Trials include those conducted in children with solid tumors and in adults with advanced hormone-refractory prostate cancer, advanced gastrointestinal (GI) cancer and small cell lung cancer (SCLC).
The pediatric trial was the first study that has tested Genasense in children. In the small scale study of 28 participants with various cancers, escalating doses of Genasense and standard chemotherapy were administered to children who had received extensive prior therapy. The authors concluded that the addition of Genasense to this chemotherapy program was well-tolerated in children at doses that were comparable to adults.
A phase II trial of Genasense plus docetaxel in patients with hormone-refractory prostate cancer achieved a major reduction in prostate specific antigen (PSA), which is a surrogate marker of prostate cancer activity. Median survival for all patients irrespective of response was 20 months.
Three phase I studies into gastrointestinal (GI) cancers have provided mixed results. A dose-response study evaluating optimum dosing of Genasense using a standard drug regimen encountered problems of prolonged neutropenia that resulted in dose delays on day 14 that were dose-limiting using full doses of Genasense. Only a handful of participants achieved a response to the treatment. The further two studies into GI cancers are either still ongoing or have not been fully reported. However, preliminary findings indicate good tolerability to the treatment.
A further study into small cell lung cancer is still underway as the primary end-point of the study was to evaluate landmark survival at 1 year. At this time, although survival data are still immature, no benefit in survival is yet apparent. Enrolment completed in October 2004 with the final results on landmark survival due to be provided with 12-months of follow-up.