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APR246 trial

Home forums Patient Message Board APR246 trial

This topic contains 4 replies, has 4 voices, and was last updated by  herb 3 weeks, 2 days ago.

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    Paul Dyer

    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?



    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.


    Paul Dyer

    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.


    Amy C

    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 luck



    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.

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