What’s The State of Immunotherapy, Transplantation in Hematologic Oncology?

The Seventh Annual Miami Cancer Institute Immunotherapies Summit for Hematologic Malignancies, hosted by Baptist Health, saw several presentations and sessions dedicated to informing hematologists, oncologists, radiation oncologists, pharmacists, and other members of the multidisciplinary team about key advances in the use of immunotherapy and transplantation. Following the program, symposium director Guenther Koehne, MD, spoke with CancerNetwork^® about notable themes and takeaways from these presentations.

Koehne, deputy director and chief of Blood and Marrow Transplant, Hematologic Oncology and Benign Hematology at Miami Cancer Institute, highlighted various developments in sessions geared towards the management of multiple myeloma, leukemia, lymphoma, and other blood cancers. He also detailed current work and potential future initiatives focused on improving outcomes among patients undergoing transplantation for their diseases.

CancerNetwork: Can you speak to the importance of having a meeting like the Miami Cancer Institute Immunotherapies Summit for Hematologic Malignancies as it relates to hematologic oncology research?

Koehne: The Miami Cancer Institute Immunotherapies Summit for Hematologic Malignancies is essential to have the lead physicians around the world or within the US in one room and update them on the rapid development of changes in the field. This has been [the seventh meeting]. If we compared the presentations and the status for treatments of these hematologic malignancies from 7 years ago to today, nothing is the same any longer. That’s why this conference is important to keep the physicians and staff members beyond physicians—APPs and lab staff—up to date with the rapid developments in the field.

Sessions on the first day of the meeting included presentations dedicated to areas like multiple myeloma, B- and T-cell malignancies, acute leukemia, and myeloproliferative disorders. Were there any standout discussions or presentations from the first day of the meeting?

I should point out the high quality presentation of Saad Usmani, MD, MBA, FACP, FASCO, from Memorial Sloan Kettering Cancer Center, who highlighted the rapid development of introducing quadruplet induction therapies to start treatment for patients with multiple myeloma, to perform an autologous stem cell transplantation thereafter, and then put the patients on maintenance therapy for a certain time period.¹ It is also an important and novel development in the treatment of multiple myeloma: to limit the time of maintenance therapy to 2 years if it’s good-risk myeloma, or many more years after that in comparison to the previous [treatment] of patients with myeloma, [which was] keeping them on maintenance therapies for as long as they tolerate it or for as long as they do not progress.

There’s a big change in the management of this one. In addition, the development, integration, and detection of minimal or measurable residual disease [MRD] leads to a limited time to administer maintenance therapies after transplant or after immunotherapies. It [also] allows us to interfere earlier if MRD returns after having been in complete remission. That’s a very important development.

I would also like to emphasize the presentation of Paul Richardson, MD, [who] I personally asked to focus on because there is a lot of novel developments, including the CELMoDs, in the treatment for patients with multiple myeloma [who] underwent immunotherapies with bispecific antibodies or CAR T cells.² These CELMoDs prevent T cell apoptosis or T cell exhaustion; with that, the duration of the immune response can be [improved].

In lymphoma, change has been significant for diffuse large B-cell lymphoma. In the induction therapy, the CAR T cells have replaced stem cell transplantations. All in all, that led to new developments in the field of lymphoma. In leukemia, [as] highlighted by Richard Stone, MD, from Dana-Farber Cancer Institute, there have been lots of changes there.³ The 7+3 induction chemotherapy has been the standard for more than 40 years, and that is changing rapidly. Stone highlighted that 7+3 may be history, and another combination of either azacitidine [Vidaza] and venetoclax [Venclexta] or 7+3 plus venetoclax will likely be the new standard.

Wendy Stock, MD, from the University of Chicago, updated us on the new developments for patients with T-ALL, which is also rapidly improving.⁴ This has been a difficult-to-treat disease in the past, and with novel approaches, it’s more promising. She also presented, for the first time, on new updates on CAR T-cell therapies for patients with T-ALL.

The second day featured talks pertaining to CAR therapies and stem cell transplant, among other topics. What were the highlights from day 2 of the conference?

Michel Sadelain, MD, PhD, presented enormous improvements in the next generations of CAR T cells, in terms of duration of the CAR T cells and the duration of the function of CAR T cells in the blood and the site.⁵ But most importantly, for the first time, he talked about CAR T-cell therapies for solid tumors, which is in development. It would be another breakthrough development in the field if CAR T cells can be specifically generated for specific solid tumors.

The Dana-Farber team—Robert Soiffer, MD, and Corey Cutler, MD—talked about improvements in allogeneic stem cell transplantation [allo-SCT] and the management of and/or prevention of acute and chronic graft-versus-host disease, which is also rapidly improving with novel treatment approaches that we didn’t even know about 2 years ago.^6,7

Are there any clinical investigations you are currently involved with that you would like to highlight? How might these efforts improve patient outcomes across different patient populations?

