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Research Week 2018: Expanding options for patients with an aggressive form of leukemia

A new clinical trial offers alternatives
Uma Borate, M.D., M.S.
Uma Borate, M.D., M.S., is working to expand treatment options for patients with an aggressive form of leukemia. (OHSU Foundation/Sarah Jenks)

One drug for seven days, one drug for three days. This treatment, aptly referred to as “7 + 3,” has long been the standard of care for patients with acute myeloid leukemia, or AML. 

Uma Borate, M.D., M.S., an assistant professor of medicine (hematology and medical oncology) in the OHSU School of Medicine and the OHSU Knight Cancer Institute, says the “7 + 3” treatment has been a mainstay for about 50 years. For a type of leukemia with many different genetic drivers, Borate and other leukemia experts agree the “one-size-fits all” approach needs an update.  

And researchers across the country, and at OHSU, are doing just that.

Like “weeds on a healthy lawn”

In order to understand why treatment options for AML have historically been limited, it’s important to understand how the disease manifests. Borate uses a metaphor to help illustrate how leukemia cells grow and crowd out normal cells.

“I liken leukemia cells to a ton of weeds on a healthy lawn,” she says. “If you don't do something about the weeds, the weeds will take over and collect all the nutrition and water the grass needs to grow. The weeds then eventually crowd out the healthy grass, similar to how leukemia cells crowd out healthy blood stem cells.”

She says patients with AML are not able to make their own blood, including white cells needed to fight infection, red blood cells that carry oxygen, and platelets that help with clotting when you have an injury.

According to the National Cancer Institute, a branch of the National Institutes of Health, an estimated 21,380 new cases of AML were diagnosed in 2017. While the disease doesn’t affect as many people as breast, lung and prostate cancers, Borate says it is highly aggressive and can quickly become fatal. And because the disease typically comes on quickly, physicians need aggressive therapies like “7 + 3.”

Not your typical clinical trial

Borate, who also directs the OHSU Knight Cancer Network, says AML patients have more reason to hope than ever before.

“Suddenly, in the last couple of years, we’ve made leaps and bounds in the different options we have both on the market and in clinical research that we can offer patients,” says Borate.

She says a typical clinical trial starts with a pharmaceutical company looking to advance a particular agent for a certain type of cancer. The trial is designed and executed by the pharmaceutical company, with the ultimate goal of getting the drug to market. Borate says it is an effective model in many cases, but “there’s an obvious bias.”

The Beat AML trial — sponsored by The Leukemia & Lymphoma Society, or LLS — takes a different approach.

The Beat AML Master Clinical Trial

AML has many different genetic changes that lead to the development of the blood cancer, Borate says; consequently, each trial has a specific genetic mutation it’s targeting.

“The goal of this study is to identify the genetic changes that lead that person to develop AML. We then want to give them a treatment that targets that particular change or mutation,” she says.

“LLS communicates with various pharma companies to contribute drugs to help us conduct these clinical trials. What LLS wanted to do is have the physicians and academic centers design the study, then they advocated with pharma companies to say, ‘This is what we think we need to do for our patients; are you willing to be a partner in this endeavor and help us do this?’”

Borate says it’s a very novel method that allows academic health centers like OHSU and others across the country to have more control in designing the study from a patient-centered perspective. A typical clinical trial studies one drug or one combination of drugs; this trial tests several different treatments, with the patient directed to a therapy based on their particular genetic mutations.

“We’re using the standard of care, but adding newer, more targeted agents to the standard of care,” she says. “Those agents are different based on the patient’s specific genetic make-up.”

Shaping future clinical trials

Borate believes that improvements in genetic technologies have led to expanded clinical trial options for patients.

“I think there’s a huge thrust right now, because finally, after all these years, we have such a good understanding of the genetic landscape that causes people to develop AML,” she says. “We understand like we never did before all the different mutations that can lead to AML. Once we understood these mutations then we have an idea of what we can target, instead of chemotherapy which just wipes out everything.”

Borate says that of the 15 to 20 different AML trials underway at OHSU, many are under the large umbrella of the Beat AML Clinical Trial, a two to five-year project that launched in 2016. Results will be shared publicly at the end of the study.

“I don’t think there’s ever been a more exciting time for the treatment of AML in the last 40 years,” Borate says.




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