Ilex Oncology Inc has launched the first combination therapy clinical trial involving the novel investigational anticancer agent clofarabine, a second-generation nucleoside analogue. The study is a Phase I/II trial of clofarabine in combination with the chemotherapy drug ara-C (cytarabine) in adults with a spectrum of hematologic malignancies.

"The activity we have seen with clofarabine as a single agent strongly warrants moving this novel drug into combination therapy with ara-C, the 'gold standard' for treating leukemias," stated Jeffrey Buchalter, ILEX President and CEO. "Since combination therapy has been clearly shown to be the most effective treatment for leukemias, this trial is the next logical phase of our clofarabine development program."

This combination study is part of a clofarabine co-development program being conducted by ILEX and its co-development partner Bioenvision Inc. The study seeks to determine the optimal dose of clofarabine when administered in combination with ara-C, the safety and tolerability of the combination of drugs, and their pharmacokinetic profile. Researchers will also be collecting important information about the activity of the combination of clofarabine and ara-C in study patients.

In July 2002, ILEX initiated a Phase I clinical trial to evaluate clofarabine in adults with advanced solid tumors. In addition, Phase II studies of clofarabine are underway in relapsed or refractory pediatric acute lymphocytic leukemia (ALL), pediatric acute myelogenous leukemia (AML) and in adult AML. Recently completed was a single-center investigator-sponsored Phase II study of clofarabine in acute leukemia and MDS.

Clofarabine is a novel investigational therapeutic designed as a second-generation nucleoside analogue to overcome the limitations of current analogues. Nucleoside analogues are antimetabolites that affect DNA synthesis and are used in patients who have failed first-line chemotherapy with alkylating agents. Researchers believe Clofarabine works by damaging DNA, disrupting the function of mitochondria and activating the caspase pathway, all of which induce cell death. Ilex obtained the right to co-develop clofarabine from Bioenvision Inc.