December = Atlanta, ASH, & Groundbreaking Work in Leukemia Clinical Research.

I’m headed to Atlanta today for the ASH Annual Meeting. As always, I eagerly anticipate this milestone in the year; a fabulous opportunity for learning of developments in hematology-oncology, and a chance to reconnect with colleagues focused on important research, clinical trials, and patient care in hematologic malignancies. This year is particularly special for me as an opportunity to round up with the many collaborators that have made a groundbreaking trial in Acute Myeloid Leukemia (AML) a reality, and the dedication that folks at The Leukemia & Lymphoma Society (LLS) have shown to drive achievement of new milestones in this innovative offensive against this intractable disease.

 Each year, more than 21,000 Americans are diagnosed with AML. Despite a remarkable wealth of knowledge about the molecular biology and drivers of this disease and a multitude of detailed clinical trials and promising drugs (and in contrast to advances in treating other blood cancers), standard treatment for AML has changed very little over the past four decades. Overall patient prognosis remains poor, with a five-year survival rate of approximately 26 percent. But there is hope on the horizon. Many biopharmas have developed targeted therapies for several of the known driver mutations involved in AML, and emerging data with several are promising (e.g. with flt 3, IDH2 etc). More than a year ago the LLS developed a “mandate for change” in AML investigations, and, convening with FDA, leading leukemia physicians, several biopharmas, NGS testing teams, and multiple service groups, has convened BeatAML; a Master Clinical Trial (umbrella) approach that seeks to make precision medicine and patient-centricity a clinical reality for patients newly diagnosed with AML. 

 Today, the LLS will announce significant progress in the 12 months or so since Beat AML “went live”:

 Participation from Leading Leukemia Centers: The most recent cancer center to join the Beat AML Master Clinical Trial is the University of Maryland Marlene and Stewart Greenebaum Comprehensive Cancer Center. Maryland joins some of the most prestigious medical centers across the country including Memorial Sloane Kettering, Ohio State University Comprehensive Cancer Center, OHSU Knight Cancer Institute, Harold C. Simmons Comprehensive Cancer Center at University of Texas Southwestern; University of Chicago Comprehensive Cancer Center; and University of Colorado Cancer Center. Several more cancer centers are expected to join in early 2018.

 More Pharma Participation: Two more companies, Astellas and Takeda, recently joined Alexion, Boehringer Ingelheim, Celgene, and Gilead Sciences, each investigating targeted therapies to treat AML patients based on their subtype. There are now nine active treatment arms, each focused on a specific subtype of AML using the following investigational drugs: Alexion: samalizumab/ALXN6000 (anti-CD200); Boehringer Ingelheim: BI 836858 (anti-CD33); Celgene: enasidenib/AG-221/CC-90007 (IDH2 inhibitor); Gilead: entospletinib (SYK inhibitor), Astellas: gilteritinib (FLT3/AXL tyrosine kinase inhibitor); Takeda: pevonedistat (NAE inhibitor). Only enasidenib has been FDA approved, but as a treatment for relapsed/refractory patients, - it is being tested in a first-line setting in Beat AML.

 Strong Patient Recruitment, and Operational Proof of Concept: At the time that ASH will commence, around 150 patients have entered the Beat AML Master Clinical Trial, and it is expected to eventually include 500 patients, continue for at least two years, and involve up to 20 clinical sites in the US. Beat AML is proving out. One of the unique aspects of this trial is that it is led by a nonprofit health organization - a "patients first" organization, LLS that is well-positioned to convene this significant collaboration of stakeholders within the cancer ecosystem, including major cancer centers, pharmaceutical companies, and several technology companies ---all helping to manage the trial. The trial protocol employs advanced genomic technology performed by Foundation Medicine to identify the cancer-driving genetic mutations in newly diagnosed AML patients age 60 and older, in order to match them with an investigational drug best suited to their disease driver mutation. For this trial, the process is being completed within seven days, a time-frame that is unprecedented in genomic technology and clinical trial setting.

 For myself, and my colleagues at INC Research- inVentiv Health, it is both a privilege and a thrill to support this groundbreaking clinical research, and to collaborate with the many dedicated and innovative people – across all teams – required to bring BeatAML forward to patients. We all anxiously await data read outs, look forward to the expansion of this trial with more targeted therapies, and to hear at ASH from the many clinical and scientific thought leaders engaged in this trial; -- progress….and potential next steps such as novel-novel combinations. Importantly, it feels like we’re truly on the cusp of meaningful change for in care options for AML patients.