MPNforum Magazine

by Jeremy Smith

On February 23^rd 2014,  I attended Susan and Sam Klepper’s Bay Area MPN Support Meeting. Usually my 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.

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