Chronic Myeloid Leukemia

December 10, 2012

ARIAD Pharmaceuticals released results from a phase I trial of ponatinib for the treatment of resistant and refractory chronic myeloid leukemia (CML) and Philadelphia chromosome-positive acute lymphoblastic leukemia (Ph+ ALL). This dose-escalation study enrolled 81 patients with resistant hematologic cancers, including 60 patients with CML and five patients with Ph+ ALL, who had become resistant to one or more tyrosine kinase inhibitors. Results showed that after 73 weeks, 72% of patients (31 of 43) with chronic-phase CML had a major cytogenetic response (MCyR), including 92% (11 of 12) who had the T315I gatekeeper mutation, the most common mutation among resistant patients. In addition, of 22 patients with accelerated-phase or blast-phase CML or Ph+ ALL, 36% (8 of 22) had a major hematologic response and 32% (7 of 22) had aMCyR. The drug was well tolerated. The most frequent adverse events were rash, thrombocytopenia, arthralgia, increased lipase, fatigue, acneiform dermatitis dry skin and nausea. ARIAD did not disclose its plans for ponatinib.

July 28, 2008

Antisoma issued positive interim results from a phase II trial of AS1411 for the treatment of Acute Myeloid Leukemia (AML). This trial planned to enroll 70 subjects with relapsed or refractory AML, in the United States. The trial was designed to evaluate the addition of low dose and high dose AS1411 to cytarabine (current standard of care) to cytarabine alone. Reported data is from 33 subjects who were enrolled in the low dose stage of the trial. These subjects were randomized to receive either 10 mg/kg/day AS1411 plus cytarabine or cytarabine alone. Treatment was well tolerated at this dose. Among 11 subjects who received AS1411 plus cytarabine, one had a complete response (CR) and one had a complete response with incomplete recovery of platelet counts (CRP); a third subject had a cytogenetic response but had leukemic blasts remaining. Among five subjects who received cytarabine alone, none had a CR, none had a CRP and there were no cytogenetic responses. The subjects who did not respond to cytarabine alone were allowed to cross over to receive AS1411 plus cytarabine. Two of the first five subjects crossed over, one of whom showed a 90% reduction in leukemic blast count after treatment with the combination. Enrollment is currently underway in the high dose stage of the trial, comparing 40 mg/kg/day AS1411 plus cytarabine with cytarabine alone.

January 14, 2002

Phase II trial results indicate that rubitecan, an oral anticancer drug, is active in both chronic myelomonocytic leukemia (CMML) and myelodysplastic syndromes (MDS). The trial included 35 evaluable subjects who had received a minimum of one prior therapy and who were diagnosed with either CMML (23 subjects) or MDS (12 subjects). Results showed an objective response rate of 28%, with four complete responses and six partial responses. An overall response rate of 42% was reported, taking into account hematological improvement in 14% of subjects. Rubitecan is being developed by SuperGen.

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