..the MPN community's hometown paper

Tefferi speaks…

by Jeremy Smith

On February 23rd 2014,  I attended Susan and Sam Klepper’s Bay Area MPN Support Meeting. Usually myJeremy paint participation is limited to Facebook and the MPNforum Magazine. I have become comfortable with using the Internet as a communication and support tool.  I do think meeting face to face is much better. It’s a pleasure to actually sit down with people and be able to share my experiences with them.

There were between forty and fifty MPN patients, spouses, family members and friends in attendance. All there to interact with  Dr. Ayalew Tefferi of the Mayo Clinic (Rochester).

 Based on the invitation many of us thought Dr. Tefferi would actually be there. Instead he connected to us via telephone and a good speaker which boomed his   voice to us. Dr. Tefferi was speaking to us regarding Geron’s new drug Imetelstat, a telomerase inhibitor.  

It was said Imetelstat has demonstrated significant activity in myelofibrosis, including complete responses and in some cases something close to remission. It was pointed out by a SCT patient who was part of the group attending that doctors rarely use words like remission. Later that day this person shared one of the most harrowing stories of a stem cell transplant I have ever heard. It’s reminded all of us of the imperfections and complications that can often arise in these situations. She gave detailed accounts of how painful the experience can be even when in the hands of some of the finest docs around. Her experiences moved and shocked many in the  audience.

In attendance for the event was a current patient of Dr. Tefferi who has MF. She has been part of a clinical trial of Imetelstat and has had amazing results. She was looking at a SCT. Today her JAK2 is no longer positive her fibrosis appears normal and she looks very healthy and full of energy. This is amazing when you consider how far she has come in her journey. Seeing similar results to an SCT transplant with her bone marrow biopsy and blood work without the risks of an SCT blew everyone away.

As she shared her story you could feel the electricity in the room as she stood before us presenting her story with bold smiles.  This was the most inspirational story I have witnessed at an MPN event and everyone expressed a heartfelt welcome to her.

At this point we do not know the long term impact of taking this drug. Or what might happen when a person discontinues taking the drug. Time will tell if this drug actually may be the drug that changes MPN lives in a big way. According to the patient, the drug is taken at an infusion center and I believe she said it can take more than three hours to administer.

Imetelstat is not FDA approved but there is some discussion it will be fast tracked. When I asked Dr. Tefferi what this means from a timeline standpoint he would not comment due to his relationship with the Geron. Not sure what this meant because it’s a quite common term but he would not answer the question.

The current clinical trial for Imetelstat is now closed. Dr. Tefferi announced a new clinical trial that will start in July, I read in a stock analyst report it will start in April. It will be launched across the Globe in twenty cities. Starting in Israel, Italy, Canada and 20 plus centers in the USA including CA, NY and the Southern part of the United States as well as the Mayo Clinic. 

ONE SIZE DOES NOT FIT ALL. OR IN OUR CASE WHAT WORKS FOR ME MAY NOT WORK FOR YOU AND VICE VERSA

Dr. Tefferi spoke of what he believes to be the biggest change in treating our MPN’s and that is the role genetics play in our lives. For example for one patient INFN may work better for MF than Jakafi while for another patient Jakafi may be the better choice for. This led  Dr. Tefferi to talk about the role genetic testing plays and the different between being JAK2 positive or CALR positive. If one is JAK2 you cannot be CALR but if you are CALR you need to be treated differently than a JAK2 patient. Dr. Tefferi recommended all MPN patients get genetic testing done.

Doctor’s have been going with the one-size fits all without taking in to account how every patient behaves, acts and responds differently to drugs. My DNA is different than yours and yours is different than mine.  Doctors have to look at each patient as individuals needing treatments that meet the specific needs of the individual patient. Patients need to start thinking about their disease, as their own and no one is the same.

 Dr. Tefferi discussed different MPN mutations for each patient with new platforms looking at over 200 genes not just JAK2.

