Lily suffers from a rare disease
Half a year later, 19 year-old frustrated sufferer Lily Wood wrote to the PM to ask why she and others in agony are still waiting. And Mr Johnson was so stirred by her words he rang her family promising to see how the NHS can reach an accord with drug firm BioMarin. Lily’s uncle Graham Anstiss, 47 – one of Mr Johnson’s constituents – revealed the PM rang him on Friday morning, moved by her letter.
Mr Anstiss told the Daily Express: “I was bowled over by how genuine, positive and concerned Mr Johnson was.
“He said he was really moved by Lily’s letter and will pursue the Kuvan situation and see what he can do for people with PKU like Lily.
“I explained how unfair it was that it you become obese you can get an NHS gastric band, yet Lily was born with PKU and cannot get Kuvan on the NHS.
“He also agreed with me that because there were so few PKU sufferers in the UK, their plight goes largely unnoticed.
“Mr Johnson said he could not promise to deliver Kuvan, as that was mainly an NHS decision, but admitted the case was overwhelming.”
Genetic condition Phenylketonuria, or PKU, forces sufferers to live on a severely-restricted dietary regime, as too much protein can cause brain damage.
Untreated, PKU may lead to intellectual disability, seizures, behavioural problems, and mental disorders. It can also result in a musty smell and lighter skin.
Sufferers’ low protein diet – topped up with synthetic protein – rules out all meat, fish, eggs, pulses, dairy, and even some vegetables and fruit.
Since 2009 US firm BioMarin’s drug – which could help 600 UK sufferers led a normal life – has been approved for use but not available on the NHS.
Meanwhile 26 other European countries including Latvia, Bulgaria and Estonia, all have it.
In February our “Supply Kuvan On the NHS” campaign launched, demanding a BioMarin deal.
Actress Lily, of West Drayton, in Middlesex, explained in her letter how unlike smokers or the obese, she had no choice about having PKU.
Lily must carefully weigh and measure every meal and snack
Yet the NHS spends billions of pounds a year treating obesity and smoking-related illnesses – but will not pay £4,000-a-year to give a little child Kuvan.
Lily said: “I have been permanently hungry all of my life.
“PKU sufferers didn’t choose to have PKU; they didn’t get this condition through making unhealthy life choices.
“Our lives are polystyrene-tasting bread and painstaking counting and measuring of food.
“Eleven years ago Kuvan was approved for use in the UK but was not funded by Nice despite being on offer in every EU country except Poland.
“Kuvan could mean most of us could enjoy food – instead of seeing it our enemy.
“I urge you now to change this, on behalf of the PKU community. We don’t want to be hungry anymore.”
Lily’s parents were shocked when she was born with PKU
Parents Chris, 56, and Hayley Wood, 44, were shocked when Lily was born with PKU, as they had never heard of it.
Each day she can only eat the protein equivalent of half a McDonald’s Chicken McNugget.
Lily must carefully weigh and measure every meal and snack and eats prescribed bread with fruit and vegetables.
She was bullied at school, with cruel kids often sprinkling their snacks on her meals knowing she could not then eat them.
At children’s parties she took her own plate of special food – as she could not munch on theirs.
Lily told us: “Once at a party I had a bit of a ham sandwich and my mum spotted me and ran over screaming!
“If I had Kuvan I could wake up and have a normal breakfast, like a bowl of cereal and a slice of toast.”
Mother Hayley said: “Kids with PKU often just want to enjoy what other people eat and it’s really difficult managing their diet.
Lily doesn’t let her disease intefere too much
“You feel like you have their future health in your hand all the time as excess protein can damage their brain.
“That weighs heavily on a parent’s mind. It’s just so unfair that Lily and others cannot have a drug that has been around a decade and could boost their quality of life.”
Earlier this year NHS England launched a draft proposal that Kuvan be “considered for routine commissioning” – a decision will be made in July.
But disgracefully, their Clinical Prioritisation Advisory Group review has refused to listen to appeals from PKU sufferers, by dropping their “impact statements”.
Campaigner Kate Learoyd, 46 – mother of PKU suffering son Alex, 12 – said: “The support of the Daily Express has meant a huge amount to us.
“This drug is already available across Europe but not in Britain. Kuvan could allow people the chance to have a healthy life.”
Any NHSE Kuvan decision will be interim depending on a final ruling by drugs appraiser the National Institute for Health and Care Excellence.
Last October BioMarin quit Nice’s appraisal of their drug but just days into our campaign this year they agreed to re-engage again.
Kuvan costs around £45,000 per adult, per year and as low as £4,000 for a child.
The drug could help most – but not all – PKU sufferers.
Downing Street declined to comment on Mr Johnson’s private call to the Wood family.
But instead a Government spokesman said: “We understand how distressing this condition is for those affected and their families.
“We’re determined to help all NHS patients access the latest treatments at a price that is fair to the NHS and the taxpayer.”
• To sign the PKU/Kuvan crisis petition, click here.