Identifying and Validating Actionable Biomarkers in MPN
Research Spotlight on Rebekka Schneider, MD, Director, Department of Cell and Tumor Biology, Aachen University, Germany, and Erasmus MC Cancer Institute, The Netherlands.
In MPNs, your blood forming stem cell acquires a mutation, and this leads to more proliferation. In the beginning, you have too many blood cells. Then, in the course of the disease, these cells somehow activate non-blood forming cells and lead to fibrosis or scarring of the bone marrow.
A pathologist by training, Dr. Schneider explains the value of her work in this way. “Once you have fibrosis, your bone marrow can’t make blood cells anymore. It’s full of the scar tissue, which you can see under the microscope. So, you can see the scar tissue, but you can’t say in a patient, this patient is at risk to develop fibrosis. You can only say the scar tissue is there, or it’s not there. What we really want to understand is what happens earlier.”
The question she and her international team is working on is this: “Can we detect something that would allow us to diagnose a patient before the scar tissue is actually there . . . to prevent the scarring from happening . . . to help identify patients at risk?” They have identified certain genes or markers that have the potential to do this. And they are now examining these specific markers, and their relationship with each other, in larger patient cohorts.
Specifically, the focus of the MPNRF-funded project is on two potential biomarkers of MPN progression, SLAMF7 and CXCL4 in blood and bone marrow cells.
As Dr. Schneider works in Germany on CXCL4, her research partner, Ann Mullally, MD, of Brigham and Women’s Hospital, is examining SLAMF7 in monocytes in bone marrow cells in her Boston lab.
For more about this project and about an upcoming clinical trial based on other MPNRF-funded work by Dr. Rebekka Schneider, click here
MPN Research Foundation Launches New Clinical Trial Finder
Now live! An easy-to-use tool that matches an MPN patient with current clinical trials they may be eligible to participate in, based on their individual medical profile. The MPNRF clinical trial finder
is powered by Trialjectory, an AI-powered technology platform for cancer patients.
Through a guided search on the MPNRF website, patients, caregivers or care partners are asked a series of questions, based on the inclusion and exclusion criteria for MPN clinical trials. Within minutes, a personalized list of potential clinical trials is generated.
Since the list is tailored to each patient’s profile, it will be shorter and more relevant than what was previously accessible. Directly through the Trialjectory platform, patients can email the physician managing their MPN, with a personalized message and specific trials they want to discuss.
A multilingual patient support team is available to help in English, Spanish,
Thank you for the sponsors supporting our efforts to raise
awareness of clinical trials
Participation in Clinical Trials: What Patients Can Learn
When new treatments enter the clinical trial phase, researchers rely on volunteers, both healthy and those with the target disease, to evaluate if they are safe and effective. Underscoring the importance of MPN patients learning the details about clinical trials, MPNRF CEO Kapila Viges joined other leaders in the MPN community at a May 7th CURE Educated Patient® MPN Summit.
Her presentation included key information about understanding and accessing MPN clinical trials, including the unique value of the MPNRF’s new clinical trial finder. Without patient volunteers, she stressed, new drugs won’t be reviewed by the Food and Drug Administration (FDA) and approved for patient use.
People living with an MPN, caregivers and others joined to learn more about the latest research in cancer care, emerging therapies, and general wellness. You can watch the entire webinar here
ESH 9th Translational Research Conference:
This year’s virtual conference by the European School of Hematology, May 12-15, hosted approximately 1600 scientists, clinicians and other supporters of the MPN research and patient community. The program focused on the latest scientific and clinical developments, presented as individual talks, live panel discussions and posters by experts in their field. The MPN Research Foundation was a proud sponsor of this conference, supporting their scholarship fund.
Of special interest were the topics of disease pathogenesis; diagnosis and prognosis of MPNs; therapeutics, including both current and novel strategies in clinical trials; and a look into the future on effective ways to determine which patients are the best candidates for a specific treatment approach.
New murine models of the disease continue to provide valuable information on the mechanism of action of drugs (see story below), and insights into signaling pathways other than the JAK2 pathway, which may impact the pathogenesis of MPNs. However, results from highly sophisticated single cell technologies that focused on bone marrow studies continue to highlight the complexity and heterogeneity of these diseases.
Lastly, there was a clear emphasis for this field of medicine to investigate beyond today’s therapies, which mostly improve blood parameters, symptoms, and reduce cardiovascular complications. The future looks toward more therapeutic options that target and eliminate the MPN stem cell clones, with the goal of minimizing disease burden, preventing progression, improving long-term survival and quality of life.
Interferon Poster Describes Work Funded by MPNRF
On day one of the ESH 9th Translational Research Conference, a project funded as part of MPNRF’s Interferon Initiative was featured on the Mentored Poster Walk. The work was presented by Jeyan Jayarajan, a PhD student from the laboratory of Michael Milsom, German Cancer Research Center in Heidelburg, Germany. The poster was titled: Interferon-alpha treatment results in the depletion of dormant JAK2-mutant HSCs in a murine model of polycythemia vera. This study provides one explanation as to how interferon-alpha can reduce the mutant JAK2 hematopoietic stem cell allele burden.
Go the Distance for MPN Research
MPN researchers are truly amazing. They are deeply invested in improving the lives of those living with an MPN, through their work in the lab, in the clinic, and through education.
Throughout May, a few MPN researchers are participating in a pilot program by committing to share their physical activity, to raise awareness and funds in the fight against these rare blood cancers.
With only a few days left in May, please support one or all of our active researchers as they Go the Distance for MPN Research:
In addition to supporting MPN research needs today with a donation, we encourage you to explore other ways to give, such as including MPN Research Foundation in your will, or giving through a retirement fund. This ensures that the foundation can meet future research needs and have an impact on those living with an MPN for years to come. Learn more here: Charitable Giving | MPN Research Foundation
Clinical Trial Highlights
Company: Constellation Pharmaceuticals (MorphoSys)
Phase 2 trial enrolling MF and high-risk ET patients. Pelabresib is a BET inhibitor that is used on its own in two arms of the trial and in combination with ruxolitinib in additional arms.
Phase 3 trial for ET patients who have had a suboptimal response or did not benefit from hydroxyurea. Response of patients on this trial is compared to response of patients on anagrelide. Note that this interferon formulation (ropeg interferon alfa-2b-njft) was approved by the FDA for use in PV in November 2021 and is marketed under the brand name BESREMi®.
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