Two young sisters with the same rare disease will finally be treated together after months in hospitals more than 400 miles apart in two separate countries.
Nicole and Jessica Rich have both been diagnosed with Batten disease, a currently-incurable condition which leaves most sufferers unlikely to survive to their teens.
Since January last year Nicole, aged six, has been receiving treatment at London’s Great Ormond Street Hospital (GOSH) after clinching a place on a trial that could add years to her life.
Two-year-old Jessica also managed to get onto a sibling trial in Hamburg, meaning the family have spent almost a year Travelling between Germany, London and their home in Throckley, Newcastle.
But now parents Gail and Matthew have finally learned that Jessica will be able to transfer to GOSH for treatment.
Gail, 40, said: “We still can’t believe it’s actually happening. It is going to be a very overwhelming and emotional day and we can’t even imagine how it will feel to see our two little girls in their beds next to each other having their infusions at the same time.
“It is going to make such a difference to our family life, not just for Matthew and I being together to support each other, but also for our son Louis to have us all home at the same time.”
The family are also continuing their campaign after the National Institute for Health and Care Excellence (NICE) recommended the drug Nicole and Jessica are on should not be made available on the NHS.
NICE decided there is not enough long-term evidence about the effectiveness of cerliponase alfa to make it available on the health service.
While the decision could yet be reversed, experts have recommended it isn’t approved as it isn’t considered a “good use of NHS resources”.
It means the sisters’ access to the drug remains in the hands of the US drug company, casting doubt over the availability of the drug for children in the future.
Gail said: “They said they couldn’t say for certain if it was value for money, but how can you put a price on a child’s life?
“This treatment is working for both of our girls, we’ve seen it ourselves.
“Right now there are families around the country who are being diagnosed and they have no hope of getting any treatment at all until this decision is made.
“When Nicole was first diagnosed and we were told there was nothing anyone could do, it was the worst feeling you can imagine.
“Getting both our girls on this trial has given us hope, and that’s the biggest thing.”
A change.org petition started by the Batten Disease Family Association against the NICE decision has now received more than 86,000 signatures, and another review is due in April.
Source : Chroniclelive