Anyone heard of Promedior?

[Jennifer1974]

I've been researching clinical trials for MPNs and I came across a drug trial for PRM-151. It's currently in phase II ( looking at safety & efficacy in a larger population) after promising Phase I results. It's some sort of biologic (protein?) that is indicated for fibrosis in the marrow..they are looking to halt or reverse fibrosis. It was originally developed for pulmonary fibrosis but this trial is for myelofibrosis (wondering if it could be applicable to other mpns as well) Just curious if anyone knew anything about it. It appears all the trials are in the US. If something like this could be developed perhaps it could be used in conjunction with Jakafi to help those whose disease has progressed?

[akonline]

Hi Jennifer, yes that's a promising new drug. I think Prof Harrison at Guys & St Thomas' is running the trial too! Worth checking. Good luck!

[eire]

Just read up on the trial - it certainly sounds very promising. I'm due to see my haem at the end of Sept and will ask her opinion. This drug is infused but looking at the literature I read it's not given daily. Great that there is such great progress with this disease when I was first diagnosed 7 years ago there was not the same amount of hope as there is today. I have no doubt it will come to Europe if it's successful.

[beetle]

I am just in the process of moving from Scotland to Devon and once there I've been offered a place on the trial. I have all the info about it but my partner is not that happy about drug trials. I am hoping to get an appointment for us both to discuss with Claire Harrison once we've settled in

[eire in reply to beetle]

They do seem a bit scary after all they are trials. The way I look at it is that they want you to live as they are investing a lot into your progress and really wouldn't offer you a place if they didn't feel you're a good prospect. I've been on my current trial nearly 4 years now and my life certainly has improved. It's a huge decision but as long as you're armed with all the info you can make an informed decision. Very best of luck to you with the move from one end of the country to the other and hopefully your partner having her mind put at ease when she attends your appointment.

[Jennifer1974 in reply to eire]

What trial are you on if I may ask?

[eire in reply to Jennifer1974]

Ruxolitnib and panobinistat have no idea of the name of the trial. I take 15mg of rux twice daily and 25mg of pan 3 times a week every other week. My spleen has stopped shrinking but at least it's not growing!!!!! The pan is quite severe on my poor old tum and I need to take lots of Imodium to relieve the discomfort!!! Fair play to you doing all this research and sharing it with us yes totally agree ' keep hope alive' !!

[Jennifer1974 in reply to eire]

Sorry about your stomach!

[beetle in reply to eire]

Yes, I see that viewpoint but a nasty little voice keeps asking me if they really are investing in my progress or in making money for themselves. Then another little voice reminds me that no drug would come to market if nobody was willing to enter trials! I will have to see which little voice shouts the loudest in due time as I am at present not doing too well on Ruxolitinib. I'll let you know the ultimate decision😏

[Jennifer1974 in reply to beetle]

Thanks for your insight.

[eire in reply to beetle]

That's a hard one to answer as its entirely feasible!!!! What a shame rux isn't helping such a big worry for you both. From posts on this page I reckon you couldn't see a better person than Claire Harrison she's very highly thought of. That's the best we have really the advice and trust we need to place in the hands of our haems and our own instincts. I wish you all the very best making your decision which I have no doubt will be very well informed. Yes please do keep us all informed as we are all very interested in each other's decisions. Likewise when I see my haem in dublin at the end of Sept maybe I'll have a little bit of insight too as I intend to ask her opinion.

[Irishmod in reply to eire]

Hi, i see from your mail you live in Ireland and attend haem in Dublin do you mind me asking which hospital you attend and if the haem specializes in MPN's, I attend Tallagh hospital have ET. It is not often you see anything from Ireland so I am curious about your reply especially as you say your on a trial I did not think anyone in the south of Ireland had this option available.

[eire in reply to Irishmod]

Hi Irishmod, I attend St. James. Eibhlin Conneally is my haem she is excellent. St. James are constantly involved in clinical trials though the trial I'm on is now closed. I live in Sligo but my haem there asked Eibhlin if she would see me with a view of my suitability for a trial. Long way to travel but I take a good book!!! That was 4 years ago and I'm doing really well. I met a couple of patients with MF but no one with ET. I have an appt at the end of Sept and can ask the question for you??? Your haem at Tallagh would definitely know her and could offer advice. Let me know what I can do to help.

