Science & Medicine

An MPN Patient Registry

myMPN — an MPN Patient Registry is coming.

Zhenya Senyak

The conference room gave off the stale smell of too many meetings over too long a period.

The presentations followed, one after another. Stories, lists, citations, white boards, slides, polite applause.

After months of bouncing ideas off her colleagues back in Chicago, after anticipating the flash and high tension energy of New York City,  Michelle Woerhle found herself  at the Patient Registry Bootcamp organized by The Genetic Alliance.   She had her place at a very ordinary conference table where something extraordinary was happening. The birth of a game changing project.

For Michelle and her colleagues at the MPN Research Foundation, a dream that seemed impossibly difficult years earlier began to take solid shape.  What if MPN patient data, demographics, clinical records and self reported symptoms could all be collected in one place to create a giant database.  Would that not clarify the need for specific new meds and quantify the available market so a drug company would put up the money to fund research and development?

And if such a master database existed couldn’t it be used to inform patients when a clinical trial was coming on stream to address those specific needs? Suddenly the hit and miss, guess and by golly approach to MPN drug development would take on new sharpened focus, And patients would have easier access to current applicable clinical trial information.   It was a win-win possibility, 

This Spring the MPN Research Foundation plans to roll out myMPN, its beta version of the MPN Patient Registry.  Michelle Woerhle,  one of the new generation leaders of the MPN Research Foundation,

Lindsey Whyte

is heading it up along with colleague Lindsey Whyte.  And you, the MPN patient, are the critical element.You’re not only invited. Your participation is essential to make the project work,

Expanding the reach

Back in 2012,  we noted a major shift in strategic direction taken by MPNRF.  The primary backer of basic research for an MPN cure, the Foundation had already racked up an astonishing  record. This small patient-funded group had already funded all or part  everything from Jakafi and the MPD Research Consortium, to tissue banks and the discovery of the CAL-R mutation. The Foundation jumpstarted myelofibrosis research with the MF Challenge and would soon lead the charge in MPN immunotherapy and CRISPR gene editing. Its pure research grants now total over $11 million.

In expanding its MPN mission, there were risks.  Could this small Chicago group go beyond backing scientific research and provide badly needed support to the MPN patient community?  Top of the list was the need to translate research into drugs that can cure myeloproliferative neoplasm… or greatly relieve its impacts. And then there was the need for independent publishing of news, scientfic findings, clinical trials.  And maybe most important, securely collect the massive data associated with existing MPN patient conditions, treatments, outcomes, disease progression to better inform biotech researchers.

Over the past half decade as new younger leadership emerged, the answer became clear. Of course they could! The website, — —  newsletter, and social media outreach all reflect  the Foundation’s direct patient engagement outside the labs and academic research settings.  Now, with the unveiling of myMPN, the MPN Patient Registry,  the Foundation is closing the loop by bringing together a collaboration of patients, researchers, drug makers, the FDA and big data into an integrated system designed to streamline the path from lab to bedside.

What is a Patient Registry anyway?

Patient registries may vary in purpose but have become nearly universal in health care systems. The first cancer patient registry was organized at the Yale-New Haven Hospital in 1926. The National Cancer Institute set up a national cancer patient registry in the 1970s and before the end of the 20th Century most States had passed laws requiring cancer registries.  But no such registry exist for the rare blood cancer, MPN.

According to the National Institutes of Health, “Registries can be used to recruit patients for clinical trials to learn about a particular disease or condition; to develop therapeutics or to learn about population behavior patterns and their association with disease development; developing research hypotheses; or for improving and monitoring the quality of health care.”

Patient registries can also be used to monitor outcomes and study best practices in care or treatment, collect data to answer a specific research question  Some patient registries may include the collection of tissue or blood samples.

And while there is little doubt of the value such a registry would add to stimulate new focused research and clarify the specific MPN patient community’s therapeutic needs, there are some major landmines buried along the pathway to successful implementation.  The practical application of all this big data is to develop and test drugs…and that means clinical trials.

The FDA is clear about the primary value of Patient Registries.The first two items heading its list are the improvement of clinical trial recruitment.

