Kristi Ouimet is hopeful her 15-year-old daughter will be admitted into a clinical trial of an experimental treatment for hyperoxaluria, a rare kidney condition that causes formation of painful and even life-threatening kidney stones. If she is enrolled in the trial, it will require a monthly commute from California to Minnesota for 2 years. But she’s not deterred. She’s already managed 2 years of a 6-day-per-week commute for 4 hours of daily dialysis plus 7 days a week of peritoneal dialysis for her younger son, who is also affected by hyperoxaluria and required a kidney and liver transplant.
“[If] driving all through snowstorms in Minnesota is going to prevent her from having to experience what my son did, I’m there,” Ouimet said in an interview with Kidney News.
Ouimet and other patient advocates shared their perspectives about the disease and the need for treatment options at a February 2018 workshop convened by ASN’s Kidney Health Initiative (KHI). KHI is partnering with the US Food and Drug Administration (FDA) and other stakeholders to pave the way for development of new treatments or the repurposing of existing medications to treat kidney diseases. Hyperoxaluria is one of the first rare diseases that KHI has set its sights on. The goal is to help identify clinical trial endpoints and designs that pharmaceutical companies or researchers can use to test potential new therapies.
“We like to work on projects where there’s a clear need and then there’s also kind of a clear opportunity,” said Melissa West, project director for KHI. Companies are interested in producing medications for hyperoxaluria, but one of the hurdles is finding ways to measure if their treatments are working in clinical trials, she said.