Cystic Fibrosis drug given green light in England

24 October 2019

A life-extending drug for cystic fibrosis will be available on the NHS in England, health bosses say.

NHS England reached a deal with Orkambi manufacturers Vertex Pharmaceuticals after months of talks.  Patients should be able to get the drug within 30 days.

The drug improves lung function and reduces breathing difficulties and can be given to children as young as two.

The firm wanted to charge £100,000 per patient per year but a compromise has been reached in a confidential deal.

It is understood to involve significantly less than the sum originally asked for.

Two other drugs made by Vertex – Symkevi and Kalydeco – will be made available as part of the deal.

These also treat cystic fibrosis symptoms.  Symkevi is restricted to over 12-year-olds, while Kalydeco can be used from 12 months.

The treatments do not work for all patients with cystic fibrosis – only those with certain mutations.

It is estimated about half of the 10,000 patients in the UK will benefit from these drugs.

Cystic fibrosis is a life-shortening genetic condition that can cause fatal lung damage.

Only about half of those with the condition live to the age of 32.

‘Best present ever’

Patients and campaigners have expressed delight at the announcement.

Christina Walker said it meant her son, Luis, eight, should be on it by Christmas, calling it the “best present ever”.

“I can’t stop smiling. I’m overwhelmingly happy. It’s absolutely wonderful,” she said.

David Louden, from Carlisle, said the decision would make a “huge difference” to his daughter, Ayda, who has cystic fibrosis.

He said the battle to get it had been “demoralising”.

“You could see this drug with all its benefits that was just hanging there in the balance, dangling like a carrot in front of you but you couldn’t access it.”

‘Good for patients and taxpayers’

More than 10,000 people in the UK have cystic fibrosis, which cause fatal lung damage

The deal comes after the Scottish government reached an agreement with the manufacturers last month.

Under the terms of the agreement both Wales and Northern Ireland can now access the drug for the same price as NHS England, but there has been no announcement over whether they will.

NHS England chief executive Simon Stevens said the deal was “good for patients and fair to British taxpayers”.

Health Secretary Matt Hancock described it as “wonderful news”.

David Ramsden, of the Cystic Fibrosis Trust, said: “This is a very special day and I want to thank people with cystic fibrosis, their families and everyone who has been part of this campaign for their persistence and determination to keep on fighting.”

He also said there could be more good news in the pipeline as a new therapy Trikafta, which 90% of people with the condition could benefit from, was getting close to be licensed for use.

‘Life-changing’ cystic fibrosis drug deal for Scotland is welcomed

12 September 2019

SNP MP Marion Fellows with granddaughter Saoirse, who was diagnosed when she was three weeks old

Families have welcomed a deal which will make two “life-changing” cystic fibrosis drugs available to eligible patients in Scotland.

A five-year agreement has been reached between the Scottish government and pharmaceutical company Vertex over the use of Orkambi and Symkevi.

The drugs improve lung health but were rejected for NHS use last month because they were not deemed cost-effective.

The medication normally costs £100,000 per patient, per year.

The Scottish government said it had secured a “confidential discount” from the manufacturer.

Patients in other parts of the UK are still not able to access the treatment, with campaigners calling for an end to the “postcode lottery”.

‘Never let up’

SNP MP for Motherwell and Wishaw Marion Fellows, whose three-year-old granddaughter Saoirse has cystic fibrosis, said it was a “big moment” for patients and their families, who had been “relentless and inspiring” in their campaign.

Ms Fellows said: “I’m so glad that so many families across Scotland will benefit from these new drugs. Hundreds will be able to lead longer and fuller lives thanks to this decision.

“The Scottish government never let up in discussions and is leading the way in cystic fibrosis treatment.

“But cystic fibrosis knows no borders. The UK government must follow the Scottish government’s lead and make it available to sufferers in England also.”

The drugs can reduce hospital admissions by stabilising lung health

Cystic fibrosis is a life-shortening genetic condition that causes fatal lung damage, and affects about 10,400 people in the UK – around 900 of them in Scotland.

