Investigator’s tiny kidney systems gain a national stage
Investigator’s tiny kidney systems gain a national stage
Organoids that model human responses to potential therapeutics will join a consortium focused on polycystic kidney disease.
Miniature in vitro systems of human cells that model kidney function have given UW Medicine entrée to an elite U.S. research alliance.
Benjamin Freedman, associate professor and nephrology investigator at the UW School of Medicine, learned recently that the organoid system whose development he has led for the past decade will become a cornerstone of the Polycystic Kidney Disease Research Resource Consortium.
The consortium’s newest member will join through a five-year award from the National Institutes of Health, which was formally announced in September.
Freedman interpreted the award as a validation of a living-systems research approach that does not involve animals, and which can more closely model human responses to therapeutics being tested.
“It's a novel idea to have tiny organoids that respond in the same exact way that a kidney would. You see (polycystic kidney) disease materialize before your eyes. The cyst structures that are characteristic of the disorder, you can see in three dimensions,” he said.
“It's more difficult to figure out disease mechanisms when you're limited to animal models because of the complexity and confounding variables that are present,” Freedman added.
Polycystic kidney disease, commonly called PKD, affects 12.5 million people worldwide and is one of the most common inherited genetic disorders. The disease is characterized by fluid-filled sacs that develop in the kidneys and curb the organs' function. Patients typically show symptoms in their 30s, progress rapidly and need kidney transplants by their 50s. Aside from the faltering kidneys, other organ systems decline in parallel.
Scientists have known PKD’s associated genes for decades, but no broadly effective therapy exists, said Freedman, adding, “I feel strongly that we don’t have full grasp of how the disorder works.”
Freedman's lab, the consortium’s first West Coast member, joins five other “core centers.”
“Our center will be dedicated to the use of in-a-dish systems for studying PKD. The idea is that we’ll help spread the [organoid] technology. In five years, hopefully we’ll be talking more about what mechanisms are driving the cysts to form, and about the ability to test many different therapeutics much more rapidly than we could before,” Freedman said.
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