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‘Super Drug’ for Pediatric Blood Cancer May Soon Be Reality

This opens up a new therapeutic approach not only for leukemia, but also for other types of cancers, say researchers

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A “super drug” that helps slow down the progression of blood cancer in children could soon become a reality, say researchers.

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.

However, the survival rate is only 30 per cent for children diagnosed with MLL-translocation leukemia, a cancer that affects the blood and bone marrow.

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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. This is a monster cancer that we’ve been dealing with for many years in children. We’re finally at a point where in five to 10 years, we can get a drug in kids that can be effective. If we can bring that survival rate up to 85 per cent, that’s a major accomplishment.
Ali Shilatifard, Professor at the Northwestern University in the US 

In the study, detailed in the journal Genes and Development, the team demonstrated that when a key protein responsible for leukemia, MLL, is stabilised, it slows the progression of the leukemia.

This MLL stabilisation process could potentially work in cancers with solid tumours, such as breast or prostate cancer.

The next step will be to combine the treatments from the past two years of research into a pediatric leukemia “super drug” to test on humans in a clinical trial, the researchers said.

“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,” the study showed.

The team also identified compounds that could slow cancer growth by interrupting a gene transcription process known as “Super Elongation Complex” (SEC).

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Topics:  Blood Cancer 

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