Dare to hope

Hope of a cure is crucial for anyone suffering with a life-threatening illness like cystic fibrosis (CF)*. My hopes have been raised and dashed in equal measure over the years. But is the outlook at last changing?

Having just turned 42, I’m keeping just above the median age of life expectancy for CF sufferers in the UK which is currently set at 41 (and 38 in the U.S.as I understand it). Based on existing UK records, each week five babies are born with CF, and two people die. More than half of the CF population will live past 41, and improved care and treatments mean that a baby born today is expected to live even longer.

The ‘c’ word of ‘cure’ is of paramount importance to all of us and our loved ones. A cure from all our ills. This word has been bandied about in many different ways by all sorts of people from scientists to the media for more than half my life.

It was way back in August 1989 that scientists from the U.S. and Canada triumphantly announced the discovery of the gene associated with CF. It was the early days of gene hunting, and the CF gene was a big prize. At the time, they predicted that a genetic test for CF was just around the corner. But they also thought a drug to treat the disease was in reach.

Patient patient
24 years and over 8500 days later a CF cure is still proving elusive. Throughout this extensive period of hope, I’ve experienced many false dawns and had to rationalise the sensationalism of many press reports. I’ve also have to manage the delight of my family and friends who tell me they’ve just heard that “a cure for CF has just been discovered!”

From 1997 I endured a fallow decade of no new drugs that could improve both my body and mind. Based on the premise that if you don’t ask, you don’t get in this life; I must have sounded like a broken record with my doctor during every appointment, “Anything new coming soon?”

Often my doctor would name a couple of drugs that I’d never heard of and tell me that we needed to ‘save them for a rainy day’. Well England has had its fair share of rain in this period, but still I had no joy. I did ask but I didn’t get.

Strangely, the years have taught me about patience. Patience is waiting. Patience is keeping going when the going is torturous and slow. Waiting hurts. But not knowing if a cure will ever come my way can sometimes be even more painful.

I know a few CFers who have withstood lung transplants and are doing pretty well but very sadly I know some that didn’t survive after receiving their new organs. I know and see others around the hospital, heavily dependent on oxygen, who are desperately holding out for the right donated organs. I can only imagine that that must require the most amazing amount of patience.

Wait for me
There are now a handful of successful drugs making their way onto the market. Like buses, you have to wait an age for one to turn up and then suddenly a couple turn up all at once.

A new generation of transformational drugs are coming. I have luckily lived long enough to see a time where drugs for the first time are available that treat the root cause of CF.

‘TOBI Podhaler’ by Novartis, consisting of a dry powder formulation of Tobramycin for oral inhalation via a handy device is one such medication. I’ve been on this for a few months and it’s improved my lung function by over 5 per cent.

Also there are two further dry powder inhalations – ‘Colobreathe’ from Forest Laboratories and ‘Bronchitol’ (mannitol) by Pharmaxis. Lastly there is an oral medication called ‘Kalydeco’ from Vertex Pharmaceuticals.

The drug that is receiving a lot of press is Kalydeco. It took Vertex a while to find a drug that would help restore the function of the protein the CF gene makes. One of those chemicals ultimately became a successful drug, but it had to be modified so patients could take it by mouth, and so it would last the right length of time in a patient’s body. Overall the success of Kalydeco has been more than two decades in the making.

Kalydeco only treats patients with CF who have at least one copy of the G551D mutation. There are around four per cent in England – about 350 patients – who are benefiting from this medicine. While not quite a cure, the drug is extremely effective for those patients who have that mutation.

It has opened up a new era of genotype specific medicines that are the closest thing to a cure yet for cystic fibrosis. It comes in the form of two blue tablets, which control the symptoms in the lungs and for some of the digestive system too. After 42 years battling with CF, taking 40 tablets a day and many other treatments, I can’t imagine having only two little pills to take a day.

I don’t have the G551D mutation. I have the most common type of CF mutation, F508Del, which 90 per cent of people with CF carry, but Kalydeco could still help me in the long run. There are further trials using Kalydeco in combination with a substance called VX-809. The first results from these trials have shown a potentially positive impact on the more common F508del mutation.

However, this is not an actual cure as the damaged CF gene still exists. The only way to fix that would be gene therapy, where a healthy form of the gene would supplant the damaged one. Although it seems simple in theory, in practice, gene therapy has been incredibly difficult to accomplish and clinical trials in the UK and US are ongoing. When perfected hopefully inside the next decade (but I don’t want to get my hopes up), the effect will be close to a cure for CF children and it will be very helpful for older adults depending on their state of health.

There is and always has been a lot of fanfare about curing CF, but this new generation of drugs offer an alternative ‘c word’ and that is ‘control’ – controlling CF at the basic level, and that offers hope while we wait for gene therapy. We are at last beginning to win a few battles while we hold out to win the whole war… but there’s still more waiting and patience required.

More can and needs to be done by pharmaceuticals to maintain the momentum on treating CF. Scientists need to have an ‘ABC mindset’ to CF – Always Be Curing!

