A therapy currently being tested in a clinical trial at the University of Wisconsin School of Medicine and Public Health to treat a common symptom of Sjögren’s disease recently got a boost from the Food and Drug Administration.
The novel experimental cell therapy, designed to treat xerostomia, the medical term for dry mouth, caused by the condition, was granted Fast Track designation by the federal agency. The therapy, which is currently being investigated in a Phase 0 clinical trial, was developed by the UW Program for Advanced Cell Therapy, a partnership between UW Health and the University of Wisconsin School of Medicine and Public Health. Researchers hope that clinical trials will show the safety and efficacy of the treatment, as it is the only therapy of its kind in the United States for this condition.
Fast Track designation speeds up the review process for drugs and devices — which can take 10 years or more — as they go through the FDA approval process. It is selectively granted to investigational therapies that treat serious conditions and fill unmet medical needs.
This is the second Fast Track designation granted to the program in the last two years. The designation is more commonly given to companies that make drugs or medical devices, making it somewhat rare that an academic institution received two such designations in such a short amount of time, according to Dr. Jacques Galipeau, associate dean for therapeutics development, and professor of medicine, UW School of Medicine and Public Health. Galipeau directs the UW Program for Advanced Cell Therapy.
“This designation shows the potential the FDA sees in cell therapies, and that the agency recognized our program’s ability to create and test these therapies safely and effectively,” he said.
Measures to accelerate approval can include more frequent meetings with the agency, a rolling review process and priority review at the time of application for a biologics license, which would allow the therapy to be marketed like any other approved drug or device.
Xerostomia is one of the most critical long-term symptoms of Sjögren’s, and it can result in problems eating, speaking and sleeping, in addition to causing pain, fatigue and contributing to tooth decay.
This new therapy offers an innovative regenerative medicine approach that leverages the immune system-stimulating and tissue-restorative potential of interferon-γ-primed autologous mesenchymal stromal cells, according to Dr. Sara McCoy, assistant professor of medicine at the UW School of Medicine and Public Health. McCoy practices at UW Health as a clinical rheumatologist.
“The FDA’s Fast Track designation demonstrates the urgent need for new therapies to help these patients who have no real treatment to relieve their dry mouth,” said McCoy, who is principal investigator of the clinical trial. “Currently, patients must rely on treatments such as special lozenges and living with the constant companionship of a water bottle; we must do better for them.”
The FDA issued an investigational new drug license, or IND, in 2023 to launch the clinical trial. The Phase 0 trial will enroll six patients to show the cell therapy is safe and promising for effectiveness. It will eventually expand to a Phase 1 trial and enroll 30 people.
The treatment uses a patient’s own interferon-gamma-activated marrow stromal cells, which are then enhanced in the program’s cell processing facility at University Hospital and injected into the patient’s salivary glands to spur saliva production.
The Phase 0 clinical trial is supported by funding from the Department of Medicine and the Institute for Clinical and Translational Research at the UW School of Medicine and Public Health. The Phase 0 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.
