The world of myeloproliferative neoplasms (MPNs) has witnessed a surge in scientific breakthroughs. From detecting genetic drivers as early as in the fetus to the unprecedented pace of new drug approvals, the momentum is changing how this family of blood cancers is understood and managed.
Increased attention and industry investment have led to a growing number of treatment options – five medicines available today and four drugs in late-stage trials testing new mechanisms, which have the potential of becoming available in the next two years. Even more exciting is the robust early-stage pipeline of novel agents, including the first potential immunotherapies targeting CALR-positive patients. Today, we can manage symptoms longer, with more treatment options, driving better outcomes. But much work lies ahead to modify the underlying disease.
Despite promising advancements in research and new treatments, MPNs are still complex and a diverse family of blood cancers. They are chronic and can be debilitating to many patients, compromising career aspirations and fulfilling family milestones. In contrast, others live for decades in relatively stable health, able to work, travel, and raise families. Yet all patients live worried and waiting for the unknown: will I get worse and when?
Though they are all rooted in mutations of blood stem cells, MPNs are heterogeneous cancers. Differences in clinical presentation lead to varied patient experiences, differences in diagnostic tests, treatment approaches across various care settings, and how the diseases progress within an individual patient. MPNs remain unpredictable, leaving many questions unanswered.
All of this has propelled MPNs into the spotlight… and now that we’re here, we must work harder, faster, and smarter.
The rapidly changing landscape and increasing complexity have required us to adapt our approach at MPN Research Foundation. We are working harder, faster, and smarter, enhancing our team and devising a research strategy to be more directive while continuing to seed the serendipitous spirit of discovery.
Our foundation is rooted in the experiences of patients and caregivers. Since the beginning, we have worked hand-in-hand with the doctors who treat these patients and wear the extra hats of scientists in search of answers to bring the best outcomes possible for MPN patients. Industry leaders look to us to bring the voice of the patient and insights from the MPN community to inform their efforts as they work to produce more effective and impactful medicines. We have the opportunity and obligation to listen and learn from all these perspectives to advance research programs that achieve better outcomes faster.
In this crucial moment, what we do next matters.
MPN Research Foundation will leverage our convening power and harness scientific leadership internally and externally to explore new frontiers of investment. We aim to explore areas such as activating the body’s natural immune system or understanding epigenetics which are the factors that affect how the gene mutation behaves, such as other diseases, environment, and nutrition. With new treatments becoming available in a short time, we have new questions to answer:
- How should patients and their doctors decide to sequence and combine treatments over the course of the disease?
- What can we learn from individual responses to treatments over time?
- Can we start to tailor personalized plans to get the right medicines to the right patients at the right time?
And we have long recognized the need for a deeper understanding of disease progression, specifically identifying patients at higher risk of advancing versus those who may stay in relatively stable health.
To address these issues, we have learned that it will require large-scale data registries and real-world evidence strategies from across this heterogeneous patient population to complement and inform the innovative lab and clinical work that leads to new treatments. Now is the time for the MPN Research Foundation to harness our convening role and engage patients, doctors, and drug-makers at every step to build a data source that might start to reveal the answers we all seek.
Anchored by the drive to study MPN disease progression, we are laying the groundwork for large-scale, data-driven research that may help us eventually uncover markers of disease progression and possibly so much more – such as finding the right treatment for the right patient at the right time.
No doubt, these are complex and compelling questions. The unknowns drew me into the world of MPNs to be part of the solution. Imagine our impact on many lives if we can unravel even a few more mysteries.
I hope you will continue to walk this road with MPN Research Foundation as we stay focused on advancing research and choose to be part of the solution.
All the best in the new year,
Kapila Viges
Chief Executive Officer