I was hired by the MPN Research Foundation in May 2016 to spearhead the development and launch of the first ever patient driven registry. At the time I was working for another disease foundation managing research projects to find a treatment or cure for an ultra orphan disease that affects less than 100 people in the US. While not life threatening, the disease (Usher Syndrome type 3) causes progressive loss of hearing and vision over many years until the patients rely on cochlear implants or tactile sign language to communicate and are completely blind. In working with the patients afflicted by Usher syndrome, I began to understand the disconnect between the patient experience and the scientific process being undertaken in the labs and universities around the world. In cases where a disease progresses slowly or where there may be triggering events that cause progression, are there ways that patients can help the scientific process along? [...]
In a few days, the month will change to September, which is recognized nationally as Blood Cancer Awareness Month. While you are already aware of MPNs, or myeloproliferative neoplasms, there are many factors indirectly but adversely affecting people living with Polycythemia Vera, Essential Thrombocythemia and Myelofibrosis. A few we hear from patients frequently are: [...]
As the American health care law continues to evolve, the MPNRF is staying on top of how it is changing, and how people living with an MPN might be affected. This article from Kaiser Health News provided what we thought was a good assessment of where we are now, and what may happen: http://khn.org/news/senate-health-bill-would-revamp-medicaid-alter-aca-guarantees-cut-premium-support/ [...]
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. [...]
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 email@example.com [...]
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. [...]
About the Blog
Check back regularly for updates about what’s happening in the MPN community, from advocacy to research..