Orkambi – A gift of hope for Maisie

“Hope is being able to see that there is light despite all of the darkness” – Desmond Tutu.

Maisie Barker has just celebrated her 20th birthday but the best present that she could have hoped for actually arrived a few months before the big day.

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Maisie took her first dose of Orkambi on 29th July

It was quite simply, the gift of hope.

Maisie who was diagnosed with cystic fibrosis (CF) at just three months old was told earlier this year that she could have access to a ‘precision medicine’ drug called Orkambi. She took her first dose of the drug on 29th July.

Orkambi is the first drug to treat the underlying cause of cystic fibrosis and not just the symptoms. Although not currently available on the NHS in the UK (because it is deemed that the benefits don’t outweigh the cost), Maisie has been granted access to Orkambi on compassionate grounds, as there are currently no other treatment options available for her.

In recent years Maisie’s health has been in serious decline. For the past two years she has been on continuous IV’s (intravenous drugs). Every day is dominated by her CF treatment regime – pills, needles, inhalers, nebulisers and physiotherapy take several hours a day to complete.   Maisie also has to take enzyme tablets before every meal as without them her body cannot digest fat and process her food to maintain a healthy weight.

Her bedtime routine can take up to an hour as she repeats her treatment regime as well as preparing her overnight feed.

“I feed through a PEG (percutaneous endoscopic gastrostomy) tube overnight to help me gain weight”, said Maisie “This means that at night I have to get the feed ready, set up machines and flush out my PEG with sterilised water.”maisie-nebuliser

“I used to be admitted into hospital every couple of weeks, as I was always very poorly. Every time I was admitted into hospital I would stay for up to two weeks, three in some cases. But now I am on home IV antibiotics and they have kept me stable for the past two years. Because of this I have only been hospitalised a few times for procedures such as needing a new port.”

Not surprisingly, Maisie has struggled both mentally and physically under this huge treatment burden, but her most crushing moment came when at the age of just 19 she was told that a lung transplant – at that point seemingly her only hope of a better quality of life – was too dangerous for her.

“I’ve been on continuous intravenous drugs for over two years” said Maisie. “My lung function was at its lowest point at 29% when I started them.  I had transplant appointments at Newcastle only to be told a lung transplant would be too dangerous for me and that they do not wish to put me on the list. So really Orkambi is plan B. If all goes well with Orkambi then there is hope that for the first time in two years I will be able to stop the IV treatment.

“I’m hoping that Orkambi will work and improve my lung function, give me more energy and allow me to do more things without getting breathless.”

Hope. Hope is what keeps all of us going.  Hope is what drives us to try to achieve certain goals.  Without hope what reason would there be to get out of bed each morning?

Maisie is completely pragmatic about her health, but she still needs hope. She is hoping for something so small, so simple – something that the rest of us take for granted every day.  Maisie hopes for the chance to be less breathless and have more energy to achieve simple tasks.  She hopes for so little and yet that hope is enough to sustain her.

No wonder then that she has pinned so much of her hope on this amazing new drug. Here in her own words, Maisie shares her initial experience of taking Orkambi:

“On the 29th of July I took my first dose of Orkambi. I started on just one tablet a day instead of the full dose, four tablets per day. This was because I took medication which could interact with Orkambi. So for one full week I took one tablet of Orkambi a day. I then went up to two tablets per day, then eventually up to the full four tablets.

“Since taking Orkambi I have suffered no side effects at all. I expected to have a few side effects but I had none. Maybe it’s because I started on such a low dose? I came home on the 8th August on the full dose.

“Within a week I started to notice massive improvements. I feel so amazing. My resting heart rate used to be around 110-120 bpm now for the first time in ages my resting heart rate is normal at 70-80 bpm.

“For the last four years I have slept sitting up with the help of a wedge as my lungs couldn’t handle me lying down flat – it caused me to struggle with my breathing.

“But for the first time last night I slept without my wedge and it was amazing to sleep lying flat. I could breathe. And the biggest change for me is that I haven’t had one of my ‘coughing fits’ since starting it. Usually my morning consists of me sitting up in bed, struggling to catch my breath whilst I cough, going bright red in the face and feeling dizzy. But that’s all stopped. I no longer cough until I can’t breathe!blows

“On the 18th of August just 10 days after coming home I did my first set of blows (lung function). When I left hospital the last FEV1 I did was 36%. The blows I did on the 18th came out at 50%!! I’ve never had an FEV1 that good since I was 8/9 years old. I never thought in a million years I would be able to get a lung function past 40%. Never mind 50%!”

Maisie is naturally delighted to have been given access to Orkambi herself. But her thoughts are with her fellow CF sufferers when she says “As happy as I am to have started on Orkambi, I still wish that more people had access to the drug and the hope of a better quality of life that it offers.”

Every person who has battled their whole life against cystic fibrosis deserves the chance to have that hope. Orkambi is not suitable for all people with cystic fibrosis, but those with the most common mutation of the disease (F508Del) stand to benefit.

That a drug like Orkambi has been developed is akin to a miracle for those people who have faced a lifelong daily battle against the many health issues cystic fibrosis can cause.

To have that miracle dangled just out of the reach of the people who could potentially have their life transformed by it seems cruel beyond belief.

We hope to bring you further updates from Maisie as her treatment continues.

In the meantime please share this article to spread awareness about the situation with Orkambi and about cystic fibrosis in general. Thank you.

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On September 17th Maisie Tweeted this photo with the message: 2014 – Told I need a lung transplant. 2015 – Denied a transplant. 2016: Given Orkambi – Climbed up Stoodley Pike (1,300 foot hill in the South Pennines).