A Wallaceburg couple wants to have the ability to choose what medication is best suited for their two young children, who both suffer from a rare disease.
Katelyn Campbell, who lives in Wallaceburg with her husband Adam, told the Sydenham Current their son Alan, 4, and daughter Nora, six-months, both live with a rare genetic disease called cystinosis.
“It’s lifelong, it’s incurable,” Katelyn said.
Cystinosis is a rare disease that is typically diagnosed prior to age 2. It is a genetic metabolic disease that causes an amino acid, cystine, to accumulate in various organs of the body. Cystine crystals accumulate in the kidneys, eyes, liver, muscles, pancreas, brain, and white blood cells.
Without specific treatment, children with cystinosis develop end stage kidney failure at approximately age nine, the Cystinosis Research Network states on their website.
“We have hopes, there is somebody working on a cure,” Katelyn said.
“We hold out hope that she will be successful. Right now with the current treatments available… you just take these medications as accurately as you can to try and prolong the damage being done to their bodies.”
Medication accessed through Special Access Programme
Katelyn said since Alan’s diagnosis when he was two years and eight months old, he has been on a drug called Cystagon to prolong his life.
Nora started this same drug at just three-weeks-old, Katelyn said.
The Campbell’s access this drug through Health Canada’s Special Access Programme.
Katelyn says both of her children are thriving with this treatment.
However, a new drug, Procysbi, has been approved to be sold by the pharmaceutical company Horizon.
This has essentially ended access to Cystagon without special approval from Health Canada for Alan, and for Nora once she is over the age of two, Katelyn said.
Just recently, Alan was given a four-month approval for Cystagon, but they will have to reapply every six months.
Katelyn said she knows a few people who have recently been denied coverage and they now live in fear that Alan will be denied access and forced to switch to the market available drug at any time.
“If they do not think your clinical reason for staying on Cystagon is strong enough, they can deny your access, and force you to switch to Procysbi,” Katelyn said.
“Both drugs have the same active ingredient, cysteamine. Procysbi can be given every 12 hours because of a coating, but means administration is particular, not making it ideal for all patients. There is also a significant price difference.”
Katelyn said switching drugs would not be in her families best interest, for a variety of reasons.
“We have built our lives around managing this disease for our children. We make hard decisions and sacrifices everyday to try to better our children’s health and provide them with the best life possible despite the complications that come with managing a rare disease.”
“We feel it should be our right to choose which treatment option is best for our children, alongside the guidance of our highly qualified medical team.”
Speaking at Lambton College
In order to raise awareness about their issue and concerns, Katelyn was set to speak at Lambton College in Sarnia on Thursday, April 12.
Katelyn, along with her friend Erin who has an eight-year-old daughter with cystinosis, were set to speak in front of 30 to 40 students about what the disease is and what is means to their families and children.
She said a photo was set to be taken and sent to some newspapers in Toronto.
Katelyn said Sarnia-Lambton MP Marilyn Gladu was set to be in attendance and a sit down meeting has been arranged with Gladu as well.
Katelyn added Ben Lobb, the MP for Huron-Bruce, is also putting forward a motion on April 16 to the standing committee on health.
“The motion is to open up an investigation or talks about the Special Access Programme and how it serves patients and rare disease patients and specifically about our access to cystagon,” she said.
Letters to MP’s
The Campbell’s are asking people to write to their local MP in order to raise even more awareness about their issue.
Katelyn said she has prepared a letter template for people to use.
It can be acquired by emailing Katelyn at firstname.lastname@example.org.
“We just want to be able to choose,” Katelyn said.
“There is two drugs that our kids can take, but they are not exactly the same. We just want the right to choose what is best for them.”
Watch for more on this story.
– Submitted photo