The Tuberous Sclerosis Association (TSA) has welcomed NHS England’s announcement that everolimus will be funded from April 2019 for patients living with Tuberous Sclerosis Complex (TSC)-related epilepsy, where the condition has not responded to standard anti-epilepsy medicines (refractory epilepsy).
TSC is a rare genetic condition affecting 1 in 6,000 people that can lead to growths in various organs of the body. These growths may be referred to as benign and non-cancerous tumours, with problems from the growths being caused mainly because of their size and where they are in the body. The organs most commonly affected by the growths are the brain, eyes, heart, kidney, skin and lungs.
Epilepsy is the most common neurological feature of TSC, affecting at least eight out of ten people living with the condition. More than 50 per cent of people with TSC who have epilepsy will not respond to standard anti-epilepsy medicines and may need an alternative form of treatment, such as everolimus. Epilepsy is generally more difficult to control for individuals living with TSC who have moderate or severe learning disabilities.
Philippa Ward, mother to seven-year-old Thomas who lives with TSC, commented on NHS England’s decision: “Everolimus has changed our lives. Thomas used to have eight seizures a day, but this went down to two a week when he started on a clinical trial investigating this medicine as a treatment for epilepsy associated with TSC. His seizures also became much more predictable, which really helped with our family life. Fewer seizures meant that Thomas’ development came on and his brilliant personality really began to shine through. It’s a fantastic Christmas present to know that more children and families living with TSC will be able to find out if this drug can help them too.”
Dr Chris Kingswood, Consultant Nephrologist and Head of Research Strategy at the Tuberous Sclerosis Association (TSA), said:
NHS England has always recognised that there is enough clinical evidence to commission everolimus for TSC-related refractory epilepsy. The 20 per cent chance of seizure freedom and 60 per cent chance of significant seizure reduction from treating this group of patients with everolimus is a massive improvement compared to using traditional anti-epileptic drugs. Clinicians supporting people living with TSC will be thrilled to add everolimus to the range of treatments that we can offer to patients with TSC-related refractory epilepsy.
TSA Chief Executive Louise Fish says:
Around 70 people in England are currently prescribed everolimus for TSC-related kidney and brain tumours. NHS England estimates that a further 300 people in England will benefit from treatment with everolimus for TSC-related refractory epilepsy. We’re delighted that NHS England has decided to fund this life-changing and potentially life-saving treatment from April 2019 onwards. We’ll be working with TSC clinics across England to help them get ready to prescribe this drug to more people who can benefit from it.
Everolimus can be life-changing for people with TSC-related refractory epilepsy. When the TSA spoke to 15 people who had experience of taking the drug:
- 14 out of 15 people reported significant improvements to their epilepsy and quality of life as a result of taking everolimus.
- 5 out of 15 people reported that the treatment was ‘life-changing’.
- 3 out of 15 people reported that they had had no seizures since taking everolimus.
Everolimus is a holistic treatment for people living with TSC. In addition to treating TSC-related refractory epilepsy, there are further benefits to patients including preventing high risk of kidney problems, lung problems and brain tumours (subependymal giant cell astrocytomas, or SEGAs). Everolimus can also be life-saving for people with TSC-related refractory epilepsy because it reduces the risk of sudden death from epilepsy (SUDEP).
The Tuberous Sclerosis Association (TSA) (http://www.tuberous-sclerosis.org) is the only UK charity dedicated to supporting people affected by TSC.