Medical Research: What is the background for this study? What are the main findings?
Dr. Darling: Many people with tuberous sclerosis complex (TSC) have skin tumors that can bleed or cause distress. Only surgical approaches were useful for treating these skin tumors in the past. Recently, drugs called mTOR inhibitors, including sirolimus, were shown to shrink internal tumors in those affected by tuberous sclerosis complex. We wanted to document what happens to the skin tumors in those being treated with oral sirolimus. We found that most patients taking oral sirolimus showed improvement in their skin tumors, and that these effects were maintained during a couple years of treatment. We did not observe any evidence for the skin tumors becoming resistant to the drug.
Medical Research: What should clinicians and patients take away from your report?
Dr. Darling: This is encouraging news to patients, since those being treated for an internal tumor can expect to see some improvement in their skin as well. However, we would not recommend oral treatment for just skin tumors due to the possibility of adverse drug effects that occur in some people.
Medical Research: What recommendations do you have for future research as a result of this study?
Dr. Darling: Research is being done on the use of mTOR inhibitors applied topically to the skin rather than taking a pill. Patients show improvement in their skin without the side effects of oral treatment. This is a promising area for further research.
Another problem is that most tumors in those with TSC tend to regrow after stopping treatment. New drugs are needed that completely eradicate the tumors.
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Thomas N. Darling, MD, PhD (2015). Sirolimus Shrinks Some Tuberous Sclerosis Tumors