Tate McDaid: Brit Boy, 5, With Rare Dementia Needs £2.95m Therapy
Tate McDaid, a five-year-old from Swansea with Sanfilippo Syndrome, could get the first approved gene therapy for the condition. His family has raised just over £200,000 of the £2.95 million cost, and his mother says time is running short before he turns six.
Tate McDaid, a five-year-old boy from Swansea with the rare dementia Sanfilippo Syndrome, could now access a US gene therapy approved in September, but his family needs almost £3m to pay for it. His mother, Tammy, says the clock is ticking because Tate turns six this month.
Tate was diagnosed at four. Sanfilippo Syndrome is a rare genetic disorder that progressively damages the brain. Children lose abilities they have already learned, including speaking, walking and eventually eating independently.
The disorder affects just over 100 children in the UK. It is often called "childhood dementia" or "childhood Alzheimer's".
The first gene therapy for the condition won approval in the US in September. The price is £2.95 million per child. Tammy said she was excited at first, but that "reality hit hard the next day" when she saw the US price had been set at $3.95m.
A race against time
Tammy said the disorder progresses more rapidly after the age of five. She described a "battle of time". Tate has "already shown signs of cognitive decline", she said, and she is "petrified" the family will run out of time.
"The older he gets the less the treatment will work and the more ability he will lose in the process," she said.
There is currently no cure for Sanfilippo Syndrome Type A.
Tammy began fundraising before the US Food and Drug Administration approved the therapy. She has raised just over £200,000 so far. That covers around 6% of the estimated cost.
"Unfortunately, we're in a world where we need millions, not hundreds of thousands," she said. She added that if a millionaire gave the family the money tomorrow, "we'd go to the US straight away".
Who pays for treatment abroad
Tammy questioned how the price works for families outside the US. "We understand what they are charging for American citizens, but they've got health insurance," she said. "What is it for us in the UK?"
She has contacted Ultragenyx, the company that developed the treatment, to ask how US families without health insurance will cover costs.
Ultragenyx said it is seeking a global regulatory strategy and FDA approval to bring the treatment to families around the world. It acknowledged the urgency families face and why some may consider travelling to the US. The company recommended families work with their child's healthcare provider to understand what access pathways may exist "before assuming this is their only option".
Noah's Ark Children's Hospital in Cardiff is liaising with officials in the US on the family's behalf.
Tammy describes Tate as "the happiest little man" who is "blissfully unaware" of his health problems. "He doesn't speak, he's never spoken," she said. "But his mobility is everything to him and that is why I'm fighting for it."
