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CU Cancer Center study leads to FDA approval of new treatment for AML

CU investigators did early work on venetoclax, recently approved for older acute myeloid leukemia (AML) patients

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Written by Cancer Center on November 10, 2020

Thanks in large part to early work by investigators at the CU Cancer Center, patients with acute myeloid leukemia (AML) have a new treatment option that has fewer side effects and has been shown to increase longevity.

On Oct. 16, 2020, the Food and Drug Administration granted regular approval to the drug venetoclax (Venclexta), in combination with a low-dose chemotherapy treatment, for the treatment of AML in older adults who are unfit for intensive chemotherapy.

The drug works by targeting and inhibiting the Bcl-2 protein, a key component of leukemia stem cells. Of 33 patients given venetoclax in a 2018 clinical trial at the CU School of Medicine, 20 experienced a complete response (aka complete remission) and eight experienced a complete response but with continued low blood counts. Of the three patients who did not respond to treatment, two discontinued the study before the first week due to personal reasons unrelated to treatment or side effects.

The FDA granted the drug preliminary approval based on those clinical trials, which were conducted by CU Cancer Center researchers Craig Jordan, PhD, and Daniel Pollyea, MD, MS. Though they weren’t involved with the phase 3 trial that led to the full FDA approval in October, the two still are researching the drug to determine how it could be even more effective.

→ Learn more about the venetoclax clinical trial with 91% response rate against newly diagnosed AML in older adults

The CU Cancer Center sat down with Pollyea to talk about the drug and his and Jordan’s role in the FDA approval.

Q: You presented your original research on venetoclax in late 2018 — what was the path from there to the drug being used elsewhere, and ultimately to the full FDA approval?

A: That’s part of the amazing story about this. The uptake of this by the leukemia community has been so incredibly rapid — this is now the standard of care, and it is so widely accepted by the community. So many patients now diagnosed with AML get venetoclax as some point in their treatment journey. It really has touched the entirety of this disease in a way that we haven’t seen anything in the decades that people have been working on this disease. It’s been almost an immediate overnight improvement in what we can do for patients.

Q: AbbVie, the developer of the drug, originally created it as a treatment for other types of leukemia. What role did the CU investigators play in getting them to consider it for AML?

A: Craig published his data back in 2013, and that really helped convince everyone that this could work in AML. We and others worked hard to get this clinical trial off the ground.

Q: What was it like to work with patients in the clinical trial, and to see the drug’s effect on them?

A: It’s incredible. The patients who volunteered to be on the clinical trial, who were the pioneers that led the way, are amazing people to be able to have that kind of altruism to volunteer their bodies and their lives for a hope — that things will be better for them, but a certainty that we would learn from their experiences and the field would improve. That’s an incredible feeling, and as soon as the drug got approved, prescribing it for the first time, that was an amazing thing, too. To see that because of the work that we had done, it translated into the ability just to write a prescription, and to know that that was the same thing that any oncologist in the country could do because of this work, that’s an amazing feeling.

Q: You are still conducting research on the drug; what are your next steps?

A: We have a couple of different studies with a bunch of different goals. One goal we have is to improve the regimen, and we have some insight into how we can make it even better. Another goal we have is to expand it beyond just the patients who are not good candidates for standard intensive chemotherapy. We think that that’s overly limiting, and that this has a very broad application throughout this disease. We also would like to make this more user-friendly, and that goes into the development of an all-oral regimen that we are going to be pioneering here very soon. Finally, a big part of what we’ve been working on for the past couple of years is trying to understand those patients who don’t respond to this and developing therapies for those patients specifically to try to improve their outcomes as well.