Study in NEJM Shows Novartis Drug AfinitorÃ‚® Reduces Size of SEGAs, Benign Brain Tumors Associated with Tuberous Sclerosis
EAST HANOVER, N.J., Nov. 3, 2010 /PRNewswire/ -- The New England Journal of Medicine (NEJM) today published a study that found patients taking Afinitor® (everolimus) tablets experienced a decrease in the size of their subependymal giant cell astrocytoma (SEGA), a benign brain tumor associated with tuberous sclerosis (TS)(1,2). This study, which was previously presented at the 46th American Society of Clinical Oncology annual meeting, is the first prospective clinical trial of a drug to show treatment benefit in these patients.
Tuberous sclerosis is a genetic disorder that is estimated to affect 25,000 to 40,000 people in the US and may cause benign tumors to form in vital organs(3). SEGAs, benign brain tumors, occur in up to 20% of patients with TS and primarily affect children and adolescents(1,4,5). SEGAs may pose a significant medical risk, including the potential for swelling in the brain, or hydrocephalus(1).
According to data published in NEJM from this Phase I/II study of 28 patients conducted by Cincinnati Children's Hospital Medical Center, treatment with everolimus was associated with a significant reduction in primary SEGA volume at six months relative to baseline on independent central review (p<0.001). Seventy-five percent of patients (21 of 28) experienced a reduction of 30% or greater in the size of their largest SEGA and 32% (9 of 28) experienced a reduction of 50% or greater at six months relative to baseline(2).
The published study findings also showed that in nine of 16 patients, everolimus therapy was associated with a reduction from baseline to six months in overall frequency of seizures per 24 hour video electroencephalograms (EEG) (n=16; median change -1 seizure, p=0.022). Additionally, no patients required surgery or developed a new SEGA while receiving everolimus(2).
The most common adverse reactions observed (incidence greater than or equal to 30%) in this trial were mouth sores,