Pediatric leukemia ‘super drug’ could be less than a decade away
Building on several years of research into a protein responsible for leukemia, a newly published paper has revealed success in slowing the progression of the deadly cancer in mouse models. The researchers suggest two new prospective therapies can now be combined into one "super drug" and progress into human clinical trials.
The research has concentrated on a type of leukemia underpinned by a translocation involving a gene called MLL. It is very common to find MLL translocations in both acute myeloid and lymphoid leukemia, particularly in infant patients. The current survival rate for young patients diagnosed with this kind of cancer is only 30 percent.
MLL is one of several proteins in a complex family referred to as COMPASS (Complex Proteins Associated with Set1). Ali Shilatifard, from Northwestern University, has been investigating the COMPASS family for almost 25 years. Mutations in the COMPASS family are frequently identified in many different cancers, and Shilatifard has been particularly focused on the molecular function of MLL for many years.
The latest research has revealed a new technique that can stabilize the MLL gene, essentially disrupting the translocation process that results in leukemia. The new study follows on from other recent work by Shilatifard and the Northwestern team uncovering compounds that disrupt the gene transcription process seen to cause cancers to grow.
The Northwestern team suggests these two successful experimental methods can be incorporated into one "super drug" and moved into human trials within the next few years. This is the culmination of four separate published studies from the last two years.
"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," says Shilatifard. "If we can bring that survival rate up to 85 percent, that's a major accomplishment."
The research of course is still untested on humans, so much more work needs to be done to establish if these treatments are safe and effective. However Zibo Zhao, first author on the new paper, is optimistic the work will effectively translate into humans, and also be applicable to a variety of other solid tumor cancers, including breast and prostate, as well as leukemia.
"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," adds Zhao.
The new paper was published in the journal Genes & Development.
Source: Northwestern University