This is a general shout out for info on either ruxolitinib or viral gene therapy For Essential thrombocythemia (ET)? Does anyone take ruxolitinib for Essential thrombocythemia (ET)? I am just on aspirin at the moment.
And I am also very interested to hear if anyone has looked into gene therapy. With modern technology including AI I would think we aren’t far off (as in can probably do now!) altering genetic mutations back to norm. I have JAK2 mutation. Do people know where there there are any gene therapy trials occurring?
Hi I will copy your post to the Blood Cancer UK Clinical Trials page for you Clinical Trials Information and Support | Blood Cancer UK
I hope others will be able to share their experiences.
Please do let us know how you get on.
Take care and really look after yourself
Thank you for sharing! I’m one of the nurses in the clinical trial support team. I’m sure you’ll receive other responses from forum members with their own experiences of Essential thrombocythemia (ET) treatment, but I wanted to share some information that might be helpful. We also have some information on standard treatment options, available here: Essential thrombocythaemia (ET) treatment and side effects | Blood Cancer UK
You may already be aware, but Ruxolitinib is not routinely available for treating Essential thrombocythemia (ET) in the UK, as it is not licensed for this indication. The MAJIC study, a trial looking at Ruxolitinib for polycythaemia vera and essential thrombocythaemia (Essential thrombocythemia (ET)), found that while Ruxolitinib improved some symptoms of Essential thrombocythemia (ET), the trial team concluded that Ruxolitinib was not more effective than other available treatments for people with Essential thrombocythemia (ET) who cannot have hydroxycarbamide. For more detailed information on the findings, you can find the study results here: A trial looking at ruxolitinib to treat polycythaemia vera and essential thrombocythaemia (MAJIC) | Cancer Research UK
For individuals who are resistant to Hydroxyurea, there may be routes to access Ruxolitinib via alternative requests through discussions with their medical team and the pharmaceutical provider. This would need to be explored with a clinician.
Currently, to our knowledge, there are no gene-editing trials available for Essential Thrombocythaemia (Essential thrombocythemia (ET)). While gene-editing treatments like exagamglogene autotemcel (Casgevy) have been approved in the UK for blood disorders like sickle cell disease and beta-thalassemia, these advancements have not yet extended to Essential thrombocythemia (ET). However, there are several ongoing trials for Essential thrombocythemia (ET), primarily aimed at patients who have not responded to, or cannot tolerate, standard treatments like hydroxyurea. Some of these trials target specific mutations (e.g., CALR).
Finally, if it’s helpful, here is a video where some of the top experts in the UK discuss the latest findings and breakthroughs in Myeloproliferative neoplasms (MPN): [Video of the Myeloproliferative neoplasms (MPN) Voice patients’ virtual forum – Video of the MPN Voice patients’ virtual forum – Post ASH 2024 update – MPN Voice
I hope this information is useful. Please don’t hesitate to get in touch if you have any further questions. You can contact our trial support service here: Clinical Trials Support Service | Blood Cancer UK