Participating in Clinical Trials:
Like Father, Like Daughter

“Once the shock of the initial diagnosis wore off, I became interested in the science of it all,” said Brooklyn resident and mother of two, Rachel Johnston, 41, a participant in a unique, JDRF-funded clinical trial of a drug called TUDCA—for people newly diagnosed with type 1 diabetes (T1D). Her father, Jeffrey Johnston, who died almost a year to the date of Rachel’s diagnosis, was also in a clinical trial but for late-stage lung cancer. “Even toward the end, when he was sick and unable to leave his bed, he was so captivated by the science. He believed in science, and so do I.”

While finding recruits for clinical trials is often difficult, especially for TUDCA, which is looking only for adult patients (ages 18-45) with new-onset (within the first 100 days) T1D—when Rachel, a patient at the Naomi Berrie Diabetes Center, heard about their pilot clinical study, she was on board almost immediately. For one thing, she stands to benefit from the trial of TUDCA, which could enhance beta cell function in the newly diagnosed: Said Rachel, “I’m in the honeymoon period of my disease which is the spot you want to be in the longest if you have type 1 diabetes. The hope of TUDCA is to prolong—in me as well as others—this period of time.”

Rachel, who runs her own design firm, is also participating in TUDCA to honor the memory of her father, an architect.  She recalled the time when they were watching the Matt Damon movie, The Martian, from his hospital bed. Said Rachel, “There’s a line in the movie where Matt Damon says, ‘I’m going to science the (heck) out of this, and my father looked at me and said, ‘That’s it! I’m going to science the (heck) out of my diagnosis.’”

Actually, Matt Damon used a four-letter expletive instead of the word heck—and the real line in the movie became Rachel’s rallying call and one she repeats over and over again about her own diagnosis and her participation in TUDCA. “It helps to have my father chiming in the background saying ‘just science the (heck) out of it’ when I’m taking my 7 TUDCA pills a day,” she said.

Like his daughter, Jeffrey Johnston would also be captivated by all the science surrounding diabetes. “There is a lot of reason for hope and optimism in the field of T1D research,” said Berrie Center Co-Director Robin Goland, MD, J. Merrill Eastman Professor of Clinical Diabetes. “The knowledge is exploding. There’s a lot of great science that is being applied that is moving us closer toward prevention and even a cure of T1D.”

Until recently, much of the type 1 diabetes research has focused on suppressing the autoimmune system to prevent or reverse T1D. Now, scientists are learning that a stress response in the insulin-producing beta cell itself may play a significant role in the development of the disease. The oral drug TUDCA (formally identified as tauroursodeoxycholic acid) approved for use in Europe and used in the treatment of liver disease, can alleviate that stress in the beta cell.  When TUDCA was given to mice in the early stages of T1D, a reduction of diabetes was observed.

Dr. Goland 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 co-investigators.

The Berrie Center at Columbia University Medical Center is the only diabetes center in the country offering the TUDCA trial. For information about participating, please contact Sarah Pollak at 212-851-5425 or sjp2174@cumc.columbia.edu.

To see an interview with Dr. Goland on TUDCA, click here: https://vimeo.com/118597801