This is a big step for us rare MPNers and I've been watching the conference news reports as they trickle in. The biggest news came from Baxter/CTIBiopharma on their Pacritinib data, a drug for myelofibrosis patients with low platelet counts. (I have the opposite problem, high platelets.) Also the conference reported information on allogenic stem cell transplants for MF. Scroll down for links to some articles from the conference.
In addition to new drug trials, researchers have started combining drugs and watching intently for effective treatments. The key to the drugs we have so far is personalized treatments. Some patients adopt a wait and watch attitude while some are more aggressive and proactive (me). One patient at the conference reported, "I feel like a thorn in my doctor's side." Exactly. Me, too. It's difficult to feel that way after appointments and many times I want to give up and say "whatever."
Much research focuses on trying to reverse the disease process, in other words, trying to keep it from transforming to MF or Leukemia. And secondary to this, is trying to create an acceptable quality of life. That's pretty much where I focus and push my doctor. I'm a HUGE believer in quality of life over quantity.
"The process of scientific advance with patients is a little bit like watching sausage being made -- ups and downs. For some, pulling back that curtain or that process can be too much information, but I do think the partnership between physicians and patients, particularly in these kinds of diseases, really is key. A lot of the advances have really been made by patients being so generous with their time or involvement in trials, or a lot of their bloodwork to be used for research -- All of these key advances have really come from those sorts of efforts." - Dr. Ruben Mesa
MF and Leukemia are the biggest fears of most patients with an MPN. PV or ET, two of the four MPNs, can evolve into MF or Leukemia, but it doesn't happen to most of us. It happens to under 10%, maybe around 3% if I remember correctly.
Also at this time, those of us in the MPN world are celebrating the tenth anniversary of the JAK2 discoveries, chiefly the JAK Inhibitors. I may not have this perfectly understood, but here's my understanding. The mutation of the JAK2 stem cell results in this rare cancer. Therefore, finding meds that inhibit the mutation are a better treatment than simply slowing down the cell production. The JAK2 mutation is how I was originally diagnosed via a bone marrow test in a small doctor's office where I was dazed and confused, surrounded by struggling chemo patients and loving care givers.
I am currently on an oral chemotherapy called Hydroxyurea. We check my blood counts every other month and adjust my dose. It's all a numbers game right now for me. When my blood counts are in a certain zone, I itch like crazy. In another zone, I am tremendously fatigued and fight foggy thinking or "chemo brain." In another zone, I suffer bone pain. All my life, my parents, husband and doctors were helping me try to find out why I suffer "leg aches." As a child, we called it "growing pains." I now know that a lot of it is bone pain from this disease. When the blood counts are in another zone, I get restless leg syndrome and can't sleep well. And always, I have abdominal pain; in fact, that is what took me to the doctor in the first place. We think it is spleen related, but not sure because my spleen isn't enlarged enough yet to show up on the MRIs. Etc. Etc. Etc.
Most hematology/oncology doctors don't know much about this and have very few patients. When my doctor was hedging some of my questions, I asked him if he currently has any other PV patients. He hedged his answer and basically admitted he has had very few in his career. When he first diagnosed me, he said it wasn't cancer. After my research, I asked him if it's cancer and he admitted yes. At first, he told me 8 years is the average life span after diagnosis. After I did research, I saw that this information is outdated. Currently, the average life span after diagnosis is around 20 years.
Upon my diagnosis, I visited specialists at Mayo and Duke and together came up with a game plan which I brought back to my small town hem/onc. I regularly check the Internet for more information and have joined several support groups. Everybody is just as confused as I am. But the camaraderie is tremendous.
MPDchat - If you or a family member has an MPN, join this Google Group, there is no fee to enter. By far, this is the best "support group" I've found. Top level questions and answers.
Allogenic transplantation for myelofibrosis: Final analysis of a prospective study after a median follow up of 5 years
ASCO 2015 - JAK Inhibitor Pacritinib Proves Effective for Easing Symptoms of Myelofibrosis
An ASCO Primer: Which Drug Makers are Pursuing Which Kinds of Therapies
Watch a Roundtable Discussion on MPNs