Is anyone involved in clinical trials for ET, specifically for CALR?

Hi Lori. For any trial there are certain criteria that has to be met, some of these will be result of what has been seen previously and protocols are quite black and white due to FDA oversight. For this particular trial I had to have extensive blood work (11 tubes for some 20+ assays, counting cbc and cmp as one each even though each measured multiple items), a bone marrow biopsy, spleen ultrasound, ekg, and a general health review. In all theses they are looking for sufficient evidence to include or exclude. Fortunately, I live near the facility so is typically about a 45 min drive depending on Denver traffic 😒.
Patty noted a bit different in our conversation, but I have been told will be a 72 hour hospital stay for each titration dose - those being at least 3 visits each a week apart, maybe more depending on tolerance. As a phase 1 study they are looking mostly at safely and efficacy of dose as these are the first administrations in humans. Following that it will be administered every 3 weeks with a full day at the clinic.

@drbart86

What a brave and noble thing you are doing, drbart86!

Everyone here thanks you and cheers you on!

@janemc thanks for the thoughts. I'm no different from any other suffering with an illness. All have their battles to face daily. I'm fortunate that I am near a research location and am able to do this. I also have very supporting friends, family, and medical staff. Those that don't have this are the truly brave ones, often dealing with this alone and in silence.

Thank you, Patty and drbart!! We are all grateful! Do you have to sign non-disclosure agreements? If not, I hope you will report out how it's going!

@drbart86

I must disagree. I would think of a million excuses not to participate.

I salute and thank you.

Day 1 completed! (Last Tuesday)

Took my first dose of immunotherapy for my cancer in a phase 1 study. I must commend the great staff at UCHealth; all have been super caring and very attentive to my needs. Today was the first dose and multi-day hospital stay; this will continue for the next several weeks, and once a safe and efficacious dose is found, there will be several years to follow.

Also, to the great work that cancer organizations do to support cancer patients. For me, Blood Cancer United (LLS) has been a key part of my journey over the last 4 years, because of a conference, I made contact with a great specialist that was local (when you have a very rare disease, there are not that many specialists, and they are typically at strong research facilities). BCU has also led me to encounter others with my class of disease - the kind of support and understanding is truly indescribable. (Please support these organizations; even a few dollars can be super meaningful.)

And of course, the pharma companies that take the risk to develop efficacious compounds are heroic in many ways. For me, there are only 3-4 approved compounds to treat symptoms (not a cure, as that is not an option yet), and at least 2 of these have not worked for me. This trial may be the closest we can get, but there are still so many for whom hope is fleeting.

The predosing protocol is quite interesting, especially mainlining benedryl 🥹. It burns going is so they administer slowly - I was asleep before they were done! Great for a several hour sleep.

Start again on Mon evening for pretests, administration on Tues and (assuming all goes well) home Thurs.

Hi @drbart86 Thank you for this update and your participation into this Clinical Trial for ET. Cancer research and development depends on these trials…along with the long awaiting cancer patients who would love to find a cure or remission from disease!
So far so good, as ‘they’ say, eh? I can relate to the mainlining of Benadryl! The best sleep I ever had. 😳 But also wired for hours later. LOL.

Sending positive vibes for smooth sailing with the next round. Keep us posted! Hugs.

@loribmt didn't get the wired part. I still wondering if somebody got the license of the Mack truck. 😂

Not in any trials, but I have been treated for 12 years for ET as a result of a CALR Exon 9 mutation. I've been treated with Hydroxyurea (500mg) and, as of the beginning of June, I am experimenting with the possibility of replacing the Hydroxyurea with unsweetened cranberry juice. On July 3, I will know the efficacy of the cranberry juice in maintaining a normal platelet count. If successful, then I will continue to use the cranberry juice instead of Hydroxyurea. The reason for this is two-fold: first, I believe in using food instead of drugs to treat or even eliminate illness (I successfully ended obesity and Type 2 diabetes, starting in 2007, by eating a paleolithic diet, for example) and, secondly, because of the side effects of the Hydroxyurea, which includes anemia because the Hydroxyurea destroys some of my red blood cells. If I'm able to stop using the Hydroxyurea, then its side effects will no longer be a problem and my red cell count can return to normal. This won't, of course, do anything to stop the CALR Exon 9 mutation, which I'll have for the rest of my life, but then, neither can the Hydroxyurea. All the drug does is suppress platelet count. If I can do that naturally with food, that's an improvement.

@drbart86 You made me laugh out loud this morning with your Mack Truck comment. I totally get the visual AND the feeling! Hang in there, you’ll survive this…though I still have some tread tracks on my body from the big tires. 😂