Once a drug is available it would help to keep it at a realistic funding price so that it’s actually benefiting those that need it. There’s nothing worse for a person with a chronic health condition then knowing of the existence of a game-changing drug, only for funding red tape to delay or eliminate it ever being taken. People talk about dangling a carrot, but this is more like suspending a bar of gold above someone’s head and then taking it away.

There’s a golden opportunity for drug companies and R&D staff to leave a lasting legacy by making a tangible difference to the quality of life and longevity of CF sufferers, so that in my lifetime the average age for a CF life is doubled.

It would also have a massive impact on my life, hopefully prolonging it so that I can enjoy a better quality of life with my wife Katie and son Felix.

I’m told that time is a great healer. Up to now that hasn’t been the case. I’m hoping that I can dare to hope for a brighter future and that time will still be on my side…

The UK CF Trust is at the heart of CF research and is holding a Research Strategy Conference on 29 April in London. Check out the CF Trust Website for more information on their strategy.

I will keep you posted on my life affirming moments, trials and tribulations as and when they happen. Please keep reading and sharing my blog and sign up (on the right hand side tab) if you have not already done so.

Yours cup half full.

Tim
Tim Wotton

* Cystic Fibrosis is one of the UK’s most common life threatening inherited diseases, affecting over 8,500 people. The condition affects the internal organs, especially the lungs and digestive system, by clogging them with thick sticky mucus. This makes it hard to breathe and digest food. Each week, five babies are born with the condition, however, each week, three young lives are also lost to it. There is currently no cure for CF. However, existing gene therapy trials in the UK are bringing people with the illness closer to a form of cure but CF is not that well known and would benefit from more public donations.For more information and to find out more view the CF Trust Website.

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About Tim Wotton

I live with my lovely wife (Katie) and spirited six year old son (Felix) in Morden, Surrey; working full-time as a communications consultant at the Fifth Business. I have played sport, particularly hockey, tennis all my life and now reguarly go to the gym. Cystic fibrosis has been a huge factor in my life, but not one that overshadows it. I have always had great support from my loving family - parents Margaret and Douglas (RIP), elder brother Chris and my twin brother Jez. I have many rich life insights based on knowing I have a reduced life expectancy and battling against the odds for over 42 years. My eyes and heart have been opened by my health struggle. I feel empowered to share my life lessons to help anyone with health and life issues to overcome. My passions include Paul Smith clothes, dress shirts (believing that it's empowering to dress like it's your last day on earth), Alfa Romeo cars, spicey food, Harlequins and England rugby, Southampton FC, Wimledon village and common, Dorset, seascapes, sunsets and military history. I am available for public speaking on this subject matter and can be contacted via timwottonAThotmailDOTcom. I have written a book based on my euphoric countdown year to 40 that is being published in 2014 and available via e-book and paperback.
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26 Responses to Dare to hope

  1. Lesley P says:

    Thankyou Tim.
    As always, an inspiration to families living with CF!
    God Bless, Lesley.

  2. Charlotte Ridout says:

    Hi Tim, from a GP’s perspective, this is such an interesting blog to read. Thank you for keeping us all up to date. You are an incredible inspiration to CFers and non-CFers.

    Lottie x

  3. Tom Pearson says:

    Hi Tim have got to say your signature definitely speaks volumes about you, cup half full says it all. Our Nathan is 15 and I am hoping in the next couple of years all CFers will get a tablet that does the same as Kalydeco. Although not a cure it will stave off the effects of CF on the lungs and in time, hopefully in my lifetime, I will see Gene Therapy be given the funding it deserves by Governments all over the world. People dont realise that CF is so prevalent all over and when you look at someone with CF it is not always apparent you are sat next to a person with it. They look the same as everyone else just like heart disease.

    Your posts are so thought provoking and to the point and its a refreshing change. Good luck for the future Tim, you deserve it.

    Regards Tom

    • Tim Wotton says:

      Hi Tom,
      As always you are very kind with your replies to my blogs. You help to inspire me to keep on sharing my news and CF insights. If my messages helped just one CF sufferer or family to have hope then it’s all been worth it…
      Send my best to Nathan.
      Best
      Tim

      • Tom Pearson says:

        Thank you I will do. Your posts give lots of inspiration and I am sure there are CFers and their relatives out there who read your posts, but will not reply as they are reluctant. However, the messages get across in a simple and easy to understand manner.

  4. i always read your blog with interest….. hopefully my daughter (Dr Charlotte O’Kane) and other cf sufferers will see a ‘cure’ … tim and your family ….take care my friend

  5. DIANA LAVALLEY says:

    Praying for a cure.

  6. Carl Ford says:

    A great blog, Tim. I’m 48 and a fellow sufferer, your post proved most illuminating. All the best, Carl

    • Tim Wotton says:

      Hi Carl,
      Good on yer Carl – for taking time to comment and for hitting the heights of 48 – I salute you and wish you all the very best with your ongoing health.

      Best
      Tim

  7. KerryLou says:

    Hi Tim – a brilliant and truthful read. I am Aunty to a 20 month old little girl with CF and we live very much in hope every day.

