Liverpool Father's Desperate Fundraising Mission for Son's Rare Disease Treatment
A father from Liverpool is engaged in a heartbreaking race against time to secure £300,000 for specialist medical treatment in Germany that could potentially slow the progression of his teenage son's rare degenerative condition before it permanently strips him of his mobility.
Jake McGregor-How, a 16-year-old from Mossley Hill in Liverpool, received a devastating diagnosis of Friedreich's Ataxia (FRDA) in December 2025. This life-limiting genetic disorder progressively damages the nervous system and can ultimately affect cardiac function, presenting a severe threat to Jake's future health and independence.
Years of Misdiagnosis and Sudden Decline
For five long years, Jake's emerging symptoms were mistakenly attributed to coeliac disease and anaemia, allowing him to continue living a relatively normal teenage life. During this period, he successfully completed his GCSE examinations and embarked on his A-Level studies with optimism and determination.
However, last summer brought a troubling deterioration in Jake's condition as his balance noticeably worsened and he began struggling to walk in a straight line. His father, Paul McGregor-How, aged 48, persistently pushed medical professionals for answers, leading to six months of intensive testing that culminated in the life-altering FRDA diagnosis.
The diagnosis arrived just three years after the family endured another profound tragedy. In 2023, Jake's mother Gaynor passed away from multifocal glioblastoma, an aggressive and rare form of brain cancer, leaving the McGregor-How family grappling with loss before facing this new medical crisis.
The Treatment Dilemma: Available Abroad but Not in Britain
While there remains no definitive cure for Friedreich's Ataxia, a drug called omaveloxolone has demonstrated significant effectiveness in slowing the disease's progression. This medication has received approval for use in various European countries and the United States, offering hope to patients and families affected by FRDA.
In the United Kingdom, however, the treatment remains inaccessible through the National Health Service. The National Institute for Health and Care Excellence (NICE) has been unable to recommend its use on the NHS, creating what Paul describes as an agonising reality for British families facing this condition.
Paul, who works as a claims manager, now confronts an impossible choice: raise £300,000 to fund a year of treatment at University Hospital Heidelberg in Germany or risk watching his son's condition deteriorate further without intervention.
A Treatment Within Touching Distance Yet Out of Reach
Speaking about the family's situation, Mr. McGregor-How expressed profound heartbreak at being unable to access a treatment that feels frustratingly close yet remains unobtainable. He revealed that following Jake's diagnosis just before Christmas 2025, he began researching the condition online and discovered the sobering prognosis for FRDA patients.
"People who have FRDA end up in a wheelchair within seven to nine years, and have sensory loss and heart problems," he explained. "To treat the disease, Jake was prescribed vitamins - calcium, vitamin D, vitamin E, and enzyme Q10. That's the only treatments offered on the NHS."
Determined to explore alternatives before Jake's condition worsened, Paul discovered omaveloxolone through online research. "It is heartbreaking because there is a treatment within touching distance - it's not like we are living in the third world, this is Great Britain," he lamented.
The German Treatment Option and Fundraising Challenge
Omaveloxolone represents an oral medication specifically designed to slow Friedreich's Ataxia progression by improving mitochondrial function in patients aged 16 and over. When combined with supportive therapies like physical therapy, speech therapy, and occupational therapy, the drug has shown potential to significantly improve both quality of life and life expectancy for FRDA patients.
The McGregor-How family has identified University Hospital Heidelberg as their best option for accessing this treatment. After an initial consultation and submission of Jake's medical records, the German clinic accepted his case, marking what Paul describes as "the first milestone" in their journey.
"If Jake was living in Germany, he would've had almost three months of Omaveloxolone already," Paul noted. "Unfortunately, that is not the case, and whilst he is managing, I can see him more unstable than he was, for sure, and getting more tired. Time is of the essence for Jake to access the drug."
The quoted cost for one year of omaveloxolone treatment at the Heidelberg clinic stands at £300,000, with additional expenses for travel and accommodation in Germany. Through fundraising efforts coordinated with the charity Tree of Hope, the family has raised approximately £100,000 toward their goal but faces a substantial remaining shortfall.
Political Advocacy and Family Resilience
Faced with what he describes as an "unattainable and unsustainable" long-term financial burden, Paul has questioned why the UK government has not reconsidered making omaveloxolone accessible for NHS patients. He has enlisted the support of his local MP, Paula Barker, the Labour representative for Liverpool Wavertree, to advocate for policy change.
Ms. Barker has described Jake's story as "absolutely heartbreaking" and has committed to raising the issue in Parliament. She has written to the Department of Health and is now preparing to contact Biogen, the pharmaceutical company manufacturing omaveloxolone, to urge resubmission of the drug for NHS appraisal.
According to NICE, their inability to recommend the drug stems from Biogen's withdrawal of its "evidence submission," with a spokesperson indicating they would "review this decision if the company decides to make a new submission."
A Family Navigating Grief and Hope
The quest for treatment carries additional emotional weight for the McGregor-How family, coming just as they were beginning to emerge from the profound grief following Gaynor's death. Paul described how he and Jake were "just crawling out of the deep grief of his mother dying and looking a little ahead to the future when we received the FRDA diagnosis."
Throughout both crises, Paul has noted Jake's remarkable resilience. "Jake was there the whole time his mother was ill – it's obviously upsetting. But he was stoic, he carried on, he didn’t go off the rails. And through his FRDA diagnosis, he has also been stoic."
Described by his father as a "talented artist and writer," Jake continues his A-Level studies in art, politics, and psychology while contemplating future possibilities including university education in Edinburgh or journalism studies. However, these plans remain uncertain as the family navigates their medical and financial challenges.
"We are basically in limbo," Paul admitted. "We don’t know and can’t plan ahead." Despite these uncertainties, he emphasised that he has not "given up hope" of eventually accessing omaveloxolone through the NHS permanently, allowing Jake to pursue his future aspirations like any other teenager.
