Since AML is most common in older adults, why do we need pediatric-specific AML research?
Targeted therapies developed in seniors may not benefit young patients. AML is, genetically, a different disease in young people – so, drug targets will differ. It’s imperative that we find targets and corresponding targeted therapies for ages 0-35 – the goal of Target Pediatric AML.
While some new AML drugs, like CPX 351, could benefit patients across all ages, all research cannot be cross-applied. The exciting, high-potential, targeted therapies (like modified T-cells) must strike targets present in young patients. AML’s genetic makeup, drivers and targets vary by age. Targeted agents approved for older adults may be of little benefit to young people. One example is a recently approved AML drug that targets IDH2, a protein highly expressed in seniors but rare in young patients.
Genomic and disease biology studies have proven that AML is a different disease in young patients (ages 0 to approximately 35). Yet, because AML is primarily a disease of older adults (avg. age of diagnosis is 67), most research and clinical trials are focused on AML patients ages 60+. In fact, today’s childhood AML treatments were developed almost solely based on information gathered in adults.
The underlying causes and drivers of AML in young people differ significantly from older adults (who have accumulated a lifetime of cell damage and genetic mutations from lifestyle, environmental exposures, etc.). Often, a single, catastrophic event triggers AML in a young person. If that mutation or variant can be targeted, outcomes can be improved. One great example is recent progress in patients with FLT-3 mutations.
Pediatric AML research remains underfunded due to several lingering MISCONCEPTIONS:
AML in seniors and young people is the same disease
“Just wait” -- promising discoveries in seniors can be passed down to kids (the “trickle-down” approach)
ALL (acute lymphoblastic leukemia) is more common and kills more children, so most pediatric leukemia research/funding should be focused there
The MLL rearrangement is essentially the same across ages and different leukemias (“the ALL or adult MLL research will benefit you, so wait”)
With recent advances (ex. CAR T-cell therapy) in the most common form of childhood leukemia, ALL, the time is right to invest more in pediatric AML. AML remains one of the poorest prognosis pediatric cancers and, though rare, now kills more children than all other leukemias.
AGE IN AML MATTERS. Target Pediatric AML seeks to fundamentally change the way AML is diagnosed, monitored, and treated in YOUNG PATIENTS – informed by “big data” collected in young patients, not older adults.