The first person in the world to receive a dose of a novel cell therapy for a symptom of the immune disorder Sjögren’s disease has been treated during a clinical trial conducted by researchers at the University of Wisconsin School of Medicine and Public Health.
Bodily functions most take for granted, like the ability to cry or swallow, are a daily struggle for Julianne McGowan, the first research participant and patient in the trial. She suffers from xerostomia, or severe dry mouth, caused by Sjögren’s disease, a disorder that affects moisture-producing glands in the eyes, mouth and other parts of the body. The disease often accompanies other conditions like rheumatoid arthritis and lupus but can occur alone.
“Early on, before I knew what it was, I’d drink an entire glass of water and still feel thirsty, thinking there was no amount of water I could drink to prevent being thirsty,” McGowan said.
In 2014, she started noticing other symptoms like joint pain, dry eye and rapid weight loss, and learned her condition could be Sjögren’s disease. In 2017, her diagnosis was confirmed by a medical professional.
For people living with dry mouth due to Sjögren’s disease, the daily routine involves keeping special lozenges on hand that help promote saliva production as well as the constant companionship of a water bottle.
Severe dry mouth limits the ability to eat and speak and is a major cause of tooth decay, resulting in a dramatically reduced quality of life for many patients, according to Dr. Sara McCoy, assistant professor of medicine, UW School of Medicine and Public Health, and a rheumatologist at UW Health. McCoy is an expert in the care of Sjögren’s disease patients and runs one of the few dedicated Sjögren’s disease clinics in the Midwest. She also conducts clinical research to help improve treatment options for those who are facing the disease.
McCoy noted that McGowan’s dry mouth had worsened due to scarring of her salivary glands. There are no Food and Drug Administration-approved treatments for Sjögren’s disease, although multiple drugs are in Phase 2 or Phase 4 studies. Those drug candidates focus on active inflammation. None currently focus on the chronic damage that has already occurred in the salivary glands.
“For people like Julianne, there’s nothing to help them, and I felt there has to be something we can do,” McCoy said.
In 2022, McCoy partnered with the UW Program for Advanced Cell Therapy, or PACT, a joint venture between the UW School of Medicine and Public Health and UW Health. The program develops novel immunotherapy treatments using cells, like white blood cells or types of stem cells, from a patient or a sibling, activating them in the PACT cell manufacturing lab and then injecting them into the patient.
With this cell therapy, we are hopeful we can do more than offer temporary relief and give these people back these critical human functions.
- Sara McCoy, MD
McCoy wondered if the use of a patient’s own enhanced stem cells could be used to improve salivary gland function in Sjögren’s disease patients and began working with PACT to start a clinical trial. She received FDA approval to initiate the trial in 2023. The approach extracts cells from the patient’s own bone marrow, activates them and injects them into salivary glands.
“The current standard of care is not addressing the root cause of dry mouth for these patients,” she said. “With this cell therapy, we are hopeful we can do more than offer temporary relief and give these people back these critical human functions.”
This Phase 0 trial is enrolling six patients and will be followed by a larger Phase 1 trial to establish safety and dose levels. Preliminary data on efficacy will be obtained in a cohort of 12 participants in an expansion phase of the study. The trial is open to people 18 and older with graft-versus-host disease, which also impacts saliva production, or Sjögren’s disease. Each participant will be given the therapy once, and the results will be tracked by McCoy’s research team for about two years.
While the goal of the first phase of the trial is to show that the therapy is safe, McGowan said she has noticed some improvement.
“I had been taking several lozenges a day to help with my symptoms, but after the treatment I used them once or twice a week,” McGowan said.
Sjögren’s disease impacts women more than men, about eight to one, according to McCoy, but there seems to be a lack of awareness of this condition, McGowan said.
For this reason, she has found participation in the trial to be a meaningful opportunity to help spread the word about the difficulties Sjögren’s patients experience, she said.
“I want to increase awareness of the disease and what the symptoms are so others don’t suffer,” McGowan said.
Read more about what sparks Dr. McCoy’s drive to innovate at UW–Madison.
Banner image by Clint Thayer, courtesy of the UW Department of Medicine
This work has been funded by an Institute for Clinical and Translational Research pilot grant (ATRS) and a UW Department of Medicine pilot grant.
