Miami Cancer Institute

Two Clinical Trials at Miami Cancer Institute Investigate Novel Approaches for the Treatment of Acute Myeloid Leukemia

April 15, 2024

MIAMI, FL – April 15, 2024 – Researchers at Miami Cancer Institute, part of Baptist Health South Florida, are currently conducting two clinical trials using novel therapeutic approaches to treating patients with acute myeloid leukemia (AML), a form of blood cancer that can be aggressive or classified at high-risk based on certain molecular structures detected within the leukemic cell population. Patients with high-risk AML are at risk of developing disease recurrence. Therefore, novel and innovative approaches are needed to improve their outcomes.

Patients in these two trials undergo initial chemotherapy followed by an allogeneic (donor-derived) stem cell transplantation. The first trial, which recently started enrolling patients, is using CRISPR technology to down-regulate a donor’s gene as part of a treatment regimen in those patients with high-risk AML who are at risk of relapsing after transplant. The second trial is using CAR-T cells – a form of immunotherapy – generated from the stem cell donor to specifically target the leukemic cell population at time of disease recurrence following the stem cell transplant. 

“We have made significant progress in the treatment of leukemias in the last decade,” said Guenther Koehne, M.D., Ph.D., deputy director of Miami Cancer Institute, chief of blood & marrow transplantation and hematologic oncology, and co-investigator of the studies. “The results of this latest research has enabled us to deliver more sophisticated and customized care and to treat many individuals who remain at risk of leukemia recurrence.”

One of the most common leukemias in adults, AML is diagnosed in approximately 20,000 patients in the U.S. each year. About half of those who receive a hematopoietic stem cell transplant go on to relapse and their prognosis is poor. Koehne and his team have developed a unique technique to manipulate donor cells in the lab to reduce the risk of graft-versus-host disease (GVHD), a serious, sometimes life-threatening complication of transplantation.

At the recent Miami Cancer Institute Global Summit on Immunotherapies for Hematologic Malignancies in March, Koehne, the symposium’s director, shared the latest updates from the principles on CAR T-cell therapies in AML patients as well as the early results on the CRISPR technology based clinical trial that recently started enrolling AML patients – “Allogeneic Engineered Hematopoietic Stem Cell Transplant (HCT) Lacking the CD33 Protein, and Post-HCT Treatment With Mylotarg, for Patients With CD33+ AML” (NCT04849910).

“Through CRISPR, which is a gene-editing technology, we remove CD33 from healthy cells so that the leukemia cell is now the only cell producing CD33,” Koehne said. “The resulting transplant product, known as tremtelectogene empogeditemcel, or trem-cel, protects the healthy cells and allows drugs to better target and kill the cancer cells. Early results in eight patients so far are promising.”

Miami Cancer Institute is the only Florida trial site for both of these AML trials, which are both enrolling patients.

About Miami Cancer Institute

Miami Cancer Institute brings to South Florida access to personalized clinical treatments and comprehensive support services delivered with unparalleled compassion. No other cancer program in the region has the combination of cancer-fighting expertise and advanced technology—including the first proton therapy center in South Florida, Latin America and the Caribbean, and one of the only radiation oncology programs in the world with each of the newest radiation therapies in one place—to diagnose and deliver precise cancer treatments that achieve the best outcomes and improve the lives of cancer patients. The Institute offers an impressive roster of established community oncologists and renowned experts, clinical researchers and genomic scientists recruited from the nation’s top cancer centers. Selected as Florida’s only member of the Memorial Sloan Kettering Cancer (MSK) Alliance, Miami Cancer Institute is part of a meaningful clinical collaboration that affords patients in South Florida access to innovative treatments and ensures that the standards of care developed by their multidisciplinary disease management teams match those at MSK.

Miami Cancer Institute is part of Baptist Health Cancer Care, the largest cancer program in South Florida, with locations from the Florida Keys to the Palm Beaches.


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