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.”

QBW251 – Day 28: second to last

Originally published on Monday, 16 November 2015

Today was my second to last visit to the hospital in regards to the drug trial.

The visit consisted of:

  • Body weight/BMI
  • Vital signs
  • Body temperature
  • Urine collection
  • Blood sampling
  • Lung function
  • Sputum sampling
  • Sweat chloride assessment
  • Pulse oximetry
  • CFQ-R questionnaire

In 2 weeks time I will be attending my last visit to the hospital.

This has been what feels like a short journey for me. Simply because I’m a very busy man and time flies when you’re busy.

Though as I said, I’m pretty sure there will be more to say on this trial in the months to come.

As an interesting last word to today’s news, through making friends with many members of the CF community around the world, I’ve had many good conversations with people who ask how the trial is going.

One conversation lead to the interesting mention that for CF people with the F508del mutation, there is a drug on the market called Orkambi.

Information about this drug can be found on this website:

There is a video on the website that elaborates on what this drug does.

QBW251 – Day 17: getting more interesting by the minute

Originally published on Sunday, 8 November 2015

Thursday, 5 November 2015
You may recall that on days 2, 3 and 4 of taking QBW251 I started to have some form of effect in one way or the other.

Whether this was side effects such as feeling sick or having headaches or other effects such as having the feeling my sinuses were more clear and able to breath better whilst exercise, this gave me some form of indication the drug was doing its job…or maybe a coincidence? Who knows?

Now today (day 17 into the trial; day 3 of coming off QBW251), I’ve started to have effects as the drug is making its way out of my body.

I noticed a slight increase in coughing in particular first thing in the morning.

Day 20 (Sunday, 8 November 2015)
Today I’ve been to the gym to do a chest workout. Breathing is as normal as it was when on QBW251.

Is this an indication the drug is still active in my body?

Could it be that in the last month I’ve simply become stronger and the effect of the drug be a coincidence?

I have 2 more visits to the hospital in regards to the trial:

  • Day 28 (Monday, 16 November 2015)
  • Day 42 (Monday, 30 November 2015)

And then…drums rolling here…3-4 months after the final date, I will know whether or not I had the placebo and results of the various tests I’ve had during my 42 days.

QBW251 – Day 14: bye bye QBW251

Originally published on Monday, 2 November 2015

Today I had an appointment at Wythenshawe Hospital in regards to the drug trial.

The sad part of today is that it’s the last day I’m on QBW251.

However, this doesn’t mean I’m at the end of the trial. I still have to return on day 28 and 42 for further tests…I’m guessing to see changes in the body as the drug leaves the system.

Weight: 68.3kg (up by 1.2kg since last Monday).

I’ve gained since 18 March 2015 6.1kg (1 stone)…amazing.

This morning a repeat of some screening tests as followed:

  • CFQ-R questionnaire
  • Bodyweight/BMI
  • Vital signs
  • Body temperature
  • ECG
  • Urine collection
  • Blood sampling
  • Sweat chloride assessment
  • Pulse oximetry

With blood sampling, they had to take blood pre-dose and 2 hours post-dose.

Again they managed to take the first lot of blood first time. Yay!

However, I’m yet to wait if the second lot of blood will be taken without a glitch.

An interesting comment made about the drug is although the tests will help see how my body reacts to the drug as it enters the system, no change in lung function doesn’t necessarily mean the drug isn’t doing its job. There could be a chance I’ve simply not been on the drug long enough for it to have an effect on lung function.

It will be strange not having the drug after my last dose tonight. However, I am looking forward to “what happens next”.

I would say this isn’t the end of the story, but the beginning.

QBW251 – when is publicity too much?

Originally published on Saturday, 31 October 2015

This isn’t an attack on anybody, nor should this be used as an attack.

Someone made a comment about whether or not I should quiet things down on the publishing front about the trial of QBW251 in case it turns out I’ve created all this hype when it could be I’m on the placebo.

Although I can see where they are coming from, what I highlighted was I made it clear from the beginning nobody knows if I’m on the drug or placebo.

All I can do is guess merely by the effects the drug is having that I could possibly well be on the actual drug.

Although there is a 50% chance I’m wrong, I have felt subtle changes since I started this trial, which could indicate I am not on the placebo.

Whether I am correct or this is by chance is another matter.

The fact the trial is ongoing to me is the main thing.

Whether or not I’m on the placebo doesn’t matter. It’s an unknown journey for me and I love sharing this journey with you all.

We all know there are drugs out there already for other genotypes doing the same work.

What we need to do is raise awareness about Cystic Fibrosis, its effects on the CF community and the importance this trial will have on our lives.

It’s about time CF got exposed as it seems to be something not enough people know about.

QBW251 – Breathing easy in Whitby

Originally published on Saturday, 31 October 2015

It’s been a week since I last put fingers to keyboard for updating people with the goings-on in the QBW251 trial.  So what has been happening?  I hear you all say.

Here is a lowdown of events from day 6 to today (day 12)…

DAY 6 (Sunday, 25 Oct 2015)
Earlier this week I reported I had a sensation in my lungs and sinuses as though they were clearing themselves more.

Today at around 2:30pm whilst doing a workout at the gym, I noticed I was breathing easier than normal as I pushed the weights.

Could this be further evidence of me being on the drug and not the placebo or coincidence?

