Recent headlines proclaim a ‘potential’ or ‘functional’ cure for multiple myeloma, but the fight against the disease must continue.
Nothing is more representative of medical success than eradicating a disease completely. However, during those crucial final stages, when a cure feels imminent, the danger of complacency becomes greatest. This loss of momentum can lead to a stalling or reversal of progress.
I fear this complacency is taking hold in multiple myeloma, a blood cancer that the Multiple Myeloma Research Foundation (MMRF), where I am president and CEO, has fought relentlessly to eradicate for more than 25 years. When the MMRF was founded, a multiple myeloma diagnosis was essentially a death sentence—fewer than 35% of patients survived five years, and the handful of available treatments offered little more than false hope.
The transformation since then has been extraordinary. Today, over 15 FDA-approved therapies exist, and the five-year survival rate has nearly doubled to over 60%. These advances represent thousands of lives saved and families given precious additional time together. But here’s the sobering reality: multiple myeloma remains incurable. This year alone, about 36,000 Americans will receive this diagnosis, and more than 12,000 patients—mothers, fathers, friends, colleagues—will lose their battle with multiple myeloma. We have come far, but we are not finished.
Recent headlines proclaiming a “potential” or “functional” cure for multiple myeloma reveal a troubling disconnect between public perception and scientific reality. While these therapeutic advances have undeniably transformed patient outcomes, they have simultaneously created new obstacles. The very treatments that now extend lives and improve quality of life have also introduced complex scientific, financial and logistical challenges that are difficult to conquer. The path forward requires confronting three critical realities.
1. Higher Bar, Higher Stakes
As multiple myeloma therapies become more effective, subsequent advances will cost more, take longer and yield smaller, more incremental benefits.
Early progress in multiple myeloma was achieved by targeting the disease’s most obvious, tractable mechanisms. Today, this creates two problems. First, the potential targets that remain are neither well known nor well understood. Second, clinical trials must show a greater effect size versus standard of care, which requires tracking more patients over a longer time period. Together, this creates an unfavorable environment for drugmakers, adding complexity, cost and risk while limiting commercial opportunities.
Overcoming this challenge will require thinking differently about drug development. Early-stage companies often have the most promising innovations but the hardest time raising capital. Without intervention, transformative therapies will die in laboratories and never reach patients who need them.
We must therefore provide firms that are willing to pursue new targets in multiple myeloma with more funding and strategic guidance to help bridge the valley of death. Through the Myeloma Investment Fund (MIF), for example, the MMRF deploys venture philanthropy capital—more than $23 million since 2019—to advance new therapies for myeloma patients regardless of market conditions or commercial timelines. Other similar initiatives could propel early-stage science into the clinic.
Additionally, biopharmaceutical companies should focus their drug development efforts in the areas of greatest need—for example high-risk multiple myeloma—to increase the likelihood of showing superior efficacy in a shorter amount of time. This will speed drug development and improve the probability that any one new agent will show a meaningful benefit to the patients that need it most.
Finally, the myeloma community should continue to work with the FDA to create faster, smarter approval processes that reflect how urgently patients need new treatments. A recent breakthrough shows this is possible: last April, an FDA advisory committee unanimously agreed to accept a new way of measuring treatment efficacy called minimal residual disease (MRD). Instead of waiting years to see an improvement in progression-free survival—time that many patients don’t have—clinical trialists can now, with great sensitivity, measure whether cancer cells have been eliminated from the body much sooner to support future accelerated drug approvals.
2. Embracing Complexity and Data Sharing
The “low-hanging fruit” in multiple myeloma are gone. The next generation of scientific discovery will be more biologically and technologically complex.
Early breakthroughs in multiple myeloma were informed by the most well-understood aspects of disease biology such as the cancer genome, unlocked by the advent of next-generation sequencing. Genetics doesn’t tell the whole story, however, and scientists are increasingly turning to the much more complex immune microenvironment for answers. Immune cells are highly diverse, single-cell analysis is expensive and the data generated are orders of magnitude larger than genomic datasets. This means significant computational and analytical challenges.
To meaningfully advance this field, we must invest in large-scale collaborative data generation projects that can achieve the scope necessary for breakthrough discoveries. The MMRF spearheads initiatives designed precisely for this purpose.
Equally critical is robust data sharing—another area where the MMRF has led. Through landmark initiatives like CoMMpass and the upcoming Virtual Lab, the MMRF has repeatedly demonstrated that organizations typically viewed as competitors can unite for transformative scientific progress.
When datasets from pharmaceutical companies, academic institutions and nonprofits are pooled—and especially now with the applications of AI—the collective intelligence generates actionable therapeutic insights with unprecedented speed and precision, far beyond what any single organization could accomplish independently.
3. Advancing Access
We must be relentless in overcoming “last mile” delivery and access barriers to get patients cures.
The cruel irony of modern multiple myeloma treatment is that our most powerful weapons remain out of reach for those who need them most. CAR T cell therapies represent some of the most groundbreaking advances in the past decade, capable of rendering multiple myeloma undetectable. Yet these treatments require specialized facilities, complex manufacturing processes and weeks of preparation, making them inaccessible to patients in rural areas or those without the means to travel to major medical centers. Removing risk evaluation and mitigation strategies will help but won’t do enough.
A breakthrough treatment that can’t reach patients isn’t a breakthrough at all. The next wave of multiple myeloma innovation must prioritize delivery and access with the same intensity we’ve applied to scientific discovery. We need therapies that are not just effective, but deliverable—treatments that can reach every patient who could benefit, regardless of their zip code or economic circumstances.
The progress in multiple myeloma treatment has been remarkable, but we cannot mistake progress for victory. Patients are still dying. Families are still losing loved ones. A cure remains elusive, and settling for “good enough” is not an option when lives hang in the balance.
Yet within this stark reality lies our greatest opportunity. The very challenges that seem insurmountable today—misperceptions about commercial opportunities, the complex biology, the fragmented data, the access barriers—are precisely the problems that demand the kind of bold, collaborative action the MMRF was built to deliver. We have the tools, the knowledge and the partnerships to break through these final barriers. What we need now is the support and unwavering commitment to use them.
The patients counting on us deserve nothing less than our most urgent, relentless effort to turn today’s treatments into tomorrow’s cures. The time for incremental progress has passed. The time for transformation is now.
