According to results presented at the 2003 annual meeting of the American Society of Clinical Oncology, the oral agent PKC 412 appears promising for the treatment of acute myeloid leukemia.

Acute myeloid leukemia (AML) is a cancer of the bone marrow and blood characterized by the uncontrolled growth of immature white blood cells (immune cells), which never develop into functioning cells. Besides not being able to carry out the functions of mature immune cells, AML cells may also crowd out normal blood cells in the bone marrow and blood. AML is considered to be an aggressive cancer and patients are often at a high risk of developing a cancer recurrence following therapy, particularly if they are not able to undergo high doses of therapy. Chromosomal variables of AML cells, as well as levels of cancer cells in the blood, further distinguish patients into being at a high-risk, standard-risk or low-risk of developing a cancer recurrence, and treatment may be altered according to these stratifications. Standard therapy for AML includes chemotherapy, typically utilizing high doses. Since some patients may not be eligible for intensive treatment with chemotherapy due to the associated side effects, researchers are evaluating novel therapeutic approaches for the treatment of this disease.

Approximately 30% of patients with AML have a genetic mutation within the FLT3 gene. A mutation within the FLT3 gene results in continuous signals to the cancer cells, causing them to grow in an uncontrolled manner, and preventing them from natural death. These patients have a poorer survival than other patients with AML treated with standard therapeutic approaches. PKC 412 is an agent that blocks the growth signal caused by the FLT3 mutation, resulting in cellular death.

A recent, small clinical trial evaluating PKC 412 was conducted by researchers from the Dana-Farber Cancer Institute. This trial included 14 patients with the FLT3 mutation who had advanced AML. Patients were treated with PKC 412 in the form of an oral pill, 3 times per day. Following treatment, 12 of the patients had at least a 50% reduction in their cancer cell levels in their circulating blood, and 5 patients experienced at least a 50% reduction in their cancer cell levels in their bone marrow. Future clinical trials are planned to evaluate PKC 412 in addition to chemotherapy, as well as in patients with AML who do not have the FLT3 mutation.

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Patients with AML who have the FLT3 mutation may wish to speak with their physician about the risks and benefits of participating in a clinical trial further evaluating PKC 412 or other promising therapeutic approaches. Two sources of information regarding ongoing clinical trials include the National Cancer Institute (cancer.gov) and www.cancerconsultants.com. Personalized clinical trial searches on behalf of patients are also performed by cancerconsultants.com.

Reference: Stone R, et al. Oral chemotherapy drug shows promise in treating people living with AML. Proceedings from the 2003 annual meeting of the American Society of Clinical Oncology. May 2003. Abstract #2265.

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