This technology utilizes tyrosine kinase inhibitors that block neural crest cell growth as a potential therapeutic treatment option for LAM.
Current treatment for LAM and TSC utilize a drug called rapamycin, which targets the mammalian target of rapamycin (mTOR). While treatment with rapamycin has been found to be associated with stabilized lung function, reduced chylous effusions, and regression of renal angiomyolipomas, it is limited in its mode of action. Rapamycin is a non-specific treatment and, as such, only blocks proliferation of LAM cells without actually killing them, restricting its therapeutic potential. Currently, the common recurrence of symptomatic LAM upon discontinuation of rapamycin treatment highlights the need for a more effective and sustained LAM and TSC therapy.
This technology proposes an improved method for treating LAM and TSC that utilizes tyrosine kinase inhibitors that block neural crest cell growth. Tyrosine kinase inhibitors have been found to pharmacologically inhibit the platelet-derived growth factor (PDGF) receptor signaling pathway, which is involved in the proliferation of LAM-associated stem cells and metastasis. These compounds can either be used alone or in combination with rapamycin as a co-adjuvants to rapamycin-induced mTOR inactivation, highlighting an increased therapeutic versatility. As such, this technology provides an improved and sustainable therapeutic strategy for patients with LAM and TSC.
This technology has been validated with tumor models in mice.
Jeanine D’Armiento, M.D., Ph.D.
Patent Pending
IR CU18003
Licensing Contact: Sara Gusik