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This is why you should be excited for upcoming cancer research


Acute myeloid leukemia experts share promising new developments on the oncology horizon.

It’s an exciting time for cancer research, say oncology experts.

Understanding how acute myeloid leukemia (AML) occurs, as well as investigating new therapies to treat the disease, can help scientists derive new insights that can be translated into other disorders, says Bruno Medeiros, MD, associate professor of medicine (hematology) at the Stanford University School of Medicine.

“If you’re able to optimize the utilization of these therapeutic interventions for patients with acute leukemia, for example, you could potentially translate that into different modalities that can be used in a broader patient population,” he says.

One of the areas of particular interest to Medeiros, an expert with the American Society of Clinical Oncology, is delving into how leukemia occurs. This research is valuable because it will enable researchers to understand the biology of cancer transformation, which is the first step in trying to develop novel treatment strategies.

He’s also hopeful about recent research into the genomics of how and potentially why leukemia develops, which will be valuable for providers to improve outcomes for patients.

Other developments

Also promising is research into therapeutic areas, such as chimeric antigen receptor T-cell (CAR-T) therapy. While only limited effectiveness data are available in AML, it’s promising based on the data that’s been presented for other but similar diseases, says Medeiros. CAR-T therapy has been approved by the FDA for acute lymphoblastic leukemia (ALL). There’s hope that CAR-T therapy can be translated into effective treatment regimens for AML, he says.

CAR-T therapy is also currently being tested in a number of different solid tumors, he adds. For example, the treatment regimen is currently being tested on ovarian cancer by Stanford researchers.

Thus, Medeiros says that a strategy that was originally developed to treat a relatively limited number of patients can be potentially used on the treatment of other solid tumors. ALL occurs in fewer than 6,000 people every year, and, thus, a fraction of the number of patients who present with far more common cancers of the breast, colon, lung, or prostate. 

Gene editing is another area that is being researched for the management of myeloid malignancies, he adds. “There’s a unique opportunity for these strategies to apply to hematological malignancies. There’s hope that early recognition of advanced myeloid disorders-before transformation-and potentially preventing those from happening with gene editing,” Medeiros says.

Two drugs have shown great promise in treating FLT3-mutated AML, says Robert Hromas, MD, chair and professor of the department of medicine at the University of Florida (UF) College of Medicine and leader of the UF Health Cancer Center Cancer Genome and Epigenome Integrity research program. FLT3 is a protein that’s mutated in about a third of AML patients.

They are:

• Midostaurin (Rydapt), was FDA approved in April 2017 for the treatment of adult patients with newly diagnosed AML who are FLT3 mutation-positive (FLT3+), as detected by an FDA-approved test, in combination with standard cytarabine and daunorubicin induction and cytarabine consolidation.

• Quizartinib, an investigational small-molecule receptor tyrosine kinase inhibitor, shows promise as a monotherapy for patients with FLT3-ITD-positive relapsed AML, and Hromas, an expert with the American Society of Hematology, describes the agent as “likely just as good [as Midostaurin]”

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