Only around half of those with the condition live to the age of 40.

NHS Scotland estimates that one in 24 Scots have a CFTR mutation which, if carried by both parents, would lead to a child being born with cystic fibrosis.

Glasgow mum Gail Gilmour, whose son has cystic fibrosis, tweeted that there were “no words adequate” to thank those who helped secure the deal for Scottish patients.

Others have said that they hoped it would lead to progress in negotiations for England, Wales and Northern Ireland.

Earlier this year campaigners appealed to the UK government to use its powers to break a deadlock in making Orkambi available on the NHS in England.

Vertex refused a £500m offer for the drug over five years – which was described as the “largest commitment” NHS England had ever made.

Those affected want other drug firms to be asked to make a cheaper version.

The UK government’s Department of Health said its approach was still to urge Vertex to accept NHS England’s “generous offer”.

‘Landmark moment’

The Scottish Medicines Consortium (SMC) had rejected the routine use of the drug in August, saying there were uncertainties about the long-term health benefits of Orkambi and Symkevi in relation to their costs.

David Ramsden, chief executive of the Cystic Fibrosis Trust, said the deal was a “landmark moment” for the hundreds of people with CF and their families across Scotland.

“This breakthrough is a victory for their perseverance and enduring hope. It means 350 eligible people living in Scotland will have access to drugs that stabilise their lung health and reduce the need for hospital admissions.”

He said the trust would continue to campaign for access to the drugs in England, Wales and Northern Ireland, adding: “Scotland’s success must now be replicated across the UK without further damaging delay.”


What are Orkambi and Symkevi?

Since 2015, the drug Orkambi has been licensed to treat cystic fibrosis in patients from two-year-olds to adults, who have a specific genetic mutation known as F508del.

It was not available on the NHS, except for certain people on compassionate grounds.

Symkevi is used to treat the same mutation in patients age 12 and older.

The mutation causes the production of an abnormal protein that disrupts how water and chloride are transported in the body.

Orkambi has been shown in clinical trials to improve lung function and respiratory symptoms in people with cystic fibrosis.

It is the first of a string of drugs that have been developed, with newer ones expected to be even more effective.

Scotland’s Health Secretary Jeane Freeman said the “fantastic news” would allow cystic fibrosis patients to “live fuller lives for longer”.

“The agreement has been reached after extensive discussions between the Scottish government and Vertex Pharmaceuticals and means the medicines will now be made available to patients on the NHS in Scotland, subject to a confidential discount,” she added.

Vertex has also committed to collecting real-time data on the medicines to support any future submissions to the SMC.

Ludovic Fenaux, Vertex senior vice president, said: “We would like to thank the Scottish authorities for their partnership and the collaborative and flexible way that we have worked together to find this access solution.

“It means that approximately 400 eligible cystic fibrosis patients in Scotland now have access to Orkambi and Symkevi.”

Some Scottish patients have previously had access to Orkambi and Symkevi through the Peer Approved Clinical System Tier 2 (PACS Tier 2), which allows doctors to apply for access on behalf of individual patients.

A spokesperson for SMC said the pharmaceutical industry had responsibility for bringing their medicines to market at a fair price.

“The SMC members make these difficult decisions based on extensive clinical experience and taking all of the available evidence into account, including that provided by patient groups. We look forward to receiving new evidence from Vertex on the impact of these medicines following the agreement they have reached with the Scottish government.”

World Mental Health Day

Thursday, 10 October 2019

Today is World Mental Health Day. Some will know what it means to raise the importance of talking about what is on your mind, which is one way of dealing with mental health. Another is doing things that make you happy such as being in a job you love, being around people who appreciate who you are, going the gym to train and fight through the effects through cardio and strength, hobbies, etc.

