NCA033989 Trial - 3rd Infusion Update (ET+CALR1)

[Zeppelin11]

Good morning! I'm happy to report that I had my third infusion last week, and my platelets are at 265,000 and seem to be stabilizing. My WBC, RBC, and LDH levels are normal, and my RDW is almost within the normal range as well!

Currently, my regimen is 750mg every two weeks, and I'll continue this indefinitely. Before the trial, my platelets were over 1 million. Over the last three weeks, as my numbers have normalized, I've noticed a significant increase in energy, a decrease in spleen size (I don't get full as quickly when I eat), and reduced brain fog, leading to more clear-headedness.

My next infusion is a week from tomorrow, at which point I will provide another update. I also plan to ask my doctors for the latest information on the trial, especially now that they've been approved to start patients at a higher dose, given the lack of significant side effects observed so far.

I'm very pleased with the results thus far. I try not to get ahead of myself, but I've been through 3 medications in 8 years and haven't seen these numbers since before I was diagnosed. Hoping for regression in my BMB in August, however I know this will take time. Chat soon!

[dbus1417]

awesome news. Thank you again for sharing and participating. 🥳

[hunter5582]

Great news! Thanks for keeping us up to date on this promising new option.

[EPguy]

The new stage of the trial you refer may be "Experimental: Part 1b" in the "study plan" of:

clinicaltrials.gov/study/NC...

This is a good sign there are no signif safety signals in the cohort. If your results are typical this agent is likely to qualify for a set of FDA procedures, below, that can bring it to market faster since it looks to address some currently unsolvable issues. (assuming the FDA is allowed to keep functioning)

--

Types of special FDA status:

Priority Review

Breakthrough Therapy

Accelerated Approval

Fast Track

fda.gov/patients/learn-abou...

[socrates_8]

Hey Zeppelin11...

Those results are sounding nothing short of spectacular...

In Australia, similar CTs are at a similar stage, as I understand things...

Being CALR (2) myself naturally I am also most interested in the progress of these Trials...

Thanks so much for Posting! Wishing you ongoing & continued success, which appears on the surface to be remarkable outcomes. Thanks for keeping us all in the loop too...

Best wishes

Steve

(Sydney)

[ETket]

It all sounds like it's going really well!

[CanadaG]

amazing results! This is very encouraging, thanks for sharing, I'm glad to hear it's working well for you.

[Mtnlife]

wonderful news. Thank you for keeping us posted on the progress.

[ETinNYC]

That's amazing news Zeppelin! I hope the bone marrow results also yield some positive news. It would be truly amazing if this drug not only reduced platelets but also had effects on the bone marrow. Do keep us posted! Hope you continue to feel well on this medication. Hugs from NYC!

[ETinTX]

So good to hear! I'm entering a clinical trial at MDAnderson April 7 for a bispecificic antibody treatment for CALR. I am CALR type 2 and have had crazy swings in my platelets. I'm cautiously optimistic and excited ....with just a touch of dread.

[dbus1417 in reply to ETinTX]

Keep us updated! Sounds like we go to the same place.

[Fandango_1 in reply to ETinTX]

Hi, my wife has ET Calr 2 mutation as well. Can i know how old were you when you found out the ET?Have you take any drugs so far?

What were the highest platelet levels you had?

And asociated ti the ET, have you other symptoms?

Thank you and Best wishes 🤗

[ETinTX in reply to Fandango_1]

I had high platelets from the time that I was 40, but didn't see an oncologist until I was 57 at which time I was diagnosed. I have been on Hydroxyurea since 2019 which didn't do a great job of controlling my platelets. I have also taken Anagrelide when my platelets exceeded 1,600 Highest level was 2,200 at which point I had a small stroke that resolved itself within a week. Right now I am on HU and Anagrelide for a short time to drive my platelets down before starting the Clinical Trial. Does your wife see an MPN specialist?

[Fandango_1 in reply to ETinTX]

Hi sorry i have been abroad for a period. Yes she Is followed in a hospital. One question pls:During that period when you were 40 years old, until 57 years old, what was the maximum number of platelets you have had? Thank you!

[Mieshie]

It’s a great news Friday! Congratulations to you and thank you for sharing your journey.

[welshhuw]

Thank you for sharing this update, very pleaded to hear such positive results. Please do continue to post your updates......I'm sure many of us CALR patients will be following your journey with interest and wishing you all the best.