Two studies of the mTOR inhibitor everolimus in Peutz-Jeghers syndrome

Two studies of the mTOR inhibitor everolimus in Peutz-Jeghers syndrome

A patient-view report by Stephanie Sugars

My 2009 report on mTOR inhibitors in PJS

Drug directions in PJS – RAD001 (aka everolimus and Afinitor), an mTOR inhibitor
A patient-view report by Stephanie Sugars 
January 17, 2009  

Part one of three: These are the thoughts of a PJS patient, not a medical professional. I'm thinking like a patient advocate and wondering what we might want to consider about using these drugs and participating in clinical trials for them. 

I don't mean to discourage anyone who'd like to participate in a clinical trial, it's a sacrifice that some will have to make to determine whether new drugs are both safe and effective for other patients. 

I do want us to consider possible risks and benefits and not get carried away in the excitement of a new era of chemoprevention in PJS.  

I've been following mTOR, rapamycin and RAD001 (everolimus) in PJS and our gene, LKB1 for many years. I heard about the Salt Lake City trial (http://www.clinicaltrials.gov/ct2/show/NCT00811590) in October 2007 at the Collaborative Group of Americas on Inherited Colorectal Cancer conference.