Fedratinib

[Paul123456]

This webcast from January.

Fedratinib was successful trialed as a JAK2 inhibitor and possibly could have been of great benefit to patients who didn’t respond to Ruxo or for whom Ruxo stopped working. However trial was suspended due to health scare re side effects.

Has now been sanctioned for use again and since there is already significant clinical data re efficacy, could be available soon. At the moment I believe Ruxo is the only first line treatment for MF. Hence anyone having a problem (incl falling Platelets) with Ruxo has few options. However there are now several other drugs in the near term pipeline and only time will tell which treatment options are the most superior.

patientpower.info/video/a-n...

The survival time statistics re MF are now historic and mostly pre date Ruxo, these new drugs should dramatically improve quality of life and buy time for more game changing breakthroughs.

My normal hem retired this week, he send me text saying don’t panic! The difference between now and 10 years ago is amazing and the science improving every year.

[pj1963]

Another interesting and positive nugget from you, Paul. Many thanks.

Paula

[Ovingite]

Thanks Paul. It's good to keep up with drug development.

[Meatloaf9]

Thank you so much for keeping us informed. A positive attitude is invaluable.

[Mazcd Partner MPNVoice]

We have noted a couple of threads recently on the forum. Just a few comments below:

Fedratinib:

This drug was reported initially to be associated with risks of Wernickes encephalopathy. Recently Profs Harrison and Mesa reviewed the data which led to new data reducing these concerns.

The status of fedratinib is awaited from licensing authorities.

Concern about whether JAKAFI might accelerate new mutations?

A provocative article has been published on MPNforum. This reviews data showing that patients with mutations in genes other than JAK2 CALR MPL responses appear shorter and if patients develop new mutations on drug and stop the drug they do not live long.

However we have also seen that patients rarely develop new mutations, and have 2 large studies showing that JAKAFI prolongs life in patients with any mutation profile. Much other data is also consistent with patients having a better quality of life and longer life on JAKAFI.

Please bear in mind that the well written article is provocative but does not prove any risk of JAKAFI worsening disease. Do not suddenly stop taking your drug on the basis of this article.

Metformin

There has also been some speculation about whether adding metformin to ruxolitinib might be beneficial. This is on the basis of non human work - mice and cells in the lab only. It is exciting provisional data. This will probably be properly tested in a clinical trial in the future.