I have the odd bout of fearing not my future, but my demise. Why? Some will already know one reason why. Unlike some, I am not in denial of my mental health. I’m rather open about it and deal with it by talking or doing something that helps. My mental health issue is something that comes and goes. What helps is keeping busy with things like work, weekends at the cottage and the gym. Sometimes I can be motivated. Sometimes not and just don’t want to do anything.

With the gym, a lot of motivation comes from my PT, who helps by encouraging me through a good cardio and strength workout. The last few sessions have been amazing compared to a year ago. My breathing control and technique have improved vastly. My lung function reaching nearly the same score I was at 5 years ago.

I’ll be blunt, but had it not been for the much needed improved treatment for CF, I question whether I’d still be here, or whether my health would be as good as it was 10 years ago, which is why I take my meds and have the fitness lifestyle I have.

The meds and healthy lifestyle have maintained my health well, so the thing that baffles me is despite how well I am and how well I’m doing, I still have the odd mental fall.

It sort of pisses me off more than anything because I shouldn’t be feeling like this with how well I’m doing. Though I’m positive that I’m not the only one who is like this.

One thing for sure is I talk about how I feel rather than shut myself off and not talk at all about it. I’d much rather have people around me than push people out my life as some have done.

There is great importance to have people around you as had they not been there, I don’t think I would be here.

Though sadly not everyone is in the same way of thinking.

Cystic fibrosis sufferer told he will not be awarded PIP by DWP

Source: Warrington Guardian
Date: Saturday, 1 April 2017
Written by: Adam Everett

Peter Trengove, who has Cystic Fibrosis, has been told that he will not be awarded Personal Independence Payments by the Department for Work and Pensions.

A CYSTIC FIBROSIS sufferer whose lungs function at as low as 31 per cent capacity has been told that benefits he receives in order to help pay with healthcare costs will be stopped.

Peter Trengove from Woolston nearly died at three days old because of the condition, which makes it difficult for sufferers to breathe and digest food.

Cystic Fibrosis also claimed the life of Peter’s older brother when he was aged six.

The 37-year-old had received Disability Living Allowance in order to help to pay care costs, but this system is currently being replaced by Personal Independence Payments as the Department for Work and Pensions believes it is ‘outdated’.

Peter, of Cynthia Avenue, received noticed on New Year’s Eve that he would have to attend an assessment interview as part of his PIP application, with forms on how Cystic Fibrosis affects his day-to-day life taking three hours to fill out.

On Saturday, Peter, whose lungs function at between 31 and 45 per cent capacity, was told that his DLA will end next month and that he will not be awarded PIP. He said: “According to the unqualified professionals at the DWP, Cystic Fibrosis isn’t a disability and has no effect on the sufferer at all.

“I was informed by the assessor that the decision would not be based on the assessment but on the written evidence given by my consultant – this was something I was relieved about because the assessor saw me on a good day.

“The decision cannot be based on one hour spent with a Cystic Fibrosis patient, which is why the decision-makers should be focussing more on the evidence given by the consultant.

“The assessor claimed I can walk 200m – however, no physical evidence was given then I can do this and the assumption was made based on me walking from the waiting room to the assessor’s office, which is less than 5m.”

Evidence that Peter believes his assessor disregarded includes medical notes on a chest infection in October that took three months to treat and a further month to recover from.

Peter had used his DLA for health costs including prescriptions, travel to specialists clinics a 40-mile round trip away and the cost of a Personal Trainer who helps to keep his lungs working as well as possible.

He is now appealing the decision but feels his health has been affected by the PIP assessment process and the subsequent cut in payments.

Peter added: “Since the assessment, I’ve had bad days, which mainly includes not leaving my house unless I really have to in fear of exposure to illness, plus I’ve had no motivation.

“I even have to force myself to get ready to go to the gym because I know I’ll basically become ill again if I don’t go.”

“Whether I will be motivated tomorrow with the weight of my PIP application outcome lying heavy on my shoulders is a question yet to be answered.

“The only hope I have at present is that by appealing the decision, this will result in the answer I had hoped for on this occasion.”