[Irishmod in reply to eire]

Hi Eire, thank you so much for you reply and your offer to speak to Eibhlin anything you can find out would be great. When I was diagnosed just over 4 years my platelets went to 1700 and my haem put in Tallaght put me on hydrea and aspirin I then asked my own doctor to refer me to Eibhlin Conneally as I had researched and found she was MPN specialist. I had a one consult at the time as she just said she agreed with the treatment plan. Part of me would have liked to change as she is the expert but I have to say Tallaght haem is very nice but not experts in MPN in January he did change my medication to Anagrilide as Hydrea gave me loads of probems. I cost me 144 euro each month and wonder if I should be on Long Term Illness just to get my drugs free I don't need anything else. It would be good to find out if any trials available with Eibhlin for ET patients and I'm lucky I live in Kildare so Dublin very convenient for me. At the moment the hospital is finding it difficult to control my platelets now have me on a mixture of Hydrea and Anagrlide to see if this will work its been a long 4 years to get them into normal range. If you can find out anything that would be great, All the best for now

[eire in reply to Irishmod]

Hi Irishmod, sorry for my late reply. There are no clinical trials at James at the moment for ET or PV I asked for you at my consultation. James is going to become top hosp for cancer care so who knows what is down the line. Your consultant will be fully aware of new trials when they come along I'm sure. If you did get on a trial your drugs would be free but other than that they're means tested. My clinical trial drugs appear not to be working anymore so it's back to the drawing board!!!!!! My spleen doubled in size in a very short time - so uncomfortable!!!! I look permanently pregnant not a good look at my age!!!!! Seems your haem at Tallagh is on top of your situation and all the haem's have meetings etc to discuss best options available. Good luck.

[Irishmod in reply to eire]

First thank you ever so much for finding out if any trials available and hopefully something in the future I will keep on checking.

That is not good news at all for you at all and must be worrying as to what options are now available for you? what drugs were you on? I do think you are lucky to be in James hospital as you are dealing with an MPN expert.

What do you think they will now do?

I know you live in Sligo which is a long way away from Dublin so if you ever need a place to stay overnight at any time in the future and especially if you have to go back the next day or early morning appointments you are more than welcome to stay with myself and my husband we have loads of room and I know you have never met us but you and I have something in common so I'm sure we would all get to know each other. My offer is genuine and would like to help if possible.

Take care and let me know how you are getting on.

Thanks again for you help

Maura

[eire in reply to Irishmod]

Oh gosh Maura you are so very good making such a generous offer. Words just fail me - that doesn't happen very often!!!!!!! My spleen doubled in size, the fibrosis is in my now enlarged liver and to top it I have gallstones!!!! I was in Sligo hosp and they told me they could take out the gallbladder but Eibhlinn has told me it's too dangerous. When the ultra sound was done my spleen took up 2 monitors the biggest they've ever seen. My claim to fame??????? There are no other options in James but she's going to contact Claire Harrison in Guys about the Promedior. Scary times I just was not expecting that on my Dublin day out. It's a very stressful waiting game for me at the moment and all so very sudden. I was so shocked didn't ask any questions but have been given the wonderful offer of being seen in dublin anytime. If it wasn't for your post about Promedior I wouldn't even have that hope as James had never heard of it!! Anyway I remain hopeful there is so much going on in the MPN world medically at the moment so fingers crossed!!!!!!!

I'll keep you posted and again many thanks for your more than generous offer.

Pat

[Irishmod in reply to eire]

Hi Pat

The offer has no closing date so if you ever need B&B just let me know.

You really are having a hard time I have never heard of someone with such a large spleen and it is unfortunate that the drugs no longer working I really hope Claire Harrison will have some suggestions she is the best and really cares for her patients and the MPN community in general and gives her time freely. I have attended a number of the patient forums organised and got loads of information from the different groups. I really feel we have to keep ourselves informed and to bring it to our consultants attention otherwise we could be losing out.

I will be thinking of you and hope you get on another treatment option will you please keep me posted on your progress? In Ireland I suspect we are a small group and especially in the South in Belfast they are much better organised.

Take care

Maura

[eire in reply to Irishmod]

Thank-you so much Maura I truly appreciate your kind offer. Yes you are right it is up to ourselves to be as informed as possible. I attended a forum in dublin 4 quite a few years ago it was run by Eibhlinn and others. People came over from the UK to help and advise also a senior consultant from Belfast hosp. Her name escapes me but all questions were answered it was just so informative. I hadn't done much if any research into my disease and was genuinely horrified I had the worst of the MPN's!!!! My head is well and truly out of the sand now!!!! I've had no further updates as yet but I live in hope. I'm feeling really good at the moment which is great as its my d'ters grad next Thursday and I have my 2 other d'ters coming over from Wales. Something nice to look forward to.

