The new study may open up new treatment possibilities for LAM and other interstitial lung diseases.
Deleting the TSC2 gene in specific lung cells of mice led to the activation of the mTORC1 signaling pathway and pulmonary disease characteristics consistent with human LAM disease, particularly in female breeder mice. These mice without TSC2 also exhibited a dysfunctional WNT cellular signaling pathway, which is also tied to lung development and is activated in multiple common pulmonary diseases, according to researchers in the Perelman School of Medicine at the University of Pennsylvania. This new research suggests targeting mTORC1 and WNT pathways may be a way to treat LAM. The findings are published in the latest issue of Nature Communications.
LAM, a rare pulmonary disease that predominantly affects women of child-bearing age and is exacerbated by pregnancies, is a cancer-like disease that leads to cysts developing in the lungs, respiratory failure, and increasingly severe and potentially fatal complications. Sirolimus (rapamycin) is the first, and only, treatment approved by the FDA for the treatment of LAM. However, this drug is not effective or well tolerated by some LAM patients, and declining lung function has been shown to continue after treatment. Therefore, there is an urgent unmet clinical need for new LAM therapies.
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Nature Communications. Research paper:
