Research by Type:
| Leukemia Research |
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BACKGROUND AND CHALLENGES It is estimated that more than 44,500 new cases of leukemia will be diagnosed and nearly 22,000 deaths will occur in the United States in 2011. Leukemia is the leading cause of cancer deaths among children under age 15, accounting for at least 34% of all cancer deaths in this age group. Although these numbers are still daunting, the outlook for patients with leukemia is much better now than it was thirty years ago. In the mid-1970s, only 35% of patients suffering from leukemia experienced five year survival. Today, that number has increased to 50%. Much of the improvement in patient survival was made possible by the continuous development of new and more effective anti-cancer treatments, which has been largely driven by the rapid advancement of cancer research and the application of new technologies in oncology. Achieving further advances in clinical care for this devastating disease will require sustained support of creative new research approaches - research that will enlarge our understanding of the disease process in leukemia and lead to more effective strategies for leukemia treatment and prevention. In 2011, NFCR continues to fund several leukemia research projects, each led by a pioneer in the field. Through their efforts to design new anti-cancer drugs that target only cancer cells and develop novel immunotherapy and other new therapeutic approaches for leukemia patients, NFCR scientists are mounting a solid attack on this deadly cancer. This is the type of innovative research that may well produce new treatments for saving and extending the lives of leukemia patients. Listed below are a few notable research programs that NFCR funds. INNOVATIVE RESEARCH Developing new anti-cancer drugs that target only cancer cells NFCR scientist Dr. Alan Sartorelli is a world-renowned pharmacologist who designs, synthesizes, and evaluates novel anti-cancer drugs. He and his team developed laromustine, a promising drug for the treatment of various types of leukemia, brain tumors, lung and other types of cancer. In recent early stage clinical studies, laromustine demonstrated encouraging treatment effectiveness in patients with Acute Myeloid Leukemia (AML). laromustine belongs to a class of chemotherapy agents called guanine O6-targeting drugs which modify specific molecules in the DNA of cancer and other rapidly dividing normal cells, leading to cell death. Dr. Sartorelli's team is now developing a second generation of laromustine-like agents that only target cancer cells. The team has designed an inactive form of the drug that converts to the active, cell-killing form only after it enters a cancer cell; this conversion process does not take place in normal, healthy cells. In addition, the drug's unique conversion mechanism may also allow targeting of metastatic cancer cells that have spread to distant sites in the body. With his innovative drug design, Dr. Sartorelli envisions that these new targeted drugs will cause little toxicity while effectively treating patients whose tumors are resistant to existing therapies. MicroRNAs - master multi-switches in formation of normal blood cells and leukemia A mysterious group of tiny cellular molecules, called microRNAs, do not participate in protein production in the cell as other RNA molecules do. Puzzled by the existence and functions of these molecules for a long time, scientists have recently discovered that microRNAs play an important regulatory role in controlling complex processes such as normal human development and cancer. NFCR scientist, Dr. Curt I. Civin, is one of the first to take a close look at the functions of microRNAs in the formation of normal blood cells and leukemia. Using cutting-edge research techniques, the Civin team found that certain microRNA molecules are powerful "master regulators" that switch critical genes on and off during normal blood cell and leukemia formation. To date, the team has pinpointed two microRNAs that regulate maturation of normal blood cells, and two others that even suppress the development of leukemia. Their research further demonstrated that one of these tumor suppressor microRNAs may slow the growth, increase spontaneous death, and increase drug sensitivity of human leukemia cells. With continued efforts, Dr. Civin's team intends to decipher how these microRNAs work to target normal blood formation and impact leukemia growth and survival. Dr. Civin's work is laying a solid foundation for developing novel anti-leukemia treatments and enhanced transplantation and transfusion therapies. Developing novel immunotherapy T cells are powerful white blood cells that track down and kill cancer cells. However, cancer cells can still grow and spread by tricking T cells and escaping from their immune attack. NFCR scientist, Laurence Cooper, M.D., Ph.D., is a pioneer in developing adoptive immunotherapy - a novel approach that treats cancer patients' white blood cells in the laboratory to stimulate their immune systems and enhance their cancer-fighting capacity. Certain leukemia and lymphoma cells express molecules called CD19 on their surface. In Dr. Cooper's new approach, T cells are collected from these patients' blood and engineered to express an antenna-like molecule called CAR (chimeric antigen receptor). Since CAR readily binds to the CD19 molecules, the CAR T cells, once infused back into patients, should be able to target and mount a full-blown immune attack on CD19+ cancer cells. Dr. Cooper's first clinical study demonstrated that CAR T cells are safe and feasible for use in patients with CD19+ lymphoma. With continued NFCR funding, Dr. Cooper and his team at MD Anderson Cancer Center are further improving the safety of CAR T cells for their patients. This promising novel immunotherapy could be the basis for a new and more effective approach to treating leukemia and lymphoma patients in the not too distant future.
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