Acute myeloid leukemia (AML) remains one of the poorest prognosis pediatric cancers. AML therapies have not changed in decades, are notoriously toxic, and harrowing side effects routinely send kids to the ICU. Treatment can be as dangerous as the cancer itself. Young survivors often face significant long-term health issues. Entire families are traumatized and even torn apart. Treating a single, complex patient can cost millions. We can and must do better, FASTER. [SEE VIDEO BELOW]
Cutting-edge, targeted drugs and T-cell therapies offer the greatest hope for breakthroughs. Powerful new tools, like genomic sequencing, can enable the discovery of new targets and more rapid, informed expansion our treatment "arsenal". Even old drugs designed for other diseases can be uncovered and repurposed to treat AML! These same tools can help us match the right therapies, in the right combinations, to patients -- delivering personalized, precision medicine. This is important because AML is a complex group of diseases, and every patient's AML can be unique -- like a fingerprint. One "magic bullet" cure for AML is unlikely. Tailored treatment regimens are widely regarded as key to improving outcomes and reducing toxic side effects.
Many of the exciting, new cancer weapons you hear about in the news are "targeted therapies", so they depend on GOOD TARGETS to work. "Target Pediatric AML" seeks to perform deep sequencing on 1000 patient samples to build a comprehensive list of targets for AML patients ages 0-35. Identifying more targets will help us maximize both existing and new treatments, including promising T-cell therapies. The project will also help sick kids NOW by offering genomic sequencing testing to both pediatric AML and biphenotypic (mixed) leukemia patients to uncover all current treatment options, enable precision diagnosis/monitoring, and inform current research.
Contact Julie Guillot email@example.com or Michael Copley firstname.lastname@example.org (AML parents and TpAML supporters/volunteers) for more information about this project and to discuss how your family or foundation can collaborate. Many thanks to current/early supporters of this and related work -- the NCI (National Cancer Institute), St. Baldrick's Foundation, Alex's Lemonade Stand, and Hyundai Hope on Wheels. We also thank our RECENT collaborators from all over the United States and Canada: