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Acute myeloid leukemia (or AML) is the deadliest form of leukemia in both children and adults. Today's treatments have changed little in decades and are notoriously brutal. In fact, therapy can be as life-threatening as the cancer itself, and visits to ICU for treatment-related complications are not uncommon. Both young patients and their families suffer terribly, as some struggle for years through multiple rounds of intense inpatient therapy, dangerous complications, and relapses.
This is an exciting time in cancer research. Targeted treatments, like T-cell immunotherapy and precision drugs, can take aim at the cancer with fewer side effects. You may have seen CAR T-cell therapy all over the news. It's been an incredible breakthrough, boasting 93% remission rates, for kids fighting the most common form of leukemia -- acute lymphoblastic leukemia (or ALL). However, almost one quarter of young leukemia patients have AML, one of the poorest prognosis pediatric cancers, and access to these exciting new therapies lag. But WHY?
In a nutshell, the promising targeted therapies require one critical thing to work -- GOOD TARGETS. Targets can be genetic mutations that cause a normal cell to "behave badly" and become cancerous. Targeted drugs can stop the root cause of the "bad behavior", and thus, stop the cancer. Targets can also be markers that make cancer look different from normal cells...drugs and cancer-killing T-cells can be programmed to look for these specific markers and avoid harm to normal tissues.
To take full advantage of cutting-edge, targeted therapies, we need a comprehensive list of targets, because knowing these targets can lead us to new treatment weapons -- even drugs and T-cells designed to treat other diseases and cancers! Some very cool discoveries have been made just this year...but we know there are more.
The majority of research funding and effort continues to be focused in seniors with AML, where the disease is more common. But we know that AML is a DIFFERENT disease process in pediatric patients (defined as ages 0-35) -- so separate study and target discovery in young people is critical. In fact, this TARGET PEDIATRIC AML effort has been identified as the "highest priority and greatest need" AML research project by the Children's Oncology Group, the world's largest organization dedicated to pediatric cancer research (signed endorsement letter available).
The largest repository of pediatric AML samples in the world resides in Seattle at Fred Hutch. Cells from many kids who are no longer here can help unlock the secrets of AML and identify the vulnerabilities of the enemy. Large-scale genomic sequencing and other cutting-edge testing and analysis can help us identify a big list of targets, and thus, build a bigger and more creative treatment arsenal.
Beyond finding the "big list of targets", we also want to offer genomic testing to sick kids RIGHT NOW. This special test can help identify novel treatment options and clinical trials, based on a kid's specific cancer profile (mutations), that their medical team may never have considered!
HELP US PIONEER PERSONALIZED, PRECISION MEDICINE for young people with AML! It will be up to us -- friends, family and foundations who care about kids and young adults fighting AML -- to accelerate this work. We are also working hard to educate and rally foundations across the country, even the big ones, about this strategic effort. But while we're doing that, we're getting started RIGHT NOW.
100% of your GIFTS will help us accelerate target discovery and provide genomic testing that can help desperate kids, RIGHT NOW -- with NO OVERHEAD. You heard it right....no overhead. 100% will be used to sequence and analyze patient samples....every penny!!!
For more detailed information about this very strategic AML research or to discuss larger gifts, contact firstname.lastname@example.org. Personal presentations to your organization can also be arranged that include key researchers.