The MPN Research Foundation (MPNRF) was founded over two decades ago to stimulate the acceleration of new therapeutics to treat and one day cure myeloproliferative neoplasms (MPNs), a group of rare, heterogeneous blood cancers. Kapila Viges, CEO of MPNRF, and Barbara Vanhusen, chairman of MPNRF’s board of directors, share how the organisation has flourished through its commitment to collaboration, bridging patients and caregivers, researchers and clinicians, industry and advocates

The founder of MPNRF was a man named Robert Rosen, who was diagnosed with polycythaemia vera in 1997. “At the time, there was nothing known about it; there was little to no research happening, and the myeloproliferative neoplasms (MPNs) weren’t even classified as cancers. It was at a time when rare disorders did not receive much attention,” Barbara explains. Finding little scientific inquiry into the underlying cause of his disease, Robert knew he was unlikely to see new treatments on the market in his lifetime. With the help of other patients, Robert founded the MPNRF to fill a gap in medical research. Sadly, Robert died in 2018 following complications from a stem cell transplant, but the valuable work he started continues today.

“We started raising money to fund research, finding out who the best and brightest blood cancer researchers were and chasing them down,” Barbara says. “The first project the foundation ever funded focused on understanding the genetic origins of myeloproliferative disorders. This set the groundwork for much of the MPN research that followed.”

 “Since the first project, the MPNRF has become increasingly influential in the research community by identifying the highest unmet needs in research and collecting funds to put towards them,” Kapila adds.

Many rare disease organisations do incredible work supporting patients to live their day-to-day lives. The MPNRF approach is predominantly focused on having a long-term impact through scientific research. “By focusing on research, we can help many more patients as opposed to a few at a time,” Kapila says.

Thanks to the hard work of the MPNRF’s global network of collaborators and the generous funding received, the MPNRF has funded over 80 projects and awarded $16 million to MPN blood cancer research since 2000. This research has explored research gaps that can help accelerate the understanding of MPNs, finding answers that can drive treatment options and open the possibility of a cure. “It’s exciting to see the research landscape change and evolve rapidly over the last few years. We believe the MPNRF had a role in activating that evolution when the field was nascent,” Kapila says.

By visiting the MPN Research Foundation’s website patients can search for a clinical trial that matches their MPN diagnosis and treatment history using a personalised clinical trial finder, powered by Trialjectory.

“Our role in patient advocacy is to ensure we are bringing the patient voice into the earliest stages of research and development.”

Kapila

The MPNRF held an externally-led patient-focused drug development meeting in 2019 with the FDA. The meeting provided patients with a long-awaited opportunity to educate representatives of the FDA, as well as biopharmaceutical representatives and researchers in the field, about the challenges and needs of the MPN patient community.

“We produced a Voice of the Patient report in 2021 which captures 300 patient voices to better understand their experience and what their respective journeys have been. It illustrates the unmet needs that patients have identified in terms of treatment and the uncertainties of the disease to bring those insights into the research paradigm.”

MPNRF hopes the report will help stakeholders understand that patients have several concerns and unmet needs:

1. Uncertainty about disease progression and unpredictability of symptoms instil a fear in patients
2. Heterogeneity of clinical experiences within indications and across the spectrum of MPNs
3. Treatment options are severely limited and those that have shown efficacy can be difficult to tolerate
4. Patients report symptoms that are not well controlled with current therapies
5. MPNs are not adequately understood by health care providers
6. The myth that MPNs only affect people of middle or advanced age

The MPNRF brings patient voice into pharmaceutical industry development programmes and uses opportunities such as reviewing clinical trial protocols as a chance to leverage the patient experience and facilitate new approaches.

“We convene the communities and actively bring together the patient, research, clinical, and pharma industry voices to align our agendas and solve unmet needs.”

Kapila

Kapila explains that the MPNRF has taken a new approach this year to not only fund high-impact translational research projects, but also fill funding gaps and help restart projects that have come to a standstill for various reasons—for example, the pandemic caused projects to stall. The 2022 Thrive Initiative aims to address these other systemic gaps in funding. “A few more steps, maybe, of data collection or further validation, could enable a very promising idea or an innovation to get closer to the clinic instead of languishing,” Kapila explains. “We want to make sure our most promising projects have a chance to move forward and not get stuck because of circumstances that could be resolved with a small boost in funding.” In terms of innovation, the 2022 Thrive initiative also aims to attract new innovators to study MPNs. Experts who have had breakthroughs or addressed scientific problems in related blood cancers have an opportunity to apply those ideas to the field of MPNs, giving patients more thought leaders working on their behalf.

The 2022 Thrive Initiative also has a funding category for junior investigators. “It can be difficult for young investigators to get their foot firmly established in a disease area and sometimes they end up pursuing research where the funding is and not necessarily where their interests lie. We want to encourage more of those younger, innovative, fresh-thinking perspectives to come into the MPN space and help change the professional trajectory of young researchers,” Kapila says. This gap funding serves as a nice compliment to ongoing funding mechanisms such as the MPN Challenge and collaborative initiatives such the MPN Interferon Initiative completed last year. For several years MPNRF has also been focused on funding studies to better understand MPN disease progression, perhaps the single greatest area of concern for patients and doctors.

Stepping into the role less than two years ago, Kapila is committed to leading the MPNRF into its next chapter and influencing new directions for the organisation. “We have our eye on expanding our reach in both the patient community and the clinical and research community,” Kapila explains. “It is about being able to build partnerships, collaborations and relationships between more stakeholders and bringing our global community together more cohesively.”

What energises and motivates me is knowing the scale of the impact it would have on so many patients if we can break through the scientific uncertainties.”

Kapila

Many organisations throughout the world, including leading cancer organisations, support patients with myeloproliferative neoplasms (MPNs). Here we introduce just some of the organisations that specifically focus on the MPNs—there are many more around the world. Their activities include advocacy, educational programmes and involvement in research

United Kingdom

United States

Australia

Disclaimer: The list above is not exhaustive. Inclusion or omission from it does not reflect an endorsement or otherwise of that group by RARE Revolution or GSK.

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NP-GBL-MML-WCNT-220004 / Date of preparation: November 2022 Funded by GSK

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