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Novel Drug Discovery Eradicates Deadly Leukemia in Mice

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drug discovery wipes out leukemia in mice Paul Hakimata/PhotoSpin

UCLA researchers may be one step closer to finding a cure for a fast-growing and deadly blood cancer, acute lymphoblastic leukemia (ALL) that affects infants, children, and more rarely, adults.

In a remarkable discovery, a diverse team of researchers from UCLA’s Jonsson Comprehensive Cancer Center has developed an experimental treatment that eradicates ALL in mice without any detectable toxic side effects.

The drug works by blocking two important metabolic pathways that the leukemia cells need to grow and spread, most often to the liver, spleen, and throughout the body.

ALL is an acute type of leukemia that affects the white blood cells called lymphocytes. White blood cell growth is accelerated, which eventually crowds out healthy bone marrow, preventing it from making the normal red blood cells, white blood cells, and platelets that your body needs.

There are about 6,000 new cases of ALL diagnosed in the United States each year. About 1,400 of those cases are fatal. ALL can cause a variety of symptoms, but the most common are fever, fatigue, loss of appetite or weight, pale skin and night sweats.

The new experimental drug works deep within the cells to block specific chemical pathways that feed out-of-control cancerous blood cell growth. When drug molecules were used to block these pathways, the cancer cells died.

The study was led by Dr. Caius Radu, associate professor of the biomedical physics interdepartmental program, and molecular and medical pharmacology, and Dr. David Nathanson, assistant professor of molecular and medical pharmacology. It was published online ahead of print on February 24, 2014 in The Journal of Experimental Medicine.

The researchers found that two pathways, the de novo pathway and the nucleoside salvage pathway, produce an important chemical called deoxycytidine triphosphate.

When an existing drug that’s commonly used in treating leukemia was given to block the de novo pathway in a leukemia cell, the cancer cell simply chose an alternative route through the nucleoside salvage pathway (NSP). Once the cell had a clear path, it began producing the chemicals it needed to survive.

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