Tuesday, March 10, 2015

2015 MPN Challenge Grant Program

The 2015 MPN Challenge is a grant program supported jointly by the MPN Research Foundation and the Leukemia & Lymphoma Society. This grant program represents an important partnership between MPNRF and LLS, who share a mission to advance the scientific understanding of MPNs and bring new treatments and the hope for a cure to patients with these rare diseases.

We hear from patients with MF, PV and ET and they have concerns about symptoms, treatment options, and quality of life, but their most pressing question is – will my disease progress, will I get MF, will I convert to AML?

The emergence of new treatments for MPN patients is encouraging, but until there is proof that these emerging treatments actually change the course of these diseases, these fears will remain. The MPNRF and LLS want to change this with a focus on new approaches towards MPN research that may change the trajectory of these diseases. We hope that independent researchers will join us in an attempt to achieve that goal.

The 2015 MPN Challenge

The goal of the 2015 MPN Challenge is to ensure that MPNs are evaluated as candidates for new transformative technologies currently being investigated for other cancers. We want to attract investigators in other fields whose research may apply to MPN research.

Why this Approach is Appropriate Now

Based on the response to the MPN Challenge in recent years, MPNRF and LLS are encouraged that ‘paradigm-shifting’ strategies identified in other areas of cancer and basic biology research can be useful in the search for MPN treatments. In 2014 alone, there have been significant advances in immunotherapy, gene editing and other technologies as applied to solid tumor and other cancers. If applied to MPNs, these new strategies, in combination with currently emerging treatments, could truly change the course of the MPNs.

Focus Areas for 2015 MPN Challenge Grants

To guide us, in November 2014, the MPN Research Foundation convened a group of scientists from both academia and industry to discuss the unmet needs in MPN research. This diverse group of current MPN Challenge grantees, academic scientists and representatives of companies currently involved in MPN treatment development held a lively discussion that resulted in the following list of focus areas for the 2015 MPN Challenge:

1. Targeting the Malignant JAK2 Clone

Research in this area will provide a better understanding of the importance of the JAK2 allele burden and mechanisms for reducing it in MPN patients, including the following areas:

• studies aimed at determining the significance of JAK2 allele burden as a diagnostic tool.
• research aimed at identifying and testing JAK inhibitors that inhibit the mutant and not the wild-type allele.
• preclinical testing of new agents that demonstrate potential to reduce the JAK2 allele burden.
• basic research and preclinical testing of agents that target the malignant stem cell specifically.

2. Application of Immuno-therapy Approaches to MPNs
This research will investigate the potential for immunotherapy or immune manipulation. Proposals for immune-therapeutic approaches to the broader spectrum of MPNs could include:

• Assessment of the potential role of chimeric antigen receptor therapy (CAR-T) to MPNs.
• Exploration of the role of immune checkpoint regulators in MPNs, e.g., CTLA-4, PD-1, PD-L
• Preclinical testing of antibodies that target the CALR mutated allele.
• Therapy targeted to inflammatory cytokines and/or cytokine receptors and their downstream effectors.
• An understanding of how to combine any of these with evolving therapies for MPN and in particular, since newly approved agents such as pan-JAK inhibitors have potential to impair immune cell activation and function, how or should they be used with immune-modulatory agents.

Proposals that have clear translational potential will be prioritized. The best proposals will include a strong collaboration between current immunotherapy experts and MPN specialists.

3. Gene Editing as Applied to MPNs.
We want to study whether CRISPR or other gene editing technologies can be applied to MPNs. Our primary objective is to make sure that MPNs are on the table as these technologies are developed. The best proposals will include a strong collaboration between current CRISPR experts and MPN specialists.

4. Additional New Mechanisms of Action.
We continually seek proposals for basic research and preclinical testing of new and innovative mechanisms of action to bring them to the point where other funding options are being directed to these new approaches.

Proposal applications are due April 1, 2015. All proposals will receive an initial review. A Scientific Peer Review Committee will meet May 29, 2015, conduct a final review and make recommendations. It is anticipated funding start date October 1, 2015.

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.