"Super drug" for pediatric leukemia may be developed in coming years: study

Source: Xinhua| 2018-12-23 00:40:39|Editor: yan
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CHICAGO, Dec. 22 (Xinhua) -- Northwestern Medicine researchers have discovered two successful therapies that slowed the progression of pediatric leukemia in mice.

They have published three studies on this over the last two years in the journal Cell, and the fourth one, or the final paper was published Thursday in Genes & Development.

The latest study shows that when a key protein responsible for leukemia, MLL, is stabilized, it slows the progression of the leukemia. The researchers will combine the treatments from the past two years of research into a pediatric leukemia "super drug" to test on humans in a clinical trial in the next three to five years.

The survival rate is only 30 percent for children diagnosed with MLL-translocation leukemia, a cancer that affects the blood and bone marrow. Patients with leukemia have a very low percentage of red blood cells, making them anemic, and have approximately 80 times more white blood cells than people without cancer.

"These white blood cells infiltrate many of the tissues and organs of the affected individuals and is a major cause of death in leukemia patients," said senior author Ali Shilatifard, a Northwestern University professor of biochemistry and molecular genetics and pediatrics. "This is a monster cancer that we've been dealing with for many years in children."

There are several types of leukemia. The Northwestern Medicine study focused on the two most common found in infants through teenagers: acute myeloid leukemia (AML) and acute lymphocytic leukemia (ALL).

For the past 25 years, Shilatifard has been studying the molecular function of MLL within its complex known as COMPASS (Complex Proteins Associated with Set1). The most recent study found that COMPASS components are one of the most frequently identified mutations in cancer.

Northwestern Medicine is the collaboration between Northwestern Memorial Healthcare and Northwestern University Feinberg School of Medicine, which includes research, teaching and patient care.