Cincinnati Children's Blog

New Tuberous Sclerosis Study: Drug Can Shrink Tumors and Save Kidneys

New Tuberous Sclerosis Study: Drug Can Shrink Tumors and Save Kidneys

“I’m active.  I feel better.  I’m happy to be alive.”  These are words from Misty Barnhart, a patient of the Tuberous Sclerosis Clinic at Cincinnati Children’s.

Before coming to Cincinnati Children’s, Misty had one of her kidneys removed after a procedure to resect masses within the kidney didn’t leave much functioning organ. Later, she was told she would need the same surgery to remove tumors from her one remaining kidney. “At that point, I was just hysterical,” she said. Before she said yes to a surgery that could leave her without any kidney function, Misty researched other options. In that research, she found Dr. John Bissler and Cincinnati Children’s.

Misty is one of about a million people worldwide living with tuberous sclerosis complex (TSC). It’s a genetic disease that causes tumors to grow on vital organs like the heart, lungs, kidneys and brain. The tumors are non-malignant, but can interfere with normal organ function.

“I can’t tell you how many times I’ve heard from patients who say their doctors told them a kidney looks bad, is full of tumors, isn’t working and has to come out,” says Dr. Bissler, who co-directs the Tuberous Sclerosis Clinic. “But you can do studies on these patients and find out that they have normal kidney function. The kidney looks bad, but it works.”

When Misty came to Cincinnati Children’s, she was enrolled in a study led by Dr. Bissler involving a drug that is now shown to shrink and successfully limit growth of tumors related to TSC. We think you’ll enjoy meeting Misty in the video below and hearing how her life has transformed.  “I call it my miracle,” says Misty.

Dr. Bissler’s study was just published in The Lancet and a news release detailing the findings is available in our Newsroom.

The Tuberous Sclerosis Clinic at Cincinnati Children’s is the world’s largest such center dedicated to identifying and treating this disorder.

Read more about the study in U.S. News & World Report.

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