GlycoGenesys cancer trial shows promise

The investigation has been useful in establishing a maximum tolerated dose for GCS-100 and providing safety and pharmacokinetic data that supports the company’s blood-borne cancer programs. Furthermore, of the 14 patients for which response data is available, 11 experienced periods of stable disease.

The primary purpose of the multi-center clinical trial is to evaluate the safety and tolerability of escalating doses of GCS-100 in patients with advanced solid tumors to determine a maximum tolerated dose. Secondary objectives included evaluation of pharmacokinetics and response.

Patients participating in the study had previously received multiple treatment regimens, including chemotherapy, immunotherapy, radiation or hormone therapy. Of note, this is the first clinical trial using the low ethanol formulation of GCS-100. This formulation has eliminated ethanol-related side effects, allowed shorter infusion times and made it possible to deliver higher quantities of drug.

The trial achieved its primary objective of establishing a maximum tolerated dose for this dosing regimen of five consecutive days of therapy followed by two weeks off. Preliminary safety results available for 17 of the 22 enrolled patients demonstrate that GCS-100 is well-tolerated with low toxicity. The dose-limiting side-effect of GCS-100 when given intravenously in this regimen was a rash. The rash was manageable with steroid therapy and did not preclude additional dosing cycles. The majority of the remaining adverse events reported to date have been mild and reversible.

The pharmacokinetics of GCS-100 was linear throughout all doses tested. The data indicates that GCS-100 can reach the serum levels sufficient to kill several types of solid tumors in laboratory studies. Furthermore, the pharmacokinetic data suggests that more convenient dosing regimens may be possible in future solid tumor trials.