One question that always comes up when Bob and I talk is this: “Why don’t we have new drugs for the MPNs?” It is a difficult question for me to answer. Is it for lack of trying? No, I’m sure that’s not it. Basic and clinical researchers are working harder than ever to identify new targets and develop new therapies. Is it that the best researchers are working on other diseases? Certainly not. No, I think it is the usual challenge of translating discoveries into therapies that work not just in animal models, but also in humans. Let’s consider the mainstay of therapy, JAK inhibitors. We are fortunate to have ruxolitinib available for PV and PMF patients. Despite its shortcomings, which include myelosuppression at higher doses and limited activity in some individuals, it has helped so many people. Why don’t we have a second JAK inhibitor in our arsenal to treat those who are intolerant or resistant to ruxolitinib? There are a few reasons, such as the challenge of accruing large numbers of patients to studies and severe side effects in a small percentage of patients. With respect to the latter, it isn’t for me to say whether the benefits outweigh the risks, but it’s a critical issue for patients and their advocates to consider.
Where is the field headed? Perhaps the therapy with the most potential is a JAK2V617F selective inhibitor. This would be a drug that inhibits the mutant kinase while leaving the normal (or wild-type) protein alone. Although such a drug would be effective in only the 50 percent of PMF patients with the V617F driver mutation, it would be applicable for nearly all patients with PV who need therapy. It is likely that this drug would be more active in patients than ruxolitinib, as the doses could be increased with less toxicity than drugs that inhibit both the mutant and wild-type versions. JAK2V617F selective inhibitors are not in the clinic, but it is likely that companies and academics are aggressively searching for these. If they aren’t, they should be. Finally, type II JAK inhibitors, which target the kinase in a slightly different way from ruxolitinib, are under investigation. I expect that we will see one or more of these novel JAK inhibitors in patients in the future. [...]
When I was first diagnosed with Essential Thrombocythemia in my mid-thirties, I read through the many sources of conflicting information found online. It left me asking many questions such as: Was this a big deal? Am I over-reacting? Am I under-reacting? Do I tell anyone? Is this because I was raised beside a chemical plant? Because I previously smoked? Because I microwaved my food in plastic? These types of unproductive thoughts flooded my mind and consumed entirely too much of my time. I turned to online forums filled with people who were equally confused, scared and frustrated and also, unfortunately, sharing misinformation. This was gasoline on an existing depression fire that I already had burning. I knew I had to change my focus quickly or my biggest fears would materialize. I would be sickly because honestly, in life, we get what we focus on the most. [...]
The patient registry, or natural history database, coming in 2016, will allow patients to report data spanning symptoms, demographics, treatments, and more. MPNRF is seeking to add to our small but mighty team by recruiting for a newly created role: the Registry Manager. [...]
Even though MPN patients who have a rare disease many may have never heard of "Rare Disease Day" and know next to nothing about the impact rare disorders have on society. Rare diseases play a larger role in public health than most people realize and deserve consideration from the medical community, policy makers, and the general public. [...]
There has been, from time to time, a question about whether myeloproliferative neoplasms are cancers. Let’s be clear, they are. But don’t take our word for it. [...]
For the past several months we've kept you informed and taking action on Medicare's pending decision of whether to provide coverage for people with myelofibrosis seeking a stem cell transplant. Currently there is no official ruling from Medicare on whether this treatment is covered, leaving many patients to risk paying out of pocket (running potentially several hundred thousand dollars) or scrambling to find alternative ways to cover it. [...]
According to a paper published in Nature Medicine, the drug MLN8237 (or Alisertib) has reduced tumor cells, blood counts and fibrosis in mouse models of myelofibrosis. Reports John Crispino, PhD, "This new paper shows that Alisertib has an anti-tumor effect in samples of myelofibrosis, similar to what we saw in megakaryocytic leukemia."
John Crispino is also the Scientific Advisor for the MPN Research Foundation. [...]
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Check back regularly for updates about what’s happening in the MPN community, from advocacy to research..