A family are campaigning for NHS bosses to fund a wonder drug which can save the lives of people with are rare genetic condition.
Darcy Holt and dad Simon both have Tuberous Sclerosis Complex (TSC), which causes mainly benign tumours to form around the body, affecting the brain, skin, kidneys, heart, eyes and lungs.
As a result of the condition, around 20 youngsters a year develop inoperable brain tumours.
The drug Everolimus can shrink the tumours and stop them growing - but is not routinely available on the NHS for the tumours, known as SEGAs.
Darcy, four, is on a clinical trial for Everolimus because she has epilepsy caused by the condition.
The Pontefract family fear that Darcy and dad Simon, 29, could potentially develop SEGAs and be left to make a funding request to the NHS for a drug which could be refused.
Mum Jess Holt, 30, said: “We know that Darcy is going to have to come off it when the trial ends.
“I know after that if she needs it we’ll have to fight for it. It’s a ticking time bomb.”
Mrs Holt said Darcy no longer needed frequent hospital visits after starting on Everolimus.
She said: “Darcy has gone from having up to 90 seizures a day to one or two a week. She hasn’t been to hospital as much and we haven’t had to call ambulances.”
On October 26 the family took part in a protest outside Parliament in London calling for Everolimus to be funded.
The Tuberous Sclerosis Association has been urging the NHS to approve the drug.
Chief executive Jayne Spink said: “The medicine these young people need in order to continue living is sitting on NHS pharmacy shelves.
“It is totally abhorrent that their families and their doctors should be forced into planning for the end of life for these patients and unthinkable that a young person be allowed to die when the treatment that could save them costs less than kidney dialysis. “
A cross-party group of MPs led by Yorkshire parliamentarians Julian Sturdy and Greg Mulholland have written to the NHS chief executive Simon Stevens urging him to make the Everolimus drug available.
Mr Sturdy, the York Outer MP, said: “Everolimus is a very effective drug which is already used to treat other symptoms of this disease, such as kidney tumours, and it is crucial that NHS England acts to ensure that sufferers of inoperable brain tumours are able to access the treatment they desperately need.
Mr Mulholland, the Leeds North West MP said: “For the last two years, I have worked with families, campaigners and MPs and led the #FundOurDrugsNOW campaign.
“We received a written guarantee that Everolimus should have been approved in April for treating brain tumours. Unfortunately NHS England then changed the way it decides which drugs to prioritise for funding, and drugs for ultra-rare diseases were unfairly disadvantaged. Everolimus, as a result, was then not approved.”