A mother who found a life-saving treatment for her critically ill son has made UK medical history – and given new hope to children diagnosed with the disease.
Catherine Baker was told by doctors that her son George’s only hope of survival for his disorder – Langerhans’ cell histiocytosis (LCH), which affects the liver – was chemotherapy, even though it would return after treatment.
Though seven-year-old George’s condition is not cancer, specialists treat LCH with chemotherapy, which is best known as a cancer drug. But every course put further strain on his liver, worsening his prognosis.
Ms Baker was so determined to help her son that she searched for her own solution. She spoke to a friend whose grandson had been on a medical trial at Cincinnati Children’s Hospital and found a US oncologist who uses a different approach – an inhibitor drug to block gene mutations that cause the condition.
Her discovery of the alternative treatment, trametinib, means that the family have just celebrated George’s seventh birthday – a landmark they feared they would never see. His survival also resulted in a UK first: a new method for treating children diagnosed with the condition.
After being told by doctors that her son George’s only hope of survival was chemotherapy, Catherine Baker discovered an another solution called trametinib
The family have just celebrated George’s seventh birthday thanks to the alternative treatment
George with his two sisters, Alice, aged 11 (left) and Beatrice, aged nine (right)
Ms Baker, from Sevenoaks in Kent, said: ‘George wouldn’t be here without the inhibitor treatment.’
The family’s nightmare began when he developed a rash on his head aged six months. Despite numerous visits to the GP, none of the treatments worked.
Ms Baker, a former tax lawyer, said: ‘Just after his second birthday, I noticed spots on his tummy. I took him straight to the hospital.’ Eventually it was found to be LCH, which affects about 50 children a year in the UK.
‘We were in total shock,’ she added. ‘It was devastating.’
George began chemotherapy, which appeared to work. But as soon as treatment finished in April 2020, the condition came back.
Ms Baker said: ‘The doctors said he had to have five rounds of chemo, but as it put so much strain on his liver, it was unlikely to cope.’
Frantic with worry, she contacted the oncologist in the US and the family flew out. She said: ‘He put George on the inhibitor drug and he improved straight away.’
George’s treatment is now being proposed in the NHS for children with LCH which affects their liver
But George’s liver was so badly damaged that in late 2021 the family was told he needed a liver transplant.
Again, Ms Baker saved her son’s life, donating part of her own liver. George underwent the operation in April last year – it was such a success he ran in his school sports day weeks later.
George is now receiving the drug through the Novartis compassionate access scheme, which makes unapproved treatments in the UK accessible to those eligible.
Ms Baker said: ‘George is a pioneer for this treatment in the UK – it’s now being proposed in the NHS as a first line of treatment for children with LCH where the liver is affected.’
George is also being supported by the Children’s Liver Disease Foundation. A spokesman said: ‘His story is amazing.’