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,
‘/>”/>
