In 2007, over 13,000 Americans were expected to be diagnosed with acute myelogenous leukemia (AML), a fast-growing cancer of the blood and bone marrow.  Standard treatment is a month or more in the hospital for intensive chemotherapy to achieve remission, followed by periodic chemotherapy to prevent a recurrence.

Leukemia stem cells are thought to play a central role in both the development and recurrence of acute leukemia, and are relatively resistant to current chemotherapy.  This research has demonstrated that a compound called DMAPT destroys stem cells for 2 types of leukemia without damaging normal blood cells, and can be utilized effectively by the body. Phase 1 clinical trials in humans of this new approach to fighting leukemia are scheduled to begin shortly.  SWCRF is funding investigation of additional compounds with similar functions.

Leukemia stem cells share many characteristics with healthy blood-producing cells, which has made targeting only the cancer cells challenging. The team led by Craig T. Jordan had previously demonstrated that a naturally occurring compound called parthenolide (PTL) kills stem cells for 2 types of leukemia in a test tube without damaging normal blood cells. However, its use as a medicine is prevented by its poor solubility, which limits the amount of PTL which can be delivered to levels that don’t kill stem cells.

Working with collaborators at the University of Kentucky, Jordan’s team evaluated a family of modified-PTL compounds to find one that had the anti-cancer effect of PTL, but greater bioavailability and solubility. Utilizing a variety of different measures of efficacy, they demonstrated that a version of PTL called DMAPT meets both criteria. Using several mechanisms, DMAPT destroys leukemia stem cells and the cancer cells they produce. It both stresses the leukemia cells and stops their built-in damage repair mechanisms.

Based on these tests of effectiveness, its high bioavailability when administered orally and its low toxicity, DMAPT will be proceeding to human Phase 1 clinical trials in the near future. SWCRF is funding the search for additional effective and usable compounds to increase the likelihood that this innovative approach to stopping leukemia will eventually result in a medicine.

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