CIRM Planning Grant in Leukemia
Leukemias are cancers of the blood forming cells that afflict both children and adults. Many drugs have been developed to treat leukemias and related diseases, but in many cases of adult leukemia, the diseases are not curable, and cause disability and eventual death. More than in other cancers, scientists understand the exact molecular changes in the blood forming cells that cause leukemias, but it has been very difficult to translate the scientific results into new and effective treatments. The main difficulty has been the failure of existing agents to eliminate small numbers of leukemia stem cells that persist in patients, despite therapy, and that continue to grow, spread, invade and kill normal cells. In fact, the models used for drug development in the pharmaceutical industry have not been designed to detect drugs or drug combinations capable of destroying the leukemia stem cells. Drugs against leukemia cell stems may already exist, or could be simple to make, but there is not an easy current way to identify them.
Recently, physicians and scientists at universities and research institutes have developed tools to isolate and to analyze leukemia stem cells taken directly from patients. By studying the leukemia stem cells, physicians and scientists have learned what molecules these cells need to survive. We have identified key switches that reprogram normal stem cells into leukemia stem cells. The experimental results strongly suggest that it will be possible to destroy leukemia stem cells with drugs or drug combinations, without harming most normal cells. Now we need to translate the new knowledge into practical treatments.
The goal of the CIRM Planning Grant in Leukemia is to forge a team, and to implement a planning process that will bring together highly experienced scientists and physicians who have developed the leukemia stem cell test systems, with our existing collaborators in the vigorous California pharmaceutical industry, who already have drugs, but lack the cells and model systems to assess their efficacy against leukemia stem cells. The requested funds will support the establishment of an interactive group, a review of projects by expert external advisors, the integration of selected projects according to a practical timeline, and the generation of a reporting structure for CIRM. The new CIRM-supported interactive group will have the resources to introduce into the clinic, within four years, new drugs for leukemias and to change the ways that these diseases are treated for the benefit of the citizens of California.
University of California, San Diego
Disease Team Planning