Give our kids the Parents fight for Batten’s wonder drug on the NHS
WITH smiles on their faces these beautiful children seem carefree and happy.
But their future hangs in the balance because a drug that has given many a new lease of life may be taken away at any moment.
Brineura is a revolutionary enzyme replacement therapy. Without it these children suffering from CLN2 Batten disease will die.
It was trialled in the UK and licensed in 2017. But it is £500,000 per child per year – and has been deemed too costly by the
NHS and Nice, t he National Institute for Health a n d Care Excellence.
The incurable neurodegenerative condition afflicts 38 people in the UK and 300 worldwide, robbing children of speech, sight and the ability to walk.
Brineura can slow the disease and increase life expectancy by 20 years. Many kids taking it are leading a normal life as a result – with virtually no symptoms of Batten’s.
Some children in England have taken part in a trial or are being given the drug on compassionate grounds by US developers pharmaceutical firm Biomarin. But this could end at any time.
Those just diagnosed and not on the drug will die unless it is made available soon. Two kids waiting for treatment have already passed away. It is currently available in 21 countries, including Wales. Now families are fighting back, bringing a judicial review against the funding decision.
As a result the action Nice and NHS England are back in discussions with Biomarin.
But the delay has a ngered families, prompting one dad to quit Britain and move his son abroad for treatment to save his life. Brineura is delivered directly into the brain, via a surgically implanted device, twice a month in hospital.
Without this drug the average life expectancy of a child with Batten is ten years.
The drug was meant to slow down the disease but parents of those on Brineura under the age of three have seen no signs of Batten’s.
Officially the guidelines say the drug gives the child 30 quality years. It has been shown to cut the rate of decline in ability to speak and move.
CLN2 disease is an inherited condition that causes progressive irreversible decline in ability to spea speak and move, loss of bal balance, convulsions, blin blindness and ultimately, de death in children.
People with CLN2 di disease do not have any e enzyme called TPP1 or th they have too little of it an and this causes a build-up o of substances called ly lysosomal s t orage m materials.
In people with CLN2 di disease, these materials bu build-up in certain parts of the body, mainly the br brain. Without the drug, pe people start to lose their abilities as their condition deteriorates and the cells in the brain die. Sufferers can die from a variety of conditions – including seizure that leads to coma, pneumonia, or gut failure.
After a recent meeting NHS England and the drugs company have agreed with Nice to undertake a review of the drug.
Samantha Barber, chief executive of the Batten Disease D Family Association, said: “Brineura is the first ever ev treatment for any form of Batten disease. It stabilises children at the point at w which they are treated. It is not a cure but it dramat dramatically improves t he qu quality of our children children’s lives and the lives of their families. For thos those who know the devas devastation of Batten dis disease, it is like
n night and day.”