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MPN Workshop of the Carolinas 2025 | The rationale for investigating T-regulatory cell therapy in myelofibrosis
In this interview, Lucia Masarova, MD, The University of Texas MD Anderson Cancer Center, Houston, TX, provides insight into the rationale for exploring T-regulatory cell therapy in myeloproliferative neoplasms (MPNs), specifically myelofibrosis. This therapeutic approach aims to regulate the dysfunctional immune system of patients and enhance the efficacy of currently approved therapies, such as ruxolitinib. Dr Masarova mentions a Phase Ib study investigating an add-on therapy with cord blood-derived, CXCR4-enriched T regulatory cells (CK0804) in patients with myelofibrosis (MF) who have a suboptimal response to ruxolitinib treatment. This interview took place at the 2nd Annual MPN Workshop of the Carolinas, held in Charlotte, NC.
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Transcript
That’s a very innovative study that we launched and started to do in patients with myelofibrosis, currently as an add-on approach to a stable dose of ruxolitinib. There were some first data that came from using regulatory T-cells in patients with inherited bone marrow failure syndromes, which also included patients with myelofibrosis. Then we were kind of encouraged by seeing responses in the cytopenias, so it was taken into the Phase Ib study of exploring specifically regulated or engineered T-regs that have CXCR4 enrichment, so they could home to the bone marrow and then there are six infusions added into patients that have ruxolitinib for myelofibrosis every month, 100 million cells in every single bag...
That’s a very innovative study that we launched and started to do in patients with myelofibrosis, currently as an add-on approach to a stable dose of ruxolitinib. There were some first data that came from using regulatory T-cells in patients with inherited bone marrow failure syndromes, which also included patients with myelofibrosis. Then we were kind of encouraged by seeing responses in the cytopenias, so it was taken into the Phase Ib study of exploring specifically regulated or engineered T-regs that have CXCR4 enrichment, so they could home to the bone marrow and then there are six infusions added into patients that have ruxolitinib for myelofibrosis every month, 100 million cells in every single bag. And then there was a Phase Ib study, so we were looking for safety.
It’s mostly to tackle the inflammation and to see whether we can somehow challenge the microenvironment. Regulatory T-cells are the gatekeepers. They pretty much are playing with the bad players and the good players and somehow regulate the imbalanced immune system in patients with various diseases, for example, autoimmune diseases, neuroinflammation. So that’s MPN, because particularly myelofibrosis, but also other MPNs are kind of a hallmark of inflammation. That’s why we thought that the T-regs, they are being brought to these patients from either healthy individuals or cord blood, as this study has, would help to regulate and navigate the dysfunctional immune system and maybe enhance the efficacy of the current therapies or even help us with the progression. So there are data from previous studies showing synergism with ruxolitinib that could also be employed in preventing infections or adverse outcomes that could come with the therapy, specifically more immune dysregulation, such as the impact on NK-cells, T-regs, or even innate immunity. So that’s why the rationale for using the regulatory adaptive T-cell therapy came from.
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