    You are great to read about and thank you for blogging x

    • Tim Wotton says:

      Hi KerryLou,
      It sounds like you are being a very loving and thoughtful aunty. It’s as tough some times for the extended family of CFers as they want to help and understand what’s going on. Thank you for reaching out and for your kind words. I hope I was able to give you and your family some peace of mind…

      Best
      Tim

  8. kami says:

    Dear Tim

    My little brother Paul was just diagnosed with cf a few years ago. He has his good time and his bad. He is doing well right now he just turned 17 years old. Reading this helps me to understand something’s. I pray one day that they wil have a cure for CF. Thank you .

    Kami

    • Tim Wotton says:

      Hi Kami,
      Bless you for your kind comment and I’m sure with a brother like you to support / look out for Paul, he will keep defying the odds and benefit from this new generation of drugs that are becoming available.
      Best wishes
      Tim

  9. David Bayliss says:

    Hi Tim, its when i hear cf sufferers in your age group doing so well it gives me hope that my 2 year old daughter will live to see a cure found.
    The biggest problem with CF is the lack of awareness which reduces the funding the CFTrust recieves, so blogs like this can only help.
    Good luck for the future Tim amd lets pray that CF can stand for Cure Found.

    Dave, a cf dad.

    • Tim Wotton says:

      Hi Dave,
      You’re not just a CF dad, it sounds like you are a very caring dad who is making sure that his lovely daughter is going to be looked after and made better. We can all pray for that.
      Yes, CF needs more profile and it’s one of the reasons I write this blog and the book I’m finishing off – to help the CF community but also to increase the awareness of this grave condition to the masses.

      God bless
      Tim

  10. Mostlogical says:

    Hi Tim
    A wonderfully thoughtful and informative post. I too have CF – late diagnosis; I am now 51. It was great to finally get a diagnosis after years of ill health & Dr’s shoulder shrugging at me; I thought I was going mad! But it was a bitter sweet relief. They say that adult CF is usually ‘mild’ but after a year averaging 10 hospital appts per week, sinus surgery & numerous other procedures I think ‘mild’ needs a new dictionary description! Anyway, I’m a very procactive person, a self employed web designer and I have a great family who have been amazing.

    If you ever need any support getting the message ‘out there’ then please let me know. I’d love to do do something more constructive as I feel CF is the ‘quiet one’ still compared to cancer, heart disease and many other conditions. I live in beautiful Dorset :)

    Good luck with your health, your family & your life.
    Karen X

    • Tim Wotton says:

      Hi Karen,
      Thanks for your note and sharing your diagnosis journey. Thanks also for your kind communication offer which I shall connect with you about as I’m keen to increase the visibility of my blog and forthcoming book.
      Dorset is my mum’s home county and I love it – I’m very envious that you live there!

      Speak with you soon and take good care
      Tim

  11. Jane Watson says:

    Hi Tim thank you for taking the time to write your thoughts on your condition. I found it both heartwarming and informative. My son is twenty four and has CF, like you he makes the most of his life and Im sure like your family we are very proud of the way he deals with it.
    Here’s Hoping
    Jane x

    • Tim Wotton says:

      Hi Jane,
      It sounds like your son and I have a similar way of handling this relentless condition – there’s 24 hours in a day – CF gets 2 of those hours which leaves 22 for Tim Wotton – who’s the winner there! It’s all about keeping on top of your meds, respecting the illness but not letting it dictate too much what I want to achieve each day.

      Please send your boy my regards…
      Best
      Tim

  12. lmk87 says:

    Hi Tim,

    Thanks for sharing this. It is good to see someone clearly explaining where we are with treatments and how/who they might help. As always, your positive attitude shines through in your words.

    I know it is a TOTALLY different kettle of fish as a condition itself … but your words make me consider the journey the science world has gone through in its quest to control/cure HIV.It is at the top of my mind at present as I have been doing some research on the subject recently through work and learning how treatments have changed just so much in the last 20 years. Back in the day having HIV meant almost certain death at a young age from a HIV related illness, 40 tablets a day was of the norm and control was poor/non existent. Now, it is mainly a different story for people who contract the virus, though still not an easy ride (what ever is?) most can be well controlled by taking in some cases just ONE tablet a day. Just one tablet a day, to control a disease that up until fairly recently, was a major killer. There is talk of a once a month injection coming out too….having HIV does not mean what it used to mean. Okay, there is not cure, but it no longer is a condition that just can’t be helped and spells a grim future, there is hope and people are living “normal” (again….what ever is normal?!) lives regardless.

    I am not comparing CF to HIV in any other way, than that I feel that we are in an age where we are seeing treatments for CF take a similar route, follow a similar path – even in the last 5 years, since I first was thrown into the world of CF after my sisters diagnosis – so much has changed, there is so much more to hope for, even if it will never cure her and others such as your good self. If we can’t sort a cure out, I live in hope that one day everyone with CF can just pop their daily pill and get on with life just like so many others living with other chronic and currently incurable conditions can too.

    Wshing you and family the best. Keep writing X

    • Tim Wotton says:

      Hi Alix,
      Thanks for your interesting reply and fascinating insight about HIV.
      Do pass on my best to your sister and I do hope she’s doing as well as possible and that the new era of CF drugs will benefit both of us!

      Kind Rgds
      Tim

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