Phenylketonuria: Treatment and Support

Lord Austin of Dudley Excerpts

Tuesday 26th June 2018 Westminster Hall

[Liz Twist (Blaydon) (Lab)]

I beg to move,

That this House has considered access to treatment, support and innovative new medicines for phenylketonuria patients.

It is a pleasure to serve under your chairmanship in this important debate, Mr Robertson. It is just over 12 months since I was elected Member of Parliament for Blaydon, and only a few days less than that since I first heard of phenylketonuria—commonly known as PKU, which is easier to pronounce. One of my constituents, Barbara McGovern, had called at my office and spoken to a member of my staff about whether I would attend an event in Westminster on 28 June. Barbara’s son Archie has PKU, and she described his condition and some of the restrictions that he has to cope with every day. Her explanation of what PKU means, and her determination to get the best for her son, impressed my staff, who made sure that I attended the reception to learn more about the condition. Barbara and her husband David, and Archie, have travelled from the north-east to listen to the debate, and I welcome them.

Since that first encounter, I have been lucky enough to be introduced to the officers of the National Society for Phenylketonuria, and have had the chance to work with them and its other members to raise the profile of PKU, get people to understand it and its effects, and press for access to treatment and support. I will give a few of the many names that I could mention. Kate Learoyd and Caroline Graham both have children with PKU, and they have dedicated much of their time to talking to Members about the condition and the need for action for people who live with PKU. Professor Anita MacDonald OBE, of Birmingham Children’s Hospital, also does much to raise awareness of the condition and goes above and beyond in advising families affected by PKU on diet.

[Ian Austin (Dudley North) (Lab)]

I commend my hon. Friend on securing the debate and on her leadership of the all-party parliamentary group on phenylketonuria. There are lots of health-related campaigns for more resources, new treatments and drugs. Many have plenty of resources and are funded by major pharmaceutical companies, which hire expensive lobbyists and all the rest of it. The remarkable thing about Kate Learoyd and Caroline Graham is that they are parents of children with the condition, and their campaign is entirely voluntary. They have to fit it around their family and other commitments, which makes their work all the more remarkable.

--- Later in debate ---

[Ian Austin (Dudley North) (Lab)]

Like other hon. Members, I had never heard of this condition until I was contacted by a constituent, Kirsty Thornton from Dudley. She is the reason why I am taking part in the debate. To hear how the condition had affected her was very moving, and as a result of meeting her, I met Kate Learoyd and Caroline Graham, to whom I paid tribute earlier. I want to mention one other person at the outset. Professor Anita MacDonald OBE, who is with us today, is the brilliant head of a dedicated team of dieticians caring for children with rare inherited metabolic disorders at the Birmingham Children’s Hospital. I want to tell the Minister that Kate Learoyd, Caroline Graham and Anita MacDonald are here today. I hope that, at the conclusion of the debate, he might find a moment to say hello to them and arrange to meet them properly and at length subsequently.

It is deeply moving to hear how families manage this condition, particularly for toddlers, who cannot understand why they are not allowed to eat the same food as their siblings or have the same food as their friends at a birthday party. One parent has said of their child:

“She resents the fact that her family can eat normally and she can’t. At mealtimes, she will go into a depression. Often she will ask to eat the crumbs of normal bread off our plates or we catch her licking our plates. It is awful to see.”

It is very distressing to hear how a simple mistake can have huge ramifications, affecting a young person’s concentration and even their mental health and then their ability to study or to work. One young person said:

“When I have high phe levels I slur my words, struggle with balance, lose my train of thought and stop speaking...I am hit with such fatigue that I lose sight of what it is like to feel awake.”

I would therefore like to encourage the Minister and the Opposition spokesperson, my hon. Friend the Member for Washington and Sunderland West (Mrs Hodgson), to join us on the PKU diet challenge this Thursday—indeed, I hope that as many hon. Members as possible will take part—to highlight the difficulty of adhering to the strict rules that people with this condition have to follow. We have to do this properly and stick to the rules. We have to check all the ingredients, as we heard earlier, weigh foods properly and keep a tally throughout the day of the amount of protein that we have eaten. Most of all, as we have just heard, we have to remember that we are doing it only for a day and not a lifetime, like the constituents on whose behalf we are speaking today.

I want to ask the Minister three other questions before I conclude. First, as he has heard, we are calling for an examination of the failure to use Kuvan in the UK. That treatment can transform people’s lives. It has been licensed for almost 10 years and is used in lots of other countries in the EU. We would like the Minister to take personal charge of this matter and work out what can be done to sort out provision of it in the UK, too.

Secondly—we have raised this issue in respect of other conditions and other drugs—will he look at the appraisal rules for new treatments for PKU? This is a very rare disease, but it has a wide impact outside direct health costs. It is not a criticism of NICE or the Minister or Government, but the fact is that these are conditions and treatments that NICE was not really designed to deal with. How can the appraisal system be altered to work for conditions such as this?

Finally, will the Minister work with the NSPKU to review the provision of treatment to patients, including basic dietary treatments? How can that be made consistent across the country? That is a very urgent thing that it should be possible to sort out without too much difficulty.