Drugs: Ultra-rare Diseases

Caroline Nokes Excerpts

Tuesday 16th June 2015 Westminster Hall

[Caroline Nokes (Romsey and Southampton North) (Con)]

It is a pleasure to serve under your chairmanship, Mr Crausby.

I am pleased to be the first to congratulate the hon. Member for Leeds North West (Greg Mulholland) on securing this important debate. I also pay tribute to him for the enormous amount of work he has done on Morquio syndrome, which he has raised many times in the House and in Westminster Hall. He has also held numerous meetings and led delegations to Downing Street. He has worked assiduously on behalf of his constituent, Sam Brown, and, as we have heard this afternoon, he has worked on not only Morquio syndrome, but a range of ultra-rare diseases. He has done an excellent job today of highlighting the problems, the delays in funding and the amount of time it has taken simply to get these drugs through the approval process.

Rather than focusing on those aspects, I will talk about the human cost of these diseases, highlighting the case of my constituent, Jagger Curtis, who is just seven years old—he will be eight in August—and a pupil at Romsey Abbey primary school in my constituency.

Last Wednesday, Jagger was one of the brave boys who walked up Downing Street to hand-deliver his letter to the Prime Minister, which was an incredible experience for him and his parents. It was a really important part of their campaign to highlight the need for funding and approval of Translarna, because Jagger suffers from Duchenne muscular dystrophy.

Of course, Translarna is a relatively new drug. I say “relatively”, because it has been used in European countries since last year; it received conditional approval from the European Commission in August 2014. Yet here in the UK, as the hon. Gentleman has said, we are still waiting.

Duchenne muscular dystrophy is a very serious condition that affects about 2,500 people in the UK, almost all of them boys. It causes muscle weakness, leading to a dramatic loss of muscle function. Typically, patients will lose the ability to walk in their early teens; they will require respiratory support by their mid teens, and they are likely to die either of heart failure or respiratory failure before they reach 30. I cannot emphasise enough what a devastating condition it is and how brave families are when they have to face up to and deal with the reality of a Duchenne diagnosis.

Currently, the only treatments available address the symptoms, rather than the cause of Duchenne. They include the prescription of steroids, which of course have some very severe side effects, including sudden and dramatic weight gain, mood swings, which can be particularly difficult to contend with in teenage boys, and thinning bones.

As has been said this afternoon, Duchenne is a rare condition, with very few sufferers in the UK, and only about 10% to 15% of them have what is referred to as “the nonsense mutation”, which makes them eligible for treatment with Translarna. In some respects, Jagger is very lucky, because he is one of the boys with the nonsense mutation and is therefore eligible for Translarna. Currently, he is still mobile, which is absolutely critical when the use of Translarna is being considered, because it cannot be prescribed after a patient has lost their mobility. Translarna has the best chance of having a beneficial effect while the boys can still move around. Once they have lost their ambulation, it is too late and the opportunity has been missed.

Jagger’s parents, Julie and James, were told late last year that he was a suitable candidate for Translarna, and they genuinely believed that they were within a few weeks of going to the hospital and picking up a prescription for the one drug that they had been told could make a difference to their son. In November 2014, they had no idea that they would still be waiting for the drug now and that it still would not have finished going through the administrative process by the end of June. We are now seven months on from the day that they had expected to go and collect a prescription, but there has still not been a decision and they simply do not know what the outcome of this process will be.

During that time, of course, Julie and James have watched their son lose some of his mobility; his muscles have wasted away further. More than anything else, they desperately want an extension of the time in which Jagger is able to move around by himself, without the need for a wheelchair.

In his letter to the Prime Minister last week, Jagger wrote that he wanted to keep on playing football forever, just like his friends. He is an enormous Saints fan, and one of his proudest moments was going on to the pitch at St Mary’s to lead the team out. There is a fantastic photograph that he included in his letter to the Prime Minister, showing him shaking hands with the Saints manager, Ronald Koeman. In every other way, Jagger is a lively, lovely, normal little boy, who has a massive love for football, but, and it is a huge but, unlike most seven-year-olds Jagger has already been fitted for a wheelchair. His parents have had to make the necessary preparations—it was difficult, even heartbreaking, but they had to do it—to ensure that when Jagger’s mobility is more restricted a wheelchair will be ready and waiting for him so that he can still get around.

For Jagger and every other boy with Duchenne muscular dystrophy who has the nonsense mutation, the clock is ticking. In fact, it has been ticking since last August, when Jagger’s parents and others had their hopes raised that there was a treatment that was about to become available on the market. That treatment could give boys such as Jagger the chance to see out their time at primary school without needing a wheelchair, so that, as Jagger himself puts it, he can run around with his friends and be like any other normal little boy.

My hon. Friend the Member for Leeds North West—I should refer to him as the hon. Gentleman now, but old habits die hard, and on this subject he has been a great friend and a great campaigner; I pay tribute to him for that—along with Muscular Dystropy UK and Action Duchenne, has done great work to highlight the problems that people have faced in getting approval for Translarna in the UK. We expect a decision on Translarna at the end of June, and the company that manufactures it, PTC Therapeutics, indicated last week that it was ready to go, had stocks available and could supply it as and when it was needed.

If that drug is given the green light at the end of June, it will be distributed here, but the boys I have mentioned today have already waited for far too long, and this drug is the only one that is giving them any hope. I know the Minister has been most diligent for some months; he has listened to all we have had to say in this Chamber, in the House, on Twitter and indeed in the media. However, as we have heard, there are real concerns about how long the approval process has taken and about how complicated it has been, as well as about some of the inconsistencies and contradictions about when the drug might be made available. I hope that the Minister will make some comment on that.

I am conscious that there are many Members here in Westminster Hall this afternoon who want to contribute, so I have deliberately kept my remarks short. I will conclude with the words of Jules Geary, because I do not think anyone else could better summarise how her family feels:

“It is hard enough watching your child have to go through losing their muscles. For the drug to work, Jagger still needs to be mobile, so we simply don’t have time to wait. We have been given hope through this drug. We just can’t let it be taken away again.”