After careful consideration, I decided to join this trial. Since I was diagnosed in 2017 I have been on Hydrea, Interferon and Angerlide. Recently my platelets spiked over the millions so my MPN Specialist, Dr Gotlib at Stanford, suggested this trial. I was hesitant at first but the more research I did, I realized I didn't want to pass up this opportunity. So I muscled through the screening process and was approved. They did notice in my BMB, a slight progression, but my diagnosis was still the same. All the more reason to move forward with the trial. After my first dose, I'm happy to report that my platelets have gone from 1 million to 618,000 and my LDH level went down 50% in just one week. Wow! I've never seen my numbers shift this much. I feel great, just dealing with some fatigue. Because my numbers were excellent, Dr. Shomali (head of the trial) took me off of Anagrelide. What a relief! My next infusion is this Monday and going forward I will receive infusions every 2 weeks. In 6 months I will get another BMB to see if the disease is essentially reversing.
Stanford is looking for more patients to join phase one of the trial. If you live in California, or are willing to travel and are interested in this opportunity, below is the link.
This is one of the first to go to trial. It's for CALR only. Quite exciting to be in and your good early results are great news for CALR's.
As in prior posts, there are several concepts targeting CALR specifically. CALR mutation effects are is exposed on the cell and more readily identified in comparison to Jak2. This report has the earlier work on it.
" the discovery of a mutCALR-specific inhibitory antibody marks an important breakthrough for the field, offering new hope for patients who currently have limited options beyond JAK inhibitors and other cytoreductive therapy."
We now know they may have solved the ID problem for Jak2, so possibly similarly effective drugs are getting started for Jak2.
That's wonderful. So happy it seems to be working well for you and hoping you also see some improvement in that BMB, when it's repeated. Please keep us posted as to how it's going. I'm super curious about this drug and I think it's great that it's been good for you, so far!
I'm hoping there will be something similar available for those of us with MPL mutations. As I understand it, the MPL target is also an extracellular target. But I believe there's not as much research into the mutation because it's not as common. (Hope I'm wrong about that!)
That’s fantastic, I’m so pleased for you, and thank you for sharing, it gives me hope in dark times. Does anyone know if that’s the same phase one trial that is being run by Guys? I thought of volunteering for it but difficult to get to London on a weekly basis, I’m hoping to join it at phase 2. Best wishes Jo
I was offered a trial (not sure which one) but also would find it difficult to get to Guys very often, it’s a pity they don’t spread the trials around the country.
More hospitals across the country are likely to join in running a trial as time goes on. It's also worth asking about what expenses the trial organisers will cover. Travel to and from the trial hospital is very likely to be paid for and maybe even arranged for you.
I'm waiting for the screening process to join this INCA 033989 trial at Christie's in Manchester. Lot of travel involved but I think well worth it, I've been offered door-to-door taxi for each visit! Some of the results from existing trials are very encouraging.
They did say I would probably get some expenses back, I live quite a distance from London and train fares are very expensive, plus I have severe arthritis which affects my mobility a great deal. We have some good hospitals in a 30 mile radius of where I live, it would be great if any of those took part in trials.
I didn't want to assume that all trials are the same. I joined a trial that has opened up outside London, about 40 miles from home. They arrange door to door taxis and refreshments for all the hospital visits.
That’s good! As I was seeing the team at Guys it was only the ones on the go there that I was offered. If I could get one nearby I would definitely consider it. I don’t think my local haematologist would put me forward though! He’s not very interested!
For my trial at Stanford they do cover travel/expenses. There might be a cap on flights but I know there are people flying out for infusions for my trial.
That takes guts and commitment to sign up for a first in human trial so a big thank you from me, also a Calr 1. Here’s willing you on for the best outcome possible and we’ll all be looking out for your updates.
Thanks so very much for sending this update. Can you share a bit more about how you applied for and were admitted to this study? Do you know what specifically they are looking for in those they pick to participate? Again, thank you for sharing.
In my case, I became intolerant to Hydrea and had a hard time with the side effects of Interferon and Anagrelide. My platelets had gone over one million recently, and I've had Von Willebrand Disease in the past. They typically select more high risk patients for phase one, however this how I qualified. My Dr is at Stanford and he and his colleague are head of the trial so this was my ticket in. I will give you the link to the trial as well as my trial Coordinators info. Not sure if you are in the U.S. or near California. See below. Not sure when phase 2 begins (could take a couple of years) but they might open up the criteria more at that time.
I've been on this in London since November. Like you, since being diagnosed in 2014, I'd been tried on Interferon, Hydroxycarbamide and then Hydroxycarbamide and Anagrelide. My platelets have dropped right down and I am feeling good. The staff at Guy's are lovely and I am able to claim for (expensive) train fares to and from the hospital.
Can you elaborate a bit on your experience in a new post? How many doses? How quickly did your platelets drop and by how much? Lots of CALR folks on here would love to hear / know more. 👍🏼
That's great news, I was also on the trial. I have post ET myelofibrosis and didn't get any response at all and only lasted on the trial for six months. My doctor tells me that all the ET patients are having good a response.
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Fatigue 
Improvement in lab results with natural products 
ET and Von Willibrands
Spleen slightly enlarged on the upper side of normal. UPDATE on results.
Feeling disheartened and upset 
