Four new treatments for acute myeloid leukemia to watch

There are four new treatments for acute myeloid leukemia (AML) that have recently been approved by the FDA.

That’s a huge breakthrough, considering that there were no new drug approvals between 2000 and 2017-and there wasn’t a commercially-available new drug for the treatment of AML available for the past three or four decades because the approved drug was withdrawn from the market, says Bruno Medeiros, MD, associate professor of medicine (hematology) at the Stanford University Medical Center and director of Cancer Center Infusion Area Treatment Services at Stanford Cancer Center.

It’s likely that at least one more AML drug will be approved next year, and maybe more, he adds.

Because of the momentum around these new drugs for the treatment of AML, Managed Healthcare Executive (MHE) recently spoke to Medeiros about how the breakthroughs are affecting patients with AML. 

MHE: As a physician who treats patients with AML, why are you enthusiastic about the four new-and impending-drug approvals? What impact could these treatments have on patient care?

Medeiros: The approval of these and future new agents is very exciting as it should translate into an improvement in the long-term survival for patients with AML.

The statistics are important for how to interpret clinical trial data, but if I can tell a patient that they have a 5%, 10%, or 20% higher chance of being cured and never have to deal with the disease again, that’s really very palpable. That really is the implication of these novel therapies, that you can cure more patients with this otherwise deadly disease.

MHE: Do you notice any specific trends or similarities between these newly-approved and future AML drugs that are likely to be approved by the FDA?

Medeiros: Several classes of novel drugs have been approved, but some patterns can be detected, such as the approval of tyrosine kinase inhibitors, small molecule inhibitors, and immunotherapies.

We’ve had Midostaurin approved for the treatment of patients with newly diagnosed AML who are FLT3 mutation-positive (FLT3+). There are two very large randomized clinical trials that are very close to completing accrual for patients with relapsed or refractory mutated AML-and it’s possible that one or both of those would be positive trials, which would significantly impact those patients.

Enasidenib was approved by the FDA for the treatment of IDH2-mutated AML patients in a relapsed or refractory setting. There’s also a very similar clinical trial that completed accrual earlier this year for IDH-1-mutated AML patients in a relapsed or refractory setting, and it’s anticipated that that drug will receive approval by the FDA.

In an earlier stage of development, there are some immunotherapies for AML, and they fall within the category of drugs similar to Gemtuzumab, which is one of the three drugs that were approved earlier this year as well.

MHE: If you could design the best possible way to partner with payers on streamlining the introduction of such new drugs into patients’ care paths, what would that look like?

Medeiros: Patients should be treated in centers that have experience with large volumes of patients. An example of this is with centers of excellence with bone marrow transplants. There’s a level of expertise that you get out of centralizing specific aspects of leukemia care.

This is a system that works in European countries. For example, there are only five hospitals in Denmark that provide care for patients with AML. Everyone is referred to those hospitals because they can maintain a level of expertise in the optimization and the use of new drugs and technology that really allows them to optimize care for these patients.

Another example is Kaiser Permanente, which has decided that only one of three Kaiser institutions will treat patients with acute leukemia. That’s to minimize cost, optimize quality of care, and ideally improve patient outcomes.

MHE: What does the future hold for these and treatments for patients with AML?

Medeiros: These new drugs improve outcomes for patients who are alive at the end of their treatments. Hopefully, the development of novel treatment options sets the bar higher for how many patients can be cured with these diseases and, consequently, that translates into an improvement in patients’ outcomes in general.