Campaign update at NSPKU2022.

Kate Learoyd, Caroline Graham, and special guest Liz Twist MP.

Liz Twist MP spoke to the conference in her role as Chair of the All Party Parliamentary Group (APPG) for PKU. She began by extolling the difference which Sapropterin makes for some, but she also acknowledged the need to work for those who it will not help.

Discover the genetics of PKU and how sapropterin works here

or find my lessons from a sapropterin trial here.

Liz Twist began campaigning for access to sapropterin on behalf of one of her constituents, but she said: “No one story would ever have made this happen. It was everyone’s effort.”

“No one story would ever have made this happen. It was everyone’s effort.”

NSPKU took individual stories and the results of their surveys to parliament, where they had real impact. The real-life stories are the ones which make a difference to the MPs, and the “penny drops when they see the exchange amounts”. The NSPKU display showing the amount of different foods allowed as one exchange had a notable impact and garnered support for the campaign.

The conference session did segue into a discussion on the protocol for defining responsiveness to sapropterin. (I will post on this soon.) Speaking as Chair of the APPG for PKU, Liz said that the group and the NSPKU need to work out how to move into the next phase of campaigning.

Looking forward to a continued campaign for new treatments, Liz Twist noted a key lesson which the APPG had learned was don't give up.

Don’t give up!

Kate Learoyd acknowledged that MPs may be bored with hearing about PKU now, but that they are stuck with the NSPKU and the ongoing campaign for better treatment for those with PKU. A positive and ongoing effect from the sapropterin campaign is that relationships have been made. There was an emphasis among all speakers, that the networks will be maintained, as these will aid the fight for future treatments.

Professor Anita MacDonald, speaking from the audience, added that another big lesson from the sapropterin campaign was that any future treatment should be trialled in patients to inform response protocols, rather than the other way around. This was well received in the room.

Prof. MacDonald went on to say that health professionals must take a scientific approach to assessing any evidence for new treatment. But they must also remember that patients must be given a chance to access the treatments which they deserve.

This session was a little fraught at times, with much discussion on sapropterin protocols. It was reassuring that those who do not respond to sapropterin have not been forgotten. The lessons of persistence and collaboration learnt in the campaign for treatment will strengthen the continuing fight for better PKU treatments in the UK.