Tuesday, February 24, 2015

Research Fuels Progress Against Cancer

Research Fuels Progress Against Cancer


Research continues to be our best defense against cancer. It improves survival and quality of life for millions of people by spurring the development of new and better ways to prevent, detect, diagnose, treat, and, increasingly, cure some of the more than 200 diseases we call cancer.


This progress against cancer is the result of the dedicated efforts of many people working together as part of the biomedical research community. These stakeholders include patients, survivors, family members and friends. Also included in this community are the clinicians and academic researchers. The biotechnology, pharmaceutical and diagnostic companies play an important role in the development of ways to diagnose, treat, detect, prevent and cure cancers. Policymakers and citizen advocates, advocacy and philanthropic organizations have an important role to play. Without continued investments in biomedical research through funding agencies like NIH and NCI, progress against cancer is in jeopardy.


Although the Foundation does not fund clinical trials directly, we have helped put a spotlight on the need for an increased focus on ET, PV and MF research. Our efforts have seen results. While fewer than 20 clinical trials for MPN treatments were conducted from 1995-2005, there have been over 750 trials since 2005.


Clinical trials give patients a chance to try new medical treatments not yet available in the marketplace. During the course of a trial, data is collected recording all observations related to lab results, the patient’s progress (or lack of progress), effectiveness, and side effects of the treatment. Clinical trials are conducted at many sites but usually they are conducted at specialty clinics or teaching medical centers.


There are 4 types or phases.


Clinical trial phases

Phase I

Phase I studies are designed to determine the best dose of a therapy and how humans process it, as well as to identity any potential toxicities. These first-in-human studies can also demonstrate early effectiveness or clinical results.


Phase II

Phase II studies determine continued effectiveness of a therapy in a particular disease or a larger group of patients, in addition to continually monitoring for adverse events or potential toxicities.


Phase III

Phase III studies are large trials designed to determine curative value as compared to standard of care (placebos are rarely used in cancer clinical studies).


Phase IV

Phase IV studies are also known as post-marketing studies. They provide additional effectiveness or “real-world” data on the therapy and are conducted after approval by the FDA.


There are many factors to consider when thinking about joining a clinical trial. There are no guarantees that the treatment being tested will be successful. Travel to a clinical trial site may be difficult and costly. The trial may involve more frequent diagnostic tests, such as a bone marrow biopsies. Participants may be asked to stop taking certain medications that would interfere with trial results. 


Whether a trial is right for you is a personal decision that should be discussed with care-givers and family members. The final decision to enroll in a trial should be considered carefully.

Tuesday, February 10, 2015

The Strength of the MPN Community

The strength of a community is determined by how well it is able to take care of itself.


How strong is the MPN community?


When you do a little research, you immediately see how vibrant this community is. There are so many MPN chat groups, advocacy organizations, Facebook pages, Twitter accounts, and forums we cannot count or list them here. People can get encouragement, information, friendship. People can also get support from organizations not just focused on MPNs. Patient Power has included MPNs in their focus. The National Organization of Rare Disorders (NORD) also provides support for MPN patients. The Leukemia and Lymphoma Society is one of our important partners.


It is the spirit and generosity of MPN patients, family and friends that enables the MPN Research Foundation to fund research that promotes medical innovations that have potentially high rewards. The MPN community is small in population, but immense in spirit and generosity.


When you read this Blog will get a snapshot of the MPN community. You may have heard that the MPN Research Foundation has helped fund every major advancement in MPN science. In the coming months this blog will provide readers with information about how our current research focus is pushing MPN Science. MPN Research Foundation leadership will be providing their perspective on the where MPN science is going and why. You will receive firsthand accounts from patients and families who are “Leading by Example” by hosting events that help raise funds and MPN awareness. We are inviting MPN patients to “Share Your Journey”. 


We also need to hear from you. We want to address the things what will be helpful for you. The only time a comment is not helpful is when there is no comment.


Want to learn more now?


Our website has an article from the New England Journal of Medicine report on randomized trial of Ruxolitinib vs phlebotomy in Hydroxyurea-resistant PV - See more at: http://www.mpnresearchfoundation.org/NEJM-evaluates-ruxolitinib-for-hydroxyurea-resistant-polycythemia-vera-vs-phlebotomy#sthash.RpkqaN5l.dpuf


Did you know February 4th was designated as World Cancer Day by the United Nations. If you visited our Facebook page you would have.


Thanks for reading.


Enjoy our community.

Thursday, March 13, 2014

FDA issues verbal hold for Geron's Imetelstat aplication

Geron has been given verbal notification from the U.S. Food and Drug Administration (FDA) that its application for imetelstat has been placed on full clinical hold, affecting all ongoing company-sponsored clinical trials. A full clinical hold is an order that the FDA issues to a trial sponsor to suspend an ongoing clinical trial or delay a proposed trial.

