“Three years ago, my lung function was 11% and I was hours from death, then I received the gift of life – which was Trikafta.”
These are the words of Nicole Adams, from Newtownabbey, Northern Ireland as she joins other cystic fibrosis (CF) patients to call for global affordable access to the drug that saved her life in December 2019.
The determined group of CF patients from across the UK have recorded a video – https://www.instagram.com/p/CsBOgpPpFNA/ – adding their voices to a campaign calling for a global access plan, for a cystic fibrosis modulator therapy called Trikafta (also known in the UK and Europe as Kaftrio), to be made available to cystic fibrosis patients around the world.
Personal trainer Ben Mudge (33), also from Northern Ireland has for years inspired others with CF to keep up with regular exercise to help them maintain their health. But even he has been blown away by the effectiveness of the new CF modulator.
“Trikafta has given me something that I genuinely never thought I would have” he said, “and that’s a future.”
Cystic fibrosis is a progressive disease. As recently as the 1960’s, children born with CF were not expected to live to adulthood.
Thick sticky mucus builds up in the lungs and other organs of CF patients, causing inflammation that can lead to infections. Damage occurs over time and can lead to a wide range of issues.
This creates a heavy, daily burden of treatment, that often increases with age. But the many nebulisers, medicines and hospitalisations only treat the symptoms of the disease.
CF modulators such as Orkambi, Symkevi, Kalydeco and Trikafta work at the root cause of cystic fibrosis. Trikafta consists of three drugs and is the most effective of the modulator treatments – around 90% of CF patients are eligible to use Trikafta.
In 2020 CF patients in the UK received the life-changing news that this revolutionary new drug was approved to be used on the NHS.
For many this has given them a new lease of life and a better baseline standard of health. However, although patients in the UK, USA and other high income countries are now benefiting from this new drug, there are still tens of thousands of people with CF in low and middle income countries who do not have access – and that situation is not likely to change in the near future.
The sticking point is the cost of the treatment – even in the UK, the approval of Trikafta (Kaftrio) followed several years of high-profile campaigning for access to earlier modulator therapies – notably Orkambi. For this reason, Nicole and other patients like Marc Cotterill were granted ‘compassionate’ access to Trikafta – before it was available on the NHS.
The breakthrough for Nicole, came after fellow CF campaigners in the UK alerted media to Nicole’s predicament and her story became very high profile.
Nicole filmed her contribution to the ‘global access’ video with her baby Colby nestled at her side. “Trikafta has dramatically changed my life,” said Nicole. “I am sitting here with my own son, something that I never thought I would have.”
“Where you live should not dictate the level of healthcare you receive or the drugs that are available to you.” – Marc Cotterill
Marc Cotterill, a fellow CF patient who also edited the ‘call to action’ video – asking people around the world to sign and ‘pass it on’ by sharing the petition link with friends, family and followers.
People who have cystic fibrosis have to deal with an increasing burden of treatment as they grow older, because cystic fibrosis is a progressive disease. The thick sticky mucus that builds up in their lungs and other organs causes damage over time, eventually in most cases leading to premature death.
Bianca Nicolas who as one half of duo Electro Velvet represented the UK in the 2015 Eurovision Song Contest said:
“Trikafta has been life-changing for me. I wake up with so much more energy, every single morning and I am just so grateful for the fact that I can fill my lungs with air. It’s given me that feeling of normality – it’s just a hope for the future that wasn’t there before. It has also for me, increased my fertility which means that I’m now a mum. It gave me my son and there’s no words for that and how grateful I am and what it means to me.
I was horrified when I learnt that only 40 out of 195 countries have this drug approved.
It must be so hard to be someone in a country where it’s not approved and see the amazing effects it’s having on everyone (in other countries) and not have the chance to experience that yourself. That is why I want to do everything I can to help other people with CF experience this second chance that I’ve been given.”
Beatrice White, a 12-year-old actress who has previously appeared alongside Benedict Cumberbatch in The Child In Time said “Kaftrio has given me freedom – I don’t worry about CF anymore.”
Referring to the petition, she then continued “I’m asking you all to sign it, share it, tag as many friends as you want. Even make your own video. Pass It On.”
Other contributors to the video include Aaron Aby, from Wrexham, a mixed martial arts fighter with CF, who has also battled testicular cancer. Aaron said:
“Since taking Kaftrio, I feel a lot better in myself. I’ve put on weight, I’ve got better energy and I feel healthier.”
The campaign for fair global access to Trikafta and other CF modulator therapies is coordinated by grass roots organisation Vertex Save Us and patient advocacy group Just Treatment.
Gayle Pledger, a spokesperson for Vertex Save Us said:
“We started this campaign because families from other countries contacted us after CF families in the UK successfully fought for access to these life-saving drugs. We have now formed a large global community of CF patients. Since joining forces with Just Treatment, who helped us gain access in the UK, we have consulted with organisations and experts on four continents.
We have a series of demands for both Vertex Pharmaceuticals and national governments and these can be found on Just Treatment’s website: https://justtreatment.org/news/cf-patients-launch-global-challenge-to-vertex
We are currently supporting CF patients and families to challenge Vertex Pharmaceutical’s patents in South Africa, Brazil, Ukraine and India in order for them to be able to access the medicines that they desperately need.
The link to the global access petition can be found here: https://bit.ly/vsupetition