I am heavily engaged now with a focus on patients with very high-risk acute myeloid leukemia. We know about TP53-mutated AML, or even more bialylic TP53-mutated AML. That disease has been very difficult to control. Even allo-SCT does not necessarily provide the outcome that we hope for in patients who relapse and have a short duration of overall survival thereafter. I started to focus on the novel approach to hopefully improve the outcomes for these patients, although we had to overcome certain limitations to develop immunotherapies for AML. In contrast to multiple myeloma or lymphoma, the AML cells express surface markers that are shared with healthy tissue. Therefore, it’s very difficult to specifically target the AML cells unless you have a way to downregulate markers on healthy tissue, leaving the AML cells with the marker expressed, which can be targeted specifically.

In short, there is a marker called CD33 that is expressed on hematopoietic stem cells but is highly overexpressed in the leukemic cell population. If you want to target the CD33 on the leukemic cell, you will also eliminate the function of the hematopoietic stem cells. The novel approach that we developed is to downregulate the expression of CD33 on the hematopoietic stem cell so that the transplant product is CD33 negative but [includes] selected stem cells based on CD34 markers; you can infuse these stem cell products after the chemotherapy. We could show in recently published data in [JCO Precision Oncology] that the recovery of the cells after this transplant shows monocytes or myeloid cells that are all CD33 negative, leaving an only CD33-positive AML population.⁸

Now, we have immunotherapeutic approaches with monoclonal antibodies or donor-derived CAR T cells that can eliminate the residual leukemic cell population, or even to get the patient back into remission upon relapse. That’s very exciting and a bit complex, but we have to go through the complexity to help these patients in the long run. I’m excited about this opportunity, and [I] will likely continue to intensely focus on this patient population.

Looking ahead, how must the field evolve with respect to the use of autologous and/or allo-SCT in leukemia, MDS, multiple myeloma, and other lymphoproliferative diseases?

Autologous stem cell transplant itself is basically limited to patients with multiple myeloma who still benefit from autologous transplant. The integration of autologous stem cell transplant for these patients is still the standard of care. The donor-derived or allo-SCT are the ones performed for patients with [AML], [MDS], and myeloproliferative disorders. We now have risk stratification for all these diseases [in] deciding whether the patient will benefit from transplantation. You don’t want to overtreat a [patient with] relatively good-risk MDS or relatively good-risk [AML] with an [allo-SCT] if the [adverse] effects would basically reduce the outcome.

But if it’s a high-risk disease or intermediate-risk disease, patients still benefit from an allo-SCT, and [it] provides the only curative approach for these diseases... The question was, "Do the patients need the transplant?" Yes, they do need the transplant. Now, the focus is, "How can we improve the outcome of these transplants [by] improving progression-free and overall survival, but also improving and maintaining quality of life?"

What do you hope others take away from the Summit overall? If there was a central theme conveyed at the meeting, what would it be?

My personal recommendation to healthcare providers would be—and that’s the theme of this meeting and beyond—stay on top of the developments and continue to learn [how] the new standard of care has rapidly and significantly changed over the last few years. That’s one of the take-home messages: that you do not miss out on the novel treatment approaches, which induce a better outcome. But as I mentioned with the immunotherapies, yes, they have [adverse] effects, but they are all controllable. Those [adverse] effects don’t compare to what patients had to go through with chemotherapy a few years ago and had to recover [with] a limited quality of life for a long period. That has changed significantly. It’s not necessary only to improve outcomes. If you have a good outcome already, then the focus should be, "How can we maintain the outcome with good quality of life?"

References

1. Usmani S. Maintenance therapy post autologous transplants: does MRD status affect the treatment choice? Presented at the Seventh Annual Miami Cancer Institute Immunotherapies Summit for Hematologic Malignancies; March 6-7, 2026. Miami, FL.
2. Richardson P. Integration of CELMoDs into treatment approaches for multiple myeloma. Presented at the Seventh Annual Miami Cancer Institute Immunotherapies Summit for Hematologic Malignancies; March 6-7, 2026. Miami, FL.
3. Stone R. AML 2026: is 7+3 still the standard of care? Presented at the Seventh Annual Miami Cancer Institute Immunotherapies Summit for Hematologic Malignancies; March 6-7, 2026. Miami, FL.
4. Stock W. Overcoming treatment resistance in T-ALL: translation to the clinic. Presented at the Seventh Annual Miami Cancer Institute Immunotherapies Summit for Hematologic Malignancies; March 6-7, 2026. Miami, FL.
5. Sadelain M. Next steps in CAR T-cell therapies. Presented at the Seventh Annual Miami Cancer Institute Immunotherapies Summit for Hematologic Malignancies; March 6-7, 2026. Miami, FL.
6. Soiffer R. Donor and graft source optimization for allo transplant. Presented at the Seventh Annual Miami Cancer Institute Immunotherapies Summit for Hematologic Malignancies; March 6-7, 2026. Miami, FL.
7. Cutler C. Novel approaches to GVHD prevention and therapy. Presented at the Seventh Annual Miami Cancer Institute Immunotherapies Summit for Hematologic Malignancies; March 6-7, 2026. Miami, FL.
8. Koehne G, Mushtaq MU, Muffly LS, et al. Remission of TP53-mutant AML after transplantation with trem-cel, a CRISPR/Cas9 gene-edited allograft lacking CD33, followed by a donor-derived anti-CD33 chimeric antigen receptor T (VCAR33). JCO Precis Oncol. 2025;9:e2500556. doi: 10.1200/PO-25-00556