3/15/2018
Participating in a Unique Clinical Trial

“I was struggling to figure out my treatment plan,” said Johanna Ohm, who was diagnosed with type 1 diabetes (T1D) last year—and she wasn’t sure if the people she was seeing (mainly a Physician Assistant on a college campus where she is an entomologist) were helping. There was a three-month wait for Johanna to see a local endocrinologist.

So Johanna, a graduate student in infectious disease biology, spent the first 100 days of her diabetes in a state of confusion. But luckily, during that time, she went to www.clinicaltrials.gov where she found the Naomi Berrie Diabetes Center and a clinical trial for a JDRF-funded study of a drug called TUDCA, (formally identified as tauroursodeoxycholic acid) that could preserve beta cell function in adults who were recently diagnosed with T1D.

“I’m a scientist by training, so I thought it would be great to participate as a patient in science at an academic institution,” said Johanna, a recipient of a Bill and Melinda Gates Foundation award to work on controlling the spread of mosquito-borne illnesses.

Once Johanna found out she met the eligibility criteria for the TUDCA trial (the trial is enrolling patients between the ages of 18 and 45 in the first 100 days of T1D) she drove approximately 250 miles from University Park, PA to the Berrie Center to participate in the clinical trial. She took that same road trip back and forth for a year, the duration of the trial.

She also moved her care to the Berrie Center where doctors discovered that she was taking ten times more insulin than she needed. “It had been a hard situation and the Berrie Centre has been a godsend to me.”

The TUDCA trial is somewhat unique since it focuses on the insulin-producing beta cells directly. Until recently, much of T1D research has focused on suppressing the autoimmune system to prevent or reverse T1D. Now scientists are learning that a stress response in beta cells also plays a significant role in the development of the disease.

Specifically, stress on the endoplasmic reticulum, (ER), the structure within the beta cells that folds and packages insulin from its precursor form, can lead to the dysfunction and ultimate loss of beta cells. It was recently discovered that the oral drug TUDCA approved for use in Europe, and used in the treatment of liver disease, can alleviate ER stress.  When TUDCA was given to mice in the early stages of T1D, an improvement in insulin production was observed.

Since TUDCA is a double-blind study, Johanna is curious to find out whether she received the real drug or a placebo.  “I feel great, and my management is completely under control,” said Johanna who is also a long-distance runner.

“I hope it’s an effect of the TUDCA.”

Berrie Center Co-Director, Robin Goland, MD, the J. Merrill Eastman Professor of Clinical Diabetes, is the principal investigator on the clinical trial of TUDCA. Rudolph Leibel, MD, Christopher J. Murphy Professor of Diabetes Research and Co-Director of the Berrie Center and Dieter Egli, PhD, Maimonides Professor of Diabetes Research, are study PIs.