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Scientists have been exploring whether the drug cerliponase alfa could be a suitable treatment for Batten Disease, also known as CLN2.
The National Institute for Health and Care Excellence (NICE) has chosen not to recommend the drug for NHS use in an initial draft recommendation, but its developers believe there is evidence to show that it can improve some of the symptoms of Batten Disease, if only in the short term.
Symptoms of the rare genetic illness include slurred speech, decline in communication skills, loss of mobility, the development of seizures, progressive dementia and muscle spasms. Currently the average life expectancy for patients is just ten years.
Initial trials involving cerliponase alfa have shown that it can temporarily help to stop some of these symptoms from progressing as rapidly, but NICE has decided not to recommend it for NHS use as there is no evidence of any long-term benefits of the drug.
Cerliponase alfa would need to be administered directly into patients' brains via a surgically implanted device, with each year of treatment costing more than £500,000.
Although it could potentially help to reduce the frequency of seizures suffered by Batten Disease patients and subsequently improve some of the communication problems they experience, access to care from healthcare professionals including speech therapists and occupational therapists would still be required even if NICE turned its initial decision around.
Dr Peter Jackson, chair of the independent Highly Specialised Technology Committee at NICE, commented: "Although cerliponase alfa is not a cure for CLN2 disease, it is an innovative treatment that is effective in the short term in slowing the rate at which it progresses.
"Given the significant burden this disease places on parents and carers of children with the condition, and the subsequent negative impact this can have on the quality of their lives, anything that can help to improve the treatment of these children is to be welcomed."
Written by Martin Lambert
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