New 'Super Drug' In Development Could Become A Powerful Tool Against Pediatric Leukemia

Currently, leukemia is the world’s most common form of cancer in children and teens affecting thousands across the globe.

A devastating malignant, and progressive disease, leukemia is when bone marrow and other blood-forming organs produce increased numbers of immature or abnormal leucocytes, leading to the suppression of white blood cells.

The sad reality with leukemia is that there is really nothing you can do to prevent the disease. This cancer of your blood cells takes the lives of hundreds of thousands of people around the world.

"These white blood cells infiltrate many of the tissues and organs of the affected individuals and are a major cause of death in leukemia patients," says Ali Shilatifard, the Robert Francis Furchgott Professor of Biochemistry and Molecular Genetics and Pediatrics.

"This is a monster cancer that we've been dealing with for many years in children."

Recently, researchers from Northwestern University have discovered two successful therapies that slowed the progression of pediatric leukemia, opening the doors to the potential creation of a super drug that could be used to combat the disease in the coming years.

The Fight Against MLL

Sadly, children diagnosed with MLL-translocation leukemia have only a survival rate of around 30%. The two years, a three-part study conducted by Northwestern Medicine aims to reduce the mortality rate of the disease by attacking the key protein responsible for leukemia.

Published in the scientific journal, Cell, Northwestern researchers have discovered that stabilizing the key protein of MLL, could slow the spread of the disease throughout the affected body. As mentioned above, there are various types of leukemia.

However, for the Northwestern Medicine study, researchers focused on the two most commonly found in infants through teenagers: acute myeloid leukemia and acute lymphocytic leukemia.

The research itself builds off of previously published reports in which Shilatifard and her team identified compounds that could slow cancer growth by interrupting a gene transcription process known as "Super Elongation Complex".

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This new leukemia stabilization process could be a powerful weapon against the disease, revolutionizing treatments for solid tumors, such as breast or prostate cancer.

"This opens up a new therapeutic approach not only for leukemia, which is so important for the many children who are diagnosed with this terrible cancer but also for other types of cancers that plague the population," says first author Zibo Zhao, a postdoctoral research fellow in Shilatifard's lab.

Medical research like this holds tremendous promise and could be used to save the lives of millions of children in the near future.