Target Pediatric AML
Together We Can Fund the Cure

Initiative Overview

Speeding personalized, precision medicine to young AML patients


Endorsed by the Children's Oncology group as the "highest potential, greatest need" Aml-related project

TpAML is endorsed by the Children’s Oncology Group (COG) as the “highest potential, greatest need” AML-related research effort. It is both a fundraising initiative and a large-scale research project. It’s a grassroots, collaborative PROJECT, not a foundation. All gifts are directed to a dedicated TpAML account managed by Children’s Oncology Group.

TpAML will speed personalized, precision medicine to young AML patients with a two-pronged approach:

  1. Expand our treatment arsenal ($6 million) by maximizing use of all available targeted agents, even those designed for other diseases via comprehensive target discovery across all AML subtypes.
  2. Jumpstart personalized medicine NOW ($2.7 million) by providing clinical genomic testing to all active AML patients treated at COG-affiliated institutions.

zach. a childhood AML patient

Targeted therapies (precision agents that identify and attack cancer, like modified T-cells) hold great promise for improving outcomes and reducing treatment-related toxicities in the most challenging, resistant cancers. Personalized medicine, or combined usage of drugs and biologics according to the patient’s specific cancer profile (genetics), is another high-potential therapeutic approach gaining momentum.

The Target Pediatric AML Project (TpAML) seeks to maximize and speed access to personalized, precision treatment regimens for young acute myeloid leukemia (AML) patients ages 0-35. Leveraging the power of deep, omic sequencing and big-data analysis, TpAML will expose, in a methodical and comprehensive manner, the key drivers, enablers, unique identifiers (biomarkers) and vulnerabilities of AML in young patients, whose disease differs significantly from older adults. This will enable expansion of our treatment toolkit and jumpstart more informed, individualized therapy.

pediatric aml hand holding

TpAML is, essentially, a “military intelligence” project that will identify a big list of AML vulnerabilities that can be exploited by targeted therapies – even agents used to treat other diseases. It is unlikely that pediatric-AML-specific drugs for AML will ever be developed. So, maximizing, repurposing, and refining existing agents – and delivering them in the right combinations to patients based on their unique leukemia profile – will be key.

Because AML is a complex, diverse group of diseases, one or two “magic bullet” cures is unlikely. Safely targeting AML has also been challenging. The more AML-specific therapeutic targets we identify, the more targeted agents we’ll have in our treatment toolkit.

We are seeing both short and long-term wins from this largest-ever, comprehensive biology study that will fully define the ‘disease landscape’ of pediatric AML and build upon the NCI-funded Target Initiative (and the work of the late Dr. Bob Arceci).

Young AML patients keep falling through the cracks. This initiative mirrors and counterbalances the Leukemia & Lymphoma Society’s nationwide, multimillion-dollar “Beat AML” campaign, which is currently focused on patients 60 years and older with similar goals – targeted drug discovery and tailored treatment. TpAML is also similar to the new NCI-COG Pediatric Match trial.  

TpAML seeks to maximize the use of all existing and emerging targeted agents -- like modified T-cells, antibody therapies, and small molecule inhibitors -- via comprehensive target discovery (since effective targeting in AML remains a significant barrier).

TpAML will also fund clinical genomic sequencing of all young AML (and mixed phenotype or MPAL leukemia) patients nationwide for 2 years to both discover novel drugs that could help these patients, right now, and support research efforts. We also hope to drive clinical genomics to “standard of care”, reimbursed by insurance and other payors. This study has the potential to fundamentally change how we diagnose, monitor, and treat AML in young patients. Genomic sequencing testing is different from today’s standard cytogenetic testing. It provides deeper insights into the exact genomic alterations driving a patient’s AML mapped to potentially beneficial targeted therapies and clinical trials.

Because TpAML is an $8.7 million, multi-phase effort, no single organization likely can nor will fund this project in its entirety. So, a collaborative, creative “funds-pooling” campaign is needed to accelerate this strategic, high-potential work. A group of parent volunteers has stepped up to reach out to friends, families and organizations worldwide, seeking funding and research/working partners. Approximately $2.5 million has been pledged/raised to date (first gift received July 2016).