Thanks again.

Pat

[Jennifer1974]

I'm just beginning my journey with ET Jak2+ but I'm always thinking about the future. Why wait until our poor bone marrow gets scarred before doing anything about it? I hate the uncertainty of it all! Beetle, I wish you the best of luck. It sounds like the drug is a protein that is naturally present in our bodies (don't know all the details) so maybe there's limited side effects? Also I'm assuming that not all my stem cells have the JAK2+ mutation..couldn't immune system be modified to identify the clone and destroy it? More research is needed...I know here in the states the FDA has a " fast track" program for drug development for.diseases/conditions that really need treatment NOW instead of the years and years it typically takes for approval. One could argue that there are more risks involved with this approach..but the MPN life is risky business anyway! Keep hope alive!

[Paul42 in reply to Jennifer1974]

I must admit i had those thoughts myself when i was diagnosed.

I had thoughts along the lines of i know stem cell transplant is considered curative, why wait until im really sick before trying it, surely if i did it now when fit and healthy i would stand a better chance of it working. Obviously i know from research that its considered curative but doesnt always work, can make you very sick etc.

I am on Rux (Jakafi) and I know some early results showed fibrosis had halted or even reversed in some people, i am not sure if there is any further evidence of this though.

This new drug looks great, but i fully understand why MF patients get trialled first (like they did with Jakafi), because they are at most risk, then PV patients come next and unfortunately in most cases ET patients last, its just because the conditions carry the most risk in that order.

PV patients only have a 15% chance of progress to MF, and ET 10% so you have to weigh the up with the risks associated with any treatments.

I do think we will get to the point where these new drugs we are talking about become the prime treatment for all patients, but we are some way off that, and cost will be a massive factor to.

[Jennifer1974 in reply to Paul42]

I do want my brother screened as a stem cell donor but pretty certain insurance won't cover it. 10-15% seems pretty high to me considering I've already survived thyroid cancer at 26 (rare at that age) and now this ....I have to hope for the best but prepare for the worst. I remember being told at 26 that thyroid cancer was the best cancer to get and it wouldn't affect my life expectancy...at 41 I'm told I have an acquired mutation in my stem cells but it shouldn't affect my life expectancy. How do you accept no control over your body? It's hard for me. If JAKAFI (Rux) may stop progression then why wait? I do think there may be at trial for JAKAFI for ET patients. And what about Pegasys interferon? I've read about molecular remission for some ET patients. I don't think it is approved for treatment of ET here in the US. And yes it comes down to cost. My mother's Enbrel (biologic) used to treat her rheaumatoid arthritis is thousands of dollars a month and insurance doesn't cover all of it.

[Paul42 in reply to Jennifer1974]

You are right there is a trial on Jakafi for ET patients. Not sure how its going but before i started it my Platelets were usually high end normal, and even had been slightly over 450, they reduced slowly and have balanced out around the 190-200 mark now for the past 9 months.

I read too about Pegasys interferon and remission for PV patients but i think this is very very rare, we are talking a handful of cases out of hundreds of thousands.

[Jennifer1974 in reply to Paul42]

My platelets are low 600s lately..just aspirin for now. I'm going to my next hematologist appointment this coming Friday armed with all of these questions. I also wonder if I hadn't found out by "accident" during my prenatal visit whether I would have had any symptoms. I felt pretty good until they told me something was "abnormal" 😜

[Paul42 in reply to Jennifer1974]

Plenty of people dont find out unless by accident, one woman i met with ET has counts of about 3500 but only found out when giving blood.

You are right to ask questions, i did and still do. Try and look at your blood counts on the screen or even get a print out. You may find they concentrate on your platelets but this will tell you if there are any other discrepancies too.

If there are any then you can pose the questions about those too, hopefully there wont be.

[Jennifer1974 in reply to Paul42]

My Dr said at some point I may switch to PV...assuming it's cause my JAKD status

[Paul42 in reply to Jennifer1974]

With MPN's a lot of people sometimes have overlapping symptoms, a lot of PV patients cant have elevated platelets etc.

I know its not easy not to worry, but the majority of people do stay 'as they are' and do not progress to anything else.

And progress in the past few years treatment wise has been fantastic, we are both young and i really wouldnt be surprised in 10-15 years time if we arent looking at a drug which totally controls the conditions that is readily available.