Experimental gene therapy functionally cures SS in mice

It’s been a long time since my last post, due to a combination of holiday travel and issues with the software that I use to manage this site. Luckily things appear to be back on track, and I’m ready to address a backlog of potential posts.

This promising study was published in 2016, but I only noticed it recently. This is a very early study with mice, so there are many hurdles and uncertainties to overcome before this therapy could potentially be available. Still, it is quite promising. From the abstract:

Primary Sjögren’s syndrome (pSS) is a chronic autoimmune disease that is estimated to affect 35 million people worldwide. Currently, no effective treatments exist for Sjögren’s syndrome, and there is a limited understanding of the physiological mechanisms associated with xerostomia and hyposalivation. The present work revealed that aquaporin 5 expression, a water channel critical for salivary gland fluid secretion, is regulated by bone morphogenetic protein 6. Increased expression of this cytokine is strongly associated with the most common symptom of primary Sjögren’s syndrome, the loss of salivary gland function. This finding led us to develop a therapy in the treatment of Sjögren’s syndrome by increasing the water permeability of the gland to restore saliva flow. Our study demonstrates that the targeted increase of gland permeability not only resulted in the restoration of secretory gland function but also resolved the hallmark salivary gland inflammation and systemic inflammation associated with disease. Secretory function also increased in the lacrimal gland, suggesting this local therapy could treat the systemic symptoms associated with primary Sjögren’s syndrome.

What is most promising to me is that the treatment, which was meant to address the sicca symptoms of SS, also resolved systemic inflammation. This demonstrates that the two are certainly linked, and that while systematic treatments like biologic drugs often fail to address all symptoms (especially sicca), future treatments may provide full relief. I will admit that I am not an expert in gene therapy, and much of the study was too advanced for me, but injecting specific proteins into salivary glands seems like a reasonable vector for a human treatment. Of course, this is very early research, and even in the best case a potential treatment is likely a decade away. Still, it is always nice to see potential treatments come out of the gate with full symptom resolution, even if only in animal studies.


Posted in Research, Treatment
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