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Leukemia & Lymphoma Society announces launch of genomic profiling–based trial for AML
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The Leukemia & Lymphoma Society today announced the launch of a groundbreaking clinical trial that will employ the use of comprehensive genomic profiling to change the approach for acute myeloid leukemia treatment.
Despite advancements in cancer research, the standard of care to treat AML has changed little in 40 years, according a press release issued by Leukemia & Lymphoma Society. The Beat AML Master Trial is designed to identify specific therapies for patients based on the biological or genetic features of their disease.
“When I started my medical training in the [1980s], we treated patients with AML with standard chemotherapy, which remains the same and the treatment outcomes remain the same,” Brian Druker, MD, director of Knight Cancer Institute at Oregon Health & Science University, said during a press conference. “We needed to apply some of the same principles that we applied to chronic myeloid leukemia to AML. But, we also recognize that AML is a far more complicated disease than CML. By combining different agents from different classes, we think we actually provide the foundation to bring new drugs into the treatment for AML. So we’re delighted to be driving the science behind this and accelerate progress against this deadly disease.”
AML is the most lethal of the blood cancers, which together are the third-leading cause of cancer death in the United States. AML is responsible for more than 10,000 deaths each year and has a 5-year survival rate below 20% for patients aged older than 60 years. Nearly 74% of all patients diagnosed with AML die within 5 years, according to SEER statistics.
The Beat AML Master Trial was announced Monday by Vice President Joe Biden as part of the national cancer moonshot initiative report. The trial will be conducted across multiple centers and include academic researchers, pharmaceutical companies, a genomics company, a clinical research organization and multiple arms of patients.
With support and guidance from the FDA, the Master Trial uses an innovative precision medicine protocol in which comprehensive genomic profiling is used to identify specific AML genetic mutations in newly diagnosed patients aged older than 60 years. Based on results of that profiling, an investigational drug or drug combination is administered to best attack the specific molecular mutations causing the cancer.
Typically, most patients with AML are rushed into standard treatment immediately upon diagnosis. In the Master Trial, newly diagnosed patients will have their genomic data analyzed within 7 days and will be entered into the appropriate study arm.
“Currently, when AML patients are diagnosed, they often get a call from their doctor and immediately go to the emergency room,” John Byrd, MD, D. Warren Brown chair of leukemia research at The Ohio State University Comprehensive Cancer Center, professor of medicine and medicinal chemistry and director of the division of hematology at The Ohio State University Wexner Medical Center, and a HemOnc Today Editorial Board member, said during the press conference. “The diagnosis is often rushed and they’re given an intensive chemotherapy course and a great majority of patients are not cured and left with little hope for long-term survival and a lot of side effects from chemotherapy.”
Studies have shown that outcomes are no different whether patients are treated immediately or after a period of stabilization, Byrd said. Further, many patients who had survived AML reported having “almost a posttraumatic stress disorder” from having to move forward with therapy so soon.
“This study is very different in that [the patients] are stabilized,” Byrd said. “After the diagnosis is made, a sample is sent to a very renowned reference lab, where the exact mutation that defines the subtype of leukemia is determined. During those 7 days, the patient is given time to get affairs in order, learn more about the disease that they have to fight and get ready for battle, rather than run into battle before they are prepared.”
The trial will launch initially at five leading cancer centers — The Ohio State University Comprehensive Cancer Center, Memorial Sloan Kettering Cancer Center, Knight Cancer Institute at Oregon Health & Science University, Dana-Farber Cancer Institute and Massachusetts General Hospital Cancer Center.
Four biopharmaceutical companies — Alexion, Boehringer Ingelheim, Celgene and Gilead Sciences — are participating in the trial and offering the following investigational drugs: samalizumab (ALXN600, Alexion Pharmaceuticals), BI 836858 (Boehringer Ingelheim), enasidenib (AG-221, Agios and Celgene) and entospletinib (GS-9973, Gilead Sciences). Other pharmaceutical companies and additional treatment arms should be added to the study over time, according to the press release.
The first patients are expected to be enrolled by December 2016. The Leukemia & Lymphoma Society anticipates that 500 patients will be treated on the trial for 1 to 3 years.
“Ultimately, the goal of this trial, over time, is to give multitargeted drugs and move away from chemotherapy in as many patients as we can while preserving the chance of long-term remission,” Byrd said.
The Master Trial is unique in that as a neutral party, The Leukemia & Lymphoma Society coordinates the collaboration of multiple pharmaceutical companies, allowing different subtypes of AML to be treated simultaneously, according to the release.
At the recommendation of the cancer moonshot blue ribbon panel report, the trial protocol will include patient-reported outcomes.
“This is about showing the way for other blood cancers, about matching patients to a specific treatment based on the biological or genetic features of their disease,” Ross Levin, MD, director of Memorial Sloan Kettering Center for Hematologic Malignancies, said during the press conference. “Within 7 days we’re going to bring patients treatment for their disease, and we think this can really show the way about how such a trial can be done.” – by Chuck Gormley