National Laboratory of Translational Neuroscience (TINL)(RRF-2.3.1-21-2022-00011)
(Recovery and Resilience Facility of the European Union within the framework of Programme
Széchenyi Plan Plus grant number RRF-2.3.1-21-2022-00011)
(RRF-2.3.1-21-2022-00011)
National Institute of Diabetes and Digestive and Kidney Diseases(DK58538)
L-thyroxine (L-T4) monotherapy is the standard treatment for hypothyroidism, administered
daily to normalize TSH levels. Once absorbed, T4 is converted to T3 to alleviate most
symptoms. However, this treatment abnormally elevates plasma T4 levels in over 50%
of patients. Using L-T4-treated Thyroid Hormone (TH) Action Indicator mice, which
express a T3-regulated luciferase (Luc) reporter, we examined whether these T4 elevations
disrupt TH signaling. Hypothyroid mice exhibited reduced Luc expression across brain
regions, and L-T4 treatment failed to restore T3 signaling uniformly. There was also
variability in the activity of type 2 deiodinase (D2), the enzyme that generates most
brain T3. Intracerebroventricular T4 administration achieved higher elevation of Luc
expression in the mediobasal hypothalamus compared to the cortex, and studies on cultured
cortical astrocytes and hypothalamic tanycytes revealed cell-type-specific responses
to T4. In tanycytes, exposure to T4 sustained D2 activity, leading to progressive
T3 signaling, whereas in astrocytes, T4 exposure triggered a drop in D2 activity,
limiting T3 production through a ubiquitin-dependent, self-limiting mechanism. The
sustained D2 activity in tanycytes was linked to rapid deubiquitination by USP33,
as confirmed using a ubiquitin-specific protease (USP) pan-inhibitor and USP33 knockout
mice. In conclusion, the brain’s response to L-T4 treatment is heterogeneous, influenced
by cell-specific regulation of D2-mediated T3 production. While cortical astrocytes
exhibit limited T3 signaling due to D2 ubiquitination, tanycytes coexpressing USP33
amplify T3 signaling by rescuing ubiquitinated D2 from proteasomal degradation. These
findings provide mechanistic insights into the limitations of L-T4 therapy and highlight
the need for tailored approaches to managing hypothyroidism.