New rules on data sharing: how will they impact MPN trials?

ASH Clinical News ran an article last month about new rules from the NIH that dictated that outcomes from clinical trials operating with NIH funding must be shared within 12 months of the close of the trial. This is significant for clinicians who run trials, companies who provide drugs for trials, institutions that host trials and most importantly, patients who put their health and lives on the line when they participate.

MPNRF has long felt that whatever the outcome of a trial, the community is served by finding out what happened. We've seen trials come and go, always with reports of some patients experiencing benefit. While our focus is funding research that can effect patients' lives and change their prognosis, we also want to advocate for the release of information that could provide clues to what drugs work, in what patients, and why, in the event that another researcher or company may pick up the ball, even for failed trials.  [...]


Thalidomide and Me by Landon Y. Jones

Note: Below is a first person account by an MPN patient of his experience with a clinical trial. This is not meant as an endorsement of the drug, company or institution involved. If you would like to share your story of being on a clinical trial please send an email to [...]


Goodbye Marge, dear friend



January 11 is International Thank You Day!

January 11th is International Thank You Day, and we couldn't help but reflect on how patients and family members trust us to give them the latest information about clinical trials, new therapies and innovative research. We in turn learn from YOU about what is of highest concern and interest.  [...]


Novel Therapies: Close but not close enough

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.  [...]


Patient guest blogger Melissa Wilder talks diagnosis,health wellness & nutrition

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. [...]

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