The clinical hold affects the remaining eight patients in the company's Phase 2 study in essential thrombocythemia (ET) or polycythemia vera (PV) and the remaining two patients in the company's Phase 2 study in multiple myeloma. Geron released astatement as well as a recording of a conference call to go over details.

This comes only a few months after Sanofi's clinical trial was discontinued due toneurotoxicity issues.

Tuesday, February 25, 2014

Crafting the MPN environment

The World Orphan Drug Congress recently put out an independent study of the most researched rare diseases. We were shocked to see that the MPNs (MPDs on the list) ranked No. 10 of 25. 

This was not the world we knew in 2000. 

Ten years ago there was no infrastructure for driving research into PV, ET, and MF. There was no buzz around MPNs. No organization existed that was solely focused on funding research into MPNs. 

We had to build our own house. 

There were already some big names invested in MPN (at the time MPD) research who did foundational work. But there was no buzz around MPNs, the kind that attract and keep the interest of young researchers. The kind of buzz that will get the attention of pharmaceutical companies. 

The JAK2 discovery changed all that. 

In 2006 the MPN Research Foundation initiated the MPD Research Alliance, a program that formalized collaboration among researchers at Harvard, UIC and Mayo Clinic who were tasked with translating the discovery of a genetic marker for the majority of PV patients and half of all MF and ET patients into something that helped them.  As a result, today we have one FDA approved drug for symptoms of MPN - Jakafi - with many more drugs in clinical trials. 

But the Foundation didn't stop with the JAK2 discovery. In 2008 we opened up our research programs to any ideas that showed promise in combating PV, ET, and MF. Through this work we've helped discover yet another genetic mutation - CALR - that explains nearly all the non-JAK2 MPN. The CALR discovery has been characterized as a "game changer". 

Just as we didn't stop with the discovery of JAK2 or CALR, we see no reason to stop now. We are in the middle of the war on MPNs, not at the end, and we won't rest until all those with PV, ET, and MF have the answers and the medications they need. 

Thursday, January 2, 2014

Video shows the impact of the Foundation on PV, ET, and MF

Watch the video explaining the need for MPN research and meet patients living with PV, ET, and MF, and the researchers helping to find a cure. 

Sunday, November 24, 2013


by Guest Blogger Lina http://linampn.com/

I am sure many of us have seen the daily Facebook posts during the month of November about all of the things our friends are thankful for. Don’t get me wrong. I’m happy that people are recognizing how blessed they are; that is a wonderful thing. My argument is that you should be thankful every day of the year, not just one month out of the year.

Now, I realize that sometimes it’s hard to be thankful. It is much easier to think of the negatives: the bad day at work, the argument with your spouse, the bills that need to be paid, or any of the other things that may be happening. The thing is, though, all of those negatives are what make the positives that much better. Without them, we would have no idea how good our lives really are. 

Often, someone who has just learned about my health situation  will say something like “Oh, I’m so sorry!” Well, I’m not. I’m not sorry about it at all. My ET has changed my life. Not all for the good, I’ll admit, but it has helped to make me who I am. It has made me a much bolder person than I was before I was diagnosed. It has helped me to see the brighter side of things more often, which has made me more able to see the things for which I am thankful. 

I am thankful for my family, the people who have known me my entire life, and have supported me through all of my choices (good, bad or otherwise). They were there for my diagnosis, and treatment, and all of the other excitement that came along with it. Without them I don’t know what I would have done. 
Lina and her husband

I am thankful for my husband. I was diagnosed just after we met in college, and even after 
seeing what I mess I was, he still stuck with me. I love you, M. 

I am thankful for my friends. Even though I’m not much fun sometimes, and can’t go out as often as I’d like, they’re still there for me.

I am thankful for my dog and writing-buddy, Tesla. Whenever I’m having a bad day, not feeling well, he’s there. No matter how long I’ve been gone he is always happy to see me, and ready to greet me with a wagging tail and a slobbery kiss. 
This is just the short list, but I am thankful for so many things in my life, and I am thankful for these things every single day.

Even if you can’t see it right now, there are things to be thankful for. My suggestion is to find the things in your every day life for which you are thankful. The girl at the coffee shop remembered your order. The lights were all green on your drive to work. The cop let you off with a warning. Whatever it may be, there are things in your life that are wonderful.

I would like to add that I am thankful for each and every one of you who may be reading this right now. I hope in some small way I have helped you to look at your life with a different perspective, and see the great things in it!

As always, be assertive. You are your own best advocate.

Until next time,