Hi Janet, Great question it seems not to have as many side effects as other options. Hope it will be approved soon!

Hi Cindy, I’m doing well enough. I say well enough because I do still have fatigue some days. I’ve had ET since 2007 but the fatigue didn’t start until about seven years ago. The medicine does reduce platelets. It can also increase hgb a little but that’s not consistent. It does not reduce red blood cells and hgb like HU does. I do have a side effect, nausea. I take anti nausea medication. I don’t mind this side effect because it’s lessening over time and overall I’m doing much better on the bomedemstat than previous medications. I take 50 mg a day. I was started on 25 mg. I was increased and agreed to increase to 55 mg three times but it wiped me out within three days. So, it’s agreed I’ll stay at 50 mg. What I mean by agree is….the most recent time I agreed I told the PA and research coordinator that I would only try an increase again with the understanding that if my body couldn’t take it I would stop. I didn’t need to say it but I needed some sense of control. We have an understanding, thanks to Dr Ruben Mesa, that there is a quality of life that needs to be maintained. I know I’m obsessive about my quality of life but this is because of dealing with my past local hematologist and the bad side effects I experienced on previous medications. And, them not giving a hoot about it.

I’m wondering how the FDA, merkt, and who ever, will decide what the dosing will be once approved. The trial called for increases, based on mg per kg of weight. That is about a ten mg increase. My body could not tolerate a ten mg increase. After the initial weeks of increases, we realized I had to start all over and wait weeks between increases and only increase 5 mg at a time. How was this possible given the trial protocol, you might be thinking? It’s because Dr Mesa was over the trial. He took care of me. He is no longer at the trial site, Mays Cancer Center in San Antonio. He is at Wake Forest in NC. I will see him there every six months or so but he is not over the trial.

I didn’t know there were any phase 1 trials for ET occurring at this time. What is the medication and has it reduced your platelets? Any side effects? Hope you are doing well.

And how are you doing, in the trial for 3 years? I am finishing a trial for ET, but I guess I didn't realize it is phase 1 so therefore not focused on efficacy and now I'm wondering why my doctor seemed so hopeful it might bring about more improvement. I was asymptomatic before the trial, still am but just disappointed

If something is working, no need to change. I'm just wanting to connect within anyone who is on the trial.

Based upon your reply....does your daughter also have an MPN?

My daughter doesn’t want me to be a trial Guinea pig. I don’t want to be one either. Pegasys is working great with me!