Target Pediatric AML
Together We Can Fund the Cure

Goals & Objectives

why target pediatric aml?

Fueling a fundamental shift in how pediatric AML is treated

TpAML will speed access to promising targeted therapies and begin the shift from the virtual “one-size-fits-all” protocols of today to more personalized, precision treatment regimens tailored to a young AML patient’s specific cancer profile (genetics). This should not only improve outcomes, but reduce both short and long-term treatment-related toxicities. 

TpAML will enable:

  • Genomic testing at diagnosis and relapse, listing all genetic alternations that could be driving the patient’s AML mapped to all known targeted agents and clinical trials (moving beyond today’s basic cytogenetic testing).
  • Improved risk classification and prognosis, which can guide aggressiveness of treatment and disease monitoring
  • A “virtual tumor boardto interpret genomic testing results and provide local oncologists with the most cutting-edge information and treatment options available
  • Expanded treatment options (including options available via compassionate use)
  • Molecular-level disease monitoring (PCR testing) that can identify 1 in 1 million cancer cells vs. today’s FISH/flow cytometry tests (that can identify only 1 in 10,000 cells).
TpAML vision for success

The initial SCOPE of TpAML consists of two major activities with both near and long-term benefits:

 

To Unravel the disease

Build a comprehensive list of therapeutic targets via discovery-phase, large-scale, deep “omic” sequencing and analysis of 1000 pediatric patient samples. This will enable us to expand and maximize our treatment toolkit.


To Begin a shift to personalized care

Profile each newly diagnosed patient’s AML, beginning the shift to personalized care, with the goal of offering clinical genomic sequencing to every young patient nationwide. Testing will also be extended to biphenotypic leukemia patients (mix of ALL & AML), a high-risk group routinely excluded from many trials. Genomic testing allows us to match all available (and emerging) therapies to a patient based on their specific cancer profile. It can also allow us to better understand why some patients respond more than others to a given therapy and will provide information for real-time, molecular level disease monitoring (more sensitive and personalized than today’s routine tests).

 

The TpAML discovery effort has five primary goals:

DEFINE THE DISEASE LANDSCAPE

Obtain a comprehensive, detailed view of the drivers, vulnerabilities, and unique identifiers (biomarkers) of AML in patients 0-35 years, finishing and expanding the work started by the NCI’s “Target AML” project and highlighted/prioritized in the Cancer Moonshot Blue Ribbon Panel Report 2016 (p.21-30).


EXPAND TREATMENT OPTIONS

More rapidly and more rationally expand our treatment toolkit (spawning new clinical trials) by

  1. Discovering and repurposing existing drugs designed for other diseases
  2. Guiding the development (and creative use) of new agents.

JUMPSTART PERSONALIZED, PRECISION MEDICINE

Turn over every stone, for EVERY child, nationwide, NOW by offering clinical genomic sequencing testing delivered by FoundationOne Here. This specialized testing will identify a comprehensive list of trial and treatment options based on the patient’s cancer profile (enabling best matching of all possible known therapies to a patient).


IMPROVE VALUE OF CURRENT RESEARCH

By including patients most likely to respond (based on their cancer genetics) and using genetic profiling to better understand why certain patients responded and others did not, we can enable improved therapy matching/application and inform future research. 


FUNDAMENTALLY CHANGE & IMPROVE Outcomes

Improve outcomes and reduce treatment-related toxicities leveraging more personalized, precision medicine (including molecular-level disease monitoring to (a) guide treatment and (b) detect and intervene in relapse early).

 

The future vision of pediatric AML treatment – the new patient experience:

Target Pediatric AML's vision