SUMMARY:
UCLA researchers in the Department of Molecular and Medical Pharmacology have identified a novel and safe therapeutic to ameliorate symptoms for the autoimmune disorder, Sjögren’s Syndrome.
BACKGROUND:
Sjögren’s syndrome (SS) is an autoimmune disorder that affects over 4 million people in the United States. Most commonly, SS causes chronic dry eyes and dry mouth. Some SS patients also suffer from a range of other symptoms, such as skin conditions and lymphoma. Research has evidenced that the underlying cause of SS is the invasion of immune cells to salivary and tear glands that results in progressive tissue damage. Despite this progress in research, there are currently no clinically approved therapies to slow the progression of SS. Treatment for SS is limited to therapies that provide relief, rather than cure, symptoms, such as with eye drops and mouthwash. Even so, prescribed clinical-grade eye drops have been reported to be beneficial for only a limited number of SS patients. While research efforts for developing treatments with therapeutic effects are ongoing, none have yet reached clinical testing. Thus, there is an urgent need to identify more effective palliative treatments, especially those with established safety data, for patients living with SS.
INNOVATION:
Researchers at UCLA led by Dr. Daniel Kaufman in the Department of Molecular and Medical Pharmacology have developed a novel strategy for treatment of SS by repurposing of a drug with globally established safety data. In the US, China, and much of Europe, GABA is regarded as safe for consumption. GABA has been shown to inhibit the autoreactive response of immune cells, stimulate anti-inflammatory activities, and, more recently, ameliorate a bevy of chronic autoimmune diseases in murine models. To test efficacy of GABA for treatment of SS, the researchers used two spontaneous murine models of SS. They found that, after a few weeks of treatment beginning after onset of SS symptoms, the mice showed pronounced restoration or preservation of saliva and tear production with no adverse effects. This strong evidence indicates that GABA is a promising intervention for SS.
POTENTIAL APPLICATIONS:
• Novel treatment for Sjögren’s Syndrome to aid in saliva and tear production
• Prospective therapeutic strategy for other autoimmune disorders
ADVANTAGES:
• Increased efficacy compared to other immunosuppresants because GABA has broad range anti-inflammatory actions so it can target multiple immune cell populations
• Safe for human consumption
• Preserves exocrine function
DEVELOPMENT-TO-DATE:
The UCLA researchers have successfully demonstrated the efficacy of GABA for ameliorating dry mouth and dry eyes symptoms of Sjögren’s Syndrome by oral administration in murine models.
Related Papers (from the inventors only):
Song, M., Tian, J., Middleton, B., Nguyen, C. Q., & Kaufman, D. L. (2022). GABA Administration Ameliorates Sjogren's Syndrome in Two Different Mouse Models. Biomedicines, 10(1), 129.