A Reporter’s Notebook
by Julie Libon
It all started with a phone call to my sister Lori. I had just seen an article on the upcoming CR&T Symposium and thought it would be an informative event to attend. She was intrigued and the next thing I know, she had signed us both up. She would take the train down from Boston and I would take a quick ferry ride across the Hudson River.
As the day got closer, I heard from those whom I have met through the MPN virtual world. Was I going? Who else was going? Where are you staying? The excitement grew as I realized that I would finally meet these “friends” in real life.
It all began Tuesday night when I made the half mile trek, in my heels, to the ferry to meet my husband Bob, fellow MPNer Mary Cotter and her husband Brian for dinner in New York City. We hit it off right away, no awkward small talk because after all we knew each other from MPNforum. It was a wonderful evening.
The next morning, I boarded the ferry again. I was anxious to start the day. In 8 minutes I was in New York, I grabbed a cab and was on my way. I met up with Lori and we made our way to the Belfer Research Building. It was a beautiful sunny seventy degree fall day in the the northeast.
We registered and laughed when we got our IDs, because mine said media. She proceeded to call me the roving reporter. The first person I recognized
was Bill Crowley from the MPN Research Foundation. I had met him last May, so it was great to catch up. A few minutes later I saw that he was talking to David Boule, the President of CR&T. I made my way back over and managed to get some hard facts on exactly who was attending this Symposium. Here is what I learned. There were 175 attendees from 20 states and several foreign countries. 40% of the people were there for MF, 40% for PV and 20% for ET. I also met a few people who have CML.
Out of the side of my eye I caught a glimpse of Marina Sampanes Peed. I rushed over to say hello, for I had only met her through skype. It was like meeting an old friend for the first time.
I could feel the excitement of so many not feeling alone in their disease anymore.
There was so much to take in. Seeing the doctor’s that I had only heard about, or seen in print from their research was humbling. You could feel the buzz in the room as more and more doctors arrived. People were instantly drawn to them. You would have thought a rock star entered the building when the infamous Dr. Silver arrived. It was a sight to see. The most impressive part was how accessible all of these doctors were. Their willingness to take the time to speak with whomever approached them really stood out to me.
It was time for the presentations to begin. I walked in and saw a man calling me over.
It was Tom Brennan and his wife Diane. We have been in touch because we both go to Sloan Kettering, but we had never met. It was so nice to finally meet in person. Amy Brown came in a little later and it was great to meet her. I recruited her to speak at an event I am hosting in the spring, so I hope she doesn’t regret meeting me. The time had finally come for us to take our seats.
Dr. Robert Kralovics spoke first. He spoke on the genetics of MPNs which was so informative. He told us that because MPNs are liquid tumors they are hard to stage and classify. We also learned that no two patients DNA is the same, which is why some patients don’t respond to treatment. An answer to a question many patients have.
Dr. Ann Mullally is a physician scientist who chose a career in the lab because she was frustrated by the current therapies. Her presentation was very interesting because she discussed the need to destroy the mutated cells. Current therapies target mutated cells and normal cells, which is one reason why we can’t eradicate the disease. She also went on to talk about CRISPR and gene editing. I will leave that subject to someone who understands it better than me. One fact that I found fascinating is that there is a small percentage of the population who have the JAK2 mutation but no disease. She felt that understanding this would be an opportunity for treatment.
Dr. Richard Silver has an amazing sense of humor. He had us all laughing out loud. Dr. Silver and Interferon go hand in hand. He presented that Interferon can significantly decrease the JAK2 Allele Burden in early disease. PV and ET should be treated early, before splenomegaly, MF and fibrosis.
I loved Dr. Verstovsek’s opening line and his answer even better.
How long do I have to live? Everybody could relate to that question. His answer, as long as possible with new medications. Progress is being made and people are living longer. Currently the only cure is a stem cell transplant. He informed us that less than 10% of patients have a transplant for a variety of reasons. We also learned that Ruxolitinib is the only medication approved for MF because the others don’t work very well. There are studies being done combining other medications with Ruxolitinib and new drugs being studied alone to optimize MF therapy. The two hottest drugs being studied are Imetelstat and PRM-151.
Dr. Jerry Spivak had some interesting things to teach us. He told us that the WHO criteria for ET is a myth and that you really need a red mass cell test. Also, platelet counts do not correlate to thrombosis. In ET age makes no difference except for those over 65. A person over 65 has a greater chance of developing AML because of age not ET. A person needs to lower their platelets to get rid of migraines. In ET, Hydroxyurea is not more effective than Anagrelide. If you have ET and are asymptomatic there should be no treatment. He also questioned whether Imetelstat was going a step too far since it can cause neutropenia and anemia. His presentation was definitely food for thought for many patients.