A spokesman for the DWP said: “Decisions for PIP are made following consideration of all the information provided by the claimant, including supporting evidence from their GP or medical specialist.

“Anyone that disagrees with a decision can ask us to look at it again and if they’re still unhappy with it they can appeal to an independent tribunal.”

Cystic Fibrosis sufferer finds big health improvement weeks into trial of a new, drug Orkambi

Source: Warrington Guardian
Date: Monday, 27 February 2017
Written by: Adam Everett

Peter Trengove has found a noticeable improvement in his health weeks into the trial of a new drug called Orkambi.

A Cystic Fibrosis sufferer says he has seen a noticeable improvement in his health weeks into a trial of a new breakthrough drug.

Woolston resident Peter Trengove nearly died at three days old because of Cystic Fibrosis, which makes it difficult for sufferers to breathe and digest food.

His older brother Paul died at the age of six due to the life-limiting condition.

But 37-year-old Peter has seen noticeable effects after three weeks of a four-month trial into a new drug called Orkambi.

Peter had earlier trialled a drug called QBW251, which was hoped to be close to a cure for the disease but proved to be not effective as predicted.

However, Orkambi, which sees two tablets taken twice a day at mealtimes, has given him fresh hope.

He said: “Although the outcome of the QBW251 drug trial concluded with the drug not being as effective as predicted in lab tests, there have been other similar medicines that have made it onto the market and used by people who meet criteria due to high costs for the drug.

Orkambi is on the market in America but is yet to come on the market in the UK due to high costs whereby for one person costs mount up to £100,000 per year.

However, all is not lost.

There have been continual drug trials happening over the last couple of years to find a drug that either works in the same way as Orkambi or will work alongside it to make Orkambi more effective.

I am currently doing a drug trial whereby I have to take Orkambi, which is being tested alongside a trial drug that’s inhaled to see the effects these combined drugs have on Cystic Fibrosis.”

Peter has noticed fewer breathing difficulties, a ‘dramatic’ reduction in coughing and greater lung function.

He added: “I’ve been able to breath better and when I cough, the feeling I have in my lungs is as though nothing is there.

My coughing that occurs in the morning after a night’s sleep has reduced dramatically to being almost non-existent.

When I’m at the gym I am able to work that little bit harder on the exercise bike.

When I wasn’t well last year my lung function dropped right down to 31%, which is a dangerous level in Cystic Fibrosis and can result in being placed on the transplant list.

Since then my lung function has lingered around 40%, which although is better, it isn’t where I’d like it to be.

As a result of being on Orkambi for three weeks and one day, my lung function has increased to 49% – and this is just Orkambi.

I am yet to be placed back on Hypertonic Saline and this new trial drug which will compliment Orkambi.

My reckoning is once I’m on them my lung function will increase even more.”

Cheshire Dogs Home

Date: Wednesday, 1 February 2017

The title of this blog most probably surprised my readers given I usually talk about CF, drug trials and film making. However, this is a special blog as a result of paying a visit to Cheshire Dogs Home today on my way home from work.

I’ve been a dog owner for 9 years and always loved dogs. Mainly because they give unconditional love.

My visit to Cheshire Dogs Home was to see what sort of voluntary work they offer. I had a look around the home to get a feel of the home. I was amazed and impressed by how lovely the home looked.

Whilst visiting I bumped into someone I worked within the Council. Her visit was merely to take her mind off daily life and also to see what dogs were there with the intention of getting one. This would be her first dog too.

It was a pleasure to share tips about what foods are suitable for dogs (puppy as well as adult) as well as other tips.

We saw three beautiful 8-year-old Retrievers (two black and one chocolate brown). All sisters, who were taken there yesterday as their owners weren’t able to look after them anymore for whatever reason.

Obviously I don’t know the story as to why, so can’t make a judgement.

However, as a dog owner, I was able to read their body language and they were ever so happy and welcoming. Had I been able to, I would’ve had adopted all three.

