Rutgers Health researchers have discovered that a weekly injection of diabetes medication may replace the daily hormone injections for those suffering from congenital generalized lipodystrophy (CGL). This rare genetic disorder, which affects only a few thousand individuals globally, leads to severe metabolic disease, diabetes, insulin resistance, and reduced life expectancy. Due to the absence of fat tissue for proper storage, fat accumulates in organs like the liver.
“These patients are severely ill and face markedly reduced life expectancy due to profound insulin resistance,” said Christoph Buettner, chief of endocrinology at Rutgers Robert Wood Johnson Medical School and senior author of the study.
The current standard treatment involves daily injections of metreleptin. However, these shots are costly and painful for CGL patients. “When you inject yourself with, for example, insulin, you inject into subcutaneous fat, but these patients don’t have that,” explained Svetlana Ten, associate professor of pediatrics and first author of the study.
Researchers tested tirzepatide as an alternative treatment. It is administered through a weekly injection and is more affordable than metreleptin. In one case study, a 23-year-old man who had refused daily treatments saw significant improvements in blood glucose levels after three weeks on tirzepatide. Another patient, a 64-year-old woman requiring supplemental insulin injections alongside leptin therapy, achieved normal blood glucose levels using tirzepatide alone.
“The surprise here was that when we stopped leptin and gave tirzepatide, the patient was very well controlled,” Buettner noted.
Both tirzepatide and leptin work in the brain but through different pathways. This indicates potential overlaps between leptin and GLP1 signaling.
A larger trial is planned to confirm these findings despite recruitment challenges due to CGL’s rarity. If successful in broader studies, tirzepatide could offer CGL patients an easier and potentially more affordable treatment option. Nonetheless, further research is necessary to assess long-term efficacy and safety in this population.
