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Rutgers Health researchers have found that a weekly injection of a diabetes medication could replace the painful and expensive daily hormone shots currently used to treat a rare genetic disorder called congenital generalized lipodystrophy (CGL). The groundbreaking study, published in The New England Journal of Medicine, explores the potential of tirzepatide, a drug commonly used for diabetes and obesity, to improve conditions for people with CGL, a disease that causes severe metabolic problems and insulin resistance.
CGL, which affects only a few thousand people globally, leaves patients with almost no fat tissue, impairing their ability to store fat properly and causing it to accumulate in organs such as the liver. This results in extreme insulin resistance, diabetes, and a shortened life expectancy. The current standard treatment for CGL involves daily injections of metreleptin, a synthetic version of the leptin hormone produced by fat tissue. However, the daily injections are not only expensive but also painful, as patients lack subcutaneous fat for easy injection.
Dr. Christoph Buettner, chief of endocrinology at Rutgers Robert Wood Johnson Medical School and senior author of the study, explained, “These patients are severely ill and face markedly reduced life expectancy due to profound insulin resistance.”
In their recent study, the research team tested tirzepatide (brand names Zepbound and Mounjaro), which has been shown to improve insulin resistance. Administered as a weekly injection, tirzepatide could provide an alternative to daily, painful leptin shots and is significantly more affordable.
The initial results were promising. A 23-year-old patient who had refused daily leptin and insulin treatments for two years saw his blood glucose levels drop significantly after just three weeks on the maximum dose of tirzepatide. His blood glucose, which had previously been high at 252 mg/dL, fell to 128 mg/dL. In addition, 93% of his blood glucose readings were within the healthy range of 70 to 140 mg/dL, a notable improvement without the need for insulin injections.
A second patient, a 64-year-old woman who had been using both leptin and insulin to manage her glucose levels, found that tirzepatide alone was sufficient to bring her blood glucose into normal range, eliminating the need for supplemental insulin.
“The surprise here was that when we stopped leptin and gave tirzepatide, the patient was very well controlled, probably better than while she was taking leptin,” Dr. Buettner noted.
Both leptin and tirzepatide work in the brain, but through different pathways. While leptin is produced by fat tissue, tirzepatide mimics the hormone GLP-1, which is not produced in fat. This unexpected result suggests a possible overlap in the mechanisms of leptin and GLP-1 signaling.
While the results from this small study are encouraging, researchers acknowledge that larger trials are needed to confirm the findings. Due to the rarity of CGL, recruiting enough patients for a comprehensive study may be challenging.
If tirzepatide proves effective in larger studies, it could offer CGL patients a more accessible and less painful treatment option. However, further research is required to understand the long-term efficacy and safety of tirzepatide for this condition and other leptin-deficient disorders.
For more information, visit the New England Journal of Medicine here.
Disclaimer: This article summarizes findings from a recent study published in the New England Journal of Medicine. While initial results are promising, further research is needed to confirm the safety and efficacy of tirzepatide for CGL patients. Always consult a healthcare professional before making any treatment decisions.