For any owner who has to give up their dog, they will have their reasons and will probably have difficulty making such a decision. Especially if like these three beauty’s they’ve had the dogs for eight years.

Some dogs my family have are from a dog home. Their previous owners weren’t able to look after them for one reason or another. However like with the dogs I saw today, my family’s dogs were well looked after prior to going into a dog home, and are very happy and well looked after now. The way they behave now is as though they were in the family from puppy age.

I’ve got two Cocker Spaniels who have Progressive Retinal Atrophy (PRA), the effects of which is slowed down with three doses of eye-drops and one tablet per day. They’re very happy dogs despite having lost their sight as a result of PRA and are very clever as they have drawn a mental map of the layout of my house and where obstacles are i.e. furniture.

Taking on another dog/s at this time wouldn’t be fair on my two dogs or the dog/s taken on. If I could help re-home dogs left at Cheshire Dogs Home, especially like the ones I saw today, I’d be a happy man.

Cystic fibrosis sufferer tries a breakthrough drug for the first time

Source: Warrington Guardian
Date: Saturday, 24 October 2015
Written by: Adam Everett

Peter Trengove is taking part in the trial of a new drug called QBW251

A CYSTIC fibrosis suffered held back tears of happiness as he tried a new breakthrough drug for the first time.

Peter Trengrove suffers from the life-shortening inherited disease which is caused by a faulty gene, but the 36-year-old has become one of the first people to try QBW251 – a drug which could help reverse the effects caused by cystic fibrosis.

The Woolston resident has reported no side effects thus far, with people in the USA and Australia watching on via Peter’s live blogging of the trial.

QBW251 could help reverse the effects of Cystic Fibrosis

On the blog, Peter describes how he held back the tears as he tried the drug for the first time and described it as a new dawn.

Cystic fibrosis causes respiratory and digestive problems and the disease claimed Peter’s older brother’s life at the age of six, while Peter himself nearly died when he was just three days old because of it.

He said: “There’s real excitement in the cystic fibrosis community about this drug.”

“Since I started telling people on Facebook about this trial I’ve had people from the UK, USA and Australia contacting me asking about the trial drug and wishing me the best of luck.”

“No one knows what’s going to happen so all eyes are on me for answers of this historic breakthrough – I feel I’ve become a celebrity.”

Cystic fibrosis sufferer trials breakthrough drug

Source: Warrington Guardian
Date: Friday, 2 October 2015
Written by: Adam Everett

Peter Trengove will trial a new drug called QBW251, which he says he has been waiting for all his life.

A CYSTIC fibrosis sufferer will trial a new breakthrough drug that he says he has been waiting for all his life.

Peter Trengove from Woolston suffers from the life-shortening inherited disease which is caused by a faulty gene.

This faulty gene controls the movement of salt and water in and out of cells, meaning that the lungs and digestive system of cystic fibrosis sufferers become clogged with mucus – making it hard to breathe and digest food.

Peter’s older brother Paul died of cystic fibrosis aged six, and Peter himself nearly died at three days old because of the disease.

But now the 36-year-old will trial a new drug called QBW251, which could help reverse the cause of the effect by the faulty gene and become the closest thing to a cure for cystic fibrosis yet.

Peter, who has a prescription of 15 medications to battle the disease, said: “The condition can cause life-threatening lung infections and serious digestive problems where reduced absorption of fats and proteins results in vitamin deficiency and malnutrition.

“It is one of the most commonly inherited chronic lung diseases in children and young adults.”

“The affect cystic fibrosis can have on a person’s life depends on the strain – in my case I have a mild strain on both lungs and digestive system whereby you can’t tell I have cystic fibrosis.”

“Although I lead as normal a life as possible there have been occasions in my life whereby even I nearly passed way from severe illness.”

“The earliest age was at three days old following a major operation to remove black sticky stools from my bowel – this left me seriously ill whereby it was touch and go.”

“Luckily I’ve lived to tell the tale.”

