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Something for everybody…some people like chocolate ice cream, some people vanilla.  Some people only want to read about family assistance, and some people are only concerned with research.  For those of you interested in research, today’s update is for you!  Here’s an update on one of the 96 research projects that we’ve been able to help fund because of you.  Thank you!

The “Target Pediatric AML” (TpAML) project is the largest, most comprehensive and methodical disease biology study ever conducted in Acute Myeloid Leukemia. It is also the “highest potential, greatest need” AML-related research project as determined by the Children’s Oncology Group (COG).  The COG, supported by the National Cancer Institute (NCI), is the world’s largest organization devoted exclusively to pediatric cancer research. The COG has over 7,500 members from 200 institutions in North America, Australia, New Zealand, and Europe.

We are proud to report that The Andrew McDonough B+ (Be Positive) Foundation was the first funder of TpAML. Subsequently, the TpAML project, under the incredible leadership of Julie Guillot and Michael Copley, has raised millions of dollars.  Here is a picture of one of the check presentations with two of the key players – Dr. Peter Adamson, Head of the Children’s Oncology Group and Dr. Andy Kolb, Chair of the Myeloid Committee of the COG.  Both Peter and Andy are members of The B+ Foundation Scientific Advisory Board as well.

This focused discovery effort is sorely needed due to the complexity and diversity of this challenging disease, which has one of the poorest prognosis rates among childhood cancers. TpAML will leverage large-scale, deep, ‘omics’ sequencing of over 1,000 banked patient samples to, for the first time, fully define the landscape of pediatric AML. TpAML plus NCI-generated TARGET data, combined, will provide researchers with insights into the disease biology of over 1,500 pAML patients. The project will identify new subtypes, drivers, enablers, unique identifiers (biomarkers) and vulnerabilities. This process will uncover new therapeutic targets, which will be mapped to all existing/emerging targeted agents and inform development of new treatments, resulting in a much-needed, expanded treatment arsenal. TpAML also seeks to provide clinical genomic testing to all active pediatric AML patients to identify existing, novel agents that could benefit young patients, right now, as well as better assess a patient’s true risk, and adjust treatment accordingly. The data can also inform findings of clinical trials to enable more personalized treatment regimens in the future.

A new and exciting evolutionary development is the recent decision by the Leukemia & Lymphoma Society (LLS) to invest in this project. LLS’s related project, titled “PedAL” (yes, everything has to have an acronym) is budgeted at $68.7MM, with $24.3MM of that coming from LLS directly and the organization hopes to secure the balance from pharmaceutical companies. This has the potential to be a particularly significant development not only because of these monies, but also because it is a strategic sea change at LLS. Historically, LLS has allocated very little funding dedicated to pediatric blood cancers. I met with LLS senior executives last month and they have assured me that there is a new and sincere commitment to fighting childhood cancers. LLS’s connections with pharma and the FDA could prove very valuable in our battle. I am cautiously optimistic and pretty excited.

Live Like Andrew!

B+

Ali & Andrew’s Dad

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