Dr. Patricia Kropf

Hematology experts at Novant Health are always looking for better therapies for acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS) to prolong patient survival. "Outside of an allogeneic transplant, if patients have relapsed disease, they have a very poor prognosis," said Patricia Kropf, MD, a hematology physician and principal investigator on several clinical trials at Novant Health Cancer Institute in Charlotte. "The only way to find better therapies is to enroll in a study. The earlier patients are referred, the more options they have with respect to treatment. Patients shouldn't wait until they're on their second-line or third-line therapy."

Novant Health Cancer Institute locations in both Charlotte and Winston-Salem are participating in AML and MDS clinical trials and actively enrolling.

Dr. James Dugan

"After the first-line therapy, the subsequent therapies are not very good," said James Dugan, MD, a hematology/oncology specialist and the physician lead for the transplant and cellular therapy department for Novant Health Cancer Institute in Winston-Salem. "We need to use the data and the resources that we have, and often the resource we have is a clinical trial. We need to move the needle on relapsed refractory AML and MDS."

While there is an NK cell therapy that is open to relapsed or refractory MDS and AML, most active therapies for AML and MDS research at Novant Health are not cellular therapies.

"With AML and MDS trials, most are targeted toward small molecule inhibitors," Dr. Dugan said. "Usually, they're targeted toward a specific mutation. AML has had a large area of research devoted to what is driving the molecular processes of the disease, so we get these next-generation sequencing panels, they come back and there's a handful of mutations for every patient. Everybody's AML has a different fingerprint, so these clinical trials now are trying to capitalize on individual areas of AML."

Here are two examples of actively enrolling trials targeted toward small molecule inhibitors:

  • Novant Health is participating in a trial with an IRAK-4 inhibitor that is open to those patients with a FLT3,N SF3B1, or U2AF1 mutations.
  • Novant Health is also participating in a trial with a menin inhibitor that's open to NPM1 mutated disease or KMT2A gene rearrangements.

"Recently we had a patient that was primary refractory to the traditional chemotherapy, who got onto a menin inhibitor clinical trial and was in complete remission within one cycle," Dr. Dugan said. "Before these types of treatments, these patients either got more chemo or went to hospice. Now they're helping patients get to transplant, or for patients who can't get to transplant, they're helping them have a better-quality life with a greater longevity than they normally would have had."

"We also do a lot of work with epigenetic therapy to decrease aberrant hypermethylation in the genome," Kropf said. "We have a currently enrolling study using VIDAZA and combining it with a new BCL2 inhibitor. Our goal is to determine if this new BCL2 inhibitor is better than the standard of care, venetoclax. We've seen great results, with multiple complete remissions, which are minimal residual disease negative."

Novant Health continues to expand its clinical trial portfolio

"We are being pulled into the world of solid malignancies because more CAR T-cell therapies, more TILs therapies, more cellular therapies are going to start to be used in solid malignancies," Dr. Dugan said. "At Novant Health Forsyth Medical Center, we just did the first TILs therapy in the state for lung cancer. It was a discussion between solid oncologists and malignant hematologists like myself, because those cellular therapies lend themselves from blood cancer."

"We're always adding more trials," Dr. Kropf said. "We have meetings once a month to discuss what trials we have, how they're enrolling, what the outcomes are, and new potential trials. Every year we'll add more studies and change the mix depending on the patient population and the outcomes.

Learn more about clinical trials or enroll a patient. Visit Clinical Trials | Cancer Treatments | Novant Health or