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MPN Workshop of the Carolinas 2024 | Telomerase inhibition as a novel targeted therapy for myelofibrosis
Rami Komrokji, MD, Lee Moffitt Cancer Center and Research Institute, Tampa, FL, discusses the potential of telomerase inhibition as a novel targeted therapy for treating patients with myelofibrosis (MF). Dr Komrokji mentions imetelstat, an agent which has been approved for the treatment of myelodysplastic syndromes (MDS) with transfusion-dependent anemia. The drug exhibited activity in MF in a Phase II trial and is being tested in an ongoing Phase III trial. This interview took place at the 1st Annual MPN Workshop of the Carolinas in Asheville, NC.
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Transcript
This had been a wonderful meeting. We just had a session on novel therapies. I discussed the role of telomerase inhibition, particularly focusing on data on imetelstat, which is a drug that’s been approved by the FDA for myelodysplastic syndromes, and we were looking at the data in myelofibrosis.
I tried to also highlight the similarities and differences between the two diseases...
This had been a wonderful meeting. We just had a session on novel therapies. I discussed the role of telomerase inhibition, particularly focusing on data on imetelstat, which is a drug that’s been approved by the FDA for myelodysplastic syndromes, and we were looking at the data in myelofibrosis.
I tried to also highlight the similarities and differences between the two diseases.
Imetelstat was tested in myelofibrosis in a Phase II study, where a higher dose reported activity and the lower dose tended to be like the control. It had modest activity, but the main signal out of the Phase II trial and the randomized one, was that it appears that there is a survival advantage of the imetelstat.
So based on that, we have an ongoing Phase III trial looking at overall survival after JAK2 failure, which is actually the only ongoing study that has a primary endpoint of overall survival and the audience in the room actually were excited and applauding that this study is looking at overall survival. We discussed a little bit also the PD data in the study, some of the disease modification suggestion with allele burden reduction and correlation with outcome, and the possibility that the mechanism of action could be different between MDS and MPN.
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