The Courier & Advertiser (Perth and Perthshire Edition)
Little Max doing well thanks to wonder drug.
Dundee medic helps 18-month-old access experimental treatment
A Tayside tot will live far longer than the year he was given by medics when diagnosed with a devastating illness, thanks to the help of a Ninewells medic who secured access to a wonder drug.
Perth youngster Max Farmer is now 18 months old and looking forward to a future it looked as though he would never have.
With the help of an NHS neurology medic, the toddler has become the first Scottish child to receive an experimental treatment.
Mum Elaine, a teacher at Oakbank Primary, said she had noticed problems in Max from a very young age.
She said: “When Max was nine weeks old, myself and his dad, Stuart, noticed a lack of movement in his arms and legs.
“He was diagnosed with spinal muscular atrophy type 1 at Ninewells and we were told he might live for a year at the most. The situation was that his muscles would degenerate and Max would not be able to breathe or eat.”
Just as Elaine, Stuart and 10-year-old sister Beth, were preparing for the worst, help came in the form of Ninewells neurology medic Professor Martin Kirkpatrick. Mr Kirkpatrick managed to get Max onto an expanded access programme which grants patients access to medical treatments which are still to be formally approved.
He was given the drug Spinraza. It was specifically developed to target spinal muscular atrophy, and as such is known as an “orphan drug”.
“Professor Kirkpatrick has been just amazing,” said Elaine. “Getting Max on that expanded access programme has been a lifesaver.”
Max has been receiving Spinraza treatments since he was 14 weeks old.
Biogen, the company providing the drug, says a single dose of Spinraza costs $125,000 (£89,500).
However, it is being provided to the family without charge.
“Biogen are providing the drug on a compassionate basis as Max missed out on their trial phase,” Elaine said.
“He is the first Scottish child to be given this treatment and, due to his swallowing being deemed unsafe, he is fed through a tube in his stomach.
“However, Max has come on so well. He can now sit unaided and move his arms and legs about, his arms to above his head.
“He can roll over and sign to us to let us know what he wants. He is becoming a very clever wee boy.
“His speech might be delayed but he shouts and cries like any other child.
“As far as we are concerned, they are noises we thought we would never hear. Noises of sheer joy.”
The Scottish Medicines Consortium (SMC) have met to discuss the benefits of Spinraza to those with SMA and have made a recommendation to the NHS in Scotland about funding the drug on the NHS.
Professor Kirkpatrick has been just amazing. Getting Max on that expanded access programme has been a lifesaver. ELAINE DONOGHUE