Although the only people to confirm this are those in charge of the trial, the effects I’m experiencing do bring me to hope it’s not a coincidence and the drug is doing as it should.

Again time will tell. In the meantime, one can’t help but be excited by all this. It’s about time the Cystic Fibrosis community had some form of hope of a better life thanks to modern medicine.

DAY 7 (Monday, 26 Oct 2015)
Today I had an appointment at Wythenshawe Hospital in regards to the drug trial. Basically to see how things are ticking.

Weight: 67.1kg (up by 1.3kg since trial started).

FEV1: 2.06; FVC: 4.52 (normal readings – the question is, should this have gone up as a result of drug working?).

The best part of today is the nurse was able to take blood samples the first time. Yay!

DAY 12 (Friday, 30 Oct 2015)
Last day of holiday in Whitby.

Today I went to Whitby town centre and climbed 199 steps up to Whitby Abbey.

Guess what?

No breathless feeling.

Could this be another sign of drug working?

Or has doing fitness for the last couple of years paid off?

Again only time will tell.

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.”

QBW251 – Day 5: scent sense

Originally published on Saturday, 24 October 2015

I had an interesting morning today.
I woke up at 8:30am with a headache slightly worse than yesterday.

Holly (my Cocker Spaniel) was laid beside me sniffing at my face.  It was as though she could smell something new about me.  As though she was detecting the drug working.

I once read about dogs and cats being able to sense things.  Whether a coincidence or she is picking up changes to my body as a result of this drug…this gets more interesting by the day.

At 8:45am I had a yoghurt and then took the drug with water.

By 9:00am the headache subsided, which shows the importance of the water intake as instructed.

QBW251 – Placebo or not Placebo?…that is the question

Originally published on Friday, 23 October 2015

DAY 2 (Wednesday, 21 October 2015)
Well, so far I’ve had no side effects or any other effects from the drug…though there’s still time.

This was the first thought of today. However as the day passed, things started to happen. Whether a coincidence was another question.

At 11:20am I had a sensation in my lungs and sinuses as though they were clearing themselves more. I could feel what felt like thick mucus making its way up and out. However, I chose to remain sceptical, though thought it was worth noting as feedback for my next study trail visit.

I was in the CF clinic at 2:00pm. All is looking good. Effect of the drug hasn’t shown in lung function.  Though it’s early days yet. The drug will probably take approx 4 days before effects, if any, are noticed. Whether I will notice them without tests or not is yet to be known.

I talked with the Doctor about the Kalydeco video I shared in an earlier post. I mentioned to him that over the years of being asked what CF is, I found it hard to put into words what it’s all about. However having watched the video, this made CF so easy to understand and for me to describe the basic function that I’m surprised a drug like this hasn’t been found much much sooner. This made the Doctor laugh.

Obviously had the human body been mechanical, switching on the faulty gene would be a lot easier.

DAY 3 (Thursday, 22 October 2015)
At 9:10am felt a little sick, which lasted approximately 3 hours…maybe a coincidence, but this is one of the side effects of taking QBW251.

No other side effects were experienced.

DAY 4 (Friday, 23 October 2015)
Weight 65.7kg.

The staff found it astonishing that I was able to take 6 tablets in 1 go.

When I said I’m used to swallowing a good mouthful, they started laughing. People have such filthy minds when they’re around me! LOL

At 11:50am whilst at work, I started to feel a little sick again like yesterday.

At 1:50pm I started with a mild headache – another known side effect. Followed 5 minutes later with slight dizzy feelings.

Again maybe a coincidence, but today I’ve had 3 different known side effects to the drug.

Whether signs this is THE drug or is the placebo and a coincidence is another matter.

QBW251 – Day1: The First Dose

Originally published on Tuesday, 20 October 2015

Here is an account for what happened today:

Arrived at the hospital.

Oximeter check is done.

Paperwork check is done.

Cannula put in and blood taken.

It took 2 attempts to get a cannula in.

Ate toast prior to the first dose.
I never thought how much this trial would affect me mentally.
So many thoughts and emotions to deal with.
It’s lovely to have so many supporters.
What’s more is, the support is worldwide.
I’m overwhelmed.

I’m staring at the new drug! Holding back tears here.

Final checks are being made prior to the first dose.

I’ve seen the tablet. It’s a capsule. Looks like the blood-red capsules similar to Rifampicin, but probably same size as Creon.

The nurses are counting the capsules to make sure all is present and correct.

The first dose is taken. I’m cured…or am I?
Placebo or not placebo, that is the question.

A first blood sample is taken.

A second blood sample is taken.

Blood pressure and pulse rate is taken.

ECG is done.

A blood sample is taken.

I’ve lost count to how many people are following me today.
This has gone viral in the USA and Australia as well as the UK.

Blood pressure and pulse rate is taken.

ECG and blood sample were taken.

Mmmm yummy…this didn’t last long!

Lunchtime…chicken roast. Mmmm!

A blood sample is taken.

Blood pressure and pulse rate is taken.

A blood sample is taken.

A urine sample is taken and physical examination done.

Blood pressure and pulse rate is taken.

ECG is done.

A urine sample is taken.

Tuna salad baguette “snack”. Yummy!

Allowed home.

This concludes the first day of my trial.

How do I feel?

On top of the world!!!