LAM Pilot Study with Imatinib Mesylate (LAMP 1)
Technical Report,30 Sep 2015,29 Sep 2016
Medical University of South Carolina Charleston United States
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Lymphangioleiomyomatosis LAM is a rare disease in which tumor cells LAM cells proliferate and destroy healthy lung tissue, leading to respiratory compromise or failure. Vascular endothelial growth factor-D VEGF-D is generated by LAM cells and is a robust biomarker for LAM disease activity and therapeutic response. Studies in the laboratory of Dr. DArmiento suggest that imatinib mesylate imatinib could completely block the growth of LAM cells through initiation of targeted cell death. Currently, most LAM patients are treated with Sirolimus rapamycin. Rapamycin growth inhibits but does not kill LAM cells. This pilot trial employs a dual agent design intended to generate safety and efficacy data sufficient to power and design a phase 3 study of imatinib vs placebo for LAM. The hypothesis is that imatinib will be equivalent to rapamycin in short term efficacy and safety. Importantly, VEGF-D level will be used as a marker for LAM disease activity in this small clinical trial design using 20 participants at two institutions. At the time of this abstract participants are not yet enrolled. IRB approvals are in place and HRPO submission is ready.
- Medicine and Medical Research