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Clinical Trial Provides New Treatment Options for Older AML Patients

By William G. Blum, MD
Director, Acute Leukemia Program, Winship Cancer Institute of Emory University

In September 2018, a 74-year-old woman was referred to Winship Cancer Institute of Emory University after her long-standing, well-managed, myeloproliferative neoplasm, specifically JAK-2 mutated polycythemia vera, progressed to acute myeloid leukemia (AML).

Given her age and medical history, her local oncologist did not believe she would do well with conventional therapy. He sent her to the Leukemia Program at Winship for advice on the best next step.

At Winship, treatment options were discussed in the context of her own desires and priorities, her medical history and examination, and the disease features that were known at that time. She was not a good candidate for aggressive chemotherapy or stem cell transplants at Winship at Emory. In fact, her difficult disease had no promising conventional treatment.

A New Clinical Trial Option

Another option, however, was a clinical trial with an innovative new approach. The BEAT AML clinical trial (sponsored by the Leukemia & Lymphoma Society) consists first of genetic characterization of the leukemic cells in each AML patient (sampling blood and bone marrow). Then, once results are available the following week, the patient and physician carefully consider available novel trials based on the molecular aberrations found in that single patient’s AML as well as conventional approaches. Additional clinical trials are also part of the BEAT AML overall approach, as together with the genetic testing, BEAT AML offers clinical trials in several different genetic subsets of AML.

With advances in genetics, it is possible to genetically characterize each AML patient, at least for common mutations. We can begin to move beyond one-size-fits-all therapies. All patients who enter the BEAT AML trial are screened for genetic mutations that may be driving their disease, then when possible, each patient is offered a therapy specifically designed to target a lesion. If no molecularly targeted therapy is available, alternative clinical trials are offered instead.

As a leader in using and developing new drugs for AML and other blood and bone marrow cancers, Winship at Emory was one of 14 academic research institutions chosen to participate in the BEAT AML trial.

Electing to Enroll in the BEAT AML Trial

After considering her options, she chose the cancer clinical trial described above. First, she consented to sending her blood and bone marrow for this specialized testing. A week later, genetic testing found several specific abnormalities in the leukemic cells, including a mutation of a gene called IDH2. She agreed to the clinical trial targeting this abnormality and was promptly placed on an experimental, oral targeted therapy.

Prior to her arrival at Emory’s Winship Cancer Institute in September 2018, she had (accurately) been told that she had perhaps three months to live with standard approaches. Two years later, until September 2020 she remained in remission and required no further therapy until February 2021.

Advances in Genetics, Individualized and Targeted Treatments

This case illustrates Winship at Emory’s strengths as a clinical and research institution and as a partner with referring hematologists/oncologists in the region. The Leukemia Program at Winship sees approximately 150 new AML patients each year, while following hundreds more. All new patients undergo genetic testing to characterize their leukemia and allow individualized treatment whenever a targeted treatment exists.

Some patients participate in one of the 15+ clinical trials currently underway at Winship (with 50+ additional trials for other types of blood and bone marrow cancers). Many patients receive new therapies that can be given only at tertiary care facilities like Winship at Emory, Georgia’s first and only National Cancer Institute-Designated Comprehensive Cancer Center. Many patients are treated in conjunction with hematologists/oncologists located closer to their homes.

In both research and treatment, the goal at the Leukemia Program at Winship is to improve outcomes, survival and quality of life for patients with AML, especially older patients, like this wonderful lady, who may not tolerate the toxicity and strength of conventional treatments.

Winship Cancer Institute Clinical Trials

Winship at Emory is currently participating in more than 400 interventional clinical trials for patients with a wide range of cancers, both early and late stage.

Since Winship is the only National Cancer Institute-Designated Comprehensive Cancer Center in Georgia, our clinical research portfolio contains innovative cutting-edge trials not offered elsewhere in the state.

To find clinical trials at Emory’s Winship Cancer Institute, visit or call 1-888-WINSHIP.

Winship Cancer Institute of Emory University is committed to the continued health and safety of all patients. During this time, we are taking all necessary precautions to screen for COVID-19 and to prevent its potential spread. We continue to monitor the evolving COVID-19 situation and are working with experts throughout Emory Healthcare to keep your patients safe. For the most up-to-date information for our referring partners, click here.

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