New Biological Insights and Recent Therapeutic Advances in the Management of Acute and Chronic Leukemias and Myelodysplastic SyndromesClinical landscape of emerging research in MDS
2:12 minutes.
TRANSCRIPTION:
DR STEENSMA: I think it’s fair to say that we are in a lean period in terms of drug development. We have not had a new drug approved for MDS since 2006. That was decitabine. So 9 years. And it’s unlikely that anything is going to be approved in the next year or two. That being said, there are a number of new compounds with activity. We saw at the most recent ASH meeting activin receptor antibodies that are being developed in different diseases, patients for whom an ESA had failed or was unlikely to work because their epo level was over 500, had over 40% hemoglobin response rate. Small numbers, early phase study, but that’s promising. What we have learned in the last 5 years is a lot more about the biology of this disease. And drugs like azacitidine, decitabine were shots in the dark. There was some rationale for using them in various hematologic malignancies, but it’s not really understood how these agents were working in MDS. But they are effective in a subset of patients. We didn’t know how lenalidomide worked when it was approved in 2005, and finally, it looks like, have figured that out within the last year. Now that we’re learning about recurrent gene mutations, there’s more than 30 genes that are recurrently mutated in MDS, and novel pathways like the spliceosome or cohesins, which are involved in chromatid separation during cell division. This at least helps us, perhaps diagnostically, perhaps prognostically, ultimately in terms of development of novel therapies. So in sum, I think biology has come a long way in the last 4 to 5 years, but treatment has not. And that’s a bit of a frustration, I think, for everybody. |