QBW251 – the trial study

Originally published on Monday, 21 September 2015

On Wednesday, 16 September 2015 I had a port flush appointment at Wythenshawe Hospital.

During my appointment I was approached about taking part in a study to find out how the study drug, QBW251, works in the bodies of patients with CF.

I was given the opportunity to discuss this with family and friends before making up my mind.  However at the time of asking it wasn’t made clear to me what drug I would be testing.

I requested more information on this to understand what was to happen.

Based on little information given at the time, I looked back over the last year or so to news items related to breakthrough medication for CF.

A story came to mind about a breakthrough in CF treatment for the lungs whereby the medication reversed or corrected the faulty gene (or something to that effect).

I suddenly had a feeling had I declined this opportunity, I would regret it.  So having spoken quickly to my dad and partner (by text), their encouragement confirmed this felt like the right thing to do.

Attempts were made to ring the hospital to confirm my interest in the study and it was a matter of time before I received the call I had been waiting for…the call that would be the beginning of a possible life changing experience.

Tomorrow (Tuesday, 22 September 2015), I will be attending my first appointment to go through processes and officially give my consent to be part of this study.

Come October, I will start trials of QBW251, a drug that may, when given to humans, improve the activity of the defective chloride channel CFTR, helping it to work better resulting in improved salt transport at the cells surface, lessen the thick, sticky mucus, and reduce lung disease in CF patients.

The only twist in this study is there is a chance I will be given a placebo…watch this space to see what happens over the next 2 months…

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