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ESH AL 2018 | Is there hope for treating TP53-mutated MDS?

In this interview, Mark Levis, MD, PhD, of the Sidney Kimmel Comprehensive Cancer Center, Baltimore, MD, speaks about TP53-mutated myelodysplastic syndromes (MDS) and the results of the Phase Ib/II clinical trial (NCT03072043) headed by the MDS Clinical Research Consortium, which showed promising response rates for the treatment of this condition with the mutant p53 activator APR-246 when given in combination with azacitidine. This interview was recorded at the 2018 European School of Hematology (ESH) Clinical Updates on Acute Leukemias, held in Budapest, Hungary.

Transcript (edited for clarity)

Perfectly dreadful disease. I refer to it as liquid pancreatic cancer. At the present time, I don’t think there’s anything that changes the natural history of that disease. I know there’s a lot of excitement about decitabine, and this and that, but remissions in that disease don’t equal survival, necessarily.

There is a very interesting new agent: APR-246, which is being studied in combination with azacitidine, and the MDS consortium is heading up that trial...

Perfectly dreadful disease. I refer to it as liquid pancreatic cancer. At the present time, I don’t think there’s anything that changes the natural history of that disease. I know there’s a lot of excitement about decitabine, and this and that, but remissions in that disease don’t equal survival, necessarily.

There is a very interesting new agent: APR-246, which is being studied in combination with azacitidine, and the MDS consortium is heading up that trial. The results of that were just presented at ACR a couple of weeks ago and they were an extraordinarily high response rate. Again, a response rate doesn’t mean an improve an overall survival, but in this case this is an actual agent targeting the p53 aberrancy itself, so I think that actually warrants enthusiasm.

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