Mum campaigns for new drug on the NHS to help slow progression of son's incurable disease

Mum campaigns for new drug on the NHS to help slow progression of son's incurable disease Sarah Officer, 41, said the approval process is "taking too long" and that her son Jake is "not the kid he used to be" as his incurable condition progresses A desperate mum is campaigning for a new drug to be made available on the NHS that could radically slow the progression of her teen son's incurable disease. Sarah Officer, 41, is part of a group of parents of children with Friedreich’s Ataxia - a rare inherited disorder that primarily affects the nervous system, causing progressive damage to the spinal cord, peripheral nerves, and cerebellum, leading to impaired muscle coordination. Jake, now 18, was diagnosed aged nine in September 2017. While there is currently no known cure for the condition, new drug Omaveloxolone has been found to slow progression by as much as 55 per cent and was approved for use by the MHRA in April last year. The drug was under evaluation by NICE but is is yet to be made available for use after manufacturer Biogen withdrew its submission last year, discussions around the appraisal process. Sarah said the approval process is "taking too long" and just 18 months later Jake is "not the kid he used to be." Sarah, from Tunbridge, Kent, has now joined with other parents to launch a petition to fund NHS access to Omaveloxolone for patients with Friedreich's Ataxia, which was published in March. The petition has now reached over 100,000 signatures and will be considered for debate in parliament. "To know there is something out there that could help is tough," said customer assistant Sarah. "This is all taking too long. "There’s a drug out there that was approved in April 2025, we’re a year down the line. I’d like things to move quicker than they are really. "I’m a really positive person and I think there is something out there that could be the gamechanger, but this is the gamechanger for now. Jake is not the kid he used to be. It’s affecting everything, his mental health." Sarah said Jake was in "fine health" until he reached 17 when one day she was getting him out of the bath and noticed he was leaning to one side. Doctors ran tests before initially diagnosing Jake with scoliosis, and he wore a spinal jacket for two years to try and stop the curvature of his spine. Jake also has "umbrella problems caused by Friedreich’s ataxia" other conditions including scoliosis, asthma, FND, sleep apnoea, anxiety and depression, and hypertrophic cardiomyopathy – where the muscle wall in the heart becomes thicker than it should causing it to beat faster than normal. "It’s really cruel, it’s like a death sentence,” Sarah says. It’s heart-breaking. Your world is turned upside down and you go through the stages of grief. "It’s a terrible diagnosis for a child. Nobody wants to hear in the future what your child is not going to have and what you’re not going to have. "Like all the milestones everybody else has like getting married, having children, having a job. It’s been a really tough few years. "For me, you get the news and I packed it away in the back of my brain and focus on the day-to-day stuff just to get through it. If I think too much and dwell on it it’s not nice – I couldn’t manage without compartmentalising it and caring for Jake." Currently, there is no cure or treatment for Friedreich’s ataxia in the UK, with physio and hydrotherapy offered to help with mobility. "As you can imagine the lists for physiotherapy are really long and services are overstretched so it’s quite hard to get physio,” Sarah said. "Jake qualified for hydrotherapy through the NHS but we only managed to get five or six sessions – you need a continuous physio plan for it to work." As part of a Facebook group for parents of children with Friedreich’s ataxia, a petition was published urging the NHS to fund access to Omaveloxolone. It currently sits at 102,354 signatures. You can read the petition here. A NICE spokesperson said: "NICE was unfortunately unable to make a recommendation on omaveloxolone (Skyclarys) for treating Friedreich’s ataxia in people 16 years and over because Biogen withdrew its evidence submission. "We provide rigorous, independent assessment of complex evidence for new health technologies to make sure the NHS can continue to offer the most effective treatments to everyone. "NICE must balance the benefits that a new medicine offers with the savings that have to be made from other vital patient services elsewhere in the system to pay for it. "Unfortunately, NICE cannot recommend a drug if the company does not take part in our work, and the outcome of an evaluation cannot be assumed. "NICE stands ready to work with the company and will reopen the appraisal if the company decides to make a new evidence submission."