An Armagh man acting as the only Northern Irish member of a UK-wide charity for people living with Phenylketonuria (PKU) is preparing for another “uphill battle” in campaigning for the roll-out of a new lifechanging medication.
Chris Cassidy – father of 14-year-old Daragh Cassidy, who lives with PKU – has long been an advocate for better awareness and access to treatment.
PKU is a rare medical condition which restricts the sufferer’s daily protein intake, as the body lacks the ability to properly break down the protein in food. In Daragh’s case, he was unable to consume more than 13g of protein a day prior to the introduction of medication.
Overconsumption of protein for someone with PKU could lead to permanent intellectual disability, seizures, developmental delays and psychiatric disorders among many other serious symptoms.
It affects on average 1-in-10,000 people in the UK and around 1-in-4,000 in Northern Ireland.
Two years ago, Chris began working closely with nationwide charity The National Society for Phenylketonuria (NSPKU) to help change legislation around the drug ‘Kuvan’ (saproterin), which has been hailed as potentially ‘life-changing’ for those with PKU.
The drug – which around 30-40% of PKU sufferers respond to – had demonstrated through trial that it allowed those who were responsive to significantly increase their protein intake. With Daragh, he increased from 13g of protein per day to a much more manageable 36g.
However, a new drug known as sepiapterin (Sephience) has now proven to be even more beneficial than Kuvan.
Related: ‘Power to the people’: Armagh campaigner celebrates ‘life-changing’ drug policy change
Speaking to Armagh I, Chris explained: “It’s a new better version of the old one which will respond to more patients with PKU. The old drug is only responded to by around 40% of the population.
“This shows better, more promising signs of results and at least 60% of the population will respond to it but it is a lot more costly… very much so.”
And, knowing first-hand the process involved with getting Kuvan legislation changed in NI, Chris is ready for an “uphill struggle” in getting sepiapterin across the line… especially given the price increase.
He added: “It’s at the stage where, as a charity, we are engaged with the pharmaceutical company who are making it to see if we can do something about that.”
The organisation have also booked a space at Westminster on November 11, to discuss the benefits of the new drug and process involved in securing the treatment on the NHS with MPs for all parties.
“This event in Westminster is to make the MPs in Parliament aware of what’s going to be coming down the line,” said Chris. “We want to tell them that there is a new drug and we need NICE to approve the medication.
“We did this as a charity when Kuvan was first introduced to the UK market. Back then it was to make people aware of what was going on so that MPs weren’t receiving emails from constituents and not knowing what was happening.
“Jeremy Hunt, the former UK Health Minister, will be in attendance and I can confirm there are 10 local MPs from all parties in NI to attend or have a representative from their offices attend, if the diaries permit. All six parties that are represented in Westminster and an independent have replied.”
Chris’s main aim is to secure sepiapterin for anyone living in the UK as an alternative to Kuvan, if they have shown no responsiveness to the treatment.
He said: “My son has been blessed and been lucky enough to have access to the old drug. He won’t be offered the new drug because it’s not going to be any more beneficial to him but there are other people out in the community that aren’t getting any benefit from the old drug but will from the new one that’s coming down the line.”
He knows they have a long road ahead but he’s hopeful that MPs will give the matter “just five minutes of their time” to pave the way for more detailed discussions, accumulated support and potential lobbying further down the line.
The public can also get involved by using templates provided on the NSPUK website to write to your MP, tell your story or write on behalf of a friend, neighbour or family member.
