By Michael Andreini, President and CEO, Multiple Myeloma Research Foundation
Twenty-six years ago, when the Multiple Myeloma Research Foundation® (MMRF) was started, a multiple myeloma diagnosis was nearly synonymous with a death sentence. Less than 35% of people were alive five years after diagnosis, and the few treatment options that existed were largely ineffective. Today, thanks to groundbreaking research and medical advancements – many spearheaded by the MMRF and our partners – we’ve made tremendous progress in the battle against this disease. With over 15 FDA-approved therapies now available, the five-year survival rate has climbed to over 60 percent.
However, with progress comes complexity. While treatments and patient outcomes have improved, multiple myeloma remains an incurable disease with significant unmet medical needs. This year, nearly 36,000 people in the U.S. will be diagnosed. For some, existing treatments may be ineffective from the start, while others will struggle to find the right option. Furthermore, the very advancements that have transformed care are now creating new barriers to further breakthroughs, presenting new and equally pressing challenges that I’ll discuss here.
One of the most dangerous myths about multiple myeloma is that it is now a manageable disease and no longer a pressing medical challenge because we have so many approved therapies.
Ironically, the success of new treatments has made it harder to develop additional therapies. Large biopharma companies, seeing a crowded market with limited commercial opportunity, are deprioritizing and investing less in myeloma research. Early-stage, venture-backed biotech companies, who take on the majority of new drug development, are prioritizing other disease areas like solid tumors with greater perceived clinical unmet need. Moreover, these companies face significant scientific and financial risks – challenges that are even greater in today’s difficult funding environment. That’s why the MMRF started the Myeloma Investment Fund®: to provide financial and strategic support to companies advancing novel classes of medicines and advocate for these innovative platforms to be tested in myeloma. Today, our portfolio includes 20 companies, ensuring that promising breakthroughs have the resources to reach myeloma patients.
At the same time, U.S. federal funding for multiple myeloma is declining. The federal government has historically been the largest funder of biomedical research, but proposed cuts to the National Institutes of Health (NIH) put future budgets at risk.
Even before these potential cuts, funding for myeloma lagged behind. Although the National Cancer Institute (NCI) budget has grown over time, the myeloma specific budget has decreased significantly. Myeloma is almost 2% of all cancers, yet receives less than 1% of the NCI’s budget.
Funding for institutions is essential to bridge the gap to translate basic science into treatments that can be tested in clinical trials. Recognizing this critical need, the MMRF has long been the largest private funder of myeloma research, with annual investments now approaching the NCI’s myeloma-specific funding levels. Without sustained investment, innovation will stall, leaving patients with fewer options when they need them most.
While there have been significant advances in our understanding of myeloma, critical knowledge gaps remain. No one institution treats enough patients to represent the full patient population, and valuable clinical data is often locked away in institutional silos. This fragmentation prevents researchers from drawing broad, population-wide conclusions that could accelerate breakthroughs, including determining the most effective combinations, dosages, and treatment sequences for different patient populations.
Breaking down these data silos is essential for advancing myeloma research. Through decades of work and initiatives like the MMRF CoMMpassSM Study and Immune Atlas program, we’ve made our data freely available, aggregating patient data to identify patterns, uncover new treatment opportunities, and ultimately accelerate the path toward a cure. Our latest initiative in this space is the MMRF Virtual Lab: an online research environment that provides the most comprehensive and open-source data in multiple myeloma research on a single platform.
While the most recent novel therapies like CAR T and bispecific antibodies have transformed multiple myeloma treatment, they are not one-size-fits-all solutions. Given the genetic complexity of the disease and systemic barriers to quality care, many patients don’t benefit equally. Significant disparities persist among patients, especially those who have high-risk disease, live in rural or community settings, and are of lower socio-economic status. These disparities also impact racial/ethnic groups such as Black patients, who continue to experience lower survival rates.
While not unique to multiple myeloma, clinical trial representation remains another key barrier. Historically, trial participants have been younger, healthier, and not racially diverse. This makes it critical to conduct trials that reflect the real-world makeup of the U.S. at large to ensure new therapies work for all patients.
To help bridge these gaps, we place an emphasis on providing patient navigation services and education as we believe that patients who are informed about their disease engage actively in treatment decisions, leading to better adherence to therapeutics, quality of life, and outcomes. We also lower barriers to patient participation in clinical trials conducted by our Multiple Myeloma Research Consortium® (MMRC®) by having broad inclusion/exclusion criteria and providing financial and travel assistance to patients.
For more than 25 years, the MMRF has remained steadfast in its mission – to accelerate cures for all multiple myeloma patients. Since our founding, we’ve raised over $600 million for research, opened nearly 100 clinical trials, and helped bring more than 15 FDA-approved therapies to market, which have helped triple the life expectancy of myeloma patients and improved patient outcomes.
While the progress made is remarkable, there is still much work to be done. In times of uncertainty, organizations like the MMRF – unbound by federal funding constraints and able to serve as an unbiased third party – play a unique and critical role in advancing research that might not otherwise be possible. The MMRF will continue to lead with urgency, breaking down barriers and working with the academic, industry, and patient communities to drive meaningful progress and impact in myeloma.
