WARRINGTON baby Theo Greenall, who was given less than two years to live, has bounced back from a rare genetic disorder thanks to the “world’s most expensive drug.”
Parents Natalie and Sean, both aged 33, had been given the devasting news that little Theo probably only had 18 months to live after he was diagnosed with spinal muscular atrophy (SMA), which restricted his breathing, caused his limbs to become floppy and prevented him from swallowing
But now thanks to “wonder-drug” Zolgensma Theo now aged 14 months has been giving a fighting chance of a long and happy life.
With a list price of £1.795 million per single dose, the “wonder drug” was made available after the NHS struck a deal with US-based manufacturer Novartis Gene Therapies.
The one-off infusion uses a harmless virus to deliver a healthy version of the SMN1 gene, which is defective in those with spinal muscular atrophy.
While qualifying for the drug last July, Theo still had to bounce back from breathing problems after doctors discussed pulling his treatment when he was admitted to hospital for six months with breathing problems.
Fortunately, he recovered enough to start the treatment in November.
Since then, Natalie said Theo’s mobility has increased dramatically – and the couple are now hopeful he will enjoy a long, happy life with them.
Natalie, who is a full-time carer for Theo said: “My son Theo Greenall was born on 3rd November 2020. Just 10 days later a midwife noticed his reflexes were absent and he was floppy.
“Theo was then admitted to Warrington hospital where they did various checks including genetic bloods.
“On the 27th November, we got the devastating news that the genetic results were back and Theo had Spinal Muscular Atrophy Type 1.
“Spinal muscular Atrophy can affect feeding, breathing and can be fatal.
“Without treatment, this is a terminal condition with a life expectancy of no more than 18 months. A few days later Theo started a disease-modifying treatment called Spinraza which is given through a spinal injection.
“In May Theo lost the ability to swallow and had surgery to have PEG fitted. His breathing also deteriorated and he suffered respiratory distress in July where he ended up being intubated in Intensive care in Alder Hey Children’s Hospital. In June a new gene therapy treatment called Zolgensma came out as another treatment for SMA and the NHS has agreed to fund it for some children who meet the criteria written by NICE.
“In July when Theo was in intensive care we got a phone call telling us that our son was eligible for the treatment but at that moment he was fighting for his life on a ventilator.
“As parents, we didn’t know how to react as it was such amazing news but on the other hand we saw our son struggling and lay in a cot in the paediatric intensive care unit.
“Thankfully Theo recovered from this episode of respiratory distress and he weaned of the ventilation and he had the new £1.8 million pound treatment in November.
“He is doing amazing and he is showing signs of progression and he is moving so much more.
“He is now able to move his legs against gravity which he has never been able to do and he has much better head control. We have been doing lots of assisted sitting so we are hopeful that in the next few months he will be able to sit unassisted. Both Zolgensma and Spinraza treatments have given us more hope in Theo’s life! We are grateful for the NHS and for the chance for Theo to have such amazing treatments.”
Natalie said she wanted to share Theo’s story to help raise awareness of his condition and the new “wonder drug.”