And clinical trial means going to bed with Big Pharma, the deep pockets funding trials.  One of the risks faced by MPNRF in expanding its mission was the expansion of its donor base. To fund its more aggressive research activities plus patient outreach services, MPNRF turned to partnerships with corporate sponsors, companies “in the biotech, pharmaceutical and related industries.”  A web page outlining its policies, identifying its sponsors and describing steps it has taken to erect a firewall to prevent undue influence can be accessed here.

These partnerships — and expanded relationships with other foundations like the Leukemia and Lymphoma Society —  have permitted a much higher level of investment in a more diversified range of research without any contr0l or influence from sponsors. The MPN Patient Registry however enables patients to learn of applicable clinical trials while providing drug companies with a powerful tool to organize clinical trials likely to succeed and enhance patient recruitment.

“By establishing a centralized repository for patient data that is secure and available 24/7,”  says Michelle ” we hope that the numbers of patients with specific MPNs suffering specific symptoms or meeting other criteria will provide compelling proof to… pharmacetuical companies and other sponsors to move forward with a therapy.”

The other side of that coin is improved access to clinical trials for patients. “What if we let patients know … when there was a clinical trial that was relevant to them? Patients could find out sooner that there are trials that they are eligible for and contact their doctors office to proactively move the process along more quickly.”

Together these two elements clearly make the clinical trial process more efficient. Both elements however have MPNRF collaborating with drug companies to enable more effective design of  clinical trial based on need while funneling patients more rapidly into the cohort through enhanced recruiting.

One could well argue that is either a very good or very bad thing depending on one’s view of the clinical trial process.  

On the down side: The track record for clinical trials in general is fairly abysmal but in MPN related trials it has been disastrous. The most famous recent case might be the abrupt abortion of the Sanofi Fedratinib (SAR302503) trial at the end of Phase III leaving a wake of dead and injured patients due to unreported Wernicke Syndrome. Major trials — Pacritinib and Imetelstat — have been interrupted by the FDA mid-course.  Other trials with no clear benefit to patients simply drone on taxing patient time and resources.  Beyond a single new drug approved in 2011 the clinical trial process has yielded no addition to the MPN formulary despite the expense of several hundred millions of dollars involving thousands of sick MPN patients.


And yet, the hard truth:  No trials, no drugs.  Although true enough, that may not be entirely true forever.  For one thing the two main MPN drugs — hydroxyurea and the interferons — are both prescribed off label. They have never been FDA approved for MPN application.  And as routine genetic sequencing continues along with electronic record keeping, it is not hard to imagine a computer-based step between mouse studies and human experimentation, a step leading to much more precise assessment of likely efficacy.

MPNforum asked Michelle about privacy issues in myMPN and about going direct to patients offering clinical trial information, by-passing the hematologist.

On privacy:  We will be able to push out notifications to people without seeing their identifying information (name, address). At the start of entering myMPN they will set their privacy settings, and can decide whether to share their contact information with us and others (or not). There’s also an “ask me first” setting, which allows them to be anonymous until they want not to be.

On direct notification: We will approach notifying patients in the registry about trials the same way we’ve approached letting patients know before; which is that we will share all pertinent information about any MPN related study whether or not we have received sponsorship from a company or not. The companies we meet with are all told this.   We will keep the Registry sponsors up to date about the status of the project, including some general reports to tell them how things are going. Beyond that, sponsors won’t have more or less access to individual patient information than anyone else. Anyone who wishes to run a query of the system will need to submit a request and be subject to the same standards.”

What about patient qualification to participate in a trial? “If a patient is alerted to a trial by us based on information they provide in the registry, they will be screened by the investigators running the trial to confirm their eligibility.  For our part, we will run routine quality checks on data within the registry based on guidelines set with our Steering Committee advisors (which includes Verstovsek, Mesa, Harrison, et al).  Any data we would present directly to the FDA would be accompanied by the caveat that it is self-reported.”   The FDA is looking more at patient reported data, quality of life and other measures as a means of assessing how to evaluate potential therapies.

Robert Rosen, founder of the MPN Research Foundation and CEO,  believesThe registry symbolizes new and expanded directions for the MPNRF. In addition to our core mission of promoting research we will now be providing a structure to analyze, evaluate and recruit patients for longterm studies. While we are starting with patient reported data we look forward to a time when we can add genetic sequencing and other clinically reported information thereby filling out the picture for patients clinicians, researchers and pharmaceutical companies seeking to improve our outcomes.”


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