A costly drug that holds promise to keep Brienna Young’s son from getting brain damage should be subsidized by the province, she said Friday.
Her son Hayden Knox, now five, was born with PKU, or phenylketonuria, a genetic disorder that makes his body unable to process phenylalanine — an amino acid in most protein foods.
As a result, Hayden — and an estimated 150 other people like him in B.C. — has to stick to a low-protein diet so he doesn’t suffer brain damage from phenylalanine accumulating in his brain. In Kamloops, four kids and a few adults have PKU.
Pharmaceutical company BioMarin has developed a drug, Kuvan, that helps. But it’s expensive — it can cost up to $500 a day or $180,000 a year. Up to half of people with PKU can be helped by Kuvan.
BioMarin is backing a publicity effort by parents with children with PKU to raise awareness about the need for the B.C. government to help them cover the cost of treating their children with Kuvan. The company also funds a support group, Canadian PKU and Allied Disorders Inc.
Hayden, a bright-eyed and busy youngster, has been on Kuvan for four months as a trial. If his single mom, who’s just completed her nursing degree, had to pay the cost of that medication, it would run $50 to $100 a day.
She does pay for his specialized, low-protein food and he gets formula to ensure his nutritional requirements. Hayden can eat some fruits and vegetables (and Skittles, he was quick to point out), but much of the rest of his food is synthetic and has to be bought at specialty stores in children’s hospitals or via mail order.
Everything he eats has to be measured into exact amounts. And although most people would find the diet bland if not unappetizing, such as hot dogs and burgers made from synthetic powder, her son accepts it — so far.
“He loves having PKU. He’s special. He loves to tell people what he can and cannot eat,” she said. Still, she worries about the day he becomes rebellious or tired of the measuring and limited foods.
Young said it isn’t just Kuvan that parents of kids with PKU and PKU adults want covered; they want help with some of the food expenses. She spends almost $3,000 a year on specialized foods. They’d also like future drugs covered as they are discovered, like one that’s expected to be coming forward in five to 10 years, she said.
The only people who get coverage for Kuvan are those with private drug plans.
The B.C. Ministry of Health reviewed the drug this past summer and determined there were unclear benefits and high drug costs, which was also the recommendation of the Common Drug Review and the B.C. Drug Benefit Council.
The ministry also pointed out that no other provincial drug plan covers Kuvan, but it is provided in B.C. at no cost to those requiring the metabolic formula for proper body development.
Young said Hayden will probably be taken off Kuvan soon, because he’s not considered a responder. That is, he’s not having the desired reaction as far as his phenylalanine levels are concerned.
But she’s seen a difference in his behaviour; he listens better, his mood is improved and he’s more responsive. Unfortunately, it’s a difference that’s hard to prove.
Some other provinces in Canada are covering food and Ontario’s looking at providing Kuvan for pregnant PKU women.
With a provincial election looming, Young said the effort to get Kuvan covered is being ramped up. Even though she’s not usually political.
“We’re going to grassroots right now,” she said. “We went to the Ministry of Health. It didn’t do anything.”