pound that is needed? Thanks x

  10. Karen Dewar says:

    Is there a fund we can donate to/ fundraise for?

  11. sethaj says:

    thats great news, my nephew and niece have CF and i’d love to see them cured.

    is there a link to donate for the £1.3mil needed ?

  12. Lee H says:

    Mr. Snow,
    I am a Yank, but was a resident of your fine land for over 15 years and remember watching you on the telly. My Argyll born son, nearly 27 now, also has CF. My son also participated in a similar nasal cell study fourteen or so years ago at the Johns Hopkins Hospital in Baltimore, Maryland. It just so happens that I am attending a CF Family Day at the Johns Hopkins Hospital (JHH) this coming Saturday. I would love to learn more of the treatment you described in your 11 Nov blog post. Some of the lead researchers for the US efforts in treating and finding a cure for CF will be there and I would like to ask them if they know of Dr. Mehta work. Add a blog post or contact me directly please.

    Regards,

    Lee H.

    1. Jon Snow says:

      Lee if you could possibly send me a private mesage on Twitter..i can get back to you if you provide me with your email and I will give you mine..best, Jon

  13. Lee H says:

    Mr. Snow,

    Just in case you are not aware of the work of the Cystic Fibrosis Foundation drug development pipeline, I thought you would like a look. No doubt you will recognize some of drugs, even if they may be named differently on your side of the pond. http://www.cff.org/treatments/Pipeline/

    Particularly encouraging is the recent submission of the drug combo Lumacaftor + ivacaftor to the US Food and Drug Administration (FDA) for approval for those CF patients that have the F508del CF mutation. As you will already know, greater than seventy percent of the CF population has a least one copy of the F508del mutation.

    It makes me happy that CF is being tackled on both sides of the pond.

    CF = Cure Found

    Regards,

    Lee H.

  14. Kirsteen says:

    is there any way people could donate to this specific research?

  15. Katrina says:

    Thank you so much, for allowing your daughters cells to be taken, all those years ago. You and everyone else who has done the same. My 9yr old daughter has CF and, although we are extremely fortunate to have Ivacaftor for her, today’s news is just wonderful! Hopefully, this treatment will be suitable for everyone with cf, not just specific gene types.

  16. William beattie says:

    This is amazing and makes me hopeful and positive,as this is a condition affecting a family member and dear friend giving me a better understanding of the condition and what they and there family’s go through.

  17. Jeanette Hall says:

    This is amazing news! I hope something big comes of this and that adults with CF can benefit too.

  18. Neville TOWNSEND says:

    On the page concerning progress in finding a treatment for cystic fibrosis, you at Channel 4 are carrying an ad for American Express: how about asking that all Amex card holders forgo their cash back for one day: would that make enough to start a fund for Dr Mehta to continue the trials he is propposing?

  19. John Bull says:

    Twentytwo years ago we lost our son to Cystic Fibrosis and although its too late for us this is probably the most groundbreaking piece of news I’ve head in the fight against this disease! Trust the research continues apace and that the funding target is met sooner rather than later.

  20. Dave Kellems says:

    Awesome news! Look me up if you need a volunteer for trials.

  21. Tony Harrison says:

    This is amazing news. I have a 6 yr old grand daughter (at present fit and well) with cf. The strange thing is I haven’t recently watched channel 4 news, but today turned it on just as Jon Snow announced this news. A long way to go, I know, but for me this was the best news I have heard for a very long time.

  22. Claire West says:

    This is wonderful news for CF sufferers like my 6 year old son. Thanks to all involved.

  23. Amber says:

    I have CF can I donate my cells?

  24. glady says:

    May God lead you with this wonderful cure!I have two Grandchildren with this horrible Disease.God Bless!!!

  25. Peter T says:

    What an emotional day it has been for us Cystic Fibrosis sufferers.

    I’ve lost many fellow CF friends to this deadly disease. Nearly lost my own life a few times, but have managed to somehow keep on fighting.

