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Can Precision Medicine Help Patients With AML?

Can Precision Medicine Help Patients With AML?
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Published on January 29, 2021

Precision Medicine Proves Beneficial for AML Patients

Patients with acute myeloid leukemia (AML) who participated in a precision medicine clinical trial lived longer than those who elected standard of care treatment, according to a study published in Nature Medicine.

The median overall survival for patients in the Leukemia & Lymphoma Society's (LLS) Beat AML Master Clinical Trial was 12.8 months vs. 3.9 months for patients who received cytarabine plus daunorubicin or a hypomethylation agent. The trial also found that genetic and cytogenetic analysis of leukemia cells to identify AML subtypes can be completed in seven days. That is enough time for patients, care partners and their healthcare providers to make a more personalized treatment decision without risking the patient's chance for survival.

Corresponding study author Dr. John C. Byrd, the D. Warren Brown Chair in Leukemia Research at The Ohio State University, said the results were more than encouraging.

"This patient-centric study shows that we can move away from chemotherapy treatment for patients who won't respond or can't withstand the harsh effects of the same chemotherapies we've been using for 40 years and match them with a treatment better suited for their individual case," Dr. Byrd said in a press release announcing the findings.

He added: “The study shows that delaying treatment up to seven days is feasible and safe and that patients who opted for the precision medicine approach experienced a lower early death rate and superior overall survival compared to patients who opted for standard of care.”

Precision Medicine in AML

AML is an extremely fast-moving cancer. Patients generally receive their first therapy immediately upon diagnosis to keep the cancer cells from growing out of control. That treatment usually involves an infusion of a combination of cytarabine and daunorubicin, or treatment with a hypomethylating agent (such as decitabine or azacytidine) for those patients who are not fit for intensive chemotherapy.

The Beat AML Trial uses advanced genomic technology to identify each patient’s cancer-driving genetic mutations and then matches patients to targeted therapies. This leads to better survival rates than the traditional one-size-fits-all treatment approach, the Nature Medicine study found.

The study reinforces the importance of molecular and genetic testing to identify specific genes, proteins and other factors involved in leukemia, according to Dr. Jessica Altman, associate professor of medicine in hematology and oncology at the Robert H. Lurie Comprehensive Cancer Center of Northwestern University.

“There are some mutations and some set[s] of genetic abnormalities that impact the initial treatment,” Dr. Altman said in a Patient Power program earlier this month. “It both informs prognosis and may impact initial therapy and treatment if the disease fails to respond or if the disease responds and then comes back.”

For example, she said the disease may express an FLT3 mutation, which is present in about 30% of adults with newly diagnosed AML. Midostaurin (Rydapt) is used in combination with certain chemotherapy medicines to target that mutation. Another targeted therapy, gilteritinib (Xospata), is used to treat relapsed or refractory FLT3-mutated AML.

“The importance of assessing patients’ goals and understanding their lifestyle and what motivates them, helps us frame the conversation when there's choice in treatment,” Dr. Altman said. “And we're in an era in acute myeloid leukemia therapy, where there is choice. And we haven't been in that arena before.”

About the Beat AML Trial

The LLS launched the Beat AML Trial in fall 2016 to test multiple novel targeted therapies in newly diagnosed AML patients ages 60 years and older. The trial has screened more than 1,000 patients at 16 cancer centers. The data presented in Nature Medicine represents patient enrollment between November 17, 2016, and January 30, 2018.

For the study, screening and analysis were successfully completed within the seven-day timeline for 374, or 94.7% of eligible patients. Ultimately, 224 of those patients opted to participate in one of the 11 study arms. The patients who chose not to join the study opted for standard of care, palliative care or an alternative clinical trial.

“The study is changing significantly the way we look at treating patients with AML, showing that precision medicine, giving the right treatment to the right patient at the right time, can improve short and long-term outcomes for patients with this deadly blood cancer,” said Dr. Louis J. DeGennaro, president and CEO of LLS, in the press release. “Further, Beat AML has proven to be a viable model for other cancer clinical trials to emulate.”

For more information on the Beat AML Master Clinical Trial, contact the LLS Information Resource Center or call 800-955-4572.

~Megan Trusdell

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