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Clinical Trials at Miami Cancer Institute Investigate Novel Approaches for Treating Blood Cancers

Guenther Koehene, MD, PhD

A new clinical trial awaiting final preparations at Miami Cancer Institute, part of Baptist Health, will test premanufactured blood products that researchers hope will lead to “off the shelf” solutions for some patients who require chimeric antigen-specific T-cell therapy to treat their blood cancers. CAR T-cells provide new treatment approaches targeting hematologic malignancies, specifically to induce remissions for patients with Non-Hodgkin Lymphoma. While CAR T-cells are effective, it takes several weeks to generate these products from the patient’s own blood. With a premanufactured product, patients may no longer have to wait weeks to receive treatment or for the time it takes to collect and manipulate these cells in a lab.

“An off-the-shelf solution would make CAR T-cells obtainable within 48 hours and with a single phone call,” Dr. Koehne said. “This would make a huge difference for patients who don’t have the time to wait.”

The study is one of several being led by Guenther Koehne, M.D., Ph.D., deputy director of the Institute and chief of Blood & Marrow Transplantation and Hematologic Oncology, that may hold the key to the next treatment breakthrough for patients with acute myeloid leukemia (AML), multiple myeloma, acute lymphoblastic leukemia (ALL) and other blood cancers.

Dr. Koehne and his team are recognized for developing 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.

To improve outcomes of patients identified as high-risk for developing disease recurrence based on certain molecular structures detected within the leukemic cell population, there is a need for novel and innovative approaches. Two such trials are being conducted at Miami Cancer Institute to treat patients with AML.

The first trial, which began enrolling patients recently, uses CRISPR technology to down-regulate a donor’s gene. The second trial uses CAR T-cells - a form of immunotherapy - generated from the stem cell donor to specifically target the leukemic cell population at the time of disease recurrence following the stem cell transplant.

“We have made significant progress in the treatment of blood cancers in the last decade,” said Dr. Koehne, who is co-investigator of the trials. “The results of this latest research are enabling us to deliver more sophisticated and customized care and to treat many individuals who remain at risk of leukemia recurrence.”

Approximately 20,000 patients in the U.S. each year are diagnosed with AML. About half of those who receive a hematopoietic cell transplant go on to relapse and their prognosis is poor.

At the Miami Cancer Institute Global Summit on Immunotherapies for Hematologic Malignancies in March, Dr. 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,  “Allogeneic Engineered Hematopoietic Stem Cell Transplant (HCT) Lacking the CD33 Protein, and Post-HCT Treatment With Mylotarg, for Patients With CD33+ AML” (NCT04849910).

“With CRISPR’s gene-editing technology, we remove CD33 from healthy cells so that the leukemia cell is now the only cell producing CD33,” Dr. 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 site for the two AML trials, which are both enrolling patients.

The Summit, in its fifth year, featured prominent researchers, including:

·        Michel Sadelain, M.D., Ph.D., Memorial Sloan Kettering Cancer Center

·        Robert Soiffer, M.D., Dana-Farber Cancer Institute/Harvard Medical School

·        Aleksandra Filipovic, M.D., Ph.D., Imperial College of London

·        Eytan Stein, M.D., and Raajit Rampal, M.D., Memorial Sloan Kettering Cancer Center

·        Marcel van den Brink, M.D., Ph.D., City of Hope

·        Leo Luznik, M.D., Johns Hopkins Medicine

·        Charles Dimitroff, Ph.D., Herbert Wertheim College of Medicine/Florida International University

“It’s an ideal environment for top researchers to share their knowledge,” Dr. Koehne said. “Research is moving very rapidly, and when we bring together the leaders in the field, we have the opportunity to integrate our data, work more efficiently and develop new lines of treatment. Treatment is no longer one size fits all. We are seeing more patients go into remission so that they can have a transplant. We are improving outcomes and survival. Working together, we are making progress.”

For more information on Miami Cancer Institute’s blood cancer program, click here.


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