Drug shrinks brain tumors in children with tuberous sclerosis complex, study suggests

In continuation of my update on Everolimus

 "Every patient in this study experienced a decrease in size of their tumors, and no patient required surgery for their tumors after treatment with everolimus," says Dr. Franz, co-director of the TSC Clinic at Cincinnati Children's and the study's main author. "Thirty-five percent of patients in this study on everolimus had at least a 50 percent reduction in tumor volume after an average of 42 weeks on medication."

The phase III study was conducted among 117 patients with TSC who were randomly assigned to either everolimus or a placebo. Patients were 9 ½ years old on average but ranged from infants to adults. No patient on placebo showed improvement in their tumors. Tumor volume was measured by MRI assessment of the brain.

Dr. Franz conducted an earlier, phase II study of everolimus published in The New England Journal of Medicine in 2010. Based on that data, the U.S. Food and Drug Administration granted accelerated approval of everolimus for patients with these tumors, known as subependymal giant cell astrocytomas, or SEGAs. The new, placebo controlled study was conducted to confirm these earlier results.

Prior to FDA approval, surgery was considered standard therapy for SEGAs, but everolimus is a potential alternative to surgery and the first targeted medical therapy for TSC, says Dr. Franz.

"Children and teens may not only avoid surgery but they also may see improvement in other aspects of this disease, including a reduction or even elimination of hydrocephalus  a buildup of fluid inside the skull leading to increased intracranial pressure. Hydrocephalus is commonly associated with these tumors because they are located deep within the brain in spinal fluid pathways, or ventricles."

In Dr. Franz's 2010 study, patients reported their quality of life, as measured by a validated quality of life and neuropsychological assessments, improved at three months and six months after treatment with everolimus...

Ref : http://www.thelancet.com/journals/lancet/article/PIIS0140-6736(12)61134-9/fulltext

Drug shrinks brain tumors in children with tuberous sclerosis complex, study suggests

FDA approves Afinitor drug for tuberous sclerosis complex

We know that Everolimus (RAD-001), marketed by Novartis under the tradenames Zortress (USA) and Certican (Europe and other countries) in transplantation medicine and Afinitor in oncology is the 42-O-(2-hydroxyethyl) derivative of sirolimus and works similarly to sirolimus as an mTOR (mammalian target of rapamycin) inhibitor. It is currently used as an immunosuppressant to prevent rejection of organ transplants. Much research has also been conducted on everolimus and other mTOR inhibitors for use in a number of cancers. The FDA has approved everolimus for the treatment of advanced kidney cancer on March 30, 2009 and for organ rejection prophylaxis on April 22, 2010. Now the same drug has been approved for Tuberous sclerosis or tuberous sclerosis complex (TSC a rare, multi-system genetic disease that causes benign tumours to grow in the brain and on other vital organs such as the kidneys, heart, eyes, lungs, and skin ) ….more