The CALR gene is a novel biomarker that was reported to be specific for myeloproliferative neoplasms (MPN) for the first time at the annual meeting of the American Society of Hematology (ASH) last month. If you are a JAK2 patient who converted from PV to MF you would still be JAK2. He also said PV patients are not CALR either. However its important if you’re a MF patient you should be tested to see if you CAL-R or Jak2. CALR patients have a better prognosis than JAK2 from some of the data he is tracking. Mutated patients with a more aggressive MF may need to go earlier to an SCT than patients with less aggressive MF.

Imetelstat seems to work molecularly in MF patients but not on others. They don’t know why some patients respond well and others do not. Dr. Tefferi was quick to point out Jakafi does not do this. A big difference between the two drugs.

Tefferi  announced and invited everyone to his April 12 patient conference in Miami.  According to Dr. Tefferi no drug companies have been invited or allowed to participate in any sponsorship of the event. He and others on the panel will be reviewing all clinical trials. He said he will ask the other Doctors there who will be presenting their findings the following question: “Would you put your daughter or son on this clinical trial.”

Towards the end of the event a newly diagnosed PV patient was sharing her story about switching from Hydrea to INFN and how happy she was. And this is when the Tefferi show went off the rails for me. After spending most of his almost two hour call talking about how one drug does not fit all and it may take testing to find out which drugs are best for an individual he stated pegylated INFN is a drug he would not use. He does not believe there is clinical evidence supporting the use of the PEG INFN and the claims that it inhibits the progression of an MPN disease. He left this person stunned by his response as she quickly sat back in her chair.

Dr. Tefferi went on to say that busulfan is a great drug for PV patients and he does not understand why more doctors aren’t using it for the treatment in PV patients. In his opinion studies do not show INFN is more effective than Hydrea. Then Dr. Tefferi went on to say  “Incyte hates me.” He has not put a single patient on Jakafi.”  Too many side effects and for MF patients who have anemia, Jakafi makes it worse. Nor does Jakafi help with fibrosis. After he made this statement a patient who is on Jakafi spoke up saying how well he is doing on the drug and he has returned to a normal life.

A Tefferi town hall type setting is always entertaining and he answers almost everyone’s questions. In my humble opinion Dr. Tefferi does a disservice to his reputation when he slams the use of pegylated INFN and Jakafi. Other than his critique of INFN and Incyte it was a fantastic meeting.

Full disclosure: Jeremy Smith is a shareholder in Geron Corporation.

Take me back to the Contents

© MPNforum.com, 2014. Unauthorized use and/or duplication of this material without express and written permission is prohibited. Excerpts and links may be used, provided that full and clear credit is given to MPNforum.com with appropriate and specific direction to the original content.

Comments on: "Tefferi speaks…" (27)

  1. Very interesting, thanks for sharing it with us.

  2. We get it…Tefferi must be the worst physician there is. He uses carcinogens, limits his patients’ therapeutic options, slams approved FDA drugs, and list goes on and on.

    • Perhaps a heavy-handed sarcastic response to a comment is not always appropriate when dealing with issues of patient concern. Busulphan may have a place in MPN treatment and SCT preparation. My comment simply supplied one reason the drug has fallen into disfavor. Those were facts backed up by provided citations.

      Dr. Tefferi is an eminent and powerful voice in American hematology. He is not, however, infallible. Your description of his activities and preferences are based entirely on Tefferi’s own public statements. Your conclusion as to his abilities as a physician are entirely your own and hardly shared, I imagine, by anyone.

      Refusal to prescribe Jakafi limits his patients’ therapeutic options. Tefferi has indeed attacked Jakafi publicly on several occasions and diminished interferon as a viable MPN drug. Jakafi is by no means a perfect drug, but it is FDA approved after rigorous clinical trial and the best we have to relieve constitutional symptoms of MF. It may possibly have other significant benefits. Thousands of us are enjoying a higher QOL because of it.

      For us, as patients, the interferon issue is more serious. Widely used in Europe, interferon remains an off label drug for MPN patients in the U.S. It has been ignored and misrepresented based on side effects from much earlier high dosage levels until recently. Based on published research and anecdotal reports from our own family of MPN patients therapeutic response to Pegasys and Intron A has been enormously encouraging.

