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Newest Advances in Stem Cell Transplantation Offer Promise for Complex Hematologic Malignancies


Guenther Koehne, M.D., Ph.D.

Guenther Koehne, M.D., Ph.D.

New stem cell transplantation research conducted by physicians at selected stem cell transplant centers and at Miami Cancer Institute, part of Baptist Health, is showing promise in the fight against relapse, a leading cause of death in patients with hematologic cancers. At the European Hematology Association 2023 meeting in June, Guenther Koehne, M.D., Ph.D., deputy director and chief of Blood & Marrow Transplantation and Hematologic Oncology at the Institute, presented initial results from a first-in-human study in patients with acute myeloid leukemia (AML).

“Patients with very complex acute myeloid leukemia who have relapsed, typically have poor outcomes,” Dr. Koehne says. “So, we are very excited that the initial results of this innovative approach are promising.” The study included a small number of AML patients. While initial results are bringing hope to patients and the healthcare team alike, more patients need to be enrolled onto the trial to confirm these results, Dr. Koehne says.

The VBP101 (NCT04849910) trial, still open for enrollment, involves CRISPR gene-editing technology, deleting CD33 protein that is also found on the surface of leukemia cells, but also on the normal blood-producing stem cells. By eliminating the protein on the stem cells with this technology, there is a chance to specifically target residual leukemia, expressing CD33, with the donor’s immune system following a stem cell transplant.

“The manipulation of the cells is very innovative and can only be performed at institutions that have sophisticated labs such as ours,” Dr. Koehne explains.

Over the past few decades, the landscape of blood cancer treatment has undergone a remarkable transformation, thanks to a deeper understanding of the molecular and genetic basis of blood cancers and developments in immunotherapy and targeted therapies. New, innovative lab techniques have bolstered progress. Improved patient outcomes, reduced toxicities, fewer side effects, and a better quality of life for those facing these complex and challenging cancer diagnoses are the result.

Among the most revolutionary therapies for hematologic malignancies is stem cell transplantation, which had its beginnings in the 1950s. As the technology has advanced, what was once believed impossible ? a cure for many and for others a much longer survival ? is now possible. Allogeneic transplant is, today, a curative therapy for a variety of blood cancers, including many cases of acute myeloid leukemia (AML), acute lymphoblastic leukemia (ALL and myelodysplastic syndromes.

“In addition to improving outcomes, we are now able to able to offer treatment to much older patients who would not have qualified for transplantation in the past,” Dr. Koehne says.

Moving forward, he says, it’s important to remain focused on key aspects that greatly affect outcomes, including:

  • Conditioning regimens – to reduce pre-transplantation toxicities and complications
  • Graft-versus-host disease (GVHD) risk – to lessen the chance of a life-threatening complication through the use of graft manipulation or post-transplantation cytotoxin administration
  • Relapse – to enhance relapse management by integrating maintenance treatments and early management at the first sign of minimal residual disease or of relapse, as well as potential infusions of CAR T cells derived from the original or from a third-party donor as an approach to prevent or treat relapse post stem cell transplant
  • Infectious complications – to reduce the risk of viral infections an reactivations, using antimicrobial prophylaxis, by continuing close monitoring and administration of anti-viral specific immune cells

Efforts to tailor treatment to the individual must also remain a priority, he says. “Just five years ago, we treated all patients with AML as a relatively homogeneous group. We now understand that there are differences based on molecular profiling and, based on risk stratification, we personalize treatment.”

With the complexities of treatments with novel agents, particularly with immunotherapeutic approaches, and with rapidly emerging data, it is through collaboration that the most significant gains can be achieved. Join the experts at the Miami Cancer Institute Global Summit on Immunotherapies for Hematologic Malignancies, March 8-9, 2024, at the JW Marriott Brickell, 1109 Brickell Ave., in Miami, to learn more. More information will be forthcoming soon.

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