    Here’s to good health. X

  26. MICHAEL WELCH says:

    My fantastic cousin sadly lost his battle with CF some years ago. If this a drug that can help any CF sufferer then please , please support its use.

  27. Isla Dowds says:

    A fantastic story that demonstrates how even the smallest level of participation in clinical research trials even at their earliest stages can help provide live changing and life saving breakthroughs! Check out NATIONAL Institiute for Health Research and People in Research websites for more information!

  28. Clive Griffiths says:

    this is truly wonderful news that will bring hope to the thousands of children suffering from this life threatening and life disrupting disease. It is in part due to the timeless effort of those who work to raise funds for research into this illness and of course to the amazing patience and perseverance of brilliant individuals like Dr. Mehta. Thank you all of them

  29. Pippa says:

    My daughter Rosie is 16. She has cystic fibrosis and is so far doing well as only her lungs affected and not her pancreas. A friend sent her a text to tell that it was shortly coming on the the news that there was hope of a cure for her and others. She was so excited it was heart warming and tear jerking as she rarely shows concern for herself. We rushed home to make it in time to listen to your report. We hardly dare to allow ourselves to be too hopeful but it does sound very encouraging if the necessary funds can be raised to further the research.

  30. Lee Leatherland says:

    Good morning,

    My fiancé age 21 is a current sufferer of the disease cystic fibrosis. This research come as a glimmer of hope for us. My question is what is the likely hood that funding will be met and trails will go ahead? Also what is the anticipated time line?

    Kind regards

    Lee

  31. Martin Adams says:
    1. Ruth Gordon says:
  32. carole shaw says:

    will this break through help suferers of c.o.p.d.

  33. stacy connor says:

    How can we donate to this research?

  34. Liz says:

    And they say the media never report on good news. This is amazing. So glad to hear about it.

  35. Jane Hamilton says:

    What amazing news :) My Grand daughter Poppy has CF and I hope and pray this cure comes in her life time. She is the most amazing little girl, beautiful inside and out, we all adore her so much. Will keep fund raising until the cure is found.
    A very hopeful Nana xxx

  36. Ruth Gordon says:

    Thank you Channel 4 for broadcasting this wonderful development and to all the viewers who have contacted us since, and to those of you who have kindly made donations, all of which will go directly to Dr Mehta’s research in Dundee.

  37. lee leatherland says:

    i have jusr looked at the comment stating that for adult CF patients this treatment will not cure the disease, but would the treatment in adult stop any further damage?

  38. Jo Stevens says:

    People who take part in research are few and far between. I hope this encourages people to consider taking part even in the most difficult circumstances.
    It is an amazing contribution to science and healthcare. A greater understanding of research among the public is an important start.

  39. Dave Smith says:

    This sounds amazing news,my newly born granddaughter has just been diagnosed with CF.
    What I cannot understand is that the major drugs companies are not knocking at the door to fund this research, surely they have much to gain if a and the amount of funding must be a drop in the ocean to them.

  40. christine says:

    Wonderful news that such a break through for CF seems likely during my life time.

    It would be my greatest dream to see my Grandaughter cured of this.

    Where can I send a donation to help?

  41. Sally Hughes says:

    I was just doodling (time-wasting) on Facebook and caught this link from a friend. You and I were marginally acquainted a while ago, so I’ve always followed your career with interest and I am hugely appreciative of what you are doing here and wanted to record that. My niece, now 31, has CF and is currently under the care of Papworth, Cambridge. She and her parents keep up a lot better than I with current research, so I have to confess to huge ignorance here. Some are sounding despairing because cell regeneration would not help older people with CF. But, I imagine that anyone with a heart-lung transplant plus this kind of therapy might stand a chance of overcoming the long-term impact of progressive lung damage (which is what usually kills). So well done to the scientists and to you for such knowledgeable and persuasive dissemination. I’ll be giving.