“Other cystic fibrosis people with a more severe strain could spend what feels like most of their life in hospital.”

“They could find it hard to secure a job due to how ill they are, find it hard to keep their weight up, be on intravenous antibiotics, be on oxygen or sadly die at a young age.”

“Whilst I was attending a health check I was approached by a nurse who told me a trial study of a new drug was taking place whereby the drug worked with cystic fibrosis of my genotype.”

“I was later given further information once I showed interest in taking part whereby I was delighted to find out it was a breakthrough I had waited a lifetime to see happen.”

“In my parent’s case they’ve been waiting 44 years as they lost their first son Paul to cystic fibrosis in 1977 at six years old.”

“Cystic fibrosis doesn’t only affect me, but my parents will have suffered sleepless night worrying about my health in times when I was ill.”

“Should this drug work this will have a huge impact on over 10,000 cystic fibrosis sufferers who regularly face the risk of chronic lung infections and spend a lot of time in hospital as a result.”

If you have Cystic Fibrosis or know of someone with Cystic Fibrosis and interested in taking part in this study, contact 0800 655 6553 for more information.

Knock, knock, Who’s there!

Source: Warrington Guardian
Date: Thursday, 29 March 2007

DOCTOR’S ORDERS: Peter Trengove emerges from his TARDIS to be confronted by the deadly Dalek (well, one he made earlier, actually)

A NEW series of Doctor Who begins this Saturday.

And while children up and down the country will be hiding behind their sofas, super-fan Peter Trengove will be watching from the comfort of his very own TARDIS!

Peter, of Cynthia Avenue in Woolston, has been a fan of the hit BBC series since 1983.

The 27-year-old said: “I’ve been a fan since watching the Children in Need special The Five Doctors.

“Back in 2002, I built myself a full-size Dalek based on blueprints I downloaded from the internet.”

“It’s mainly plywood, aluminium, wire mesh and fibreglass with bits of household material.”

The 5ft 7ins Dalek features a sink plunger, car indicator lights and even a jet gun that can fire water.

After building the Dalek, Peter went a step further – splashing out £1,000 on the replica time machine!

He said, “I bought a police box off eBay that had been used in stage plays and concerts. To buy a real one costs about £2,500, so it was a good price.”

Peter is currently redrafting his own Doctor Who novel, Genesis of a Time Lord, for a film adaptation.

He hopes it will tell the tale of new sciences mirroring real developments in the treatment of Cystic Fibrosis, from which he suffers.

Who is the inspiration for Peter’s artistic display on brotherly love

Source: Warrington Guardian
Date: Thursday, 6 July 2006

OUT OF THIS WORLD: Peter shows off his entry and certificate with a little help from a star of his favourite TV show.

The work of a Warrington artist, inspired by the bond he shares with his late brother, has won him a place in the final of a national competition.

Although Peter Trengove never knew his brother Paul, who died aged six – two years before Peter was born, the pair share many common bonds.

Both were born with Cystic Fibrosis and both shared a passion for Doctor Who, with Tom Baker as their favourite Doctor.

Only a week before he died, Paul fulfilled his ambition to meet Mr Baker.

This inspired Peter to choose the image of Jodrell Bank for his entry in the Back the Kid arts and photography competition, as it was the location where Tom Baker departed from an episode of Doctor Who.

Peter, aged 27, from Cynthia Avenue, Woolston, took the picture on April 17 last year – 16 years to the day after Doctor Who returned to the television. He chose to title the image From this world to the next.

Peter said: “Not long before his departure from this world to the next, Paul met our hero, Tom Baker. Paul may have died at a young age but he left this world happy that he had met Tom Baker.”

The judges chose Peter as a finalist for the powerful idea behind the picture, a symbol that links Peter to his dead brother.

Back the Kid is a nationwide campaign to help ensure people with Cystic Fibrosis, particularly children and teens, have access to the best possible care and treatments, to improve their quality of life and to help them live longer.