We as MPN patients hear and read so much about clinical trials. Dr. Jason Gotlib answered the question many of us ask. Should I enter a clinical trial? He spoke about the personal motivation of improving symptoms and having a better quality of life. We learned that you have to balance that with more frequent testing, doctors visits and travel. You have to think about the adverse effects you might encounter or that you may be put on a placebo. A drug development timeline is 5-10 years clinical trial and then approval. Ruxolitinib from patient to approval was 4 years, which is very unusual. My take away was if you choose to be a part of a clinical trial, do your homework and learn as much as possible about the trial.
MPN complications affect us all. Dr. Andrew Schafer touched on many of these. Some of the most common problems are thrombotic and vascular, including DVT, pulmonary embolism and clotting problems. There are also arterial issues including stroke, heart attack, miscarriage and digital ischemia affecting hands and feet. Left untreated digital ischemia can lead to pre-gangreneous changes. We are far behind in thrombotic and vascular problems. The only drug is 100 mg low dose aspirin. After a 3 year follow up with PV patients there is a reduction in cardiovascular death, non-fatal stroke and venous thromboembolism. Fun fact – aspirin comes from the bark of the willow tree.
Dr. Mesa was meant from a young age to be a doctor who specializes in Leukemia. He put up the most adorable slide of himself as a small child giving a presentation at a science fair on Leukemia. We all got a kick out of seeing that. Dr. Mesa spoke about what we should expect from MPN therapy. He said that MPN patients are not all the same and nobody is just a disease. Also, symptom burden is not indicative of disease burden. MPNs impact a patients quality of life, including employment, early retirement, disability, anxiety, reproduction uncertainty of the future and choosing not to participate. There will be a quality of life study presented at ASH comparing MPN patients against other groups, for example lupus. The goal of treatment is to help in any way possible, including physical, mental and emotional. There are new evolving therapies being studied at this time.
One of the most interesting parts of attending the CR&T Symposium was hearing other patients stories. Although we are all diagnosed with MPNs our experiences are very different. Lori, Bob, and I went to the MF breakout group. This group was led by Dr. Verstovsek, Dr. Gotlib and Dr. Schafer. They would only answer questions in general terms since we were not their patients.
I talked to a few patients in this group and they had some interesting stories to tell.
There was a gentleman I had spoken to during the registration period who was just diagnosed with MF a month ago. He is currently taking thalidomide and prednisone and has severe neuropathy. He does not have an enlarged spleen. He does have anemia, fatigue and is JAK2 positive. His Dr. put him on Jakafi. He brought this up at our Q&A session for MF and Dr. Verstovsek said that Jakafi worsens anemia, lowers platelets and is primarily used for an enlarged spleen and to improve quality of life. Dr. Gotlib added in that it can sometimes improve fatigue.
I met a patient from New York who has been diagnosed with Myelofibrosis. She has an enlarged spleen and is going to have a stem cell transplant. Her doctor wants to remove her spleen before she has the transplant. It was made clear from every doctor at the symposium that it is the rare case where someone needs to undergo a splenectomy. She left that evening with a lot of questions.
The PRM151 trial holds a lot of promise for many patients. There was however, a woman who was taken off the trial after a year because she did not see any results. When she brought this to Dr. Verstovsek’s attention, he asked if she had progressed at all in that year. She said no. He then told her that it might have been helpful in keeping her disease stable for a year. I thought this was an interesting point.
There was a patient who had a SCT that was unsuccessful. She said she has zero donor cells, but the JAK2 mutation is gone. She is asymptomatic with low platelets and normal spleen. Dr. Gotlib recommended the watch and wait approach in that case and to not start anything new.
Interferon was a hot topic at the symposium and came up again at our MF Q&A.
The question was asked, why are some MF patients given interferon. Dr Verstovsek suggested that it can improve bone marrow production if used early. Dr. Schafer said that advanced cases don’t respond. He also said that data will be presented at ASH about combining Interferon and Jakafi. An important tidbit I learned is that interferon is not approved for MF and there are no plans to pursue approval. This can cause an issue with getting the drug covered by insurance.
The last bit of information I will leave you with is that a scoring system for Pre-PV and Pre-ET should be published soon.
By the time the symposium had wrapped up, I had met new people, learned from top MPN specialists and was a roving reporter. It was a great day.
With the day done, here are my take aways that were similar to others at the symposium. There are currently no big breakthroughs that we haven’t heard of before, you can put the top MPN specialists in the same room and get many different opinions on treatment and some of the WHO criteria. Patients I spoke to were confused because of differing opinions between some of these doctors and their own doctor. My last takeaway is that research is being done and these doctors are frustrated, like us, by a lack of good treatments and are invested in finding good therapies and a cure. Overall, I am very optimistic!
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