MDS updates from iwMDS-iwMPN 2024 | Ongoing research in the NIH Myeloid Malignancies Program

My name is Alain Mina. I’m a staff clinician at the National Institute of Health with a focus on adult myeloid malignancies and stem cell transplantation. The goal of my talk was to tell you a little bit about the Myeloid Malignancies Program. It’s a relatively new program, but I think it’s becoming increasingly more powerful and far-reaching. It stemmed from the decision, the pivotal decision, to harness the intramural research capacity of the National Cancer Institute, the NCI, back in November of 2020; and since then, we have been able to initiate a number of different collaborative efforts that manifested in several symposia, three NCI National symposia, one NCI FDA workshop to put out there key consideration and recommendations for clinical trial design in myelodysplastic syndromes. And most recently, an effort we’re really proud of, is the NCI Translational Workshop that happened pretty recently, in the last couple of months. 

All these different collaborative endeavors are at the service of patients. Our goals are not only to serve patients but community oncologists, to collaborate with the scientific community, and build a strong clinical trial portfolio in order to do so. This portfolio will focus on the spectrum that is MDS, be it the low-risk, the high-risk, the de novo, and the relapsed/refractory, all the way into the transplant, and a personal interest to me, the post-transplant relapsed setting. 

We have a number of clinical trials that have been initiated over the last couple of years, some of which are the eltanexor or the XPO1 inhibitors in combination with decitabine in higher-risk MDS, the pacritinib in relapsed/refractory MDS, be it the low-risk and the high-risk, and that protocol covered the pediatric and the adult. We have our natural history protocol for not only RUNX1 germline, but also for pediatric and adult suspected MDS, diagnosed MDS and CCUS. Other protocols are more focused on transplant, be it the myeloablative engineered graft stem cell transplant for CD33-positive relapsed/ refractory AML and high-risk MDS, both in the pediatric and the adult, in patients with matched sibling and matched unrelated donor. Another protocol we’re proud of is going to be the palifermin combined with post-transplant cyclophosphamide. It is a reduced-intensity regimen for any patient who is not eligible for myeloablative transplantation, but it will be of particular interest and benefit to our patient population with MDS who have increased comorbidities, are at an increased advanced age, and whose GvHD is a kind of the main transplant-related mortality they have to deal with. So this combination of palifermin/PTCy should theoretically annihilate a lot of toxicities. 

And then finally I’d like to mention our autologous CD123 engager T-cells. This is part of conditioning or induction. This is an autologous effort that came with collaboration with outside institutions, and will hopefully be opening up soon in patients with relapsed/refractory CD123-positive AML. In conclusion, I’d like to thank the organizers here and our team at the NIH and the MMP. The NIH is a neutral ground for academic endeavors and collaborative efforts. We are dedicated players, team players, and we are excited for more and more collaborations down the road.