Human Papillomavirus (HPV) is a “family” of more than 200 related viruses. Some people know they should vaccinate their children against the virus – but too many don’t. While these HPV infections clear in most people, for reasons scientists still don’t fully understand, some individuals do not, which can then lead to illnesses for which there are few or no treatments.

Recurrent respiratory papillomatosis (RRP) is just one of those unexpected HPV-driven conditions. RRP is not a sexually transmitted disease and patients are not contagious. An estimated 15,000 to 20,000 people in the United States and more than 125,000 globally have RRP. This burdensome disease takes several forms and impacts people’s upper or lower respiratory tracts or presents as recurrent lesions on the vocal cords or adjacent tissues that require endless corrective surgeries. The treatment often results in permanent damage to a person’s voice.

RRP falls into two demographic subtypes: juvenile-onset (even toddlers) RRP and adult-onset RRP. Each presents unique medical management and lifestyle difficulties, and in addressing these challenges, patient advocacy—raising awareness and building a supportive community—is critically important.

Since it has no Food and Drug Administration (FDA)- approved treatment or cure, patients and scientists devote energy and resources to ensuring people with RRP have access to information. They are in the loop about clinical possibilities for this rare disease. No cure doesn’t mean there is no action!

Beyond the physical challenges of dealing with the disease – and the missed life events and career detours resulting from repeated surgeries, patients also face significant and demoralizing administrative challenges, such as battling payers to cover care using drugs not indicated by the Food and Drug Administration (FDA) for RRP or deemed “not sufficiently proven.”

The lack of treatment does not mean the RRP community is without hope. They are resilient and courageous and are making meaningful connections through the patient advocacy efforts of the Recurrent Respiratory Papillomatosis Foundation. They are reaching and inspiring researchers at the National Institutes of Health to pursue breakthrough research and oversee clinical trials. They also connect with scientists advancing possible therapies at discovery and clinical-stage biopharmaceutical companies like Precigen and encourage them to move forward by enrolling in clinical trials.

Collaboration Accelerates Change

When people unite, their presence creates energy. The Recurrent Respiratory Papillomatosis Foundation, biotech company Precigen, the National Cancer Institute (NCI), and RRP patients and their caregivers met on June 11th at the National Press Club for the Inaugural International Recurrent Respiratory Papillomatosis Awareness Day. This was an inflection point for those who follow the rare disease category.

The gathering wasn’t about hype or baseless optimism; it was a meeting that brought people together, prepared and ready to roll up their sleeves and get to work. It was a day that reaffirmed a commitment to transparency and a truthful assessment of the current situation and path forward.

Virginia Senator Mark Warner, chair of the powerful Senate Intelligence Committee, which oversees cybersecurity efforts that are key to healthcare and innovation data protection, kicked off RRP Awareness Day by expressing his support for people with rare diseases and his desire to help RRP patients find their voice. Senator Warner stated his desire to advance research and innovation and ensure access to care, an expression of determination that reflected his long-standing record on behalf of people seeking treatment options and improved outcomes.   

RRP Foundation President Kim McClellan also spoke as an advocate for the RRP community and as a patient. “We are here to raise awareness about RRP and bring together critical stakeholders in a dialogue on important aspects impacting individuals living with RRP,” she said. “We invite and encourage anyone living with RRP, either as a patient, family member or caregiver, to join us in spreading the word about RRP and participate in clinical trials and advocacy efforts.”

The date of this groundbreaking gathering has special meaning for the RRP community. June 11th (6/11) corresponds to HPV variants 6 and 11 associated with RRP. As the date symbolizes, the gathering united people with the disease, their family members, congressional leaders, and researchers from government agencies and corporate partners in a community united in a common cause.

The opportunity to share and hear multiple perspectives enriched discussions and underscored the importance of taking a comprehensive approach to tackling this condition. Panels of experts and patients sharing personal stories about their journeys gave attendees an unmatched opportunity to delve into the intricacies and impacts of RRP.

Helen Sabzevari, PhD, President and CEO of Precigen, expressed that she and her company were “proud to join forces with the RRP Foundation to establish the first global RRP Awareness Day to bring visibility to the many challenges experienced by RRP patients and to help forge connections among patients, clinicians and government officials.”

A former NCI team leader, Dr. Sabzevari’s commitment to RRP awareness and patient well-being as an animating principle is a model biopharma company C-Suite executives would be wise to emulate. For her and her Precigen colleagues, patients are the focal point of every decision, action, and investment.

RRP Awareness Day was an inspiring platform for discussing struggle, stigma, and science. Lunch was optional, but tissues were required as attendees in the filled-to-capacity room listened to a patient panel on how RRP impacts people and their families. They learned how some individuals living with RRP have needed hundreds of surgeries over the years, beginning when they were toddlers or young children in primary school.

Culture Drives Clinical Performance

Therapeutic innovations are needed to ensure that future generations living with RRP have options reviewed and indicated by the FDA for treating this viral condition.

During the event, a panel of representatives from advocacy and research reflected on how their collaborative approach centering around patients – from the design of clinical trials to allocating resources that have enabled patients to participate in those trials – has been vital in accelerating the R&D process toward identifying and developing viable treatments. The panel included James Gulley, MD., PhD., NIH, Senior Investigator, Center for Immuno-Oncology, Acting Co-Director, National Cancer Institute/Center for Cancer Research; Scott M. Norberg, DO., NIH, Associate Research Physician, Center for Immuno-Oncology; Helen Sabzevari, PhD, CEO, Precigen; and Kim McClellan, President, RRP Foundation.

Dr. Gulley, who is part of the NCI team and has been instrumental in advancing research on RRP and its connection to HPV, emphasized the pressing need for innovative therapies. In his panel comments, Dr. Gulley highlighted the importance of collaborative research efforts to explore potential immunotherapeutic approaches that could offer new hope for patients suffering from this debilitating condition.

No Disagreement – Harmony

Panelists Gulley, Norberg, and Sabzevari applauded the patient community, acknowledging the courage of their readiness to volunteer to participate in clinical trials to speed possible therapeutics forward. It was a reassuring presence and a reminder that public-private collaborations, particularly for rare diseases, do more than spark hope; they spur action. The patient-panel takeaways were: (1) Connect with the RRP Foundation, (2) Support ongoing clinical trial efforts, (3) Prevention through HPV vaccination is key.

While there is still no FDA-approved treatment to manage RRP, this community remains resilient and upbeat, inspiring everyone facing the challenge of rare conditions. The RRP Foundation, Precigen, and NCI are on the same page—science is essential. People living with RRP can remain hopeful that this collaboration will continue until actions result in better options for this patient community.