  42. rachael roberts says:

    I have the same question as Dave. I have a 2 year old with cf and our family and friends are lining up to contribute financially to this research. I am, however, confused as to why drug companies are not fighting amongst themselves to fund anything remotely resembling a cure for cf.. This is pocket change to drug companies. Is there an explanation?

  43. Ginny says:

    I do not know how the funds raised by Children in Need are allocated but perhaps there would be some funding available from them. This is exciting news for all those people affected by CF and with this potential treatment/cure on the horizon it is a cause that would benefit many many children all over the world.

  44. Tiffany white says:

    Hi we have a 6 year old with cf she’s well only had one admission in 6 years was diagnosed at birth we never miss a treatment ie drugs physio nebs how can we register for any trials or fundraising to help xxxx Tiffany white mom of Rosie wcf

  45. Angelo says:

    I am in my 50’s living with CF. almost died in 2011 after tune-ups four times a year did nothing to help. I had adult stem cell treatment that brought me back from the dead. I have had 3 now, and a few other CF patients are getting the treatments. This treatment is available for adults, not just children, and I am proof that it works. Coincidently, my doctor is also in Naples area, but not Italy. He is in Florida. More CF patients need to know about this treatment as well. But the cost is high, and insurance does not cover it. We have to do a lot of fundraising to get our treatments.

  46. Angelo says:

    I am in my 50’s living with CF. almost died in 2011 after tune-ups four times a year did nothing to help. I had adult stem cell treatment that brought me back from the dead. I have had 3 now, and a few other CF patients are getting the treatments. This treatment is available for adults, not just children, and I am proof that it works. Coincidently, my doctor is also in Naples area, but not Italy. He is in Florida. More CF patients need to know about this treatment as well. But the cost is high, and insurance does not cover it. We have to do a lot of fundraising to get our treatments.
    You can read about my own journey at http://www.angelodistefano.com

  47. Pam says:

    Have you considered requesting funding from the Cystic Fibrosis Foundation in the United States? They are a super collector of funds, seeking and evaluating potential therapies in which to invest . . . .

  48. Rebecca says:

    Hi everyone, my brother is in his 50’s and has CF. I and a couple of other people, one, crowd fund raiser were meeting at the end of this month with Dr Anil Mehta to discuss the best way to raise the funds needed. Unfortunatly we couldn’t fix a date so we will be on Skype instead. I shall ask the question about Children in Need funds as I also don’t know how people qualify for receiving money from them. The CF trust in this country can’t support final stage research as it is the most expensive stage. I don’t know about the US trust so this question will also be raised. We have all heard of various treatments over the years and got excited only to be disappointed. I hope this is not the case here and I believe its worth every penny to try it and see. Even though this line of treatment is not suitable for my brother, thousands of children could be saved from a long battle through their lives.
    I am very interested in Angelos shared experience as an adult recieving stem cell treatment and will be looking into that to help my brother. I thank every one of you who have donated through the charity choice Stuart Gorden link so far and will post on here once the meeting (skype) has taken place to fill you in on any further fundraising. I feel the money could be raised pretty quickly from some of those big boys out there. From what I know, the trials would take 18 months. Let’s get them started now!

  49. Berryman Bunch says:

    How has the research came along now its almost April, 2015??? Is it going to be available for worldwide CF patients being prescribed it anytime soon?? Was just wondering thanks

  50. mary cassidy says:

    my 2 year old greatgrandson has cf. I would like to set up a direct debit to make a donation to the cf trust. My greatest wish is that a cure can be found soon, as this disease is so distressing. I watch my darling little boy suffering, and in and out of hospital, it really breaks my heart.
    He is the most beautiful, cheery, and loving little boy. May God Bless all those who are suffering from cf

  51. Grant maxwell says:

    Has anyone heard any news on this yet? I can only hope the necessary money has been raised!

    1. Rod says:

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