Iowa City Non-Profit Uses Adult Stem Cells to Search for Cures, Treatments of Rare Diseases
Jay and Anant Kamath at the John Paul II Medical Research Institute based in Iowa City are using adult stem cells as a weapon to fight rare genetic diseases. They keep thousands of stem cell samples stored at 200 degrees below zero at their lab in Coralville.
"We can harvest those from tissue biopsies when children go in for various routine surgeries," said CEO Jay Kamath. "We can also harvest those from as little as 7 milliliters of blood."
They can use the stem cells to recreate any type of cell in the human body.
"We can take your own cells and reprogram them within a month, and then create new tissue types," said Anant Kamath, research scientist.
Jay Kamath said The John Paul II Medical Research institute started in 2006 with the goal of finding cures for cancer, and neurological and chronic diseases. Now, the Kamaths are turning their attention to rare genetic diseases, like Niemann-Pick Type C, a fatal disorder that slowly degrades brain function.
Two of Bryan Hadley's kids, Peyton and Kayla, were diagnosed with it in 2007. He described the symptoms over Skype from his home in Medford, Oregon.
"Slow speech, they have ataxia, balance problems, movement," Hadley explained.
In his search for a treatment, Hadley found Kamath's team, which isolated stem cells from his children to be sent off for testing. Hadley says an already-discovered drug called Cyclodextrin was found to be effective in treating their condition, and the results were almost immediate.
"They regained their speech back, their balance was better, swallowing difficulties got a little better," Hadley recalled. It's not a cure, but Hadley said it's buying his family time until researchers can find one.
"95 percent of rare diseases currently have no therapies available to them," Jay Kamath told us. "And roughly 70 percent of those children will die before the age of 5."
Anant said they want to find as many cures to as many diseases as they can, but they need blood and tissue samples from patients to do that. Eventually, he hopes to be able to cure fatal diseases like end-stage cardiac failure and Alzheimer's.
"Our goal is to create a toolkit for patients, so they can be treated for things that are now terminal," Anant told us.
If you are a patient interested in helping the institute by donating a blood or tissue sample, you can sign up for their