Clinical Trials Fuel Hereditary Cancer Guidelines and Hope

by Diane Rose, VP of Research, FORCE

Every May, National Cancer Research Month invites us to celebrate scientific progress. For those of us navigating hereditary cancer, the message is more than celebratory, it is urgent. The research conducted through clinical trials is the key to progress. Without them, we would have no new drugs, no updated guidelines and far fewer treatment options.

Hereditary cancers represent a subset of all cancers. For the individuals and families affected, the stakes are disproportionately high. These cancers may appear earlier, behave more aggressively and carry risks that extend from one generation to the next. And because they affect a relatively small population, breakthroughs don’t happen frequently or by chance. They happen because of the researchers, clinicians and most importantly, patients willing to participate in clinical trials.

For many of us in this field, the work is deeply personal. Cancer risk has affected my aunt, my father, my siblings and myself. It is now shaping the questions my sons, daughter and granddaughters will one day have to ask. For families like mine, clinical trials are not something that just happen in a far-off lab. They are beacons of hope.

What progress looks like for cancer treatment

In addition to my personal connection, hereditary cancer research is also my professional focus. I help lead collaborations that connect researchers, clinicians and patient advocates to accelerate progress in this area. In my experience, the story of PARP inhibitors and breast cancer is one of the clearest examples of what sustained research can deliver.

PARP inhibitors are a class of targeted therapies that have transformed treatment for people with BRCA-related cancers. They were developed through a series of large, complex global clinical trials requiring intensive collaboration among academic institutions, government-funded research networks and industry partners. Trial results showed improved progression-free survival, and, in some cases, better quality of life compared to standard chemotherapy.

More breakthroughs occurred when researchers considered what could happen if these therapies were used to treat people earlier in the trajectory of the disease. The OlympiA clinical trial answered that question and changed the field forever. In patients with BRCA mutations and early-stage, HER2-negative breast cancer with a high risk of recurrence, adding a year of treatment with the oral PARP inhibitor olaparib significantly increased survival. In other words, a drug once reserved for advanced cancer moved into the curative setting. Today, PARP inhibitors are standard of care for treating certain breast, ovarian, pancreatic and prostate cancers.

The shift from treating late-stage disease to preventing recurrence is integral to cancer treatment research. It only happened because thousands of patients enrolled in a rigorously designed, international clinical trial. And, because research constantly builds on prior successes, this trial will help drive future progress.

Prevention and detection

Prevention research is another powerful frontier in hereditary cancer. The early-phase NOUS-209 vaccine study for people with Lynch syndrome is a recent example. Rather than treating cancer after it appears, the “cancer interception” approach of this study sought to train the immune system to recognize abnormal proteins seen in Lynch-associated tumors before cancer develops. The trial showed encouraging signs that prevention may one day include more than intensive screening and risk-reducing surgery; it may include a vaccine.

More people, more progress

Clinical trials remain an underutilized tool in cancer care. Too few patients are informed that a clinical trial might be an option. In a recent FORCE survey of people with inherited cancer risk, only 16% of respondents were told about research options, including clinical trials. Conversely, when asked about research opportunities, 75% indicated they were interested in participating in clinical research studies.   

Other geographic, financial and logistical barriers to participation make enrollment difficult or impossible. For hereditary cancer research, there are added challenges. Identifying eligible patients requires access to genetic testing, which is not available to everyone.

The result is a system that does not include many people who could contribute and benefit from it.

If we want to accelerate progress, we need to treat clinical trial participation as a standard component of care rather than a last resort. That means integrating trials into preventive medicine and oncology practices, expanding access through decentralized and community-based models, making information accessible and understandable to patients, and ensuring that people have the information they need to make informed health decisions. It also means recognizing the important role of patient advocacy organizations. FORCE plays a critical role in bridging the gap between research and the people it is meant to serve. It helps educate patients, supports informed decision-making and ensures that the lived experience of hereditary cancer is reflected in research priorities.

Progress takes time and the ability to reach people who are eligible and interested in participating. It is methodical and dependent on participation at every level. But when it happens, it is breathtaking.

During National Cancer Research Month, it is worth celebrating how far we’ve come. But celebration alone is not enough. Every major advance in hereditary cancer, from targeted therapies to earlier interventions, exists because patients chose to participate in clinical trials. The next generation of breakthroughs will depend on whether we make it easier, more equitable and more expected for others to do the same. For those facing hereditary cancer, clinical trials don’t just drive progress; they offer hope.