      We have an obligation to explore therapeutic assertions of drug companies, scientists, physicians, and fellow patients and get to the bottom of things. Challenging claims, whether mine or the Emperor of Hematology’s, is an essential undertaking. We can’t afford to believe anyone these days walks on water.

    • Jeremy Smith said:

      Hi Panos,

      Thank you for responding to the article. I was not saying Dr. Tefferi is a bad Doctor at all but there are some questions that need to be answered on his part. He refuses to use both Jakafi and INFN with patients claiming they are not effective while also stating we have to try all drugs available but he does not follow his own recommendations. It’s also fair to question whether or not he is too close to Geron because he never mentioned any of the side effects of Geron on a conference call I was on with him yet he knew about them.

  3. “Dr. Tefferi went on to say that BUSULPHAN is a great drug for PV patients and he does not understand why more doctors aren’t using it for the treatment in PV patients.”
    Reported by Jeremy Smith

    Two Good Reasons: Busulphan is a known carcinogen and is known to increase risk of acute leukemia. And, interferon is available, non toxic and effective.

    “Radioactive phosphorus and alkylating agents such as BUSULPHAN are effective at controlling the platelet count but are associated with an increased risk of progression to acute leukemia, particularly when used sequentially with hydroxycarbamide.”
    Anthony R. Green. Blood, 2011

    BUSULPHAN is a bifunctional alkylating agent, having a selective immunosuppressive effect on bone marrow…According to the Fourth Annual Report on Carcinogens (NTP 85-002, 1985), busulfan is listed as a known carcinogen. [PubChem]

    Acute leukemia and myelodysplasia in patients with a Philadelphia chromosome negative chronic myeloproliferative disorder treated with hydroxyurea alone or with hydroxyurea after BUSULPHAN. Am J Hematol 2003;74(1):26-31. Nielsen, Hasselbalch

  4. Kelley Lanier said:

    I have been through two clinical trials with Dr. Tefferi and I respect his undoubted passion for finding a cure for myelofibrosis. However, I believe that his passion can negatively effect the doctor-patient relationship. In my case, when things were going well, he was happy and supportive. However, when the results were not so good, he was much less supportive and communicative.

    I would urge anyone considering enrolling in a clinical trial to get an opinion from a hematologist who is familiar with you and your disease and not involved in the trial. It would also be important to keep that hematologist in the loop as you proceed through any clinical trial.

    Kelley Lanier

    • Jeremy Smith said:

      Hi Kelly,

      I had not heard previously anything like that “However, when the results were not so good, he was much less supportive and communicative.”. Thank you for sharing.

      Jeremy

      • Let me join Jeremy in expressing gratitude to Kelley and all patients who share their experience. The medical fraternity might disagree vocally on treatment options but we rarely hear them criticize their colleagues’ professional actions. As patients, we’re on our own in this most critical arena. Unfortunately that reliance on each other needs to extend to choices of therapies as well. When one doctor slams FDA approved and widely used drugs and no specialist rises to defend their use, what can we do? We need to depend, as Kelley points out, on our hematologists and, equally, trust each other to provide honest reports of our progress in therapy. (The List of Hematologists is an invaluable resource for all of us, a place to find physicians recommended to MPN patients by MPN patients based on direct experience. Please add to that List if you have a good, supportive, communicative doctor.http://mpnforum.com/list-of-100-hematologists/) Thanks, Kelly…

  5. jsandresen1 said:

    Hi Dore,

    Tefferi stated imetelstat works molecularly with some patents but with other patients it does not. So some patients respond well and it impacts the patient molecularly, a good thing. In other patients the drug did not.

    Tefferi was pointing out one of the big differences between the two drugs, imetelstat and Jakafi, is Jakafi does not impact an MPN patient molecularly. He was making the point to prove the benefits of imetelstat over Jakafi.

