New drug helps young Ottawa girl fight ‘ultra rare’ disease

By Chris Hofley, Ottawa Sun

Morgan MacIntyre is literally one a million.

As a baby, the now 12-year-old was diagnosed with what the medical community calls an “ultra rare” disease: atypical Hemolytic Uremic Syndrome (aHUS).

But despite the rarity — aHUS affects only about 60 Canadians — of the illness and the impact it has had on her family for a decade, there is no woe-is-me attitude coming from the sixth grader.

At CHEO Wednesday for her regular, bi-weekly treatment to keep the disease from ravaging her internal organs, the preteen is more likely to burst into a fit of giggles than complain about another trip to the hospital.

The procedures, hospital visits and the port-a-cath attached to her have, after all, been part of her life since before she can remember.

Morgan’s illness causes part of the immune system to turn on the body, attacking both healthy and unhealthy cells, which causes abnormal blood clotting and vessel damage, severely damaging vital organs in the process.

“I cried for two straight days, and I’m not a crier,” said Morgan’s mom, Tracy, of the moment Morgan was diagnosed.

But things are getting better for Morgan and her folks, the founders of a national support group for people with the disease.

Her bi-weekly treatment using a just-approved drug has allowed Morgan to visit CHEO less frequently and the side effects are almost non-existant. Health Canada’s approval of Soliris means Morgan can go to school regularly, ride horses and enjoy being a kid.

But don’t let her age fool you; Morgan’s experience with aHUS has turned her into a wise-beyond-her-years kid who isn’t the least bit sorry for herself. She chooses to focus on the positives and looks at CHEO as a big extended family, rather than a hospital.

She’s also is getting a few years worth of science class experience outside of school by keeping a keen eye on the doctors and nurses.

“We (studied) the human body last year and I knew stuff they weren’t even talking about at school,” she said.

The MacIntyres have been able to get the new treatment covered through their insurance companies, but with an average price of $500,000, they have to re-apply every year as the treatment isn’t covered by OHIP.

Even as managing the disease gets easier, it’s never out of mind and never will be, said dad Kevin.

“It involves the whole family,” he said. “It’s tough.”

atypical Hemolytic Uremic Syndrome

  • WHAT IS IT: A life-threatening, chronic disease that primarily affects kidney function. It causes abnormal blood clots to form in small blood vessels in the kidneys.
  • WHAT CAUSES IT: It often results from a combination of environmental and genetic factors. Most cases are sporadic, which means that they occur in people with no apparent history of the disorder in their family. Less than 20 percent of all cases have been reported to run in families.
  • SYMPTOMS: nausea and vomiting, confusion, shortness of breath, fatigue, anemia (low red blood cell/platelet count in the blood), thrombocytopenia (low platelet count in the blood)
  • TREATMENT: There is no cure. Treatments include blood transfusions, plasma therapies, dialysis and medications.