This ASH Special Report touches on some of the science, the reports, the doctors and drug industry circling our MPN world. What was not in evidence at ASH were MPN patients. To bring it home, we sat down with one of our regular columnists, Harvey Gould, to ask him about his experience as a participant in the YM Biosciences CYT387 clinical trial.
MPNForum: What’s your diagnosis and prognosis?
Harvey: I was diagnosed with PMF more than twelve years ago when I was told that my prognosis was three to five years. One thing I’ve learned is not to pay attention to prognoses, but rather to get on with life and fight this monster. I love confounding the medical community.
MPNForum: Who suggested the idea of you becoming a participant in the clinical trial?
Harvey: My hem/onc.
MPNForum: Did he explain why he thought you might benefit from it?
Harvey: At the time (early 2011) I’d become transfusion dependent; my spleen was extremely large and my hemoglobin was dropping back into the 7s within days of me receiving two to three units. He said the two primary desired effects of Cytopia (CYT387) were shrinking the spleen and elevating hemoglobin.
MPNForum: How easily did you get into the clinical trial?
Harvey: Not easily. One of the protocols for admission was that your creatinine could not be above 2.0. At the time of my application my creatinine was then about 2.5, so I didn’t qualify even without further investigation.
MPNForum: But you were admitted. How did that happen?
Harvey: In April 2011, as my spleen was continuing to grow and it seemed clear that I was going to continue being transfusion dependent I had a splenectomy. Of the many consequences, one was that my creatinine dropped below 2.0. I still had to undergo other testing, including a bone marrow biopsy, but ended up qualifying and was admitted to Phase II of the Cytopia clinical trial when all participants receive the actual medication—no placebos.
MPNForum: When did you enter the program and are you still in it?
Harvey: I entered in August 2011 and I remain a participant though you’re told that you can leave the trial at any time.
MPNForum: What does participating entail?
Harvey: In the early phase I was required to return for monitoring I think weekly. Now I return once every three months. “Monitoring” means having vitals taken, an EKG (in the earlier time into the program), having blood drawn for numerous tests, answering a series of questions regarding any side effects, quality of life, etc., and a neurological exam.
MPNForum: Do you take the medication only while at the site for a follow up?
Harvey: No. You’re given sufficient medication to have enough to take two capsules daily.
MPNForum: What benefits, if any, have you realized?
Harvey: The first time I went there I said, “It’s a miracle! You can’t even palpate my spleen!” The doctor said, “Very funny.” I said, “Oh, yeah. I don’t have a spleen anymore.” So the only real effect for me is on my hemoglobin.
Harvey: I think it’s helped elevate my hemoglobin, but it’s impossible to be sure.
Harvey: Some of the less desirable consequences of my splenectomy were that my platelets and WBCs elevated to unacceptably high levels. Though I’ve permanently lost partial vision in one eye because of the elevated platelets and related hemorrhaging in the eyes and swollen optic disks, the treatment was for me to go on Hydroxyurea to bring down those levels. Though my dose varies depending on my WBC and platelet levels, I’ve been told I’ll remain on HU, or some related drug for the rest of my life.
MPNForum: And what’s the correlation between your hemoglobin and the HU?
Harvey: While the HU has brought down my WBCs and platelets, it also brings down my hemoglobin at the same time that the Cytopia is trying to elevate it so you have competing forces.
MPNForum: So why do you think the Cytopia has helped your hemoglobin?
Harvey: Because even while I continue to take the HU my hemoglobin remains in the mid 9s and I haven’t needed a blood transfusion for the last year and a half.
MPNForum: Have you had any side effects that you attribute to the Cytopia?
MPNForum: How long do you anticipate remaining on the drug?
Harvey: I was told upon admission to the trial at I’d continue to receive it as long as I was willing to take it and as long as it appeared that I was benefiting from it so unless it loses its efficacy I intend to continue to take it forever.
MPNForum: Have you learned of any potential benefits of Cytopia as compared to other JAK2I inhibitors?
Harvey: Dr. Jason Gotlib heads up the study at the medical facility at which I’m a participant (Stanford.) It’s my understanding that he has recently reported that from studies thus far, Cytopia appears not to negatively impact RBCs while there have been some reports of such negative impact from other JAK 2 inhibitors.
MPNForum: Are you generally encouraged by developments in scientific studies into MPNs?
Harvey: I am greatly encouraged. Look, the JAK 2 mutation wasn’t even discovered until five years after I was diagnosed and since then a number of new drugs are being tested and some are now on the market. To be honest, after my diagnosis I’d figured, “OK, this is so rare there won’t be any serious scientific investigation into causes or possible cures.” I’ve been pleasantly surprised.
© Harvey Gould and MPNforum.com, 2012. Unauthorized use and/or duplication of this material without express and written permission from this blog’s author and/or owner is strictly prohibited. Excerpts and links may be used, provided that full and clear credit is given to Harvey Gould and MPNforum.com with appropriate and specific direction to the original content.