UPDATE (July 2024): The Opti-AML study, which LLS launched just last year as part of our groundbreaking Beat AML® Master Trial (see below), has reached an important milestone.
The first review of data from the study is reporting that treatment with a shorter cycle of venetoclax and azacitidine is safe and is as effective as the current FDA-approved standard of care in older adults with newly diagnosed acute myeloid leukemia (AML).
Investigators will continue enrolling patients into the study to validate these early findings. Full study results are expected by September 2025.
Rye Brook, N.Y., June 22, 2023 — The Leukemia & Lymphoma Society (LLS) today announced that the first patient was dosed in a clinical trial investigating whether a shorter course of combination therapy is less toxic while remaining as effective as the FDA-approved regimen in older patients with newly diagnosed acute myeloid leukemia (AML).
The aim of this phase 2 sub study within LLS’s Beat AML® Master Clinical Trial is to analyze if a 14-day cycle of venetoclax and azacitidine, followed by a 14-day break, elicits a similar complete remission rate in AML patients aged 60 years and older who are unable to tolerate intensive induction chemotherapy, versus the FDA-approved two 28-day cycles in a back-to-back sequence, which can prove too toxic for older patients. Approximately 200 patients will be enrolled onto the trial and will be randomized into either treatment arm. Treatment responses will be compared following two months of therapy.
“We theorize that by shortening the dosing interval, patients will recover their blood counts sooner, which we hope will reduce the risk of infections, as well as the need for hospitalization, and minimize the need for transfusion support while still maintaining the effectiveness,” said Principal Investigator Uma Borate, MBBS, physician and associate professor, Division of Hematology, The Ohio State University Comprehensive Cancer Center — Arthur G. James Cancer Hospital and Richard J. Solove Research Institute. “Ultimately, we are hopeful that patients on this shortened treatment cycle will experience fewer toxic side effects while maintaining the effectiveness of the therapy and thus have better outcomes.”
“The promise of a shorter, less toxic regimen for treating AML can be a gamechanger for older patients,” said E. Anders (Andy) Kolb, MD, president and CEO, LLS. “Infections among this patient population are a major concern and often lead to a disruption in treatment which we want to avoid when we’re dealing with this very aggressive and fast-moving form of leukemia.”
Making clinical trials more patient friendly
As the largest nonprofit funder of blood cancer research, LLS is committed to saving lives and improving quality of life. Through innovation within the Beat AML Master Clinical Trial, the infrastructure is pushing the boundaries in making clinical trials more patient centric.
Through partnerships with several leading clinical research technology software companies, patients enrolled in this sub study will benefit from a wearable patch linked to a smartphone that continuously monitors vital signs as well as symptoms and side effects. The device allows clinical researchers to remotely monitor a patient’s overall health. Additionally, phlebotomists will be sent to the patient’s home to reduce the patient burden of traveling for frequent blood draws.
“With this framework, LLS is embracing technology to create a trial that’s more manageable for older patients and their families,” said Dr. Kolb. “But this is just the beginning. We are continually looking for even more ways to make blood cancer care more patient centered.”
LLS is partnering with Korio, a company that provides randomization and trial supply management, as well as Narrativa to use the company’s artificial intelligence models and automated data analyses. These technologies will help reduce the time to market for potential new treatments by accelerating the approval process and will streamline the user experience during patient enrollment.
Changing how AML is treated
Recognizing the urgent need to do better for AML patients, LLS launched the Beat AML Master Clinical Trial in fall 2016 to test multiple novel targeted therapies at major cancer centers across the U.S. in newly diagnosed AML patients aged 60 and older.
In a historic first, LLS was the first non-profit health organization to sponsor a blood cancer clinical trial, which laid the groundwork for LLS to launch its Pediatric Acute Leukemia (PedAL) master clinical trial in 2022.
LLS has assembled a wide-ranging network of Beat AML partners, including the U.S. Food and Drug Administration (FDA), top cancer centers, multiple pharmaceutical companies, and select technology experts, who have united to find cures for AML.
To date, the ongoing trial has screened more than 1,300 patients at more than a dozen leading cancer centers. The trial works to swiftly diagnose a patient’s specific subtype and identify the most effective treatment for them and their form of AML. This has led to superior treatment and survival outcomes compared to acute myeloid leukemia (AML) patients treated with standard chemotherapy, according to findings published in the medical journal Nature Medicine.
About The Leukemia & Lymphoma Society
The Leukemia & Lymphoma Society® (LLS) is the global leader in the fight against blood cancer. The LLS mission: Cure leukemia, lymphoma, Hodgkin’s disease and myeloma, and improve the quality of life of patients and their families. LLS funds lifesaving blood cancer research around the world, provides free information and support services, and is the voice for all blood cancer patients seeking access to quality, affordable, coordinated care.
Founded in 1949 and headquartered in Rye Brook, N.Y., LLS has regions throughout the United States and Canada. To learn more, visit www.LLS.org. Patients should contact the Information Resource Center at (800) 955-4572, Monday through Friday, 9 a.m. to 9 p.m. ET.
For additional information, visit lls.org/lls-newsnetwork. Follow us on Facebook, X, and Instagram.
About the Beat AML® Master Clinical Trial
The Beat AML® Master Clinical Trial is the first collaborative precision medicine clinical trial in a blood cancer. Launched in 2016 and focused on newly diagnosed patients aged 60 or older, the trial uses advanced genomic technology to match patients to the most promising targeted treatment based on their unique genetic mutations. The trial tests multiple therapies in multiple study arms simultaneously under a “Master Trial” protocol that not only has the power to bring new therapies to acute myeloid leukemia patients faster, but also has the potential to stand as a model for future clinical trials. The trial has already generated strong results, showing superior survival rates and better quality of life when genomic analysis is used to match patients to targeted therapies. For more information, visit ClinicalTrials.gov.
This substudy within the Beat AML® Master Clinical Trial would not be possible without the support of donors, including Emily and Neil Kishter, Judy and Charlie Lynch, and Drenda Vijuk.