    Another point Tefferi made is the Jakafi can be a poor choice for MF patients because Jakafi makes people with anemia worse and many MF patients have Anemia.

    I hope that clears things up for you.

  6. can you clarify: “Intelestat (you mean imetelstat) seems to work in molecularly in MF patients but not on others. They don’t know why some patients respond well and others do not. Dr. Tefferi was quick to point out Jakifi does not do this. A big difference between the two drugs.”

    imetelstat does not work on other what?

    Jakifi does not do what?

    Big diff betw the two is imetelstat disease modifying effect but what are you referring to as big difference?

    thanks

    • Thanks Dore for picking up that typo. Jeremy Smith provided copy and edits at the very last minutes of the publishing cycle of MPNforum and the errors in that sentence were ours not his and since corrected. We’ll let him respond if he chooses but we should underscore the conclusions in your last sentence are yours and not his.

      Although, based on Dr. Tefferi’s pilot study, there are some positive indications in certain cases, we don’t yet know whether or not Imetelstat will have a durable impact on underlying myelofibrosis; Jakafi has never claimed to effect the disease, but to alleviate the worst of symptoms and improve QOL and, possibly, longevity. We all look forward to the results of clinical trial.

  7. I am really curious now about Imetelstat (sorry if spelling is off). I cannot take Hydrea, Angrelide, or any form of Interferon. I took p32 twice at Mayo Scottsdale (hello Kathy).
    It lowered my platelets dramatically without apparent side effects, while side effects on the other drugs were at ER level. I have done a lot of traveling, however, both to Mayo and also to Hopkins. I don’t know if I want to go to a third venue, Mayo Rochester, to see Dr. Tefferi. One can spend all of one’s money doing this! But I’m not sure I want a third dose of p32. My platelets are pretty well controlled now, 600,000 abou

    I am partial to Dr. Spivak, and if he knows about Imetelstat, I might go there to discuss.

    I am having phlebs rarely now, as my hematocrit is rising only sporadically now. My hematologist is concerned about low Ferritin from phlebs.

    As for Teferri’s personality, I heard that he was not very copacetic from Porphyria patients who consulted him. He basically brushed them off saying they didn’t have it if they didn’t have really high test scores. They were hurt by this, and most got dx’s later from other people who looked at the whole picture. He, however, comes from South Africa and is considered a Variegate Porphyria expert. Most docs in SA know about Porph because it is
    in 5% of the population, highest ini the world.

    Dr. Cammoriano tested my porph while I was seeing him at Mayo and he said it was positive. It probably was not high positive on a test given without me being in “attack”, but he was satisfied with the results and went on to discuss the PV which I’d come to see him about.
    Having Porph as well as PV is the ultimate double jeopardy when it comes to drugs, but it is an advantage in that we Porphs know a lot about what triggers illness in the areas of drugs, foods, and the environment.

    Mariel

  8. Jeremy Smith said:

    Well said Dave,

    When I converted from PV to MF I stopped the Hydrea. My Doctor was leaning towards Jakafi and I said no I want to try INFN. I had to put my pit down on tips because he was in the Tefferi camp and no at Stanford was being treated with INFN with MF. He had been surprised so far at the stability but time will tell.

    As far as Tefferi I don’t understand his position.

    Thank you for your comments.

  9. I’m in a love/hate relationship with Jakafi. I’m not sure where I would be without it, but I seem to be on a long term downward spiral of my platelets and I think this is true for a lot of us long term users. Your platelets likely won’t come up again even if you stop. This makes joining another drug trial including Imetelstat unlikely since your platelets are too low to qualify. Treat MF early with Pegasys and give it time to work. It may take a year or more before you see results. I wish I had given Pegasys more time before starting Jakafi. Now I’m kinda stuck and my counts actually fall If I lower my dose of Jakafi.

    I used to criticize Tefferi for his comments on Jakafi. I think he is wrong to deny his patients who are really suffering, and I don’t understand how he would deny Pegasys either. He seems to have a big ego and was upset that Incyte took Jakafi away from him and handed it to Verstovsek at MD Anderson to set it up for FDA approval. In spite of all this power play and egomania, I still believe Mayo Rochester is doing great work. Tefferi is correct on how our genetics will play into future treatment of MF, and if you don’t know your mutational status, you will be at a disadvantage for determining what treatments you should start and when.

