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New CLL Therapy Showing Promise for AML

By Staff blogger | April 21, 2016

LLS-funded researcher Anthony Letai, MD, PhD, talks about how his work led to clinical trials of venetoclax for acute myeloid leukemia (AML) and a priority review by the FDA, and how these developments could ultimately lead to the first new therapy approval for AML in decades.

An associate professor in medicine at the Dana-Farber Cancer Institute, Letai has been the recipient of several LLS grants in recent years – supported as a Fellow and Scholar, and most recently, receiving Translational Research Program funding.

SUCCESS WITH CLL

We’ve seen some exciting new clinical advances in chronic lymphocytic leukemia (CLL), in particular the spectacular activity of venetoclax, formerly known as ABT-199. The FDA approved the investigational medicine earlier this month for treatment of previously treated CLL patients who are at higher risk, and in January, the FDA granted a second Breakthrough Therapy Designation in combination with rituximab for the same population.

Venetoclax will likely be a key component of CLL therapy moving forward, and your work has played a significant role over the past 10 years.

What has been your motivation and involvement in these developments?

Letai: I am an oncologist as well as a lab researcher, so my greatest motivation has been to figure out a way to do better for our cancer patients.  It is interesting to probe the secrets of cancer biology, but the real gratification comes in actually improving the care of living, breathing cancer patients.  The concept of learning things in the lab to apply them to clinical care seems straightforward, but experience has taught me how challenging it can be.  The support of foundations like LLS that are interested not only in scientific hypothesis testing, but also in real clinical progress, is essential for this kind of translation to the clinic.