Teen denied 'miracle drug' in postcode lottery

A 19-year-old woman diagnosed with a rare SOD1 gene mutation of motor neurone disease (MND) faces a critical challenge in accessing a new drug, Tofersen. This medication has proven effective in slowing the progression of the debilitating condition. However, access to Tofersen is determined by regional NHS services, creating a postcode lottery that has left the teenager, Lillia Jakeman, unable to receive the life-changing treatment in her local area, despite the pharmaceutical company supplying it free to the NHS.

Lillia's family expressed deep frustration over the denial of access, highlighting that the drug is available in some parts of the country but not others. The treatment involves monthly spinal injections, and local health services cite insufficient funding for staffing and associated costs as reasons for not providing it. This situation has led the family to set up a fundraising page to cover the costs of private treatment, as local NHS trusts have rejected requests for the drug, emphasizing financial constraints and the need for NICE approval and wider NHS England funding.

Experts predict that Tofersen can significantly stall MND progression for years, with patients reporting improved mobility and lung function. The family is now considering relocating to access the drug, a difficult prospect given Lillia's mobility issues and adapted home. The situation underscores the emotional toll on patients and families when life-saving treatments are inaccessible due to systemic and financial barriers within the healthcare system, leaving them in a state of 'daily mental torture.'