Pediatric Leukemia “Super-Drug” Could Become Reality in Coming Years


With three studies over the last two years, researchers at Northwestern University Feinberg School of Medicine have published the final paper on mixed-lineage leukemia 1 (MLL1), a key protein responsible for the disease’s growth. In this time they have discovered two successful therapies that slowed the progression of pediatric leukemia, and are now looking at ways of combining their effect.

Children diagnosed with MLL-translocation leukemia, a cancer that affects the blood and bone marrow, generally have only a 30% survival rate. Patients experience a drop in red blood cells, making them anemic and increasing the number of white blood cells by about 80 times.


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”, said senior author Ali Shilatifard, Professor of Biochemistry and Molecular Genetics and Pediatrics. “This is a monster cancer that we’ve been dealing with for many years in children”.

For the last 25 years, Dr. Shilatifard’s work has revolved around studying the molecular function of MLL within its complex known as COMPASS (Complex Proteins Associated with Set1). These COMPASS components have been identified as core mutations within cancers and tumors.

Previous findings from the research team involved compounds that could slow cancer growth by interrupting a gene transcription process known as “super elongation complex” (SEC).  At that time, it was a world first and SEC became the first compound in its class to do so.

I’ve been working on this translocation for more than two decades, and we’re finally at the point where in five to 10 years, we can get a drug in kids that can be effective”, noted Shilatifard. “If we can bring that survival rate up to 85 percent, that’s a that’s a major accomplishment”.

More importantly, this new therapeutic approach opens the door not only for leukemia treatments, but also other cancers with solid tumors, such as breast or prostate cancer.

The next step is setting up clinical trials, which the team hopes to achieve in the next three to five years.