Telford mum’s call for drugs to help her son
The mother of a three-year-old suffering from cystic fibrosis said her son's life expectancy is "hanging in the balance" as the family waits for vital treatment.
Emma Hughes of Telford is urging the health secretary Matt Hancock to push for medicines that could save Chester's life by treating the underlying causes of his genetically-inherited condition, after a Parliamentary inquiry last week into the drugs.
Chester has already spent 25 weeks in hospital because of complications from cystic fibrosis (CF), and Emma spoke of the pain of families she has seen lose a child to the illness.
She now campaigns for the NHS to provide Orkambi, a drug that tackles the causes of CF rather than the symptoms, by thinning the mucus that clogs the lungs of sufferers.
While it isn't compatible with Chester's mutation of CF, Emma wants to see more treatments for the disease made available on the NHS.
Emma, who serves in the army, said in her open letter to Matt Hancock: "For years the cystic fibrosis community has waited with bated breath for a much-needed cure, or at the very least have held out for a treatment that treats the underlying cause of cystic fibrosis as opposed to just treating the symptoms.
"Imagine the excitement when Orkambi came along. Yet we find ourselves in a predicament where despite a treatment being readily available in countries across the world, we still do not have access in this country.
"In the three and a half years since my son was diagnosed I have witnessed too many parents having to bury their children, and as a small and extremely close family-like community the death of a child with cystic fibrosis affects our whole family.
"We mourn and our hearts break for those families, knowing all too well that as things stand that could one day be us.
"Having watched the House of Commons Health and Social Care Committee inquiry on the availability of Orkambi on the NHS, I was left feeling incredibly deflated and let down. Somehow the welfare of my son and his future have become lost in a battle over finances.
"I think it’s time that the focus was shifted back to what this medicine can do for those up and down the country who are living and fighting this cruel condition on a daily basis."
Chester needs 20 to 25 tablets a day to stay well, and has regular physiotherapy sessions to help clear his airways.
He has a port to allow for intravenous antibiotics, and is vulnerable to infection.
Emma explained: "Chester was diagnosed after suffering from a bowel obstruction, meconium ileus, which resulted in surgery and a 15-week stay at Birmingham Children’s Hospital.
"He can get out of breath and tires more quickly than a child without CF and he is susceptible to infection.
"We do all his treatments to keep him well but unfortunately a lot of the risks are environmental and therefore we cannot prevent infection.
"Each infection causes more damage and scarring to his lungs and each time he has to have antibiotic treatments he runs the risk of antibiotic immunity."
She is hopeful that Orkambi will be made available for widespread prescription for people with CF, and that that will open the door for other drugs that could give Chester relief.
Chester's older brother Oakley, seven, helps Emma care for his brother.
Emma said: "Cystic fibrosis has a huge impact on Chester, but also on our family.
"There isn’t one aspect of our life that cystic fibrosis doesn’t affect or impact on, from holidays, days out, activities or a normal daily routine cystic fibrosis always finds a way to interfere and wreak havoc.
"As much as I dislike the word, cystic fibrosis is a ‘burden’ on our family and the impact is felt by all of us involved in Chester’s life – from his brother having to take a backseat whilst he has treatments, to having to take time off work because Chester has appointments or is admitted to hospital because he is unwell.
"Chester is still too young to understand the implications of his condition, and I dread the day I will have to tell him, because right now I find it incredibly difficult to find the words to explain the enormity of his condition in a way that he will understand.
"I worry about his health, I worry about how rapid any decline may be and on top of the physical implications I worry about his mental health. If I am struggling to deal with his diagnosis, how will he deal with something this big?
"I wouldn’t wish this condition on my worst enemy, it's cruel and it’s heart-breaking.
"I should be excited to watch my child grow up, to sit and wonder what he will be and what he will achieve, and I should be able to enjoy celebrating his birthdays, but I live in fear that I could lose him before he reaches adulthood, the fear he may never get to experience life as it should be.
"No parent should live in fear of having to bury their own child.
"The argument for Orkambi and other precision medicines has gone on so long, leaving the cystic fibrosis community emotionally drained, this shouldn’t even be a fight.
"Just when we think we are getting closer to a deal, our hopes are shattered and we end up back at the start. Time is passing far too quickly and for those that need Orkambi now, time is running out, and sadly for many it is already too late.
"Whilst I am fully appreciative of the argument of clinical effectiveness versus cost effectiveness, especially when our NHS is on its knees financially, finding that we are being priced out of a drug is unacceptable and having no one to fight that battle is even worse. My child’s life is worth more than this.
"No one in this negotiation can seem to come to an agreement, and no one seems to want to take charge. We need someone to get the parties involved around the table, take ownership and responsibility to ensure this deadlock is overcome.
"My son didn’t ask for this condition, and I would give all I have to take it away, but we are where we are.
"Nevertheless, he should be able to live with this condition with the same kind of normality as any other child, and where there is a drug that could help, that is widely available, there is no doubt in my mind that access to it should be a given."