Tagged: APR246 Trial
December 3, 2018 at 6:50 pm #44615Paul DyerParticipant
I am a 50 year old man, diagnosed in mid July with high risk poor outcome multiple mutated genes. Further cytogenetic study showed P53 mutation.
Luckily I went to Dana Farber, and enrolled in a new clinical trial with a drug called APR246 & azacitidine. After 2 rounds blasts were less than 1% (down from 9%). I was immediately scheduled for an allo transplant which occurred on Nov 3, after another trial of venetoclax pre transplant. Now at day +30 and improving daily.
Is there anyone else here in the APR246 trial? Results?March 25, 2019 at 10:31 am #46362chicaloca9999Participant
My dad was just diagnosed with MDS. Not sure of all the details at this point. I just know he has a tp53 mutation and a partial exon 9 duplication not sure if all other results have come back yet or if he has more issues like that. Not sure risk level yet. 1% blasts we have been told but that did not come from his main hematologist. We have not been able to see her yet due to him being inpatient.He is 70. Anyone else have this mutation or duplication? Not even sure what to expect. He is currently in hospital due to infection to which they cannot figure out what bacteria, fungus, or virus is causing it. He almost did not make it. He is better now lab wise but now is having issues with confusion and lethargy. They stopped voriconazole due to possibility of that med causing the mental status changes but day 4 out, he is still not any better in that area. On cefepime now along with many other meds such as vancomycin. Not really sure what is causing the mental status changes and lethargy that seem pretty severe. Any advice or info would be greatly appreciated. I saw you also had tp53 so I thought maybe you would have knowledge.March 25, 2019 at 3:34 pm #46366Paul DyerParticipant
Unfortunately, I don’t have much insight for you. My diagnosis/treatment/transplant happened so quickly. I was in very good shape when diagnosed. I had no problems with the clinical trial/treatment. The drug APR-246 (Prima-1) is now in its phase 3 trial. I was involved in the 1b/2 phase. It is showing great promise to a mutation (TP53) that has had poor success to date. The trial is only happening at a few “centers of excellence” such as MD Anderson, Moffitt Center, Dana Farber? I hope this info was of help, and wish your Dad the best.May 21, 2019 at 2:13 pm #46775Amy CParticipant
Paul- how is everything going now? I explored this trial for my Dad with a Dr. at the Moffitt center.
He has MDS Tp 53 as well. Thanks and good luckAugust 28, 2019 at 7:57 am #48757
I’m 63 yr old male. Diagnosed with MDS P53 mutation 2 months ago. Also went to Dana Farber. Was hopeful to get accepted into APR246 trial but not a candidate because I had throat cancer successfully removed 10 months ago. Because this is a blind trial and was told you cannot move between groups, I elected Decitabine in hopes of remission (12% blasts) for stem cell transplant.October 17, 2019 at 9:09 pm #49186
Paul, my husband is 56 and was diagnosed in July with high risk MDS with excess blasts and p53 mutation. He is being treated at Dana Farber and is also in the APR246 clinical trial. After 2 rounds his blasts went from 15% to less than 5% in the marrow but his doctor feels the MRD is higher than what he’d like before transplant. We were also told he still has p53 mutation. He is just finishing round 3 and will be having another BMB on the 29th to decide if he will go right to transplant or have more of this regimen or change to a different one. (He does have a perfect match) How are you doing now?October 17, 2019 at 9:24 pm #49187
Herb, how are you doing with your treatment? We are also being treated at Dana Farber.October 20, 2019 at 7:09 am #49197
Colleen & John,
I hope you are doing well and pray for your success. Did 10 days of decitamine which landed me in hospital for 12 days with several infections. I am now on a chemo cocktail of decitamine and Venetaclax. Tolerating it well this far. BMB scheduled for 10/23 locally (Western MA) and going to DFCI on 10/25 to discuss results and next step. DFCI has found SCT donor, hoping and praying chemo reduces blastsOctober 22, 2019 at 6:11 pm #49219
We will keep you in our prayers that this regimen is working! John has a BMB on the 29th. Those results will tell us if he’s ready for transplant. Need minimal residual disease to be much lower than last one. We won’t have results till that Friday at earliest. Good luck & best wishes to you.October 22, 2019 at 6:24 pm #49220
The regimen you are on is the alternate one he will be going to if they can’t get him low enough so we’re curious to see how well it works for you.October 23, 2019 at 7:05 am #49223
Colleen & John,
BMB will be this morning. Been hoping and praying for good results. I have had more energy and started exercising again. Will let you know as soon as I get results. Good luck on the 29th. I’ll be at DFCI on the 25th to discuss next steps.October 23, 2019 at 8:54 pm #49227
Hope it went well today. Will keep you in our prayers for a great result! Please let us know how it goes!October 26, 2019 at 1:21 pm #49267
Venetoclax did its job! BMP shows blasts went from 11% to 3%. Dfci is tentatively thinking sct in December. I didn’t realize that tp53 mutation means we have a higher chance of recurrence of mds even after sct. Good luck on the 25th and please let me know how it goes. Fingers crossed for youOctober 26, 2019 at 5:43 pm #49268
That’s awesome news!! Did they say anything to you about minimal residual disease (MRD)? John’s marrow showed less than 5% but MRD -measured by flow cytometry- was high still. We’re anxious to see what next weeks marrow shows. Praying and hoping so much that this last round did it! Yes we were told that he has a higher recurrence rate so that’s why we need to get him to lowest number possible. Will you be having another round of chemo?October 26, 2019 at 6:44 pm #49269
Because BMB was done in Western MA, DFCI didn’t have full report at the time that would have revealed MRD. To be honest, I don’t recall any doctor speaking to us about that. I had biopsy at day 15 of Venetaclax whereas they want you to do a full 23-25 days for full effect. Because SCT will not be for over a month, I will be having another round of decitamine and another month or so of Venetaclax. Hoping and praying for good news for John. Can’t help but think it would be something if we were in at same time. Please feel free to email me at firstname.lastname@example.org. I wish you all the best.
Register for an account, or login to post to our message boards. Click here.
- You must be logged in to reply to this topic.