exciting research: mpnresearchfoundation.org/n... - MPN Voice
exciting research
This is a fast developing technology with the teams working on it making public progress just the last year or so. CALR MPN is likely to be curable for many in the foreseeable future.
These methods take advantage of a unique feature of the CALR mutation: it is exposed outside the cell, as described in that report: "A mutation in CALR (CALR-FS) causes an alteration in the cell surface landscape" So the defective cell can be identified and removed using recently developed methods.
For Jak2 (near all PV and many ET) the ID method won't work because the Jak2 mutation hides within the cell. So the cell cannot be targeted. There are efforts to pre-treat the cells to force the Jak2 allele to speak up. Then they can nail it with these methods. When/if I come across that report again I'll post on it.
One item I think is not correct: "Around a third of MPNs are driven by a mutant protein called... (CALR)." It's about that for the subset with ET/MF, but much less when including PV. So these new methods will not help most MPN patients, but it is a big deal for CALR patients.
got it. Thank you for the info & is good news that there’s all this research going on. Hopefully it’s promising 🤞🏼
Big deal indeed! Thank you very much.
this is potentially wonderful news, I think there will be trials first which I hope I’ll be feeling brave and healthy enough to sign up to. Jo
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