  10. I am confused on his comments re PV, yes to imetelstat or no?

  11. I had the same question as guy. Your answer still has me scratching my head a little. So did Dr. Tefferi say the things in the earlier paragraph and then contradict himself? That’s my question and I’m still not clear. Here’s the part I’m confused about: “Dr. Tefferi spoke of what he believes to be the biggest change in treating our MPN’s and that is the role genetics play in our lives. For example for one patient INFN may work better for MF than Jakafi while for another patient Jakafi may be the better choice for.” Thanks for any additional clarification you can provide.

    • Looks like two separate issues. As an illustration of genetic differences, Dr. Tefferi could correctly point out “For example for one patient INFN may work better for MF than Jakafi while for another patient Jakafi may be the better choice,” and stil maintain,as Jeremy reported, “pegylated INFN is a drug he would not use.” (Wasn’t Dr. Tefferi also reported as saying he hadn’t put a single patient on Jakafi?) The limitation of his patients’ therapeutic options may not square with his deep understanding of how genetics drives our responses to drugs, but that’s a different question.

  12. jsandresen1 said:

    Guy,

    Sorry I thought my article was clear.
    Tefferi would not use Jakafi or INFN. He does not believe INFN is a better drug option than Hydrea or Bulsfan.

    By the way I was not sharing my personal opinion only his. I hope this clear things up.

    • jsandresen1 said:

      Guy,

      My other point in the article was Tefferi made the statement we must consider all drugs because what works for one patient may not work for another patient. But then he said he would not use INFN or Jakafi which means he would eliminate two drugs that work very well for others. Which contradicts his original statement.

  13. Thanks for the nice report. I gathered that you were moved by Dr. Tefferi’s comments regarding his opinions/use of Jakifi and INFN. However, I am a little confused because of apparent inconsistent viewpoints that were conveyed in your report. What caught my attention was that in one place you write “For example for one patient INFN may work better for MF than Jakafi while for another patient Jakafi may be the better choice for.” Am I correct to conclude that it was Dr. Teferri who said something to this effect? If so, it would imply that he considers each of these could be a suitable treatment choice in some patients. In this case, this seems at odds with the statement that “He does not believe there is clinical evidence supporting the use of the PEG INFN and the claims that it inhibits the progression of an MPN disease” and of “He has not “put a single patient on Jakafi.””. Perhaps I am misunderstanding the context or something else, or perhaps Dr. Tefferi’s statements were entirely consistent. Any clarification you can give would be appreciated. Thanks.

  14. Jeremy Smith said:

    Kathy well said

  15. Kathy Van Meter said:

    I am saddened by the slamming. Those of us who are on INF or Jakafi really do not need to hear, in Dr.Tefferi’s “absolute terms” that we are being mistreated. There are a variety of reasons that we are taking these drugs, maybe to “get your life back” as in reduce the constitutional symptoms, or allergy or intolerance to other drugs. In this social worker/PV patient’s humble opinion, support SHOULD BE the name of the game no matter who you are. Peace…….

  16. jsandresen1 said:

    Hi Chuck,

    Many patients rave about him as a Doctor and this is the second time I have seen him speak. I am always riveted by the way he presents. I did not like his views in INFN not because he disagree with some of the science. It was the way he slammed INFN.

  17. Chuck Varnado said:

    Thanks Great Article Jeremy. I am really starting to wonder what is really motivating Dr Tefferi.

  18. jsandresen1 said:

    By the way at the end of my article it states I own shares in Geron. for the record I also own shares in Incyte.

  19. jsandresen1 said:

    Hi Bonnie Thank you. I could not agree with you more about Tefferi he is never boring.

  20. Bonnie Evans said:

    Thanks for the article. I always am interested in what Dr Tefferi has to say.

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