New paper on current understanding of MF and impact of mutations

[Paul123456]

Bit hard work but very interesting update re MF

nature.com/articles/s41408-...

[socrates_8]

Thanks Paul...

Steve

[Cookiebaker]

This maybe a great paper but it is way over my head. I am very interested in the mutations.

[Miriammusic]

Paul this was amazing. Changed my thinking with regards to ‘remission ‘ and managing this disease. Thank you for sharing all of your info with us.

[Aneliv9 in reply to Miriammusic]

Can i ask you in what way your thinking about remission and managing the disease changed? You mean that you understand now that these two meanings are different?

[Miriammusic in reply to Aneliv9]

Hi! Yes certainly that. And also just a different mindset. Sometimes I get down and feel burdened, as one can with a chronic disease. I am tolerating my treatment and am very grateful for that but the interferon is not a picnic. But there was something in the paragraph under treatment standards that stood out to me. The part that says “disease management is focused on the relief of symptoms and the improvement of quality of life”. So that rather than focusing on a molecular remission or some magic number in general, I should probably focus on treating my symptoms and managing this condition. Much in the same way that a diabetic does. The journey not the destination maybe? Learn to “manage” my disease. Does that make sense? Sorry so long!

[Aneliv9 in reply to Miriammusic]

I think you were clear. But i can't understand if this disease management does give you a survival advantage, so you have not to worry about remission..

[stillkicking]

Thanks Paul, it looks fascinating. I'm glad that there are people around who actually understand this stuff... reading it was a bit like listening to music..., for me it was mostly abstract, but every now and then there were little glimpses of something that almost made sense! I must have another read/listen and see if I start to comprehend some more!

[Bluetop]

Thanks Paul.

[jointpain]

Thanks Paul, anybody know why Anagrelide is not mentioned in the whole paper? My lovely wife who has MF jak2 pos. Is on Hydroxy and Anagrelide to keep her platelets in check. The combination is extremely effective, and keep them to mid normal range. Very difficult reading though.

[Paul123456 in reply to jointpain]

You may find this interesting since just switch to Peg from HU

bloodjournal.org/content/ea...

Best Paul

[Aneliv9]

I read the article and i would like to ask you,if you could point a finger on any treatment that seems the most promising or disease modifying except vaccines that are only for Calr MF.. i have been confused,cause even a drug that could reverse bone marrow fibrosis,seems that is not enough..

[Paul123456]

My very limited understanding is that there are new drugs for MF such as AVID, I believe now in Phase 2 trials, that are looking promising and other drugs that appear effective for improving fibrosity alone. However this latter group requires a combination with another drug to limit the ‘proliferation’ element of our disease. Otherwise fighting an uphill battle.

I don’t know whether interferons are a suitable combo drug, they appear to be disease modifying on a molecular level for some people.

[Aneliv9 in reply to Paul123456]

What do you think a drug should achieve in order to be considered as a drug that controls the disease progression? Px have an impact on number of cancer cells ( is this what proliferation means ?)

[Aneliv9 in reply to Paul123456]

In theory, if we had a drug that would reverse fibrosity with a drug that would limiting proliferation, we would have halt of the disease??? Why am i so confused about all that drugs??? It seems to me that while so many drugs are tested and have an impact on fibrosis, or proliferation, this disease can't be modified significant... Am i wrong? Also maybe i cannot exactly understand all the meanings of the words cause i am from Europe

[Paul123456]

You are right. Outright cure still seems a while away but we appear to be getting close to disease slowing drugs and, if able to reverse fibrous, we can buy even more time. Hence pull back more advanced patients

The consensus seems to be that such a drug/combo will be available within 5 years.

Pegasys appears to be the best bet at the moment but only a subset achieve a good molecular response. And we don’t have any long term clinical date. Some trials are trying to spread its efficacy to a wider group of patients.

The great news is that lots going on and Phase 2 trials now looking better risk:reward if advanced MF

[Stella57]

Thanks for this. I should be more active in keeping up with new research, but I'm pleased that you can share on this forum.