Figure 6.

Combination treatment with a mTOR (mammalian target of rapamycin) inhibitor and a calcium channel blocker. A, Everolimus restored differentiation of SM22α (smooth muscle 22α) positive cells to the control cell level. Combination treatment with everolimus and verapamil (purple) was not superior to treatment with everolimus alone (orange) in any patient. Although there is a strong trend towards significance, we note that verapamil treatment (green) was less effective on Williams syndrome (WS)2 (P=0.068) and WS3 (P=0.059) than in Figure 5D. Verapamil was also more effective on ELN (elastin)-1 (P=0.037). This has no effect on the conclusion that everolimus alone rescued all patients. B, Everolimus rescued proliferation in all 4 patients exhibiting the hyperproliferation phenotype. Combination treatment was not significantly different to treatment with everolimus. C, Everolimus rescued endothelin-induced calcium flux in 4 patients but not to control levels, and WS3 did not respond. Combination treatment was not significantly different to treatment with everolimus. n=3 independent experiments, using 3 technical replicates for each experiment. A–C, Supporting data are provided in Table IIIC in the Data Supplement. *P<0.05, drug treatment vs DMSO.