NHS ‘abandons’ tumour children in drug U-turn

THE NHS rationing body has been accused of abandoning children with a life threatening genetic condition after it refused funding for a breakthrough drug that will let them live ‘long and healthy lives’.

Hopes were raised after ‘spectacular’ trial results in which youngsters with the tumourcausing disease tuberous sclerosis complex (TSC), some of whom had been given as little as 12 months to live, were saved by the one-a-day tablet.

But i n July, NHS England reversed an earlier decision to recommend prescribing the breakthrough drug everolimus, claiming that it was ‘not currently affordable’.

The U-turn came at the same time as judges ruled that NHS England should pay for the HIVprevention medication Truvada.

Last month a letter signed by 13 of Britain’s leading specialists from the fields of kidney health, genetics and cancer was handed to Health Ministers, expressing ‘shock and disappointment’. This was backed by a petition launched by UK charity the Tuberous Sclerosis Association.

‘Without this therapy [up to 20 children a year] will die, whereas with it almost all will live long and healthy lives,’ wrote consultant kidney specialist and TSC expert Dr Chris Kingswood, of Brighton & Sussex University Hospitals and St George’s Hospital Trusts.

TSC causes non-cancerous growths to form in the brain, eyes, heart, kidney, skin and lungs. Current estimates are that it affects one in 6,000 births.

Tumours can be slow-growing but in some cases patients may suffer potentially fatal seizures, kidney and brain infections.

First-line treatment includes seizure medication and surgery to remove growths and repair other kinds of damage.

Particularly hard to tackle is a type of brain tumour that affects up to one in five patients, known as subependymal giant cell astrocytomas (SEGAs). In patients with severe, progressive and inoperable SEGA, life expectancy can be as little as 12 months.

Trials of the new once-a-day tablet halved the size of SEGAs in patients who took it. Seizures were also significantly reduced.

Studies that followed hundreds of sufferers for five years led to the drug being hailed by paediatricians as ‘astonishingly successful’, with ‘minimal’ side effects.

Dr Kingswood said: ‘We have had young patients who have been given less than a year to live, but will now have a good chance of living a long and healthy life thanks to the medication.’

The price privately is £32,000 per patient per year, but the NHSnegotiated cost was said to be less than half that. The cost of treating a child with kidney failure caused by TSC is at least £30,000. ‘It makes no sense to deny them this treatment,’ said Dr Kingswood.

ONE youngster saved by the drug is 16-year-old Joe Baum, who in 2013 was given months to live when routine scans revealed SEGA tumours in his brain had grown considerably. His mother Mandy, a GP, said: ‘Joe was diagnosed with TSC when he was a baby and has epilepsy and is autistic, and muscle weakness in his left side. To be told we could lose him was indescribably painful.’

Within six months of everolimus treatment, the tumours had shrunk by half and Joe is now stable. His mother said: ‘With my doctor’s hat on, I can see everolimus is expensive but it is heartbreaking that other families might lose their child because they are denied this drug.’

NHS England has said funding will be reconsidered in 2017. ‘The Individual Funding Request route remains open to clinicians,’ it added in a statement.